Clinical trial NCT01961921: The Study of ALN-TTR02 (Patisiran) for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran)
Official title: A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Safety, Clinical Activity, and Pharmacokinetics of ALN-TTR02 in Patients With Familial Amyloidotic Polyneuropathy Who Have Previously Received ALN-TTR02
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: TTR-mediated Amyloidosis
Keywords: RNAi therapeutic
Lead sponsor: Alnylam Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2013-10-14
Enrollment: 27
Sex: ALL
Minimum age (years): 18
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the safety and tolerability of long-term dosing with ALN-TTR02 (patisiran) in patients with transthyretin (TTR) mediated amyloidosis (ATTR).


Eligibility criteria: Inclusion Criteria:

* Previously received and tolerated ALN-TTR02 (patisiran) in Study ALN-TTR02-002.
* Adequate Karnofsky performance status, liver function, and renal function.

Exclusion Criteria:

* Pregnant or nursing.
* Has had a liver transplant.
* Has a New York Heart Association heart failure classification \>2.
* Has unstable angina.
* Has uncontrolled clinically significant cardiac arrhythmia.

Primary outcomes:
measure: The Number of Participants Experiencing Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation
timeFrame: From Baseline up to 56 days post last dose
Secondary outcomes:
measure: Percentage Change From Baseline in Serum TTR Levels
timeFrame: From Baseline up to 56 days post last dose
measure: Change From Baseline in the Modified Neuropathy Impairment Score +7 (mNIS+7)
timeFrame: Baseline, Month 24
measure: Change From Baseline in Quality of Life and Disability as Assessed by the EuroQoL (Quality of Life)-5 Dimensions (EQ-5D), EuroQoL Visual Analog Scale (EQ-VAS) Questionnaires and Rasch-built Overall Disability Scale (R-ODS)
timeFrame: Baseline, Month 24
measure: Change in Gait Speed With 10-meter Walk Test
timeFrame: Baseline, Month 24
measure: Mean Change From Baseline in Hand Grip Strength
timeFrame: Baseline, Month 24
measure: Change From Baseline in Nutritional Status (Modified Body Mass Index, mBMI)
timeFrame: Baseline, Month 24
Other outcomes:
Locations:
city: Boston
country: United States
facility: Clinical Trial Site
state: Massachusetts
city: Rio de Janeiro
country: Brazil
facility: Clinical Trial Site
city: Le Kremlin-bicetre
country: France
facility: Clinical Trial Site
city: Marseille Cedex
country: France
facility: Clinical Trial Site
city: Munster
country: Germany
facility: Clinical Trial Site
city: Lisbon
country: Portugal
facility: Clinical Trial Site
city: Porto
country: Portugal
facility: Clinical Trial Site
city: Palma De Mallorca
country: Spain
facility: Clinical Trial Site
city: Umeå
country: Sweden
facility: Clinical Trial Site
Study officials:
affiliation: Alnylam Pharmaceuticals
name: Jared Gollob, MD
role: STUDY_DIRECTOR
References:
citation: Coelho T, Adams D, Conceicao I, Waddington-Cruz M, Schmidt HH, Buades J, Campistol J, Berk JL, Polydefkis M, Wang JJ, Chen J, Sweetser MT, Gollob J, Suhr OB. A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis. Orphanet J Rare Dis. 2020 Jul 8;15(1):179. doi: 10.1186/s13023-020-01399-4.