BridgeMD
Hematopoietic stem cell gene therapy study
EU CTIS ID: 2024-515253-25-00
What this study is testing
Evaluation of the safety of gene therapy in MLD subjects, considering both the conditioning regimen safety and the safety of LV-transduced cell infusion, short and long-term after the treatment (for details see safety primary endpoint);"Evaluation of the efficacy of gene therapy, assessed as reduction in the progression of the clinical motor impairment in treated subjects as compared to the progression measured in untreated MLD patients in our disease natural history study, accompanied by a significant increase of residual ARSA activity as compared to pre-treatment subjects’ values. Motor functions will be measured by the clinically relevant GMFM scoring system (for details see efficacy primary endpoint). Indeed, there is a clear causal relationship between the potential beneficial outcome measured with the GMFM and the treatment, being motor impairment consequent to the involvement of both central and peripheral nervous system, and less influenced by other variables. Residual ARSA activity will be measured on hematopoietic cells (PBMC and BM cells)
- Phase I and Phase II (Integrated)- Other
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
- Pre-symptomatic late infantile patients;
- Pre- or early-symptomatic early juvenile patients;
- Parental/guardian/patientsigned informed consent.
You likely can't join if
- • HIVRNA and/or HCVRNA and/or HBVDNA-positive patients; • Patients affected by neoplastic diseases; • Patients with cytogenetic alterations typical of MDS/AML; • Patients with end-organ functions or any other severe disease which, in the judgment of the investigator, would make the patient inappropriate for entry into this study; • Patients enrolled in other trials;
- • Patients who underwent allogeneic hematopoietic stem cell transplantation in the previous 6 months; • Patients who underwent allogeneic hematopoietic stem cell transplantation with evidence of residual cells of donor origin.
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.