ISRCTN47106904进行中（未招募）Unknown
HORACE (Halting Ornithine transcarbamylase deficiency with Recombinant AAV in ChildrEn). Phase I/II prospective open-label, multicentre, dose-finding clinical trial to assess the safety and efficacy of AAVLK03hOTC for paediatric patients with ornithine transcarbamylase deficiency
niversity of London0 个研究点目标入组 12 人开始时间: 2023年10月13日最近更新:
适应症
试验速览
- 阶段
- Unknown
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 12
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •1. Patient (male or female) aged =16 years old at the time of written informed consent. For the dose escalation phase patients must be aged 6-16 years, for the dose expansion phase patients must be aged 0-16 years (at the time of written informed consent).
- •2. OTC deficiency confirmed via enzymatic or molecular analysis. This may include the identification of pathogenic mutations or liver OTC activity that is <20% of normal activity.
- •3. Patient has severe disease defined by reduced protein allowance and is prescribed at least one ammonia scavenger drug
- •4. Patient (if capable of signing) and parent or legal representative have signed a written informed consent form
- •5. Females of childbearing potential must have a negative pregnancy test in serum or urine at the screening and day 0 infusion visits, and use a highly effective contraception method from the screening visit until 4 weeks after the first negative plasma sample monitoring vector genomes copies or the week 52 visit, whichever comes first
- •6. 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 monitoring vector genomes copies or the week 52 visit, whichever comes first
- •7. Patient’s ammonia level at the baseline visit (pre-gene therapy infusion) is <100 ?mol/L and is within the range of historical ammonia levels obtained when the patient was clinically stable
- •8. Patient has been on a stable dose of ammonia scavenger and stable protein allowance for the last 4 weeks at the baseline visit
- •9. Patient is willing to commit to an additional 4 years of long-term safety follow-up on a separate trial following the first 52 weeks of the HORACE trial
排除标准
- •1. Titres of the neutralising antibodies against AAV-LK03 >1:40 serum dilution
- •2. 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 ULN
- •3. Evidence of severe unexplained liver disease (unexplained by OTC deficiency) including but not limited to liver malignancy, liver cirrhosis, or acute liver failure
- •4. Evidence of active hepatitis B or C virus (HBV and HCV respectively) documented by hepatitis B surface antigen (HBsAg) or HCV RNA positivity
- •5. Positive PCR for human immunodeficiency virus (HIV)
- •6. Liver transplant including hepatocytes/cells infusion
- •7. Current participation in another clinical trial of an investigational medicinal product or medical device, or participation within the previous 12 months
- •8. Patient has contraindications to sodium (1,2-13C2) acetate or immunosuppression including prednisolone
- •9. Active infection (bacterial or viral)
- •10. Pregnant or breastfeeding females
- •11. 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 considered as sequelae of previous hyperammonaemic decompensation and which are considered stable (i.e. not evolving), a grade 3 will be acceptable. Grades 4 and 5 will preclude inclusion
- •12. Patients with any other significant condition or disability that, in the investigator's opinion, may interfere with the patient’s optimal participation in the study
研究者
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