MAA-304
Research type
Research Study
Full title
Phase 3 Study to Evaluate the Efficacy and Safety of Subcutaneous Marzeptacog alfa (activated) For On-Demand Treatment and Control of Bleeding Episodes in Subjects with Haemophilia A or Haemophilia B, with Inhibitors: The Crimson 1 Study.
IRAS ID
286475
Contact name
Cathy Farrelly
Contact email
Sponsor organisation
Catalyst Biosciences, Inc.
Eudract number
2020-000996-19
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
14,789, IND
Duration of Study in the UK
1 years, 11 months, 6 days
Research summary
Research Summary
Marzeptacog alfa (activated), or MarzAA, is being tested by Catalyst Biosciences, Inc. to be used as a subcutaneous treatment (i.e., a needle injection below the skin) for the on-demand treatment and control of bleeding in patients with Haemophilia A or B with inhibitors.MarzAA is a modified form of activated factor FVII protein. This modification may provide a greater ability to activate the clotting system and increase its duration of action.
This study will evaluate whether MarzAA can treat a bleed when given subcutaneously. MarzAA is an experimental drug and is not currently approved for treatment in the European Union or the United States. Subcutaneous injection is also an experimental way of injecting clotting factors to treat a bleed.
The aim is to enrol and randomise approximately 60 participants at approximately 50 sites worldwide. The estimated duration of the study is approximately 23 months with a projected maximum study duration of approximately 15 months for each participant, including the 30-day screening period.
The study will enrol male or female participants with congenital HA or HB with inhibitors who are ≥12 years of age, with an annualised bleeding rate (ABR) ≥8. Subjects will be randomized to begin treatment with either a sequence of MarzAA or Standard of Care (SOC) followed by treatment with SOC or MarzAA.
Summary of Results
No subjects have been enrolled in the UK. As the sponsor has terminated development of the investigational product, there will be no results.REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
20/EM/0248
Date of REC Opinion
18 Jan 2021
REC opinion
Further Information Favourable Opinion