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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/NCT03549871
Registration number
NCT03549871
Ethics application status
Date submitted
25/05/2018
Date registered
8/06/2018
Date last updated
6/02/2023
Titles & IDs
Public title
A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis
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Scientific title
ATLAS-PPX: an Open-label, Multinational, Switching Study to Describe the Efficacy and Safety of Fitusiran Prophylaxis in Patients With Hemophilia A and B Previously Receiving Factor or Bypassing Agent Prophylaxis.
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Secondary ID [1]
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2016-004087-19
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Secondary ID [2]
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EFC15110
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Universal Trial Number (UTN)
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Trial acronym
ATLAS-PPX
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Hemophilia
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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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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Fitusiran
Treatment: Drugs - BPA prophylaxis
Treatment: Drugs - Factor (FVIII or FIX) prophylaxis
Experimental: Fitusiran - Cohort A [inhibitor]: participants with severe hemophilia A/B and inhibitory antibodies to coagulation factor VIII (FVIII)/factor IX (FIX) previously received BPA prophylaxis. Cohort B [non-inhibitor]: participants with severe hemophilia A/B without inhibitory antibodies to FVIII/FIX previously received factor prophylaxis. Participants from both cohorts was enrolled into 6-month factor/BPA prophylaxis period and continued their pre-study, regularly scheduled prophylaxis regimen with factor/BPAs. This period could be skipped by subgroup of Cohort A (hemophilia B with inhibitors to FIX and historical annualized bleeding rate [ABR] >=20) that started directly with fitusiran. Post completing factor/BPA prophylaxis period, participants entered 7-month fitusiran treatment period (1-month onset+6-month efficacy) followed by AT follow-up/roll-over into LTE15174 (NCT03754790). Throughout study, participants could receive on-demand treatment for breakthrough BE with factor/BPAs, as appropriate.
Treatment: Drugs: Fitusiran
Pharmaceutical form: solution for injection Route of administration: subcutaneous
Treatment: Drugs: BPA prophylaxis
Pharmaceutical form: solution for injection Route of administration: Intravenous
Treatment: Drugs: Factor (FVIII or FIX) prophylaxis
Pharmaceutical form: solution for injection Route of administration: Intravenous
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Intervention code [1]
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Treatment: Drugs
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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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Estimated Annualized Bleeding Rate (ABR)
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Assessment method [1]
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Bleeding episodes (BE): any occurrence of hemorrhage might require administration of factor/BPA. BE start time: time at which 1st BE symptoms develop; bleeding/any symptoms at same location occurred within 72 hours of last injection used to treat BE at that location was considered part of original BE and counted as 1 BE towards ABR. Bleeding began after 72 hours of last injection at that location was considered as a new event. ABR = total number of qualifying BE/total number of days in the respective period*365.25. Estimated data were derived by using repeated measures negative binomial (NB) regression model.
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Timepoint [1]
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Factor/BPA prophylaxis period: Day -168 to Day -1 or up to last day of bleeding follow up (any day up to Day -1); 6-month fitusiran efficacy period: Day 29 to Day 190 or up to last day of bleeding follow up (any day up to Day 190), whichever was earliest
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Primary outcome [2]
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Observed Annualized Bleeding Rate (ABR)
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Assessment method [2]
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A bleeding episode (BE): any occurrence of hemorrhage might require administration of factor/BPA. BE start time: time at which 1st BE symptoms develop; bleeding/any symptoms at same location that occurred within 72 hours of last injection used to treat BE at that location was considered a part of original BE and counted as 1 BE towards ABR. Any bleeding that began after 72 hours of last injection at that location was considered as a new event. ABR = total number of qualifying BE/number of days in the respective period *365.25.
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Timepoint [2]
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Factor/BPA prophylaxis period: Day -168 to Day -1 or up to last day of bleeding follow up (any day up to Day -1); 6-month fitusiran efficacy period: Day 29 to Day 190 or up to last day of bleeding follow up (any day up to Day 190), whichever was earliest
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Secondary outcome [1]
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Estimated Annualized Spontaneous Bleeding Rate
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Assessment method [1]
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BE: any occurrence of hemorrhage that might require administration of factor/BPA infusion. BE start time: time at which 1st BE symptoms develop; bleeding/any symptoms at same location that occurred within 72 hours of last injection used to treat BE at that location was considered part of original BE and counted as 1 BE towards ABR. Bleeding began after 72 hours of last injection at that location was considered as a new event. Spontaneous BE: BE occurrence for no apparent/known reason, particularly into joints, muscles, and soft tissues. ABR = total number of qualifying BE/number of days in respective period *365.25. Estimated data was derived using repeated measures NB regression model.
