Clinical Trial Evaluating the Effects of Ganaxolone in Children With Autism
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age5-17
SponsorStanford University
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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)
Disqualifiers
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
Trial design
Design model
Parallel
Treatments tested in this trial
Ganaxolone (Ztalmy)
DrugLiquid 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
Placebo
DrugThe placebo suspension will contain the same components as the active compound, except for ganaxolone.
Treatment groups
66 Participants
are divided into 2 treatment groupsGroup A: Ganaxolone ArmActive comparator 1 intervention
Group B: Placebo ArmPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
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.
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.Secondary outcomes
1
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.
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 16.2
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.
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 16.3
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.
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.Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Stanford University
Lead sponsor
SPARK NS
Collaborator
Source ClinicalTrials.gov
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