Liver disease drug trial pulled before it even started
NCT ID NCT02900183
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study aimed to test an experimental drug called ARC-AAT in people with Alpha-1 Antitrypsin Deficiency (AATD), a genetic condition that can cause liver and lung damage. The drug was designed to reduce harmful protein buildup in the liver. However, the trial was withdrawn before any participants were enrolled, so no data on safety or effectiveness were collected.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- ARC-AAT (an RNA interference drug)
- What this could lead to
- If it had worked, this could have pointed toward a treatment to reduce harmful protein buildup in the liver for people with Alpha-1 Antitrypsin Deficiency.
- What could go wrong
- This trial was withdrawn before enrolling anyone, so no results are available. The approach was still early (Phase 2) and required invasive liver biopsies, which carry risks.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Expected to start
-
Oct 2016
An estimate. Start dates often move.
- Finished
-
Nov 2016
- Lead sponsor
-
A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 to 75 years
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Male or non-nursing female patients 18-75 years of age, inclusive, at the time of Screening * Previous diagnosis of PiZZ genotype Alpha-1 Antitrypsin Deficiency (AATD) * Non-smoker (not a daily cigarette smoker) for at least three years with current non-smoking status confirmed by urine cotinine at screening. * Highly effective, double barrier contraception (both male and female partners) during the study and for 3 months following the last dose of ARC-AAT Injection * Suitable venous access for blood sampling Exclusion Criteria: * Known diagnosis of hepatic fibrosis from a cause other than AATD * History of poorly controlled autoimmune disease, or any history of autoimmune hepatitis * Human immunodeficiency virus (HIV) infection * Seropositive for Hepatitis B virus (HBV) or Hepatitis C virus (HCV) * Uncontrolled hypertension * History of cardiac rhythm disturbances * Symptomatic heart failure, unstable angina, myocardial infarction, severe cardiovascular disease, transient ischemic attack (TIA) or cerebrovascular accident (CVA) within 6 months prior to study entry * History of malignancy within the last 2 years except for adequately treated basal cell carcinoma, squamous cell skin cancer, superficial bladder tumors, or in situ cervical cancer * History of major surgery within 1 month of Screening * Regular use of alcohol within one month prior to the Screening visit * Use of illicit drugs (such as cocaine, phencyclidine \[PCP\] and crack) within 1 year prior to Screening or positive urine drug screen at Screening * Use of an investigational agent or device within 30 days prior to dosing or current participation in an investigational study involving a therapeutic intervention * Any clinically significant history/presence of an uncontrolled systemic disease * Blood donation (≥500 mL) within 7 days prior to study treatment administration
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Research Site 1
Toronto, Ontario, M5T3A9, Canada
-
Research Site 2
Dublin, Ireland
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Research Site 3
Pavia, 27100, Italy
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Research Site 4
Malmö, SE-205 02, Sweden
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Could a simple shot replace IV drips for Alpha-1 patients?
- Hidden liver harm: study tracks silent damage in genetic disorder
- Promising liver drug trial halted early: what it means for patients
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