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 21 sites in 5 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
- 21 sitesin 5 countries
- Study dates
- 30 Jul 2026to 30 Aug 2029
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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
21 sites in 5 countries. Contact the study team or a site near you to ask about taking part.
21 sites in 5 countries
United States10 sites, 10 recruiting
- Not yet recruiting
Clinical Trial Site
Augusta, Georgia, 30912
- Not yet recruiting
Clinical Trial Site
Chapel Hill, North Carolina, 27514
- Not yet recruiting
Clinical Trial Site
Charleston, South Carolina, 29425
- Not yet recruiting
Clinical Trial Site
Cleveland, Ohio, 44195
- Not yet recruiting
Clinical Trial Site
Lexington, Kentucky, 40536
- Recruiting
Clinical Trial Site
Miami, Florida, 33143
- Not yet recruiting
Clinical Trial Site
Orange, California, 92868
- Not yet recruiting
Clinical Trial Site
Pittsburgh, Pennsylvania, 15213
- Not yet recruiting
Clinical Trial Site
San Diego, California, 92123
- Not yet recruiting
Clinical Trial Site
San Francisco, California, 94143
United Kingdom5 sites, 5 recruiting
- Not yet recruiting
Clinical Trial Site
Birmingham, B15 2GW
- Not yet recruiting
Clinical Trial Site
Bradford, BD9 6RJ
- Not yet recruiting
Clinical Trial Site
London, SE1 7EH
- Not yet recruiting
Clinical Trial Site
Oxford, OX3 7HE
- Not yet recruiting
Clinical Trial Site
Truro, TR1 3LJ
Turkey (Türkiye)3 sites, 3 recruiting
- Not yet recruiting
Clinical Trial Site
Istanbul, 34093
- Not yet recruiting
Clinical Trial Site
Şahinbey, 27070
- Not yet recruiting
Clinical Trial Site
Samsun, 55200
Netherlands2 sites, 2 recruiting
- Not yet recruiting
Clinical Trial Site
Nijmegen, 6525 GA
- Not yet recruiting
Clinical Trial Site
Utrecht, 3584 CX
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 completion30 Aug 2029 (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
- Oct 2026
- First submitted
- 4 May 2026
- Last updated
- 26 Oct 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.
Study at a glance
From the registry record, last updated by the sponsor on 26 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT07575308
- Acronym / sponsor ID
- HMBeacon / ALN-6400-002
- Keywords
- siRNA, RNAi therapeutic, Plasminogen, PLG, VWD, HMB
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- Alnylam PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 30 Jul 2026 (actual)
- Primary completion
- 30 Jun 2027 (estimated)
- Study completion
- 30 Aug 2029 (estimated)
- First posted
- 8 May 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ALN-6400 (Treatment Group A) | Experimental | Drug: ALN-6400Participants will be administered multiple doses of ALN-6400. |
| ALN-6400 (Treatment Group B) | Experimental | Drug: ALN-6400Participants will be administered multiple doses of ALN-6400. |
Who may qualify
- Ages
- 16 to 45 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- 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
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ALN-6400ALN-6400 will be administered subcutaneously (SC). | Drug | ALN-6400 (Treatment Group A), ALN-6400 (Treatment Group B) |
Primary outcome
Frequency of Adverse Events
Time frame: Up to Week 72
5 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Oct 2026.
- Individual participant dataSharing planned: 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Medical Director study director
Alnylam Pharmaceuticals
21 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ALN-6400 in people with Von Willebrand Disease (VWD) and Heavy Menstrual Bleeding (HMB), run by Alnylam Pharmaceuticals at 21 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 26 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 21 sites in United States, United Kingdom, Turkey (Türkiye), Netherlands, Sweden. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Alnylam Clinical Trial Information Line (1-877-ALNYLAM, clinicaltrials@alnylam.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Frequency of Adverse Events (time frame: Up to Week 72). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Oct 2026; AzTrials last refreshed its copy on 31 Oct 2026.