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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT03861273
Registration number
NCT03861273
Ethics application status
Date submitted
1/03/2019
Date registered
4/03/2019
Date last updated
3/05/2024
Titles & IDs
Public title
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
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Scientific title
Phase 3, Open-label, Single-arm Study to Evaluate Efficacy and Safety of FIX Gene Transfer With PF-06838435 (rAAV-Spark100-hFIX-R338L) in Adult Male Participants With Moderately Severe to Severe Hemophilia B (FIX:C =2%) (BeneGene-2)
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Secondary ID [1]
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2018-003086-33
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Secondary ID [2]
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C0371002
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Universal Trial Number (UTN)
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Trial acronym
BENEGENE-2
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Hemophilia B
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Condition category
Condition code
Blood
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Clotting disorders
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Human Genetics and Inherited Disorders
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0
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Other interventions - PF-06838435/ fidanacogene elaparvovec
Experimental: PF-06838435/ fidanacogene elaparvovec -
Other interventions: PF-06838435/ fidanacogene elaparvovec
Gene Therapy
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Intervention code [1]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Annualized Bleeding Rate (ABR) for Total Bleeds (Treated and Untreated) From Week 12 to Month 15
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Assessment method [1]
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ABR = number of total bleeding episodes on study during the given time period) *365.25/ (Date of last day - date of first day +1) in that time period. Surgical procedures were excluded from summary/analyses. Treated Bleed: An event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding (protocol definition, unless specifically referring to untreated bleed). Untreated Bleed: A bleeding event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002.
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Timepoint [1]
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Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm)
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Secondary outcome [1]
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ABR for Treated Bleeds From Week 12 to Month 15
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Assessment method [1]
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ABR = number of total bleeding episodes on study during the given time period) *365.25/ (Date of last day - date of first day +1) in that time period. Surgical procedures were excluded from summary/analyses. Treated Bleed: An event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding (protocol definition, unless specifically referring to untreated bleed). This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002.
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Timepoint [1]
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Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm)
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Secondary outcome [2]
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Annualized Infusion Rate (AIR) of Exogenous FIX From Week 12 to Month 15
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Assessment method [2]
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AIR = number of exogenous infusions (for any reason) received during given time period *365.25/ (Date of last day - date of first day +1) in that time period. This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002.
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Timepoint [2]
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Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm)
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Secondary outcome [3]
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Steady State Circulating Factor IX (FIX:C) From Week 12 to Month 15
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Assessment method [3]
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The certified central clinical laboratory analyzed the sample by two one-stage assays and a chromogenic assay. The first one-stage assay was performed on BCSXP analyzer with Actin-FSL reagent. The second one-stage assay used the same analyzer but SynthAsil as reagent. Data reported in this Outcome Measure is the geometric mean of all assessments from week 12 to Month 15.
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Timepoint [3]
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Week 12 to Month 15
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Secondary outcome [4]
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Circulating Factor IX (FIX:C) at Week 12, Week 24, Week 65
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Assessment method [4]
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The certified central clinical laboratory analyzed the sample by two one-stage assays and a chromogenic assay. The first one-stage assay was performed on BCSXP analyzer with Actin-FSL reagent. The second one-stage assay used the same analyzer but SynthAsil as reagent.
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Timepoint [4]
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Week 12, Week 24, Week 65
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Secondary outcome [5]
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Annualized Factor IX (FIX) Consumption From Week 12 to Month 15
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Assessment method [5]
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Annualized FIX consumption was reported by International Units per kilogram (IU/kg). This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002. Data reported in this Outcome Measure is average of all assessments from Week 12 to Month 15.
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Timepoint [5]
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Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm)
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Secondary outcome [6]
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ABR for Spontaneous Bleeds From Week 12 to Month 15
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Assessment method [6]
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ABR = number of total bleeding episodes on study during the given time period) *365.25/ (Date of last day - date of first day +1) in that time period. Surgical procedures were excluded from summary/analyses. Surgical procedures were excluded from summary/analyses. Spontaneous Bleeds: Bleeding for no apparent/known reason particularly into the joints, muscles, and soft tissues. This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002.
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Timepoint [6]
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Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm)
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Secondary outcome [7]
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ABR for Traumatic Bleeds From Week 12 to Month 15
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Assessment method [7]
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ABR = number of total bleeding episodes on study during the given time period) *365.25/ (Date of last day - date of first day +1) in that time period. Surgical procedures were excluded from summary/analyses. Surgical procedures were excluded from summary/analyses. Traumatic Bleeds: Bleeding event occurring for an apparent/known reason. This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002.
