Trials / Active Not Recruiting
Active Not RecruitingNCT03307980
Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B
A FACTOR IX (FIX) GENE TRANSFER, MULTI CENTER EVALUATION OF THE LONG TERM SAFETY AND EFFICACY STUDY OF PF 06838435 AND A DOSE ESCALATION SUBSTUDY IN INDIVIDUALS WITH HEMOPHILIA B
- Status
- Active Not Recruiting
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 21 (actual)
- Sponsor
- Pfizer · Industry
- Sex
- Male
- Age
- 18 Years – 65 Years
- Healthy volunteers
- Not accepted
Summary
Long-term safety and efficacy follow-up for participants with Hemophilia B who were previously treated in the C0371005 (formerly SPK-9001-101) study, and a dose-escalation sub-study evaluating safety, tolerability, and kinetics of a higher dose with long-term safety and efficacy follow-up. Participants in the substudy do not need to have participated in C0371005.
Detailed description
Evaluation of the long-term level of persistence and potential late or delayed adverse events associated with PF-06838435 (formerly SPK-9001), assessment of the durability of the transgene expression, and determination of the effects of PF-06838435 on clinical outcomes in individuals who have previously received a single administration of PF-06838435 in the C0371005 study. Amendment 2 of this study incorporates a dose-escalation substudy to evaluate the safety, tolerability, and kinetics of a single IV infusion of PF-06838435 at a higher dose than that used in the C0371005 study. The dose-escalation participants will also be followed for long-term safety and efficacy.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | PF-06838435 (formerly SPK-9001) | Gene Therapy: A novel, bioengineered adeno-associated viral vector carrying human factor IX variant |
Timeline
- Start date
- 2017-06-22
- Primary completion
- 2029-05-29
- Completion
- 2029-10-25
- First posted
- 2017-10-12
- Last updated
- 2026-03-18
Locations
25 sites across 4 countries: United States, Australia, Canada, Turkey (Türkiye)
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03307980. Inclusion in this directory is not an endorsement.