OTC DeficiencyPhase 2NCT06488313
A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD
A Phase 2 study testing ARCT-810 in people with OTC Deficiency and Ornithine Transcarbamylase Deficiency, run by Arcturus Therapeutics, Inc. at 1 site in United States.
Trial summary
Evaluate the safety and pharmacodynamics of multiple doses of ARCT-810 in adolescent and adult participants with OTC deficiency. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- OTC Deficiency, Ornithine Transcarbamylase Deficiency, OTCD
- What is being tested?
- ARCT-810
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteUnited States
- Study dates
- 4 Nov 2024to 1 Sep 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Willingness and ability to comply with all the protocol requirements, complete all study visits and sign informed consent.
- Males and Females aged ≥12 years, at Screening.
- Documented clinical diagnosis of OTC deficiency.
- History of symptomatic hyperammonemia or elevated plasma ammonia or glutamine with clinical stability for at least 1 month prior to Screening.
- Medically managed for OTC deficiency and receiving a stable protein-restricted diet, dietary supplements, and/or ammonia scavenger regimen (if applicable) for at least 28 days.
- Good general health with no clinically significant abnormal findings that would interfere with study procedures (including plasma ammonia within participant's historical range).
- Must be willing to adhere to contraception guidelines.
Who cannot join
- Uncontrolled hypertension.
- Symptoms of infection for at least 7 days prior to dosing.
- Malignancy within 5 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated.
- History of any OTC gene therapy, or history of liver-derived stem cell therapy in the past 2 years.
- History of any organ transplant.
- History of severe allergic reaction to a liposomal or PEG-containing product.
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- History of congenital or acquired cardiac disorders.
- Abuse of medications, illicit drugs or alcohol.
- Blood donation of 50 to 499 mL within 30 days of screening or of >499 mL within 60 days of screening.
- Clinically significant laboratory abnormalities on screening labs including INR >1.5, eGFR< 60 mL/min/1.73m2 or positive test results for HIV, HBV, or HCV.
- Inadequately controlled diabetes.
- Clinically significant anemia.
- Changes in maintenance therapies for OTC deficiency with 28 days prior to dosing.
- Medical history requiring continuous or intermittent systemic corticosteroid administration.
- Receipt of inhibitors of urea synthesis or drugs that significantly affect renal clearance.
- Recent treatment with another investigational drug, biological agent, or device.
- Treatment with any oligonucleotide (siRNA or mRNA) within 6 months prior to screening. COVID-19 vaccines are not exclusionary.
- Involved in study conduct or an immediate family member of an individual involved in the study.
- Participated in another dosing cohort of the study.
- Any other conditions, in the opinion of the investigator, that would interfere with participation.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Uncommon Cures
Chevy Chase, Maryland, 20815
Marshall Summar, MD, 541-232-7818, arcturus@uncommoncures.com
About this study
Official title: A Phase 2a, Open-label, Multiple Ascending Dose Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Adolescent and Adult Participants With Ornithine Transcarbamylase Deficiency
Evaluate the safety and pharmacodynamics of multiple doses of ARCT-810 in adolescent and adult participants with OTC deficiency.
Read the detailed description
This a Phase 2a, open-label study of ARCT-810 in participants 12 years of age and older living with OTC deficiency. After a diet stabilization period of at least 4 weeks, all participants will be enrolled to receive ARCT-810 every two weeks, for up to five doses, at one of three dose levels. Clinic visits will occur during screening and at Days 1, 15, 29, 36, 43, 57, 60, 71, and 85. During the study, participants will remain on their current clinical management for OTC deficiency. Dose escalation or cohort expansion may occur following completion of three participants at each dose level.
- Conditions
- OTC Deficiency, Ornithine Transcarbamylase Deficiency, OTCD
- Being tested
- ARCT-810
- Medical subject headings
- Ornithine Carbamoyltransferase Deficiency Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ARCT-810Biological
ARCT-810 is an investigational medicinal product comprising Ornithine Transcarbamylase (OTC) messenger RNA (mRNA) formulated in a lipid nanoparticle (LNP).
