Autism Spectrum DisorderPhase 2NCT03156153
A Study of Bumetanide for the Treatment of Autism Spectrum Disorders
A Phase 2 study testing Bumetanide in people with Autism Spectrum Disorder, run by Xinhua Hospital, Shanghai Jiao Tong University School of Medicine at 1 site in China.
Trial summary
The investigators are going to carry out a randomized double-blind placebo-controlled trial to study the efficiency and mechanism of bumetanide on the treatment of children with Autism Spectrum Disorder. Read more about this study
- Who is it for?
- Ages 3 to 6 years
- Conditions the study is for
- Autism Spectrum Disorder
- What is being tested?
- Bumetanide
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 120 participants
- Where
- 1 siteChina
- Study dates
- 24 May 2017to 10 Jul 2019
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 6 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- The patients, aged from 3 to 6 years old, were given the diagnosis of ASD according to the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-V) , by a team of autism experts; Scores for Children Autism Rating Scale (CARS) were more than 30; Signed Informed Consents were provided by parents.
Who cannot join
- Liver and kidney dysfunction; With a history of allergy to sulfa drugs; abnormal ECG; chromosomal abnormality; suffering from nervous system diseases (such as epilepsy, schizophrenia, and so on); using the melatonin treatment for sleep disorders or withdrawal less than three weeks.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Study of Bumetanide for the Treatment of Children With Autism Spectrum Disorder：a Randomized Double-blind Placebo-controlled Trial
The investigators are going to carry out a randomized double-blind placebo-controlled trial to study the efficiency and mechanism of bumetanide on the treatment of children with Autism Spectrum Disorder.
Read the detailed description
In consideration of the increasing number of autistic children and poor intervention effect in China, it is an urgent to find some effective medicine. Some studies have reported bumetanide, a classic diuretic, could improve autistic behaviors in both animal model and humans; while the efficiency of bumetanide on Chinese autistic chilren is unkonwn and the underlying mechanisms remain unfolding. The investigators aim at investigating whether bumetanide would improve the clinical symptoms in Chinese children with autism within a safe dosage and further study the physiological mechanism beneath.The investigators will regularly assess the participants' autism-related symptoms during medication, as well as the adverse effects of each patient. The investigators will carry out genome-wide association analysis (GWAS) from blood sample, related metabolites in nervous system and compare the concentration of the neurotransmitter in autistic brain before and after 3 months' treatmeat,and also will collect the EEG signal in autistic chilldren when the participants performing certain tasks before and after 3 months' treatment.
- Conditions
- Autism Spectrum Disorder
- Being tested
- Bumetanide, Placebo
- Medical subject headings
- Autism Spectrum Disorder
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BumetanideDrug
bumetanide tablets, oral intake, 0.5mg, twice daily, respectively at 8 am and 4 pm
PlaceboDrug
placebo tablets, oral intake, 0.5mg, twice daily, respectively at 8 am and 4 pm
| Study group | Role | What they receive |
|---|---|---|
| Bumetanide group | Experimental | Double-blind phase: in the first 3 months, patients will receive the experimental treatment - bumetanide, oral intake, 0.5mg/time, twice a day; Open-label phase: after 3 month double-blined treatment, all the patients will receive 3-month bumetanide treatment - oral intake, 0.5mg/time, twice a day. Receives: Drug: Bumetanide |
| Control group | Placebo comparator | Double-blind phase: in the first 3 months, patients will receive the placebo - oral intake, 0.5mg/time, twice a day; Open-label phase: after 3 month double-blined treatment, patients in this group will receive 3-month bumetanide treatment - oral intake, 0.5mg/time, twice a day. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Childhood Autism Rating Scale(CARS)
CARS is a behaviour-rating scale used to assess the presence and severity of the symptoms of autism spectrum disorder
Measured: Day 0 and Day 90
Secondary outcomes (6)
Clinical Global Impressions Scale (CGI)
CGI is a scale used to assess the severity of the illness and the global improvement of the patient under intervention
Measured: Day 0 and Day 90
Social Responsiveness Scale (SRS)
SRS is a questionnaire used to assess the presence and severity of social impairment.
Measured: Day 0 and Day 90
Autism Diagnostic Observation Schedule (ADOS)
The ADOS is a structured play session conducted by clinicians, with the total score combines the Social and Communication domain items.
Measured: Day 0 and Day 90
Short Sensory Profile Report
The Sensory Profile is a measure of children's responses to sensory events in daily life. The caregiver completes the Sensory Profile by assessing the frequency of the child's responses to certain sensory processing, modulation, and behavioral/emotional events as described in the 125 items.
Measured: Day 0 and Day 90
Symbolic Play Test
Symbolic Play Test is a nonverbal measure of symbolic functioning in participants aged 12-36 months. The test does not require any expressive speech, and is therefore appropriate for use with all participants with ASD. Participants are sequentially presented with four sets of toys, and their spontaneous manipulation of the objects is observed and recorded on a standardized checklist.
Measured: Day 0 and Day 90
Chinese Communicative Development Inventory
The Chinese Communicative Development Inventories (CCDI) is a questionnaire that is used to direct measures of the participants' language.Change between day 0 and day 90 of the result of the Chinese Communicative Development Inventory
Measured: Day 0 and Day 90
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 May 2017
- Study start24 May 2017
- Primary completion8 Jul 2019
- Study completion10 Jul 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autism Spectrum DisorderStudies of the same condition, open and under way.
- More trials run by Xinhua Hospital, Shanghai Jiao Tong University School of MedicineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 120 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Shanghai Jiao Tong University School of Medicine
- Responsible party
- Sponsor
- Investigators
- Fei Li, doctor, Principal investigator, Xinhua hospital Affilated to Shanghai Jiao Tong University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03156153
- Sponsor's protocol ID
- XH-16-048
- Status last verified
- Jul 2019
- First submitted
- 15 May 2017
- Last updated
- 1 Mar 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 1 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study of Bumetanide for the Treatment of Children With Autism Spectrum Disorder：a Randomized Double-blind Placebo-controlled Trial
- ClinicalTrials.gov ID
- NCT03156153
- Acronym / sponsor ID
- XH-16-048
- Conditions
- Autism Spectrum Disorder
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 120 participants (actual)
- Lead sponsor
- Xinhua Hospital, Shanghai Jiao Tong University School of MedicineUniversity, hospital or other
- Collaborators
- Shanghai Jiao Tong University School of Medicine
- Study start
- 24 May 2017 (actual)
- Primary completion
- 8 Jul 2019 (actual)
- Study completion
- 10 Jul 2019 (actual)
- First posted
- 17 May 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The investigators are going to carry out a randomized double-blind placebo-controlled trial to study the efficiency and mechanism of bumetanide on the treatment of children with Autism Spectrum Disorder.
Detailed description
In consideration of the increasing number of autistic children and poor intervention effect in China, it is an urgent to find some effective medicine. Some studies have reported bumetanide, a classic diuretic, could improve autistic behaviors in both animal model and humans; while the efficiency of bumetanide on Chinese autistic chilren is unkonwn and the underlying mechanisms remain unfolding. The investigators aim at investigating whether bumetanide would improve the clinical symptoms in Chinese children with autism within a safe dosage and further study the physiological mechanism beneath.The investigators will regularly assess the participants' autism-related symptoms during medication, as well as the adverse effects of each patient. The investigators will carry out genome-wide association analysis (GWAS) from blood sample, related metabolites in nervous system and compare the concentration of the neurotransmitter in autistic brain before and after 3 months' treatmeat,and also will collect the EEG signal in autistic chilldren when the participants performing certain tasks before and after 3 months' treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Bumetanide group | Experimental | Drug: BumetanideDouble-blind phase: in the first 3 months, patients will receive the experimental treatment - bumetanide, oral intake, 0.5mg/time, twice a day; Open-label phase: after 3 month double-blined treatment, all the patients will receive 3-month bumetanide treatment - oral intake, 0.5mg/time, twice a day. |
| Control group | Placebo comparator | Drug: PlaceboDouble-blind phase: in the first 3 months, patients will receive the placebo - oral intake, 0.5mg/time, twice a day; Open-label phase: after 3 month double-blined treatment, patients in this group will receive 3-month bumetanide treatment - oral intake, 0.5mg/time, twice a day. |
Who may qualify
- Ages
- 3 to 6 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- The patients, aged from 3 to 6 years old, were given the diagnosis of ASD according to the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-V) , by a team of autism experts; Scores for Children Autism Rating Scale (CARS) were more than 30; Signed Informed Consents were provided by parents.
Key exclusion criteria (1 listed)
- Liver and kidney dysfunction; With a history of allergy to sulfa drugs; abnormal ECG; chromosomal abnormality; suffering from nervous system diseases (such as epilepsy, schizophrenia, and so on); using the melatonin treatment for sleep disorders or withdrawal less than three weeks.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Bumetanidebumetanide tablets, oral intake, 0.5mg, twice daily, respectively at 8 am and 4 pm | Drug | Bumetanide group |
| Placeboplacebo tablets, oral intake, 0.5mg, twice daily, respectively at 8 am and 4 pm | Drug | Control group |
Primary outcome
Childhood Autism Rating Scale(CARS)
Time frame: Day 0 and Day 90
CARS is a behaviour-rating scale used to assess the presence and severity of the symptoms of autism spectrum disorder
6 secondary outcomes
Clinical Global Impressions Scale (CGI)
Time frame: Day 0 and Day 90
Social Responsiveness Scale (SRS)
Time frame: Day 0 and Day 90
Autism Diagnostic Observation Schedule (ADOS)
Time frame: Day 0 and Day 90
Short Sensory Profile Report
Time frame: Day 0 and Day 90
Symbolic Play Test
Time frame: Day 0 and Day 90
Chinese Communicative Development Inventory
Time frame: Day 0 and Day 90
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Mar 2021.
- Individual participant dataSharing planned: Undecided
Related trials for Autism Spectrum Disorder
RecruitingPhase 1
Cannabidiol (CBD) in Adults With ASD
Autism Spectrum Disorder
RecruitingPhase 3
A Study to Evaluate the Long-term Safety and Tolerability of Xanomeline and Trospium Chloride for the Treatment of Psychiatric Disorders in Children and Adolescents
Schizophrenia, Autism-Related Irritability
Not yet recruiting
Sensory-integration Therapy Verses Augumented Reality-based Training in ASD
Autism Spectrum Disorder
Recruiting
Cumulative and Booster Effects of Multisession Prefrontal tDCS in ASD Adolescents
Transcranial Direct Current Stimulation, Autistic Spectrum Disorder
Not yet recruiting
"Family Resilience And Mind-body Enhancement" (FRAME): A Randomised Controlled Trial
Autism
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Fei Li, doctor principal investigator
Xinhua hospital Affilated to Shanghai Jiao Tong University School of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Bumetanide in people with Autism Spectrum Disorder, run by Xinhua Hospital, Shanghai Jiao Tong University School of Medicine at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Childhood Autism Rating Scale(CARS) (time frame: Day 0 and Day 90). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Mar 2021.