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NCT04201418 · ClinicalTrials.gov registry record
A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation
A clinical trial of Polyneuropathy and Hereditary Transthyretin-mediated (ATTRv) Amyloidosis, sponsored by Alnylam Pharmaceuticals.
- Completed
- Registry status
- 67
- Enrollment target
- 20
- Study locations
NCT04201418: Completed study of Polyneuropathy and Hereditary Transthyretin-mediated (ATTRv) Amyloidosis, sponsored by Alnylam Pharmaceuticals.
NCT04201418 is a study of Polyneuropathy and Hereditary Transthyretin-mediated (ATTRv) Amyloidosis that has completed, run by Alnylam Pharmaceuticals. The registered enrollment target is 67 participants. The trial reports 20 study locations across 14 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04201418, a study of Polyneuropathy and Hereditary Transthyretin-mediated (ATTRv) Amyloidosis, has completed, sponsored by Alnylam Pharmaceuticals.
- COMPLETED
- Registry status
- 67 participants
- Enrollment target
- 20
- Study locations
Study Summary
To evaluate the effectiveness of patisiran in patients with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation.
Primary Outcome
PND Scores: Stage 0=No symptoms, Stage 1=Sensory disturbances but preserved walking capability, Stage 2=Impaired walking capacity, but ability to walk without a stick or crutches, Stage 3A/B=Walking with the help of 1 or 2 sticks or crutches, Stage 4=confined to wheel chair or bedridden.
Conditions Studied
Interventions
- DRUG Patisiran
Study Locations (20)
California
- Clinical Trial Site - Los Angeles
- Clinical Trial Site - San Diego
Florida
- Clinical Trial Site - Jacksonville
- Clinical Trial Site - Tampa
Georgia
- Clinical Trial Site - Braselton
- Clinical Trial Site - Macon
Illinois
- Clinical Trial Site - Chicago
- Clinical Trial Site - Chicago
North Carolina
- Clinical Trial Site - Chapel Hill
- Clinical Trial Site - Charlotte
Pennsylvania
- Clinical Trial Site - Allentown
- Clinical Trial Site - Bethlehem
Colorado
- Clinical Trial Site - Aurora
Connecticut
- Clinical Trial Site - Farmington
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 67 participants |
| Start Date | 2019-12-18 |
| Est. Completion | 2022-05-24 |
What the finished NCT04201418 record still lists
NCT04201418 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 67 participants, a relatively small participant target.
The record links to 2 conditions, with Polyneuropathy appearing as the primary indexed condition, and to 1 intervention - of which Patisiran is the first listed.
NCT04201418 names 20 study sites across 14 states, led by California, Florida, Georgia.
Frequently Asked Questions
What is clinical trial NCT04201418 about?
NCT04201418 is a clinical study titled "A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation". To evaluate the effectiveness of patisiran in patients with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation.
What is the current status of trial NCT04201418?
This trial is currently completed. The enrollment target is 67 participants. The study started on 2019-12-18. Estimated completion is 2022-05-24.
What conditions does trial NCT04201418 study?
This clinical trial studies the following conditions: Polyneuropathy, Hereditary Transthyretin-mediated (ATTRv) Amyloidosis.
What interventions are being tested in trial NCT04201418?
The interventions under investigation include: Patisiran (DRUG).
Who is sponsoring clinical trial NCT04201418?
This trial is sponsored by Alnylam Pharmaceuticals, which has 49 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04201418 being conducted?
This trial has 20 study locations across California, Colorado, Connecticut, Florida, Georgia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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