CTIS2022-500306-17-00进行中（未招募）1 期
A multi-center, randomized, active controlled clinical trial to evaluate the efficacy and safety of OTL-203 in subjects with mucopolysaccharidosis type I, Hurler syndrome (MPS-IH) compared to standard of care with allogeneic hematopoietic stem cell transplantation (allo-HSCT) - OTL-203-02
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 40
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
- 年龄范围
- 0 至 17（—）
- 性别
- All
入选标准
- •Written informed consent by parent/legal guardian., Male or female subject with age at enrolment: = 28 days to =30 months. Note: In addition, subjects aged above 30 months shall be considered for enrolment and treatment with OTL-203 in EU/UK only, if they meet all the eligibility criteria and upon agreement with the Medical Monitor (MM), to comply with the EU/UK PIP. Any subject aged > 30 months enrolled and treated with OTL-203 will be followed as per the SoA of the study but will not be included in the primary statistical analysis., Norm-referenced cognitive standard score of =70 evaluated within the Screening period of the study and measured by age-appropriate cognitive domains of Bayley Scale of Infant Development (BSID)-III or Wechsler Preschool and Primary Scale of Intelligence (WPPSI)-IV (visual spatial index” for WPPSI-IV)., Confirmed laboratory diagnosis of MPS-IH as demonstrated by biallelic mutation(s) in the gene coding for IDUA enzyme (at a CLIA-accredited laboratory): a. homozygosity or compound heterozygosity for two known severe alleles, both of which must be associated with a severe (Hurler) phenotype according to the literature, or b. If mutation(s) are unknown/novel, either of the following: i. sibling known to have MPS-IH with severe phenotype, or ii. presence of somatic features (confirmed by an MPS-I specialist) presenting in the first two years of life, which are consistent with MPS-IH severe phenotype [including but not limited to frequent ear infections, frequent upper respiratory infections, umbilical/inguinal hernia, kyphosis/gibbus, corneal clouding, hepatosplenomegaly, dysostosis multiplex, cognitive impairment, cardiac valve abnormalities, joint contractures], Final confirmation of MPS-IH diagnosis by Diagnostic Review Committee (DRC), following the review of: a. gene mutation analysis b. documented biochemical evidence of a deficiency in IDUA enzyme activity c. documented evidence of altered GAG metabolism d. somatic manifestations of the disease.
排除标准
- •Previous allo-HSCT or gene therapy., Subjects who have contraindications for MRI scans (for example, but not limited to, cardiac pacemaker, metal implants)., Current enrollment or past treatment in any other study/trial using a novel investigational agent for which the washout cannot be confirmed by the time of anticipated treatment., Evidence of: a. Positivity to serological testing for: i. Human immunodeficiency virus (HIV-1 or HIV-2), ii. Human T lymphotropic virus (HTLV-1 or HTLV-2), iii. Hepatitis B virus (HBV) core. Subjects positive for Hepatitis B core antibodies due to prior resolved disease may be enrolled, if a confirmatory negative Hepatitis B surface antigen and negative HBV deoxyribonucleic acid test are obtained iv. Hepatitis C virus (HCV). Subjects who have previously tested positive for antibodies against HCV may be enrolled, provided ongoing infection is excluded using nucleic acid testing v. Mycoplasma, unless ongoing infection can be excluded (e.g., on the basis of a negative result on a repeat serological testing or molecular testing (polymerase chain reaction) and the investigator’s assessment); b. Active tuberculosis (TB) c. Not meeting the microbiology biological screening requirements for drug product (DP) manufacturing., Malignant neoplasia (except local skin cancer). 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 Sponsor’s MM., Myelodysplastic syndrome (MDS)/ acute myeloid leukemia (AML)., History of uncontrolled seizures., Subjects with an active infection not responsive to treatment, end-organ damage, or any other disease that contraindicates performance of any of the procedures detailed in the protocol (for example, but not limited to, general anesthesia, mobilization of CD34+ cells from the bone marrow into the peripheral circulation using G-CSF ± plerixafor, leukapheresis and myeloablative conditioning with busulfan and fludarabine)., Subjects, who in the opinion of the Investigator, may not be able to comply with protocol requirements or cooperate fully with the study procedures and necessary long-term follow up., Subjects with any of the following: • Alanine transaminase (ALT) or aspartate aminotransferase (AST) >3 × upper limit of normal (ULN), • Total bilirubin >1.5 × ULN at screening unless the subject has a previously known history of Gilbert's syndrome and a fractionated bilirubin that shows conjugated bilirubin <35% of total bilirubin, • Renal creatinine clearance <30 mL/min, • Left ventricular ejection fraction (LVEF) < 45% by echo or diagnosis of severe pulmonary hypertension, • Medical conditions or extenuating circumstances that, in the opinion of the Investigator, might compromise the subject’s well-being or safety, or the interpretability of the subject’s clinical data.
研究者
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