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Completed

NCT Number: NCT04201418

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

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

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Key information

Conditions

Hereditary Transthyretin-mediated (ATTRv) Amyloidosis Amyloid Neuropathies Amyloid Neuropathies, Familial Amyloidosis Amyloidosis, Familial Amyloidosis, Hereditary, Transthyretin-Related Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Nutritional and Metabolic Diseases Peripheral Nervous System Diseases Polyneuropathies Polyneuropathy Proteostasis Deficiencies

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Clinical Trial Site, Los Angeles, California, United States

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Patisiran

Drug

Patisiran-lipid complex injection, for intravenous use

Other names: ONPATTRO, ALN-TTR02

Primary outcomes

  1. 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.

Sponsors and collaborators

Lead sponsor

Alnylam Pharmaceuticals

Industry

Registry information

Official study 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

Important dates

Study start
2019
Primary completion
2022
Study completion
2022
First posted
Dec 17, 2019
Registry last updated
Jun 6, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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