← Back to Search

Gene Therapy

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

Study 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.Check my eligibility

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.See study design

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

Select...

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

Select...

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

Select...

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Incidence of PF-06838435 related adverse events

Secondary outcome measures

Annualized (factor FIX) infusion rate

Annualized bleeding rate (ABR)

Brief Pain Inventory

+11 more

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

Find a Location

Who is running the clinical trial?

PfizerLead Sponsor

4,567 Previous Clinical Trials

10,911,852 Total Patients Enrolled

20 Trials studying Hemophilia B

4,947 Patients Enrolled for Hemophilia B

Pfizer CT.gov Call CenterStudy DirectorPfizer

3,474 Previous Clinical Trials

8,093,020 Total Patients Enrolled

21 Trials studying Hemophilia B

1,973 Patients Enrolled for Hemophilia B

Media Library

PF-06838435 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03307980 — Phase 2

Hemophilia B Research Study Groups: PF-06838435 Dose-Escalation

Hemophilia B Clinical Trial 2023: PF-06838435 Highlights & Side Effects. Trial Name: NCT03307980 — Phase 2

PF-06838435 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03307980 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are the goals that this experiment is looking to achieve?

"According to the clinical trial sponsor, Pfizer, the main objective of the study is to assess the number of participants with clinical thrombotic events over a 52-week period. Additionally, the study will also collect data on secondary outcomes including mean and standard deviation of FIX Antigen levels, vector-derived FIX Activity levels, and quality-of-life assessment (measured using the Brief Pain Inventory)."

Answered by AI

In how many different areas is this experiment being done today?

"This trial is being conducted at 6 different medical facilities, which are located in Montreal, Slidell, New Orleans and other locations."

Answered by AI

Does this experiment test elderly individuals?

"The age restrictions for this study are that participants must be between 18 and 65 years old."

Answered by AI

How many people are being recruited for this experiment?

"In order for the trial to proceed as planned, 55 patients who fit the clinical profile must be recruited. The sponsor of the study, Pfizer, will manage recruitment from various sites - such as McGill University Health Center - Research Institute in Montreal, Quebec and McMaster University Medical Centre in Slidell, Louisiana."

Answered by AI

Are there any available vacancies for this clinical trial?

"Yes, this trial is still searching for patients. The original posting date was June 22nd, 2017, and the most recent update was on October 4th, 2022."

Answered by AI

Are there any know adverse effects of PF-06838435?

"There is only preliminary data supporting the safety of PF-06838435, as this medication is still in Phase 2 clinical trials."

Answered by AI

Who does this research involve?

"Up to 55 patients, that currently suffer from hemophilia b and are within the ages of 18 to 65, can be admitted into this trial. Furthermore, it is mandatory that participants meet the following qualifications: Male, have had no prior history with inhibitors towards factor IX protein, willing to provide informed consent, agree to not donate sperm, and must use contraception if they plan on engaging in intercourse."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B

2017. "Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03307980.

All > Hemophilia A > Hemophilia