NCT07503444招募中3 期
A Phase 3 Randomized, Double-Blind, Placebo Controlled, Parallel Group, Multicenter Study With Open-Label Extension to Evaluate the Efficacy And Safety of Fenfluramine Hydrochloride in Study Participants With Rett Syndrome
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 200
- 试验地点
- 28
研究概览
简要总结
The purpose of this study is to investigate the efficacy of fenfluramine hydrochloride (HCl) versus placebo in study participants with Rett syndrome (RTT).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
盲法说明
The study consists of a Double-Blind and an Open-Label (OL) Intervention Period. During OL Intervention Period, the sponsor, participants, caregivers, and Investigators will be unblinded to treatment assignment.
入排标准
- 年龄范围
- 5 Years 至 35 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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:
- •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
- •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
研究组 & 干预措施
Placebo
Placebo Comparator
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.
干预措施: Placebo (Other)
fenfluramine hydrochloride
Experimental
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.
干预措施: fenfluramine hydrochloride (Drug)
结局指标
主要结局
未指定
次要结局
- Incidence of treatment-emergent adverse events (TEAEs) during the double-blind intervention period(From Baseline (Day 1) up to Week 14)
- Incidence of serious TEAEs during the double-blind intervention period(From Baseline (Day 1) up to Week 14)
- Incidence of TEAEs leading to discontinuation during the double-blind intervention period(From Baseline (Day 1) up to Week 14)
- Incidence of related TEAEs during the double-blind intervention period(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(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(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(From Baseline (Day 1) up to Week 14)
- Incidence of treatment-emergent adverse events (TEAEs) from baseline to end of safety follow up(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(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(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(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(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(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(From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months))
研究者
研究点 (28)
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