PF-06838435 for Hemophilia B

Not currently recruiting at 32 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial focuses on testing a treatment called PF-06838435 for individuals with Hemophilia B, a condition where blood doesn't clot properly due to low levels of factor IX protein. Researchers aim to determine if increasing the dose of this treatment is safe and effective over a long period. The trial is open to men with Hemophilia B who have previously used factor IX products, excluding those with certain medical conditions like active hepatitis or significant liver disease. As a Phase 2 trial, the research measures how well the treatment works in an initial, smaller group of participants.

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.

Is there any evidence suggesting that PF-06838435 is likely to be safe for humans?

Research shows that PF-06838435, a gene therapy for hemophilia B, has been studied for its safety and tolerability. Earlier studies have found that adults with hemophilia B generally tolerate this treatment well. Serious side effects are rare, with most participants experiencing mild to moderate reactions that were manageable.

This gene therapy uses a virus to deliver a missing clotting protein. It has been tested in several studies, including one that showed promising safety results and another currently examining its long-term safety and effectiveness.

For those considering a trial for PF-06838435, past research supports its safety in humans. However, as with any medical treatment, discussing potential risks and benefits with a healthcare provider is important. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Hemophilia B?

Unlike the standard treatments for Hemophilia B, which typically involve regular infusions of clotting factor IX concentrates, PF-06838435 is a gene therapy that aims to provide a long-term solution with just a single intravenous infusion. This innovative treatment uses a viral vector to deliver a functional copy of the factor IX gene directly into the patient's cells, potentially allowing their body to produce the necessary clotting factor on its own. Researchers are excited about PF-06838435 because it could significantly reduce or even eliminate the need for frequent infusions, offering patients a more convenient and potentially more effective way to manage their condition.

What evidence suggests that PF-06838435 might be an effective treatment for Hemophilia B?

Research has shown that PF-06838435, a gene therapy treatment under study in this trial, could benefit people with Hemophilia B. In an earlier study, patients who received this treatment had higher levels of factor IX, a key protein for blood clotting. This increase led to fewer bleeding episodes and reduced the need for regular clotting factor infusions. Early results also suggest that PF-06838435 can offer long-lasting benefits by maintaining factor IX levels at or near normal. Overall, these findings support its potential effectiveness in managing Hemophilia B. ^{1 2 3 6 7}

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. clinicaltrials.gov clinicaltrials.gov/study/NCT03861273

NCT03861273 | A Study to Evaluate the Efficacy and ... 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

2. pfizer.com pfizer.com/sites/default/files/plsr-studies/C0371002%20Plain%20Language%20Study%20Results%20Summary%20%28PLSRS%29%20Phase%202-4.pdf

Clinical Study Results Hemophilia B is a bleeding disease that happens due to lack of a protein which helps in blood clotting. This protein is called coagulation ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT03307980

NCT03307980 | Long-term Safety and Efficacy Study and ... 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 ...

4. ashpublications.org ashpublications.org/bloodadvances/article/9/14/3543/536584/Completion-of-phase-2b-trial-of-etranacogene

Completion of phase 2b trial of etranacogene dezaparvovec ... Etranacogene dezaparvovec induced stable, sustained normal/near-normal FIX levels and eliminated the need for prophylaxis over 5 years.

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10341900/

Gene Therapy Approaches for the Treatment of Hemophilia B As of May 2023, two AAV-based drugs were in phase 3 clinical trials: SPK-9001 (PF-06838435/Fidanacogene elaparvovec, Pfizer, New York, NY, USA) ...

6. orpha.net orpha.net/en/research-trials/clinical-trial/605308

BENEGENE 3: Phase 3 open-label study to evaluate ... BENEGENE 3: Phase 3 open-label study to evaluate efficacy, safety, and tolerability of FIX gene transfer with fidanacogene elaparvovec (PF-06838435) in ...

7. fda.gov fda.gov/media/178737/download

Clinical Review Memo - BEQVEZ BEQVEZ is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with moderate to severe hemophilia B ...