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Experimental therapy for rare metabolic disease shows early promise

NCT ID NCT05526066

Disease control Sponsor: Arcturus Therapeutics, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested a new medicine called ARCT-810 in 8 people aged 12 to 65 with ornithine transcarbamylase (OTC) deficiency, a rare genetic disorder that causes dangerous ammonia buildup. The main goal was to check the drug's safety and how the body processes it. The trial was stopped early, so results are limited.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

8 people

The number who actually took part.

Started

Oct 2022

Finished

Oct 2024

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 to 65 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: 1. Adequate cognitive ability to understand study requirements and give informed consent 2. Males and females aged 12 to 65 years inclusive, at Screening 3. Documented diagnosis of OTC deficiency 4. Clinical stability (no clinical symptoms of hyperammonemia within 1 month, no hospitalizations for metabolic decompensation within 3 months, ≤ 2 hospitalizations within 1 year) 5. Stable protein-restricted diet, dietary supplements, and ammonia scavenger regimen (if applicable) for at least 28 days. 6. BMI = 18.0 - 32.0 kg/m2, inclusive for adults, and \>5th percentile for adolescents ≥12 to 17 years 7. Must be willing to adhere to contraception guidelines Key Exclusion Criteria: 1. History of any OTC gene therapy, or history of liver-derived stem cell therapy in the past 3 years 2. History of other medical conditions that may make the participant unsuitable for inclusion or could interfere with study participation (e.g., uncontrolled hypertension or diabetes, malignancy, HIV, hepatitis B or C) 3. History of severe allergic reaction to liposomal or PEG-containing products 4. Abuse of illicit drugs, medications or alcohol 5. Clinically significant laboratory abnormalities on screening labs

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Conditions

The condition(s) this trial relates to.

ornithine carbamoyltransferase deficiency

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Ornithine transcarbamylase deficiency Otc deficiency OTCD

Contacts and locations

Locations

  • Assistance Publique - Hôpitaux de Paris (AP-HP) - Hôpital Necker-Enfants Malades

    Paris, France

  • Azienda Ospedaliera di Padova

    Padova, 35128, Italy

  • Cliniques Universitaires Saint Luc

    Brussels, 1200, Belgium

  • Complejo Hospitalario Universitario de Santiago (CHUS) - Hospital Clínico Universitario

    Santiago de Compostela, 15706, Spain

  • Great Ormond Street Hospital for Children NHS Foundation Trust

    London, UK, WC1N 3JH, United Kingdom

  • Hospital Clínic de Barcelona

    Barcelona, Spain

  • Hospital Sant Joan de Déu

    Barcelona, Spain

  • Hospital Universitario 12 de Octubre

    Madrid, 28041, Spain

  • Hôpitaux Universitaires de Marseille - Hôpital de la Timone

    Marseille, 13005, France

  • IRCCS Ospedale Pediatrico Bambino Gesu

    Rome, 00165, Italy

  • Karolinska Universitetssjukhuset - Astrid Lindgrens Barnsjukhus

    Stockholm, SE- 171 64, Sweden

  • Salford Royal NHS Foundation Trust - Salford Royal Hospital

    Salford, M6 8HD, United Kingdom

  • University College London Hospitals NHS Foundation Trust - National Hospital for Neurology and Neurosurgery

    London, United Kingdom

  • University Hospitals Birmingham NHS Foundation Trust - Queen Elizabeth Hospital Birmingham

    Birmingham, UK, B15 2PR, United Kingdom

More trials for these conditions

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