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

Completed

• Conditions: Hereditary Transthyretin-mediated (ATTRv) Amyloidosis; Polyneuropathy
• Interventions: Drug: Patisiran

• Registration Number: NCT04201418
• Lead Sponsor: Alnylam Pharmaceuticals

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 available

Study Timeline

• Study First Submitted: 2019-12-13
• Study First Submitted QC: 2019-12-13
• Study First Posted: 2019-12-17
• Study Start: 2019-12-18
• Primary Completion: 2022-05-24
• Study Completion: 2022-05-24
• Status Verified: 2022-06
• Last Update Submitted: 2022-06-03
• Last Update Posted: 2022-06-06

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 67

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

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Patisiran Prospective Cohort	Patisiran	Patients who are naive to patisiran at study enrollment with the intention to initiate commercial patisiran therapy.
Patisiran Mixed Cohort	Patisiran	Patients who are currently on commercial patisiran therapy for less than 12 months at study enrollment.
Patisiran Retrospective Cohort	Patisiran	Patients who have been on commercial patisiran therapy for at least 12 months prior to study enrollment, regardless of current treatment status at enrollment.

Primary Outcome Measures

Name	Time	Method
Percentage of Participants with Stable or Improved Polyneuropathy Disability (PND) Score at 12 Months Relative to Baseline	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.

Trial Locations

Locations (1)

Clinical Trial Site; 🇺🇸 Milwaukee, Wisconsin, United States