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A pilot, multicentre, controlled, open-label study evaluating 24 months of lithium carbonate treatment in patients with TBR1-related neurocognitive disorder. ESALIT (Efficacy- Safety-Lithium-TBR1)
EU CTIS ID: 2024-513955-34-00
What this study is testing
After a 6 to 12 months observational period, evaluate the effect of 24 months of lithium carbonate treatment on adaptative behaviour in patients with TBR1-related neurocognitive disorder.
- Phase I and Phase II (Integrated)- Other
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Written informed consent from the patient, parent or legal representative
- ≥ 6 years old at the time of consent
- Proven pathogenic or probably pathogenic TBR1 variant (SNV confirmed by Sanger sequencing or CNV including only TBR1)
- If applicable: Stable concomitant psychoactive medication regimen (dose and schedule) ≥2 months prior to lithium initiation
- Affected individuals able to take tablet /capsules orally
- Highly effective method of contraception in affected female individuals of childbearing age (Combined hormonal contraception, progestogen-only hormonal contraception, intrauterine device, intrauterine hormone-releasing system or abstinence) during treatment and for at least 3 months after the final dose of lithium
You likely can't join if
- Renal/liver insufficiency (disturbed liver function, abnormal creatinine clearance)
- Parent/guardian incapable of expressing consent
- Person not affiliated to a national health insurance scheme
- Person subject to a court order
- Cognitivo-behavioural therapy focused on ASD in 6 weeks previous to inclusion
- Other genetic pathogenic variant associated to neurocognitive disorders
See the full eligibility criteria
- Written informed consent from the patient, parent or legal representative
- ≥ 6 years old at the time of consent
- Proven pathogenic or probably pathogenic TBR1 variant (SNV confirmed by Sanger sequencing or CNV including only TBR1)
- If applicable: Stable concomitant psychoactive medication regimen (dose and schedule) ≥2 months prior to lithium initiation
- Affected individuals able to take tablet /capsules orally
- Highly effective method of contraception in affected female individuals of childbearing age (Combined hormonal contraception, progestogen-only hormonal contraception, intrauterine device, intrauterine hormone-releasing system or abstinence) during treatment and for at least 3 months after the final dose of lithium
- 1 available parent/guardian able to attend all visits having acceptable reading skills
- Highly effective method of contraception in affected men individuals of childbearing age (condom or abstinence) during the treatment and for at least 5 days after the final dose of lithium
- Renal/liver insufficiency (disturbed liver function, abnormal creatinine clearance)
- Parent/guardian incapable of expressing consent
- Person not affiliated to a national health insurance scheme
- Person subject to a court order
- Cognitivo-behavioural therapy focused on ASD in 6 weeks previous to inclusion
- Other genetic pathogenic variant associated to neurocognitive disorders
- Any introduction of psychotropic molecules within 2 months prior to the trial, including neuroleptics, monoamine oxidase inhibitors, stimulants, antidepressants.
- Concomitant use of Angiotensin-Converting Enzyme (ACE) inhibitor, angiotensin II receptor antagonists, Nonsteroidal anti-inflammatory drugs, diuretics.
- Current lithium treatment
- Severe behavioural disorder or refusal to take drug treatment not allowing for compliance with medication;
- Impossibility to perform blood tests to check the lithiaemia when the patient is included.
- Unbalanced thyroid or diabetic pathology
- Participation in another therapeutic trial
- Long QT/Brugada syndrome or familial antecedent of Brugada syndrome, cardiac insufficiency
- Addison disease, dehydration, sodium restriction
- Non-stabilized epileptic disease.
- Patient with concomitant diseases for which the experimental treatment by lithium could alter the tolerance
- Hypersensitivity to lactose, lithium or one of its excipients
- Patient with a wheat allergy (other than celiac disease)
- Pregnant or breastfeeding woman
The study team makes the final eligibility decision.
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, 0-17 years, 65+ years, 18-64 years. The study team makes the final eligibility decision.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.