Authorised Therapeutic exploratory (Phase II) Von Willebrand Disease (VWD) and Heavy Menstrual Bleeding (HMB)

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

EU CTIS ID: 2025-524967-19-00

What this study is testing

To evaluate the safety and tolerability of multiple doses of ALN-6400 in female patients with VWD and HMB

  • Therapeutic exploratory (Phase II)

A plain-language read of the study's public EU CTIS listing. The study team confirms the details.

Who can take part

You may be able to join if

  • 1. Females age 18 to 45 years, inclusive, at the time of initial informed consent. Or age 18 to 34 years, inclusive, at the time of initial informed consent if receiving COCs (≤35 µg estrogen per day, ie, low dose, as described in Section 5.9.1).
  • 2. Historical diagnosis of VWD of the following types, with or without inhibitors: a. Type 1: Type 1 with historical VWF:Act* <30 IU/dL; OR Type 1 with historical VWF:Act* ≥30 and <40 IU/dL and International Society on Thrombosis and Haemostasis Bleeding Assessment Tool (ISTH-BAT) [Rodeghiero 2010] score >5 at screening. *VWF:Act may be assessed as platelet-dependent VWF activity (VWF:platelet glycoprotein 1b mutant [GP1bM] binding or VWF:platelet glycoprotein 1b recombinant [VWF:GP1bR] binding) or via the VWF ristocetin cofactor assay. VWF:Act may be reported in %. b. Type 2 (any subtype) c. Type 3 d. Platelet-type
  • 3. HMB with 2 cycles with pictorial blood assessment chart (PBAC) scores >100 during screening.
  • 4. Stable standard-of-care treatment for HMB, including no therapy, during screening and no planned changes in therapy during the Double-blind Period, except for rescue treatments (Section 5.7.1) and as noted below: a. TXA and other antifibrinolytic treatments must be discontinued prior to Day 1. b. Scheduled factor for menstrual bleeding prophylaxis must be discontinued prior to Day 15. Note that “factor” in this protocol refers to any VWF-containing product or any Factor VIII-containing product.
  • 5. Menses every 21 to 35 days, as reported by the patient, over the 6 months before screening.
  • 6. If receiving COCs, body mass index <30 kg/m2.

