Transthyretin-mediated Amyloidosis (ATTR Amyloidosis)
Conditions
Keywords
TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Familial Amyloid Polyneuropathies, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related, Familial Amyloidosis, RNAi therapeutic
Brief summary
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-TTRSC02 in healthy subjects.
Interventions
DRUGALN-TTRSC02
Ascending doses of ALN-TTRSC02 by subcutaneous (SC) injection
DRUGSterile Normal Saline (0.9% NaCl)
Calculated volume to match active comparator
Sponsors
Alnylam Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Subject)
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
Yes
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety of ALN-TTRSC02, evaluated by the proportion of subjects experiencing adverse events (AEs) | Day 1 through to Day 314 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Profile of pharmacokinetics (PK) of ALN-TTRSC02 | Day 1 through to Day 3 | Cmax |
| Effect of ALN-TTRSC02 on serum TTR levels as measured by reduction from baseline in serum TTR | Day 1 through to Day 314 | — |
| Effect of ALN-TTRSC02 on serum Vitamin A levels as measured by reduction from baseline in serum Vitamin A | Screening through to Day 314 | — |
Countries
United Kingdom
Outcome results
None listed