Recruiting
Phase 2

ALN-6400

Sponsor:

Alnylam Pharmaceuticals

Code:

NCT07575308

Conditions

Von Willebrand Disease (VWD)

Heavy Menstrual Bleeding (HMB)

Eligibility Criteria

Sex: Female

Age: 16 - 45

Healthy Volunteers: Not accepted

Interventions

ALN-6400

Study Details

Brief 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

Conditions

Von Willebrand Disease (VWD)

Heavy Menstrual Bleeding (HMB)

Study ID

NCT07575308

Start date

Jul 30, 2026

Status verified date

Sep, 2026

Completion date

Nov 29, 2028

Anticipated

Primary completion date

Jun 30, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: Female

Age: 16 - 45

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

24 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: ALN-6400 (Treatment Group A)

Participants will be administered multiple doses of ALN-6400.

experimental: ALN-6400 (Treatment Group B)

Participants will be administered multiple doses of ALN-6400.

Interventions

ALN-6400

ALN-6400 will be administered subcutaneously (SC).

Primary outcome measure

  • Frequency of Adverse Events [ Time Frame: Up to Week 72 ]

Central Contacts and Locations

Central contacts

Alnylam Clinical Trial Information Line

1-877-ALNYLAMclinicaltrials@alnylam.com

Alnylam Clinical Trial Information Line

1-877-256-9526clinicaltrials@alnylam.com

Locations

Clinical Trial Site

Recruiting

Orange, California, United States, 92868

Clinical Trial Site

Recruiting

San Diego, California, United States, 92123

Clinical Trial Site

Recruiting

San Francisco, California, United States, 94143

Clinical Trial Site

Recruiting

Miami, Florida, United States, 33024

Clinical Trial Site

Recruiting

Lexington, Kentucky, United States, 40536

Clinical Trial Site

Recruiting

Pittsburgh, Pennsylvania, United States, 15213

More Information

Sponsor

Alnylam Pharmaceuticals

Last update posted

Sep 18, 2026

Last verified

Sep, 2026

Keywords

  • siRNA, RNAi therapeutic, Plasminogen, PLG, VWD, HMB

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-10. This information was provided to ClinicalTrials.gov by Alnylam Pharmaceuticals on 2026-09-18. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.