Heavy Menstrual Bleeding (HMB), Von Willebrand Disease (VWD)
Conditions
Keywords
siRNA, RNAi therapeutic, Plasminogen, PLG, VWD, HMB
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
Interventions
DRUGALN-6400
ALN-6400 will be administered subcutaneously (SC).
Sponsors
Alnylam Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
FEMALE
Age
16 Years to 45 Years
Healthy volunteers
No
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 /
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Frequency of Adverse Events | Up to Week 72 |
Secondary
| Measure | Time frame |
|---|---|
| Change from Baseline in Plasminogen (PLG) Plasma Activity Levels | Screening and up to Week 72 postdose |
| Change from Baseline in PLG Plasma Protein Levels | Screening and up to Week 72 postdose |
| Change from Baseline in Menstrual Blood Loss Via Pictorial Blood Assessment Chart (PBAC) | Screening and up to Week 72 postdose |
| Patient-reported Outcome (PRO) assessed by Patient Global Impression of Change (PGI-C) Score | Week 4 up to Week 72 |
| Change from Baseline in PRO assessed by Patient Global Impression of Severity (PGI-S) Score | Baseline up to Week 72 |
Countries
United States
Contacts
CONTACTAlnylam Clinical Trial Information Line
STUDY_DIRECTORMedical Director
Alnylam Pharmaceuticals
Outcome results
None listed