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Timepoint [7]
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Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm)
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Secondary outcome [8]
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ABR for Untreated Bleeds From Week 12 to Month 15
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Assessment method [8]
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ABR = number of total bleeding episodes on study during the given time period) *365.25/ (Date of last day - date of first day +1) in that time period. Surgical procedures were excluded from summary/analyses. Surgical procedures were excluded from summary/analyses. Untreated Bleed: A bleeding event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002.
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Timepoint [8]
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Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm)
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Secondary outcome [9]
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Number of Target Joint Bleeds From Week 12 to Month 15
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Assessment method [9]
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Target Joint: Defined as a major joint (e.g., hip, elbow, wrist, shoulder, knee, and ankle) into which repeated bleeds occurred (three or more spontaneous bleeds into a single joint within a consecutive 6-month period). A target joint was considered resolved when there were =<2 bleeds into the joint within a 12-month period. Joint Bleed: A bleeding episode characterized by rapid loss of range of motion as compared with baseline that was associated with any combination of the following: pain or an unusual sensation in the joint, palpable swelling, and warmth of the skin over the joint. This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002.
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Timepoint [9]
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Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm)
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Secondary outcome [10]
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Percentage of the Participants Without Bleeds From Week 12 to Month 15
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Assessment method [10]
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Percentage of participants without bleeds (total bleeds and treated bleeds) were summarized by type from Week 12 to Month 15.
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Timepoint [10]
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Week 12 to Month 15
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Secondary outcome [11]
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Change From Baseline in Joint Health as Measured by the Hemophilia Joint Health Score (HJHS) Instrument at Month 12
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Assessment method [11]
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A qualified healthcare professional assessed six joints (left ankle, right ankle, left elbow, right elbow, left knee, right knee) scored from 0 to 20 based on: duration of swelling, muscle atrophy, crepitus, flexion loss, extension loss, instability, joint pain, and strength. Gait was scored (0 to 4) based on walking, stairs, running, hopping on one leg. Total score = sum of scores from all joints + gait score ranged from 0 to 124, with the higher the number equating to more severe joint damage.
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Timepoint [11]
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Baseline, Month 12
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Secondary outcome [12]
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Change From Baseline in Hemophilia Quality of Life (Haem A QoL) Physical Health Domain at Month 12
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Assessment method [12]
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The Haem-A-QoL questionnaire contained 46 items with ten domains that assessed health in the following areas: Physical Health; Feelings; View of Self; Sports and Leisure; Work and School; Dealing with Haemophilia; Treatment; Future; Family Planning; and Partnership and Sexuality. The physical health domain was considered as the primary domain in this questionnaire, had a transformed score range from 0 to 100, with lower scores representing higher quality of life. In this Outcome Measure Physical Health domain scores of Haem A QoL are reported.
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Timepoint [12]
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Baseline, Month 12
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Secondary outcome [13]
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Change From Baseline in Hemophilia Activities List (HAL) Complex Lower Extremity Activities Component Score at Month 12
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Assessment method [13]
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The HAL was a multiple domain measure of the impact of hemophilia on functional abilities in adults. The 7 domains of this instrument contained 42 items in total, as follows: lying/sitting/kneeling/standing; lower (leg) functioning; upper (arm) functioning; transportation; self-care; household tasks; and sports/leisure. Selected items from five of the domains were used to create three components: upper extremity; basic lower extremity; and complex lower extremity activities. The component score of "complex lower extremity activities" was the most important in this questionnaire, had a transformed score range from 0 to 100, higher values indicated less functional limitations in performing tasks.
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Timepoint [13]
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Baseline, Month 12
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Secondary outcome [14]
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Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs
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Assessment method [14]
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Results would be posted at secondary completion date.
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Timepoint [14]
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Maximum up to 6 years (Week 312) after PF-06838435 infusion
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Secondary outcome [15]
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Number of Participants With Adverse Events of Special Interest
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Assessment method [15]
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Results would be posted at secondary completion date.
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Timepoint [15]
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Maximum up to 6 years (Week 312) after PF-06838435 infusion
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Secondary outcome [16]
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Number of Participants With Positive Neutralizing Antibody (nAb) to Adeno-associated Virus Vector (AAV) and Anti-Drug Antibody (ADA)
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Assessment method [16]
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Results would be posted at secondary completion date.
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Timepoint [16]
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Maximum up to 6 years (Week 312) after PF-06838435 infusion
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Secondary outcome [17]
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ABR for Total Bleeds (Treated and Untreated) Through the Study
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Assessment method [17]
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Results would be posted at secondary completion date.
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Timepoint [17]
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Maximum up to 6 years (Week 312) after PF-06838435 infusion
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Secondary outcome [18]
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ABR for Treated Bleeds Through the Study
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Assessment method [18]
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Results would be posted at secondary completion date.
