BASIS KIDS
Research type
Research Study
Full title
AN OPEN-LABEL STUDY IN PEDIATRIC (<18 YEARS OF AGE), SEVERE HEMOPHILIA A PARTICIPANTS (COAGULATION FACTOR ACTIVITY <1%) WITH OR WITHOUT INHIBITORS OR MODERATELY SEVERE TO SEVERE HEMOPHILIA B PARTICIPANTS (COAGULATION FACTOR ACTIVITY ≤2%) WITH OR WITHOUT INHIBITORS COMPARING 12 MONTHS OF HISTORICAL STANDARD TREATMENT TO MARSTACIMAB PROPHYLAXISA
IRAS ID
1006432
Contact name
Annemarie Gregg
Contact email
Sponsor organisation
Pfizer Inc.
Clinicaltrials.gov Identifier
Research summary
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 haemophilia in paediatric patients.
This study will enrol paediatric participants from ages 1 to 17 years in a sequential manner. The study will open enrolment to adolescent participants aged 12 to 17 years first. Then children aged 6 to 11 years will be permitted to enrol. Lastly, children aged 1 to 5 years will be permitted to enrol.
This study will enrol participants who have severe haemophilia A or moderately severe to severe haemophilia B (with or without inhibitors).
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 during the 12-month treatment period.
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 haemophilia.
Participants will be in this study for approximately 14-15 months, including a screening period that may last up to 45-days, a 12month treatment phase during which the participant receives weekly prophylaxis treatment with marstacimab, and approximately 1-month follow-up for safety monitoring.REC name
South Central - Hampshire A Research Ethics Committee
REC reference
23/SC/0014
Date of REC Opinion
4 Aug 2023
REC opinion
Further Information Favourable Opinion