All treatments
Treatment
Libmeldy 2-10 x 10^6 cells/mL dispersion for infusion clinical trials
2 indexed studies · 0 currently recruiting
- Ongoing, recruitingPhase I and Phase II (Integrated)- Other
A Phase I/II clinical trial of hematopoietic stem cell gene therapy for the treatment of Metachromatic Leukodystrophy
This study looks at a new gene therapy, Libmeldy, for Metachromatic Leukodystrophy (MLD). It's testing if the treatment is safe and if it helps improve movement and how well the body works, aiming to slow down the disease.
For: Metachromatic Leukodystrophy (MLD)Italy - Ongoing, recruitingTherapeutic confirmatory (Phase III)
A single arm, open label, clinical study of cryopreserved autologous CD34+ cells transduced with lentiviral vector containing human ARSA cDNA, for the treatment of early onset Metachromatic Leukodystrophy (MLD)
This study is testing a new gene therapy called Libmeldy for children with Metachromatic Leukodystrophy (MLD). It looks at how well the treatment helps with movement and overall health over 24 months, as well as how safe it is.
For: Metachromatic Leukodystrophy (MLD)Italy