TSHA-105 for SLC13A5 Deficiency
(SLC13A5 Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called TSHA-105, an experimental therapy for individuals with SLC13A5 Citrate Transporter Disorder, a genetic condition affecting brain function. The study aims to determine if a single administration of this treatment into the spine is safe and effective. It seeks participants with this specific genetic disorder who exhibit related symptoms. As a Phase 1, Phase 2 trial, this research focuses on understanding how the treatment works in people and measuring its effectiveness in an initial, smaller group, offering participants a chance to contribute to groundbreaking research.
Will I have to stop taking my current medications?
The trial does not clearly state if you need to stop your current medications, but it mentions that if you have a chronic condition requiring ongoing medication, it might pose unnecessary risks for the study. It's best to discuss your specific medications with the trial team.
Is there any evidence suggesting that TSHA-105 is likely to be safe for humans?
Research shows that TSHA-105 is undergoing its first safety tests in humans. This gene therapy targets SLC13A5 deficiency, a condition that can cause seizures and developmental issues. Although human data is not yet available, animal studies have shown positive results, prompting human trials.
As an early-stage clinical trial, the safety of TSHA-105 in humans receives close monitoring. These initial trials focus on ensuring participant safety. While specific human data is not yet available, the trial's progress indicates confidence in its potential safety.
Participants will receive the treatment through an injection into the spinal canal, a common method for delivering gene therapies. As with any new treatment, side effects may occur, but these are still under study. The trial aims to gather clearer information on how well participants tolerate this treatment.12345Why do researchers think this study treatment might be promising?
TSHA-105 is unique because it targets the underlying genetic cause of SLC13A5 deficiency, a rare disorder that affects brain metabolism. Unlike standard treatments that mainly address symptoms like seizures, TSHA-105 is designed to deliver a corrected version of the gene directly to the brain cells. This approach offers the potential to not just manage symptoms but to actually modify the disease itself, which is why researchers are excited about its possibilities.
What evidence suggests that TSHA-105 might be an effective treatment for SLC13A5 Deficiency?
Research has shown that TSHA-105, a gene therapy administered through the spinal fluid, appears promising in early studies. In mice lacking the SLC13A5 gene, TSHA-105 significantly lowered blood citrate levels and reduced seizures. SLC13A5 Citrate Transporter Disorder is a rare condition affecting brain development and causing seizures. This treatment targets the SLC13A5 gene, which facilitates the movement of citrate, an important molecule for energy production. Although research in humans is ongoing, early results from animal studies are encouraging.12367
Are You a Good Fit for This Trial?
This trial is for individuals with SLC13A5 Citrate Transporter Disorder. Specific eligibility criteria are not provided, but typically include factors like age range, disease severity, and overall health status.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Single lumbar intrathecal administration of TSHA-105 to assess safety and efficacy
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- TSHA-105
Find a Clinic Near You
Who Is Running the Clinical Trial?
TESS Research Foundation
Lead Sponsor