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NCT Number: NCT07575308

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

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

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Key information

Conditions

Von Willebrand Disease (VWD) Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Blood Platelet Disorders Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Genetic Diseases, Inborn Genital Diseases Genital Diseases, Female Heavy Menstrual Bleeding (HMB) Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhage Hemorrhagic Disorders Menorrhagia Menstruation Disturbances Pathologic Processes Pathological Conditions, Signs and Symptoms Urogenital Diseases Uterine Diseases Uterine Hemorrhage von Willebrand Diseases

Age range

16 year–45 year

Sex eligibility

Female

Study type

Interventional

Phase

Phase 2

Primary location

Clinical Trial Site

Miami, Florida, 33024, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

ALN-6400

Drug

ALN-6400 will be administered subcutaneously (SC).

Primary outcomes

  1. Frequency of Adverse Events

    Time frame: Up to Week 72

Secondary outcomes

  1. Change from Baseline in Plasminogen (PLG) Plasma Activity Levels

    Time frame: Screening and up to Week 72 postdose

  2. Change from Baseline in PLG Plasma Protein Levels

    Time frame: Screening and up to Week 72 postdose

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

    Time frame: Screening and up to Week 72 postdose

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

    Time frame: Week 4 up to Week 72

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

    Time frame: Baseline up to Week 72

Study contacts

Contact information is provided by the study sponsor or research team.

Alnylam Clinical Trial Information Line

CONTACT

[email protected]

1-877-ALNYLAM

Alnylam Clinical Trial Information Line

CONTACT

[email protected]

1-877-256-9526

Sponsors and collaborators

Lead sponsor

Alnylam Pharmaceuticals

Industry

Registry information

Official study 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)

Acronym: HMBeacon

Important dates

Study start
2026
Primary completion
2027
Study completion
2028
First posted
May 8, 2026
Registry last updated
Jul 14, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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