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Timepoint [1]
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Factor/BPA prophylaxis period: Day -168 to Day -1 or up to last day of bleeding follow up (any day up to Day -1); 6-month fitusiran efficacy period: Day 29 to Day 190 or up to last day of bleeding follow up (any day up to Day 190), whichever was earliest
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Secondary outcome [2]
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Observed Annualized Spontaneous Bleeding Rate
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Assessment method [2]
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BE: any occurrence of hemorrhage that might require administration of factor/BPA. BE start time: time at which 1st BE symptoms develop; bleeding/any symptoms at same location that occurred within 72 hours of last injection used to treat BE at that location was considered part of original BE and was counted as 1 BE towards ABR. Bleeding began after 72 hours from last injection at that location was considered as a new event. Spontaneous BE: bleeding event occurred for no apparent or known reason, particularly into joints, muscles and soft tissues. ABR = total number of qualifying BE/number of days in respective period *365.25.
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Timepoint [2]
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Factor/BPA prophylaxis period: Day -168 to Day -1 or up to last day of bleeding follow up (any day up to Day -1); 6-month fitusiran efficacy period: Day 29 to Day 190 or up to last day of bleeding follow up (any day up to Day 190), whichever was earliest
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Secondary outcome [3]
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Estimated Annualized Joint Bleeding Rate
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Assessment method [3]
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BE: any hemorrhage that required administration of factor/BPA. BE start time: time at which 1st BE symptoms develop; bleeding/any symptoms at same location that occurred within 72 hours of last injection to treat BE at that location was considered part of original BE; counted as 1 BE towards ABR. Bleeding after 72 hours from last injection at that location was considered as a new event. Joint BE: characterized by unusual sensation in joint ("aura") + increasing swelling/warmth over joint skin, increasing pain/progressive loss of range of motion/difficulty in limb use compared to Baseline. ABR = total number of qualifying BE/number of days in respective period *365.25. Estimated data were derived by using repeated measures NB regression model.
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Timepoint [3]
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Factor/BPA prophylaxis period: Day -168 to Day -1 or up to last day of bleeding follow up (any day up to Day -1); 6-month fitusiran efficacy period: Day 29 to Day 190 or up to last day of bleeding follow up (any day up to Day 190), whichever was earliest
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Secondary outcome [4]
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Observed Annualized Joint Bleeding Rate
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Assessment method [4]
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BE: any occurrence of hemorrhage that might require administration of factor/BPA. BE start time: time at which 1st BE symptoms develop; bleeding/any symptoms at same location that occurred within 72 hours of last injection used to treat BE at that location was considered part of original BE and counted as 1 BE towards ABR. Bleeding began after 72 hours from last injection at that location was considered as a new event. Joint BE: characterized by unusual sensation in joint (aura) increasing swelling/warmth over joint skin, increasing pain or progressive loss of range of motion/difficulty in limb use compared to Baseline. ABR = total number of joint BE/number of days in respective period*365.25.
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Timepoint [4]
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Factor/BPA prophylaxis period: Day -168 to Day -1 or up to last day of bleeding follow up (any day up to Day -1); 6-month fitusiran efficacy period: Day 29 to Day 190 or up to last day of bleeding follow up (any day up to Day 190), whichever was earliest
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Secondary outcome [5]
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Change in Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QOL) Physical Health Score in the Fitusiran Treatment Period and the Factor or BPA Prophylaxis Period
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Assessment method [5]
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Haem-A-QoL: participant-reported questionnaire for adults aged >=17 years with hemophilia and comprised of 46 items covering 10 domains. Physical health domain (PHD) is assessed with 5 items rated on 5-point Likert scale: never, rarely, sometimes, often or all the time. Raw score for PHD were transformed to scale ranged from 0 to 100, where lower scores = better physical health. Least square (LS) mean and 95% confidence interval (CI) by mixed model for repeated measure (MMRM) analysis with robust sandwich covariance matrix: change from Month -6 to Day 1 and to Month 7 as response variable; period (factor/BPA prophylaxis & fitusiran treatment) & Baseline score (Month -6) as fixed effects.
