HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB

Alnylam Pharmaceuticals

Status and phase: Begins enrollment this month; Phase 2

Conditions: Von Willebrand Disease (VWD); Heavy Menstrual Bleeding (HMB)

Treatments: Drug: ALN-6400

Study type: Interventional

Funder types: Industry

Identifiers

NCT07575308

ALN-6400-002

2025-524967-19-00 (EU Trial (CTIS) Number)

Details and patient eligibility

About

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

Enrollment: 24 estimated patients

Sex: Female

Ages: 16 to 45 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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24 participants in 2 patient groups

ALN-6400 (Treatment Group A)

Experimental group

Description:

Participants will be administered multiple doses of ALN-6400.

Treatment:

Drug: ALN-6400

ALN-6400 (Treatment Group B)

Experimental group

Description:

Participants will be administered multiple doses of ALN-6400.

Treatment:

Drug: ALN-6400