search
Back to results

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

Primary Purpose

Amyloidosis

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Patisiran (ALN-TTR02)
Sponsored by
Alnylam Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Amyloidosis focused on measuring RNAi therapeutic, FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis

Eligibility Criteria

18 Years - 85 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Have completed a patisiran study (i.e., completed the last efficacy visit in the parent study) and, in the opinion of the investigator, tolerated study drug
  • Be willing and able to comply with the protocol-required visit schedule and visit requirements and provide written informed consent

Exclusion Criteria:

  • Any new or uncontrolled condition that could make the patient unsuitable for participation

Sites / Locations

  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Patisiran (ALN-TTR02)

Arm Description

Outcomes

Primary Outcome Measures

Safety and tolerability of long-term dosing of patisiran as measured by the proportion of subjects with adverse events leading to discontinuation of study drug

Secondary Outcome Measures

Assessment of changes from baseline in neurologic impairment assessed using the Neuropathy Impairment Score (NIS) associated with long-term dosing of patisiran
Assessment of changes from baseline in neurologic impairment assessed using the Modified NIS (mNIS +7) composite score associated with long-term dosing of patisiran
Assessment of changes from baseline in neurologic impairment assessed using NIS+7 associated with long-term dosing of patisiran
Assessment of changes from baseline in quality of life using the Norfolk Quality of Life-Diabetic Neuropathy (QOL-DN) questionnaire associated with long-term dosing of patisiran
Assessment of changes from baseline in quality of life using the EuroQOL (EQ-5D) questionnaire associated with long-term dosing of patisiran
Assessment of changes from baseline in autonomic function assessed using the Composite Autonomic Symptom Score (COMPASS 31) with long-term dosing of patisiran
Assessment of changes from baseline in serum TTR lowering associated with long-term dosing of patisiran
Assessment of changes from baseline in nutritional status using modified body mass index (mBMI) associated with long-term dosing of patisiran
Assessment of changes from baseline in disability reported by patients using the Rasch-built Overall Disability Scale (R-ODS) associated with long-term dosing of patisiran
Assessment of changes from baseline in motor function assessed by NIS-Weakness (NIS-W) with long-term dosing of patisiran
Assessment of changes from baseline in motor function assessed by timed 10-meter walk test with long-term dosing of patisiran
Assessment of changes from baseline in motor function assessed by grip strength test with long-term dosing of patisiran

Full Information

First Posted
July 16, 2015
Last Updated
January 19, 2023
Sponsor
Alnylam Pharmaceuticals
search

1. Study Identification

Unique Protocol Identification Number
NCT02510261
Brief Title
The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran)
Official Title
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 Patisiran Clinical Study
Study Type
Interventional

2. Study Status

Record Verification Date
January 2023
Overall Recruitment Status
Completed
Study Start Date
July 2015 (undefined)
Primary Completion Date
November 23, 2022 (Actual)
Study Completion Date
November 23, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alnylam Pharmaceuticals

