Deze pagina is automatisch vertaald en de nauwkeurigheid van de vertaling kan niet worden gegarandeerd. Raadpleeg de Engelse versie voor een brontekst.

Clinical Trial Evaluating the Effects of Ganaxolone in Children With Autism

3 juni 2026 bijgewerkt door: Antonio Hardan, Stanford University

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.

Studie Overzicht

Toestand

Nog niet aan het werven

Conditie

Interventie / Behandeling

Studietype

Ingrijpend

Inschrijving (Geschat)

66

Fase

  • Fase 2

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studiecontact

Studie Contact Back-up

Studie Locaties

  • Verenigde Staten
    • California
      • Stanford, California, Verenigde Staten, 94305
        • Stanford University
        • Contact:
        • Contact:
        • Hoofdonderzoeker:
          • Antonio Hardan, M.D.

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

  • Kind

Accepteert gezonde vrijwilligers

Nee

Beschrijving

Inclusion Criteria:

  1. children between the ages of 5 years and 17 years old at enrollment
  2. 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)
  3. medical stability based on clinical interview
  4. stable medication regimens (2 weeks, with the exception of fluoxetine for 4 weeks)
  5. stable psychosocial therapies (4 weeks) prior to randomization and no plans to change treatments or intensity during the trial
  6. high rates of irritability defined as the Aberrant Behavior Checklist irritability subscale score > 18
  7. 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
  8. for females, negative urine pregnancy test at baseline
  9. no planned changes in school placement
  10. 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
  11. availability of a reliable informant who interacts with the participant regularly and can reliably complete assessments in English regarding their behaviors throughout the trial
  12. ability to participate in the testing administered in English to the extent that valid standard scores and biological samples can be obtained.

Exclusion Criteria:

  1. any unstable medical condition, such as unstable seizure disorder or heart disease
  2. any lifetime diagnosis of severe psychiatric (e.g., schizophrenia) or neurodegenerative conditions
  3. concomitant use of any neuroactive steroids or corticosteroids.
  4. history of substance abuse or active/planned use of alcohol, opioids, or cannabinoids
  5. recent history or current suicidal ideation assessed with the Columbia-Suicide Severity Rating Scale (C-SSRS) and by clinical interview with the study physician
  6. pregnancy and mothers who are breastfeeding
  7. prior participation in any clinical trial in the 30 days prior to study entry
  8. known intolerance or hypersensitivity to ganaxolone or similar analogs
  9. Concomitant use of medications that are inducers of CYP450 3A4/5, such as rifampin, carbamazepine, phenytoin, phenobarbital, and St. John's wort

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Behandeling
  • Toewijzing: Gerandomiseerd
  • Interventioneel model: Parallelle opdracht
  • Masker: Verviervoudigen

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Actieve vergelijker: Ganaxolone Arm
Participants receives up to 20 weeks of Ganaxolone treatment
Geneesmiddel: 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
Placebo-vergelijker: Placebo Arm
Participants receives up to 20 weeks of placebo treatment
Geneesmiddel: Placebo
The placebo suspension will contain the same components as the active compound, except for ganaxolone.

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
The score on irritability subscale on the Aberrant Behavior Checklist (ABC)
Tijdsspanne: 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.
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.

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
The score of Insistence on Sameness subscale of the Dimensional Assessment of Repetitive Behaviors (DARB)
Tijdsspanne: 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 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.
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)
Tijdsspanne: 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 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.
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)
Tijdsspanne: 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.
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.
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.

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

Stanford University

Medewerkers

SPARK NS

Onderzoekers

  • Hoofdonderzoeker: Antonio Y. Hardan, M.D., Stanford University School of Medicine-Psychiatry and Behavioral Sciences - Child and Adolescent Psychiatry and Child Development

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Geschat)

1 januari 2027

Primaire voltooiing (Geschat)

1 januari 2030

Studie voltooiing (Geschat)

1 januari 2030

Studieregistratiedata

Eerst ingediend

3 juni 2026

Eerst ingediend dat voldeed aan de QC-criteria

3 juni 2026

Eerst geplaatst (Werkelijk)

9 juni 2026

Updates van studierecords

Laatste update geplaatst (Werkelijk)

9 juni 2026

Laatste update ingediend die voldeed aan QC-criteria

3 juni 2026

Laatst geverifieerd

1 juni 2026

Meer informatie

Termen gerelateerd aan deze studie

Trefwoorden

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

  • IRB-84478

Plan Individuele Deelnemersgegevens (IPD)

Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?

NEE

Informatie over medicijnen en apparaten, studiedocumenten

Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel

Ja

Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct

Nee

Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .

Abonneren