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Completed

NCT Number: NCT03862807

Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant

The purpose of this study is to evaluate the efficacy, safety and pharmacokinetics of patisiran in participants with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) with disease progression after liver transplant.

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Key information

Conditions

Amyloidosis, Familial Amyloidosis Amyloidosis, Hereditary, Transthyretin-Related Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases Proteostasis Deficiencies Transthyretin Amyloidosis

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Clinical Trial Site, Créteil, 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

  • Received liver transplant for treatment of hATTR amyloidosis ≥12 months before study start
  • Has increase in polyneuropathy disability (PND) score after liver transplant
  • Has received stable immunosuppressive regimen with ≤10 mg/day of prednisone for at least 3 months before study start
  • Has Karnofsky Performance Status (KPS) of ≥70%
  • Has vitamin A level greater than or equal to lower limit of normal

Exclusion criteria

  • Has previously received inotersen or patisiran
  • Has clinically significant liver function test abnormalities
  • Has known portal hypertension with ascites
  • Has estimated glomerular filtration rate (eGFR) ≤30 mL/min/1.73 m^2
  • Has known leptomeningeal amyloidosis
  • Has infection with hepatitis B, hepatitis C or human immunodeficiency virus (HIV)
  • Has New York Heart Association heart failure classification of >2
  • Is wheelchair bound or bedridden
  • Has received organ transplants other than liver transplant
  • Will be using another tetramer stabilizer during the study

Treatment and study plan

Patisiran

Drug

Patisiran was administered via IV infusion.

Other names: ALN-TTR02, ONPATTRO

Primary outcomes

  1. Average of Month 6 and Month 12 Percentage Reduction From Baseline in Serum Transthyretin (TTR)

    Time frame: Baseline, Months 6 and 12

    Serum TTR was assessed using enzyme linked immunosorbent assay (ELISA). The average of the percentage reduction in serum TTR observed at Month 6 and at Month 12 is first calculated for each patient and then the median (95% CI) of these averaged values is summarized for the Safety Analysis Set.

Secondary outcomes

  1. Change From Baseline in the Neuropathy Impairment Score (NIS) at Month 12

    Time frame: Baseline, Month 12

    The NIS is a composite neurologic impairment score that assesses motor weakness (NIS-W), sensation (NIS-S) and reflexes (NIS-R) by physical exam. The minimum and maximum values are 0 and 244, respectively. A higher score indicates a worse outcome.

  2. Change From Baseline in Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Score at Month 12

    Time frame: Baseline, Month 12

    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.

  3. Change From Baseline in the Rasch-Built Overall Disability Scale (R-ODS) at Month 12

    Time frame: Baseline, Month 12

    The R-ODS is comprised of a 24-item linearly weighted scale that specifically captures activity and social participation limitations. The minimum and maximum values are 0 and 48, respectively. A higher score indicates a better outcome.

  4. Change From Baseline in the Composite Autonomic Symptom Score (COMPASS-31) at Month 12

    Time frame: Baseline, Month 12

    The COMPASS-31 questionnaire is a measure of autonomic neuropathy symptoms. The questions evaluate 6 autonomic domains (orthostatic intolerance, vasomotor, secretomotor, gastrointestinal, bladder, and pupillomotor). The minimum and maximum values are 0 and 100, respectively. A higher score indicates a worse outcome.

  5. Change From Baseline in the Modified Body Mass Index (mBMI) at Month 12

    Time frame: Baseline, Month 12

    Nutritional status of participants was evaluated using the mBMI, calculated as BMI (kg/m^2) multiplied by albumin (g/L). An increase from baseline in mBMI suggests improvement, and a decrease from baseline suggests worsening.

  6. Percentage of Participants With Adverse Events

    Time frame: From baseline to end of study at Month 13

    An AE is any untoward medical occurrence in a participant or clinical investigational patient administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment.

Sponsors and collaborators

Lead sponsor

Alnylam Pharmaceuticals

Industry

Registry information

Official study title

An Open-label Study to Evaluate Safety, Efficacy and Pharmacokinetics (PK) of Patisiran-LNP in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) With Disease Progression Post-Orthotopic Liver Transplant

Important dates

Study start
2019
Primary completion
2020
Study completion
2020
First posted
Mar 5, 2019
Registry last updated
Apr 22, 2024

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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