Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
MY TRIALS
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Register a trial
The ANZCTR website will be unavailable from 1pm until 3pm (AEDT) on Wednesday the 30th of October for website maintenance. Please be sure to log out of the system in order to avoid any loss of data.
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT03861273
Registration number
NCT03861273
Ethics application status
Date submitted
1/03/2019
Date registered
4/03/2019
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
Query!
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)
Query!
Secondary ID [1]
0
0
2022-502844-11-00
Query!
Secondary ID [2]
0
0
C0371002
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
BENEGENE-2
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Hemophilia B
0
0
Query!
Condition category
Condition code
Blood
0
0
0
0
Query!
Clotting disorders
Query!
Human Genetics and Inherited Disorders
0
0
0
0
Query!
Other human genetics and inherited disorders
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Treatment: Other - PF-06838435/ fidanacogene elaparvovec
Experimental: PF-06838435/ fidanacogene elaparvovec -
Treatment: Other: PF-06838435/ fidanacogene elaparvovec
Gene Therapy
Query!
Intervention code [1]
0
0
Treatment: Other
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Annualized Bleeding Rate (ABR) for Total Bleeds (Treated and Untreated) From Week 12 to Month 15
Query!
Assessment method [1]
0
0
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.
Query!
Timepoint [1]
0
0
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)
Query!
Secondary outcome [1]
0
0
ABR for Treated Bleeds From Week 12 to Month 15
Query!
Assessment method [1]
0
0
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.
Query!
Timepoint [1]
0
0
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)
Query!
Secondary outcome [2]
0
0
Annualized Infusion Rate (AIR) of Exogenous FIX From Week 12 to Month 15
Query!
Assessment method [2]
0
0
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.
Query!
Timepoint [2]
0
0
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)
Query!
Secondary outcome [3]
0
0
Steady State Circulating Factor IX (FIX:C) From Week 12 to Month 15
Query!
Assessment method [3]
0
0
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.
Query!
Timepoint [3]
0
0
Week 12 to Month 15
Query!
Secondary outcome [4]
0
0
Circulating Factor IX (FIX:C) at Week 12, Week 24, Week 65
Query!
Assessment method [4]
0
0
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.
Query!
Timepoint [4]
0
0
Week 12, Week 24, Week 65
Query!
Secondary outcome [5]
0
0
Annualized Factor IX (FIX) Consumption From Week 12 to Month 15
Query!
Assessment method [5]
0
0
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.
Query!
Timepoint [5]
0
0
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)
Query!
Secondary outcome [6]
0
0
ABR for Spontaneous Bleeds From Week 12 to Month 15
Query!
Assessment method [6]
0
0
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.
Query!
Timepoint [6]
0
0
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)
Query!
Secondary outcome [7]
0
0
ABR for Traumatic Bleeds From Week 12 to Month 15
Query!
Assessment method [7]
0
0
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.
Query!
Timepoint [7]
0
0
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)
Query!
Secondary outcome [8]
0
0
ABR for Untreated Bleeds From Week 12 to Month 15
Query!
Assessment method [8]
0
0
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.
Query!
Timepoint [8]
0
0
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)
Query!
Secondary outcome [9]
0
0
Number of Target Joint Bleeds From Week 12 to Month 15
Query!
Assessment method [9]
0
0
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.
Query!
Timepoint [9]
0
0
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)
Query!
Secondary outcome [10]
0
0
Percentage of the Participants Without Bleeds From Week 12 to Month 15
Query!
Assessment method [10]
0
0
Percentage of participants without bleeds (total bleeds and treated bleeds) were summarized by type from Week 12 to Month 15.
Query!
Timepoint [10]
0
0
Week 12 to Month 15
Query!
Secondary outcome [11]
0
0
Change From Baseline in Joint Health as Measured by the Hemophilia Joint Health Score (HJHS) Instrument at Month 12
Query!
Assessment method [11]
0
0
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.
Query!
Timepoint [11]
0
0
Baseline, Month 12
Query!
Secondary outcome [12]
0
0
Change From Baseline in Hemophilia Quality of Life (Haem A QoL) Physical Health Domain at Month 12
Query!
Assessment method [12]
0
0
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.
Query!
Timepoint [12]
0
0
Baseline, Month 12
Query!
Secondary outcome [13]
0
0
Change From Baseline in Hemophilia Activities List (HAL) Complex Lower Extremity Activities Component Score at Month 12
Query!
Assessment method [13]
0
0
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.
Query!
Timepoint [13]
0
0
Baseline, Month 12
Query!
Secondary outcome [14]
0
0
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs
Query!
