A phase I/II study evaluating safety and efficacy of autologous hematopoietic stem and progenitor cells genetically modified with IDUA lentiviral vector encoding for the human a-L-iduronidase gene for the treatment of patients affected by Mucopolysaccharidosis Type I, Hurler variant

Ospedale San Raffaele S.r.l.0 个研究点目标入组 8 人开始时间: 2024年6月11日最近更新: 2024年8月21日

适应症

试验速览

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•Written informed consent by parent/legal guardian, Sex: Males or Females, Age: = 28 days and = 11 years old, Biochemically and molecularly proven MPS IH, Lansky Index > 80 %, Indication to HSCT, Lack of a non-heterozygous (for mutated IDUA) HLA-matched sibling donor or a =7/8 (4 digits high-resolution typing) HLA-matched cord blood donor with a cellularity =5 x 107 Total Nucleated Cells (TNC)/Kg after 1-month search. This criterion will not apply to patients whose country of origin does not offer unrelated donor cord blood transplantation., Adequate cardiac, renal, hepatic and pulmonary functions

•Use of other investigational agents within 4 weeks prior to study enrolment (within 6 weeks if use of long-acting agents), Controindications to PeIMP (G-CSF, Plerixafor, Busulfan, Fludarabine, Rituximab), Severe, active viral, bacterial, or fungal infection at eligibility evaluation, Patients affected by malignant neoplasia or family history of familial cancer syndromes, Cytogenetic alterations associated with high risk of developing hematological malignancies, History of uncontrolled seizures, Patients with end-organ damage or any other severe disease which, in the judgment of the investigator, would make the patient inappropriate for entry into this study, Positivity for HIV (serology or RNA), and/or HbsAg and/or HBV DNA and/or HCV RNA and/or Treponema Pallidum or Mycoplasma active infection, Patients with DQ/IQ <70 (Cognitive Scale for Bayley Scale of Infant Development and Performance IQ for wppsi and wisc), Previous allogeneic HSCT or gene therapy with a different product

发起方

Ospedale San Raffaele S.r.l.