Efficacy and Safety of Bumetanide Oral Liquid Formulation in Children Aged From 2 to Less Than 7 Years Old With Autism Spectrum Disorder.

Phase 3TerminatedNCT03715153

Institut de Recherches Internationales Servier211 enrolled

Overview

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.

The present study (CL3-95008-002) was performed in children from 2 to less than 7 years old presenting with ASD. A 6-month double-blind treatment period was performed in which efficacy and safety of bumetanide 0.5mg BID were assessed versus placebo. This double-blind period was followed by a 6-month open label treatment period of bumetanide 0.5mg twice daily in which long term safety was evaluated.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

211

Conditions

Autism Spectrum Disorder (ASD)

Interventions

BUMETANIDE (S95008) for week 0 - 26DRUG

Oral solution dosed at 0.5 mg/mL Taken twice daily.

PLACEBO for week 0 - 26DRUG

Oral solution Taken twice daily.

Open-Label BUMETANIDE (S95008) for weeks 26 - 52DRUG

Oral solution dosed at 0.5 mg/mL Taken twice daily.

Eligibility

Sex: ALLMin age: 2 YearsMax age: 6 Years

Medical Language ↔ Plain English

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

Locations (50)

Outcomes

Primary Outcomes

Childhood Autism Rating Scale, Second Edition (CARS2) Total Raw Score

The CARS2 total raw score range from 15 to 60. This scale is a behaviour rating scale intended to diagnose autism. A total score of 15 indicates that an individual behaviour is within normal limits, whereas a value of 60 indicates that the individual's behaviour is severly abnormal. In term of change from baseline, the greater the mean value decreases, the better it is.

Time frame: Change from baseline to Week 26

Secondary Outcomes

Social Responsiveness Scale, Second Edition (SRS-2) Total Raw Score

The SRS-2 total raw score serves as an index of severity of social deficits in the autism spectrum. The total raw score ranges from 65 to 260. A value of 65 represents no symptoms disorders, a value of 260 represents a severe autism spectrum disorder. In terms of change from baseline, the greater the mean value decreases, the better it is.

Time frame: Change from baseline to Week 26

Clinical Global Impression - Global Improvement (CGI-I) Score

Scale which assesses the severity of the illness and the global improvement of the patient under study treatment. It ranges from 1 (normal) through to 7 (amongst the most severely ill patients).

Time frame: At Week 26

Vineland Adaptative Behaviour Scale II (VABS II)

Scale designated to measure adaptative behaviour The scale for behaviour ranges from 1 to 67. The more the score decreases, the better it is.

Time frame: Change from baseline to Week 26

Number of Patients With Abnormalities in 12-leads Electrocardiogram (ECG) Parameters

Number of patients with clinically significant ECG abnormalities The 12-lead electrocardiogram (ECG) is a medical test that is recorded using leads, or nodes, attached to the body. Electrocardiograms (ECGs), capture the electrical activity of the heart and transfer it to graphed paper where abnormalities are reported and interpretated by the cardiologist.

Data from ClinicalTrials.gov

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