ALD-104 (CALD) Clinical study of the Lenti-D drug product (gene therapy) in boys with cerebral adrenoleukodystrophy - temporarily closed for inclusion
MetVasA (SCT survivors) Clinical study of specific late effects of hematopoietic stem cell transplantation in childhood - open for inclusion
Long-term HIT (Stem cell transplantation) Clinical study investigating blood production in stem cell transplantation survivors - open for inclusion
OMS721-HCT-002 narsoplimab (high-risk HSCT-TMA) Clinical study of narsoplimab in children with thrombotic microangiopathy following allogeneic hematopoietic stem cell transplantation - open for inclusion
OTL-203-02 / HURCULES (MPS-IH) Clinical study of a new gene therapy (OTL-203) compared to a standard treatment as a safe and effective alternative to treat mucopolysaccharidosis Type I, Hurler syndrome (MPS-IH) - open for inclusion
PIKACHU (Stem cell transplantation) Clinical study of precision dosing of prednisolone as either prophylaxis or treatment of graft-versus-host disease after hematopoetic stem cell transplantation - open for inclusion
SCRIPT-AML (Stem cell transplantation in Acute Myeloid Leukemia) Clinical study of conditioning regimen in stem cell transplantation - open for inclusion
VANISH Clinical study of scans of fungal infections in the lungs - open for inclusion