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

NCT07575308 | Not Yet Recruiting Phase 2 FDA Drug

• 2 other identifiers: ALN-6400-0022025-524967-19-00
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief 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

Conditions

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

Keywords

siRNA, RNAi therapeutic, Plasminogen, PLG, VWD, HMB

Outcome Measures

Primary Outcomes (1)

• Frequency of Adverse Events

  Up to Week 72

Secondary Outcomes (5)

• Change from Baseline in Plasminogen (PLG) Plasma Activity Levels

  Screening and up to Week 72 postdose

• Change from Baseline in PLG Plasma Protein Levels

  Screening and up to Week 72 postdose

• Change from Baseline in Menstrual Blood Loss Via Pictorial Blood Assessment Chart (PBAC)

  Screening and up to Week 72 postdose

• Patient-reported Outcome (PRO) assessed by Patient Global Impression of Change (PGI-C) Score

  Week 4 up to Week 72

• Change from Baseline in PRO assessed by Patient Global Impression of Severity (PGI-S) Score

  Baseline up to Week 72

Study Arms (2)

ALN-6400 (Treatment Group A)

EXPERIMENTAL

Participants will be administered multiple doses of ALN-6400.

Drug: ALN-6400

ALN-6400 (Treatment Group B)

EXPERIMENTAL

Participants will be administered multiple doses of ALN-6400.

Drug: ALN-6400

Interventions

ALN-6400 DRUG

ALN-6400 will be administered subcutaneously (SC).

ALN-6400 (Treatment Group A); ALN-6400 (Treatment Group B)

Eligibility Criteria

• Age: 16 Years - 45 Years
• Sex: female
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• Alnylam Pharmaceuticals: lead

Study Sites (1)

Clinical Trial Site

Miami, Florida, 33024, United States

Location

MeSH Terms

Conditions

von Willebrand Diseases; Menorrhagia

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Blood Platelet Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Uterine Hemorrhage; Uterine Diseases; Genital Diseases, Female; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Genital Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Menstruation Disturbances

Study Officials

• Medical Director

  Alnylam Pharmaceuticals

  STUDY DIRECTOR

Central Study Contacts

Alnylam Clinical Trial Information Line

CONTACT

1-877-ALNYLAM; clinicaltrials@alnylam.com

Alnylam Clinical Trial Information Line

CONTACT

1-877-256-9526; clinicaltrials@alnylam.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 4, 2026
• First Posted: May 8, 2026
• Study Start: May 5, 2026
• Primary Completion (Estimated): June 30, 2027
• Study Completion (Estimated): November 29, 2028
• Last Updated: May 8, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will share

Locations

US (1)