HypertriglyceridemiaPhase 1NCT03783377
Study of ARO-APOC3 in Healthy Volunteers, Hypertriglyceridemic Patients and Patients With Familial Chylomicronemia Syndrome (FCS)
A Phase 1 study testing ARO-APOC3 and sterile normal saline (0.9% NaCl) in people with Hypertriglyceridemia and Familial Chylomicronemia, run by Arrowhead Pharmaceuticals at 10 sites in 3 countries.
Trial summary
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple doses of ARO-APOC3 in healthy adult volunteers and in patients with severe hypertriglyceridemia and familial chylomicronemia syndrome (FCS). Read more about this study
- Who is it for?
- Ages 18 to 70 yearsHealthy volunteers can take part
- Conditions the study is for
- Hypertriglyceridemia, Familial Chylomicronemia
- What is being tested?
- ARO-APOC3 and sterile normal saline (0.9% NaCl)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 112 participants
- Where
- 10 sitesin 3 countries
- Study dates
- 8 Mar 2019to 11 Feb 2021
- Phase
- Phase 1
- 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
- Women of childbearing 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
- Normal electrocardiogram (ECG) at screening
- Hypertriglyceridemic patients must have a history of fasting serum triglycerides of at least 300 mg/dL (3.38 mmol/L) at screening or verifiable diagnosis of FCS
Who cannot join
- 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 more than two tobacco/nicotine containing or cannabis products per month within 6 months prior to drug administration (applicable only to Normal Healthy Volunteers)
- Note: additional inclusion/exclusion criteria may apply, per protocol
Trial locations and contacts
10 sites in 3 countries.
10 sites in 3 countries
Australia4 sites
Research Site 3
Adelaide, South Australia, 5000
Research Site 2
Camperdown, New South Wales, 2050
Research Site 4
Perth, Washington, 6009
Research Site 5
Sippy Downs, Queensland
Canada3 sites
Research Site 6
Chicoutimi, Quebec, G7H 7K9
Research Site 7
London, Ontario, N6A 5B7
Research Site 8
Montreal, Quebec, H2W 1R7
New Zealand3 sites
Research Site 11
Christchurch, 8011
Research Site 9
Grafton, Auckland, 1010
Research Site 10
Papatoetoe, Auckland, 2025
About this study
Official title: A Phase 1 Single and Multiple Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamic Effects of ARO-APOC3 in Adult Healthy Volunteers as Well as in Severely Hypertriglyceridemic Patients and Patients With Familial Chylomicronemia Syndrome
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple doses of ARO-APOC3 in healthy adult volunteers and in patients with severe hypertriglyceridemia and familial chylomicronemia syndrome (FCS).
- Conditions
- Hypertriglyceridemia, Familial Chylomicronemia
- Being tested
- ARO-APOC3, sterile normal saline (0.9% NaCl)
- Medical subject headings
- Hypertriglyceridemia, Hyperlipoproteinemia Type I
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ARO-APOC3Drug
single or multiple doses of ARO-APOC3 by subcutaneous (sc) injections
sterile normal saline (0.9% NaCl)Drug
calculated volume to match active treatment
| Study group | Role | What they receive |
|---|---|---|
| ARO-APOC3 | Experimental | Not described Receives: Drug: ARO-APOC3 |
| Placebo | Placebo comparator | Not described Receives: Drug: sterile normal saline (0.9% NaCl) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Adverse Events (AEs) Possibly or Probably Related to Treatment
Measured: Up to Day 113 (+/- 3 days)
Secondary outcomes (6)
Pharmacokinetics (PK) of ARO-APOC3 in Normal Healthy Volunteers (NHVs): Maximum Observed Plasma Concentration (Cmax)
Measured: Single dose phase: Up to 48 hours post-dose
PK of ARO-APOC3 in NHVs: Time to Maximum Plasma Concentration (Tmax)
Measured: Single dose phase: Up to 48 hours post-dose
PK of ARO-APOC3 in NHVs: Terminal Elimination Half-Life (t1/2)
Measured: Single dose phase: Up to 48 hours post-dose
PK of ARO-APOC3 in NHVs: Area Under the Plasma Concentration Versus Time Curve From Zero to 24 Hours (AUC0-24)
Measured: Single dose phase: Up to 48 hours post-dose
PK of ARO-APOC3 in NHVs: Area Under the Plasma Concentration Versus Time Curve From Zero to Infinity (AUCinf)
Measured: Single dose phase: Up to 48 hours post-dose
Reduction in Fasting Serum APOC3 from Pre-Dose Baseline
Measured: Up to Day 113 (+/- 3 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Dec 2018
- Study start8 Mar 2019
- Primary completion11 Feb 2021
- Study completion11 Feb 2021
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 HypertriglyceridemiaStudies 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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 112 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
- NCT03783377
- Sponsor's protocol ID
- AROAPOC31001
- Status last verified
- Dec 2025
- First submitted
- 19 Dec 2018
- Last updated
- 24 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Gaudet D, Clifton P, Sullivan D, Baker J, Schwabe C, Thackwray S, Scott R, Hamilton J, Given B, Melquist S, Zhou R, Chang T, San Martin J, Watts GF, Goldberg IJ, Knowles JW, Hegele RA, Ballantyne CM. RNA Interference Therapy Targeting Apolipoprotein C-III in Hypertriglyceridemia. NEJM Evid. 2023 Dec;2(12):EVIDoa2200325. doi: 10.1056/EVIDoa2200325. Epub 2023 Nov 17.
