Ignite Creation Date:
2025-12-24 @ 4:23 PM
Ignite Modification Date:
2026-01-22 @ 9:06 AM
Study NCT ID:
NCT05526066
Status:
TERMINATED
Last Update Posted:
2025-09-26
First Post:
2022-08-08
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study for Adolescents and Adults With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D020163', 'term': 'Ornithine Carbamoyltransferase Deficiency Disease'}], 'ancestors': [{'id': 'D056806', 'term': 'Urea Cycle Disorders, Inborn'}, {'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D000592', 'term': 'Amino Acid Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR'], 'maskingDescription': 'Double-Blinded'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL', 'interventionModelDescription': 'On Day 1, participants will receive a single dose of Study Drug via intravenous infusion.\n\nIf the safety observations are considered acceptable, that participant will enter the multiple-dose portion of the study in which they will receive a further 5 doses of Study Drug on Days 15, 29, 43, 57 and 71.'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 8}}, 'statusModule': {'whyStopped': 'The study was terminated after the completion of Cohort 1 (0.3 mg/kg) due to slow enrollment in this placebo-controlled study.', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2022-10-17', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-09', 'dispFirstSubmitDate': '2025-09-22', 'completionDateStruct': {'date': '2024-10-31', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-09-22', 'studyFirstSubmitDate': '2022-08-08', 'studyFirstSubmitQcDate': '2022-08-30', 'dispFirstPostDateStruct': {'date': '2025-03-25', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2025-09-26', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2022-09-02', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2024-10-31', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidence, severity and dose-relationship of adverse events (AEs)', 'timeFrame': 'Week 23', 'description': 'Safety and tolerability of ARCT-810 assessed by determining the number and severity of AEs by dose level'}], 'secondaryOutcomes': [{'measure': 'Plasma concentration area under the curve after first and last doses of ARCT-810', 'timeFrame': 'Up to 17 Weeks', 'description': 'Area under the plasma concentration versus time curve (AUC) from time zero to the last quantifiable time point'}, {'measure': 'Maximum observed plasma concentration (Cmax) after first and last doses of ARCT-810', 'timeFrame': 'Up to 17 Weeks', 'description': 'The maximum observed plasma concentration (Cmax)'}, {'measure': 'Time at which Cmax occurred after first and last doses of ARCT-810', 'timeFrame': 'Up to 17 Weeks', 'description': 'The time at which Cmax occurred (Tmax)'}, {'measure': 'AUC0-inf after first and last doses of ARCT-810', 'timeFrame': 'Up to 17 Weeks', 'description': 'Plasma AUC from time zero extrapolated to infinity'}, {'measure': 'AUCExtrap after first and last doses of ARCT-810', 'timeFrame': 'Up to 17 Weeks', 'description': 'The relative portion of AUC0-inf extrapolated beyond AUC0-t'}, {'measure': 'T1/2 after first and last doses of ARCT-810', 'timeFrame': 'Up to 17 Weeks', 'description': 'Terminal half-life'}, {'measure': 'MRT0-inf after first and last doses of ARCT-810', 'timeFrame': 'Up to 17 Weeks', 'description': 'The mean residence time extrapolated to infinity'}, {'measure': 'CL after first and last doses of ARCT-810', 'timeFrame': 'Up to 17 Weeks', 'description': 'Total body clearance, calculated as dose divided by AUC0-inf'}, {'measure': 'Vss after first and last doses of ARCT-810', 'timeFrame': 'Up to 17 Weeks', 'description': 'Volume of distribution'}, {'measure': 'Urea Cycle Function', 'timeFrame': 'Week 12', 'description': 'Change from baseline in urea cycle function as measured by 13C-urea assay'}, {'measure': 'Plasma Ammonia', 'timeFrame': 'Week 11', 'description': 'Change from baseline in urea cycle function as measured by 24-hour plasma ammonia profile'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['OTC', 'OTCD', 'Ornithine', 'Transcarbamylase', 'mRNA', 'Ornithine Transcarbamylase Deficiency', 'OTC Deficiency'], 'conditions': ['Ornithine Transcarbamylase Deficiency', 'OTC Deficiency', 'OTCD']}, 'descriptionModule': {'briefSummary': 'The primary objective is to evaluate the safety and tolerability of repeated doses of intravenously administered ARCT-810.', 'detailedDescription': 'This study is a Phase 2, randomized, placebo-controlled study of ARCT-810 in people living with OTC deficiency 12 years of age and older. After an at least 4 week screening and diet stabilization period, participants will be randomized 3:1 to receive ARCT-810 or placebo. Following the first dose and safety evaluation, participants will receive up to an additional 5 doses of ARCT-810 or placebo, each separated by 14 days. The treatment period is followed by a 12-week observation period.