Overview

This trial is active, not recruiting.

Conditions ttr-mediated amyloidosis, amyloidosis, hereditary, amyloid neuropathies, familial, familial amyloid polyneuropathies, amyloid neuropathies, amyloidosis, hereditary, transthyretin-related
Treatments patisiran (aln-ttr02), sterile normal saline (0.9% nacl)
Phase phase 3
Sponsor Alnylam Pharmaceuticals
Start date November 2013
End date July 2017
Trial size 225 participants
Trial identifier NCT01960348, 2013-002987-17, ALN-TTR02-004

Summary

The purpose of this study is to evaluate the safety and efficacy of patisiran (ALN-TTR02) in patients with transthyretin (TTR) mediated amyloidosis

United States No locations recruiting
Other countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking double blind (subject, investigator)
Primary purpose treatment
Arm
(Active Comparator)
patisiran (aln-ttr02)
administered by intravenous (IV) infusion
(Placebo Comparator)
sterile normal saline (0.9% nacl)
administered by intravenous (IV) infusion

Primary Outcomes

Measure
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

Secondary Outcomes

Measure
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

Eligibility Criteria

Male or female participants from 18 years up to 85 years old.

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

Additional Information

Official 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)
Trial information was received from ClinicalTrials.gov and was last updated in October 2016.
Information provided to ClinicalTrials.gov by Alnylam Pharmaceuticals.
Location data was received from the National Cancer Institute and was last updated in October 2016.