Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)

NCT02939820 | Unknown

• 1 other identifier: ALN-TTR02-007
• Study Type: expanded_access
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

The purpose of this study is to provide expanded access of patisiran to patients with hereditary transthyretin-mediated amyloidosis (hATTR).

Conditions

TTR-mediated Amyloidosis; Amyloidosis, Hereditary; Amyloid Neuropathies, Familial; Familial Amyloid Polyneuropathies; Amyloid Neuropathies; Amyloidosis, Hereditary, Transthyretin-Related

Keywords

RNAi therapeutic; FAP; Familial Amyloid Polyneuropathies; TTR; Transthyretin; Amyloidosis

Interventions

patisiran (ALN-TTR02) DRUG

patisiran (ALN-TTR02) administered by intravenous (IV) infusion

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Alnylam Pharmaceuticals: lead

Related Publications (1)

• Miah KM, Hyde SC, Gill DR. Emerging gene therapies for cystic fibrosis. Expert Rev Respir Med. 2019 Aug;13(8):709-725. doi: 10.1080/17476348.2019.1634547. Epub 2019 Jun 27.

  PMID: 31215818 DERIVED

Related Links

• https://ghr.nlm.nih.gov/condition/transthyretin-amyloidosis
• https://www.nlm.nih.gov/medlineplus/amyloidosis.html

MeSH Terms

Conditions

Amyloidosis, Familial; Amyloid Neuropathies, Familial; Amyloid Neuropathies; Amyloidosis, Hereditary, Transthyretin-Related; Amyloidosis

Interventions

patisiran

Condition Hierarchy (Ancestors)

Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases; Proteostasis Deficiencies; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Peripheral Nervous System Diseases; Neuromuscular Diseases

Study Officials

• Medical Director

  Alnylam Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 18, 2016
• First Posted: October 20, 2016
• Last Updated: May 20, 2024

Record last verified: 2024-05