PubMed 38320498
Study at a glance
From the registry record, last updated by the sponsor on 24 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Single and Multiple Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamic Effects of ARO-APOC3 in Adult Healthy Volunteers as Well as in Severely Hypertriglyceridemic Patients and Patients With Familial Chylomicronemia Syndrome
- ClinicalTrials.gov ID
- NCT03783377
- Acronym / sponsor ID
- AROAPOC31001
- Conditions
- Hypertriglyceridemia, Familial Chylomicronemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 112 participants (actual)
- Lead sponsor
- Arrowhead PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 8 Mar 2019 (actual)
- Primary completion
- 11 Feb 2021 (actual)
- Study completion
- 11 Feb 2021 (actual)
- First posted
- 21 Dec 2018 (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 this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple doses of ARO-APOC3 in healthy adult volunteers and in patients with severe hypertriglyceridemia and familial chylomicronemia syndrome (FCS).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ARO-APOC3 | Experimental | Drug: ARO-APOC3 |
| Placebo | Placebo comparator | Drug: sterile normal saline (0.9% NaCl) |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Women of childbearing 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
- Normal electrocardiogram (ECG) at screening
- Hypertriglyceridemic patients must have a history of fasting serum triglycerides of at least 300 mg/dL (3.38 mmol/L) at screening or verifiable diagnosis of FCS
Key exclusion criteria (6 listed)
- 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 more than two tobacco/nicotine containing or cannabis products per month within 6 months prior to drug administration (applicable only to Normal Healthy Volunteers)
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-APOC3single or multiple doses of ARO-APOC3 by subcutaneous (sc) injections | Drug | ARO-APOC3 |
| sterile normal saline (0.9% NaCl)calculated volume to match active treatment | Drug | Placebo |
Primary outcome
Number of Participants with Adverse Events (AEs) Possibly or Probably Related to Treatment
Time frame: Up to Day 113 (+/- 3 days)
6 secondary outcomes
Pharmacokinetics (PK) of ARO-APOC3 in Normal Healthy Volunteers (NHVs): Maximum Observed Plasma Concentration (Cmax)
Time frame: Single dose phase: Up to 48 hours post-dose
PK of ARO-APOC3 in NHVs: Time to Maximum Plasma Concentration (Tmax)
Time frame: Single dose phase: Up to 48 hours post-dose
PK of ARO-APOC3 in NHVs: Terminal Elimination Half-Life (t1/2)
Time frame: Single dose phase: Up to 48 hours post-dose
PK of ARO-APOC3 in NHVs: Area Under the Plasma Concentration Versus Time Curve From Zero to 24 Hours (AUC0-24)
Time frame: Single dose phase: Up to 48 hours post-dose
PK of ARO-APOC3 in NHVs: Area Under the Plasma Concentration Versus Time Curve From Zero to Infinity (AUCinf)
Time frame: Single dose phase: Up to 48 hours post-dose
Reduction in Fasting Serum APOC3 from Pre-Dose Baseline
Time frame: Up to Day 113 (+/- 3 days)
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 24 Dec 2025.
- Derived from this study: PubMed 38320498Gaudet D, Clifton P, Sullivan D, Baker J, Schwabe C, Thackwray S, Scott R, Hamilton J, Given B, Melquist S, Zhou R, Chang T, San Martin J, Watts GF, Goldberg IJ, Knowles JW, Hegele RA, Ballantyne CM. RNA Interference Therapy Targeting Apolipoprotein C-III in Hypertriglyceridemia. NEJM Evid. 2023 Dec;2(12):EVIDoa2200325. doi: 10.1056/EVIDoa2200325. Epub 2023 Nov 17.
- 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.
10 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ARO-APOC3 and sterile normal saline (0.9% NaCl) in people with Hypertriglyceridemia and Familial Chylomicronemia, run by Arrowhead Pharmaceuticals at 10 sites in 3 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 4 inclusion and 6 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 24 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 10 sites in Australia, Canada, New Zealand. 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 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.
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) Possibly or Probably Related to Treatment (time frame: Up to Day 113 (+/- 3 days)). 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 NCT03783377, 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 24 Dec 2025.