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This study evaluates a gene therapy in which patients' own stem cells are genetically modified with a lentiviral vector carrying the IDUA gene, for patients with Mucopolysaccharidosis Type I, Hurler variant. It includes patients from 28 days to 11 years old with a biochemically and molecularly confirmed diagnosis. It is in Phase 1/Phase 2 and is active, not recruiting.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * Written informed consent by parent/legal guardian * Sex: Males and Females * Age: ≥ 28 days and ≤ 11 years old * Biochemically and molecularly proven MPS IH * Lansky index \>80% * Indication to hematopoietic stem cell transplant * 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 10\^7 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 Exclusion Criteria: * Use of other investigational agents within 4 weeks prior to study enrolment (within 6 weeks if use of long-acting agents) * Severe, active viral, bacterial or fungal infection at eligibility evaluation * Patients affected by 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 * Previous allogeneic hematopoietic stem cells transplantation or gene therapy with a different product * Contraindications to PeIMP (G-CSF, Plerixafor, Busulfan, Fludarabine, Rituximab)