drugset / Trial / NCT02363946
A Study of ARC-AAT in Healthy Volunteer Subjects and Patients With Alpha-1 Antitrypsin Deficiency (AATD)
RandomizedParallel-groupTriple-blindTreatment
Summary
The purpose of the study is to determine the safety and tolerability of escalating doses of ARC-AAT and to evaluate the pharmacokinetics of ARC-AAT and the effect of ARC-AAT on circulating levels of alpha-1 antitrypsin (AAT). The study will consist of two parts, Part A (conducted in healthy volunteers) and Part B (conducted in AATD patients) at up to 9 escalating dose levels with 6 participants per dose level.
Timeline
- Start
- 2015-02
- Primary completion
- 2016-11
- Completion
- 2016-11
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | ARC-AAT | Small interfering RNA (siRNA) | 0.38 mg/kg | Intravenous |
| Subject | ARC-AAT | Small interfering RNA (siRNA) | 1 mg/kg | Intravenous |
| Subject | ARC-AAT | Small interfering RNA (siRNA) | 2 mg/kg | Intravenous |
| Subject | ARC-AAT | Small interfering RNA (siRNA) | 3 mg/kg | Intravenous |
| Subject | ARC-AAT | Small interfering RNA (siRNA) | 4 mg/kg | Intravenous |
| Subject | ARC-AAT | Small interfering RNA (siRNA) | 5 mg/kg | Intravenous |
| Subject | ARC-AAT | Small interfering RNA (siRNA) | 6 mg/kg | Intravenous |
| Subject | ARC-AAT | Small interfering RNA (siRNA) | 7 mg/kg | Intravenous |
| Subject | ARC-AAT | Small interfering RNA (siRNA) | 8 mg/kg | Intravenous |
| Background | Diphenhydramine | Unknown | 50 mg | Oral |