C3 GlomerulopathyPhase 1/2NCT05083364
Study of ARO-C3 in Adult Healthy Volunteers and Patients With Complement Mediated Renal Disease
A Phase 1/2 study testing ARO-C3 in people with C3 Glomerulopathy and IgA Nephropathy, run by Arrowhead Pharmaceuticals at 16 sites in 7 countries.
Trial summary
The purpose of AROC3-1001 is to evaluate the safety, tolerability, pharmacokinetics and/or pharmacodynamics in adult healthy volunteers (HVs) and in adult patients with complement-mediated renal disease (C3 Glomerulopathy [C3G] and IgA Nephropathy [IgAN]). Read more about this study
- Who is it for?
- Ages 18 to 70 yearsHealthy volunteers can take part
- Conditions the study is for
- C3 Glomerulopathy, IgA Nephropathy
- What is being tested?
- ARO-C3
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 62 participants
- Where
- 16 sitesin 7 countries
- Study dates
- 1 Feb 2022to 10 Sep 2025
- Phase
- Phase 1/2
- 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 70 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Willing to provide written informed consent and to comply with study requirements
- Female participants must be non-pregnant/non-lactating
- Healthy volunteers must be willing to be vaccinated with a meningococcal and pneumococcal vaccine. C3G and IgAN participants must have been vaccinated or willing to undergo vaccination
- All participants must be willing to be vaccinated or have a history of vaccination for Haemophilus influenzae
- Body Mass Index (BMI) between 18.0 and 35.0 kg/m2
- 12-lead electrocardiogram (ECG) at Screening with no abnormalities that may compromise participant's safety at discretion of investigator
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- Participants of childbearing potential must use highly effective contraception during the study and for at least 12 weeks following the end of the study or last dose of study drug, whichever is later. Males must not donate sperm during the study and for at least 12 weeks following the end of the study or last dose of study drug, whichever is later.
- No abnormal finding of clinical relevance at the Screening evaluation that, in the opinion of the investigator, could adversely impact participant safety or study results
- Diagnosis of C3G or IgAN
- Clinical evidence of ongoing disease based on significant proteinuria
- Estimated glomerular filtration rate ≥30 mL/Min/1.73 m2 at Screening and currently not on dialysis
- Must be on a maximally recommended or tolerated dose of an angiotensin converting enzyme inhibitor (ACEI) or angiotensin receptor blocker (ARB)
Who cannot join
- Seropositive for human immunodeficiency virus (HIV) infection,hepatitis B virus, or hepatitis C virus
- History of recurrent or chronic infections
- Uncontrolled hypertension
- Regular use of alcohol within 30 days prior to Screening
- Use of illicit drugs within 1 year prior to Screening or positive urine drug screen at Screening
- History of meningococcal infection
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- History of asplenia or splenectomy
- Known contraindication or history of anaphylactic reaction to any vaccine or vaccine component or prophylactic antibiotics planned for use in the study
- Any medical or surgical condition that, in the opinion of the investigator, would expose the participant to a significant safety risk or compromise the results of the study
- Note: Additional Inclusion/Exclusion criteria may apply per protocol
Trial locations and contacts
16 sites in 7 countries.
16 sites in 7 countries
South Korea6 sites
Research Site 3
Daegu, 42601
Research Site 1
Gamcheon, Busan, 49267
Research Site 4
Goyang-si, Gyeonggi-do, 10444
Research Site 2
Haeundae, Busan, 48108
Research Site 6
Soeul, 03722
Research Site 8
Soeul, 05030
Australia3 sites
Research Site 1
Camperdown, New South Wales, 2050
Research Site 2
Clayton, Victoria, 3168
Research Site 3
Concord, New South Wales, 2139
Thailand2 sites
Research Site 2
Bangkok, 10400
Research Site 3
Chiang Mai, 50200
About this study
Official title: A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and/or Pharmacodynamics of ARO-C3 in Adult Healthy Volunteers and in Adult Patients With Complement-Mediated Renal Disease
The purpose of AROC3-1001 is to evaluate the safety, tolerability, pharmacokinetics and/or pharmacodynamics in adult healthy volunteers (HVs) and in adult patients with complement-mediated renal disease (C3 Glomerulopathy [C3G] and IgA Nephropathy [IgAN]). In Part 1 of the study, HVs will receive either one or two doses of ARO-C3 or placebo. In Part 2 of the study, adult patients with C3G/IgAN will receive 3 open-label doses of ARO-C3. Dose levels in Part 2 will be determined based on cumulative safety and pharmacodynamic data from Part 1.
