Other Clinical Studies
- Read moreThe TREE Clinical Study in Rett Syndrome
The TREE trial is a fully recruited phase 2a, double-blind and placebo-controlled clinical study that has been designed to assess the safety and tolerability of leriglitazone in paediatric patients with Rett Syndrome (RTT).
The TREE study enrolled 23 female patients aged between 5 and 12 years who present with classical or typical Rett syndrome (RTT) based on the 2010 criteria, and with a confirmed mutation in the MECP2 gene. Participants receive either placebo or leriglitazone and are closely monitored for safety. Additionally, patients will undergo assessments using different Rett scales and questionnaires to assess preliminary efficacy results. Top-line results from TREE will be available by the end of 2026.
For further information visit:
https://euclinicaltrials.eu/ctis-public/view/2024-514684-26-00?lang=en
Hospitals involved in TREE Clinical StudySee mapHospital Sant Joan de DéuBarcelonaSpainHospitals involved in TREE Clinical StudySee list
Hospital Sant Joan de DéuBarcelonaSpainHideRead moreThe FRAMES Clinical Study in FRDAThe FRAMES trial is a completed phase 2 randomized, double-blind, placebo-controlled, clinical study that was designed to assess the efficacy and safety of leriglitazone in patients with Friedreich's ataxia (FRDA) with input from EU and US clinical experts and patient advocacy groups.
The study enrolled 36 patients and concluded in 2020. Results showed clinical benefit in this population including stopping brain Iron accumulation (QSM) (Pandolfo et al., 2022).
For further information visit:
https://clinicaltrials.gov/ct2/show/NCT03917225?term=MIN102&rank=2Hospitals involved in FRAMES Clinical StudySee mapHôpital Erasme-ULBBrusselsBelgiumICM, Groupe Hospitalier Pitié SalpêtrièreParisFranceRWTH Aachen UniversityAachenGermanyHospital Universitario La PazMadridSpainHospital Sant Joan de DéuBarcelonaSpainHospitals involved in FRAMES Clinical StudySee list
Hôpital Erasme-ULBBrusselsBelgiumICM, Groupe Hospitalier Pitié SalpêtrièreParisFranceRWTH Aachen UniversityAachenGermanyHospital Universitario La PazMadridSpainHospital Sant Joan de DéuBarcelonaSpainHide
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