CompletedPhase 1

Phase 1 Safety, Pharmacokinetics And Pharmacodynamics Study Of Recombinant Factor VIIa Variant (813d) In Adult Subjects With Hemophilia

United States, Belgium, Hungary29 participantsStarted 2011-12

Plain-language summary

This study hypothesizes that the study drug, PF-05280602 (at the selected doses) will be safe to administer to subjects with severe Hemophilia A or B with or without inhibitors and will demonstrate evidence of hemostatic activity. This is supported by the preclinical findings in hemophilic animal models.

Who can participate

Age range

18 Years – 64 Years

Sex

MALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Male subjects 18 to \<65 years old with severe hemophilia A or B with or without inhibitors to FVIII or FIX. * Subjects is willing and able to comply with the mandatory washout periods prior to screening and prior to dosing and through 48 hours post dosing. At screening this includes a washout of FIX for 96 hours and FVIII for 72 houts. At dosing this includes a washout of FIX for 96 hours and FVIII and other hemostatic agents for 72 hours through 48 hours post dosing. * Subjects must agree and commit to using a a highly effective method of birth control from the time of screening through four weeks after study drug administration. Exclusion Criteria: * Presence of a bleeding disorder in addition to hemophilia A or B. * Regular, concomitant therapy with immunomodulating drugs (eg, intravenous immunoglobulin, and routine systemic corticosteroids). * History of coronary artery disease, thrombolic disease or diagnosis of prothrombic disorder.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Change From Baseline in Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP)

Timeframe: Baseline, Day 2, Day 3, and Day 15

Change From Baseline in Body Weight

Timeframe: Baseline, Day 2, Day 3, and Day 15

Change From Baseline in Body Temperature

Timeframe: Baseline, Day 2, Day 3, and Day 15

Change From Baseline in Respiration Rate

Timeframe: Baseline, Day 2, Day 3, and Day 15

Change From Baseline in Supine Pulse Rate

Timeframe: Baseline, Day 2, Day 3, and Day 15

Number of Participants With Changes Since Previous Physical Examination

Timeframe: Baseline (Day 0), Day 1, Day 2, Day 3, Day 15

Number of Participants With Potentially Clinically Significant Electrocardiogram (ECG) Findings

Timeframe: Baseline through Day 15

Trial details

NCT IDNCT01439971

SponsorCatalyst Biosciences

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2015-10

Results submitted2016-10-20

View on ClinicalTrials.gov More Hemophilia A trials

Plain-language summary

Who can participate

Age range

18 Years – 64 Years

Sex

MALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Change From Baseline in Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP)

Timeframe: Baseline, Day 2, Day 3, and Day 15

Change From Baseline in Body Weight

Timeframe: Baseline, Day 2, Day 3, and Day 15

Change From Baseline in Body Temperature

Timeframe: Baseline, Day 2, Day 3, and Day 15

Change From Baseline in Respiration Rate

Timeframe: Baseline, Day 2, Day 3, and Day 15

Change From Baseline in Supine Pulse Rate

Timeframe: Baseline, Day 2, Day 3, and Day 15

Number of Participants With Changes Since Previous Physical Examination

Timeframe: Baseline (Day 0), Day 1, Day 2, Day 3, Day 15

Number of Participants With Potentially Clinically Significant Electrocardiogram (ECG) Findings

Timeframe: Baseline through Day 15