NCT05526066 - Study for Adolescents and Adults With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810 | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

October 17, 2022

Primary Completion Date

October 31, 2024

Study Completion Date

October 31, 2024

Conditions

Ornithine Transcarbamylase DeficiencyOTC DeficiencyOTCD

Interventions

BIOLOGICAL

ARCT-810

ARCT-810 is messenger RNA (mRNA) coding for Ornithine Transcarbamylase (OTC) formulated in a lipid nanoparticle (LNP).

OTHER

Placebo

Normal Saline

Trial Locations (14)

Cliniques Universitaires Saint Luc, Brussels

Hôpitaux Universitaires de Marseille - Hôpital de la Timone, Marseille

Complejo Hospitalario Universitario de Santiago (CHUS) - Hospital Clínico Universitario, Santiago de Compostela

Hospital Universitario 12 de Octubre, Madrid

Azienda Ospedaliera di Padova, Padua

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

Hospital Clínic de Barcelona, Barcelona

Hospital Sant Joan de Déu, Barcelona

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

IRCCS Ospedale Pediatrico Bambino Gesu, Rome

Karolinska Universitetssjukhuset - Astrid Lindgrens Barnsjukhus, Stockholm

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

Great Ormond Street Hospital for Children NHS Foundation Trust, London

Salford Royal NHS Foundation Trust - Salford Royal Hospital, Salford