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

ConditionsVon Willebrand Disease (VWD)Heavy Menstrual Bleeding (HMB)
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexFemale
Age16-45
SponsorAlnylam Pharmaceuticals

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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/1.73m² at screening

Is not willing to comply with the contraceptive requirements during the study period

Trial design

Design model

Parallel

Treatments tested in this trial

  • ALN-6400

    Drug

    ALN-6400 will be administered subcutaneously (SC).

Treatment groups

24 Participants
are divided into 2 treatment groups
Group A: ALN-6400 (Treatment Group A)Experimental treatment 1 intervention
Group B: ALN-6400 (Treatment Group B)Experimental treatment 1 intervention

Trial outcomes

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

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Alnylam Pharmaceuticals

Lead sponsor

Source ClinicalTrials.gov
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