| Study group | Role | What they receive |
|---|---|---|
| ARCT-810 | Experimental | Participants will receive up to 5 IV infusions of ARCT-810 administered at 14-day intervals. Receives: Biological: ARCT-810 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence, severity and dose-relationship of adverse events (AEs)
Safety and tolerability of ARCT-810 assessed by incidence, severity, and dose-relationship of AEs
Measured: Day 85
Secondary outcomes (5)
Stable isotope ureagenesis assay values (AUC of first isotope)
Change from Baseline in stable isotope ureagenesis assay values (AUC of first isotope) following multiple doses of ARCT-810
Measured: Up to Day 85
Stable isotope ureagenesis assay values (AUC of second isotope)
Change from Baseline in stable isotope ureagenesis assay values (AUC of second isotope) following mulitple doses of ARCT-810
Measured: Up to Day 85
Fasting plasma ammonia
Proportion of participants maintaining normal morning fasting plasma ammonia
Measured: Up to Day 85
Plasma Glutamine
Change from Baseline in plasma glutamine and labeled glutamine as available
Measured: Up to Day 85
Plasma pharmacokinetics
The noncompartmental plasma pharmacokinetics of ARCT-810 will be assessed based on the observed plasma concentration of ARCT-810 mRNA and lipid
Measured: Up to Day 57
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jul 2024
- Study start4 Nov 2024
- Primary completion1 Jun 2026 (estimated)
- Study completion1 Sep 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for OTC DeficiencyStudies of the same condition, open and under way.
- 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
- Sequential
- Model description
- All participants will receive 5 doses of Study Drug via intravenous infusion at one of the three dose levels. Dose levels will be enrolled sequentially.
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Arcturus Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06488313
- Sponsor's protocol ID
- ARCT-810-04
- Status last verified
- Mar 2025
- First submitted
- 28 Jun 2024
- Last updated
- 17 Mar 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2a, Open-label, Multiple Ascending Dose Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Adolescent and Adult Participants With Ornithine Transcarbamylase Deficiency
- ClinicalTrials.gov ID
- NCT06488313
- Acronym / sponsor ID
- ARCT-810-04
- Conditions
- OTC Deficiency, Ornithine Transcarbamylase Deficiency, OTCD
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Arcturus Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 4 Nov 2024 (actual)
- Primary completion
- 1 Jun 2026 (estimated)
- Study completion
- 1 Sep 2026 (estimated)
- First posted
- 5 Jul 2024 (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.
Evaluate the safety and pharmacodynamics of multiple doses of ARCT-810 in adolescent and adult participants with OTC deficiency.
Detailed description
This a Phase 2a, open-label study of ARCT-810 in participants 12 years of age and older living with OTC deficiency. After a diet stabilization period of at least 4 weeks, all participants will be enrolled to receive ARCT-810 every two weeks, for up to five doses, at one of three dose levels. Clinic visits will occur during screening and at Days 1, 15, 29, 36, 43, 57, 60, 71, and 85. During the study, participants will remain on their current clinical management for OTC deficiency. Dose escalation or cohort expansion may occur following completion of three participants at each dose level.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- All participants will receive 5 doses of Study Drug via intravenous infusion at one of the three dose levels. Dose levels will be enrolled sequentially.
| Arm | Type | Receives |
|---|---|---|
| ARCT-810 | Experimental | Biological: ARCT-810Participants will receive up to 5 IV infusions of ARCT-810 administered at 14-day intervals. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Willingness and ability to comply with all the protocol requirements, complete all study visits and sign informed consent.
- Males and Females aged ≥12 years, at Screening.
- Documented clinical diagnosis of OTC deficiency.
- History of symptomatic hyperammonemia or elevated plasma ammonia or glutamine with clinical stability for at least 1 month prior to Screening.
- Medically managed for OTC deficiency and receiving a stable protein-restricted diet, dietary supplements, and/or ammonia scavenger regimen (if applicable) for at least 28 days.
Key exclusion criteria (20 listed)
- Uncontrolled hypertension.
- Symptoms of infection for at least 7 days prior to dosing.
- Malignancy within 5 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated.
- History of any OTC gene therapy, or history of liver-derived stem cell therapy in the past 2 years.
- History of any organ transplant.
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 |
|---|---|---|
| ARCT-810ARCT-810 is an investigational medicinal product comprising Ornithine Transcarbamylase (OTC) messenger RNA (mRNA) formulated in a lipid nanoparticle (LNP). | Biological | ARCT-810 |
Primary outcome
Incidence, severity and dose-relationship of adverse events (AEs)
Time frame: Day 85
Safety and tolerability of ARCT-810 assessed by incidence, severity, and dose-relationship of AEs
5 secondary outcomes
Stable isotope ureagenesis assay values (AUC of first isotope)
Time frame: Up to Day 85
Stable isotope ureagenesis assay values (AUC of second isotope)
Time frame: Up to Day 85
Fasting plasma ammonia
Time frame: Up to Day 85
Plasma Glutamine
Time frame: Up to Day 85
Plasma pharmacokinetics
Time frame: Up to Day 57
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Mar 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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ARCT-810 in people with OTC Deficiency and Ornithine Transcarbamylase Deficiency, run by Arcturus Therapeutics, Inc. at 1 site in United States.
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 7 inclusion and 20 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: looking for participants now, as of the sponsor's last update on 17 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence, severity and dose-relationship of adverse events (AEs) (time frame: Day 85). 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 17 Mar 2025.