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| ID | Type | Description | Link |
|---|---|---|---|
| CSH-3660 |
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| Name | Class |
|---|---|
| Children's National Research Institute | OTHER |
OBJECTIVES:
Evaluate the safety and feasibility of administering recombinant adenovirus containing the ornithine transcarbamylase gene to adults with partial ornithine transcarbamylase deficiency.
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.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| recombinant adenovirus containing the ornithine transcarbamylase gene | Genetic |
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
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| Name | Affiliation | Role |
|---|---|---|
| Mark Batshaw | Children's National Research Institute | Study Chair |
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| ID | Term |
|---|---|
| D020163 | Ornithine Carbamoyltransferase Deficiency Disease |
| D008661 | Metabolism, Inborn Errors |
| D035583 | Rare Diseases |
| D056806 | Urea Cycle Disorders, Inborn |
| ID | Term |
|---|---|
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D000592 | Amino Acid Metabolism, Inborn Errors |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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