Polyneuropathy Clinical Trial
Official title:
A Phase 4 Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of Hereditary Transthyretin-Mediated (ATTRv) Amyloidosis With a V122I or T60A Mutation
Verified date | June 2022 |
Source | Alnylam Pharmaceuticals |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
To evaluate the effectiveness of patisiran in patients with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation.
Status | Completed |
Enrollment | 67 |
Est. completion date | May 24, 2022 |
Est. primary completion date | May 24, 2022 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility | Inclusion Criteria: - Diagnosed with ATTRv amyloidosis with polyneuropathy, with a documented V122I or T60A mutation - PND score of I-IIIB at baseline. - Exposure to commercial patisiran in one of the 3 cohorts: - Prospective Cohort: Naive to patisiran treatment at the time of enrollment with intention to initiate treatment with patisiran. - Mixed cohort: Currently on commercial patisiran therapy for less than 12 months at study enrollment. - Retrospective cohort: Exposed to commercial patisiran treatment for at least 12 months prior to study enrollment, regardless of current treatment status at enrollment. Exclusion Criteria: - New York Heart Association (NYHA) heart failure classification =3 - Karnofsky Performance Status (KPS) <60% - Unstable congestive heart failure (CHF) - Known primary amyloidosis (AL) or leptomeningeal amyloidosis - Prior major organ transplant - Previously received patisiran - Previous treatment with a TTR silencing therapy |
Country | Name | City | State |
---|---|---|---|
United States | Clinical Trial Site | Allentown | Pennsylvania |
United States | Clinical Trial Site | Aurora | Colorado |
United States | Clinical Trial Site | Austin | Texas |
United States | Clinical Trial Site | Bethlehem | Pennsylvania |
United States | Clinical Trial Site | Boston | Massachusetts |
United States | Clinical Trial Site | Braselton | Georgia |
United States | Clinical Trial Site | Chapel Hill | North Carolina |
United States | Clinical Trial Site | Charlotte | North Carolina |
United States | Clinical Trial Site | Chicago | Illinois |
United States | Clinical Trial Site | Chicago | Illinois |
United States | Clinical Trial Site | Columbus | Ohio |
United States | Clinical Trial Site | Detroit | Michigan |
United States | Clinical Trial Site | Farmington | Connecticut |
United States | Clinical Trial Site | Germantown | Tennessee |
United States | Clinical Trial Site | Jackson | Mississippi |
United States | Clinical Trial Site | Jacksonville | Florida |
United States | Clinical Trial Site | Kansas City | Missouri |
United States | Clinical Trial Site | Lancaster | Pennsylvania |
United States | Clinical Trial Site | Los Angeles | California |
United States | Clinical Trial Site | Macon | Georgia |
United States | Clinical Trial Site | Milwaukee | Wisconsin |
United States | Clinical Trial Site | Omaha | Nebraska |
United States | Clinical Trial Site | Pittsburgh | Pennsylvania |
United States | Clinical Trial Site | Pittsburgh | Pennsylvania |
United States | Clinical Trial Site | San Antonio | Texas |
United States | Clinical Trial Site | San Diego | California |
United States | Clinical Trial Site | Tampa | Florida |
Lead Sponsor | Collaborator |
---|---|
Alnylam Pharmaceuticals |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Percentage of Participants with Stable or Improved Polyneuropathy Disability (PND) Score at 12 Months Relative to Baseline | 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. | Baseline, Month 12 |
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