NCT07575308PHASE2Recruiting
HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB
Alnylam Pharmaceuticals
Start Date
7/30/2026
Completion Date
11/29/2028
Summary
The purpose of this study is to evaluate the safety, tolerability, efficacy, and pharmacodynamics (PD) of multiple doses of ALN-6400 in adult and adolescent patients with VWD and HMB
Eligibility Criteria
Age Range: 16 years to 45 years
Inclusion Criteria -
* Is an adolescent or adult patient with a diagnosis of VWD (including Type 1, Type 2, Type 3, and platelet-type VWD) and HMB for 2 cycles during screening
Exclusion Criteria -
* Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) greater than 2 times the upper limit of normal (ULN)
* Has total bilirubin greater than 1.5×ULN (except for patients with Gilbert's syndrome)
* Has an estimated glomerular filtration (eGFR) less than 30 mL/min/1.73m\^2 at screening
* Is not willing to comply with the contraceptive requirements during the study period
* Used routine factor prophylaxis (greater than or equal to 1 infusion of factor per week over 12 weeks) within 4 weeks of screening or plans use for routine factor prophylaxis during the study
* New placement of any hormonal or nonhormonal intrauterine device (IUD) or hormonal implants within 24 weeks of screening
* Known current gynecological conditions causing abnormal uterine bleeding (including infection, fibroids, endometriosis, polycystic ovary syndrome, or dysplasia)
Note: other protocol defined inclusion / exclusion criteria apply
Interventions
DRUG
ALN-6400
Conditions
Von Willebrand Disease (VWD)Heavy Menstrual Bleeding (HMB)
Locations
Clinical Trial Site
Orange, California 92868
United States
Clinical Trial Site
San Diego, California 92123
United States
Clinical Trial Site
San Francisco, California 94143
United States
Clinical Trial Site
Miami, Florida 33024
United States
Clinical Trial Site
Lexington, Kentucky 40536
United States
Clinical Trial Site
Pittsburgh, Pennsylvania 15213
United States
Clinical Trial Site
Nijmegen, 6525 GA
Netherlands
Clinical Trial Site
Stockholm, 171 76
Sweden
Clinical Trial Site
Bradford, BD9 6RJ
United Kingdom
Clinical Trial Site
Oxford, OX3 7HE
United Kingdom
Clinical Trial Site
Truro, TR1 3LJ
United Kingdom