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.