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レット症候群におけるフェンフルラミン塩酸塩の第3相試験

2026年10月30日 更新者：UCB BIOSCIENCES, Inc.

レット症候群の研究参加者におけるフェンフルラミン塩酸塩の有効性と安全性を評価するための、オープンラベル延長を伴う第3相無作為化二重盲検プラセボ対照並行群比較多施設共同研究

The purpose of this study is to investigate the efficacy of fenfluramine hydrochloride (HCl) versus placebo in study participants with Rett syndrome (RTT).

調査の概要

状態

募集

条件

介入・治療

研究の種類

介入

入学 (推定)

200

段階

  • フェーズ 3

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

研究連絡先のバックアップ

研究場所

  • アメリカ
    • Arkansas
      • Little Rock、Arkansas、アメリカ、72202
        • 募集
        • Ep0247 21010
    • Florida
      • Miami、Florida、アメリカ、33155
        • 募集
        • Ep0247 21009
      • Orlando、Florida、アメリカ、32806
        • 募集
        • Ep0247 21011
    • Georgia
      • Atlanta、Georgia、アメリカ、30322
        • 募集
        • Ep0247 21007
    • Pennsylvania
      • Philadelphia、Pennsylvania、アメリカ、19104-4318
        • 募集
        • Ep0247 21003
    • Texas
      • Houston、Texas、アメリカ、77030
        • 募集
        • Ep0247 21005
      • Irving、Texas、アメリカ、75062
        • 募集
        • Ep0247 21013
    • Washington
      • Seattle、Washington、アメリカ、98105
        • 募集
        • Ep0247 21004
  • イギリス
      • Manchester、イギリス
        • 募集
        • Ep0247 11303
      • Nottingham、イギリス
        • 募集
        • Ep0247 11304
  • イスラエル
      • Ramat Gan、イスラエル
        • 募集
        • Ep0247 16001
  • イタリア
      • Genova、イタリア
        • 募集
        • Ep0247 17003
      • Milan、イタリア
        • 募集
        • Ep0247 17001
      • Naples、イタリア
        • 募集
        • Ep0247 17008
      • Pisa、イタリア
        • 募集
        • Ep0247 17002
      • Troina、イタリア
        • 募集
        • Ep0247 17006
  • グルジア
      • Tbilisi、グルジア
        • 募集
        • Ep0247 13001
      • Tbilisi、グルジア
        • 募集
        • Ep0247 13002
  • スペイン
      • Madrid、スペイン
        • 募集
        • Ep0247 11105
      • Madrid、スペイン
        • 募集
        • Ep0247 11101
      • Málaga、スペイン
        • 募集
        • Ep0247 11103
      • Seville、スペイン
        • 募集
        • Ep0247 11102
  • ドイツ
      • Vogtareuth、ドイツ
        • 募集
        • Ep0247 14001
  • ハンガリー
      • Budapest、ハンガリー
        • 募集
        • Ep0247 15001
      • Debrecen、ハンガリー
        • 募集
        • Ep0247 15002
  • フランス
      • Brest、フランス
        • 募集
        • Ep0247 12005
      • Bron、フランス
        • 募集
        • Ep0247 12003
      • Marseille、フランス
        • 募集
        • Ep0247 12004
      • Paris、フランス
        • 募集
        • Ep0247 12006
      • Paris、フランス
        • 募集
        • Ep0247 12001
  • ベルギー
      • Brussels、ベルギー
        • 募集
        • Ep0247 11001
      • Edegem、ベルギー
        • 募集
        • Ep0247 11002
  • ポーランド
      • Gdansk、ポーランド
        • 募集
        • Ep0247 19003
      • Krakow、ポーランド
        • 募集
        • Ep0247 19002
  • 日本
      • Chiyoda-ku、日本
        • 募集
        • Ep0247 43005
      • Kodaira、日本
        • 募集
        • Ep0247 43004
      • Kurume、日本
        • 募集
        • Ep0247 43001
      • Musashimurayama-shi、日本
        • 募集
        • Ep0247 43006
      • Osaka、日本
        • 募集
        • Ep0247 43002
      • Setagaya-ku、日本
        • 募集
        • Ep0247 43003

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

5年～35年 (子、大人)

健康ボランティアの受け入れ

いいえ

受講資格のある性別

全て

説明

Inclusion Criteria:

