- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00004386
Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency
OBJECTIVES:
Evaluate the safety and feasibility of administering recombinant adenovirus containing the ornithine transcarbamylase gene to adults with partial ornithine transcarbamylase deficiency.
Study Overview
Status
Intervention / Treatment
Detailed Description
PROTOCOL OUTLINE: This a dose escalation study to estimate the maximum tolerated dose of recombinant adenovirus encoded with the ornithine transcarbamylase gene.
Patients receive a single dose of virus infused into the liver under fluoroscopic guidance. Groups of 3 patients receive successively higher doses of virus; each cohort is observed for safety for 3 weeks before entry of the next group.
Patients are followed weekly for 1 month, then every 3 months until stable.
Study Type
Phase
- Phase 1
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics-- Confirmed partial ornithine transcarbamylase deficiency Asymptomatic, i.e., at least 1 month since hyperammonemia --Prior/Concurrent Therapy-- Not specified --Patient Characteristics-- No pregnant or nursing women Negative pregnancy test required of fertile women
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
Collaborators and Investigators
Collaborators
Investigators
- Study Chair: Mark Batshaw, Children's National Research Institute
Study record dates
Study Major Dates
Study Start
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Nutrition Disorders
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Malnutrition
- Metabolism, Inborn Errors
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Amino Acid Metabolism, Inborn Errors
- Urea Cycle Disorders, Inborn
- Ornithine Carbamoyltransferase Deficiency Disease
- Deficiency Diseases
Other Study ID Numbers
- 199/12054
- CSH-3660
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Ornithine Transcarbamylase Deficiency Disease
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Ultragenyx Pharmaceutical IncCompletedOrnithine Transcarbamylase (OTC) DeficiencyUnited Kingdom, United States, Canada, Spain
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Ultragenyx Pharmaceutical IncActive, not recruitingOrnithine Transcarbamylase (OTC) DeficiencyCanada, United States, France, Spain, United Kingdom
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Ultragenyx Pharmaceutical IncTerminatedOrnithine Transcarbamylase DeficiencyUnited States
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iECURE, Inc.Not yet recruitingUrea Cycle Disorders, Inborn | Ornithine Transcarbamylase Deficiency | Ornithine Transcarbamylase Deficiency Disease | Ornithine Carbamoyltransferase Deficiency (Disorder)
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University of PennsylvaniaTerminatedOrnithine Transcarbamylase Deficiency Disease
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Arcturus Therapeutics, Inc.RecruitingOTC Deficiency | Ornithine Transcarbamylase Deficiency | OTCDSpain, Belgium, United Kingdom, Sweden
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University College, LondonRecruitingOrnithine Transcarbamylase DeficiencyUnited Kingdom
-
Arcturus Therapeutics, Inc.Active, not recruitingOrnithine Transcarbamylase DeficiencyUnited States
-
Ultragenyx Pharmaceutical IncCompletedOrnithine Transcarbamylase DeficiencyUnited States
-
Arcturus Therapeutics, Inc.CompletedOrnithine Transcarbamylase DeficiencyNew Zealand
Clinical Trials on recombinant adenovirus containing the ornithine transcarbamylase gene
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Shanghai Fengxian District Central HospitalShanghai Yuansong Biotechnology Co., LTDRecruitingMelanoma | Breast Cancer | Head and Neck Cancer | Lung Cancer | Bladder Cancer | Cervical Carcinoma | Ovarian CarcinomaChina
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ArthrogenUnknown
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ArthrogenCentre for Human Drug Research (CHDR)UnknownArthritis, Rheumatoid | Osteo ArthritisNetherlands
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University of ManchesterUniversity College, London; Manchester University NHS Foundation Trust; Orchard... and other collaboratorsActive, not recruitingMucopolysaccharidosis Type IIIAUnited Kingdom
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Fujian Cancer HospitalRecruitingUntreated Advanced Non-small Cell Lung CancerChina