Assessment method [14]
0
0
Results would be posted at secondary completion date.
Query!
Timepoint [14]
0
0
Maximum up to 6 years (Week 312) after PF-06838435 infusion
Query!
Secondary outcome [15]
0
0
Number of Participants With Adverse Events of Special Interest
Query!
Assessment method [15]
0
0
Results would be posted at secondary completion date.
Query!
Timepoint [15]
0
0
Maximum up to 6 years (Week 312) after PF-06838435 infusion
Query!
Secondary outcome [16]
0
0
Number of Participants With Positive Neutralizing Antibody (nAb) to Adeno-associated Virus Vector (AAV) and Anti-Drug Antibody (ADA)
Query!
Assessment method [16]
0
0
Results would be posted at secondary completion date.
Query!
Timepoint [16]
0
0
Maximum up to 6 years (Week 312) after PF-06838435 infusion
Query!
Secondary outcome [17]
0
0
ABR for Total Bleeds (Treated and Untreated) Through the Study
Query!
Assessment method [17]
0
0
Results would be posted at secondary completion date.
Query!
Timepoint [17]
0
0
Maximum up to 6 years (Week 312) after PF-06838435 infusion
Query!
Secondary outcome [18]
0
0
ABR for Treated Bleeds Through the Study
Query!
Assessment method [18]
0
0
Results would be posted at secondary completion date.
Query!
Timepoint [18]
0
0
Maximum up to 6 years (Week 312) after PF-06838435 infusion
Query!
Secondary outcome [19]
0
0
AIR of Exogenous Factor IX Through the Study
Query!
Assessment method [19]
0
0
Results would be posted at secondary completion date.
Query!
Timepoint [19]
0
0
Maximum up to 6 years (Week 312) after PF-06838435 infusion
Query!
Secondary outcome [20]
0
0
FIX: C Level Through the Study
Query!
Assessment method [20]
0
0
Results would be posted at secondary completion date.
Query!
Timepoint [20]
0
0
Maximum up to 6 years (Week 312) after PF-06838435 infusion
Query!
Secondary outcome [21]
0
0
Annualized Factor IX Consumption Through the Study
Query!
Assessment method [21]
0
0
Results would be posted at secondary completion date.
Query!
Timepoint [21]
0
0
Maximum up to 6 years (Week 312) after PF-06838435 infusion
Query!
Secondary outcome [22]
0
0
ABR for Spontaneous and Traumatic, and Untreated Bleeds Through the Study
Query!
Assessment method [22]
0
0
Results would be posted at secondary completion date.
Query!
Timepoint [22]
0
0
Maximum up to 6 years (Week 312) after PF-06838435 infusion
Query!
Secondary outcome [23]
0
0
Change From Baseline in Joint Health as Measured by the HJHS Instrument Through the Study
Query!
Assessment method [23]
0
0
Results would be posted at secondary completion date.
Query!
Timepoint [23]
0
0
Baseline, 6 years
Query!
Secondary outcome [24]
0
0
Number of Target Joint Bleeds Through the Study
Query!
Assessment method [24]
0
0
Results would be posted at secondary completion date.
Query!
Timepoint [24]
0
0
Maximum up to 6 years (Week 312) after PF-06838435 infusion
Query!
Secondary outcome [25]
0
0
Change From Baseline in Haem A QoL Physical Health Domain Through the Study
Query!
Assessment method [25]
0
0
Results would be posted at secondary completion date.
Query!
Timepoint [25]
0
0
Baseline, Maximum up to 6 years (Week 312) after PF-06838435 infusion
Query!
Secondary outcome [26]
0
0
Change From Baseline in HAL Complex Lower Extremity Activities Component Score Through the Study
Query!
Assessment method [26]
0
0
Results would be posted at secondary completion date.
Query!
Timepoint [26]
0
0
Baseline, 6 years
Query!
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
Query!
Minimum age
18
Years
Query!
Query!
Maximum age
65
Years
Query!
Query!
Sex
Males
Query!
Can healthy volunteers participate?
No
Query!
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
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Non-randomised trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Open (masking not used)
Query!
Who is / are masked / blinded?
Query!
Query!
Query!
Query!
Intervention assignment
Single group
Query!
Other design features
Query!
Phase
Phase 3
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Active, not recruiting
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
29/07/2019
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
30/10/2030
Query!
Actual
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
51
Query!
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC,WA
Query!
Recruitment hospital [1]
0
0
Royal Prince Alfred Hospital - Camperdown
Query!
Recruitment hospital [2]
0
0
Royal Brisbane and Women's Hospital - Brisbane
Query!
