Metachromatic Leukodystrophy (MLD) research hub
3 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.
Italy - active not recruitingPHASE2
A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy
This study is looking at a new medicine called SHP611 for children with Metachromatic Leukodystrophy (MLD). Doctors want to see if SHP611, given into the spinal fluid, can help children keep their ability to move around for longer. It also looks at how it affects speech and if it's safe.
United States · Argentina · Belgium - 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.
Italy