  • Participant has typical or classic Rett Syndrome (RTT) according to the RettSearch Consortium 2010 revised criteria
  • Participant has a documented disease-causing mutation or deletion in the methyl-CpG-binding protein 2 (MECP2) gene
  • Participant meets criteria for postregression for at least 6 months prior to Screening, defined as:
  • No loss or degradation of ambulation (including gait, coordination, or independence of walking/standing);
  • No loss or degradation of hand function; no loss or degradation of speech (including babbling, words, or previously developed communicative vocalizations);
  • No loss or degradation of nonverbal communicative or social skills (including eye gaze, using body to indicate communicative intent, or social attentiveness)
  • Participant has an Rett Syndrome Clinical Severity Scale (RTT-CSS) rating of 10 to 36 (inclusive)
  • Participant has a Clinical Global Impression-Severity (CGIS) score of ≥4
  • Participant has a legal representative capable of providing signed informed consent on behalf of the participant as described in the protocol, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol.
  • Participant is aged 5 to 35 years of age (inclusive) at the time of first administration of investigational intervention.
  • Male or female.
  • Participant has a consistent caregiver who is ≥18 years of age at the Screening Visit. The caregiver needs to be able to complete the caregiver assessments defined for the entire study. Every attempt should be made to have the same evaluator complete the assessments for the duration of the study.

Exclusion Criteria:

  • Participant has a history of lymphoma, leukemia, or any malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years.

  • Participant has clinically significant abnormality in vital signs according to the Investigator

  • Participant has an exclusionary cardiovascular or cardiopulmonary abnormality based on echocardiogram (ECHO), electrocardiogram (ECG), or physical examination, and is not approved for entry by the central cardiac reader. Exclusionary abnormalities include, but are not limited to:

    1. Greater than trace aortic valve regurgitation.
    2. Greater than mild mitral valve regurgitation.
    3. Possible signs of pulmonary arterial hypertension (PAH) with abnormal pulmonary artery systolic pressure (PASP) or PASP ≥35 mmHg.
    4. Evidence of left ventricular dysfunction (systolic or diastolic).
    5. Clinically significant structural cardiac abnormality, including but not limited to mitral valve prolapse, atrial or ventricular septal defects, or patent ductus arteriosus with reversal of shunt (right to left shunt). Note: Patent foramen ovale without a reversal of shunt or a bicuspid aortic valve is not considered exclusionary

  • Participant has a clinically significant medical condition, including chronic obstructive pulmonary disease, interstitial lung disease, portal hypertension, or need for invasive mechanical ventilation (eg, via tracheostomy), or has had clinically relevant symptoms or a clinically significant illness currently or in the 4 weeks prior to the Screening Visit that would negatively impact study participation, collection of study data, or pose a risk to the participant

  • Participant is taking >4 concomitant antiseizure medications (ASMs). Rescue medications are not included in the count

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：トリプル

武器と介入

参加者グループ / アーム
介入・治療
実験的：fenfluramine hydrochloride

Participants will receive fenfluramine hydrochloride for 14 weeks in a double-blind period (including titration and maintenance), followed by a 2-week double-blind transition period. This is followed by a 52-week open-label treatment period. Participants may continue treatment until alternative access is available. Those who discontinue without continued access will undergo an 8-day taper and a 26-week follow-up.
薬：fenfluramine hydrochloride

Oral solution

他の名前：
  • フィンテプラ、ZX008
プラセボコンパレーター：Placebo

Participants will receive a matching placebo for 14 weeks in a double-blind period (including titration and maintenance). After this period, they transition into a 2-week double-blind period with fenfluramine hydrochloride, followed by a 52-week open-label treatment period. Participants may continue treatment until alternative access is available. Those who discontinue without continued access will undergo an 8-day taper and a 26-week follow-up.
他の：Placebo

Oral solution

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
Change from Baseline to Week 14 in Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score
時間枠：From Baseline (Day 1) to Week 14

The RSBQ is a caregiver-completed, instrument assessing behavioral and emotional features in RTT. The RSBQ consists of 45 items, including 8 subscales: General mood (8 items); Breathing problems (5 items); Hand behavior (6 items); Face movements (4 items); Body rocking and expressionless face (6 items); Nighttime behaviors (3 items); Fear/anxiety (4 items); and Walking/standing (2 items). Caregivers are asked to evaluate each RTT feature based on the current status of the patients on a 3-point scale as 0 ("not true"), 1 ("somewhat or sometimes true"), or 2 ("often true"), with the total score ranging from 0 to 90. Higher scores indicate increased disease severity. Seven items that do not belong under any of the subscales are classed as "uncategorized" but contribute to the overall total score.
From Baseline (Day 1) to Week 14
Clinical Global Impression of Change (CGIC) Score at Week 14
時間枠：At Week 14

The CGIC is a clinician-rated single item evaluating the degree of improvement or worsening of a participant's condition from Baseline following treatment or intervention. The CGIC uses a 7-point response scale, with the following ratings: "1: Very Much improved", "2: Much improved", "3: Minimally improved", "4: No change", "5: Minimally worse", "6: Much worse", "7: Very Much Worse".
At Week 14

