Safety and tolerability of SC ALN-TTRSCO2 in healthy volunteers
Research type
Research Study
Full title
A Phase 1, Randomized, Single-Blind, Placebo-Controlled, Single-Ascending Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-TTRSC02 in Healthy Subjects
IRAS ID
196337
Contact name
Jorg Taubel
Contact email
Sponsor organisation
Alnylam Pharmaceuticals, Inc
Eudract number
2015-005803-98
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 5 months, 3 days
Research summary
We are conducting a First-in-Human clinical study with an investigational medicine known as ALN-TTRSC02 which is being developed as a subcutaneous (SC) injection by Alnylam Pharmaceuticals Inc. (the sponsor of the study) for treatment of patients with transthyretin-mediated amyloidosis (ATTR amyloidosis). \n\nThe main aim of the study is to see if a single dose of ALN-TTRSC02 is safe and well tolerated. This includes a review of any symptoms or changes in health status after administration of ALN-TTRSC02. In addition, the study will determine if ALN-TTRSC02 has any impact on the body by assessing blood transthyretin (TTR) and Vitamin A levels and will look at the amount of ALN-TTRSC02 that gets into the blood and urine after dosing. We will assess the safety parameters including vital signs measurements, physical examinations, laboratory evaluations, electrocardiogram and monitor any adverse events. \n\nThe study is divided into 10 groups and the doses are scheduled to start with a low dose of ALN-TTRSC02 and increase to a higher dose once the previous doses have been found to be safe. The starting dose will be 25 mg of ALN-TTRSC02 and the maximum dose will not be more than 600 mg. \n\nIt is anticipated that up to 110 healthy volunteers (Caucasian and Japanese) will be included in this study. This study will take up to 4.7 months to complete for each volunteer, including screening, study conduct and follow up. If additional TTR monitoring is required, time can be extended for up to a maximum of 8 months after the last post-dose follow up visit (Day 90). Participation will require an in-patient period of 4 days/3 nights and 8 outpatient visits.\n\nThis study will be conducted at Richmond Pharmacology Ltd. and is funded by Alnylam Pharmaceuticals Inc., which is a biopharmaceutical company based in the USA.\n
REC name
London - London Bridge Research Ethics Committee
REC reference
16/LO/0241
Date of REC Opinion
20 May 2016
REC opinion
Further Information Favourable Opinion