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '12 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria:\n\n1. Adequate cognitive ability to understand study requirements and give informed consent\n2. Males and females aged 12 to 65 years inclusive, at Screening\n3. Documented diagnosis of OTC deficiency\n4. Clinical stability (no clinical symptoms of hyperammonemia within 1 month, no hospitalizations for metabolic decompensation within 3 months, ≤ 2 hospitalizations within 1 year)\n5. Stable protein-restricted diet, dietary supplements, and ammonia scavenger regimen (if applicable) for at least 28 days.\n6. BMI = 18.0 - 32.0 kg/m2, inclusive for adults, and \\>5th percentile for adolescents ≥12 to 17 years\n7. Must be willing to adhere to contraception guidelines\n\nKey Exclusion Criteria:\n\n1. History of any OTC gene therapy, or history of liver-derived stem cell therapy in the past 3 years\n2. 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)\n3. History of severe allergic reaction to liposomal or PEG-containing products\n4. Abuse of illicit drugs, medications or alcohol\n5. Clinically significant laboratory abnormalities on screening labs'}, 'identificationModule': {'nctId': 'NCT05526066', 'briefTitle': 'Study for Adolescents and Adults With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810', 'organization': {'class': 'INDUSTRY', 'fullName': 'Arcturus Therapeutics, Inc.'}, 'officialTitle': 'Phase 2, Randomized, Double-Blind, Placebo-Controlled, Nested Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability and Pharmacokinetics of ARCT-810 in Adolescent and Adult Participants With Ornithine Transcarbamylase Deficiency', 'orgStudyIdInfo': {'id': 'ARCT-810-03'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'ARCT-810', 'description': 'Participants receive an initial intravenous (IV) infusion ARCT-810. If considered safe and well tolerated, participants will receive up to 5 additional IV infusions of ARCT-810 administered at 14-day intervals.', 'interventionNames': ['Biological: ARCT-810']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo, Normal Saline', 'description': 'Participants receive an initial IV infusion of placebo. If considered safe and well tolerated, participants receive up to 5 additional IV infusions of placebo administered at 14-day intervals.', 'interventionNames': ['Other: Placebo']}], 'interventions': [{'name': 'ARCT-810', 'type': 'BIOLOGICAL', 'description': 'ARCT-810 is messenger RNA (mRNA) coding for Ornithine Transcarbamylase (OTC) formulated in a lipid nanoparticle (LNP).', 'armGroupLabels': ['ARCT-810']}, {'name': 'Placebo', 'type': 'OTHER', 'description': 'Normal Saline', 'armGroupLabels': ['Placebo, Normal Saline']}]}, 'contactsLocationsModule': {'locations': [{'zip': '1200', 'city': 'Brussels', 'country': 'Belgium', 'facility': 'Cliniques Universitaires Saint Luc', 'geoPoint': {'lat': 50.85045, 'lon': 4.34878}}, {'zip': '13005', 'city': 'Marseille', 'country': 'France', 'facility': 'Hôpitaux Universitaires de Marseille - Hôpital de la Timone', 'geoPoint': {'lat': 43.29695, 'lon': 5.38107}}, {'city': 'Paris', 'country': 'France', 'facility': 'Assistance Publique - Hôpitaux de Paris (AP-HP) - Hôpital Necker-Enfants Malades', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '35128', 'city': 'Padua', 'country': 'Italy', 'facility': 'Azienda Ospedaliera di Padova', 'geoPoint': {'lat': 45.40797, 'lon': 11.88586}}, {'zip': '00165', 'city': 'Rome', 'country': 'Italy', 'facility': 'IRCCS Ospedale Pediatrico Bambino Gesu', 'geoPoint': {'lat': 41.89193, 'lon': 12.51133}}, {'city': 'Barcelona', 'country': 'Spain', 'facility': 'Hospital Clínic de Barcelona', 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}, {'city': 'Barcelona', 'country': 'Spain', 'facility': 'Hospital Sant Joan de Déu', 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}, {'zip': '28041', 'city': 'Madrid', 'country': 'Spain', 'facility': 'Hospital Universitario 12 de Octubre', 'geoPoint': {'lat': 40.4165, 'lon': -3.70256}}, {'zip': '15706', 'city': 'Santiago de Compostela', 'country': 'Spain', 'facility': 'Complejo Hospitalario Universitario de Santiago (CHUS) - Hospital Clínico Universitario', 'geoPoint': {'lat': 42.88052, 'lon': -8.54569}}, {'zip': 'SE- 171 64', 'city': 'Stockholm', 'country': 'Sweden', 'facility': 'Karolinska Universitetssjukhuset - Astrid Lindgrens Barnsjukhus', 'geoPoint': {'lat': 59.32938, 'lon': 18.06871}}, {'zip': 'B15 2PR', 'city': 'Birmingham', 'state': 'UK', 'country': 'United Kingdom', 'facility': 'University Hospitals Birmingham NHS Foundation Trust - Queen Elizabeth Hospital Birmingham', 'geoPoint': {'lat': 52.48142, 'lon': -1.89983}}, {'zip': 'WC1N 3JH', 'city': 'London', 'state': 'UK', 'country': 'United Kingdom', 'facility': 'Great Ormond Street Hospital for Children NHS Foundation Trust', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'city': 'London', 'country': 'United Kingdom', 'facility': 'University College London Hospitals NHS Foundation Trust - National Hospital for Neurology and Neurosurgery', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'M6 8HD', 'city': 'Salford', 'country': 'United Kingdom', 'facility': 'Salford Royal NHS Foundation Trust - Salford Royal Hospital', 'geoPoint': {'lat': 53.48771, 'lon': -2.29042}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Arcturus Therapeutics, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}