Phase 2
N=12
The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver Transplant
Transthyretin (TTR)-Mediated Amyloidosis · Familial Amyloidotic Polyneuropathy (FAP) · ATTR Amyloidosis · Familial Amyloid Neuropathies
Bottom Line
View on ClinicalTrials.gov: NCT02595983 ↗Enrolled (actual)
12
Serious AEs
66.7%
Results posted
Mar 2019
Primary outcome: Primary: Percentage Change From Baseline in Serum TTR at Month 6 — -72.0 percentage change from baseline in TTR
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Revusiran (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Alnylam Pharmaceuticals
- Primary completion
- Feb 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage Change From Baseline in Serum TTR at Month 6 |
-72.0 | — |
| SECONDARY Percentage Change From Baseline in Serum TTR Over 18 Months |
-75.1; -79.0; -73.0; -73.5; -73.4; -72.0 | — |
| SECONDARY Change From Baseline in Modified Neurological Impairment Score (mNIS +7) Composite Score Over 18 Months |
90.4; 6.9 | — |
| SECONDARY Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN) Questionnaire Score |
61.3; 77.1 | — |
| SECONDARY Number of Participants in Each Polyneuropathy Disability (PND) Stage Based on Worst Post-Baseline Score |
0; 0; 0; 0; 0; 0 | — |
Summary
The purpose of this study was to evaluate the safety and effectiveness of revusiran (ALN-TTRSC) in adults with transthyretin-mediated amyloidosis (ATTR), whose disease has continued to worsen after liver transplantation. Dosing has been discontinued; patients are being followed-up for safety.
Eligibility Criteria
Inclusion Criteria
- Diagnosis of FAP (familial amyloidotic polyneuropathy) with documented TTR mutation
- Received an orthotopic liver transplant ≥12 months before the date of informed consent
- An increase in polyneuropathy disability (PND) score post-transplant
- Polyneuropathy Disability score of ≤3b
Exclusion Criteria
- New York Heart Association (NYHA) classification of >2
- Other known causes of sensorimotor or autonomic neuropathy (eg, autoimmune disease)
Data sourced from ClinicalTrials.gov (NCT02595983). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.