Actively Recruiting
Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn
Led by University College, London · Updated on 2023-11-07
12
Participants Needed
1
Research Sites
191 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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).
CONDITIONS
Official Title
Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patient aged 0 to 16 years at time of informed consent (6-16 years for dose escalation phase)
- Confirmed ornithine transcarbamylase deficiency by enzyme or genetic testing
- Severe disease with reduced protein allowance and prescribed at least one ammonia scavenger drug
- Signed informed consent by patient (if capable) and parent or legal representative
- Females of childbearing potential must have negative pregnancy tests and use effective contraception during study
- Sexually active boys must use adequate contraception from 14 days before infusion until study end
- Baseline ammonia level less than 100 �b5mol/L within stable historical range
- Stable ammonia scavenger dose and protein allowance for 4 weeks before baseline visit
- Willingness to participate in 4 years of long-term safety follow-up
You will not qualify if you...
- Neutralising antibodies against AAV-LK03 above 1:5 serum dilution
- Significant liver inflammation with lab values above specified limits
- Severe unexplained liver disease including malignancy, cirrhosis, or acute liver failure
- Active hepatitis B or C infection
- Positive test for HIV
- Previous liver transplant or hepatocyte/cell infusion
- Participation in another investigational trial within last 12 months
- Contraindication to immunosuppression
- Active bacterial or viral infection
- Pregnant or breastfeeding females
- Serious underlying medical conditions including malignancy or severe organ impairment (grade 4 or 5), except stable neurological symptoms from prior ammonia episodes
- Any other condition that may interfere with study participation as judged by investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Great Ormond Street Hospital
London, United Kingdom, WC1N 3JH
Actively Recruiting
Research Team
T
Trial Manager
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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