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Trial record 1 of 1 for:    NCT03946449
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Assessment of Changes in a Novel Histological Activity Scale in Response to ARO-AAT

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ClinicalTrials.gov Identifier: NCT03946449
Recruitment Status : Not yet recruiting
First Posted : May 10, 2019
Last Update Posted : May 10, 2019
Sponsor:
Information provided by (Responsible Party):
Arrowhead Pharmaceuticals

Brief Summary:
The purpose of this study is to evaluate the effect of ARO-AAT Injection (also referred to as ARO-AAT) on a histological liver disease activity scale in participants with AAT-associated liver disease over time. Participants will receive multiple subcutaneous doses of ARO-AAT.

Condition or disease Intervention/treatment Phase
Alpha 1-Antitrypsin Deficiency Drug: ARO-AAT Injection Phase 2

Detailed Description:
Approximately 12 participants will be enrolled to receive multiple subcutaneous injections of ARO-AAT. All eligible participants will require a pre-dose biopsy completed as part of the study within the screening window. All participants will undergo an End of Study (EOS) biopsy. Treated participants will be offered the opportunity to continue treatment in an open label extension.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Pilot Open Label, Multi-dose, Phase 2 Study to Assess Changes in a Novel Histological Activity Scale in Response to ARO-AAT in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)
Estimated Study Start Date : July 15, 2019
Estimated Primary Completion Date : July 15, 2021
Estimated Study Completion Date : July 15, 2021


Arm Intervention/treatment
Experimental: ARO-AAT Cohort 1

Administered on Day 1, Weeks 4 and 16 for a minimum of 3 doses.

Extension Cohort (optional enrollment): Administered every 12 weeks for 4 additional doses.

Drug: ARO-AAT Injection
solution for subcutaneous (sc) injection

Experimental: ARO-AAT Cohort 2

Administered on Day 1, Weeks 4, 16, 28 and 40 for a minimum of 5 doses.

Extension Cohort (optional enrollment): Administered every 12 weeks for 4 additional doses.

Drug: ARO-AAT Injection
solution for subcutaneous (sc) injection




Primary Outcome Measures :
  1. Change From Baseline Over Time in a Histological Liver Disease Activity Scale [ Time Frame: Baseline and Week 24 (Cohort 1) or Week 48 (Cohort 2); Extension Cohort: Baseline and Extension Week 44 ]

Secondary Outcome Measures :
  1. Change From Baseline Over Time in Ishak Fibrosis Score [ Time Frame: Baseline and Week 24 (Cohort 1) or Week 48 (Cohort 2); Extension Cohort: Baseline and Extension Week 44 ]
  2. Percentage Change From Baseline In Soluble Liver Z-Alpha-1 Antitrypsin (Z-AAT), Insoluble Liver Z-AAT Levels [ Time Frame: Baseline and Week 24 (Cohort 1) or Week 48 (Cohort 2); Extension Cohort: Baseline and Extension Week 44 ]
  3. Percentage Change From Baseline in Serum AAT Levels [ Time Frame: Baseline and Week 24 (Cohort 1) or Week 48 (Cohort 2); Extension Cohort: Baseline and Extension Week 44 ]
  4. Change From Baseline Over Time in Hepatic SERPINA1 mRNA Expression (if sufficient sample is available) [ Time Frame: Baseline and Week 24 (Cohort 1) or Week 48 (Cohort 2); Extension Cohort: Baseline and Extension Week 44 ]
  5. Changes From Baseline Over Time in Liver Fibrosis Gene Expression (if scientifically feasible and sufficient sample is available) [ Time Frame: Baseline and Week 24 (Cohort 1) or Week 48 (Cohort 2); Extension Cohort: Baseline and Extension Week 44 ]
  6. Number of Participants with Adverse Events (AEs) Possibly or Probably Related to Treatment [ Time Frame: Up to Week 24 (Cohort 1) or Week 48 (Cohort 2); Extension Cohort: up to Extension Week 44 ]


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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of AATD
  • Women of childbearing potential must have a negative pregnancy test, cannot be breast feeding, and must be willing to use contraception
  • Willing to provide written informed consent and to comply with study requirements
  • Non-smoker for at least 1 year
  • No abnormal finding of clinical relevance at screening

Exclusion Criteria:

  • Clinically significant health concerns other than AATD
  • Previous diagnosis or diagnosis at Screening of definitive liver cirrhosis
  • 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 involving therapeutic intervention
  • Use of illicit drugs within 1 year prior to Screening

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): NCT03946449


Contacts
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Contact: Medical Monitor 626-304-3400 medicalmonitor@arrowheadpharma.com

Sponsors and Collaborators
Arrowhead Pharmaceuticals

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Responsible Party: Arrowhead Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03946449     History of Changes
Other Study ID Numbers: AROAAT2002
2019-000068-86 ( EudraCT Number )
First Posted: May 10, 2019    Key Record Dates
Last Update Posted: May 10, 2019
Last Verified: May 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