HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: ALN-6400.
- Who it may be relevant to
- Registry conditions: Von Willebrand Disease (VWD), Heavy Menstrual Bleeding (HMB). Basic parameters: 16 years — 45 years · Female.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official 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)
Overview
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
Interventions
- Drug ALN-6400
ALN-6400 will be administered subcutaneously (SC).
Primary outcome measures
- Frequency of Adverse Events [Time frame: Up to Week 72]
Secondary outcome measures (5)
- Change from Baseline in Plasminogen (PLG) Plasma Activity Levels [Time frame: Screening and up to Week 72 postdose]
- Change from Baseline in PLG Plasma Protein Levels [Time frame: Screening and up to Week 72 postdose]
- Change from Baseline in Menstrual Blood Loss Via Pictorial Blood Assessment Chart (PBAC) [Time frame: Screening and up to Week 72 postdose]
- Patient-reported Outcome (PRO) assessed by Patient Global Impression of Change (PGI-C) Score [Time frame: Week 4 up to Week 72]
- Change from Baseline in PRO assessed by Patient Global Impression of Severity (PGI-S) Score [Time frame: Baseline up to Week 72]
Eligibility 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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Triple blind
- Primary purpose
- Treatment
Study locations
United States · 1 center
- Clinical Trial Site — Miami
Identifiers
NCT: NCT07575308 · ALN-6400-002 · 2025-524967-19-00