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NCTID
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NCT05092685 (View at clinicaltrials.gov)
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Description
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Ornithine transcarbamylase deficiency (OTCD) is an inherited metabolic liver disease which means that the body cannot maintain normal levels of ammonia. Ammonia levels can rise (called hyperammonaemic decompensations) which can be life-threatening and may result in impaired neurological development in children. OTCD is a rare genetic disorder characterised by complete or partial lack of the enzyme ornithine transcarbamylase (OTC).
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Development Status
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Active
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Indication
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Ornithine Transcarbamylase (OTC) Deficiency
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Disease Ontology Term
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DOID:9271
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Compound Name
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BGT-OTCD
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Compound Alias
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AAVLK03hOTC
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Compound Description
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AAV-based gene therapy vector (serotype LK03) encoding a human codon-optimized OTC gene under the control of the hAAT promotor and hOTC enhancer
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Sponsor
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University College, London
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Funder Type
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Other
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Recruitment Status
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Enrollment Count
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12 (ESTIMATED)
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| Results Posted |
Not Available
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