A Study of an Investigational Drug, ALN-AAT, in Healthy Adult Subjects and Patients With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease
Phase 1
Terminated
- Conditions
- Antitrypsin Deficiency Liver Disease
- Interventions
- Drug: Sterile Normal Saline (0.9% NaCl)
- Registration Number
- NCT02503683
- Lead Sponsor
- Alnylam Pharmaceuticals
- Brief Summary
The purpose of this study is to evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Subcutaneously Administered ALN-AAT in Healthy Adult Subjects and Patients with ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 26
Inclusion Criteria
- Adequate complete blood counts, liver and renal function.
- 12-lead electrocardiogram (ECG) within normal limits
- Female subjects must be of non-childbearing potential; e.g. post-menopausal or pre-menopausal with surgical sterilization
- Male subjects agree to use appropriate contraception
- Willing to provide written informed consent and willing to comply with study requirements
- Nonsmokers for at least 5 years before screening
Exclusion Criteria
- Any uncontrolled or serious disease, or any medical or surgical condition, that may interfere with participation in the clinical study and/or put the subject at significant risk
- Received an investigational agent within 90 days before the first dose of study drug or are in follow-up of another clinical study
- Active serious mental illness or psychiatric disorder requiring current pharmacological intervention
- History or evidence of alcohol or drug abuse within 12 months before screening.
- History of intolerance to SC injection
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description ALN-AAT ALN-AAT - Sterile Normal Saline (0.9% NaCl) Sterile Normal Saline (0.9% NaCl) -
- Primary Outcome Measures
Name Time Method The safety of ALN-AAT evaluated by the proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs), and AEs leading to study drug discontinuation Part A (SAD phase): through day 70; Part B (MAD) phase: through Day 154; Part C: through Day 224
- Secondary Outcome Measures
Name Time Method Profile of Pharmacokinetics (PK) of ALN-AAT Part A (SAD) phase: up to 21 days; Part B (MAD) phase: up to 105 days; Part C: up to 161 days Cmax, tmax, AUC, t1/2
The effect of ALN-AAT on serum levels of AAT protein Part A (SAD) phase: through day 70; Part B (MAD) phase: through Day 154; Part C: through Day 224
Trial Locations
- Locations (1)
Richmond Pharmacology, Ltd.
🇬🇧London, United Kingdom