Recruitment hospital [3]
0
0
Royal Adelaide Hospital - Adelaide
Query!
Recruitment hospital [4]
0
0
The Alfred Hospital - Melbourne
Query!
Recruitment hospital [5]
0
0
Fiona Stanley Hospital - Murdoch
Query!
Recruitment postcode(s) [1]
0
0
2050 - Camperdown
Query!
Recruitment postcode(s) [2]
0
0
4029 - Brisbane
Query!
Recruitment postcode(s) [3]
0
0
5000 - Adelaide
Query!
Recruitment postcode(s) [4]
0
0
3004 - Melbourne
Query!
Recruitment postcode(s) [5]
0
0
6150 - Murdoch
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
California
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
Colorado
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
Indiana
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Mississippi
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Pennsylvania
Query!
Country [6]
0
0
Brazil
Query!
State/province [6]
0
0
Espirito Santo
Query!
Country [7]
0
0
Brazil
Query!
State/province [7]
0
0
SP
Query!
Country [8]
0
0
Brazil
Query!
State/province [8]
0
0
SÃO Paulo
Query!
Country [9]
0
0
Brazil
Query!
State/province [9]
0
0
Rio de Janeiro
Query!
Country [10]
0
0
Canada
Query!
State/province [10]
0
0
Ontario
Query!
Country [11]
0
0
France
Query!
State/province [11]
0
0
Bron
Query!
Country [12]
0
0
France
Query!
State/province [12]
0
0
Paris
Query!
Country [13]
0
0
Germany
Query!
State/province [13]
0
0
Berlin
Query!
Country [14]
0
0
Germany
Query!
State/province [14]
0
0
Bonn
Query!
Country [15]
0
0
Germany
Query!
State/province [15]
0
0
Homburg/Saar
Query!
Country [16]
0
0
Greece
Query!
State/province [16]
0
0
Athens
Query!
Country [17]
0
0
Japan
Query!
State/province [17]
0
0
Hokkaido
Query!
Country [18]
0
0
Japan
Query!
State/province [18]
0
0
Nara
Query!
Country [19]
0
0
Japan
Query!
State/province [19]
0
0
Saitama
Query!
Country [20]
0
0
Japan
Query!
State/province [20]
0
0
Tokyo
Query!
Country [21]
0
0
Korea, Republic of
Query!
State/province [21]
0
0
Seoul
Query!
Country [22]
0
0
Saudi Arabia
Query!
State/province [22]
0
0
Riyadh
Query!
Country [23]
0
0
Spain
Query!
State/province [23]
0
0
Murcia
Query!
Country [24]
0
0
Spain
Query!
State/province [24]
0
0
Barcelona
Query!
Country [25]
0
0
Sweden
Query!
State/province [25]
0
0
Malmö
Query!
Country [26]
0
0
Taiwan
Query!
State/province [26]
0
0
Changhua
Query!
Country [27]
0
0
Taiwan
Query!
State/province [27]
0
0
Kaohsiung
Query!
Country [28]
0
0
Taiwan
Query!
State/province [28]
0
0
Taichung City
Query!
Country [29]
0
0
Taiwan
Query!
State/province [29]
0
0
Taichung
Query!
Country [30]
0
0
Taiwan
Query!
State/province [30]
0
0
Taipei
Query!
Country [31]
0
0
Turkey
Query!
State/province [31]
0
0
Adana
Query!
Country [32]
0
0
Turkey
Query!
State/province [32]
0
0
Gaziantep
Query!
Country [33]
0
0
Turkey
Query!
State/province [33]
0
0
Istanbul
Query!
Country [34]
0
0
Turkey
Query!
State/province [34]
0
0
Izmir
Query!
Country [35]
0
0
United Kingdom
Query!
State/province [35]
0
0
Tyne & Wear
Query!
Country [36]
0
0
United Kingdom
Query!
State/province [36]
0
0
Glasgow
Query!
Country [37]
0
0
United Kingdom
Query!
State/province [37]
0
0
London
Query!
Country [38]
0
0
United Kingdom
Query!
State/province [38]
0
0
Newcastle upon Tyne
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Query!
Name
Pfizer
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
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).
Query!
Trial website
https://clinicaltrials.gov/study/NCT03861273
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Pfizer CT.gov Call Center
Query!
Address
0
0
Pfizer
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
Query!
What data in particular will be shared?
Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
Query!
When will data be available (start and end dates)?
Query!
Available to whom?
Query!
Available for what types of analyses?
Query!
How or where can data be obtained?
IPD available at link: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests
Query!
What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/73/NCT03861273/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/73/NCT03861273/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT03861273