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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02510261




Registration number
NCT02510261
Ethics application status
Date submitted
16/07/2015
Date registered
29/07/2015
Date last updated
16/09/2020

Titles & IDs
Public 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)
Scientific 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
Secondary ID [1] 0 0
ALN-TTR02-006
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Amyloidosis 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Patisiran (ALN-TTR02)

Experimental: Patisiran (ALN-TTR02) -


Treatment: Drugs: Patisiran (ALN-TTR02)
administered by intravenous (IV) infusion

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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
Timepoint [1] 0 0
52 weeks
Secondary outcome [1] 0 0
Assessment of changes from baseline in neurologic impairment assessed using the Neuropathy Impairment Score (NIS) associated with long-term dosing of patisiran
Timepoint [1] 0 0
baseline, week 52
Secondary outcome [2] 0 0
Assessment of changes from baseline in neurologic impairment assessed using the Modified NIS (mNIS +7) composite score associated with long-term dosing of patisiran
Timepoint [2] 0 0
baseline, week 52
Secondary outcome [3] 0 0
Assessment of changes from baseline in neurologic impairment assessed using NIS+7 associated with long-term dosing of patisiran
Timepoint [3] 0 0
baseline, week 52
Secondary outcome [4] 0 0
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
Timepoint [4] 0 0
baseline, week 52
Secondary outcome [5] 0 0
Assessment of changes from baseline in quality of life using the EuroQOL (EQ-5D) questionnaire associated with long-term dosing of patisiran
Timepoint [5] 0 0
baseline, week 52
Secondary outcome [6] 0 0
Assessment of changes from baseline in autonomic function assessed using the Composite Autonomic Symptom Score (COMPASS 31) with long-term dosing of patisiran
Timepoint [6] 0 0
baseline, week 52
Secondary outcome [7] 0 0
Assessment of changes from baseline in serum TTR lowering associated with long-term dosing of patisiran
Timepoint [7] 0 0
baseline, week 52
Secondary outcome [8] 0 0
Assessment of changes from baseline in nutritional status using modified body mass index (mBMI) associated with long-term dosing of patisiran
Timepoint [8] 0 0
baseline, week 52
Secondary outcome [9] 0 0
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
Timepoint [9] 0 0
baseline, week 52
Secondary outcome [10] 0 0
Assessment of changes from baseline in motor function assessed by NIS-Weakness (NIS-W) with long-term dosing of patisiran
Timepoint [10] 0 0
baseline, week 52
Secondary outcome [11] 0 0
Assessment of changes from baseline in motor function assessed by timed 10-meter walk test with long-term dosing of patisiran
Timepoint [11] 0 0
baseline, week 52
Secondary outcome [12] 0 0
Assessment of changes from baseline in motor function assessed by grip strength test with long-term dosing of patisiran
Timepoint [12] 0 0
baseline, week 52

Eligibility
Key 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
Minimum age
18 Years
Maximum age
85 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Any new or uncontrolled condition that could make the patient unsuitable for
participation

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Clinical Trial Site - Westmead
Recruitment postcode(s) [1] 0 0
- Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Maryland
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
Michigan
Country [8] 0 0
United States of America
State/province [8] 0 0
Minnesota
Country [9] 0 0
United States of America
State/province [9] 0 0
Missouri
Country [10] 0 0
United States of America
State/province [10] 0 0
New York
Country [11] 0 0
United States of America
State/province [11] 0 0
Ohio
Country [12] 0 0
Argentina
State/province [12] 0 0
Buenos Aires
Country [13] 0 0
Brazil
State/province [13] 0 0
Rio de Janeiro
Country [14] 0 0
Brazil
State/province [14] 0 0
São Paulo
Country [15] 0 0
Bulgaria
State/province [15] 0 0
Sofia
Country [16] 0 0
Canada
State/province [16] 0 0
British Columbia
Country [17] 0 0
Cyprus
State/province [17] 0 0
Nicosia
Country [18] 0 0
France
State/province [18] 0 0
Martinique
Country [19] 0 0
France
State/province [19] 0 0
Reunion Island
Country [20] 0 0
France
State/province [20] 0 0
Creteil
Country [21] 0 0
France
State/province [21] 0 0
Le Kremlin-bicetre
Country [22] 0 0
France
State/province [22] 0 0
Lille
Country [23] 0 0
France
State/province [23] 0 0
Marseille Cedex
Country [24] 0 0
Germany
State/province [24] 0 0
Cologne
Country [25] 0 0
Germany
State/province [25] 0 0
Heidelberg
Country [26] 0 0
Germany
State/province [26] 0 0
Muenster
Country [27] 0 0
Italy
State/province [27] 0 0
Pavia
Country [28] 0 0
Italy
State/province [28] 0 0
Rome
Country [29] 0 0
Italy
State/province [29] 0 0
Sicily
Country [30] 0 0
Japan
State/province [30] 0 0
Ehime
Country [31] 0 0
Japan
State/province [31] 0 0
Fukuoka
Country [32] 0 0
Japan
State/province [32] 0 0
Hiroshima
Country [33] 0 0
Japan
State/province [33] 0 0
Kumamoto
Country [34] 0 0
Japan
State/province [34] 0 0
Nagano
Country [35] 0 0
Japan
State/province [35] 0 0
Okawasuji
Country [36] 0 0
Japan
State/province [36] 0 0
Ono
Country [37] 0 0
Japan
State/province [37] 0 0
Oita
Country [38] 0 0
Korea, Republic of
State/province [38] 0 0
Seoul
Country [39] 0 0
Malaysia
State/province [39] 0 0
Kuala Lumpur
Country [40] 0 0
Mexico
State/province [40] 0 0
Tlalpan
Country [41] 0 0
Netherlands
State/province [41] 0 0
Groningen
Country [42] 0 0
Portugal
State/province [42] 0 0
Lisbon
Country [43] 0 0
Portugal
State/province [43] 0 0
Porto
Country [44] 0 0
Spain
State/province [44] 0 0
Barcelona
Country [45] 0 0
Spain
State/province [45] 0 0
Huelva
Country [46] 0 0
Spain
State/province [46] 0 0
Madrid
Country [47] 0 0
Spain
State/province [47] 0 0
Palma De Mallorca
Country [48] 0 0
Sweden
State/province [48] 0 0
Umeå
Country [49] 0 0
Taiwan
State/province [49] 0 0
Taipei
Country [50] 0 0
Turkey
State/province [50] 0 0
Istanbul
Country [51] 0 0
United Kingdom
State/province [51] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Alnylam Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
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).
Trial website
https://clinicaltrials.gov/show/NCT02510261
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Alnylam Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications