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Gene therapy study with autologous hematopoietic stem cells for patients affected by MPS-IH
EU CTIS ID: 2024-514870-29-00
What this study is testing
To evaluate the safety and tolerability of autologous CD34+ cell enriched fraction that contains HSPC transduced with lentiviral vector (LVV) encoding the IDUA gene in pediatric patients with MPS-IH following a myeloablative and lymphoablative conditioning regimen.
- Phase I and Phase II (Integrated)- First administration to humans
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Written informed consent by parent/legal guardian
- Sex: Males and Females
- ≥ 28 days and ≤ 11 years old
- Biochemically and molecularly proven MPS-IH
- Lansky Index > 80 %
- Indication to HSCT
You likely can't join if
- Use of other investigational agents within 4 weeks prior to study enrolment (within 6 weeks if use of long-acting agents)
- Controindications to Products equivalent to the IMP (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
See the full eligibility criteria
- Written informed consent by parent/legal guardian
- Sex: Males and Females
- ≥ 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) human leukocyte antigens (HLA) -matched sibling donor or a ≥7/8 (4 digits high-resolution typing) HLA-matched cord blood donor with a cellularity ≥5x10^7 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
- Use of other investigational agents within 4 weeks prior to study enrolment (within 6 weeks if use of long-acting agents)
- Controindications to Products equivalent to the IMP (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 (also referred as, “cognitive standard score”, measured using 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
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.