二次結果の測定

結果測定
メジャーの説明
時間枠
Change from Baseline to Week 14 in Patient-Reported Outcomes Measurement Information System-Sleep Disturbance (PROMIS-SD) score
時間枠：From Baseline (Day 1) to Week 14

Sleep disturbances will be assessed by the PROMIS-SD parent proxy 8a version. Caregivers are asked to rate 8 items over the past 7 days on a 5-point scale: "1: Never", "2: Almost never", "3: Sometimes", "4: Almost always", and "5: Always", with higher scores indicating more severe sleep disturbances.
From Baseline (Day 1) to Week 14
Change from Baseline to Week 14 in Observer-Reported Communication Ability (ORCA) score
時間枠：From Baseline (Day 1) to Week 14

The ORCA is an 84-item, observer-reported measure of communication ability over the past 30 days. The majority of the items included in the ORCA measure have 3 response options: "No or only once," "Sometimes," and "Yes, almost all the time", which enable derivation of an overall communication score and scores for each form of communication (expressive, receptive, and pragmatic), with higher scores indicating greater communication ability.
From Baseline (Day 1) to Week 14
Caregiver Global Impression of Change - Seizure (CaGIC-Seizure) score at Week 14
時間枠：At Week 14

The CaGIC-Seizure is a caregiver-reported item assessing the change from Baseline in the participant's seizure status. The CaGIC-Seizure uses a 7-point response scale, with the following ratings: "1: Very Much improved", "2: Much improved", "3: Minimally improved", "4: No change", "5: Minimally worse", "6: Much worse", "7: Very Much Worse".
At Week 14
Incidence of treatment-emergent adverse events (TEAEs) during the double-blind intervention period
時間枠：From Baseline (Day 1) up to Week 14

An AE is any untoward medical occurrence in a clinical study participant temporally associated with the use of study intervention, whether or not considered related to the study intervention. Treatment emergent adverse events (TEAEs) are adverse events that are not present prior to the pharmaceutical product administration or an already present event that worsens either in intensity or frequency.
From Baseline (Day 1) up to Week 14
Incidence of serious TEAEs during the double-blind intervention period
時間枠：From Baseline (Day 1) up to Week 14

An SAE is defined as any untoward medical occurrence that, at any dose, meets 1 or more of the criteria listed:

  • Results in death
  • Is life-threatening
  • Requires inpatient hospitalization or prolongation of existing hospitalization
  • Results in persistent or significant disability/incapacity
  • Is a congenital anomaly/birth defect
  • Other important medical events which based on medical or scientific judgement may jeopardize the patients or may require medical or surgical intervention to prevent any of the above.
From Baseline (Day 1) up to Week 14
Incidence of TEAEs leading to discontinuation during the double-blind intervention period
時間枠：From Baseline (Day 1) up to Week 14

An AE is any untoward medical occurrence in a clinical study participant temporally associated with the use of study intervention, whether or not considered related to the study intervention. Treatment emergent adverse events (TEAEs) are adverse events that are not present prior to the pharmaceutical product administration or an already present event that worsens either in intensity or frequency. TEAEs leading to discontinuation will be reported.
From Baseline (Day 1) up to Week 14
Incidence of related TEAEs during the double-blind intervention period
時間枠：From Baseline (Day 1) up to Week 14

An AE is any untoward medical occurrence in a clinical study participant temporally associated with the use of study intervention, whether or not considered related to the study intervention. Treatment emergent adverse events (TEAEs) are adverse events that are not present prior to the pharmaceutical product administration or an already present event that worsens either in intensity or frequency. Related TEAEs will be reported.
From Baseline (Day 1) up to Week 14
Change from Baseline in QT interval corrected using Fridericia's formula (QTcF) interval on 12-lead ECG by visit during the double-blind intervention period
時間枠：From Baseline (Day 1) up to Week 14

Change from Baseline in QTcF interval as measured by 12-lead ECG up to Week 14 by visit will be reported
From Baseline (Day 1) up to Week 14
Incidence of treatment-emergent Doppler Echocardiogram (ECHO) results meeting the Food and Drug Administration (FDA) case definition of drug-associated valvular heart disease (VHD) during the double-blind intervention period
時間枠：From Baseline (Day 1) up to Week 14

The FDA case definition of drug-associated VHD is aortic regurgitation ≥mild and/or mitral regurgitation ≥moderate with restricted valve motion, valve thickening, and/or physical signs or symptoms attributable to valve diseases. ECHO readings related to VHD on any of the 4 valves (aortic, mitral, pulmonary, tricuspid) will be reported with grades of absent, trace, mild, moderate, or severe.
From Baseline (Day 1) up to Week 14
Incidence of treatment-emergent Doppler ECHO results meeting the FDA case definition of PAH (defined as a PASP >35mmHg) during the double-blind intervention period
時間枠：From Baseline (Day 1) up to Week 14

