Clinical Trial Evaluating the Effects of Ganaxolone in Children With Autism
2026年6月3日 更新者：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.
調査の概要
研究の種類
介入
入学 (推定)
66
段階
- フェーズ2
連絡先と場所
このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。
研究連絡先
- 名前：Robin Libove, BS
- 電話番号：(650) 736-1235
- メール：autismdd@stanford.edu
研究連絡先のバックアップ
- 名前：Briana Hernandez, BS
- 電話番号：(650) 736-1235
- メール：autismdd@stanford.edu
研究場所
-
-
California
-
Stanford、California、アメリカ、94305
- Stanford University
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コンタクト：
- Robin Libove, BS
- 電話番号：(650) 736-1235
- メール：autismdd@stanford.edu
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コンタクト：
- Briana Hernandez, BS
- 電話番号：(650) 736-1235
- メール：autismdd@stanford.edu
-
主任研究者：
- Antonio Hardan, M.D.
-
-
参加基準
研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。
適格基準
就学可能な年齢
- 子
健康ボランティアの受け入れ
いいえ
説明
Inclusion Criteria:
- 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
- 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.
Exclusion Criteria:
- 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
- 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
研究計画
このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。
研究はどのように設計されていますか？
デザインの詳細
- 主な目的：処理
- 割り当て：ランダム化
- 介入モデル：並列代入
- マスキング：4倍
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
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アクティブコンパレータ：Ganaxolone Arm
Participants receives up to 20 weeks of Ganaxolone treatment
|
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 |
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プラセボコンパレーター：Placebo Arm
Participants receives up to 20 weeks of placebo treatment
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The placebo suspension will contain the same components as the active compound, except for ganaxolone.
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この研究は何を測定していますか？
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
The score on irritability subscale on the Aberrant Behavior Checklist (ABC)
時間枠：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.
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二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
The score of Insistence on Sameness subscale of the Dimensional Assessment of Repetitive Behaviors (DARB)
時間枠：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.
|
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The score on irritability subscale on the Clinical Global Impressions Scale (CGI-IR)
時間枠：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.
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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.
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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)
時間枠：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.
|
協力者と研究者
ここでは、この調査に関係する人々や組織を見つけることができます。
スポンサー
協力者
捜査官
- 主任研究者：Antonio Y. Hardan, M.D.、Stanford University School of Medicine-Psychiatry and Behavioral Sciences - Child and Adolescent Psychiatry and Child Development
研究記録日
これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。
主要日程の研究
研究開始 (推定)
2027年1月1日
一次修了 (推定)
2030年1月1日
研究の完了 (推定)
2030年1月1日
試験登録日
最初に提出
2026年6月3日
QC基準を満たした最初の提出物
2026年6月3日
最初の投稿 (実際)
2026年6月9日
学習記録の更新
投稿された最後の更新 (実際)
2026年6月9日
QC基準を満たした最後の更新が送信されました
2026年6月3日
最終確認日
2026年6月1日
詳しくは
本研究に関する用語
キーワード
追加の関連 MeSH 用語
その他の研究ID番号
- IRB-84478
個々の参加者データ (IPD) の計画
個々の参加者データ (IPD) を共有する予定はありますか?
いいえ
医薬品およびデバイス情報、研究文書
米国FDA規制医薬品の研究
はい
米国FDA規制機器製品の研究
いいえ
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