Efficacy and Safety of Bumetanide Oral Liquid Formulation in Children Aged From 2 to Less Than 7 Years Old With Autism Spectrum Disorder. A 6-month Randomised, Double-blind, Placebo Controlled Multicentre Parallel Group Study to Evaluate Efficacy and Safety of Bumetanide 0.5mg Twice a Day Followed by an Open Label Active 6-month Treatment Period With Bumetanide (0.5mg Twice a Day) and a 6 Weeks Discontinuation Period After Treatment Stop.

CL3-95008-002

The purpose of this study was to evaluate the efficacity and the safety of bumetanide/S95008 in the improvement of Autism Spectrum Disorder core symptoms.

2018-10-04

2021-10-26

2021-10-26

Terminated

Early phase I

211

Inclusion Criteria: * Male and female patients from 2 to less than 7 * Primary diagnosis of ASD as per Diagnostic and Statistical Manual of Mental Disorders - Fifth Edition (DSM-5) criteria * Criteria met for ASD on Autism Diagnostic Observation Schedule-Generic (ADOS-2) and Autism Diagnosis Interview Revised (ADI-R) * CGI (Clinical Global Impression) - Severity rating Score ≥ 4 * Childhood Autism Rating Scale second edition (CARS2-ST or HF) total raw score ≥ 34 * Social responsiveness Scale second edition (SRS-2) total score ≥ 66 T-Score * Absence of diagnosis of Fragile X or Rett Syndrome * Absence of any clinically significant abnormality likely to interfere with the conduct of the study according to the judgment of the investigator. Exclusion Criteria: * Patients not able to follow the study assessments defined by the protocol, with the exception of self-rating questionnaires which will be assessed by parent/legal representative/caregiver for those patients unable to complete them * Patients having a high suicidal risk according to the investigator judgement * Chronic renal dysfunction * Chronic cardiac dysfunction * Patient with unstable psychotherapy, behavioural, cognitive or cognitive-behavioural therapy * Severe electrolyte imbalance that is likely to interfere with the study conduct or evaluation

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'interventionModelDescription': '6-month, randomized, double-blind, placebo-controlled, parallel groups followed by an open label active 6-month treatment period with bumetanide.', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}}, 'enrollmentInfo': {'count': 211, 'type': 'ACTUAL'}}

2023-06-05