Von Willebrand Disease (VWD)Phase 2HMBeaconNCT07575308
HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB
A Phase 2 study testing ALN-6400 in people with Von Willebrand Disease (VWD) and Heavy Menstrual Bleeding (HMB), run by Alnylam Pharmaceuticals at 11 sites in 4 countries.
Trial 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 Read more about this study
- Who is it for?
- Ages 16 to 45 years, women only
- Conditions the study is for
- Von Willebrand Disease (VWD), Heavy Menstrual Bleeding (HMB)
- What is being tested?
- ALN-6400
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 11 sitesin 4 countries
- Study dates
- 30 Jul 2026to 29 Nov 2028
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 to 45 years
- Women only
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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 locations and contacts
11 sites in 4 countries. Contact the study team or a site near you to ask about taking part.
11 sites in 4 countries
United States6 sites, 6 recruiting
- Recruiting
Clinical Trial Site
Lexington, Kentucky, 40536
- Recruiting
Clinical Trial Site
Miami, Florida, 33024
- Recruiting
Clinical Trial Site
Orange, California, 92868
- Recruiting
Clinical Trial Site
Pittsburgh, Pennsylvania, 15213
- Recruiting
Clinical Trial Site
San Diego, California, 92123
- Recruiting
Clinical Trial Site
San Francisco, California, 94143
United Kingdom3 sites, 3 recruiting
- Not yet recruiting
Clinical Trial Site
Bradford, BD9 6RJ
- Not yet recruiting
Clinical Trial Site
Oxford, OX3 7HE
- Not yet recruiting
Clinical Trial Site
Truro, TR1 3LJ
Netherlands1 site, 1 recruiting
- Not yet recruiting
Clinical Trial Site
Nijmegen, 6525 GA
Sweden1 site, 1 recruiting
- Not yet recruiting
Clinical Trial Site
Stockholm, 171 76
About this study
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)
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
- Being tested
- ALN-6400
- Keywords
- siRNA, RNAi therapeutic, Plasminogen, PLG, VWD, HMB
- Medical subject headings
- von Willebrand Diseases, Menorrhagia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALN-6400Drug
ALN-6400 will be administered subcutaneously (SC).
| Study group | Role | What they receive |
|---|---|---|
| ALN-6400 (Treatment Group A) | Experimental | Participants will be administered multiple doses of ALN-6400. Receives: Drug: ALN-6400 |
| ALN-6400 (Treatment Group B) | Experimental | Participants will be administered multiple doses of ALN-6400. Receives: Drug: ALN-6400 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency of Adverse Events
Measured: Up to Week 72
Secondary outcomes (5)
Change from Baseline in Plasminogen (PLG) Plasma Activity Levels
Measured: Screening and up to Week 72 postdose
Change from Baseline in PLG Plasma Protein Levels
Measured: Screening and up to Week 72 postdose
Change from Baseline in Menstrual Blood Loss Via Pictorial Blood Assessment Chart (PBAC)
Measured: Screening and up to Week 72 postdose
Patient-reported Outcome (PRO) assessed by Patient Global Impression of Change (PGI-C) Score
Measured: Week 4 up to Week 72
Change from Baseline in PRO assessed by Patient Global Impression of Severity (PGI-S) Score
Measured: Baseline up to Week 72
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 May 2026
- Study start30 Jul 2026
- Primary completion30 Jun 2027 (estimated)
- Study completion29 Nov 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Von Willebrand Disease (VWD)Studies of the same condition, open and under way.
- More trials run by Alnylam PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Alnylam Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Alnylam Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07575308
- Sponsor's protocol ID
- ALN-6400-002
- Other IDs
- 2025-524967-19-00, 2025
- Acronym
- HMBeacon
- Status last verified
- Sep 2026
- First submitted
- 4 May 2026
- Last updated
- 18 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Phase 2-4: Access to Anonymized individual participant data that support these results is made available 12 months after study completion and not less than 12 months after the product and indication have been approved in the United States (US) and/or the European Union (EU). Data will be provided contingent upon the approval of a research proposal and the execution of a data sharing agreement. Requests for access to data can be submitted via the website www.vivli.org.