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)
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
2 other identifiers
interventional
27
7 countries
9
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Oct 2013
Typical duration for phase_2
9 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
October 1, 2013
CompletedFirst Submitted
Initial submission to the registry
October 9, 2013
CompletedFirst Posted
Study publicly available on registry
October 14, 2013
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 1, 2016
CompletedStudy Completion
Last participant's last visit for all outcomes
August 1, 2016
CompletedResults Posted
Study results publicly available
October 30, 2018
CompletedApril 19, 2024
November 1, 2018
2.8 years
October 9, 2013
August 30, 2018
April 17, 2024
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
The Number of Participants Experiencing Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation
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.
From Baseline up to 56 days post last dose
Secondary Outcomes (6)
Percentage Change From Baseline in Serum TTR Levels
From Baseline up to 56 days post last dose
Change From Baseline in the Modified Neuropathy Impairment Score +7 (mNIS+7)
Baseline, Month 24
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)
Baseline, Month 24
Change in Gait Speed With 10-meter Walk Test
Baseline, Month 24
Mean Change From Baseline in Hand Grip Strength
Baseline, Month 24
- +1 more secondary outcomes
Study Arms (1)
ALN-TTR02 (patisiran)
EXPERIMENTALInterventions
Eligibility Criteria
You may qualify if:
- Previously received and tolerated ALN-TTR02 (patisiran) in Study ALN-TTR02-002.
- Adequate Karnofsky performance status, liver function, and renal function.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (9)
Clinical Trial Site
Boston, Massachusetts, United States
Clinical Trial Site
Rio de Janeiro, Brazil
Clinical Trial Site
Le Kremlin-Bicêtre, France
Clinical Trial Site
Marseille, France
Clinical Trial Site
Münster, Germany
Clinical Trial Site
Lisbon, Portugal
Clinical Trial Site
Porto, Portugal
Clinical Trial Site
Palma de Mallorca, Spain
Clinical Trial Site
Umeå, Sweden
Related Publications (1)
Coelho T, Adams D, Conceicao I, Waddington-Cruz M, Schmidt HH, Buades J, Campistol J, Berk JL, Polydefkis M, Wang JJ, Chen J, Sweetser MT, Gollob J, Suhr OB. A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis. Orphanet J Rare Dis. 2020 Jul 8;15(1):179. doi: 10.1186/s13023-020-01399-4.
PMID: 32641071DERIVED
MeSH Terms
Interventions
Results Point of Contact
- Title
- Chief Medical Officer
- Organization
- Alnylam Pharmaceuticals Inc
Study Officials
- STUDY DIRECTOR
Jared Gollob, MD
Alnylam Pharmaceuticals
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- LTE60
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 9, 2013
First Posted
October 14, 2013
Study Start
October 1, 2013
Primary Completion
July 1, 2016
Study Completion
August 1, 2016
Last Updated
April 19, 2024
Results First Posted
October 30, 2018
Record last verified: 2018-11
Data Sharing
- IPD Sharing
- Will share
Access to Anonymized individual participant data that support these results is made available 12 months after study completion and not less than 12 months after the product and indication have been approved in the US and/or the EU. Data will be provided contingent upon the approval of a research proposal and the execution of a data sharing agreement. Requests for access to data can be submitted via the website www.vivli.org.