Lithium shows promise for autism social deficits in small trial

NCT ID NCT04623398

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This pilot study tested whether lithium can improve social communication in children with autism spectrum disorder who also have a SHANK3 gene mutation (Phelan-McDermid syndrome). 22 children took either lithium or a placebo for 12 weeks. Researchers measured changes in social responsiveness. The goal was to see if lithium, identified from lab studies on patient-derived stem cells, could ease core autism symptoms.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
lithium carbonate
What this could lead to
If it works, this could point toward a treatment for social communication deficits in people with autism linked to SHANK3 mutations.
What could go wrong
This was a very small pilot study with only 22 participants. Results may not apply to everyone, and lithium can have side effects like kidney or thyroid issues.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

22 people

The number who actually took part.

Started

Feb 2022

Finished

Aug 2024

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

7 to 18 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Children under 18 years of age * Minimum weight of 20 kg for children aged 7 years old * Patient with haplo deficiency SHANK3, i.e. carrier of a SHANK3 deletion (CNV) or a de novo truncating mutation in SHANK3 (Phelan McDermid syndrome); * Total Social Responsiveness Scale - T score (SRS) of at least 66 * Patients of childbearing age who are sexually active must agree to use a highly effective form of contraception (estrogen-progestin or progestin-only contraception, or an intrauterine device, or contraceptive abstinence). * Affiliation to a social security system * Signature of the consent by the holders of parental authority * Non-participation in another clinical trial * Diagnosis of Autism Spectrum Disorders (DSM-5 criteria) confirmed by Autism Diagnostic Interview-Revised (ADI-R) and Autism Diagnostic Observation Scale (ADOS-II) * IQ Assessment * Beta-HCG negative Exclusion Criteria: * Hepatic or renal insufficiency (disturbed liver function tests, abnormal creatinine clearance); * Unbalanced thyroid or diabetic pathology; * Cardiac pathology: Brugada syndrome or family history of Brugada syndrome, heart failure; * Addison's disease; * Unstable epileptic disease. * Patient with concomitant diseases judged for which the experimental treatment with Li + could compromise tolerance ; * History of allergy to Li+; * Allergy to lactose, lactose being the sole diluent and excipient of the prepared form. * Initiation of co-occurring cognitive-behavioural therapy that is specifically focused on autistic symptoms within 6 weeks prior to inclusion; * Any introduction of psychotropic drugs within 2 weeks prior to trial, including neuroleptics, monoamine oxidase inhibitors, stimulants, antidepressants. For neuroleptic drugs and Fluoxetine, this delay should be 4 weeks prior to the trial; * Serious behavioural problems or refusal to take medication that does not allow for compliance; * Inability to perform blood tests to check lithemia when the patient is included.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Hôpital Robert Debré

    Paris, 75019, France

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