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Completed

NCT Number: NCT01961921

The Study of ALN-TTR02 (Patisiran) for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran)

The purpose of this study is to evaluate the safety and tolerability of long-term dosing with ALN-TTR02 (patisiran) in patients with transthyretin (TTR) mediated amyloidosis (ATTR).

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Key information

Conditions

TTR-mediated Amyloidosis

Age range

18 year–85 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Clinical Trial Site, Rio de Janeiro, Brazil

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Previously received and tolerated ALN-TTR02 (patisiran) in Study ALN-TTR02-002.
  • Adequate Karnofsky performance status, liver function, and renal function.

Exclusion criteria

  • Pregnant or nursing.
  • Has had a liver transplant.
  • Has a New York Heart Association heart failure classification >2.
  • Has unstable angina.
  • Has uncontrolled clinically significant cardiac arrhythmia.

Treatment and study plan

ALN-TTR02 (patisiran) administered by intravenous (IV) infusion

Drug

Primary outcomes

  1. The Number of Participants Experiencing Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation

    Time frame: From Baseline up to 56 days post last dose

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

Secondary outcomes

  1. Percentage Change From Baseline in Serum TTR Levels

    Time frame: From Baseline up to 56 days post last dose

    TTR levels, measured using the enzyme-linked immunosorbent assay (ELISA) method.

  2. Change From Baseline in the Modified Neuropathy Impairment Score +7 (mNIS+7)

    Time frame: Baseline, Month 24

    The mNIS+7 assessment is a composite measure of neurologic impairment that provides a comprehensive measure of large and small fiber function that encompasses the totality of the motor, sensory, and autonomic deficits seen in hereditary transthyretin-mediated amyloidosis (hATTR) patients with polyneuropathy. The minimum and maximum values are 0 and 304, respectively. A higher score indicates a worse outcome.

  3. Change From Baseline in Quality of Life and Disability as Assessed by the EuroQoL (Quality of Life)-5 Dimensions (EQ-5D), EuroQoL Visual Analog Scale (EQ-VAS) Questionnaires and Rasch-built Overall Disability Scale (R-ODS)

    Time frame: Baseline, Month 24

    The overall EQ-5D is measured on a scale from 0 to 1, with 0 being worst and 1 best. The EQ-VAS is measured on a scale of 0-100, with 0 being the worst and 100 the best. The R-ODS captures activity and social participation limitations in patients. The R-ODS score ranges from 0 (most severe activity and social participation limitations) to 100 (no activity and social participation limitations).

  4. Change in Gait Speed With 10-meter Walk Test

    Time frame: Baseline, Month 24

    The 10-meter walk test measures the time (in seconds) that it takes a patient to walk 10 meters.

  5. Mean Change From Baseline in Hand Grip Strength

    Time frame: Baseline, Month 24

    The mean (SEM) hand grip strength change from baseline at 24 months between patients who used patisiran with or without a concomitant TTR stabilizer.

  6. Change From Baseline in Nutritional Status (Modified Body Mass Index, mBMI)

    Time frame: Baseline, Month 24

    Nutritional status of patients 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.

Sponsors and collaborators

Lead sponsor

Alnylam Pharmaceuticals

Industry

Registry information

Official study title

A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Safety, Clinical Activity, and Pharmacokinetics of ALN-TTR02 in Patients With Familial Amyloidotic Polyneuropathy Who Have Previously Received ALN-TTR02

Important dates

Study start
2013
Primary completion
2016
Study completion
2016
First posted
Oct 14, 2013
Registry last updated
Apr 19, 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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