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New treatment option for Ornithine Transcarbamylase Deficiency
Official title Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn
ClinicalTrials.gov ID: NCT05092685
What this study is testing
What is AAVLK03hOTC?
AAVLK03hOTC is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for ornithine transcarbamylase deficiency.
Also referred to as Also known as ssAAV-LK03.hAAT.hcoOTC.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 0 to 16
You may be able to join if
- Patient (male or female) aged ≤16 years at time of written informed consent. For the dose escalation phase patients must be aged 6-16, for the dose...
- OTC deficiency confirmed via enzymatic or molecular analysis. This may include identification of pathogenic mutations or liver OTC activity that is...
- Patient has severe disease defined by reduced protein allowance and prescribed at least one ammonia scavenger drug.
- Patient (if capable of signing) and parents or legal representative have signed a written informed consent form.
- Females of childbearing potential must have a negative pregnancy test in serum or urine at the screening and Day 0 infusion visits, and use an...
You likely can't join if
- Titres of the neutralising antibodies against AAV-LK03 \>1:5 serum dilution.
- Significant hepatic inflammation as evidenced by the following laboratory abnormalities: alanine aminotransferase or aspartate aminotransferase or...
- Evidence of severe unexplained liver disease including but not limited to liver malignancy, liver cirrhosis, or acute liver failure.
- Evidence of active hepatitis B or C virus (HBV and HCV respectively) documented by hepatitis B surface antigen (HBsAg) or HCV RNA positivity.
- Positive PCR for human immunodeficiency virus (HIV).
- Liver transplant including hepatocytes/cells infusion.
See the full eligibility criteria
- Patient (male or female) aged ≤16 years at time of written informed consent. For the dose escalation phase patients must be aged 6-16, for the dose expansion phase patients must be aged 0-16 (at the time of written...
- OTC deficiency confirmed via enzymatic or molecular analysis. This may include identification of pathogenic mutations or liver OTC activity that is \<20% of normal activity.
- Patient has severe disease defined by reduced protein allowance and prescribed at least one ammonia scavenger drug.
- Patient (if capable of signing) and parents or legal representative have signed a written informed consent form.
- Females of childbearing potential must have a negative pregnancy test in serum or urine at the screening and Day 0 infusion visits, and use an adequate contraception method from the screening visit until 4 weeks after...
- Sexually active boys must use an adequate contraception method (abstinence or use of condom with spermicide) from at least 14 days prior to the infusion and until 4 weeks after the first negative plasma sample...
- Patient's ammonia level at baseline visit (pre-gene therapy infusion) is \<100µmol/L and is within the range of historical ammonia levels obtained when the patient was clinically stable.
- Patient has been on a stable dose of ammonia scavenger and stable protein allowance for the last 4 weeks at the baseline visit.
- Patient is willing to commit to an additional 4 years of long-term safety follow-up.
- Titres of the neutralising antibodies against AAV-LK03 \>1:5 serum dilution.
- Significant hepatic inflammation as evidenced by the following laboratory abnormalities: alanine aminotransferase or aspartate aminotransferase or bilirubin \>2 x upper limit of normal (ULN), alkaline phosphatase \>3 x...
- Evidence of severe unexplained liver disease including but not limited to liver malignancy, liver cirrhosis, or acute liver failure.
- Evidence of active hepatitis B or C virus (HBV and HCV respectively) documented by hepatitis B surface antigen (HBsAg) or HCV RNA positivity.
- Positive PCR for human immunodeficiency virus (HIV).
- Liver transplant including hepatocytes/cells infusion.
- Current participation in another clinical trial of an investigational medicinal product or medical device, or participation within previous 12 months.
- Patient has contraindication to immunosuppression.
- Active infection (bacterial or viral).
- Pregnant or breastfeeding females.
- Patients with other serious underlying medical conditions including malignancy and severe (≥ grade 3) functional organ impairment (liver, kidney, respiratory) according to CTCAE v5.0. For neurological symptoms...
- Patients with any other significant condition or disability that, in the investigator opinion, may interfere with the patient's optimal participation in the study.
The study team makes the final eligibility decision.
Where it's taking place
- London, United Kingdom
Compensation & support
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0 days to 16 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include London, United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.