Phase 2 Epilepsy Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• Children aged 2 to 9 years, or 10 to 20 years old at the time of screening
• People with a confirmed diagnosis of SLC13A5 citrate transporter disorder, proven through genetic testing at an approved laboratory, showing specific inherited changes in the SLC13A5 gene
• People whose symptoms and clinical features are consistent with SLC13A5 citrate transporter disorder
• People (or their parent or guardian) who are willing and able to give written consent to participate and attend all required study visits and procedures; children aged 10 to 17 must also provide their own agreement based on their understanding of the study
• People who are capable of having children must use a barrier method of contraception (such as condoms) for the first 12 months after receiving the study treatment, plus at least one additional highly effective birth control method if sexually active

Who may not be able to join:

• People who are unable to take part in the study procedures, as determined by the trial site investigator
• People who have a medical condition that prevents them from safely having a lumbar puncture (a procedure to collect fluid from around the spine) or from receiving anaesthesia
• People with a history of bleeding disorders or any other condition that makes a lumbar puncture medically inadvisable according to local hospital policy
• People who cannot be safely sedated, in the opinion of the anaesthesiologist
• People who have an active infection at the time they are due to receive the study treatment
• People with another illness, or who require ongoing medication, that the lead trial doctor believes would create added risks from gene therapy
• People who are unable to have an MRI scan according to local hospital policy
• People who are unable to undergo any other procedure required by the study
• People with significant brain or behavioural conditions unrelated to SLC13A5 disorder that could affect the reliability or interpretation of study results
• People who have received another experimental drug within 30 days before screening, or who plan to receive one (other than gene therapy) during the study
• People who are already enrolled in another interventional clinical trial
• People for whom the study drug TSHA-105 or any of its ingredients is medically contraindicated
• People for whom any of the immune-suppressing medications used in this study are medically contraindicated
• People with significantly abnormal blood test results before starting gene therapy, including very low or very high haemoglobin levels, high white blood cell counts, low platelet counts, or abnormal liver, kidney, or clotting function results (confirm specific thresholds with trial site)

Important: Always verify eligibility with the trial site directly before applying.