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Search / Trial NCT02939820

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 03, 2025

Approved for marketing

Keywords

Rn Ai Therapeutic Fap Familial Amyloid Polyneuropathies Ttr Transthyretin Amyloidosis

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.

Locations

Patients applied

0 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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