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
Public ClinicalTrials.gov record NCT03861273. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official 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)
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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).
Study identification
- NCT ID
- NCT03861273
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 51 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 18 Years to 65 Years
- Sex
- Male
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jul 28, 2019
- Primary completion
- Nov 15, 2022
- Completion
- Feb 24, 2031
- Last update posted
- May 31, 2026
2019 – 2031
United States locations
- U.S. sites
- 17
- U.S. states
- 5
- U.S. cities
- 6
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| UCSF IDS Pharmacy | San Francisco | California | 94143 | — |
| University of California, San Francisco - Clinical Research Center | San Francisco | California | 94143 | — |
| University of California, San Francisco - Outpatient Hematology Clinic | San Francisco | California | 94143 | — |
| The Regents of the University of California, San Francisco campus | San Francisco | California | 94158 | — |
| Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus | Aurora | Colorado | 80045 | — |
| Regents of the University of Colorado University of Colorado Denver, Office of Grants and Contracts | Aurora | Colorado | 80045 | — |
| Indiana Hemophilia & Thrombosis Center, Inc. | Indianapolis | Indiana | 46260 | — |
| Indiana Hemophilia and Thrombosis Center, Inc | Indianapolis | Indiana | 46260 | — |
| Innovative Hematology, Inc. | Indianapolis | Indiana | 46260 | — |
| St. Vincent Hospital & Health Care Center, Inc. | Indianapolis | Indiana | 46260 | — |
| Madison Radiological Group | Madison | Mississippi | 39110 | — |
| Mississippi Center for Advanced Medicine | Madison | Mississippi | 39110 | — |
| The Trustees of the University of Pennsylvania, Office of Clinical Research- Legal | Hamilton | Pennsylvania | 19104 | — |
| Center for Human Phenomic Science | Philadelphia | Pennsylvania | 19104 | — |
| Investigational Drug Service | Philadelphia | Pennsylvania | 19104 | — |
| Penn Blood Disorder Center | Philadelphia | Pennsylvania | 19104 | — |
| The Trustees of the University of Pennsylvania, Office of Clinical Research- Legal | Philadelphia | Pennsylvania | 19104 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 50 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03861273, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted May 31, 2026 · Synced Sep 1, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03861273 live on ClinicalTrials.gov.