A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation
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
1 other identifier
observational
67
1 country
27
Brief Summary
To evaluate the effectiveness of patisiran in patients with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for all trials
Started Dec 2019
Typical duration for all trials
27 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
December 13, 2019
CompletedFirst Posted
Study publicly available on registry
December 17, 2019
CompletedStudy Start
First participant enrolled
December 18, 2019
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 24, 2022
CompletedStudy Completion
Last participant's last visit for all outcomes
May 24, 2022
CompletedJune 6, 2022
June 1, 2022
2.4 years
December 13, 2019
June 3, 2022
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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
Study Arms (3)
Patisiran Prospective Cohort
Patients who are naive to patisiran at study enrollment with the intention to initiate commercial patisiran therapy.
Patisiran Mixed Cohort
Patients who are currently on commercial patisiran therapy for less than 12 months at study enrollment.
Patisiran Retrospective Cohort
Patients who have been on commercial patisiran therapy for at least 12 months prior to study enrollment, regardless of current treatment status at enrollment.
Interventions
Patisiran-lipid complex injection, for intravenous use
Eligibility Criteria
Participants with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation
You may qualify if:
- 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.
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (27)
Clinical Trial Site
Los Angeles, California, 90095, United States
Clinical Trial Site
San Diego, California, 92103, United States
Clinical Trial Site
Aurora, Colorado, 80045, United States
Clinical Trial Site
Farmington, Connecticut, 06030, United States
Clinical Trial Site
Jacksonville, Florida, 32224, United States
Clinical Trial Site
Tampa, Florida, 33609, United States
Clinical Trial Site
Braselton, Georgia, 30517, United States
Clinical Trial Site
Macon, Georgia, 31201, United States
Clinical Trial Site
Chicago, Illinois, 60611, United States
Clinical Trial Site
Chicago, Illinois, 60612, United States
Clinical Trial Site
Boston, Massachusetts, 02111, United States
Clinical Trial Site
Detroit, Michigan, 48202, United States
Clinical Trial Site
Jackson, Mississippi, 39216, United States
Clinical Trial Site
Kansas City, Missouri, 64111, United States
Clinical Trial Site
Omaha, Nebraska, 68198, United States
Clinical Trial Site
Chapel Hill, North Carolina, 27599, United States
Clinical Trial Site
Charlotte, North Carolina, 28207, United States
Clinical Trial Site
Columbus, Ohio, 43210, United States
Clinical Trial Site
Allentown, Pennsylvania, 18103, United States
Clinical Trial Site
Bethlehem, Pennsylvania, 18015, United States
Clinical Trial Site
Lancaster, Pennsylvania, 17602, United States
Clinical Trial Site
Pittsburgh, Pennsylvania, 15212, United States
Clinical Trial Site
Pittsburgh, Pennsylvania, 15232, United States
Clinical Trial Site
Germantown, Tennessee, 38138, United States
Clinical Trial Site
Austin, Texas, 78756, United States
Clinical Trial Site
San Antonio, Texas, 78229, United States
Clinical Trial Site
Milwaukee, Wisconsin, 53226, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Medical Director
Alnylam Pharmaceuticals
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 13, 2019
First Posted
December 17, 2019
Study Start
December 18, 2019
Primary Completion
May 24, 2022
Study Completion
May 24, 2022
Last Updated
June 6, 2022
Record last verified: 2022-06
Data Sharing
- IPD Sharing
- Will not share