Trial to Evaluate Safety, Tolerability, and Parmacokinetics of ALN-TTR02 in Healthy Volunteer Subjects
Phase 1
Completed
- Conditions
- TTR-mediated Amyloidosis
- Interventions
- Drug: Sterile Normal Saline (0.9% NaCl)
- Registration Number
- NCT01559077
- Lead Sponsor
- Alnylam Pharmaceuticals
- Brief Summary
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-TTR02 in healthy volunteer subjects.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 17
Inclusion Criteria
- Body mass index must be between 18.0 kg/m2 and ≤ 31.5 kg/m2;
- Non-smokers for at least 3 months preceding screening;
- Females subjects must be of non-childbearing potential e.g., post-menopausal or pre-menopausal with surgical sterilization;
- Males agree to use appropriate contraception;
- Medical history must be verified by either a personal physician or medical practitioner as appropriate;
- Willing to give written informed consent and are willing to comply with the study requirements.
Exclusion Criteria
- Known hepatitis B surface antigen (HBsAg), hepatitis B virus (HBV), hepatitis C virus (HCV) or human immunodeficiency virus (HIV) infection
- Multiple drug allergies or know sensitivity to oligonucleotide
- History of drug abuse and/or alcohol abuse
- Receiving an investigational agent within 3 months prior to study drug administration
- Subjects with safety laboratory test results deemed clinical significant by the Investigator;
- Received prescription drugs within 4 weeks of first dosing
- Subjects who have donated more than 500 mL of blood within the 3 months prior to ALN-PCS02 or placebo administration;
- Subjects who have used prescription drugs within 4 weeks of first dosing
- Considered unfit for the study by the Principal Investigator
- Employee or family member of the sponsor or the clinical study site personnel
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description ALN-TTR02 ALN-TTR02 - Sterile Normal Saline (0.9% NaCl) Sterile Normal Saline (0.9% NaCl) -
- Primary Outcome Measures
Name Time Method The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation. Up to 28 days
- Secondary Outcome Measures
Name Time Method Pharmacokinetics (PK) of ALN-TTR02 (Cmax, tmax, t1/2, AUC0-last, CL). Up to 180 days Effect of ALN-TTR02 on transthyretin (TTR), vitamin A, and retinol binding protein (RBP) (Determination of % Lowering of TTR, vitamin A and RBP to pretreatment/Baseline Levels) Up to 56 days
Trial Locations
- Locations (1)
Clinical Site
🇬🇧London, United Kingdom