Participate in clinical studies

The ROSA Study has started!

The ROSA study for children with Angelman syndrome started in June 2021. In this study we are looking for suitable and feasible outcome measures for children with Angelman syndrome, and we want to learn more about the natural history of this rare syndrome.

The TRAIN-study

In the TRAIN-study, we will conduct a study with trametinib in adult NF1 patients with plexiform neurofibromas that cause complaints.

SPECIAL X

Research on the social and mental skills and autism characteristics in children with Fragile X Syndrome.

Afgeronde klinische studies (deelname gesloten)

NEPTUNE TRIAL

Phase 3 clinical study of the effect of the drug OV101 (Gaboxadol) in children with Angelman Syndrome

EXCEL TRIAL

Phase 2 and 3 clinical study of the effect of Lamotrigine on cognition in patients with NF1

COVANT STUDY

Cognitive variability in NF1 and TSC monozygotic twins

GWPCARE6 TRIAL

Phase 3 clinical study of the effect of Cannabidiol on epilepsy in patients with TSC

RAPIT TRIAL

Phase 2 and 3 clinical study of the effect of the drug Sirolimus in children with TSC.

RATE TRIAL

Fase 2 en 3 klinische studie naar het effect van het medicijn Sirolimus in kinderen met TSC.

SIMCODA TRIAL

Phase 3 clinical study of the effect of the drug Simvastatin in children with NF1

NFSIMVA TRIAL

Phase 3 clinical study of the effect of the drug Simvastatin in patients with NF1