Safety and Efficacy of TSHA-102 in Adolescent and Adult Females with Rett Syndrome (REVEAL Adult Study)

Participation Requirements

• Sex:

  FEMALE

• Eligible Ages:

  12 and up

Participation 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 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.