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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT02510261
Ethics application status
Date submitted
16/07/2015
Date registered
29/07/2015

Titles & IDs
Public title
The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants 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
2014-003877-40
Secondary ID [2] 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

Experimental: Prior Placebo Group of Study 004 - Participants who received placebo and completed parent study ALN-TTR02-004 (NCT01960348) were enrolled to receive 0.3 milligrams per kilogram (mg/kg) patisiran intravenously (IV) once every 3 weeks (Q3W) up to 65.5 months.

Experimental: Prior Patisiran Group of Study 004 - Participants who received patisiran and completed parent study ALN-TTR02-004 (NCT01960348) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 66.9 months.

Experimental: Prior Patisiran Group of Study 003 - Participants who received patisiran and completed parent study ALN-TTR02-003 (NCT01961921) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 61.4 months.


Treatment: Drugs: Patisiran
Patisiran was administered IV.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of Participants With Adverse Events (AEs) Leading to Study Discontinuation
Timepoint [1] 0 0
First dose up to 28 days after last dose of study drug (approximately 5.6 years)
Secondary outcome [1] 0 0
Change From Baseline in the Total Neuropathy Impairment Score (NIS) at Year 5
Timepoint [1] 0 0
Baseline, Year 5
Secondary outcome [2] 0 0
Change From Baseline in the Total Modified NIS (mNIS +7) Composite Score At Year 3
Timepoint [2] 0 0
Baseline, Year 3
Secondary outcome [3] 0 0
Change From Baseline in the NIS+7 Total Score at Week 52
Timepoint [3] 0 0
Baseline, Week 52
Secondary outcome [4] 0 0
Change From Baseline in the Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN) Questionnaire Total Score at Year 5
Timepoint [4] 0 0
Baseline, Year 5
Secondary outcome [5] 0 0
Change From Baseline in the EuroQOL-5 Dimensions-5 Levels (EQ-5D-5L) Index Score at Year 5
Timepoint [5] 0 0
Baseline, Year 5
Secondary outcome [6] 0 0
Change From Baseline in the EuroQoL Visual Analogue Scale (EQ-VAS) Score at Year 5
Timepoint [6] 0 0
Baseline, Year 5
Secondary outcome [7] 0 0
Change From Baseline in the Composite Autonomic Symptom Score (COMPASS 31) Total Score at Week 52
Timepoint [7] 0 0
Baseline, Week 52
Secondary outcome [8] 0 0
Change From Baseline in the Modified Body Mass Index (mBMI) at Year 5
Timepoint [8] 0 0
Baseline, Year 5
Secondary outcome [9] 0 0
Change From Baseline in the Rasch-built Overall Disability Scale (R-ODS) at Year 5
Timepoint [9] 0 0
Baseline, Year 5
Secondary outcome [10] 0 0
Change From Baseline in the NIS+7 Component: NIS-Weakness (NIS-W) Score at Year 5
Timepoint [10] 0 0
Baseline, Year 5
Secondary outcome [11] 0 0
Change From Baseline in the 10-meter Walk Test (10-MWT) Speed at Year 5
Timepoint [11] 0 0
Baseline, Year 5
Secondary outcome [12] 0 0
Change From Baseline in the Hand Grip Strength at Week 52
Timepoint [12] 0 0
Baseline, Week 52
Secondary outcome [13] 0 0
Number of Participants With Change From Baseline in the Polyneuropathy Disability (PND) Stage
Timepoint [13] 0 0
Baseline, Year 5
Secondary outcome [14] 0 0
Number of Participants With Change From Baseline in the Familial Amyloidotic Polyneuropathy (FAP) Stage
Timepoint [14] 0 0
Baseline, Year 5
Secondary outcome [15] 0 0
Number of Participants With Change From Baseline in the New York Heart Association (NYHA) Classification
Timepoint [15] 0 0
Baseline, Year 5
Secondary outcome [16] 0 0
Change From Baseline in the Intraepidermal Nerve Fiber Density (IENFD) at Year 5
Timepoint [16] 0 0
Baseline, Year 5
Secondary outcome [17] 0 0
Change From Baseline in the Sweat Gland Nerve Fiber Density (SGNFD) at Year 5
Timepoint [17] 0 0
Baseline, Year 5
Secondary outcome [18] 0 0
Change From Baseline in the Dermal Amyloid Burden at Year 5
Timepoint [18] 0 0
Baseline, Year 5
Secondary outcome [19] 0 0
Change From Baseline in the Cardiac Biomarker: Serum Troponin I at Year 5
Timepoint [19] 0 0
Baseline, Year 5
Secondary outcome [20] 0 0
Change From Baseline in the Cardiac Biomarker: N-terminal Prohormone of B-type Natriuretic Peptide (NT-proBNP) at Year 5
Timepoint [20] 0 0
Baseline, Year 5
Secondary outcome [21] 0 0
Change From Baseline in the Echocardiogram Parameter: Average Peak Longitudinal Strain at Year 5
Timepoint [21] 0 0
Baseline, Year 5
Secondary outcome [22] 0 0
Change From Baseline in the Echocardiogram Parameter: Left Ventricular (LV) Mass at Year 5
Timepoint [22] 0 0
Baseline, Year 5
Secondary outcome [23] 0 0
Change From Baseline in the Echocardiogram Parameter: LV End-diastolic Volume at Year 5
Timepoint [23] 0 0
Baseline, Year 5
Secondary outcome [24] 0 0
Change From Baseline in the Echocardiogram Parameter: LV Relative Wall Thickness at Year 5
Timepoint [24] 0 0
Baseline, Year 5
Secondary outcome [25] 0 0
Change From Baseline in the Echocardiogram Parameter: Mean LV Wall Thickness at Year 5
Timepoint [25] 0 0
Baseline, Year 5
Secondary outcome [26] 0 0
Change From Baseline in the Echocardiogram Parameter: Cardiac Output at Year 5
Timepoint [26] 0 0
Baseline, Year 5
Secondary outcome [27] 0 0
Percent Change From Baseline in Serum TTR Levels at Year 5
Timepoint [27] 0 0
Baseline, Year 5

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
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Any new or uncontrolled condition that could make the participant unsuitable for participation

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
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
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
16/07/2015
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
23/11/2022
Sample size
Target
Accrual to date
Final
211
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
Trial website
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

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT02510261