Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)
Launched by ALNYLAM PHARMACEUTICALS · Oct 18, 2016
Trial Information
Current as of June 30, 2025
Approved for marketing
Keywords
ClinConnect Summary
Choosing to participate in an expanded access program is an important personal decision. Talk with your doctor and family members or friends about deciding to join a research study. To learn more about this study, please have your doctor contact the study research staff using the Contacts provided. For general information, see the links provided in More Information.
Gender
ALL
Eligibility criteria
- Inclusion Criteria:
- • Male or female greater than or equal to 18 years of age
- • Have a diagnosis of hATTR
- • Meet Karnofsky performance status and Polyneuropathy Disability (PND) score requirements
- • Have adequate complete blood counts, liver function tests and coagulation tests
- Exclusion Criteria:
- • Participated in an interventional hATTR amyloidosis clinical trial involving RNA interference (RNAi) therapeutics within the last 12 months
- • Are currently eligible to participate in or currently enrolled in an ongoing interventional hATTR amyloidosis clinical trial
- • Have inadequate cardiac function
- • Known primary amyloidosis (AL amyloidosis) or leptomeningeal amyloidosis
- • Have known serious comorbidities or considered unfit for the program by the investigator
- • Prior or planned liver or heart transplantation
About Alnylam Pharmaceuticals
Alnylam Pharmaceuticals is a pioneering biopharmaceutical company focused on the development of innovative therapies based on RNA interference (RNAi) technology. Founded in 2002, Alnylam is dedicated to transforming the treatment landscape for patients with genetically defined diseases by leveraging its proprietary platform to discover and develop novel therapeutics. With a robust pipeline of clinical programs targeting a range of conditions, including rare genetic disorders and prevalent diseases, Alnylam is committed to advancing scientific research and improving patient outcomes through cutting-edge medicine and rigorous clinical trials. The company emphasizes collaboration and transparency in its operations, fostering partnerships within the scientific community to drive innovation and enhance healthcare solutions globally.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
Patients applied
Trial Officials
Medical Director
Study Director
Alnylam Pharmaceuticals
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported
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