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Eine Phase-3-Studie zu Fenfluraminhydrochlorid bei Rett-Syndrom

30. Oktober 2026 aktualisiert von: UCB BIOSCIENCES, Inc.

Eine Phase-3-Studie, randomisiert, doppelblind, placebokontrolliert, mit parallelen Gruppen und mehreren Zentren, mit offener Verlängerung zur Bewertung der Wirksamkeit und Sicherheit von Fenfluramin-Hydrochlorid bei Studienteilnehmern mit Rett-Syndrom

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

Studienübersicht

Status

Rekrutierung

Bedingungen

Intervention / Behandlung

Studientyp

Interventionell

Einschreibung (Geschätzt)

200

Phase

  • Phase 3

Kontakte und Standorte

Dieser Abschnitt enthält die Kontaktdaten derjenigen, die die Studie durchführen, und Informationen darüber, wo diese Studie durchgeführt wird.

Studienkontakt

Studieren Sie die Kontaktsicherung

Studienorte

  • Belgien
      • Brussels, Belgien
        • Rekrutierung
        • Ep0247 11001
      • Edegem, Belgien
        • Rekrutierung
        • Ep0247 11002
  • Deutschland
      • Vogtareuth, Deutschland
        • Rekrutierung
        • Ep0247 14001
  • Frankreich
      • Brest, Frankreich
        • Rekrutierung
        • Ep0247 12005
      • Bron, Frankreich
        • Rekrutierung
        • Ep0247 12003
      • Marseille, Frankreich
        • Rekrutierung
        • Ep0247 12004
      • Paris, Frankreich
        • Rekrutierung
        • Ep0247 12006
      • Paris, Frankreich
        • Rekrutierung
        • Ep0247 12001
  • Georgia
      • Tbilisi, Georgia
        • Rekrutierung
        • Ep0247 13001
      • Tbilisi, Georgia
        • Rekrutierung
        • Ep0247 13002
  • Israel
      • Ramat Gan, Israel
        • Rekrutierung
        • Ep0247 16001
  • Italien
      • Genova, Italien
        • Rekrutierung
        • Ep0247 17003
      • Milan, Italien
        • Rekrutierung
        • Ep0247 17001
      • Naples, Italien
        • Rekrutierung
        • Ep0247 17008
      • Pisa, Italien
        • Rekrutierung
        • Ep0247 17002
      • Troina, Italien
        • Rekrutierung
        • Ep0247 17006
  • Japan
      • Chiyoda-ku, Japan
        • Rekrutierung
        • Ep0247 43005
      • Kodaira, Japan
        • Rekrutierung
        • Ep0247 43004
      • Kurume, Japan
        • Rekrutierung
        • Ep0247 43001
      • Musashimurayama-shi, Japan
        • Rekrutierung
        • Ep0247 43006
      • Osaka, Japan
        • Rekrutierung
        • Ep0247 43002
      • Setagaya-ku, Japan
        • Rekrutierung
        • Ep0247 43003
  • Polen
      • Gdansk, Polen
        • Rekrutierung
        • Ep0247 19003
      • Krakow, Polen
        • Rekrutierung
        • Ep0247 19002
  • Spanien
      • Madrid, Spanien
        • Rekrutierung
        • Ep0247 11105
      • Madrid, Spanien
        • Rekrutierung
        • Ep0247 11101
      • Málaga, Spanien
        • Rekrutierung
        • Ep0247 11103
      • Seville, Spanien
        • Rekrutierung
        • Ep0247 11102
  • Ungarn
      • Budapest, Ungarn
        • Rekrutierung
        • Ep0247 15001
      • Debrecen, Ungarn
        • Rekrutierung
        • Ep0247 15002
  • Vereinigte Staaten
    • Arkansas
      • Little Rock, Arkansas, Vereinigte Staaten, 72202
        • Rekrutierung
        • Ep0247 21010
    • Florida
      • Miami, Florida, Vereinigte Staaten, 33155
        • Rekrutierung
        • Ep0247 21009
      • Orlando, Florida, Vereinigte Staaten, 32806
        • Rekrutierung
        • Ep0247 21011
    • Georgia
      • Atlanta, Georgia, Vereinigte Staaten, 30322
        • Rekrutierung
        • Ep0247 21007
    • Pennsylvania
      • Philadelphia, Pennsylvania, Vereinigte Staaten, 19104-4318
        • Rekrutierung
        • Ep0247 21003
    • Texas
      • Houston, Texas, Vereinigte Staaten, 77030
        • Rekrutierung
        • Ep0247 21005
      • Irving, Texas, Vereinigte Staaten, 75062
        • Rekrutierung
        • Ep0247 21013
    • Washington
      • Seattle, Washington, Vereinigte Staaten, 98105
        • Rekrutierung
        • Ep0247 21004
  • Vereinigtes Königreich
      • Manchester, Vereinigtes Königreich
        • Rekrutierung
        • Ep0247 11303
      • Nottingham, Vereinigtes Königreich
        • Rekrutierung
        • Ep0247 11304

Teilnahmekriterien

Forscher suchen nach Personen, die einer bestimmten Beschreibung entsprechen, die als Auswahlkriterien bezeichnet werden. Einige Beispiele für diese Kriterien sind der allgemeine Gesundheitszustand einer Person oder frühere Behandlungen.

