Effect of lithium on autistic children

Lithium effect in patients with Autism Spectrum Disorder and Phelan-McDermid Syndrome (SHANK3 haploinsuffisance): pilot study. - LISPHEM

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2017-000825-11-FR

Enrollment

Registered

2018-06-06

Start date

Unknown

Completion date

Unknown

Last updated

2025-01-07

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Autistic Spectrum Disorder and Phelan-McDermid Syndrome MedDRA version: 21.1 Level: PT Classification code 10063844 Term: Autism spectrum disorder System Organ Class: 10037175 - Psychiatric disorders

Interventions

Product Name: carbonate de lithium 62,5mg Pharmaceutical Form: Capsule INN or Proposed INN: carbonate de lithium CAS Number: 554-13-2 Current Sponsor code: 5005635 Other descriptive name: lithium carb

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: -Children aged 7-17 years and 8 months; Nosological criteria: -Patient with haplo SHANK3 deficiency, ie carrying a deletion (CNV) SHANK3 or a de novo truncating mutation in SHANK3 (Phelan McDermid syndrome); -Total score to Social Responsiveness Scale - T score (SRS) of at least 66 -Affiliation to a social security scheme (no MEA) -Consentement of the holders of the parental authority -Non-participation in another clinical trial Are the trial subjects under 18? yes Number of subjects for this age range: 40 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: Hepatic or renal insufficiency (disturbed hepatic assessment, abnormal creatinine clearance); - Thyroid or unbalanced diabetic pathology; - Cardiac pathology: Brugada syndrome or family history of Brugada syndrome, heart failure; -Maladie of Addison; -Non-stabilized epileptic disease. Criteria for contraindications to any of the treatments studied: -Patient with concomitant diseases considered for which experimental treatment with Li + could compromise tolerance; -A history of allergy to Li +; -Lactose allergy, lactose being the only diluent and excipient of the prepared form -Start of a co-occurring cognitive-behavioral therapy that is specifically focused on autistic symptoms in the 6 weeks prior to inclusion; -Any introduction of psychotropic molecules in the 2 weeks preceding the test, including neuroleptics, monoamine oxidase inhibitors, stimulants, antidepressants. For delayed neuroleptics and Fluoxetine, this time should be 4 weeks prior to the study; - Serious behavioral problem or refusal to take medication that does not allow medication compliance; -Impossibility to practice blood tests to check the lithemia when the patient is included.

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate the effect of Li + at 12 weeks as a new treatment for social communication deficit in patients with Phelan-McDermid Syndrome (PMS);Secondary Objective: - To evaluate the effect of Li + at 12 weeks on all cardinal and main symptoms in patients with Phelan-McDemid Syndrome (PMS). - To evaluate the tolerance of Li + for 12 weeks in children with PMS. - Demonstrate the feasibility of a randomized controlled phase III study;Primary end point(s): Main Criterion: Severity of Autistic Symptoms - Social Responsiveness Scale - Total score at 12 weeks (SRS, Constantino 2003). Secondary evaluation criteria: -Scores at scales exploring aberrant, stereotyped, repetitive and obsessive behaviors, and comorbidities. -Scores at the scales evaluating the presence of treatment-related adverse effects. -Precision of the efficiency hypothesis for calculating the number of patients and validation of the placebo for blind evaluation.;Timepoint(s) of evaluation of this end point: 12 weeks	—

Measure

Time frame

Main Objective: To evaluate the effect of Li + at 12 weeks as a new treatment for social communication deficit in patients with Phelan-McDermid Syndrome (PMS);Secondary Objective: - To evaluate the effect of Li + at 12 weeks on all cardinal and main symptoms in patients with Phelan-McDemid Syndrome (PMS). - To evaluate the tolerance of Li + for 12 weeks in children with PMS. - Demonstrate the feasibility of a randomized controlled phase III study;Primary end point(s): Main Criterion: Severity of Autistic Symptoms - Social Responsiveness Scale - Total score at 12 weeks (SRS, Constantino 2003). Secondary evaluation criteria: -Scores at scales exploring aberrant, stereotyped, repetitive and obsessive behaviors, and comorbidities. -Scores at the scales evaluating the presence of treatment-related adverse effects. -Precision of the efficiency hypothesis for calculating the number of patients and validation of the placebo for blind evaluation.;Timepoint(s) of evaluation of this end point: 12 weeks

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Secondary

Measure	Time frame
Secondary end point(s): Secondary evaluation criteria: -Scores at scales exploring aberrant, stereotyped, repetitive and obsessive behaviors, and comorbidities. -Scores at the scales evaluating the presence of treatment-related adverse effects. -Precision of the efficiency hypothesis for calculating the number of patients and validation of the placebo for blind evaluation.;Timepoint(s) of evaluation of this end point: 12 weeks	—

Measure

Time frame

Secondary end point(s): Secondary evaluation criteria: -Scores at scales exploring aberrant, stereotyped, repetitive and obsessive behaviors, and comorbidities. -Scores at the scales evaluating the presence of treatment-related adverse effects. -Precision of the efficiency hypothesis for calculating the number of patients and validation of the placebo for blind evaluation.;Timepoint(s) of evaluation of this end point: 12 weeks

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Countries

France

Contacts

Public ContactDRCI Hôpital St Louis

ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)

laura.blanchet@aphp.fr

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026