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Clinical Trial Evaluating the Effects of Ganaxolone in Children With Autism

2026년 6월 3일 업데이트: 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.

연구 개요

상태

아직 모집하지 않음

정황

개입 / 치료

연구 유형

중재적

등록 (추정된)

66

단계

  • 2 단계

연락처 및 위치

이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.

연구 연락처

연구 연락처 백업

연구 장소

  • 미국
    • California
      • Stanford, California, 미국, 94305
        • Stanford University
        • 연락하다:
        • 연락하다:
        • 수석 연구원:
          • Antonio Hardan, M.D.

참여기준

연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.

자격 기준

공부할 수 있는 나이

  • 어린이

건강한 자원 봉사자를 받아들입니다

아니

설명

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

공부 계획

이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.

연구는 어떻게 설계됩니까?

디자인 세부사항

  • 주 목적: 치료
  • 할당: 무작위
  • 중재 모델: 병렬 할당
  • 마스킹: 네 배로

무기와 개입

참가자 그룹 / 팔
개입 / 치료
활성 비교기: Ganaxolone Arm
Participants receives up to 20 weeks of Ganaxolone treatment
의약품: 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
위약 비교기: Placebo Arm
Participants receives up to 20 weeks of placebo treatment
의약품: Placebo
The placebo suspension will contain the same components as the active compound, except for ganaxolone.

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
The score on irritability subscale on the Aberrant Behavior Checklist (ABC)
기간: 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.

2차 결과 측정

결과 측정
측정값 설명
기간
The score of Insistence on Sameness subscale of the Dimensional Assessment of Repetitive Behaviors (DARB)
기간: 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)
기간: 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)
기간: 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.

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

스폰서

Stanford University

협력자

SPARK NS

수사관

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

연구 기록 날짜

이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.

연구 주요 날짜

연구 시작 (추정된)

2027년 1월 1일

기본 완료 (추정된)

2030년 1월 1일

연구 완료 (추정된)

2030년 1월 1일

연구 등록 날짜

최초 제출

2026년 6월 3일

QC 기준을 충족하는 최초 제출

2026년 6월 3일

처음 게시됨 (실제)

2026년 6월 9일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 6월 9일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 6월 3일

마지막으로 확인됨

2026년 6월 1일

추가 정보

이 연구와 관련된 용어

키워드

추가 관련 MeSH 약관

기타 연구 ID 번호

  • IRB-84478

개별 참가자 데이터(IPD) 계획

개별 참가자 데이터(IPD)를 공유할 계획입니까?

아니요

약물 및 장치 정보, 연구 문서

미국 FDA 규제 의약품 연구

예

미국 FDA 규제 기기 제품 연구

아니

이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .

자폐 스펙트럼 장애에 대한 임상 시험

Ganaxolone (Ztalmy)에 대한 임상 시험

유사한 임상시험 검색

스폰서 및 공동 작업자

건강 상태

약물 개입

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정황

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약물 개입

식이 보충제

스폰서 / 협력자

위치

구독하다