- Conditions
- C3 Glomerulopathy, IgA Nephropathy
- Being tested
- ARO-C3, Placebo
- Medical subject headings
- Glomerulonephritis, IGA
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ARO-C3Drug
ARO-C3 for sc injection
PlaceboDrug
sterile normal saline (0.9% NaCl) for sc injection
| Study group | Role | What they receive |
|---|---|---|
| ARO-C3 (Healthy Volunteers) | Experimental | 1 or 2 doses of ARO-C3 by subcutaneous (sc) injection Receives: Drug: ARO-C3 |
| Placebo (Healthy Volunteers) | Placebo comparator | placebo calculated volume to match active treatment by sc injection Receives: Drug: Placebo |
| ARO-C3 (Adult Patients with C3G or IgAN) | Experimental | 3 doses of ARO-C3 by sc injection Receives: Drug: ARO-C3 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Adverse Events (AEs) and/or Serious Adverse Events (SAEs) at Day 169
Measured: up to day 169 (End of Study [EOS])
Secondary outcomes (7)
Pharmacokinetics (PK) of ARO-C3: Maximum Observed Plasma Concentration (Cmax)
Measured: up to 48 hours post-dose
PK of ARO-C3: Area under the Plasma Concentration Versus Time Curve from Zero to 24Hours (AUC0-24)
Measured: up to 48 hours post-dose
PK of ARO-C3: Area Under the Plasma Versus Time Concentration Curve from Zero to the Last Quantifiable Plasma Concentration (AUClast)
Measured: up to 48 hours post-dose
PK of ARO-C3: Area Under the Plasma Concentration Versus Time Curve from Zero Extrapolated to Infinity (AUCinf) PK of ARO-C3:
Measured: up to 48 hours post-dose
PK of ARO-C3: Terminal Elimination Half-Life (t1/2)
Measured: up to 48 hours post-dose
PK of ARO-C3: Apparent Total Body Clearance of ARO-C3 from Plasma (CL)
Measured: up to 48 hours post-dose
PK of ARO-C3: Volume of Distribution (Vz/F)
Measured: up to 48 hours post-dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Oct 2021
- Study start1 Feb 2022
- Primary completion10 Sep 2025
- Study completion10 Sep 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for C3 GlomerulopathyStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 62 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
- NCT05083364
- Sponsor's protocol ID
- AROC3-1001
- Other IDs
- 2023-506690-36-00, 2023
- Status last verified
- May 2026
- First submitted
- 8 Oct 2021
- Last updated
- 22 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Tunnicliffe DJ, Reid S, Craig JC, Samuels JA, Molony DA, Strippoli GF. Non-immunosuppressive treatment for IgA nephropathy. Cochrane Database Syst Rev. 2024 Feb 1;2(2):CD003962. doi: 10.1002/14651858.CD003962.pub3.
PubMed 38299639
Study at a glance
From the registry record, last updated by the sponsor on 22 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and/or Pharmacodynamics of ARO-C3 in Adult Healthy Volunteers and in Adult Patients With Complement-Mediated Renal Disease
- ClinicalTrials.gov ID
- NCT05083364
- Acronym / sponsor ID
- AROC3-1001
- Conditions
- C3 Glomerulopathy, IgA Nephropathy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 62 participants (actual)
- Lead sponsor
- Arrowhead PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 1 Feb 2022 (actual)
- Primary completion
- 10 Sep 2025 (actual)
- Study completion
- 10 Sep 2025 (actual)
- First posted
- 19 Oct 2021 (actual)
- 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.
