NCT06152237Active, not recruitingPhase 1
A Multicenter, Open Label, Randomized, Dose-Escalation and Dose-Expansion Study of the Safety, Tolerability, and Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy, for the Treatment of Pediatric Females With Rett Syndrome
Taysha Gene Therapies, Inc.5 sites in 3 countries6 target enrollmentStarted: December 12, 2023Last updated:
Conditions
Interventions
Trial Snapshot
- Phase
- Phase 1
- Status
- Active, not recruiting
- Sponsor
- Taysha Gene Therapies, Inc.
- Enrollment
- 6
- Locations
- 5
- Primary Endpoint
- Primary Safety
Study Overview
Brief Summary
The REVEAL Pediatric Study is a multi-center, Phase 1/2 open-label, dose-escalation and dose-expansion study of TSHA-102, an investigational gene therapy, in pediatric females with Rett Syndrome.
The safety, tolerability, and preliminary efficacy of two dose levels will be evaluated. The study duration is up to 6 years.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Sequential
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 5 Years to 8 Years (Child)
- Sex
- Female
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Participant has a confirmed diagnosis of classical/typical Rett Syndrome with a documented mutation of the MECP2 gene that results in loss of function.
- •Participant is between ≥5 to ≤8 years of age at the time of consent.
- •Participant must be up to date with all relevant local vaccination requirements, with last vaccination dose received at least 42 days prior to the start of the immunosuppression regimen.
- •Participant's parent/caregiver must be willing to allow participant 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 gene 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 an MECP2 mutation that does not cause Rett syndrome.
- •Participant requires non-invasive and invasive ventilatory support.
- •Participant has contraindications for IT administration of TSHA-102 or lumbar puncture procedure, other medical conditions, or contraindications to any medications required for IT administration.
- •Participant has acute or chronic hepatitis B or C infections.
Arms & Interventions
Cohort 2
Experimental
Dose Level 2
Intervention: TSHA-102 (Genetic)
Cohort 1
Experimental
Dose Level 1
Intervention: TSHA-102 (Genetic)
Outcomes
Primary Outcomes
Primary Safety
Time Frame: Baseline through week 52
The incidence of participants experiencing any treatment-emergent adverse events (AEs) and serious adverse events (SAEs)
Secondary Outcomes
- Exploratory Efficacy(Baseline through week 52)
Investigators
Study Sites (5)
Loading locations...
Similar Trials
Active, not recruiting
Phase 1
Study Of Entrectinib (Rxdx-101) in Children and Adolescents With Locally Advanced Or Metastatic Solid Or Primary CNS Tumors And/Or Who Have No Satisfactory Treatment OptionsSolid TumorsCNS TumorsNCT02650401Hoffmann-La Roche69
Recruiting
Phase 1
A Beta-only IL-2 ImmunoTherapY StudyAdvanced Solid TumorUnresectable Solid TumorTriple Negative Breast CancerNon-Small Cell Lung Cancer SquamousNon-Small Cell Lung Cancer Non-squamousColorectal Cancer (MSI-H)Squamous Cell Carcinoma of Head and NeckCutaneous Squamous Cell CarcinomaEndometrial CarcinomaBasal Cell CarcinomaClear Cell Renal Cell CarcinomaMerkel Cell CarcinomaPleural MesotheliomaSolid TumorSolid Tumor, AdultMSI-H Solid Malignant TumorCancer With A High Tumor Mutational BurdenEpithelial Ovarian CarcinomaPrimary Peritoneal CancerGastroesophageal Junction (GEJ) CancerAcral MelanomaDMMR Solid Malignant TumorFallopian Tube CancerMSI-H CancerDMMR CancerViral CancerEndometrial CancerCutaneous MelanomaPancreas Adenocarcinoma (MSI-H)Cervical CancersOvarian CancerGastric CancerCervical CancerBladder CancerEsophageal CancerSkin CancerMucosal MelanomaNCT05086692Medicenna Therapeutics, Inc.115
Completed
Phase 1
KN046 in Subjects With Advanced Solid Tumors and LymphomaAdvanced Solid TumorsLymphomaNCT03733951Jiangsu Alphamab Biopharmaceuticals Co., Ltd139
Terminated
Phase 1
A Study to Evaluate Safety, Tolerability, Dosimetry, and Preliminary Efficacy of the HER2 Directed Radioligand CAM-H2 in Patients With Advanced/Metastatic HER2-Positive Breast, Gastric, and Gastro-Esophageal Junction (GEJ) CancerAdvanced/Metastatic HER2-positive Breast, Gastric, Gastroesophageal Junction Cancer With Disease Progression Following Anti-HER2 Standard of Care TreatmentNCT04467515Precirix13
Active, not recruiting
Phase 1
Multi-Center Study of GSK2857916 in Combination With Pomalidomide and DexRelapsed and/or Refractory Multiple MyelomaNCT03715478Canadian Myeloma Research Group120