PF-06838435 for Hemophilia B

Phase 2

Waitlist Available

Research Sponsored by Pfizer

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

No measurable factor IX inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor IX protein

Received ≥50 exposure days to factor IX products

Timeline

Screening 3 weeks

Treatment Varies

Follow Up year 2 up to year 6

Awards & highlights

No Placebo-Only Group

Summary

This trial is for people with Hemophilia B who have been treated before in the C0371005 study, as well as a substudy for people who need a higher dose of the treatment.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Potential side effects may include immune reactions to the therapy, issues related to injection site, liver enzyme elevations, or development of inhibitors against factor IX which could reduce treatment efficacy.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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

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

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

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ year 2 up to year 6

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~year 2 up to year 6

This trial's timeline: 3 weeks for screening, Varies for treatment, and year 2 up to year 6 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Annualized (factor FIX) infusion rate

Annualized bleeding rate (ABR)

Brief Pain Inventory

+11 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

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.

0 / 3Eligibility criteria met