Autism in ChildrenPhase 2NCT07635862
Clinical Trial Evaluating the Effects of Ganaxolone in Children With Autism
A Phase 2 study testing Ganaxolone (Ztalmy) in people with Autism in Children and Autistic Disorder in Children and Adolescents, run by Stanford University at 1 site in United States.
Trial summary
The goal of this study is to conduct a randomized, placebo-controlled trial (RCT) of ganaxolone, a neuroactive steroid (NAS), in autistic children and adolescents aged 5 to 17 years old. Read more about this study
- Who is it for?
- Ages 5 to 17 years
- Conditions the study is for
- Autism in Children, Autistic Disorder in Children and Adolescents, Autism Spectrum Disorder
- What is being tested?
- Ganaxolone (Ztalmy)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 66 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Jan 2027to 1 Jan 2030
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 5 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- children between the ages of 5 years and 17 years old at enrollment
- diagnosis of autism spectrum disorder based on DSM-5 criteria and confirmed with the Autism Diagnostic Inventory - Revised (ADI-R) and Autism Diagnostic Observation Schedule 2nd edition (ADOS-2), or Childhood Autism Rating Scales (CARS)
- medical stability based on clinical interview
- stable medication regimens (2 weeks, with the exception of fluoxetine for 4 weeks)
- stable psychosocial therapies (4 weeks) prior to randomization and no plans to change treatments or intensity during the trial
- high rates of irritability defined as the Aberrant Behavior Checklist irritability subscale score > 18
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- for participants who are sexually active, use of an effective contraceptive (e.g., birth control medications for female participants and condoms for male participants) and no plans for pregnancy throughout the trial
- for females, negative urine pregnancy test at baseline
- no planned changes in school placement
- for participants living within 150 miles of Stanford University, have the ability to attend site visits and attempt EEG and MRI procedures before and after the trial
- availability of a reliable informant who interacts with the participant regularly and can reliably complete assessments in English regarding their behaviors throughout the trial
- ability to participate in the testing administered in English to the extent that valid standard scores and biological samples can be obtained.
Who cannot join
- any unstable medical condition, such as unstable seizure disorder or heart disease
- any lifetime diagnosis of severe psychiatric (e.g., schizophrenia) or neurodegenerative conditions
- concomitant use of any neuroactive steroids or corticosteroids.
- history of substance abuse or active/planned use of alcohol, opioids, or cannabinoids
- recent history or current suicidal ideation assessed with the Columbia-Suicide Severity Rating Scale (C-SSRS) and by clinical interview with the study physician
- pregnancy and mothers who are breastfeeding
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- prior participation in any clinical trial in the 30 days prior to study entry
- known intolerance or hypersensitivity to ganaxolone or similar analogs
- Concomitant use of medications that are inducers of CYP450 3A4/5, such as rifampin, carbamazepine, phenytoin, phenobarbital, and St. John's wort
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site
Stanford University
Stanford, California, 94305
Robin Libove, BS, (650) 736-1235, autismdd@stanford.edu
Briana Hernandez, BS, (650) 736-1235, autismdd@stanford.edu
About this study
Official title: A Randomized Controlled Trial of Ganaxolone for Children With Autism Spectrum Disorder
The goal of this study is to conduct a randomized, placebo-controlled trial (RCT) of ganaxolone, a neuroactive steroid (NAS), in autistic children and adolescents aged 5 to 17 years old. Ganaxolone is approved and effective for treating seizures in children as young as 2 years old who have CDKL5 deficiency disorder (CDD), a neurogenetic condition associated with developmental delays, seizure disorder, hypotonia, visual impairments, and autistic features. The primary outcome of interest for this trial is irritability on the Aberrant Behavior Checklist (ABC) because it is a common symptom of emotion dysregulation in ASD that impacts quality of life, including mental health, independence, educational opportunities, and integration into the community. The secondary domains of interest for this trial are restricted and repetitive behaviors (RRB), specifically insistence on sameness (IS), a subdomain of RRB characterized by inflexibility and a strong preference for predictable routines and familiar environments. Secondary outcome measures include the IS subscale from the Dimensional Assessment of Repetitive Behaviors (DARB) and subscales of the Clinical Global Impressions Scale for irritability (CGI-IR) and IS (CGI-IS). For participants living within 150 miles of Stanford University, we require participants to attend site visits and attempt EEG and MRI procedures before and after the trial, though we are recruiting nationally and the study can be completed without site vists.