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Timepoint [5]
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Month -6 of Factor or BPA prophylaxis period (Baseline), Day 1 (Month 1) and Month 7 of fitusiran treatment period
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Secondary outcome [6]
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Change in Haemophilia Quality of Life Questionnaire for Adults Total Score in the Fitusiran Treatment Period and the Factor or BPA Prophylaxis Period
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Assessment method [6]
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Haem-A-QoL: questionnaire for adults aged >= 17 years with hemophilia; and comprised of 46 items covering 10 domains: physical health, feelings, view of yourself, sports and leisure, work and school, dealing with hemophilia, treatment, future, family planning, partnership and sexuality. Items were rated on 5-point Likert scale: never, rarely, sometimes, often or all time. Domain raw score was transformed to scale ranged from 0 to 100, where lower scores=better health. LS mean & 95% CI by MMRM analysis with robust sandwich covariance matrix: change from Month -6 to Day 1 and to Month 7 as response variable; period (factor/BPA prophylaxis & fitusiran treatment) & Baseline score (Month -6) as fixed effects.
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Timepoint [6]
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Month -6 of Factor or BPA prophylaxis period (Baseline), Day 1 (Month 1) and Month 7 of fitusiran treatment period
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Secondary outcome [7]
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Estimated Annualized Bleeding Rate in the Fitusiran Onset Period
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Assessment method [7]
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BE: any occurrence of hemorrhage that might require administration of factor/BPA. BE start time: time at which 1st BE symptoms develop; bleeding/any symptoms at same location that occurred within 72 hours of last injection used to treat BE at that location was considered part of original BE and was counted as 1 BE towards ABR. Bleeding began after 72 hours from last injection at that location was considered as new event. Estimated ABR and 95% CI was derived by using repeated measures NB regression model with logarithm of duration (years) that each participant spends in 1-Month fitusiran onset period matching BE data being analyzed as offset variable. ABR = total number of qualifying BE/number of days in respective period *365.25.
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Timepoint [7]
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Day 1 up to Day 28 or up to the last day of bleeding follow up (any day up to Day 28), whichever was earliest
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Secondary outcome [8]
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Observed Annualized Bleeding Rate in the Fitusiran Onset Period
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Assessment method [8]
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BE: any occurrence of hemorrhage that might require administration of factor/BPA. BE start time: time at which 1st BE symptoms develop; bleeding/any symptoms at same location that occurred within 72 hours of last injection used to treat BE at that location was considered a part of original BE and was counted as one BE towards ABR. Bleeding began after 72 hours from last injection at that location was considered as a new event. ABR= total number of qualifying BE/number of days in the 1-month onset period *365.25. Analysis was based on on-treatment strategy which included all treated bleeding events in 1-month onset period and excluded any bleeding events in period of intercurrent events.
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Timepoint [8]
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Day 1 up to Day 28 or up to the last day of bleeding follow up (any day up to Day 28), whichever was earliest
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Secondary outcome [9]
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Estimated Annualized Bleeding Rate in the Fitusiran Treatment Period
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Assessment method [9]
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BE: defined as any occurrence of hemorrhage that might require administration of factor/BPA. BE start time was time at which 1st BE symptoms develop; bleeding/any symptoms at same location that occurred within 72 hours of last injection used to treat BE at that location was considered a part of original BE and counted as one BE towards ABR. Bleeding began after 72 hours from last injection at that location was considered as new event. Analysis was based on on-treatment strategy which included all treated bleeding events in fitusiran period and excluded any bleeding events in the period of intercurrent events. ABR = total number of qualifying BE/number of days in respective period *365.25.
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Timepoint [9]
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From Day 1 up to Day 190
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Secondary outcome [10]
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Observed Annualized Bleeding Rate in the Fitusiran Treatment Period
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Assessment method [10]
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BE: any occurrence of hemorrhage that might require administration of factor/BPA. BE start time was time at which 1st BE symptoms develop; bleeding/any symptoms at same location that occurred within 72 hours of last injection used to treat BE at that location was considered a part of original bleeding event and was counted as one BE towards ABR. Any bleeding that began after 72 hours from last injection at that location was considered as a new event. ABR= total number of qualifying BE/number of days in treatment period *365.25. Analysis was based on on-treatment strategy which included all treated bleeding events in fitusiran period and excluded any bleeding events in period of intercurrent events.
