EUCTR2019-002051-42-GB进行中（未招募）1 期
A phase I-II, study of autologous CD34+ haematopoietic stem cells transduced ex vivo with CD11b lentiviral vector encoding for human SGSH in patients with mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome type A)
niversity of Manchester0 个研究点目标入组 5 人开始时间: 2020年4月9日最近更新:
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 5
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. Provision of written informed consent prior to any study related procedures. This study consent must be provided by the parents/legal guardians
- •2. Age at baseline =3 months and =24 months
- •3. Normal cognitive function or mild cognitive deterioration at baseline (Development Quotient =80) as determined by the Bayley Scale of Infant Development-third edition (BSID-III), cognitive domain, or assessed as normal or only mildly impaired by experienced neuropsychologist and agreed by independent expert reviewer
- •4. Sibling or relative of known MPS IIIA patients with rapidly progressing phenotype or genotype associated with rapidly progressing phenotype or presence of somatic features predictive of rapid progression (including but not limited to mild facial dysmorphism, frequent ear infections, frequent upper respiratory infections, umbilical/inguinal hernia, diarrhoea, speech delay, sleeping difficulties, hyperactivity, attention deficit and hearing difficulties, early behavioural problems including perseverative chewing and difficulty with toilet training)
- •5. Diagnosis of MPS IIIA based on evidence of SGSH deficiency, defined as Sulfoglucosamine Sulfohydrolase (SGSH) activity =10% of the Lower Limit of Normal as measured in total leukocytes, plus either (1) a normal enzyme activity level of at least one other sulfatase (to rule out multiple sulfatase deficiency) as measured in leukocytes, or (2) two documented mutations in the SGSH gene
- •6. Medically stable and able to accommodate the protocol requirements (including travel) and protocol assessments without placing an undue burden on the subject/subject’s family, as determined by the CI
- •7. Subjects and their parents/legal guardians must be willing and able to comply with study restrictions and to commit to attend clinic for the required duration during the study and follow up period as specified in the protocol
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 5
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. The subject has previously received stem cell therapy, gene therapy or enzyme replacement therapy (any route of administration)
- •2. Subject currently enrolled in other interventional trial
- •3. Contraindications for MRI scans (e.g., cardiac pacemaker, metal fragment or chip in the eye, or aneurysm clip in the brain)
- •4. The subject has a history of poorly controlled seizures
- •5. Homozygous or compound heterozygous for the S298P mutation or any other mutation known to be associated to slow progressing phenotype
- •6. The patient is currently receiving psychotropic or other medications which, in the CI’s opinion, would be likely to substantially confound test results
- •7. The patient has received any investigational medication (including Genistein) within 30 days prior to the Baseline visit or is scheduled to receive any investigational drug during the course of the study
- •8. Documented Human Immunodeficiency Virus (HIV) infection (positive HIV ribonucleic acid [RNA] and/or anti p24 antibodies)
- •9. Malignant neoplasia (except local skin cancer) or a documented history of hereditary cancer syndrome. Subjects with a prior successfully treated malignancy and a sufficient follow up to exclude recurrence (based on oncologist opinion) can be included after discussion and approval by the Medical Monitor
- •10. Myelodysplasia, cytogenetic alterations characteristic of myelodysplastic syndrome and acute myeloid leukaemia, or other serious haematological disorders
- •11. Medical condition or extenuating circumstance that, in the opinion of the CI might compromise the subject’s ability to comply with protocol requirements, the subject’s well being or safety, or the interpretability of the subject’s clinical data
- •12. Visual or hearing impairment sufficient to preclude cooperation with neurodevelopmental testing
- •13. Severe behavioural disturbances due to reasons other than MPS IIIA and likely to interfere with protocol compliance, as determined by the CI
- •14. Known sensitivity to busulfan
- •15. The receipt of live vaccinations within 30 days prior to study start
研究者
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