CompletedPhase 2

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

France, Germany, Portugal12 participantsStarted 2015-10

Plain-language 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.

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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)

What they're measuring

Percentage Change From Baseline in Serum TTR at Month 6

Timeframe: Month 6

Trial details

NCT IDNCT02595983

SponsorAlnylam Pharmaceuticals

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2017-02-06

Results submitted2018-02-23

View on ClinicalTrials.gov More Transthyretin (TTR)-Mediated Amyloidosis trials