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A Phase III Open-Label, Single-Group, Extension Study to
Obtain Long-Term Safety and Tolerability Data of
Idebenone in the Treatment of Friedreich*s Ataxia Patients

- To gather long-term data on the safety and tolerability of idebenone inFriedreich*s Ataxia- To explore the effect of idebenone after longer term administration onneurological symptoms and signs as assessed by the InternationalCooperative Ataxia…

Tamoxifen in Duchenne muscular dystrophy - TAMDMD:
A multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial
The study will be extended to an open label study with the following title (OLE: Open Label Extension):
Tamoxifen in Duchenne muscular dystrophy - TAMDMD:
A 48 week open label extension of a multi centre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy trial

To test if tamoxifen treatment, compared to placebo, reduces the progression of the disease in 6.5-12 years old ambulant DMD patients by at least 50% (using the MFM D1 subscore as primary clinical endpoint in group A patients).To test if tamoxifen…

Open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies.

This study has been transitioned to CTIS with ID 2023-504520-26-00 check the CTIS register for the current data. Primary objective:To assess the long term safety and tolerability of GIVINOSTAT in patients with DMD following core protocols program…

A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of
Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe
Disease

This study has been transitioned to CTIS with ID 2023-505170-15-00 check the CTIS register for the current data. The objective of the study is to assess the long-term safety and efficacy of intravenous ATB200 co-administration with oral AT2221 in…