PF-06838435 for Hemophilia B

Recruiting at 30 trial locations

Overseen By Pfizer CT.gov Call Center

Age: 18+

Sex: Male

Trial Phase: Phase 2

Sponsor: Pfizer

Must be taking: Factor IX products

Must not be taking: Antivirals, others

Disqualifiers: Hepatitis, HIV, Liver disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

21 Participants Needed

What You Need to Know Before You Apply

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.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, if you are on antiviral therapy for hepatitis B or C, you cannot participate in the study.

What data supports the effectiveness of the drug PF-06838435 for Hemophilia B?

The effectiveness of treatments similar to PF-06838435, such as recombinant factor IX Fc fusion protein, has been demonstrated in studies showing they help manage Hemophilia B by reducing bleeding episodes and improving safety over long-term use. ^{1 2 3 4 5}

Who Is on the Research Team?

Pfizer CT.gov Call Center

Principal Investigator

Pfizer

Are You a Good Fit for This Trial?

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

If you're a male, you will need to avoid having sex or use reliable barrier contraception until three consecutive semen samples show no signs of the study's drug.

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

You understand what the study is about and agree to follow its rules.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Dose-Escalation Treatment

Single intravenous infusion of PF-06838435 with potential dose adjustments based on initial participant data

Up to 52 weeks

Long-term Follow-up

Participants are monitored for long-term safety and efficacy, including quality-of-life assessments and bleeding events

Up to 6 years

What Are the Treatments Tested in This Trial?

Interventions

• PF-06838435

Trial Overview 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.

How Is the Trial Designed?

Group I: PF-06838435 Dose-Escalation Experimental Treatment 1 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.

Who Is Running the Clinical Trial?

Pfizer

Lead Sponsor

Trials

4,712

Recruited

50,980,000+

Known For

Vaccine Innovations

Top Products

Viagra, Zoloft, Lipitor, Prevnar 13

Albert Bourla

Pfizer

Chief Executive Officer since 2019

PhD in Biotechnology of Reproduction, Aristotle University of Thessaloniki

Patrizia Cavazzoni

Pfizer

Chief Medical Officer

MD from McGill University

Published Research Related to This Trial

BAY 94-9027 demonstrated effective bleed prevention in children with severe hemophilia A, with a median annualized bleeding rate of 2.9 in the first part of the study and 2.4 in the second part, indicating its efficacy across different age groups.

No patients developed FVIII inhibitors during the study, suggesting a favorable safety profile, although 12 patients discontinued treatment due to loss of efficacy or hypersensitivity, primarily in the younger cohort.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

PROTECT VIII Kids: BAY 94-9027 (PEGylated Recombinant Factor VIII) safety and efficacy in previously treated children with severe haemophilia A. Santagostino, E., Kenet, G., Fischer, K., et al.[2020]

N8-GP, an extended half-life recombinant factor VIII, effectively maintains FVIII levels above 1 IU/dL in both adults and children with severe hemophilia A during prophylaxis, with dosing schedules of every 4 days for adults/adolescents and twice weekly for children.

The pharmacokinetic study involving 69 patients showed that N8-GP's half-life is positively correlated with von Willebrand factor levels in adults/adolescents, suggesting that this relationship may influence dosing strategies for optimal treatment outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fixed doses of N8-GP prophylaxis maintain moderate-to-mild factor VIII levels in the majority of patients with severe hemophilia A. Chowdary, P., Carcao, M., Holme, PA., et al.[2023]

Citations

1. Switzerland pubmed.ncbi.nlm.nih.gov

Joint Mobility and Physical Function of Danish Hemophilia Patients: A Three-Wave Panel Study Spanning 24 Years. [2019]

2. England pubmed.ncbi.nlm.nih.gov

Long-term safety and sustained efficacy for up to 5 years of treatment with recombinant factor IX Fc fusion protein in subjects with haemophilia B: Results from the B-YOND extension study. [2021]

3. Switzerland pubmed.ncbi.nlm.nih.gov

Improved Pharmacokinetics with BAY 81-8973 Versus Antihemophilic Factor (Recombinant) Plasma/Albumin-Free Method: A Randomized Pharmacokinetic Study in Patients with Severe Hemophilia A. [2022]

4. England pubmed.ncbi.nlm.nih.gov

PROTECT VIII Kids: BAY 94-9027 (PEGylated Recombinant Factor VIII) safety and efficacy in previously treated children with severe haemophilia A. [2020]

5. United States pubmed.ncbi.nlm.nih.gov

Fixed doses of N8-GP prophylaxis maintain moderate-to-mild factor VIII levels in the majority of patients with severe hemophilia A. [2023]