Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

Rare disease

Metachromatic leukodystrophy

  • 6 clinical trials
Quick filters

6 Clinical trials

Metachromatic leukodystrophy

Not yet recruiting

Long‑Term Monitoring of Malignancy Risk in Early‑Onset Metachromatic Leukodystrophy Patients Treated with atidarsagene autotemcel

Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Atidarsagene Autotemcel
  • Sponsor:Orchard Therapeutics (Europe) Limited

Metachromatic leukodystrophy

Not recruiting

Study of Intrathecal Cebsulfase Alfa for Children with Late Infantile Metachromatic Leukodystrophy

InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:Cebsulfase Alfa
  • Sponsor:Shire Human Genetic Therapies Inc.

Metachromatic leukodystrophy

Not recruiting

Study on Gene Therapy with Atidarsagene Autotemcel for Patients with Metachromatic Leukodystrophy

Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years
  • Substances:Atidarsagene Autotemcel
  • Sponsor:Orchard Therapeutics (Europe) Limited

Metachromatic leukodystrophy

Not recruiting

Study on Long-Term Safety and Efficacy of Cebsulfase Alfa for Children with Late Metachromatic Leukodystrophy Receiving Intrathecal Enzyme Replacement

InvestigationalNo placebo
Countries:CzechiaCzechia
  • Participants:0–17 years
  • Substances:Cebsulfase Alfa
  • Sponsor:Shire Human Genetic Therapies Inc.

Metachromatic leukodystrophy

Not recruiting

Study on the Effects of Atidarsagene Autotemcel for Patients with Late Juvenile Metachromatic Leukodystrophy

Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years
  • Substances:Atidarsagene Autotemcel
  • Sponsor:Orchard Therapeutics (Europe) Limited

Metachromatic leukodystrophy

Not recruiting

Study of Atidarsagene Autotemcel for Treating Early Onset Metachromatic Leukodystrophy (MLD) in Patients

Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years
  • Substances:Atidarsagene Autotemcel
  • Sponsor:Orchard Therapeutics (Europe) Limited
See all trials →
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.