A Phase 1 interventional study of ARCT-810 and Placebo in Ornithine Transcarbamylase Deficiency, sponsored by Arcturus Therapeutics, Inc.. Completed at 8 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-08-02.
Sponsored by Arcturus Therapeutics, Inc. · Phase 1, Interventional, and Treatment
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Among 28 studies in Ornithine Carbamoyltransferase Deficiency Disease with a site in United States — 6 open to participants now, 11 under way, 2 of them phase 3. Ornithine Carbamoyltransferase Deficiency Disease · United States.
Among the 28 studies in Ornithine Carbamoyltransferase Deficiency Disease with a site in United States, this one is the 5th largest by enrollment of the 12 interventional ones that give a count, is one of 4 that started in 2020, is one of 3 in phase 1, and was the 17th registered.
59 studies on the registry are indexed under Ornithine Carbamoyltransferase Deficiency Disease; 13 are open to participants now.
Browse Ornithine Carbamoyltransferase Deficiency Disease studies →6 of the 28 studies in Ornithine Carbamoyltransferase Deficiency Disease with a site in United States are open now. Recruiting first, the newest first:
iECURE, Inc. · Sites in United States, France, United Kingdom
Ages 7 mo–15 mo
Arcturus Therapeutics, Inc. · Sites in United States
Ages 12+
Baylor College of Medicine · Sites in United States
Abbott Medical Devices · Sites in United States, Australia, Austria and 11 more countries
Ages 18+
Children's National Research Institute · Sites in United States
Ages 7–18
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 10 Ornithine Carbamoyltransferase Deficiency Disease studies recruiting now, 3 (30%) set no upper age limit and 6 (60%) stop at 75 or younger; 7 take children under 18; 3 take healthy volunteers.
This study: 18 Years and older · all sexes · no healthy volunteers.
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28 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 5 · Phase 2 6 · Phase 3 2
First in 2007; this study’s year is marked. 4 started in 2020.
This study's enrollment of 16 is below the median of 30 across 28 interventional studies indexed under Ornithine Carbamoyltransferase Deficiency Disease.
Median enrollment 15 across the 12 interventional studies, 66 across the 16 observational. Of the 17 that completed or were terminated, 7 (41%) have results posted.
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15 of 28 have an industry lead sponsor. The 10 most frequent:
Arcturus Therapeutics, Inc. is the lead sponsor of 13 studies on the registry; 2 are open to participants now.
Of its 8 completed or terminated interventional studies of FDA-regulated products, 5 (63%) have results posted.
Arcturus Therapeutics, Inc. has 4 studies filed under Ornithine Carbamoyltransferase Deficiency Disease — fewer than ten, so there is no comparison to draw.
3 of the studies in Ornithine Carbamoyltransferase Deficiency Disease with a site in United States that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05345171Phase 3Ultragenyx Pharmaceutical IncClinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency | 2027-09 |
| NCT03655223RTI InternationalEarly Check: Expanded Screening in Newborns | 2026-11-30 |
| NCT04612764Baylor College of MedicineLiver Disease in Urea Cycle Disorders | 2026-12-31 |
Add all 3 to your calendar (.ics) → With an account.
Among the 28 studies in Ornithine Carbamoyltransferase Deficiency Disease with a site in United States, from their registry updates:
Nothing recorded among them since 2026-09-25.
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Determine the safety, tolerability and pharmacokinetics of single doses of ARCT-810 in clinically stable patients (stable on standard of care treatment, e.g. diet ± ammonia scavengers) with ornithine transcarbamylase deficiency (OTCD).
This is a single ascending dose study of ARCT-810 in which approximately 12 (up to a maximum of 20) clinically stable patients with ornithine transcarbamylase deficiency (OTCD) are planned to be enrolled.
Each study subject's participation length is approximately 8 weeks, from screening through last study visit. The study comprises an up to 4-week screening period, and a 4-week diet run-in period, to occur concurrently, followed by a 1-day dosing period and a 28-day post-treatment period.
Study participants will be allocated to one of the three single-dose treatment groups (also referred to as cohorts), to test different doses of ARCT-810. Four subjects will be enrolled in each group. Within each cohort, subjects will be randomized 3:1 to receive ARCT-810 or placebo as an IV infusion.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Subject's ornithine transcarbamylase deficiency (OTCD) is stable as evidenced by
Subjects must remain free from symptoms of hyperammonemia throughout the screening period.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Ascending single doses of ARCT-810 administered intravenously
Biological: ARCT-810
Single doses of 0.9% Saline administered intravenously
Other: Placebo
ARCT-810 is an investigational medicinal product comprising Ornithine Transcarbamylase (OTC) mRNA formulated in a lipid nanoparticle (LNP) under development.
The placebo for this study is 0.9% sterile saline.
Incidence, severity and dose-relationship of adverse events (AEs)
Safety and tolerability of ARCT-810 assessed by determining the incidence, severity and dose-relationship of AEs by dose
Time frame: 4 weeks
Change in area under the curve after single dose of ARCT-810
Area under the plasma concentration versus time curve (AUC) from time zero to the last quantifiable time point
Time frame: Up to 4 weeks
Maximum observed plasma concentration (Cmax) after single dose of ARCT-810
The maximum observed plasma concentration (Cmax)
Time frame: Up to 4 weeks
Time at which Cmax occurred after single dose of ARCT-810
The time at which Cmax occurred (Tmax)
Time frame: Up to 4 weeks
AUC0-inf after single dose of ARCT-810
AUC from time zero extrapolated to infinity
Time frame: Up to 4 weeks
AUCExtrap after single dose of ARCT-810
The relative portion of AUC0-inf extrapolated beyond AUC0-t
Time frame: Up to 4 weeks
T1/2 after single dose of ARCT-810
Terminal half-life
Time frame: Up to 4 weeks
MRT0-inf after single dose of ARCT-810
The mean residence time extrapolated to infinity
Time frame: Up to 4 weeks
CL after single dose of ARCT-810
Total body clearance, calculated as dose divided by AUC0-inf
Time frame: Up to 4 weeks
Vss after single dose of ARCT-810
Volume of distribution
Time frame: Up to 4 weeks
Ran from Nov 2020 to primary completion in Aug 2023, 33 months.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Jul 2024. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: ARCT-810
Ornithine Carbamoyltransferase Deficiency Disease →
Arcturus Therapeutics, Inc.