레트 증후군에서 펜플루라민 염산염의 3상 임상시험
레트 증후군 환자를 대상으로 펜플루라민 하이드로클로라이드의 효능 및 안전성을 평가하기 위한 3상 무작위, 이중 맹검, 위약 대조, 평행군, 다기관 연구 및 개방 연장 연구
The purpose of this study is to investigate the efficacy of fenfluramine hydrochloride (HCl) versus placebo in study participants with Rett syndrome (RTT).
연구 개요
상태
상태
정황
정황
개입 / 치료
개입 / 치료
연구 유형
연구 유형
등록 (추정된)
등록
단계
단계
- 3단계
연락처 및 위치
연구 연락처
연구 연락처
- 이름: UCB Cares
- 전화번호: 0018445992273
- 이메일: UCBCares@ucb.com
연구 연락처 백업
- 이름: UCB Cares
- 전화번호: +18445992273
- 이메일: ucbcares@ucb.com
연구 장소
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Tbilisi, 그루지야
- 모병
- Ep0247 13001
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Tbilisi, 그루지야
- 모병
- Ep0247 13002
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Vogtareuth, 독일
- 모병
- Ep0247 14001
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Arkansas
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Little Rock, Arkansas, 미국, 72202
- 모병
- Ep0247 21010
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Florida
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Miami, Florida, 미국, 33155
- 모병
- Ep0247 21009
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Orlando, Florida, 미국, 32806
- 모병
- Ep0247 21011
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Georgia
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Atlanta, Georgia, 미국, 30322
- 모병
- Ep0247 21007
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Pennsylvania
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Philadelphia, Pennsylvania, 미국, 19104-4318
- 모병
- Ep0247 21003
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Texas
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Houston, Texas, 미국, 77030
- 모병
- Ep0247 21005
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Irving, Texas, 미국, 75062
- 모병
- Ep0247 21013
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Washington
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Seattle, Washington, 미국, 98105
- 모병
- Ep0247 21004
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Brussels, 벨기에
- 모병
- Ep0247 11001
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Edegem, 벨기에
- 모병
- Ep0247 11002
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Madrid, 스페인
- 모병
- Ep0247 11105
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Madrid, 스페인
- 모병
- Ep0247 11101
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Málaga, 스페인
- 모병
- Ep0247 11103
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Seville, 스페인
- 모병
- Ep0247 11102
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Manchester, 영국
- 모병
- Ep0247 11303
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Nottingham, 영국
- 모병
- Ep0247 11304
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Ramat Gan, 이스라엘
- 모병
- Ep0247 16001
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Genova, 이탈리아
- 모병
- Ep0247 17003
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Milan, 이탈리아
- 모병
- Ep0247 17001
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Naples, 이탈리아
- 모병
- Ep0247 17008
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Pisa, 이탈리아
- 모병
- Ep0247 17002
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Troina, 이탈리아
- 모병
- Ep0247 17006
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Chiyoda-ku, 일본
- 모병
- Ep0247 43005
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Kodaira, 일본
- 모병
- Ep0247 43004
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Kurume, 일본
- 모병
- Ep0247 43001
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Musashimurayama-shi, 일본
- 모병
- Ep0247 43006
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Osaka, 일본
- 모병
- Ep0247 43002
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Setagaya-ku, 일본
- 모병
- Ep0247 43003
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Gdansk, 폴란드
- 모병
- Ep0247 19003
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Krakow, 폴란드
- 모병
- Ep0247 19002
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Brest, 프랑스
- 모병
- Ep0247 12005
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Bron, 프랑스
- 모병
- Ep0247 12003
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Marseille, 프랑스
- 모병
- Ep0247 12004
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Paris, 프랑스
- 모병
- Ep0247 12006
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Paris, 프랑스
- 모병
- Ep0247 12001
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Budapest, 헝가리
- 모병
- Ep0247 15001
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Debrecen, 헝가리
- 모병
- Ep0247 15002
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참여기준
자격 기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
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:
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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.
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Participant has clinically significant abnormality in vital signs according to the Investigator
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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:
- Greater than trace aortic valve regurgitation.
- Greater than mild mitral valve regurgitation.
- Possible signs of pulmonary arterial hypertension (PAH) with abnormal pulmonary artery systolic pressure (PASP) or PASP ≥35 mmHg.
- Evidence of left ventricular dysfunction (systolic or diastolic).
- 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
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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
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Participant is taking >4 concomitant antiseizure medications (ASMs). Rescue medications are not included in the count
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 무작위
- 중재 모델: 병렬 할당
- 마스킹: 삼루타
팔의 수
무기와 개입
참가자 그룹 / 팔참가자 그룹 / 팔 |
개입 / 치료개입 / 치료 |
|---|---|
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실험적: 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. |
Oral solution
다른 이름들:
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위약 비교기: 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. |
Oral solution |
연구는 무엇을 측정합니까?
주요 결과 측정
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Change from Baseline to Week 14 in Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score
기간: From Baseline (Day 1) to Week 14
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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
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Clinical Global Impression of Change (CGIC) Score at Week 14
기간: At Week 14
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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
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2차 결과 측정
2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Change from Baseline to Week 14 in Patient-Reported Outcomes Measurement Information System-Sleep Disturbance (PROMIS-SD) score
기간: From Baseline (Day 1) to Week 14
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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
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Change from Baseline to Week 14 in Observer-Reported Communication Ability (ORCA) score
기간: From Baseline (Day 1) to Week 14
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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
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Caregiver Global Impression of Change - Seizure (CaGIC-Seizure) score at Week 14
기간: At Week 14
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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
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Incidence of treatment-emergent adverse events (TEAEs) during the double-blind intervention period
기간: From Baseline (Day 1) up to Week 14
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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
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Incidence of serious TEAEs during the double-blind intervention period
기간: From Baseline (Day 1) up to Week 14
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An SAE is defined as any untoward medical occurrence that, at any dose, meets 1 or more of the criteria listed:
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From Baseline (Day 1) up to Week 14
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Incidence of TEAEs leading to discontinuation during the double-blind intervention period
기간: From Baseline (Day 1) up to Week 14
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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
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Incidence of related TEAEs during the double-blind intervention period
기간: From Baseline (Day 1) up to Week 14
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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
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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
기간: From Baseline (Day 1) up to Week 14
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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
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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
기간: From Baseline (Day 1) up to Week 14
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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
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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
기간: From Baseline (Day 1) up to Week 14
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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
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Incidence of treatment-emergent adverse events (TEAEs) from baseline to end of safety follow up
기간: 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)
|
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Incidence of serious TEAEs from baseline to end of safety follow up
기간: 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:
|
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
기간: 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
기간: 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)
|
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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
기간: 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
기간: 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
기간: 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)
|
공동 작업자 및 조사자
수사관
수사관
- 연구 책임자: UCB Cares, 0018445992273
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
연구 시작
기본 완료 (추정된)
기본 완료
연구 완료 (추정된)
연구 완료
연구 등록 날짜
최초 제출
최초 제출
QC 기준을 충족하는 최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
처음 게시됨
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
마지막 업데이트 게시됨
QC 기준을 충족하는 마지막 업데이트 제출
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
추가 관련 MeSH 약관
기타 연구 ID 번호
기타 연구 ID 번호
- EP0247
- 2025-523157-34 (기타 식별자: EU CTR)
- 2025 (미국 NIH 보조금/계약)
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
IPD 계획 설명
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 공유 기간
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 공유 액세스 기준
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 공유 지원 정보 유형
- 연구_프로토콜
- 수액
- CSR
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
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. .