The purpose of AROC3-1001 is to evaluate the safety, tolerability, pharmacokinetics and/or pharmacodynamics in adult healthy volunteers (HVs) and in adult patients with complement-mediated renal disease (C3 Glomerulopathy [C3G] and IgA Nephropathy [IgAN]). In Part 1 of the study, HVs will receive either one or two doses of ARO-C3 or placebo. In Part 2 of the study, adult patients with C3G/IgAN will receive 3 open-label doses of ARO-C3. Dose levels in Part 2 will be determined based on cumulative safety and pharmacodynamic data from Part 1.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| ARO-C3 (Healthy Volunteers) | Experimental | Drug: ARO-C31 or 2 doses of ARO-C3 by subcutaneous (sc) injection |
| Placebo (Healthy Volunteers) | Placebo comparator | Drug: Placeboplacebo calculated volume to match active treatment by sc injection |
| ARO-C3 (Adult Patients with C3G or IgAN) | Experimental | Drug: ARO-C33 doses of ARO-C3 by sc injection |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Willing to provide written informed consent and to comply with study requirements
- Female participants must be non-pregnant/non-lactating
- Healthy volunteers must be willing to be vaccinated with a meningococcal and pneumococcal vaccine. C3G and IgAN participants must have been vaccinated or willing to undergo vaccination
- All participants must be willing to be vaccinated or have a history of vaccination for Haemophilus influenzae
- Body Mass Index (BMI) between 18.0 and 35.0 kg/m2
Key exclusion criteria (10 listed)
- Seropositive for human immunodeficiency virus (HIV) infection,hepatitis B virus, or hepatitis C virus
- History of recurrent or chronic infections
- Uncontrolled hypertension
- Regular use of alcohol within 30 days prior to Screening
- Use of illicit drugs within 1 year prior to Screening or positive urine drug screen at Screening
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 |
|---|---|---|
| ARO-C3ARO-C3 for sc injection | Drug | ARO-C3 (Adult Patients with C3G or IgAN), ARO-C3 (Healthy Volunteers) |
| Placebosterile normal saline (0.9% NaCl) for sc injection | Drug | Placebo (Healthy Volunteers) |
Primary outcome
Number of Participants with Adverse Events (AEs) and/or Serious Adverse Events (SAEs) at Day 169
Time frame: up to day 169 (End of Study [EOS])
7 secondary outcomes
Pharmacokinetics (PK) of ARO-C3: Maximum Observed Plasma Concentration (Cmax)
Time frame: up to 48 hours post-dose
PK of ARO-C3: Area under the Plasma Concentration Versus Time Curve from Zero to 24Hours (AUC0-24)
Time frame: up to 48 hours post-dose
PK of ARO-C3: Area Under the Plasma Versus Time Concentration Curve from Zero to the Last Quantifiable Plasma Concentration (AUClast)
Time frame: up to 48 hours post-dose
PK of ARO-C3: Area Under the Plasma Concentration Versus Time Curve from Zero Extrapolated to Infinity (AUCinf) PK of ARO-C3:
Time frame: up to 48 hours post-dose
PK of ARO-C3: Terminal Elimination Half-Life (t1/2)
Time frame: up to 48 hours post-dose
PK of ARO-C3: Apparent Total Body Clearance of ARO-C3 from Plasma (CL)
Time frame: up to 48 hours post-dose
PK of ARO-C3: Volume of Distribution (Vz/F)
Time frame: up to 48 hours post-dose
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 May 2026.
- Derived from this study: PubMed 38299639Tunnicliffe DJ, Reid S, Craig JC, Samuels JA, Molony DA, Strippoli GF. Non-immunosuppressive treatment for IgA nephropathy. Cochrane Database Syst Rev. 2024 Feb 1;2(2):CD003962. doi: 10.1002/14651858.CD003962.pub3.
- 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.
16 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ARO-C3 in people with C3 Glomerulopathy and IgA Nephropathy, run by Arrowhead Pharmaceuticals at 16 sites in 7 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 12 inclusion and 10 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: finished, as of the sponsor's last update on 22 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in South Korea, Australia, Germany, Thailand, Georgia and 2 more countries. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Number of Participants with Adverse Events (AEs) and/or Serious Adverse Events (SAEs) at Day 169 (time frame: up to day 169 (End of Study [EOS])). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05083364, which also lists 1 linked publication.
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 22 May 2026.