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Trial record 3 of 3 for:    ARO-AAT Injection | AAT liver disease

Study of ARO-AAT in Normal Adult Volunteers

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.
 
ClinicalTrials.gov Identifier: NCT03362242
Recruitment Status : Completed
First Posted : December 5, 2017
Last Update Posted : August 18, 2020
Sponsor:
Information provided by (Responsible Party):
Arrowhead Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE November 30, 2017
First Posted Date  ICMJE December 5, 2017
Last Update Posted Date August 18, 2020
Actual Study Start Date  ICMJE March 12, 2018
Actual Primary Completion Date October 23, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 30, 2017)
Number of Participants With Adverse Events (AEs) Possibly or Probably Related to Treatment [ Time Frame: Part A (single-ascending dose [SAD] phase): up to 29 (+/- 2) days post-dose; Part B (multiple-ascending dose [MAD] phase): up to 113 (+/- 2) days post-dose ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 4, 2017)
  • Pharmacokinetics (PK) of ARO-AAT: Maximum Observed Plasma Concentration (Cmax) [ Time Frame: Part A (single-ascending dose [SAD] phase): up to 48 hours post-dose; Part B (multiple-ascending dose [MAD] phase): up to 48 hours post-dose ]
  • PK of ARO-AAT: Time to Maximum Plasma Concentration (Tmax) [ Time Frame: Part A (SAD phase): up to 48 hours post-dose; Part B (MAD phase): up to 48 hours post-dose ]
  • PK of ARO-AAT: Terminal Elimination Half-Life (t½) [ Time Frame: Part A (SAD phase): up to 48 hours post-dose; Part B (MAD phase): up to 48 hours post-dose ]
  • PK of ARO-AAT: Area Under the Plasma Concentration Versus Time Curve From Zero to 24 Hours (AUC0-24) [ Time Frame: Part A (SAD phase): up to 48 hours post-dose; Part B (MAD phase): up to 48 hours post-dose ]
  • PK of ARO-AAT: Area Under the Plasma Concentration Versus Time Curve From Zero to Infinity (AUCinf) [ Time Frame: Part A (SAD phase): up to 48 hours post-dose; Part B (MAD phase): up to 48 hours post-dose ]
  • Percent Change in Serum Alpha-1 Antitrypsin (AAT) Levels From Day 1 Pre-Dose Baseline to Nadir [ Time Frame: Part A (SAD phase): up to 29 (+/- 2) days; Part B (MAD phase): up to 113 (+/- 2) days ]
  • Duration of Response of Serum AAT levels From Nadir Back to Above 20% of Baseline or Above 90 mg/dL [ Time Frame: Part A (SAD phase): up to 29 (+/- 2) days; Part B (MAD phase): up to 113 (+/- 2) days ]
Original Secondary Outcome Measures  ICMJE
 (submitted: November 30, 2017)
  • Pharmacokinetics (PK) of ARO-AAT: Maximum Observed Plasma Concentration (Cmax) [ Time Frame: Part A (single-ascending dose [SAD] phase): up to 48 hours post-dose; Part B (multiple-ascending dose [MAD] phase): up to 48 hours post-dose ]
  • PK of ARO-AAT: Time to Maximum Plasma Concentration (Tmax) [ Time Frame: Part A (SAD phase): up to 48 hours post-dose; Part B (MAD phase): up to 48 hours post-dose ]
  • PK of ARO-AAT: Terminal Elimination Half-Life (t½) [ Time Frame: Part A (SAD phase): up to 48 hours post-dose; Part B (MAD phase): up to 48 hours post-dose ]
  • PK of ARO-AAT: Area Under the Plasma Concentration Versus Time Curve From Zero to 24 Hours (AUC0-24) [ Time Frame: Part A (SAD phase): up to 48 hours post-dose; Part B (MAD phase): up to 48 hours post-dose ]
  • PK of ARO-AAT: Area Under the Plasma Concentration Versus Time Curve From Zero to Infinity (AUCinf) [ Time Frame: Part A (SAD phase): up to 48 hours post-dose; Part B (MAD phase): up to 48 hours post-dose ]
  • Percent Change in Serum Alpha-1 Antitrypsin (AAT) Levels From Day 1 Pre-Dose Baseline to Nadir [ Time Frame: Part A (SAD phase): Part A (SAD phase): up to 29 (+/- 2) days; Part B (MAD phase): up to 113 (+/- 2) days ]
  • Duration of Response of Serum AAT levels From Nadir Back to Above 20% of Baseline or Above 90 mg/dL [ Time Frame: Part A (SAD phase): up to 29 (+/- 2) days; Part B (MAD phase): up to 113 (+/- 2) days ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study of ARO-AAT in Normal Adult Volunteers
Official Title  ICMJE A Phase 1 Single and Multiple Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Effect of ARO-AAT on Serum Alpha-1 Antitrypsin Levels in Normal Adult Volunteers
Brief Summary The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple-ascending doses of ARO-AAT in healthy adult volunteers.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Alpha 1-Antitrypsin Deficiency
Intervention  ICMJE
  • Drug: ARO-AAT Injection
    Single or multiple doses of ARO-AAT by subcutaneous (sc) injections
  • Other: Sterile Normal Saline (0.9% NaCl)
    Calculated volume to match active comparator
Study Arms  ICMJE
  • Active Comparator: ARO-AAT
    Intervention: Drug: ARO-AAT Injection
  • Placebo Comparator: Placebo
    Intervention: Other: Sterile Normal Saline (0.9% NaCl)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: July 9, 2018)
45
Original Estimated Enrollment  ICMJE
 (submitted: November 30, 2017)
40
Actual Study Completion Date  ICMJE March 21, 2020
Actual Primary Completion Date October 23, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Women of child bearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception
  • Willing to provide written informed consent and to comply with study requirements
  • Non-smoker for at least one year
  • Normal lung function
  • No abnormal finding of clinical relevance at Screening
  • Normal AAT level at Screening visit

Exclusion Criteria:

  • Clinically significant health concerns
  • Regular use of alcohol within one month prior to Screening
  • Use of an investigational agent or device within 30 days prior to dosing or current participation in an investigational study
  • Recent use of illicit drugs
  • Use of any drugs or dietary/herbal supplements know to interfere with liver metabolism

NOTE: additional inclusion/exclusion criteria may apply, per protocol

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 55 Years   (Adult)
Accepts Healthy Volunteers  ICMJE Yes
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE New Zealand
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03362242
Other Study ID Numbers  ICMJE AROAAT1001
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Arrowhead Pharmaceuticals
Study Sponsor  ICMJE Arrowhead Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Arrowhead Pharmaceuticals
Verification Date August 2020

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