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NCT03861273 · ClinicalTrials.gov registry record · Phase 3
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
A Phase 3 study of Hemophilia B, sponsored by Pfizer.
- Active
- Registry status
- Phase 3
- Development phase
- 51
- Enrollment target
- 20
- Study locations
NCT03861273: Active Phase 3 study of Hemophilia B, sponsored by Pfizer.
NCT03861273 is a Phase 3 study of Hemophilia B that is active but no longer recruiting, run by Pfizer. The registered enrollment target is 51 participants, below the 94-participant average among 20 other Hemophilia B trials with a reported enrollment target (46% lower). The trial reports 20 study locations across 9 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03861273, a Phase 3 study of Hemophilia B, is active but no longer recruiting, sponsored by Pfizer.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 51 participants
- Enrollment target
- 20
- Study locations
Study 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).
Primary Outcome
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 withi
Conditions Studied
Interventions
- BIOLOGICAL PF-06838435/ fidanacogene elaparvovec
Study Locations (20)
California
- UCSF IDS Pharmacy - San Francisco
- University of California, San Francisco - Clinical Research Center - San Francisco
- University of California, San Francisco - Outpatient Hematology Clinic - San Francisco
- The Regents of the University of California, San Francisco campus - San Francisco
Indiana
- Indiana Hemophilia & Thrombosis Center, Inc. - Indianapolis
- Indiana Hemophilia and Thrombosis Center, Inc - Indianapolis
- Innovative Hematology, Inc. - Indianapolis
- St. Vincent Hospital & Health Care Center, Inc. - Indianapolis
Pennsylvania
- Center for Human Phenomic Science - Philadelphia
- Investigational Drug Service - Philadelphia
- Penn Blood Disorder Center - Philadelphia
- The Trustees of the University of Pennsylvania, Office of Clinical Research- Legal - Philadelphia
Colorado
- Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus - Aurora
- Regents of the University of Colorado University of Colorado Denver, Office of Grants and Contracts - Aurora
Mississippi
- Madison Radiological Group - Madison
- Mississippi Center for Advanced Medicine - Madison
New South Wales
- Royal Prince Alfred Hospital - Camperdown
South Australia
- Royal Adelaide Hospital - Adelaide
Victoria
- The Alfred Hospital - Melbourne
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 51 participants |
| Start Date | 2019-07-29 |
| Est. Completion | 2031-02-25 |
| Phase | Phase 3 |
What the registry record for NCT03861273 still lists
NCT03861273 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 51 participants, a relatively small participant target, below the 94-participant average among 20 other Hemophilia B trials with a reported enrollment target (46% lower).
The record links to 1 condition, with Hemophilia B appearing as the primary indexed condition, and to 1 intervention - of which PF-06838435/ fidanacogene elaparvovec is the first listed.
NCT03861273 names 20 study sites across 9 states, led by California, Indiana, Pennsylvania.
Frequently Asked Questions
What is clinical trial NCT03861273 about?
NCT03861273 is a clinical study titled "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". 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 cell...
What is the current status of trial NCT03861273?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 51 participants. The study started on 2019-07-29. Estimated completion is 2031-02-25.
What conditions does trial NCT03861273 study?
This clinical trial studies the following conditions: Hemophilia B.
What interventions are being tested in trial NCT03861273?
The interventions under investigation include: PF-06838435/ fidanacogene elaparvovec (BIOLOGICAL).
Who is sponsoring clinical trial NCT03861273?
This trial is sponsored by Pfizer, which has 1,845 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03861273 being conducted?
This trial has 20 study locations across California, Colorado, Indiana, Mississippi, Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT03861273's enrollment target sits among peer trials
51 9th of 20 higher than 12 of 20 other Hemophilia B trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Hemophilia B trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
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