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Timepoint [18]
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Maximum up to 6 years (Week 312) after PF-06838435 infusion
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Secondary outcome [19]
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AIR of Exogenous Factor IX Through the Study
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Assessment method [19]
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Results would be posted at secondary completion date.
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Timepoint [19]
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Maximum up to 6 years (Week 312) after PF-06838435 infusion
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Secondary outcome [20]
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FIX: C Level Through the Study
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Assessment method [20]
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Results would be posted at secondary completion date.
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Timepoint [20]
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Maximum up to 6 years (Week 312) after PF-06838435 infusion
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Secondary outcome [21]
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Annualized Factor IX Consumption Through the Study
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Assessment method [21]
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Results would be posted at secondary completion date.
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Timepoint [21]
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Maximum up to 6 years (Week 312) after PF-06838435 infusion
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Secondary outcome [22]
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ABR for Spontaneous and Traumatic, and Untreated Bleeds Through the Study
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Assessment method [22]
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Results would be posted at secondary completion date.
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Timepoint [22]
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Maximum up to 6 years (Week 312) after PF-06838435 infusion
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Secondary outcome [23]
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Change From Baseline in Joint Health as Measured by the HJHS Instrument Through the Study
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Assessment method [23]
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Results would be posted at secondary completion date.
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Timepoint [23]
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Baseline, 6 years
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Secondary outcome [24]
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Number of Target Joint Bleeds Through the Study
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Assessment method [24]
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Results would be posted at secondary completion date.
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Timepoint [24]
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Maximum up to 6 years (Week 312) after PF-06838435 infusion
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Secondary outcome [25]
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Change From Baseline in Haem A QoL Physical Health Domain Through the Study
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Assessment method [25]
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Results would be posted at secondary completion date.
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Timepoint [25]
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Baseline, Maximum up to 6 years (Week 312) after PF-06838435 infusion
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Secondary outcome [26]
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Change From Baseline in HAL Complex Lower Extremity Activities Component Score Through the Study
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Assessment method [26]
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Results would be posted at secondary completion date.
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Timepoint [26]
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Baseline, 6 years
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Eligibility
Key inclusion criteria
Inclusion Criteria
- Males who completed 6 months of Factor IX prophylaxis therapy during the lead-in study
(C0371004) prior to providing consent at the screening visit for this study.
- Documented moderately severe to severe hemophilia B (Factor IX activity < =2%)
- Previous experience with FIX therapy (=>50 documented exposure days to a FIX protein
product)
- Suspension of prophylaxis therapy for hemophilia B after administration of the study
drug
- Laboratory values (hemoglobin, platelets and creatinine) within study specified limits
- Agree to contraception until components of the drug are eliminated from their body
- Capable of giving signed informed consent
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Minimum age
18
Years
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Maximum age
65
Years
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Sex
Males
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Can healthy volunteers participate?
No
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Key exclusion criteria
Exclusion Criteria
- Anti-AAVRh74var neutralizing antibodies (nAb) titer above the established threshold
(ie, positive for nAb).
- History of inhibitor to Factor IX or inhibitor detected during screening. Clinical
signs or symptoms of decreased response to Factor IX
- Hypersensitivity to Factor IX replacement product or IV immunoglobulin administration
- History of chronic infection or other chronic disease
- Any conditions associated with increased thromboembolic risk
- Concurrent clinically significant major disease or condition unsuitable for
participation and/or may interfere with the interpretation of study results
- Laboratory values at screening visit that are abnormal or outside acceptable study
limits
- Current unstable liver or biliary disease
- Currently on antiviral therapy for hepatitis B or C
- Planned surgical procedure requiring Factor IX surgical prophylactic factor treatment
15 months from screening visit
- Use of restricted therapies (e.g., blood products, acetylsalicylic acid [aspirin] or
ibuprofen, other investigational therapy, and by-passing agents)
- Previously dosed in a gene therapy research trial at any time or in an interventional
clinical study within 12 weeks of screening visit
- Active hepatitis B or C; hepatitis B surface antigen, hepatitis B virus
deoxyribonucleic acid positivity, or hepatitis C virus ribonucleic acid positivity
- Significant liver disease
- Serological evidence of HIV1 or HIV2 infection with either CD4+ cell count <=200 mm3
and/or a viral load >20 copies/mL
- Study and sponsor staff involved in the conduct of the study and their families
- Unable to comply with study procedures
- Sensitivity to heparin or heparin induced thrombocytopenia
- Sensitivity to any of the study interventions, or components thereof, or drug or other
allergy
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Active, not recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
29/07/2019
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
5/09/2030
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Actual
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Sample size
Target
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Accrual to date
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Final
51
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Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC,WA
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Recruitment hospital [1]
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Royal Prince Alfred Hospital - Camperdown
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Recruitment hospital [2]
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Royal Brisbane and Women's Hospital - Brisbane
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Recruitment hospital [3]
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Royal Adelaide Hospital - Adelaide
