HMBeacon: Phase 2 Study of ALN-6400 in Females with VWD and HMB
Research type
Research Study
Full title
HMBeacon: A Phase 2, Randomized, Double-blind Study of the Safety, Tolerability, Efficacy, and Pharmacodynamics of Multiple Dose ALN-6400 in Adult and Adolescent Female Patients with Von Willebrand Disease (VWD) and Heavy Menstrual Bleeding (HMB)
IRAS ID
1013589
Contact name
Maria Sotirova
Contact email
Sponsor organisation
Alnylam Pharmaceuticals, Inc.
Research summary
Researchers want to learn about the safety and effects of the study medicine (ALN-6400) for a bleeding disorder called von Willebrand Disease (VWD). People with VWD do not have enough protein (Von Willebrand Factor) or this protein doesn't work correctly in their blood, making it hard to form an “inner Band-Aid®” or blood clot. This leads to bleeding from the body's linings, like the mouth, nose, stomach, gut, uterine lining. Heavy menstrual bleeding (HMB) is one of the most difficult symptoms reported by women with VWD because it recurs each month & may not respond sufficiently to available therapies that doctors use, like medicines that impact hormone levels or those used to prevent pregnancy.
In this study, researchers are testing ALN-6400 to see if it can reduce blood levels of a protein produced in the liver called plasminogen. Plasminogen plays a key role in helping the body break down blood clots. By lowering plasminogen levels in the blood, researchers expect to stabilize the blood clots and reduce bleeding symptoms in people with VWD and HMB. Researchers are testing ALN-6400 to see if it is safe and if it can help prevent or reduce HMB and other bleeding.
The study includes up to 24 female participants between 16 and 45 years old with both VWD and HMB. Participants will be assigned by chance to receive 1 of 2 dose levels of ALN-6400. Neither the participants nor the study staff will know which dose level of study medicine each participant gets. This is called a double-blind. Study medicine will be given as an injection under the skin (called subcutaneous).
Eligible participants may receive ALN-6400 during the following periods. Double-blind Period: 2 groups of participants are assigned by chance to receive ALN-6400 at the first or the second dose level once every 3 months (total of 2 doses). Optional Extension Period: Same 2 groups of participants may receive 4 more doses of ALN-6400. These periods will be followed by a Follow-up Period up to 24 weeks.REC name
London - Brighton & Sussex Research Ethics Committee
REC reference
26/LO/0269
Date of REC Opinion
8 May 2026
REC opinion
Further Information Favourable Opinion