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 Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
24
ALN-6400 will be administered subcutaneously (SC).
Clinical Trial Site
Orange, California, United States
RECRUITINGClinical Trial Site
San Diego, California, United States
RECRUITINGFrequency of Adverse Events
Time frame: Up to Week 72
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
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Clinical Trial Site
San Francisco, California, United States
RECRUITINGClinical Trial Site
Miami, Florida, United States
RECRUITINGClinical Trial Site
Lexington, Kentucky, United States
RECRUITINGClinical Trial Site
Pittsburgh, Pennsylvania, United States
RECRUITINGClinical Trial Site
Nijmegen, Netherlands
NOT_YET_RECRUITINGClinical Trial Site
Stockholm, Sweden
NOT_YET_RECRUITINGClinical Trial Site
Bradford, United Kingdom
NOT_YET_RECRUITINGClinical Trial Site
Oxford, United Kingdom
NOT_YET_RECRUITING...and 1 more locations