ECHO readings related to pulmonary arterial hypertenstion (PAH) will be reported based on Pulmonary artery systolic pressure (PASP).
From Baseline (Day 1) up to Week 14
Incidence of treatment-emergent adverse events (TEAEs) from baseline to end of safety follow up
時間枠：From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)

An AE is any untoward medical occurrence in a clinical study participant temporally associated with the use of study intervention, whether or not considered related to the study intervention. Treatment emergent adverse events (TEAEs) are adverse events that are not present prior to the pharmaceutical product administration or an already present event that worsens either in intensity or frequency.
From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)
Incidence of serious TEAEs from baseline to end of safety follow up
時間枠：From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)

An SAE is defined as any untoward medical occurrence that, at any dose, meets 1 or more of the criteria listed:

  • Results in death
  • Is life-threatening
  • Requires inpatient hospitalization or prolongation of existing hospitalization
  • Results in persistent or significant disability/incapacity
  • Is a congenital anomaly/birth defect
  • Other important medical events which based on medical or scientific judgement may jeopardize the patients or may require medical or surgical intervention to prevent any of the above.
From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)
Incidence of TEAEs leading to discontinuation from baseline to end of safety follow up
時間枠：From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)

An AE is any untoward medical occurrence in a clinical study participant temporally associated with the use of study intervention, whether or not considered related to the study intervention. Treatment emergent adverse events (TEAEs) are adverse events that are not present prior to the pharmaceutical product administration or an already present event that worsens either in intensity or frequency. TEAEs leading to discontinuation will be reported.
From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)
Incidence of related TEAEs from baseline to end of safety follow up
時間枠：From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)

An AE is any untoward medical occurrence in a clinical study participant temporally associated with the use of study intervention, whether or not considered related to the study intervention. Treatment emergent adverse events (TEAEs) are adverse events that are not present prior to the pharmaceutical product administration or an already present event that worsens either in intensity or frequency. Related TEAEs will be reported.
From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)
Change from Baseline in QT interval corrected using Fridericia's formula (QTcF) interval on 12-lead ECG by visit from baseline to end of safety follow up
時間枠：From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)

Change from Baseline in QTcF interval as measured by 12-lead ECG up to Week 98 by visit will be reported
From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)
Incidence of treatment-emergent Doppler Echocardiogram (ECHO) results meeting the Food and Drug Administration (FDA) case definition of drug-associated valvular heart disease (VHD) from baseline to end of safety follow up
時間枠：From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)

The FDA case definition of drug-associated VHD is aortic regurgitation ≥mild and/or mitral regurgitation ≥moderate with restricted valve motion, valve thickening, and/or physical signs or symptoms attributable to valve diseases. ECHO readings related to VHD on any of the 4 valves (aortic, mitral, pulmonary, tricuspid) will be reported with grades of absent, trace, mild, moderate, or severe.
From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)
Incidence of treatment-emergent Doppler ECHO results meeting the FDA case definition of PAH (defined as a PASP >35mmHg) from baseline to end of safety follow up
時間枠：From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)

ECHO readings related to pulmonary arterial hypertenstion (PAH) will be reported based on Pulmonary artery systolic pressure (PASP).
From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)

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

UCB BIOSCIENCES, Inc.

捜査官

  • スタディディレクター：UCB Cares、0018445992273

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 (実際)

2026年7月14日

一次修了 (推定)

2029年2月6日

研究の完了 (推定)

2031年8月29日

試験登録日

最初に提出

2026年3月25日

QC基準を満たした最初の提出物

2026年3月25日

最初の投稿 (実際)

2026年3月31日

学習記録の更新

投稿された最後の更新 (実際)

2026年11月2日

QC基準を満たした最後の更新が送信されました

2026年10月30日

最終確認日

2026年10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

  • EP0247
  • 2025-523157-34 (その他の識別子：EU CTR)
  • 2025 (米国 NIH グラント/契約)

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?

はい

IPD プランの説明

Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.

IPD 共有時間枠

Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.

IPD 共有アクセス基準

Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.

IPD 共有サポート情報タイプ

  • STUDY_PROTOCOL
  • SAP
  • CSR

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

この情報は、Web サイト clinicaltrials.gov から変更なしで直接取得したものです。研究の詳細を変更、削除、または更新するリクエストがある場合は、register@clinicaltrials.gov。 までご連絡ください。 clinicaltrials.gov に変更が加えられるとすぐに、ウェブサイトでも自動的に更新されます。

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