Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)
Primary Purpose
TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial
Status
No longer available
Phase
Locations
Study Type
Expanded Access
Intervention
patisiran (ALN-TTR02)
Sponsored by
About this trial
This is an expanded access trial for TTR-mediated Amyloidosis focused on measuring RNAi therapeutic, FAP, Familial Amyloid Polyneuropathies, TTR, Transthyretin, Amyloidosis
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
Sites / Locations
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT02939820
First Posted
October 18, 2016
Last Updated
July 14, 2022
Sponsor
Alnylam Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT02939820
Brief Title
Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)
Official Title
Expanded Access Protocol of Patisiran for Patients With Hereditary Transthyretin-Mediated Amyloidosis (hATTR Amyloidosis) With Polyneuropathy
Study Type
Expanded Access
2. Study Status
Record Verification Date
July 2022
Overall Recruitment Status
No longer available
Study Start Date
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Primary Completion Date
undefined (undefined)
Study Completion Date
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3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alnylam Pharmaceuticals
4. Oversight
5. Study Description
Brief Summary
The purpose of this study is to provide expanded access of patisiran to patients with hereditary transthyretin-mediated amyloidosis (hATTR).
Detailed Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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
7. Study Design
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
patisiran (ALN-TTR02)
Intervention Description
patisiran (ALN-TTR02) administered by intravenous (IV) infusion
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Alnylam Pharmaceuticals
Official's Role
Study Director
12. IPD Sharing Statement
Citations:
PubMed Identifier
31215818
Citation
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.
Results Reference
derived
Links:
URL
https://ghr.nlm.nih.gov/condition/transthyretin-amyloidosis
Description
https://ghr.nlm.nih.gov/condition/transthyretin-amyloidosis
URL
https://www.nlm.nih.gov/medlineplus/amyloidosis.html
Description
https://www.nlm.nih.gov/medlineplus/amyloidosis.html
Learn more about this trial
Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)
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