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Recruitment hospital [4]
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The Alfred Hospital - Melbourne
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Recruitment hospital [5]
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Fiona Stanley Hospital - Murdoch
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Recruitment postcode(s) [1]
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2050 - Camperdown
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Recruitment postcode(s) [2]
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4029 - Brisbane
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Recruitment postcode(s) [3]
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5000 - Adelaide
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Recruitment postcode(s) [4]
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3004 - Melbourne
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Recruitment postcode(s) [5]
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6150 - Murdoch
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Recruitment outside Australia
Country [1]
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0
United States of America
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State/province [1]
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0
California
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Country [2]
0
0
United States of America
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State/province [2]
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0
Colorado
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Country [3]
0
0
United States of America
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State/province [3]
0
0
Indiana
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Country [4]
0
0
United States of America
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State/province [4]
0
0
Mississippi
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Country [5]
0
0
United States of America
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State/province [5]
0
0
Pennsylvania
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Country [6]
0
0
Brazil
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State/province [6]
0
0
Espirito Santo
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Country [7]
0
0
Brazil
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State/province [7]
0
0
SP
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Country [8]
0
0
Brazil
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State/province [8]
0
0
SÃO Paulo
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Country [9]
0
0
Brazil
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State/province [9]
0
0
Rio de Janeiro
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Country [10]
0
0
Canada
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State/province [10]
0
0
Ontario
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Country [11]
0
0
France
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State/province [11]
0
0
Bron
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Country [12]
0
0
France
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State/province [12]
0
0
Paris
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Country [13]
0
0
Germany
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State/province [13]
0
0
Berlin
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Country [14]
0
0
Germany
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State/province [14]
0
0
Bonn
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Country [15]
0
0
Germany
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State/province [15]
0
0
Homburg/Saar
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Country [16]
0
0
Greece
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State/province [16]
0
0
Athens
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Country [17]
0
0
Japan
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State/province [17]
0
0
Hokkaido
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Country [18]
0
0
Japan
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State/province [18]
0
0
Nara
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Country [19]
0
0
Japan
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State/province [19]
0
0
Saitama
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Country [20]
0
0
Japan
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State/province [20]
0
0
Tokyo
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Country [21]
0
0
Korea, Republic of
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State/province [21]
0
0
Seoul
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Country [22]
0
0
Saudi Arabia
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State/province [22]
0
0
Riyadh
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Country [23]
0
0
Sweden
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State/province [23]
0
0
Malmö
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Country [24]
0
0
Taiwan
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State/province [24]
0
0
Changhua
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Country [25]
0
0
Taiwan
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State/province [25]
0
0
Kaohsiung
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Country [26]
0
0
Taiwan
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State/province [26]
0
0
Taichung City
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Country [27]
0
0
Taiwan
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State/province [27]
0
0
Taichung
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Country [28]
0
0
Taiwan
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State/province [28]
0
0
Taipei
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Country [29]
0
0
Turkey
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State/province [29]
0
0
Adana
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Country [30]
0
0
Turkey
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State/province [30]
0
0
Gaziantep
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Country [31]
0
0
Turkey
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State/province [31]
0
0
Istanbul
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Country [32]
0
0
Turkey
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State/province [32]
0
0
Izmir
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Country [33]
0
0
United Kingdom
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State/province [33]
0
0
Tyne & Wear
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Country [34]
0
0
United Kingdom
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State/province [34]
0
0
Glasgow
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Country [35]
0
0
United Kingdom
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State/province [35]
0
0
London
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Country [36]
0
0
United Kingdom
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State/province [36]
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0
Newcastle upon Tyne
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Pfizer
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
This study will evaluate the efficacy and safety of PF-06838435 (a gene therapy drug) in
adult male participants with moderately severe to severe hemophilia B (participants that have
a Factor IX circulating activity of 2% or less). The gene therapy is designed to introduce
genetic material into cells to compensate for missing or non-functioning Factor IX. Eligible
study participants will have completed a minimum 6 months of routine Factor IX prophylaxis
therapy during the lead in study (C0371004). Participants will be dosed once (intravenously)
and will be evaluated over the course of 6 years. The main objective of the study will
evaluate the annualized bleeding rate [ABR] for participants treated with gene therapy versus
standard of care (SOC) therapy (FIX prophylaxis replacement regimen).
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https://clinicaltrials.gov/ct2/show/NCT03861273
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Pfizer
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Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT03861273
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