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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT01960348




Registration number
NCT01960348
Ethics application status
Date submitted
9/10/2013
Date registered
10/10/2013
Date last updated
22/04/2024

Titles & IDs
Public title
APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis
Scientific title
APOLLO: A Phase 3 Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Patisiran (ALN-TTR02) in Transthyretin (TTR)-Mediated Polyneuropathy (Familial Amyloidotic Polyneuropathy-FAP)
Secondary ID [1] 0 0
2013-002987-17
Secondary ID [2] 0 0
ALN-TTR02-004
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
TTR-mediated Amyloidosis 0 0
Amyloidosis, Hereditary 0 0
Amyloid Neuropathies, Familial 0 0
Familial Amyloid Polyneuropathies 0 0
Amyloid Neuropathies 0 0
Amyloidosis, Hereditary, Transthyretin-Related 0 0
Condition category
Condition code
Neurological 0 0 0 0
Other neurological disorders
Neurological 0 0 0 0
Neurodegenerative diseases
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Intervention/exposure
Study type
Interventional(has expanded access)
Description of intervention(s) / exposure
Treatment: Drugs - patisiran (ALN-TTR02)
Treatment: Drugs - Sterile Normal Saline (0.9% NaCl)

Active Comparator: patisiran (ALN-TTR02) -

Placebo Comparator: Sterile Normal Saline (0.9% NaCl) -


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

Treatment: Drugs: Sterile Normal Saline (0.9% NaCl)
administered by intravenous (IV) infusion
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Modified Neuropathy Impairment Score +7 (mNIS+7)
Timepoint [1] 0 0
18mo
Secondary outcome [1] 0 0
Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Questionnaire
Timepoint [1] 0 0
18mo
Secondary outcome [2] 0 0
Neurological Impairment Score-Weakness (NIS-W) Score
Timepoint [2] 0 0
18mo
Secondary outcome [3] 0 0
Rasch-built Overall Disability Scale (R-ODS) Score
Timepoint [3] 0 0
18mo
Secondary outcome [4] 0 0
Timed 10-meter Walk Test (10-MWT, Gait Speed)
Timepoint [4] 0 0
18mo
Secondary outcome [5] 0 0
Modified Body Mass Index (mBMI)
Timepoint [5] 0 0
18mo
Secondary outcome [6] 0 0
Autonomic Symptoms Questionnaire (Composite Autonomic Symptom Score [COMPASS 31])
Timepoint [6] 0 0
18mo

Eligibility
Key inclusion criteria
- Male or female of 18 to 85 years of age (inclusive);

- Have a diagnosis of FAP

- Neuropathy Impairment Score requirement of 5-130

- Meet Karnofsky performance status requirements

- Have adequate complete blood counts and liver function tests

- Have adequate cardiac function

- Have negative serology for hepatitis B virus (HBV) and hepatitis C virus (HCV)
Minimum age
18 Years
Maximum age
85 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Had a prior liver transplant or is planned to undergo liver transplant during the
study period;

- Has untreated hypo- or hyperthyroidism;

- Has known human immunodeficiency virus (HIV) infection;

- Had a malignancy within 2 years, except for basal or squamous cell carcinoma of the
skin or carcinoma in situ of the cervix that has been successfully treated;

- Recently received an investigational agent or device

- Is currently taking diflunisal, tafamidis, doxycycline, or tauroursodeoxycholic acid

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled 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
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
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
1/11/2013
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/08/2017
Sample size
Target
Accrual to date
Final
225
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
Illinois
Country [4] 0 0
United States of America
State/province [4] 0 0
Maryland
Country [5] 0 0
United States of America
State/province [5] 0 0
Massachusetts
Country [6] 0 0
United States of America
State/province [6] 0 0
Michigan
Country [7] 0 0
United States of America
State/province [7] 0 0
Minnesota
Country [8] 0 0
United States of America
State/province [8] 0 0
Missouri
Country [9] 0 0
United States of America
State/province [9] 0 0
New York
Country [10] 0 0
United States of America
State/province [10] 0 0
North Carolina
Country [11] 0 0
United States of America
State/province [11] 0 0
Oregon
Country [12] 0 0
Argentina
State/province [12] 0 0
Buenos Aires
Country [13] 0 0
Brazil
State/province [13] 0 0
Ribeirao Preto
Country [14] 0 0
Brazil
State/province [14] 0 0
Rio de Janeiro
Country [15] 0 0
Brazil
State/province [15] 0 0
Sao Paulo
Country [16] 0 0
Bulgaria
State/province [16] 0 0
Sofia
Country [17] 0 0
Canada
State/province [17] 0 0
British Columbia
Country [18] 0 0
Cyprus
State/province [18] 0 0
Nicosia
Country [19] 0 0
France
State/province [19] 0 0
Bourdeaux
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 Cedex
Country [23] 0 0
France
State/province [23] 0 0
Marseille Cedex
Country [24] 0 0
Germany
State/province [24] 0 0
Heidelberg
Country [25] 0 0
Germany
State/province [25] 0 0
Muenster
Country [26] 0 0
Germany
State/province [26] 0 0
Regensburg
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
Nagano
Country [31] 0 0
Japan
State/province [31] 0 0
Aichi
Country [32] 0 0
Japan
State/province [32] 0 0
Kumamoto
Country [33] 0 0
Korea, Republic of
State/province [33] 0 0
Seoul
Country [34] 0 0
Malaysia
State/province [34] 0 0
Kuala Lumpur
Country [35] 0 0
Mexico
State/province [35] 0 0
Mexico City
Country [36] 0 0
Netherlands
State/province [36] 0 0
Groningen
Country [37] 0 0
Portugal
State/province [37] 0 0
Lisbon
Country [38] 0 0
Portugal
State/province [38] 0 0
Porto
Country [39] 0 0
Spain
State/province [39] 0 0
Barcelona
Country [40] 0 0
Spain
State/province [40] 0 0
Huelva
Country [41] 0 0
Spain
State/province [41] 0 0
Madrid
Country [42] 0 0
Spain
State/province [42] 0 0
Palma De Mallorca
Country [43] 0 0
Sweden
State/province [43] 0 0
Umeå
Country [44] 0 0
Taiwan
State/province [44] 0 0
Taipai
Country [45] 0 0
Taiwan
State/province [45] 0 0
Taipei
Country [46] 0 0
Turkey
State/province [46] 0 0
Istanbul
Country [47] 0 0
United Kingdom
State/province [47] 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 patisiran (ALN-TTR02) in
patients with transthyretin (TTR) mediated amyloidosis. An open-label, single-arm, long-term
follow-up extension study NCT02510261 (ALN-TTR02-006) was initiated to provide participants
who completed this study with continued patisiran-LNP (lipid nanoparticle) treatment.
Trial website
https://clinicaltrials.gov/ct2/show/NCT01960348
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Jared Gollob
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

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT01960348