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Trial record 1 of 2 for:    NCT01960348
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APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01960348
Recruitment Status : Completed
First Posted : October 10, 2013
Results First Posted : September 6, 2018
Last Update Posted : December 11, 2018
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE October 9, 2013
First Posted Date  ICMJE October 10, 2013
Results First Submitted Date  ICMJE August 7, 2018
Results First Posted Date  ICMJE September 6, 2018
Last Update Posted Date December 11, 2018
Study Start Date  ICMJE November 2013
Actual Primary Completion Date August 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 7, 2018)
Modified Neuropathy Impairment Score +7 (mNIS+7) [ Time Frame: 18mo ]
The difference between the patisiran (ALN-TTR02) and placebo groups in the change from baseline in mNIS+7 at 18 months. The mNIS+7 is a composite score that quantitates motor, sensory, and autonomic neurologic impairment due to injury of large and small nerves. The minimum and maximum values are 0 and 304, respectively. A higher score indicates a worse outcome.
Original Primary Outcome Measures  ICMJE
 (submitted: October 9, 2013)
The difference between the ALN TTR02 and placebo groups in the change from baseline of modified Neuropathy Impairment Score+7 (mNIS+7) [ Time Frame: 18 months ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: August 7, 2018)
  • Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Questionnaire [ Time Frame: 18mo ]
    The difference between the patisiran (ALN-TTR02) and placebo groups in the change from baseline in Norfolk QoL-DN at 18 months. The Norfolk QoL-DN questionnaire is a standardized 35-item patient-reported outcomes measure that is sensitive to the different features of diabetic neuropathy - small fiber, large fiber, and autonomic nerve function. The minimum and maximum values are -4 and 136, respectively. A higher score indicates a worse outcome.
  • Neurological Impairment Score-Weakness (NIS-W) Score [ Time Frame: 18mo ]
    The difference between the patisiran (ALN-TTR02) and placebo groups in the change from baseline in NIS-W at 18 months. NIS-W is a measure of motor strength, comprised of cranial nerve and both upper and lower limb motor assessments. The minimum and maximum values are 0 and 192, respectively. A higher score indicates a worse outcome.
  • Rasch-built Overall Disability Scale (R-ODS) Score [ Time Frame: 18mo ]
    The difference between the patisiran (ALN-TTR02) and placebo groups in the change from baseline in R-ODS score at 18 months. The R-ODS is comprised of a 24-item linearly weighted scale that specifically captures activity and social participation limitations in patients. The minimum and maximum values are 0 and 48, respectively. A higher score indicates a better outcome.
  • Timed 10-meter Walk Test (10-MWT, Gait Speed) [ Time Frame: 18mo ]
    The difference between the patisiran (ALN-TTR02) and placebo groups in the change from baseline in 10-MWT at 18 months. Ability to ambulate (gait speed) was assessed through the 10-meter walk test (10-MWT). The walk had to be completed without assistance from another person; ambulatory aids such as canes and walkers were permitted.
  • Modified Body Mass Index (mBMI) [ Time Frame: 18mo ]
    The difference between the patisiran (ALN-TTR02) and placebo groups in the change from baseline in mBMI at 18 months. The nutritional status of patients was evaluated using the mBMI; calculated as the product of BMI (weight in kilograms divided by the square of height in meters) and serum albumin (g/L).
  • Autonomic Symptoms Questionnaire (Composite Autonomic Symptom Score [COMPASS 31]) [ Time Frame: 18mo ]
    The difference between the patisiran (ALN-TTR02) and placebo groups in the change from baseline in COMPASS 31 at 18 months. The COMPASS 31 is a measure of autonomic neuropathy symptoms. The questions evaluated 6 autonomic domains (orthostatic intolerance, vasomotor, secretomotor, gastrointestinal, bladder, and pupillomotor). The minimum and maximum values are 0 and 100, respectively. A higher score indicates a worse outcome.
Original Secondary Outcome Measures  ICMJE
 (submitted: October 9, 2013)
  • The difference between ALN-TTR02 and placebo in the change from baseline in quality of life [ Time Frame: 18 months ]
  • The difference between ALN-TTR02 and placebo in the change from baseline in motor function [ Time Frame: 18 months ]
  • The difference between ALN-TTR02 and placebo in the change from baseline in autonomic function [ Time Frame: 18 months ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis
Official Title  ICMJE 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)
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE
  • TTR-mediated Amyloidosis
  • Amyloidosis, Hereditary
  • Amyloid Neuropathies, Familial
  • Familial Amyloid Polyneuropathies
  • Amyloid Neuropathies
  • Amyloidosis, Hereditary, Transthyretin-Related
Intervention  ICMJE
  • Drug: patisiran (ALN-TTR02)
    administered by intravenous (IV) infusion
  • Drug: Sterile Normal Saline (0.9% NaCl)
    administered by intravenous (IV) infusion
Study Arms  ICMJE
  • Active Comparator: patisiran (ALN-TTR02)
    Intervention: Drug: patisiran (ALN-TTR02)
  • Placebo Comparator: Sterile Normal Saline (0.9% NaCl)
    Intervention: Drug: Sterile Normal Saline (0.9% NaCl)
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: January 29, 2016)
225
Original Estimated Enrollment  ICMJE
 (submitted: October 9, 2013)
200
Actual Study Completion Date  ICMJE August 2017
Actual Primary Completion Date August 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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)

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 85 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Argentina,   Australia,   Brazil,   Bulgaria,   Canada,   Cyprus,   France,   Germany,   Italy,   Japan,   Korea, Republic of,   Malaysia,   Mexico,   Netherlands,   Portugal,   Spain,   Sweden,   Taiwan,   Turkey,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01960348
Other Study ID Numbers  ICMJE ALN-TTR02-004
2013-002987-17 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Alnylam Pharmaceuticals
Study Sponsor  ICMJE Alnylam Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Jared Gollob Alnylam Pharmaceuticals
PRS Account Alnylam Pharmaceuticals
Verification Date November 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP