Evaluation of the Efficiency of Treatment by BUMETANIDE on Autistic Children With a Known Ethiology

NCT02947880 | Withdrawn Phase 2 DMC

• 1 other identifier: I14039
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

During a previous therapeutic trial, investigators showed that the bumetanide improved significantly autism. This trial showed that a therapeutic response was obtained in 75% of cases. These first results were reinforced by a study led with adult patients for whom the eye tracking measurements as well as the functional MRI showed a diminution of the response time and a modification (amplification) of the cerebral response during an emotions recognition test. Finally, investigators confirmed the physiological mechanism behind the action of the bumetanide in a study in two mouse models of autism.

Conditions

Autistic Disorder

Keywords

autism, Bumetanide

Outcome Measures

Primary Outcomes (1)

• Change (evolution) between day 0 and day 99 of the result of the scale CARS (Childhood Autism Rating Scale).

  Day 0 and Day 99

Secondary Outcomes (1)

• CARS (Childhood Autism Rating Scale) between D0 and D99 and between D99 and D190 which will be describe by etiology

  Day 0, Day 99 and Day 190

Study Arms (2)

Bumetanide group

EXPERIMENTAL

During 3 months in the double blind, the patient will receive the experimental treatment. For the patient of 25kg and more the bumetanide is used at the posology of 1mg in the morning and 1mg in the evening, for patient under 25kg the posology is 0.5mg in the morning and 0.5mg in the evening. After the 3 months in the double blind trial (bumetanide versus placebo), all the patient will receive (in the open phase of the trial) the bumetanide during 3 months with the posology fitting with their weights.

Drug: Bumetanide

Placebo group

PLACEBO COMPARATOR

During 3 months in the double blind, the patient will receive the placebo. For the patient of 25kg and more the bumetanide is used at the posology of 1mg in the morning and 1mg in the evening, for patient under 25kg the posology is 0.5mg in the morning and 0.5mg in the evening. After the 3 months in the double blind trial (bumetanide versus placebo), all the patient will receive (in the open phase of the trial) the bumetanide during 3 months with the posology fitting with their weights.

Drug: Placebo

Interventions

Bumetanide DRUG

For the patient of 25kg and more the bumetanide is used at the posology of 1mg in the morning and 1mg in the evening, for patient under 25kg the posology is 0.5mg in the morning and 0.5mg in the evening.

Bumetanide group

Placebo DRUG

For the patient of 25kg and more the placebo is used at the posology of 1mg in the morning and 1mg in the evening, for patient under 25kg the posology is 0.5mg in the morning and 0.5mg in the evening.

Placebo group

Eligibility Criteria

• Age: 5 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Children and teenager from age 5 to age 17, with a diagnosis of typical autism or Asperger syndrome according to the criteria of diagnosis of the WHO's classification (CIM-10),
• With a known etiology,
• Patients for whom the CARS results are strictly Superior or equal to 30,
• Of whom the parents have given their free, informed and written consent,
• Affiliated or beneficiary of the French social security.

You may not qualify if:

• Patients under treatment by inlet diuretic either at the time of the study or before,
• Patients with electrolytic disorders,
• Patients with a known hypersensitivity to sulfa drugs,
• Patients with a hepatic or renal failure,
• Patients with an epilepsy not controlled by a treatment (comitial crisis in the past 6 month at the time the trial starts despite a treatment),
• Patients under treatment by psychotropic exception made of the melatonin,
• Allergy to the bumetanide or one of its excipients,
• Patient under a treatment by lithium, diphemanil, erythromycin IV, halofantrine, pentamidine, sultopride, vincamine, aminoglycoside,
• Pregnant and lactating women.
• QT prolongation noticed on the ECG at Day0,
• Anomaly on the biological check up (Day 0) made before including the patient that would contraindicated the prescription of bumetanide,
• Patients for whom the CARS results are strictly inferior to 30.

Sponsors & Collaborators

• University Hospital, Limoges: lead

MeSH Terms

Conditions

Autistic Disorder

Interventions

Bumetanide

Condition Hierarchy (Ancestors)

Autism Spectrum Disorder; Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Sulfonamides; Amides; Organic Chemicals; meta-Aminobenzoates; Aminobenzoates; Benzoates; Acids, Carbocyclic; Carboxylic Acids; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Sulfones; Sulfur Compounds

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 25, 2016
• First Posted: October 28, 2016
• Study Start: December 1, 2016
• Primary Completion: December 1, 2019
• Study Completion: June 1, 2020
• Last Updated: June 2, 2021

Record last verified: 2017-08