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Timepoint [10]
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from Day 1 up to Day 190
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Secondary outcome [11]
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Cohort A: Annualized Weight-adjusted Consumption of BPA (Activated Prothrombin Complex Concentrates)
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Assessment method [11]
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Annualized weight-adjusted BPA consumption was calculated for each participant during prophylaxis period as: [Sum of BPA dose per body weight received during corresponding period/number of days in corresponding period]*365.25. In this outcome measure, data of annualized weight-adjusted consumption of BPA agent: aPCC (unit per kilogram [U/kg]) were reported.
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Timepoint [11]
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Factor/BPA prophylaxis period: Day -168 to Day -1 or up to last day of bleeding follow up (any day up to Day -1); 6-month fitusiran efficacy period: Day 29 to Day 190 or up to last day of bleeding follow up (any day up to Day 190), whichever was earliest
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Secondary outcome [12]
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Cohort A: Annualized Weight-adjusted Consumption of BPA (Recombinant Factor VIIa)
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Assessment method [12]
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Annualized weight-adjusted BPA consumption was calculated for each participant during prophylaxis period as: (Sum of BPA dose per body weight received during corresponding period/number of days in corresponding period)*365.25. In this outcome measure, data of annualized weight-adjusted consumption of BPA agents: rFVIIa (unit: micrograms per kg [mcg/kg]) were reported. Combined data of annualized weight-adjusted BPA consumption (mcg/kg) for both treated bleeds and prophylaxis purpose were reported in this outcome measure.
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Timepoint [12]
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Factor/BPA prophylaxis period: Day -168 to Day -1 or up to last day of bleeding follow up (any day up to Day -1); 6-month fitusiran efficacy period: Day 29 to Day 190 or up to last day of bleeding follow up (any day up to Day 190), whichever was earliest
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Secondary outcome [13]
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Cohort B: Annualized Weight-adjusted Consumption of Factor
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Assessment method [13]
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Annualized weight-adjusted BPA consumption was calculated for each participant during prophylaxis period as: (Sum of BPA dose per body weight received during corresponding period/number of days in corresponding period)*365.25. In this outcome measure, data of annualized weight-adjusted consumption of BPA agents: FVIII and FIX (unit: international Units [IU] per kg [IU/kg]) were reported.
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Timepoint [13]
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Factor/BPA prophylaxis period: Day -168 to Day -1 or up to last day of bleeding follow up (any day up to Day -1); 6-month fitusiran efficacy period: Day 29 to Day 190 or up to last day of bleeding follow up (any day up to Day 190), whichever was earliest
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Eligibility
Key inclusion criteria
- Males, >=12 years of age.
- Severe hemophilia A or B (as evidenced by a central laboratory measurement at
screening or documented medical record evidence of FVIII less than (<) 1 percent (%)
or FIX level less than or equal to (<=) 2%).
- A minimum of 2 bleeding episodes required BPA treatment within the last 6 months prior
to screening for participants with inhibitory antibodies to factor VIII or factor IX
(Cohort A). A minimum of 1 bleeding episode required factor treatment within the last
12 months prior to screening for participants without inhibitory antibodies to factor
VIII or factor IX (Cohort B).
- Met either the definition of inhibitor or non-inhibitor participant as below:
- Inhibitor: Use of BPAs for prophylaxis and for any bleeding episodes for at least the
last 6 months prior to screening, and met one of the following Nijmegen-modified
Bethesda assay results criteria:
- Inhibitor titer of >=0.6 Bethesda Unit per milliliter (BU/mL) at screening, or
- Inhibitor titer of <0.6 BU/mL at screening with medical record evidence of 2
consecutive titers >=0.6 BU/mL, or
- Inhibitor titer of <0.6 BU/mL at screening with medical record evidence of anamnestic
response
- The subgroup of participants in Cohort A participants might additionally meet the
following criteria to be eligible to start treatment with fitusiran directly after the
screening period:
- Hemophilia B with inhibitory antibody to Factor IX as defined above
- Not responding adequately to BPA treatment (historical ABR >=20) prior to
enrollment
- In the opinion of the Investigator, with approval of Sponsor Medical Monitor,
6-month BPA prophylaxis period should be omitted.