You likely can't join if

  • 1. Has any of the following laboratory parameter assessments at screening: a. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2× upper limit of normal (ULN). b. Total bilirubin >1.5×ULN. Patients with elevated total bilirubin that is secondary to documented Gilbert’s syndrome are eligible if the total bilirubin is <2×ULN. c. International normalized ratio (INR) >1.1.
  • 10. Use of etonogestrel implant within 24 weeks of screening or planned use during the study.
  • 11. Taking an anticoagulant (eg, warfarin, apixaban, rivaroxaban, dabigatran) during screening or planned use during the study.
  • 12. Taking antiplatelet medications including aspirin (other nonsteroidal anti-inflammatory drugs [NSAIDs] are permitted) during screening or planned use during the study.
  • 13. Taking routine factor prophylaxis (ie, ≥1 infusion of factor per week over 12 weeks) within 4 weeks of screening or planned use for routine factor prophylaxis during the study. Scheduled factor for menstrual bleeding prophylaxis is permitted, but must be discontinued prior to Day 15, as indicated in Inclusion Criterion 4b.
  • 14. Unable or unwilling to abstain from antifibrinolytic agents, including TXA and aminocaproic acid, during the Double-blind and Extension Periods.
See the full eligibility criteria
Who can join
  • 1. Females age 18 to 45 years, inclusive, at the time of initial informed consent. Or age 18 to 34 years, inclusive, at the time of initial informed consent if receiving COCs (≤35 µg estrogen per day, ie, low dose, as described in Section 5.9.1).
  • 2. Historical diagnosis of VWD of the following types, with or without inhibitors: a. Type 1: Type 1 with historical VWF:Act* <30 IU/dL; OR Type 1 with historical VWF:Act* ≥30 and <40 IU/dL and International Society on Thrombosis and Haemostasis Bleeding Assessment Tool (ISTH-BAT) [Rodeghiero 2010] score >5 at screening. *VWF:Act may be assessed as platelet-dependent VWF activity (VWF:platelet glycoprotein 1b mutant [GP1bM] binding or VWF:platelet glycoprotein 1b recombinant [VWF:GP1bR] binding) or via the VWF ristocetin cofactor assay. VWF:Act may be reported in %. b. Type 2 (any subtype) c. Type 3 d. Platelet-type
  • 3. HMB with 2 cycles with pictorial blood assessment chart (PBAC) scores >100 during screening.
  • 4. Stable standard-of-care treatment for HMB, including no therapy, during screening and no planned changes in therapy during the Double-blind Period, except for rescue treatments (Section 5.7.1) and as noted below: a. TXA and other antifibrinolytic treatments must be discontinued prior to Day 1. b. Scheduled factor for menstrual bleeding prophylaxis must be discontinued prior to Day 15. Note that “factor” in this protocol refers to any VWF-containing product or any Factor VIII-containing product.
  • 5. Menses every 21 to 35 days, as reported by the patient, over the 6 months before screening.
  • 6. If receiving COCs, body mass index <30 kg/m2.
  • 7. Patient is able to understand and is willing and able to comply with the study requirements, including completing the daily menstrual bleed diary with PBAC assessments (including 2 full cycles during screening), and to provide written informed consent.
What rules you out
  • 1. Has any of the following laboratory parameter assessments at screening: a. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2× upper limit of normal (ULN). b. Total bilirubin >1.5×ULN. Patients with elevated total bilirubin that is secondary to documented Gilbert’s syndrome are eligible if the total bilirubin is <2×ULN. c. International normalized ratio (INR) >1.1.
  • 10. Use of etonogestrel implant within 24 weeks of screening or planned use during the study.
  • 11. Taking an anticoagulant (eg, warfarin, apixaban, rivaroxaban, dabigatran) during screening or planned use during the study.
  • 12. Taking antiplatelet medications including aspirin (other nonsteroidal anti-inflammatory drugs [NSAIDs] are permitted) during screening or planned use during the study.
  • 13. Taking routine factor prophylaxis (ie, ≥1 infusion of factor per week over 12 weeks) within 4 weeks of screening or planned use for routine factor prophylaxis during the study. Scheduled factor for menstrual bleeding prophylaxis is permitted, but must be discontinued prior to Day 15, as indicated in Inclusion Criterion 4b.
  • 14. Unable or unwilling to abstain from antifibrinolytic agents, including TXA and aminocaproic acid, during the Double-blind and Extension Periods.
  • 15. Known current gynecological conditions causing abnormal uterine bleeding (including infection, fibroids, endometriosis, polycystic ovary syndrome, or dysplasia).
  • 16. Any bleeding disorder other than VWD.
  • 17. Acquired VWD.
  • 18. Untreated hypothyroidism, defined as elevated thyroid-stimulating hormone within 3 months of screening in patients with a history of hypothyroidism.
  • 19. Uncontrolled hypertension at screening in the opinion of the Investigator.
  • 2. Has an estimated glomerular filtration (eGFR) of <30 mL/min/1.73m2 at screening (calculation will be based on the Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI] equation
  • 20. Any unresolved acute bleeding episode which, in the opinion of the Investigator, would interfere with study compliance or data interpretation; or, in the opinion of the Investigator, taking part in the study would jeopardize the safety of the patient.
  • 21. Has known hereditary thrombophilia, thrombotic thrombocytopenic purpura, antiphospholipid antibody syndrome, or any other clotting disorder.
  • 22. Any history of thrombosis/thromboembolism confirmed by ultrasound or other imaging modalities.
  • 23. Presence of ligneous lesion(s) on physical examination; or a lesion that, in the opinion of the Investigator, might be confused with a ligneous lesion.
  • 24. Planned major surgery during the study or any planned procedure for HMB (eg, endometrial ablation).
  • 25. Has undergone liver transplantation or is anticipated to be on an active liver transplantation waiting list during the study treatment period.
  • 26. Has other medical conditions or comorbidities which, in the opinion of the Investigator, would interfere with study compliance or data interpretation; or, in the opinion of the Investigator, taking part in the study would jeopardize the safety of the patient.
  • 27. History of multiple drug allergies or history of allergic reaction to any component of or excipient in the study drug.
  • 28. History of intolerance to SC injection(s).
  • 29. Is not willing to comply with the contraceptive requirements during the study period, as described in Section 5.9.1.
  • 3. Hemoglobin <8 g/dL at screening.
  • 30. Female patient is pregnant, planning a pregnancy or breastfeeding.
  • 31. Unwilling or unable to limit alcohol consumption throughout the course of the study. Alcohol intake of >2 units/day is excluded during the study (unit: 1 glass of wine [approximately 125 mL] = 1 measure of spirits [approximately 1 fluid ounce] = ½ pint of beer [approximately 284 mL]).
  • 32. History of alcohol use disorder, within the last 12 months before screening, in the opinion of the Investigator.
  • 33. History of substance use disorder that in the opinion of the Investigator would interfere with compliance with study procedures or follow-up visits.
  • 34. If receiving COCs, tobacco use within the last 12 months before screening per medical history.
  • 4. Platelet count <100,000/μL (except for patients with VWD Type 2B or platelet-type VWD, for whom the cutoff is <50,000/μL) at screening.
  • 5. Taking gonadotropin-releasing hormone agonists or systemic estrogen-containing hormone replacement therapy within 12 weeks of screening or planned use during the study. a. COCs (≤35 µg estrogen per day, ie, low dose) are permitted if the patient has been receiving this for at least 3 months prior to screening and has been treated with a COC (with the same or higher estrogen dose) for at least 12 months at any time prior to screening. b. For patients age ≤19 years at the time of initial informed consent, COCs (≤35 µg estrogen per day, ie, low dose) are permitted if the patient has been receiving this for at least 3 months prior to screening and has been treated with a COC (with the same or higher estrogen dose) for at least 6 months at any time prior to screening. c. Also refer to inclusion Criteria 1 and 7 and exclusion Criteria 22 and 34.
  • 6. Received an investigational agent within the last 30 days or 5 half-lives, whichever is longer, before the start of screening, or are in follow-up of another clinical study before study enrollment. Any agent that has received health agency authorization (including for emergency use) by local or regional regulatory authorities is not considered investigational.
  • 7. Currently taking, taken within 24 weeks before randomization, or anticipated to receive an RNAi therapeutic or antisense oligonucleotide (approved or investigational) other than ALN-6400.
  • 8. Any planned change to permitted medications for the treatment of HMB that could impact menstruation during the study.
  • 9. New placement of any hormonal or nonhormonal IUD or hormonal implants within 24 weeks of screening.

The study team makes the final eligibility decision.

Where it's taking place

  • United States
  • United Kingdom
  • Turkey

Questions & answers

Do participants get paid in this trial?

This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.

Is it free to join, and do I need insurance?

Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.

How long does this study last?

The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.

Who can join this trial?

This study is enrolling female, 18-64 years. The study team makes the final eligibility decision.

Where is this trial taking place?

Study sites include United States; United Kingdom; Turkey. Enter your location above to see the nearest site and check your eligibility.

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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.