Zulassungskriterien

Studienberechtigtes Alter

5 Jahre bis 35 Jahre (Kind, Erwachsene)

Akzeptiert gesunde Freiwillige

Nein

Studienberechtigte Geschlechter

Alle

Beschreibung

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

Studienplan

Dieser Abschnitt enthält Einzelheiten zum Studienplan, einschließlich des Studiendesigns und der Messung der Studieninhalte.

Wie ist die Studie aufgebaut?

Designdetails

  • Hauptzweck: Behandlung
  • Zuteilung: Zufällig
  • Interventionsmodell: Parallele Zuordnung
  • Maskierung: Verdreifachen

Waffen und Interventionen

Teilnehmergruppe / Arm
Intervention / Behandlung
Experimental: 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.
Arzneimittel: fenfluramine hydrochloride

Oral solution

Andere Namen:
  • Fintepla, ZX008
Placebo-Komparator: 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.
Sonstiges: Placebo

Oral solution

Was misst die Studie?

Primäre Ergebnismessungen

Ergebnis Maßnahme
Maßnahmenbeschreibung
Zeitfenster
Change from Baseline to Week 14 in Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score
Zeitfenster: 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
Zeitfenster: 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

Sekundäre Ergebnismessungen

Ergebnis Maßnahme
Maßnahmenbeschreibung
Zeitfenster
Change from Baseline to Week 14 in Patient-Reported Outcomes Measurement Information System-Sleep Disturbance (PROMIS-SD) score
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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)

Mitarbeiter und Ermittler

Hier finden Sie Personen und Organisationen, die an dieser Studie beteiligt sind.

Sponsor

UCB BIOSCIENCES, Inc.

Ermittler

  • Studienleiter: UCB Cares, 0018445992273

Studienaufzeichnungsdaten

Diese Daten verfolgen den Fortschritt der Übermittlung von Studienaufzeichnungen und zusammenfassenden Ergebnissen an ClinicalTrials.gov. Studienaufzeichnungen und gemeldete Ergebnisse werden von der National Library of Medicine (NLM) überprüft, um sicherzustellen, dass sie bestimmten Qualitätskontrollstandards entsprechen, bevor sie auf der öffentlichen Website veröffentlicht werden.

Haupttermine studieren

Studienbeginn (Tatsächlich)

14. Juli 2026

Primärer Abschluss (Geschätzt)

6. Februar 2029

Studienabschluss (Geschätzt)

29. August 2031

Studienanmeldedaten

Zuerst eingereicht

25. März 2026

Zuerst eingereicht, das die QC-Kriterien erfüllt hat

25. März 2026

Zuerst gepostet (Tatsächlich)

31. März 2026

Studienaufzeichnungsaktualisierungen

Letztes Update gepostet (Tatsächlich)

2. November 2026

Letztes eingereichtes Update, das die QC-Kriterien erfüllt

30. Oktober 2026

Zuletzt verifiziert

1. Oktober 2026

Mehr Informationen

Begriffe im Zusammenhang mit dieser Studie

Schlüsselwörter

Zusätzliche relevante MeSH-Bedingungen

Andere Studien-ID-Nummern

  • EP0247
  • 2025-523157-34 (Andere Kennung: EU CTR)
  • 2025 (US NIH Stipendium/Vertrag)

Plan für individuelle Teilnehmerdaten (IPD)

Planen Sie, individuelle Teilnehmerdaten (IPD) zu teilen?

JA

Beschreibung des IPD-Plans

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-Sharing-Zeitrahmen

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-Sharing-Zugriffskriterien

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.

Art der unterstützenden IPD-Freigabeinformationen

  • STUDIENPROTOKOLL
  • SAFT
  • CSR

Arzneimittel- und Geräteinformationen, Studienunterlagen

Studiert ein von der US-amerikanischen FDA reguliertes Arzneimittelprodukt

Ja

Studiert ein von der US-amerikanischen FDA reguliertes Geräteprodukt

Nein

Diese Informationen wurden ohne Änderungen direkt von der Website clinicaltrials.gov abgerufen. Wenn Sie Ihre Studiendaten ändern, entfernen oder aktualisieren möchten, wenden Sie sich bitte an register@clinicaltrials.gov. Sobald eine Änderung auf clinicaltrials.gov implementiert wird, wird diese automatisch auch auf unserer Website aktualisiert .

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