Study to Evaluate the Long-term Safety and Efficacy of Patisiran (ALN-TTR02) in Patients with Familial Amyloidotic Polyneuropathy (FAP)

• Register: EUCTR
• Last refreshed on: 24 August 2015
• Main ID: EUCTR2014-003877-40-ES
• Date of registration: 03/03/2015
• Prospective Registration: Yes
• Primary sponsor: Alnylam Pharmaceuticals, Inc.
• Public title: Study to Evaluate the Long-term Safety and Efficacy of Patisiran (ALN-TTR02) in Patients with Familial Amyloidotic Polyneuropathy (FAP)
• 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 Clinical Study with Patisiran
• Date of first enrolment: 11/05/2015
• Target sample size: 228
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2014-003877-40
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 1

Countries of recruitment

Argentina, Brazil, Bulgaria, Canada, France, Germany, Italy, Japan, Korea, Republic of, Mexico, Netherlands, Portugal, Spain, Sweden, Taiwan, Turkey, United Kingdom, United States

Contacts

• Name: Monica Bermejo
• Affiliation: Medpace Spain, S.L

• Name: Monica Bermejo
• Affiliation: Medpace Spain, S.L

Key inclusion & exclusion criteria

Inclusion criteria:
1. Have completed a patisiran study
2. Adequate liver function and renal function
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 114
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 114

Exclusion criteria:
1. Pregnant or nursing
2. Have uncontrolled clinically significant cardiac arrhythmia or unstable angina


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Transthyretin mediated amyloidosis (ATTR)
MedDRA version: 17.1 Level: PT Classification code 10007509 Term: Cardiac amyloidosis System Organ Class: 10007541 - Cardiac disorders
MedDRA version: 17.1 Level: PT Classification code 10019889 Term: Hereditary neuropathic amyloidosis System Organ Class: 10010331 - Congenital, familial and genetic disorders

Intervention(s)

Product Name: Patisiran
Product Code: ALN-TTR02
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Patisiran
Current Sponsor code: Patisiran
Other descriptive name: ALN-TTR02
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 2-

Primary Outcome(s)

Primary end point(s): To assess the safety and efficacy of long-term dosing with patisiran

Timepoint(s) of evaluation of this end point: The review of AEs will be done at each visit from Day 0 through 28 days post last dose. Efficacy assessments will be performed at Day 0, annually and 28 days post last dose

Main Objective: To assess the safety and efficacy of long-term dosing with patisiran in transthyretin-mediated amyloidosis (ATTR) patients with familial amyloidotic polyneuropathy (FAP)

Secondary Objective: None

Secondary Outcome(s)

Secondary end point(s): None

Timepoint(s) of evaluation of this end point: None

Secondary ID(s)

ALN-TTR02-006

Source(s) of Monetary Support

Alnylam Pharmaceuticals, Inc