4. Oversight

Data Monitoring Committee
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyloidosis
Keywords
RNAi therapeutic, FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
211 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Patisiran (ALN-TTR02)
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Patisiran (ALN-TTR02)
Intervention Description
administered by intravenous (IV) infusion
Primary Outcome Measure Information:
Title
Safety and tolerability of long-term dosing of patisiran as measured by the proportion of subjects with adverse events leading to discontinuation of study drug
Time Frame
Up to 5 years
Secondary Outcome Measure Information:
Title
Assessment of changes from baseline in neurologic impairment assessed using the Neuropathy Impairment Score (NIS) associated with long-term dosing of patisiran
Time Frame
Baseline up to 5 years
Title
Assessment of changes from baseline in neurologic impairment assessed using the Modified NIS (mNIS +7) composite score associated with long-term dosing of patisiran
Time Frame
Baseline up to 3 years
Title
Assessment of changes from baseline in neurologic impairment assessed using NIS+7 associated with long-term dosing of patisiran
Time Frame
Baseline up to 3 years
Title
Assessment of changes from baseline in quality of life using the Norfolk Quality of Life-Diabetic Neuropathy (QOL-DN) questionnaire associated with long-term dosing of patisiran
Time Frame
Baseline up to 5 years
Title
Assessment of changes from baseline in quality of life using the EuroQOL (EQ-5D) questionnaire associated with long-term dosing of patisiran
Time Frame
Baseline up to 5 years
Title
Assessment of changes from baseline in autonomic function assessed using the Composite Autonomic Symptom Score (COMPASS 31) with long-term dosing of patisiran
Time Frame
Baseline up to 52 weeks
Title
Assessment of changes from baseline in serum TTR lowering associated with long-term dosing of patisiran
Time Frame
Baseline up to 5 years
Title
Assessment of changes from baseline in nutritional status using modified body mass index (mBMI) associated with long-term dosing of patisiran
Time Frame
Baseline up to 5 years
Title
Assessment of changes from baseline in disability reported by patients using the Rasch-built Overall Disability Scale (R-ODS) associated with long-term dosing of patisiran
Time Frame
Baseline up to 5 years
Title
Assessment of changes from baseline in motor function assessed by NIS-Weakness (NIS-W) with long-term dosing of patisiran
Time Frame
Baseline up to 5 years
Title
Assessment of changes from baseline in motor function assessed by timed 10-meter walk test with long-term dosing of patisiran
Time Frame
Baseline up to 5 years
Title
Assessment of changes from baseline in motor function assessed by grip strength test with long-term dosing of patisiran
Time Frame
Baseline up to 52 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
85 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Have completed a patisiran study (i.e., completed the last efficacy visit in the parent study) and, in the opinion of the investigator, tolerated study drug Be willing and able to comply with the protocol-required visit schedule and visit requirements and provide written informed consent Exclusion Criteria: Any new or uncontrolled condition that could make the patient unsuitable for participation
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Alnylam Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Clinical Trial Site
City
La Mesa
State/Province
California
Country
United States
Facility Name
Clinical Trial Site
City
Aurora
State/Province
Colorado
Country
United States
Facility Name
Clinical Trial Site
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32224
Country
United States
Facility Name
Clinical Trial Site
City
Chicago
State/Province
Illinois
Country
United States
Facility Name
Clinical Trial Site
City
Baltimore
State/Province
Maryland
Country
United States
Facility Name
Clinical Trial Site
City
Boston
State/Province
Massachusetts
Country
United States
Facility Name
Clinical Trial Site
City
Detroit
State/Province
Michigan
Country
United States
Facility Name
Clinical Trial Site
City
Rochester
State/Province
Minnesota
Country
United States
Facility Name
Clinical Trial Site
City
Joplin
State/Province
Missouri
ZIP/Postal Code
64804
Country
United States
Facility Name
Clinical Trial Site
City
Saint Louis
State/Province
Missouri
Country
United States
Facility Name
Clinical Trial Site
City
Cooperstown
State/Province
New York
Country
United States
Facility Name
Clinical Trial Site
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
Clinical Trial Site
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
Clinical Trial Site
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43210
Country
United States
Facility Name
Clinical Trial Site
City
Buenos Aires
Country
Argentina
Facility Name
Clinical Trial Site
City
Westmead
Country
Australia
Facility Name
Clinical Trial Site
City
Rio de Janeiro
Country
Brazil
Facility Name
Clinical Trial Site
City
São Paulo
Country
Brazil
Facility Name
Clinical Trial Site
City
Sofia
Country
Bulgaria
Facility Name
Clinical Trial Site
City
Vancouver
State/Province
British Columbia
Country
Canada
Facility Name
Clinical Trial Site
City
Nicosia
Country
Cyprus
Facility Name
Clinical Trial Site
City
Fort De France
State/Province
Martinique
ZIP/Postal Code
97261
Country
France
Facility Name
Clinical Trial Site
City
Saint-Pierre
State/Province
Reunion Island
ZIP/Postal Code
97448
Country
France
Facility Name
Clinical Trial Site
City
Creteil
Country
France
Facility Name
Clinical Trial Site
City
Le Kremlin-bicetre
Country
France
Facility Name
Clinical Trial Site
City
Lille
Country
France
Facility Name
Clinical Trial Site
City
Marseille Cedex
Country
France
Facility Name
Clinical Trial Site
City
Cologne
ZIP/Postal Code
50937
Country
Germany
Facility Name
Clinical Trial Site
City
Heidelberg
Country
Germany
Facility Name
Clinical Trial Site
City
Muenster
Country
Germany
Facility Name
Clinical Trial Site
City
Pavia
Country
Italy
Facility Name
Clinical Trial Site
City
Rome
Country
Italy
Facility Name
Clinical Trial Site
City
Sicily
Country
Italy
Facility Name
Clinical Trial Site
City
Ehime
Country
Japan
Facility Name
Clinical Trial Site
City
Fukuoka
Country
Japan
Facility Name
Clinical Trial Site
City
Hiroshima
Country
Japan
Facility Name
Clinical Trial Site
City
Kumamoto
Country
Japan
Facility Name
Clinical Trial Site
City
Nagano
Country
Japan
Facility Name
Clinical Trial Site
City
Okawasuji
Country
Japan
Facility Name
Clinical Trial Site
City
Ono
Country
Japan
Facility Name
Clinical Trial Site
City
Ōita
Country
Japan
Facility Name
Clinical Trial Site
City
Seoul
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Kuala Lumpur
Country
Malaysia
Facility Name
Clinical Trial Site
City
Tlalpan
Country
Mexico
Facility Name
Clinical Trial Site
City
Groningen
Country
Netherlands
Facility Name
Clinical Trial Site
City
Lisbon
Country
Portugal
Facility Name
Clinical Trial Site
City
Porto
Country
Portugal
Facility Name
Clinical Trial Site
City
Barcelona
Country
Spain
Facility Name
Clinical Trial Site
City
Huelva
Country
Spain
Facility Name
Clinical Trial Site
City
Madrid
Country
Spain
Facility Name
Clinical Trial Site
City
Palma De Mallorca
Country
Spain
Facility Name
Clinical Trial Site
City
Umeå
Country
Sweden
Facility Name
Clinical Trial Site
City
Taipei
ZIP/Postal Code
11217
Country
Taiwan
Facility Name
Clinical Trial Site
City
Taipei
Country
Taiwan
Facility Name
Clinical Trial Site
City
Istanbul
Country
Turkey
Facility Name
Clinical Trial Site
City
London
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
33212063
Citation
Adams D, Polydefkis M, Gonzalez-Duarte A, Wixner J, Kristen AV, Schmidt HH, Berk JL, Losada Lopez IA, Dispenzieri A, Quan D, Conceicao IM, Slama MS, Gillmore JD, Kyriakides T, Ajroud-Driss S, Waddington-Cruz M, Mezei MM, Plante-Bordeneuve V, Attarian S, Mauricio E, Brannagan TH 3rd, Ueda M, Aldinc E, Wang JJ, White MT, Vest J, Berber E, Sweetser MT, Coelho T; patisiran Global OLE study group. Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study. Lancet Neurol. 2021 Jan;20(1):49-59. doi: 10.1016/S1474-4422(20)30368-9. Epub 2020 Nov 16. Erratum In: Lancet Neurol. 2021 Feb;20(2):e2.
Results Reference
derived

Learn more about this trial

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

We'll reach out to this number within 24 hrs