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Trial record 1 of 1 for:    ALN-TTR02-012
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A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation

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ClinicalTrials.gov Identifier: NCT04201418
Recruitment Status : Active, not recruiting
First Posted : December 17, 2019
Last Update Posted : September 16, 2021
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Tracking Information
First Submitted Date December 13, 2019
First Posted Date December 17, 2019
Last Update Posted Date September 16, 2021
Actual Study Start Date December 18, 2019
Estimated Primary Completion Date June 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: December 13, 2019)
Percentage of Participants with Stable or Improved Polyneuropathy Disability (PND) Score at 12 Months Relative to Baseline [ Time Frame: Baseline, Month 12 ]
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.
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation
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
Brief Summary To evaluate the effectiveness of patisiran in patients with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation.
Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Participants with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation
Condition
  • Hereditary Transthyretin-mediated (ATTRv) Amyloidosis
  • Polyneuropathy
Intervention Drug: Patisiran
Patisiran-lipid complex injection, for intravenous use
Other Names:
  • ONPATTRO
  • ALN-TTR02
Study Groups/Cohorts
  • Patisiran Prospective Cohort
    Patients who are naive to patisiran at study enrollment with the intention to initiate commercial patisiran therapy.
    Intervention: Drug: Patisiran
  • Patisiran Mixed Cohort
    Patients who are currently on commercial patisiran therapy for less than 12 months at study enrollment.
    Intervention: Drug: Patisiran
  • 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.
    Intervention: Drug: Patisiran
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Active, not recruiting
Actual Enrollment
 (submitted: July 14, 2021)
67
Original Estimated Enrollment
 (submitted: December 13, 2019)
100
Estimated Study Completion Date June 2022
Estimated Primary Completion Date June 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria

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
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT04201418
Other Study ID Numbers ALN-TTR02-012
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party Alnylam Pharmaceuticals
Study Sponsor Alnylam Pharmaceuticals
Collaborators Not Provided
Investigators
Study Director: Medical Director Alnylam Pharmaceuticals
PRS Account Alnylam Pharmaceuticals
Verification Date September 2021