Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-25 @ 1:34 AM
Ignite Modification Date:
2025-12-25 @ 1:34 AM
NCT ID:
NCT03488394
Eligibility Criteria:
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)
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
28 Days
Maximum Age:
11 Years
Study:
NCT03488394
Study Brief:
Protocol Section:
NCT03488394