Transthyretin-mediated Amyloidosis (ATTR Amyloidosis) Clinical Trial
Official 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
| Verified date | September 2018 |
| Source | Alnylam Pharmaceuticals |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-TTRSC02 in healthy subjects.
| Status | Completed |
| Enrollment | 80 |
| Est. completion date | January 12, 2018 |
| Est. primary completion date | January 12, 2018 |
| Accepts healthy volunteers | Accepts Healthy Volunteers |
| Gender | All |
| Age group | 18 Years to 65 Years |
| Eligibility |
Inclusion Criteria: - Male and female subjects, age 18 to 65 years, inclusive. - Body mass index (BMI) =18.0 kg/m2 and =30 kg/m2 assessed at Screening. - No clinically significant health concerns, as determined by medical history and physical examination, in the opinion of the Investigator. - Women of child bearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception, willing and able to comply with the study requirements and to provide written informed consent. - For Japanese cohorts, subjects of Japanese descent are defined as people carrying a Japanese passport, descendants of 4 Japanese grandparents, and have not been outside Japan for more than 5 years. Exclusion Criteria: - Clinically relevant history or presence of respiratory, gastrointestinal, renal, cardiovascular, hepatic, hematological, lymphatic, neurological, psychiatric, musculoskeletal, genitourinary, immunological, and other inflammatory diseases, or dermatological or connective tissue diseases or disorders. - Active serious mental illness or psychiatric disorder. - Clinically significant ECG abnormalities. Abnormal for AST/ALT and any other clinical safety laboratory result considered clinically significant. - Known history of allergic reaction to an oligonucleotide or GalNAc. - History of intolerance to subcutaneous injection. |
| Country | Name | City | State |
|---|---|---|---|
| United Kingdom | Clinical Trial Site | London |
| Lead Sponsor | Collaborator |
|---|---|
| Alnylam Pharmaceuticals |
United Kingdom,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Safety of ALN-TTRSC02, evaluated by the proportion of subjects experiencing adverse events (AEs) | Day 1 through to Day 314 | ||
| Secondary | Profile of pharmacokinetics (PK) of ALN-TTRSC02 | Cmax | Day 1 through to Day 3 | |
| Secondary | Profile of pharmacokinetics (PK) of ALN-TTRSC02 | tmax | Day 1 through to Day 3 | |
| Secondary | Profile of pharmacokinetics (PK) of ALN-TTRSC02 | AUC | Day 1 through to Day 3 | |
| Secondary | Profile of pharmacokinetics (PK) of ALN-TTRSC02 | t½ | Day 1 through to Day 3 | |
| Secondary | Profile of pharmacokinetics (PK) of ALN-TTRSC02 | CL/F | Day 1 through to Day 3 | |
| Secondary | Profile of pharmacokinetics (PK) of ALN-TTRSC02 | Vss/F | Day 1 through to Day 3 | |
| Secondary | Profile of pharmacokinetics (PK) of ALN-TTRSC02 | Vz/F | Day 1 through to Day 3 | |
| Secondary | Profile of pharmacokinetics (PK) of ALN-TTRSC02 | fe | Day 1 through to Day 314 | |
| Secondary | Profile of pharmacokinetics (PK) of ALN-TTRSC02 | CLR | Day 1 through to Day 314 | |
| Secondary | Effect of ALN-TTRSC02 on serum TTR levels as measured by reduction from baseline in serum TTR | Day 1 through to Day 314 | ||
| Secondary | Effect of ALN-TTRSC02 on serum Vitamin A levels as measured by reduction from baseline in serum Vitamin A | Screening through to Day 314 |