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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn
NCTID
NCT05092685
(View at clinicaltrials.gov)
Description
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
Active
Indication
Ornithine Transcarbamylase (OTC) Deficiency
Disease Ontology Term
DOID:9271
Compound Name
BGT-OTCD
Compound Description
AAVLK03hOTC
Sponsor
University College, London
Funder Type
Other
Recruitment Status
Recruiting
Enrollment Count
12
Results Posted
Not Available
Therapy Information
Target Gene/Variant
OTC
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Intravenous
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV-LK03
Editor Type
none
Dose 1
6E11 vg/kg
Dose 2
2E12 vg/kg
Dose 3
6E12 vg/kg
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2021-09-28
Completion Date
2027-06-30
Last Update
2023-11-07
Participation Criteria
Eligible Age
0 Days - 16 Years
Standard Ages
Child
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
1
Locations
United Kingdom
Regulatory Information
Has US IND
False
FDA Designations
Orphan Drug Designation
Recent Updates
Granted ODD by FDA and EMA
Resources/Links
Clinical Publications
(Poster) First patient treated in the phase 1/2 trial of AAVLK03hOTC gene therapy for ornithine transcarbamylase deficiency in children - ESGCT 2024
News and Press Releases
Bloomsbury Genetic Therapies Announces Initiation of Recruitment in Phase 1/2 HORACE Clinical Trial of Investigational Gene Therapy BGT-OTCD for the Treatment of Ornithine Transcarbamylase Deficiency (OTCD)
Preclinical Publications
Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys
Characterization of the humanized FRG mouse model and development of an AAV-LK03 variant with improved liver lobular biodistribution