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| ID | Type | Description | Link |
|---|---|---|---|
| 2025-524967-19-00 | EU Trial (CTIS) Number |
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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
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ALN-6400 (Treatment Group A) | Experimental | Participants will be administered multiple doses of ALN-6400. |
|
| ALN-6400 (Treatment Group B) | Experimental | Participants will be administered multiple doses of ALN-6400. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ALN-6400 | Drug | ALN-6400 will be administered subcutaneously (SC). |
|
| Measure | Description | Time Frame |
|---|---|---|
| Frequency of Adverse Events | Up to Week 72 |
| Measure | Description | 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 | |
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Inclusion Criteria -
Exclusion Criteria -
Note: other protocol defined inclusion / exclusion criteria apply
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Alnylam Clinical Trial Information Line | Contact | 1-877-ALNYLAM | clinicaltrials@alnylam.com | |
| Alnylam Clinical Trial Information Line | Contact | 1-877-256-9526 | clinicaltrials@alnylam.com |
| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Alnylam Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Trial Site | Miami | Florida | 33024 | United States |
Phase 2-4:
Access to Anonymized individual participant data that support these results is made available 12 months after study completion and not less than 12 months after the product and indication have been approved in the United States (US) and/or the European Union (EU).
Data will be provided contingent upon the approval of a research proposal and the execution of a data sharing agreement. Requests for access to data can be submitted via the website www.vivli.org.
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| ID | Term |
|---|---|
| D014842 | von Willebrand Diseases |
| D008595 | Menorrhagia |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| 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 |
| D020147 | Coagulation Protein Disorders |
| D001791 | Blood Platelet Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D014592 | Uterine Hemorrhage |
| D014591 | Uterine Diseases |
| D005831 | Genital Diseases, Female |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D000091662 | Genital Diseases |
| D006470 | Hemorrhage |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D008599 | Menstruation Disturbances |