- Ongoing, recruitingTherapeutic exploratory (Phase II)
A Global, Multicenter, Single-arm, Matched External Control Study of Intrathecal SHP611 in Subjects with Late Infantile Metachromatic Leukodystrophy
This study is looking into a new treatment called rhASA for Late Infantile Metachromatic Leukodystrophy (MLD), a serious inherited illness. It's testing if rhASA can help children keep their ability to move and whether it can slow down the disease's progression.
For: Late Metachromatic Leukodystrophy (MLD)Germany · France · Belgium - Ongoing, recruitingPhase I and Phase II (Integrated)- Other
An Open-Label Extension of Study HGT-MLD-070 Evaluating Long Term Safety and Efficacy of Intrathecal Administration of HGT-1110 in Patients with Metachromatic Leukodystrophy
This study is looking at a new treatment called rhASA for a rare genetic condition in children and young people called Metachromatic Leukodystrophy (MLD). It's checking if the treatment is safe and if it helps with MLD symptoms over a long time.
For: Late Metachromatic Leukodystrophy (MLD)Czechia · Denmark · Germany