Transthyretin (TTR) mediated familial amyloidotic cardiomyopathy (FAC) MedDRA version: 18.1 Level: PT Classification code 10016202 Term: Familial amyloidosis System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: Revusiran
Product Code: ALN-TTRSC
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Revusiran
Current Sponsor code: ALN-TTRSC
Other descriptive name: ALN-51547
Concentrati
Sponsors
Alnylam Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Completed and, in the opinion of the Investigator, tolerated study drug dosing in Study ALN-TTRSC-004 (ie, completed the Month 18 visit) 2. Women of child-bearing potential must have a negative pregnancy test, cannot be breastfeeding, and must use 1 highly effective method of contraception in combination with a barrier method throughout study participation and for 28 days after last dose of study drug 3. Male patients with partners of child-bearing potential must agree to use a condom, accompanied with spermicidal foam, gel, film, cream, or suppository, except in countries where spermicide is not available for use in combination with a condom, throughout study participation and for 28 days after the last dose of study drug; males must also abstain from sperm donation after the first dose of study drug through study participation and for 28 days after the last dose of study drug 4. Willing and able to comply with the protocol-required visit schedule and requirements and provide informed consent or have a legal guardian who can provide informed consent 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 100 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 100
Exclusion criteria
Exclusion criteria: 1. AST and ALT >2 × ULN, or total bilirubin >2 mg/dL (34.2 µmol/L), unless elevation in total bilirubin is due to Gilbert's syndrome. If a patient does not meet this exclusion criterion but does not meet dose holding criteria for this study, then the patient may be enrolled after consultation with the Medical Monitor. 2. eGFR <15 mL/min/1.73 m2 (using the Modification of Diet in Renal Disease [MDRD] formula) 3. Is currently taking tafamidis, diflunisal, doxycycline, or tauroursodeoxycholic acid; if previously on any of these agents, must have completed a 14-day wash-out prior to start of study drug administration in this study 4. Received an investigational agent or device, other than revusiran or placebo in ALN-TTRSC-004, within 28 days of anticipated study drug administration or 5 half-lives of the investigational drug, whichever is longer 5. History of allergic reaction to an oligonucleotide or GalNAc 6. Any significant change in the patient’s medical status or comorbidities that, in the opinion of the Investigator, would interfere with study compliance or data interpretation.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the safety and efficacy of long-term dosing with revusiran in patients with FAC.;Secondary Objective: Not Applicable;Primary end point(s): 1. Incidence of adverse events (AEs) and serious adverse events (SAEs);Timepoint(s) of evaluation of this end point: 1. Continuous evaluation over 26 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Change from baseline in 6-minute walk distance 2. Change from baseline in serum TTR;Timepoint(s) of evaluation of this end point: 1. Every 6 months for 26 months 2. Every 6 months for 26 months | — |
Countries
Belgium, Brazil, Canada, France, Germany, Italy, Mexico, Spain, Sweden, United Kingdom, United States
Contacts
Public ContactClinical Trial Hotline
Alnylam Pharmaceuticals Inc
Outcome results
None listed