NCT02510261CompletedPhase 3
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 Patisiran Clinical Study
Alnylam Pharmaceuticals1 site in 1 country211 target enrollmentStarted: July 16, 2015Last updated:
Conditions
Interventions
Drugs
Trial Snapshot
- Phase
- Phase 3
- Status
- Completed
- Sponsor
- Enrollment
- 211
- Locations
- 1
- Primary Endpoint
- Percentage of Participants With Adverse Events (AEs) Leading to Study Discontinuation
Study Overview
Brief Summary
The purpose of this study is to evaluate the safety and efficacy of long-term dosing with ALN-TTR02 (patisiran) in participants with transthyretin (TTR) mediated amyloidosis (ATTR).
Study Design
- Study Type
- Interventional
- Allocation
- Non Randomized
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to 85 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Have completed a patisiran study (i.e., completed the last efficacy visit in the parent study) and, in the opinion of the investigator, tolerated study drug
- •Be willing and able to comply with the protocol-required visit schedule and visit requirements and provide written informed consent
Exclusion Criteria
- •Any new or uncontrolled condition that could make the participant unsuitable for participation
Arms & Interventions
Prior Placebo Group of Study 004
Experimental
Participants who received placebo and completed parent study ALN-TTR02-004 (NCT01960348) were enrolled to receive 0.3 milligrams per kilogram (mg/kg) patisiran intravenously (IV) once every 3 weeks (Q3W) up to 65.5 months.
Intervention: Patisiran (Drug)
Prior Patisiran Group of Study 004
Experimental
Participants who received patisiran and completed parent study ALN-TTR02-004 (NCT01960348) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 66.9 months.
Intervention: Patisiran (Drug)
Prior Patisiran Group of Study 003
Experimental
Participants who received patisiran and completed parent study ALN-TTR02-003 (NCT01961921) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 61.4 months.
Intervention: Patisiran (Drug)
Outcomes
Primary Outcomes
Percentage of Participants With Adverse Events (AEs) Leading to Study Discontinuation
Time Frame: First dose up to 28 days after last dose of study drug (approximately 5.6 years)
AE is any untoward medical occurrence in a participant or clinical investigational subject administered a medicinal product and which does not necessarily have a causal relationship with this treatment.
Secondary Outcomes
- Change From Baseline in the Total Modified NIS (mNIS +7) Composite Score At Year 3(Baseline, Year 3)
- Change From Baseline in the NIS+7 Total Score at Week 52(Baseline, Week 52)
- Change From Baseline in the Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN) Questionnaire Total Score at Year 5(Baseline, Year 5)
- Change From Baseline in the Total Neuropathy Impairment Score (NIS) at Year 5(Baseline, Year 5)
- Change From Baseline in the EuroQOL-5 Dimensions-5 Levels (EQ-5D-5L) Index Score at Year 5(Baseline, Year 5)
- Change From Baseline in the EuroQoL Visual Analogue Scale (EQ-VAS) Score at Year 5(Baseline, Year 5)
- Change From Baseline in the Composite Autonomic Symptom Score (COMPASS 31) Total Score at Week 52(Baseline, Week 52)
- Change From Baseline in the Modified Body Mass Index (mBMI) at Year 5(Baseline, Year 5)
- Change From Baseline in the Rasch-built Overall Disability Scale (R-ODS) at Year 5(Baseline, Year 5)
- Change From Baseline in the NIS+7 Component: NIS-Weakness (NIS-W) Score at Year 5(Baseline, Year 5)
- Change From Baseline in the 10-meter Walk Test (10-MWT) Speed at Year 5(Baseline, Year 5)
- Change From Baseline in the Hand Grip Strength at Week 52(Baseline, Week 52)
- Number of Participants With Change From Baseline in the Polyneuropathy Disability (PND) Stage(Baseline, Year 5)
- Number of Participants With Change From Baseline in the Familial Amyloidotic Polyneuropathy (FAP) Stage(Baseline, Year 5)
- Number of Participants With Change From Baseline in the New York Heart Association (NYHA) Classification(Baseline, Year 5)
- Change From Baseline in the Intraepidermal Nerve Fiber Density (IENFD) at Year 5(Baseline, Year 5)
- Change From Baseline in the Sweat Gland Nerve Fiber Density (SGNFD) at Year 5(Baseline, Year 5)
- Change From Baseline in the Dermal Amyloid Burden at Year 5(Baseline, Year 5)
- Change From Baseline in the Cardiac Biomarker: Serum Troponin I at Year 5(Baseline, Year 5)
- Change From Baseline in the Cardiac Biomarker: N-terminal Prohormone of B-type Natriuretic Peptide (NT-proBNP) at Year 5(Baseline, Year 5)
- Change From Baseline in the Echocardiogram Parameter: Average Peak Longitudinal Strain at Year 5(Baseline, Year 5)
- Change From Baseline in the Echocardiogram Parameter: Left Ventricular (LV) Mass at Year 5(Baseline, Year 5)
- Change From Baseline in the Echocardiogram Parameter: LV End-diastolic Volume at Year 5(Baseline, Year 5)
- Change From Baseline in the Echocardiogram Parameter: LV Relative Wall Thickness at Year 5(Baseline, Year 5)
- Change From Baseline in the Echocardiogram Parameter: Mean LV Wall Thickness at Year 5(Baseline, Year 5)
- Change From Baseline in the Echocardiogram Parameter: Cardiac Output at Year 5(Baseline, Year 5)
- Percent Change From Baseline in Serum TTR Levels at Year 5(Baseline, Year 5)
Investigators
Study Sites (1)
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