Safety and Efficacy of TSHA-102 in Adult Females With Rett Syndrome (REVEAL Adult Study)
Rett Syndrome
About this trial
This is an interventional treatment trial for Rett Syndrome focused on measuring Rett Syndrome, Neurodevelopmental disorder, Rett, MECP2
Eligibility Criteria
Inclusion Criteria: Participant has a clinical diagnosis of classical/typical Rett syndrome with a documented pathogenic mutation of the methyl-CpG-binding protein 2 (MECP2) gene that results in loss of function. Participants eligible to receive severe acute respiratory syndrome (SARS) coronavirus 2 (COVID-19) vaccination must be fully vaccinated against COVID-19. Participants must be willing to receive blood or blood products for the treatment of an AE if medically needed. Exclusion Criteria: Participant has another neurodevelopmental disorder independent of the MECP2 loss-of-function mutation, or any other genetic syndrome with a progressive course. Participant has a history of brain injury that causes neurological problems. Participant had grossly abnormal psychomotor development in the first 6 months of life. Participant has a diagnosis of atypical Rett syndrome. Participant has a MECP2 mutation that does not cause Rett syndrome. Participant requires invasive ventilatory support. Participant has contraindications for IT administration of TSHA-102 or lumbar puncture procedure, or other medical conditions, or contraindications to any medications required for IT administration. Participant has uncontrolled seizures or a history of status epilepticus within the 3 months prior to enrollment.
Sites / Locations
- Taysha Study SiteRecruiting
Arms of the Study
Arm 1
Arm 2
Experimental
Experimental
Cohort 1
Cohort 2
Participants will be randomized to treatment with TSHA-102 or delayed treated control at the starting dose. Dose Level 1
Participants will be randomized to treatment with TSHA-102 or delayed treated control at the higher dose. Dose Level 2