Alpha-1 Antitrypsin DeficiencyPhase 2NCT02900183
Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels
A Phase 2 study testing ARC-AAT Injection in people with Alpha-1 Antitrypsin Deficiency, run by Arrowhead Pharmaceuticals at 4 sites in 4 countries.
Trial summary
Patients with Alpha-1 Antitrypsin Deficiency (AATD) will be enrolled to receive multiple doses of ARC-AAT Injection. All subjects will require a pre-dose biopsy and a biopsy post last dose. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Alpha-1 Antitrypsin Deficiency
- What is being tested?
- ARC-AAT Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 4 sitesin 4 countries
- Study dates
- Oct 2016to Nov 2016
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 14Show fewer
- 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
Trial locations and contacts
4 sites in 4 countries.
4 sites in 4 countries
About this study
Official title: An Open-Label, Multi-dose, Phase 2 Study to Determine the Safety, Tolerability and Effect on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels of ARC-AAT as Evidenced by Changes in Liver Biopsy in Patients With Alpha-1 Antitrypsin Deficiency (AATD)
Patients with Alpha-1 Antitrypsin Deficiency (AATD) will be enrolled to receive multiple doses of ARC-AAT Injection. All subjects will require a pre-dose biopsy and a biopsy post last dose.
Read the detailed description
This is a multi-center, multi-dose study to evaluate the safety, tolerability and effect on intrahepatic and circulating Alpha-1 Antitrypsin (AAT) levels of ARC-AAT Injection, administered intravenously. Participants who have signed an Institutional Review Board/Ethics Committee (IRB/EC) approved informed consent form and met all of the protocol eligibility criteria will receive multiple intravenous doses (7 total doses administered every 28 days) of ARC-AAT Injection. Patients previously, currently or never receiving AAT augmentation therapy are acceptable for enrollment. All subjects will require a pre-dose biopsy and a post-dose biopsy completed after the last dose or at early termination as applicable. For each participant the duration of the study clinic visits is approximately 49 weeks, from the beginning of the Screening period to the Day 287 End-of-Study (EOS) visit.
The following evaluations will be performed at regular intervals throughout the study: medical history, physical examinations, bee venom allergy blood test, vital sign measurements (blood pressure, temperature, heart rate, respiratory rate), weight, adverse events monitoring, electrocardiograms (ECGs), pregnancy tests (females), concurrent medication evaluation, pulmonary function testing, clinical laboratory tests including hematology, coagulation, chemistry, FibroTest, anti-drug antibodies, drug screens, serum AAT levels, liver biopsy, FibroScan, and urinalysis.
- Conditions
- Alpha-1 Antitrypsin Deficiency
- Being tested
- ARC-AAT Injection
- Medical subject headings
- alpha 1-Antitrypsin Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ARC-AAT InjectionDrug
RNA interference (RNAi)-based, liver-targeted therapeutic
| Study group | Role | What they receive |
|---|---|---|
| ARC-AAT Injection | Experimental | Intravenous administration of ARC-AAT Injection (4 mg/kg or 6 mg/kg) every 28 days for a total of 7 doses Receives: Drug: ARC-AAT Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and frequency of adverse events as a measure of safety and tolerability of multiple doses of ARC-AAT Injection
Measured: Baseline through Day 287 End-of Study Visit
Secondary outcomes (1)
Change from baseline in circulating serum levels of alpha-1 antitrypsin
Measured: Baseline through Day 287 End-of Study Visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Sep 2016 (estimated)
- Study startOct 2016 (estimated)
- Primary completionNov 2016
- Study completionNov 2016
Why it stopped: Company decision to not initiate trial
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alpha-1 Antitrypsin DeficiencyStudies of the same condition, open and under way.
- More trials run by Arrowhead PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Arrowhead Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02900183
- Sponsor's protocol ID
- ARCAAT2001
- Other IDs
- 2016-000917-59, 2016
- Status last verified
- Dec 2025
- First submitted
- 9 Sep 2016
- Last updated
- 18 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Multi-dose, Phase 2 Study to Determine the Safety, Tolerability and Effect on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels of ARC-AAT as Evidenced by Changes in Liver Biopsy in Patients With Alpha-1 Antitrypsin Deficiency (AATD)
- ClinicalTrials.gov ID
- NCT02900183
- Acronym / sponsor ID
- ARCAAT2001
- Conditions
- Alpha-1 Antitrypsin Deficiency
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Company decision to not initiate trial
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Arrowhead PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Oct 2016 (estimated)
- Primary completion
- Nov 2016 (actual)
- Study completion
- Nov 2016 (actual)
- First posted
- 14 Sep 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Patients with Alpha-1 Antitrypsin Deficiency (AATD) will be enrolled to receive multiple doses of ARC-AAT Injection. All subjects will require a pre-dose biopsy and a biopsy post last dose.
Detailed description
This is a multi-center, multi-dose study to evaluate the safety, tolerability and effect on intrahepatic and circulating Alpha-1 Antitrypsin (AAT) levels of ARC-AAT Injection, administered intravenously. Participants who have signed an Institutional Review Board/Ethics Committee (IRB/EC) approved informed consent form and met all of the protocol eligibility criteria will receive multiple intravenous doses (7 total doses administered every 28 days) of ARC-AAT Injection. Patients previously, currently or never receiving AAT augmentation therapy are acceptable for enrollment. All subjects will require a pre-dose biopsy and a post-dose biopsy completed after the last dose or at early termination as applicable. For each participant the duration of the study clinic visits is approximately 49 weeks, from the beginning of the Screening period to the Day 287 End-of-Study (EOS) visit.
The following evaluations will be performed at regular intervals throughout the study: medical history, physical examinations, bee venom allergy blood test, vital sign measurements (blood pressure, temperature, heart rate, respiratory rate), weight, adverse events monitoring, electrocardiograms (ECGs), pregnancy tests (females), concurrent medication evaluation, pulmonary function testing, clinical laboratory tests including hematology, coagulation, chemistry, FibroTest, anti-drug antibodies, drug screens, serum AAT levels, liver biopsy, FibroScan, and urinalysis.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ARC-AAT Injection | Experimental | Drug: ARC-AAT InjectionIntravenous administration of ARC-AAT Injection (4 mg/kg or 6 mg/kg) every 28 days for a total of 7 doses |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (14 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ARC-AAT InjectionRNA interference (RNAi)-based, liver-targeted therapeutic | Drug | ARC-AAT Injection |
Primary outcome
Incidence and frequency of adverse events as a measure of safety and tolerability of multiple doses of ARC-AAT Injection
Time frame: Baseline through Day 287 End-of Study Visit
1 secondary outcome
Change from baseline in circulating serum levels of alpha-1 antitrypsin
Time frame: Baseline through Day 287 End-of Study Visit
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Dec 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
4 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ARC-AAT Injection in people with Alpha-1 Antitrypsin Deficiency, run by Arrowhead Pharmaceuticals at 4 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 18 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Canada, Ireland, Italy, Sweden. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and frequency of adverse events as a measure of safety and tolerability of multiple doses of ARC-AAT Injection (time frame: Baseline through Day 287 End-of Study Visit). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Dec 2025; AzTrials last refreshed its copy on 6 Nov 2026.