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Trial record 3 of 128 for:    AAT

Study of ARO-AAT in Normal Adult Volunteers

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ClinicalTrials.gov Identifier: NCT03362242
Recruitment Status : Active, not recruiting
First Posted : December 5, 2017
Last Update Posted : May 2, 2019
Sponsor:
Information provided by (Responsible Party):
Arrowhead Pharmaceuticals

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.

Condition or disease Intervention/treatment Phase
Alpha 1-Antitrypsin Deficiency Drug: ARO-AAT Injection Other: Sterile Normal Saline (0.9% NaCl) Phase 1

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 45 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : March 12, 2018
Actual Primary Completion Date : October 11, 2018
Estimated Study Completion Date : September 30, 2019


Arm Intervention/treatment
Active Comparator: ARO-AAT Drug: ARO-AAT Injection
Single or multiple doses of ARO-AAT by subcutaneous (sc) injections

Placebo Comparator: Placebo Other: Sterile Normal Saline (0.9% NaCl)
Calculated volume to match active comparator




Primary Outcome Measures :
  1. 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 ]

Secondary Outcome Measures :
  1. 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 ]
  2. 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 ]
  3. 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 ]
  4. 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 ]
  5. 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 ]
  6. 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 ]
  7. 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 ]


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Ages Eligible for Study:   18 Years to 55 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

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


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): NCT03362242


Locations
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New Zealand
Auckland Clinical Studies Limited
Grafton, Auckland, New Zealand, 1010
Sponsors and Collaborators
Arrowhead Pharmaceuticals

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Responsible Party: Arrowhead Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03362242     History of Changes
Other Study ID Numbers: AROAAT1001
First Posted: December 5, 2017    Key Record Dates
Last Update Posted: May 2, 2019
Last Verified: April 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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

Additional relevant MeSH terms:
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Alpha 1-Antitrypsin Deficiency
Liver Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Subcutaneous Emphysema
Emphysema
Pathologic Processes