HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB
Purpose
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
Conditions
- Von Willebrand Disease (VWD)
- Heavy Menstrual Bleeding (HMB)
Eligibility
- Eligible Ages
- Between 16 Years and 45 Years
- Eligible Sex
- Female
- Accepts Healthy Volunteers
- No
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
Study Design
- Phase
- Phase 2
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Triple (Participant, Care Provider, Investigator)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental ALN-6400 (Treatment Group A) |
Participants will be administered multiple doses of ALN-6400. |
|
|
Experimental ALN-6400 (Treatment Group B) |
Participants will be administered multiple doses of ALN-6400. |
|
Recruiting Locations
Clinical Trial Site
Los Angeles, California 90027
Los Angeles, California 90027
Clinical Trial Site
Orange, California 92868
Orange, California 92868
Clinical Trial Site
Miami, Florida 33024
Miami, Florida 33024
Clinical Trial Site
Lexington, Kentucky 40536
Lexington, Kentucky 40536
More Details
- Status
- Recruiting
- Sponsor
- Alnylam Pharmaceuticals