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New treatment option for SLC13A5 Citrate Transporter Disorder
Official title Intrathecal Gene Therapy For SLC13A5 Citrate Transporter Disorder
ClinicalTrials.gov ID: NCT07102524
What this study is testing
What is TSHA-105?
TSHA-105 is an investigational medicine, being studied as a potential treatment for slc13a5 citrate transporter disorder.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- Phase 1/2, open-label study to assess the efficacy and safety of a single lumbar intrathecal administration of TSHA-105 in individuals with SLC13A5 Citrate Transporter Disorder
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 20
You may be able to join if
- Male and females between the ages of 2 to 9 or 10 to 20 years at the time of screening
- Confirmed diagnosis of SLC13A5 citrate transporter disorder by genomic DNA mutation analysis (confirmed by a CLIA certified, CE-marked, or equivalent...
- Clinical features consistent with SLC13A5 citrate transporter disorder
- Written informed consent provided by subject/parent/guardian and willingness to participate and comply with all the study related visits and...
- people able to reproduce must use a barrier method of contraception for the first 12 months after dosing as well as at least one additional highly...
You likely can't join if
- Inability to participate in study procedures (as determined by the site investigator)
- Presence of a concomitant medical condition that precludes lumbar puncture (LP) or use of anesthetics
- History of bleeding disorder or any other medical condition or circumstance in which lumbar puncture is contraindicated according to local...
- Inability to be safely sedated in the opinion of the clinical anesthesiologist
- Active infection, at the time of dosing, based on clinical observations
- Concomitant illness or requirement for chronic drug treatment that in the opinion of the PI creates unnecessary risks for gene transfer
See the full eligibility criteria
- Male and females between the ages of 2 to 9 or 10 to 20 years at the time of screening
- Confirmed diagnosis of SLC13A5 citrate transporter disorder by genomic DNA mutation analysis (confirmed by a CLIA certified, CE-marked, or equivalent lab) demonstrating homozygous or compound heterozygous, confirmed...
- Clinical features consistent with SLC13A5 citrate transporter disorder
- Written informed consent provided by subject/parent/guardian and willingness to participate and comply with all the study related visits and procedures. Assent provided by children 10 to 17 years old based on their...
- people able to reproduce must use a barrier method of contraception for the first 12 months after dosing as well as at least one additional highly effective birth control method if sexually active
- Inability to participate in study procedures (as determined by the site investigator)
- Presence of a concomitant medical condition that precludes lumbar puncture (LP) or use of anesthetics
- History of bleeding disorder or any other medical condition or circumstance in which lumbar puncture is contraindicated according to local institutional policy
- Inability to be safely sedated in the opinion of the clinical anesthesiologist
- Active infection, at the time of dosing, based on clinical observations
- Concomitant illness or requirement for chronic drug treatment that in the opinion of the PI creates unnecessary risks for gene transfer
- Inability of the subject to undergo MRI according to local institutional policy
- Inability of the subject to undergo any other procedure required in this study
- The presence of significant non-SLC13A5 related CNS impairment or behavioral disturbances that would confound the scientific rigor or interpretation of results of the study
- Have received an investigational drug within 30 days prior to screening or plan to receive an investigational drug (other than gene therapy) during the study.
- Enrollment and participation in another treatment clinical trial
- Contraindication to TSHA-105 or any of its ingredients
- Contraindication to any of the immune suppression medications used in this study
- Clinically significant abnormal laboratory values (hemoglobin \ 20 g/dL; white blood cell \> 20,000 per cmm, platelets count \ ULN; GGT, ALT, and AST or total bilirubin \> 2x ULN, creatinine ≥ 1.5 mg/dL) prior to gene...
The study team makes the final eligibility decision.
Where it's taking place
- Dallas, Texas, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years to 20 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Dallas, Texas, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.