- Non-inhibitor: Use of factor concentrates for prophylaxis and for any bleeding
episodes for at least the last 6 months prior to screening, and met each of the
following criterion:
- Nijmegen-modified Bethesda assay inhibitor titer of <0.6 BU/mL at screening and
- No use of BPAs to treat bleeding episodes for at least the last 6 months prior to
screening and
- No history of immune tolerance induction therapy within the past 3 years prior to
screening.
- Documented prophylactic treatment with factor concentrates or BPAs for the treatment
of hemophilia A or B for at least 6 months prior to screening.
- Adherent to the prescribed prophylactic therapy for at least 6 months prior to
screening per Investigator assessment.
- Willed and complied with the study requirements and to provide written informed
consent and assent.
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Minimum age
12
Years
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Maximum age
No limit
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Sex
Males
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Known co-existing bleeding disorders other than hemophilia A or B.
- AT activity <60% at screening.
- Co-existing thrombophilic disorder.
- Clinically significant liver disease.
- Active Hepatitis C virus infection.
- Acute or chronic Hepatitis B virus infection.
- HIV positive with a CD4 count of <200 cells per microliter.
- History of arterial or venous thromboembolism.
- Inadequate renal function.
- History of multiple drug allergies or history of allergic reaction to an
oligonucleotide or N-Acetylgalactosamine (GalNAc).
- History of intolerance to subcutaneous injection(s).
- Any other conditions or comorbidities that made the participant unsuitable for
enrollment or could interfere with participation in or completion of the study, per
Investigator judgment.
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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
Completed
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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
25/07/2018
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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
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Actual
25/03/2022
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Sample size
Target
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Accrual to date
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Final
80
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Investigational Site Number 6104 - Prahran
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Recruitment postcode(s) [1]
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3181 - Prahran
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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California
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China
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Beijing
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Denmark
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Copenhagen
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France
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Lyon
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Ireland
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Crumlin
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Israel
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Ramat-Gan
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Italy
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Milano
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Japan
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Nagoya
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Japan
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Nishinomiya
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Japan
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Saitama
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Japan
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Tokyo
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Korea, Republic of
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Busan
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Korea, Republic of
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Daejeon
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Korea, Republic of
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Seoul
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Malaysia
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Ampang
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0
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Malaysia
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Johor Bahru
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Malaysia
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Kota Kinabalu
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Mexico
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San Pablo
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Turkey
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Adana
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Turkey
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Ankara
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Turkey
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Antalya
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Turkey
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Gaziantep
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0
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Turkey
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Istanbul
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0
0
Turkey
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Izmir
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0
0
Turkey
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Kayseri
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0
0
Turkey
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0
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Samsun
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0
0
Turkey
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0
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Van
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0
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Ukraine
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0
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Kyiv
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Country [29]
0
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Ukraine
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State/province [29]
0
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Lviv
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Country [30]
0
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United Kingdom
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State/province [30]
0
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Glasgow
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Country [31]
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United Kingdom
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State/province [31]
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Genzyme, a Sanofi Company
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Address
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Ethics approval
Ethics application status
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Summary
Brief summary
Primary Objective:
To characterize the frequency of bleeding episodes (BE) while receiving fitusiran treatment,
relative to the frequency of bleeding episodes while receiving factor concentrate or
bypassing agent (BPA) prophylaxis.
Secondary Objectives:
- To characterize the following while receiving fitusiran treatment, relative to receiving
factor or BPA prophylaxis:
- the frequency of spontaneous bleeding episodes
- the frequency of joint bleeding episodes
- health related quality of life (HRQOL) in participants greater than or equal to (>=) 17
years of age
- To characterize the frequency of bleeding episodes during the onset and treatment
periods in participants receiving fitusiran.
- To characterize the safety and tolerability of fitusiran.
- To characterize the annualized weight-adjusted consumption of factor/BPA while receiving
fitusiran treatment, relative to receiving factor or BPA prophylaxis.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT03549871
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
0
0
Clinical Sciences & Operations
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Address
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Sanofi
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Country
0
0
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Phone
0
0
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Fax
0
0
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Email
0
0
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Contact person for public queries
Name
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0
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Address
0
0
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Country
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0
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Phone
0
0
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Fax
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0
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Email
0
0
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT03549871
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