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NCT04201418 Clinical Trial

A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation

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

Clinical Trial Details — Status: Completed

Administrative data
NCT number NCT04201418
Other study ID # ALN-TTR02-012
Secondary ID
Status Completed
Phase
First received
Last updated
Start date December 18, 2019
Est. completion date May 24, 2022

Study information
Verified date June 2022
Source Alnylam Pharmaceuticals
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

To evaluate the effectiveness of patisiran in patients with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation.

Recruitment information / eligibility
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

Study Design

Related Conditions & MeSH terms

Intervention

Drug:
• Patisiran
Patisiran-lipid complex injection, for intravenous use

Locations
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

Sponsors (1)
Lead Sponsor Collaborator
Alnylam Pharmaceuticals

Country where clinical trial is conducted

United States, 

Outcome
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
See also
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