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Other Clinical Studies
- The TREE Clinical Study in Rett Syndrome
The TREE trial is a phase 2a, double-blind, placebo-controlled clinical study designed to assess the safety and tolerability of leriglitazone in paediatric patients with Rett Syndrome (RTT).
The TREE study aims to recruit 24 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 will receive either placebo or leriglitazone and will be closely monitored for safety. Additionally, patients will undergo assessments using different scales and questionnaires to assess preliminary efficacy results.
Hospitals involved in TREE Clinical StudySee mapHospital Sant Joan de DéuBarcelonaSpainHospitals involved in TREE Clinical StudySee listHospital Sant Joan de DéuBarcelonaSpainHideThe FRAMES Clinical Study in FRDAThe FRAMES trial was a 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 recruited 36 patients and results showed clinical benefit on this population.
Preclinical data showing potential therapeutic action and protection against neurodegenaration, in particular againts Friedereich's Ataxia, was published in Neurobiology of Disease: PPAR gamma agonist leriglitazone improves frataxin-loss impairments in cellular and animal models of Friedreich Ataxia - ScienceDirect
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 listHôpital Erasme-ULBBrusselsBelgiumICM, Groupe Hospitalier Pitié SalpêtrièreParisFranceRWTH Aachen UniversityAachenGermanyHospital Universitario La PazMadridSpainHospital Sant Joan de DéuBarcelonaSpainHide
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