PF-06838435 for Hemophilia B

Palo Alto (17 mi)

Age: 18+

Sex: Male

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Pfizer

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?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.

Eligibility Criteria

This trial is for males aged 18-65 with Hemophilia B (severe, ≤2% factor IX) who've used clotting factor IX products for at least 50 days. They must not have inhibitors to factor IX and agree to specific contraception until semen tests are clear of the vector. Prior participation in study C0371005 isn't required for the sub-study.

Inclusion Criteria

I don't have factor IX inhibitors and never had them.

I have been treated with factor IX products for 50 days or more.

I am a male aged 18-65 with severe hemophilia B.

Treatment Details

The trial studies long-term safety and effectiveness of PF-06838435 in those with Hemophilia B. It includes a follow-up from an earlier study and a new dose-escalation sub-study to assess higher doses over time.

1Treatment groups

Experimental Treatment

Group I: PF-06838435 Dose-EscalationExperimental Treatment1 Intervention

Single intravaneous infusion of PF-06838435. After 2 participants receive initial dose, data will be evaluated and a decision will be made to escalate or reduce the dose being evaluated, increase the number of participants receiving the dose, or stop dosing. Multiple iterations may be undertaken.