A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B

Part of paid clinical trials in Salt Lake City, Utah.

Sponsor
Pfizer
Study ID
NCT05611801
Phase
PHASE3
Status
Recruiting
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Conditions

  • Hemophilia A
  • Hemophilia B

Eligibility Criteria

Sex
MALE
Age
1 Year - 17 Years
Healthy Volunteers
Not accepted

Interventions

  • marstacimab — DRUG
    marstacimab

Study Details

The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called marstacimab) for the potential treatment of hemophilia in pediatric patients. This study will enroll pediatric participants from ages 1 to 17 years in a sequential manner. The study will open enrollment to adolescent participants aged 12 to 17 years first. Then children aged 6 to 11 years will be permitted to enroll. Lastly, children aged 1 to 5 years will be permitted to enroll. This study will enroll participants who: * have severe Hemophilia A or moderately severe to severe Hemophilia B (with or without inhibitors) * have accurate historical records documenting all factor VIII, factor IX, or bypass agent infusions and hemophilia bleed events for at least 1 year prior to entering the study * if a non-inhibitor patient, must be on a stable routine prophylaxis regimen with factor VIII or factor IX replacement products for at least 12 months prior to study entry * if an inhibitor patient, must be on an on-demand bypass treatment regimen during the 12 months prior to study entry All participants in this study will receive marstacimab to use prophylactically. Marstacimab will be given once a week as a subcutaneous (under the skin) shot. The first dose of marstacimab will be given at the study site by the study site staff. During the 12-month treatment period, weekly doses of marstacimab can be given at home, or if preferred, the doses may be given by the study site staff. To help us determine if the study medicine is safe and effective, we will compare participant experiences when they are taking the study medicine to a historical period when they were not. Researchers want to see if the study medicine works to prevent the bleeding episodes commonly experienced by patients with Hemophilia. Participants will be in this study for about 14 months (approximately 1 month in a Screening period, 12 months receiving treatment, and 1 month in a follow-up period) during which they will visit the study site at least 10 times. If preferred, and if local regulations allow it, 2 of the study visits can be completed at the participant's home instead of at the study site. There will also be 6 scheduled telephone calls approximately every 2 months.

Key Dates

First listed
Nov 10, 2022
Start date
Dec 9, 2022
Status verified
Aug 2026
Primary completion
Sep 10, 2028
Completion
Sep 10, 2028

Study Design

Enrollment
100 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: marstacimab (PF-06741086)
    Weekly subcutaneous injections.

Primary Outcome Measure

Annualized bleeding rate (ABR) of treated bleeding events [ Time Frame: Baseline to end of 12-month treatment period ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Intermountain - Primary Children's HospitalSalt Lake CityUtah84113-

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Intermountain - Primary Children's Hospital· Salt Lake City, UT

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