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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)

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ClinicalTrials.gov Identifier: NCT01961921
Recruitment Status : Completed
First Posted : October 14, 2013
Results First Posted : October 30, 2018
Last Update Posted : December 4, 2018
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Study Description
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Brief Summary:
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).

Condition or disease Intervention/treatment Phase
TTR-mediated Amyloidosis Drug: ALN-TTR02 (patisiran) administered by intravenous (IV) infusion Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 27 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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
Study Start Date : October 2013
Actual Primary Completion Date : July 2016
Actual Study Completion Date : August 2016

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Amyloidosis
Drug Information available for: Patisiran

Arms and Interventions
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Arm Intervention/treatment
Experimental: ALN-TTR02 (patisiran) Drug: ALN-TTR02 (patisiran) administered by intravenous (IV) infusion


Outcome Measures
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Primary Outcome Measures :
  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 Outcome Measures :
  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.


Eligibility Criteria
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Ages Eligible for Study:   18 Years to 85 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01961921


Locations
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United States, Massachusetts
Clinical Trial Site
Boston, Massachusetts, United States
Brazil
Clinical Trial Site
Rio de Janeiro, Brazil
France
Clinical Trial Site
Le Kremlin-bicetre, France
Clinical Trial Site
Marseille Cedex, France
Germany
Clinical Trial Site
Munster, Germany
Portugal
Clinical Trial Site
Lisbon, Portugal
Clinical Trial Site
Porto, Portugal
Spain
Clinical Trial Site
Palma De Mallorca, Spain
Sweden
Clinical Trial Site
Umeå, Sweden
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
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Study Director: Jared Gollob, MD Alnylam Pharmaceuticals
  Study Documents (Full-Text)

Documents provided by Alnylam Pharmaceuticals:
Study Protocol  [PDF] June 15, 2015
Statistical Analysis Plan  [PDF] September 14, 2016

More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01961921    
Other Study ID Numbers: ALN-TTR02-003
2013-001644-65 ( EudraCT Number )
First Posted: October 14, 2013    Key Record Dates
Results First Posted: October 30, 2018
Last Update Posted: December 4, 2018
Last Verified: November 2018
Keywords provided by Alnylam Pharmaceuticals:
RNAi therapeutic
Additional relevant MeSH terms:
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Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases