Clinical Trial Evaluating the Effects of Ganaxolone in Children With Autism

Conditions

• Autism Spectrum Disorder
• Autism in Children
• Autistic Disorder in Children and Adolescents

Eligibility Criteria

Sex

ALL

Age

5 Years - 17 Years

Healthy Volunteers

Not accepted

Interventions

• 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
• Placebo — DRUG
  The placebo suspension will contain the same components as the active compound, except for ganaxolone.

Study Details

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.

Key Dates

Start date

Jan 1, 2027

Status verified

Jun 2026

Primary completion

Jan 1, 2030

Completion

Jan 1, 2030

Study Design

Enrollment

66 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Active Comparator: Ganaxolone Arm
  Participants receives up to 20 weeks of Ganaxolone treatment
• Placebo Comparator: Placebo Arm
  Participants receives up to 20 weeks of placebo treatment

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. ]

Central Contacts

• Robin Libove, BS
  (650) 736-1235
  [email protected]
• Briana Hernandez, BS
  (650) 736-1235
  [email protected]

Locations (1)

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