A study to look at the long-term efficacy and safety of ALN-TTRSC in patients with an inherited condition that causes certain protein molecules to deposit in the heart
- Conditions
- Transthyretin (TTR) mediated familial amyloidotic cardiomyopathy (FAC)MedDRA version: 18.1Level: PTClassification code 10016202Term: Familial amyloidosisSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2015-005333-49-GB
- Lead Sponsor
- Alnylam Pharmaceuticals, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 200
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
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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method Secondary end point(s): 1. Change from baseline in 6-minute walk distance <br><br>2. Change from baseline in serum TTR;Timepoint(s) of evaluation of this end point: 1. Every 6 months for 26 months <br><br>2. Every 6 months for 26 months