- Conditions
- Autism in Children, Autistic Disorder in Children and Adolescents, Autism Spectrum Disorder
- Being tested
- Ganaxolone (Ztalmy), Placebo
- Keywords
- Autism, Ganaxolone, Autism disorder in children and adolescents, Ztalmy, GABA, Insistance on Sameness, Emotional Regulation Difficulties, Irritability
- Medical subject headings
- Autistic Disorder, Autism Spectrum Disorder
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ganaxolone (Ztalmy)Drug
Liquid oral suspension of ganaxolone will be administered orally with food three times daily. Dosage will be increased based on tolerability, no more frequently than every 7 days. Dosing will be flexible and occur between 4 to 8 weeks, depending on tolerability and response across each individual patient. Once the maximum tolerated dose is identified, it will be held stable for the remaining 4-8 weeks of the study, for a total of 12 weeks in the randomized, controlled phase.
The titration schedule for patients weighing 28 kg or less is:
Days 1-7: 2 mg/kg x 3 per day Days 8-14: 4 mg/kg x 3 per day Days 15-21: 8 mg/kg x 3 per day Days 22-28: 14 mg/kg x 3 per day Day 29 and thereafter: 21 mg/kg x 3 per day
The titration schedule for patients weighing more than 28 kg is:
Days 1-7: 50 mg/kg x 3 per day Days 8-14: 100 mg/kg x 3 per day Days 15-21: 200 mg/kg x 3 per day Days 22-28: 400 mg/kg x 3 per day Day 29 and thereafter: 600 mg/kg x 3 per day
PlaceboDrug
The placebo suspension will contain the same components as the active compound, except for ganaxolone.
| Study group | Role | What they receive |
|---|---|---|
| Ganaxolone Arm | Active comparator | Participants receives up to 20 weeks of Ganaxolone treatment Receives: Drug: Ganaxolone (Ztalmy) |
| Placebo Arm | Placebo comparator | Participants receives up to 20 weeks of placebo treatment Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
The score on irritability subscale on the Aberrant Behavior Checklist (ABC)
It is a common symptom of emotion dysregulation in ASD that impacts quality of life, including mental health, independence, educational opportunities, and integration into the community, and ganaxolone may provide more direct benefits or increased tolerability compared to atypical antipsychotics.
Measured: The measures will be completed at screening, baseline, week 2, week 4, week 6, week 8, week 10, week 12, week 14, and week 16.
Secondary outcomes (3)
The score of Insistence on Sameness subscale of the Dimensional Assessment of Repetitive Behaviors (DARB)
The DARB is an informant report measure developed and validated following methods outlined by the NIH Patient-Reported Outcome Measurement Information System (PROMIS) framework. Comprehensive, multi-trait, multi-method factor analyses across existing RRB instruments and meta-analysis of factor analyses identified the following eight RRB subdomains: repetitive motor behaviors, insistence on sameness, restricted interests, unusual interests, self-injurious behaviors, sensory sensitivity, obsessions and compulsions, and repetitive language.
Measured: The measures will be completed at screening, baseline, week 2, week 4, week 6, week 8, week 10, week 12, week 14, and week 16.
The score on irritability subscale on the Clinical Global Impressions Scale (CGI-IR)
The study physician will also administer the CGI scale, which includes clinical judgment of the severity of illness and global improvement. The CGI is widely used in psychopharmacological studies and has high sensitivity to measure medication effects.
Measured: The measures will be completed at screening, baseline, week 2, week 4, week 6, week 8, week 10, week 12, week 14, and week 16.
The score on Insistence on Sameness subscale on the Clinical Global Impressions Scale(CGI-IS)
The study physician will also administer the CGI scale, which includes clinical judgment of the severity of illness and global improvement. The CGI is widely used in psychopharmacological studies and has high sensitivity to measure medication effects.
Measured: These measures will be completed at screening, baseline, week 2, week 4, week 6, week 8, week 10, week 12, week 14, and week 16.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jun 2026
- Study start1 Jan 2027 (estimated)
- Primary completion1 Jan 2030 (estimated)
- Study completion1 Jan 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autism in ChildrenStudies of the same condition, open and under way.
- More trials run by Stanford UniversityThe 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
- 66 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Collaborators
- SPARK NS
- Responsible party
- Principal investigator, Antonio Hardan, Stanford University
- Investigators
- Antonio Y. Hardan, M.D., Principal investigator, Stanford University School of Medicine-Psychiatry and Behavioral Sciences - Child and Adolescent Psychiatry and Child Development
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07635862
- Sponsor's protocol ID
- IRB-84478
- Status last verified
- Nov 2026
- First submitted
- 3 Jun 2026
- Last updated
- 5 Nov 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized Controlled Trial of Ganaxolone for Children With Autism Spectrum Disorder
- ClinicalTrials.gov ID
- NCT07635862
- Acronym / sponsor ID
- IRB-84478
- Conditions
- Autism in Children, Autistic Disorder in Children and Adolescents, Autism Spectrum Disorder
- Keywords
- Autism, Ganaxolone, Autism disorder in children and adolescents, Ztalmy, GABA, Insistance on Sameness, Emotional Regulation Difficulties, Irritability
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 66 participants (estimated)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Collaborators
- SPARK NS
- Study start
- 1 Jan 2027 (estimated)
- Primary completion
- 1 Jan 2030 (estimated)
- Study completion
- 1 Jan 2030 (estimated)
- First posted
- 9 Jun 2026 (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 goal of this study is to conduct a randomized, placebo-controlled trial (RCT) of ganaxolone, a neuroactive steroid (NAS), in autistic children and adolescents aged 5 to 17 years old. Ganaxolone is approved and effective for treating seizures in children as young as 2 years old who have CDKL5 deficiency disorder (CDD), a neurogenetic condition associated with developmental delays, seizure disorder, hypotonia, visual impairments, and autistic features. The primary outcome of interest for this trial is irritability on the Aberrant Behavior Checklist (ABC) because it is a common symptom of emotion dysregulation in ASD that impacts quality of life, including mental health, independence, educational opportunities, and integration into the community. The secondary domains of interest for this trial are restricted and repetitive behaviors (RRB), specifically insistence on sameness (IS), a subdomain of RRB characterized by inflexibility and a strong preference for predictable routines and familiar environments. Secondary outcome measures include the IS subscale from the Dimensional Assessment of Repetitive Behaviors (DARB) and subscales of the Clinical Global Impressions Scale for irritability (CGI-IR) and IS (CGI-IS). For participants living within 150 miles of Stanford University, we require participants to attend site visits and attempt EEG and MRI procedures before and after the trial, though we are recruiting nationally and the study can be completed without site vists.
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 |
|---|---|---|
| Ganaxolone Arm | Active comparator | Drug: Ganaxolone (Ztalmy)Participants receives up to 20 weeks of Ganaxolone treatment |
| Placebo Arm | Placebo comparator | Drug: PlaceboParticipants receives up to 20 weeks of placebo treatment |
Who may qualify
- Ages
- 5 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (12 listed)
- children between the ages of 5 years and 17 years old at enrollment
- diagnosis of autism spectrum disorder based on DSM-5 criteria and confirmed with the Autism Diagnostic Inventory - Revised (ADI-R) and Autism Diagnostic Observation Schedule 2nd edition (ADOS-2), or Childhood Autism Rating Scales (CARS)
- medical stability based on clinical interview
- stable medication regimens (2 weeks, with the exception of fluoxetine for 4 weeks)
- stable psychosocial therapies (4 weeks) prior to randomization and no plans to change treatments or intensity during the trial
Key exclusion criteria (9 listed)
- any unstable medical condition, such as unstable seizure disorder or heart disease
- any lifetime diagnosis of severe psychiatric (e.g., schizophrenia) or neurodegenerative conditions
- concomitant use of any neuroactive steroids or corticosteroids.
- history of substance abuse or active/planned use of alcohol, opioids, or cannabinoids
- recent history or current suicidal ideation assessed with the Columbia-Suicide Severity Rating Scale (C-SSRS) and by clinical interview with the study physician
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 |
|---|---|---|
| Ganaxolone (Ztalmy)Liquid oral suspension of ganaxolone will be administered orally with food three times daily. Dosage will be increased based on tolerability, no more frequently than every 7 days. Dosing will be flexible and occur between 4 to 8 weeks, depending on tolerability and response across each individual patient. Once the maximum tolerated dose is identified, it will be held stable for the remaining 4-8 weeks of the study, for a total of 12 weeks in the randomized, controlled phase. The titration schedule for patients weighing 28 kg or less is: Days 1-7: 2 mg/kg x 3 per day Days 8-14: 4 mg/kg x 3 per day Days 15-21: 8 mg/kg x 3 per day Days 22-28: 14 mg/kg x 3 per day Day 29 and thereafter: 21 mg/kg x 3 per day The titration schedule for patients weighing more than 28 kg is: Days 1-7: 50 mg/kg x 3 per day Days 8-14: 100 mg/kg x 3 per day Days 15-21: 200 mg/kg x 3 per day Days 22-28: 400 mg/kg x 3 per day Day 29 and thereafter: 600 mg/kg x 3 per day | Drug | Ganaxolone Arm |
| PlaceboThe placebo suspension will contain the same components as the active compound, except for ganaxolone. | Drug | Placebo Arm |
Primary outcome
The score on irritability subscale on the Aberrant Behavior Checklist (ABC)
Time frame: The measures will be completed at screening, baseline, week 2, week 4, week 6, week 8, week 10, week 12, week 14, and week 16.
It is a common symptom of emotion dysregulation in ASD that impacts quality of life, including mental health, independence, educational opportunities, and integration into the community, and ganaxolone may provide more direct benefits or increased tolerability compared to atypical antipsychotics.
3 secondary outcomes
The score of Insistence on Sameness subscale of the Dimensional Assessment of Repetitive Behaviors (DARB)
Time frame: The measures will be completed at screening, baseline, week 2, week 4, week 6, week 8, week 10, week 12, week 14, and week 16.
The score on irritability subscale on the Clinical Global Impressions Scale (CGI-IR)
Time frame: The measures will be completed at screening, baseline, week 2, week 4, week 6, week 8, week 10, week 12, week 14, and week 16.
The score on Insistence on Sameness subscale on the Clinical Global Impressions Scale(CGI-IS)
Time frame: These measures will be completed at screening, baseline, week 2, week 4, week 6, week 8, week 10, week 12, week 14, and week 16.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Nov 2026.
- Individual participant dataSharing planned: No
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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.
Antonio Y. Hardan, M.D. principal investigator
Stanford University School of Medicine-Psychiatry and Behavioral Sciences - Child and Adolescent Psychiatry and Child Development
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 Ganaxolone (Ztalmy) in people with Autism in Children and Autistic Disorder in Children and Adolescents, run by Stanford University at 1 site in United States.
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 12 inclusion and 9 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: opening soon, as of the sponsor's last update on 5 Nov 2026. 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 United States. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Robin Libove, BS ((650) 736-1235, autismdd@stanford.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The score on irritability subscale on the Aberrant Behavior Checklist (ABC) (time frame: The measures will be completed at screening, baseline, week 2, week 4, week 6, week 8, week 10, week 12, week 14, and week 16.). 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 5 Nov 2026.