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A Study of an Investigational Drug, ALN-AAT, in Healthy Adult Subjects and Patients With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02503683
First Posted: July 21, 2015
Last Update Posted: June 5, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Alnylam Pharmaceuticals
July 16, 2015
July 21, 2015
June 5, 2017
July 2015
December 2015   (Final data collection date for primary outcome measure)
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 [ Time Frame: Part A (SAD phase): through day 70; Part B (MAD) phase: through Day 154; Part C: through Day 224 ]
Same as current
Complete list of historical versions of study NCT02503683 on ClinicalTrials.gov Archive Site
  • Profile of Pharmacokinetics (PK) of ALN-AAT [ Time Frame: 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 [ Time Frame: Part A (SAD) phase: through day 70; Part B (MAD) phase: through Day 154; Part C: through Day 224 ]
Same as current
Not Provided
Not Provided
 
A Study of an Investigational Drug, ALN-AAT, in Healthy Adult Subjects and Patients With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease
A Phase 1/2, Randomized, Single-blind, Placebo-Controlled, Single Ascending Dose and Multiple Dose, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Subcutaneously Administered ALN-AAT in Healthy Adult Subjects and Patients With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease
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.
Not Provided
Interventional
Phase 1
Phase 2
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Participant)
Primary Purpose: Treatment
Antitrypsin Deficiency Liver Disease
  • Drug: ALN-AAT
    Single or multiple doses of ALN-AAT by subcutaneous (sc) injection
  • Drug: Sterile Normal Saline (0.9% NaCl)
    calculated volume to match active comparator
  • Active Comparator: ALN-AAT
    Intervention: Drug: ALN-AAT
  • Placebo Comparator: Sterile Normal Saline (0.9% NaCl)
    Intervention: Drug: Sterile Normal Saline (0.9% NaCl)
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
66
November 2017
December 2015   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
18 Years to 65 Years   (Adult)
Yes
Contact information is only displayed when the study is recruiting subjects
United Kingdom
 
 
NCT02503683
ALN-AAT-001
No
Not Provided
Not Provided
Alnylam Pharmaceuticals
Alnylam Pharmaceuticals
Not Provided
Study Director: Patrick Haslett, MD Alnylam Pharmaceuticals
Alnylam Pharmaceuticals
June 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP