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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
  Purpose
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.

Condition Intervention Phase
Antitrypsin Deficiency Liver Disease Drug: ALN-AAT Drug: Sterile Normal Saline (0.9% NaCl) Phase 1 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Participant)
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:


Further study details as provided by Alnylam Pharmaceuticals:

Primary Outcome Measures:
  • 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 ]

Secondary Outcome Measures:
  • 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 ]

Estimated Enrollment: 66
Study Start Date: July 2015
Estimated Study Completion Date: November 2017
Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: ALN-AAT Drug: ALN-AAT
Single or multiple doses of ALN-AAT by subcutaneous (sc) injection
Placebo Comparator: Sterile Normal Saline (0.9% NaCl) Drug: Sterile Normal Saline (0.9% NaCl)
calculated volume to match active comparator

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02503683


Locations
United Kingdom
Richmond Pharmacology, Ltd.
London, United Kingdom
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
Study Director: Patrick Haslett, MD Alnylam Pharmaceuticals
  More Information

Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02503683     History of Changes
Other Study ID Numbers: ALN-AAT-001
First Submitted: July 16, 2015
First Posted: July 21, 2015
Last Update Posted: June 5, 2017
Last Verified: June 2017

Keywords provided by Alnylam Pharmaceuticals:
RNAi therapeutic
AAT
Alpha-1 Antitrypsin Deficiency

Additional relevant MeSH terms:
Liver Diseases
Alpha 1-Antitrypsin Deficiency
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Subcutaneous Emphysema
Emphysema
Pathologic Processes
Alpha 1-Antitrypsin
Protein C Inhibitor
Trypsin Inhibitors
Serine Proteinase Inhibitors
Protease Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action