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Completed

NCT Number: NCT02595983

The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver Transplant

The purpose of this study was to evaluate the safety and effectiveness of revusiran (ALN-TTRSC) in adults with transthyretin-mediated amyloidosis (ATTR), whose disease has continued to worsen after liver transplantation. Dosing has been discontinued; patients are being followed-up for safety.

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Key information

Conditions

Transthyretin (TTR)-Mediated Amyloidosis ATTR Amyloidosis Amyloid Neuropathies Amyloid Neuropathies, Familial Amyloidosis Amyloidosis, Familial Congenital, Hereditary, and Neonatal Diseases and Abnormalities Familial Amyloid Neuropathies Familial Amyloidotic Polyneuropathy (FAP) 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 Proteostasis Deficiencies

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Clinical Trial SIte, Paris, France

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Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Diagnosis of FAP (familial amyloidotic polyneuropathy) with documented TTR mutation
  • Received an orthotopic liver transplant ≥12 months before the date of informed consent
  • An increase in polyneuropathy disability (PND) score post-transplant
  • Polyneuropathy Disability score of ≤3b

Exclusion criteria

  • New York Heart Association (NYHA) classification of >2
  • Other known causes of sensorimotor or autonomic neuropathy (eg, autoimmune disease)

Treatment and study plan

Revusiran

Drug

500mg Revusiran by subcutaneous (sc) injection

Other names: ALN-TTRSC

Primary outcomes

  1. Percentage Change From Baseline in Serum TTR at Month 6

    Time frame: Month 6

    A negative percentage change from baseline at Month 6 indicates a reduction in serum TTR level.

Secondary outcomes

  1. Percentage Change From Baseline in Serum TTR Over 18 Months

    Time frame: Weeks 3, 7, 12, 18, 24, 26 (Month 6), 39 (Month 9), 52 (Month 12), 57, 78 (Month 18)

    A negative percentage change from baseline indicates a reduction in serum TTR level.

  2. Change From Baseline in Modified Neurological Impairment Score (mNIS +7) Composite Score Over 18 Months

    Time frame: Baseline, Months 6, 12, 18

    The mNIS+7 is a composite score that measures neurologic impairment which includes the following components: physical exam of lower limbs, upper limbs and cranial nerves to assess motor strength/weakness, electrophysiologic measurement of small and large nerve fiber function, sensory testing and postural blood pressure. The minimum and maximum values are 0 and 304, respectively. A higher score indicates a worse outcome. A negative change from baseline indicates an improvement.

  3. Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN) Questionnaire Score

    Time frame: Baseline, Months 6, 12, 18

    The Norfolk QoL-DN questionnaire is a standardized 35-item patient-reported outcomes measure that is sensitive to the different features of diabetic neuropathy - small fiber, large fiber, and autonomic nerve function. The minimum and maximum values are -4 and 136, respectively. A higher score indicates a worse outcome.

  4. Number of Participants in Each Polyneuropathy Disability (PND) Stage Based on Worst Post-Baseline Score

    Time frame: Baseline, Months 6, 12, 18

    PND Scores: Stage 0: No symptoms, Stage 1: Sensory disturbances but preserved walking capabilities, Stage 2: Impaired walking capacity, but ability to walk without a stick or crutches, Stage 3A/B: Walking with help of 1 or 2 sticks or crutches, Stage 4: confined to wheel chair. For each stage (0-4) at baseline, the number of participants is presented at their worst post-baseline score for each stage (0-4) post-baseline. Worst post-baseline is defined the highest PND classification for a participant recorded after the first dose of study drug.

Sponsors and collaborators

Lead sponsor

Alnylam Pharmaceuticals

Industry

Registry information

Official study title

An Open-label Study to Evaluate the Efficacy and Safety of Revusiran in Patients With Transthyretin-mediated Familial Amyloidotic Polyneuropathy With Disease Progression Post-Orthotopic Liver Transplant

Important dates

Study start
2015
Primary completion
2017
Study completion
2017
First posted
Nov 4, 2015
Registry last updated
Mar 28, 2019

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

View the official ClinicalTrials.gov record (opens in a new tab)

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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