Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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EUCTR |
Last refreshed on:
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9 January 2023 |
Main ID: |
EUCTR2014-003877-40-SE |
Date of registration:
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01/04/2015 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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Study to Evaluate the Long-term Safety and Efficacy of Patisiran (ALN-TTR02) in Patients with Familial Amyloidotic Polyneuropathy (FAP)
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Scientific title:
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A Multicenter, Open-Label, Extension Study to Evaluate the Long-term Safety and Efficacy of Patisiran in Patients with Familial Amyloidotic Polyneuropathy Who Have Completed a Prior Clinical Study with Patisiran |
Date of first enrolment:
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29/05/2015 |
Target sample size:
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228 |
Recruitment status: |
Not Recruiting |
URL:
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https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2014-003877-40 |
Study type:
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Interventional clinical trial of medicinal product |
Study design:
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Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 1
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Phase:
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Human pharmacology (Phase I): no
Therapeutic exploratory (Phase II): no
Therapeutic confirmatory - (Phase III): yes
Therapeutic use (Phase IV): no
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Countries of recruitment
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Argentina
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Australia
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Brazil
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Bulgaria
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Canada
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Cyprus
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France
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Germany
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Italy
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Japan
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Korea, Republic of
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Malaysia
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Mexico
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Netherlands
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Portugal
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Spain
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Sweden
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Switzerland
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Taiwan
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Turkey
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United Kingdom
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United States
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Contacts
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Name:
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Clinical Trials Information Line
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Address:
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300 Third Street
02142
Cambridge MA
United States |
Telephone:
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0018772569526 |
Email:
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clinicaltrials@alnylam.com |
Affiliation:
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Alnylam Pharmaceuticals, Inc. |
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Name:
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Clinical Trials Information Line
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Address:
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300 Third Street
02142
Cambridge MA
United States |
Telephone:
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0018772569526 |
Email:
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clinicaltrials@alnylam.com |
Affiliation:
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Alnylam Pharmaceuticals, Inc. |
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Key inclusion & exclusion criteria
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Inclusion criteria: 1. Have completed a patisiran study
2. Adequate liver function and renal function
Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 114 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 114
Exclusion criteria: 1. Pregnant or nursing
2. Have uncontrolled clinically significant cardiac arrhythmia or unstable angina
Age minimum:
Age maximum:
Gender:
Female: yes Male: yes
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Health Condition(s) or Problem(s) studied
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Transthyretin mediated amyloidosis (ATTR) MedDRA version: 20.0
Level: PT
Classification code 10007509
Term: Cardiac amyloidosis
System Organ Class: 10007541 - Cardiac disorders
MedDRA version: 20.0
Level: PT
Classification code 10019889
Term: Hereditary neuropathic amyloidosis
System Organ Class: 10010331 - Congenital, familial and genetic disorders
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Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
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Intervention(s)
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Trade Name: Onpattro Product Name: Patisiran Product Code: ALN-TTR02 Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: PATISIRAN Current Sponsor code: Patisiran Other descriptive name: ALN-TTR02 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 2-
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Primary Outcome(s)
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Timepoint(s) of evaluation of this end point: The review of AEs will be done at each visit from Day 0 through 28 days post last dose. Efficacy assessments will be performed at Day 0, annually and 28 days post last dose
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Primary end point(s): To assess the safety and efficacy of long-term dosing with patisiran
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Secondary Objective: None
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Main Objective: To assess the safety and efficacy of long-term dosing with patisiran in patients with transthyretin-mediated amyloidosis (ATTR) amyloidosis
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Secondary Outcome(s)
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Timepoint(s) of evaluation of this end point: None
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Secondary end point(s): None
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Secondary ID(s)
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2014-003877-40-ES
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ALN-TTR02-006
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Source(s) of Monetary Support
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Alnylam Pharmaceuticals, Inc
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Ethics review
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Status: Approved
Approval date: 05/05/2015
Contact:
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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