ACTRN12618001063202已完成2 期
A Randomized, Double-Blind, Placebo-Controlled Multiple-Center, Efficacy andSafety Study of ZYN002 Administered as a Transdermal Gel to Children andAdolescents with Fragile X Syndrome – CONNECT-FX
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 212
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomised controlled trial
- 主要目的
- Treatment
- 盲法
- Blinded (masking used)
入排标准
- 年龄范围
- 3 Years 至 17 Years（—）
- 性别
- All
入选标准
- •1.Male or female children and adolescents aged 3 to less than 18 years, at the time of Screening.
- •2.Judged by the Investigator to be in generally good health at Screening based upon the results of a medical history, physical examination, 12-lead ECG, and clinical laboratory test results. Laboratory results outside of the reference range must be documented as not clinically significant by both the Investigator and Sponsor.
- •3.Patients must have a diagnosis of FXS through molecular documentation of FMR1 full mutation.
- •4.Patients must be assessed by the Investigator as being moderately to severely impacted due to FXS.
- •5.Patients with a history of seizure disorders must currently be receiving treatment with a stable regimen of one or two AEDs, or must be seizure-free for one year if not currently receiving AEDs.
- •6.Patients who are taking psychotropic medication(s) should be on a stable regimen of no more than two such medications for at least four weeks preceding study Screening and must maintain that regimen throughout the study.
- •7.If patients are receiving non-pharmacological, behavioral, and/or dietary interventions, they must be stable and have been doing so for three months prior to Screening.
- •8.Patients have a body mass index between 12–30 kg /m2 (inclusive).
- •9.Females of childbearing potential must have a negative serum pregnancy test at the Screening Visit and a negative serum or urine pregnancy test at all designated visits.
- •10.Patients and parents/caregivers must be adequately informed of the nature and risks of the study and give written informed consent (and assent if applicable) prior to Screening.
- •11.Parents/caregiver(s) must provide written consent to assist in trial drug administration.
- •12.In the Investigator’s opinion, patients and parents/caregivers are reliable and willing and able to comply with all protocol requirements and procedures.
排除标准
- •1.Females who are pregnant, nursing, or planning a pregnancy; females of childbearing potential and male patients with a partner of childbearing potential who are unwilling or unable to use an acceptable method of contraception for the duration of therapy and for three months after the last dose of study medication.
- •2.History of significant allergic condition, significant drug-related hypersensitivity, or allergic reaction to any compound or chemical class related to ZYN002 or its excipients.
- •3.Exposure to any investigational drug or device <=30 days prior to Screening or at any time during the study.
- •4.Patient has alanine aminotransferase (ALT), aspartate aminotransferase (AST), or total bilirubin levels greater than or equal to 2 times the upper limit of normal (ULN) or has alkaline phosphatase levels greater than or equal to 3 times the ULN as determined from Screening safety laboratories.
- •5.Use of cannabis or any THC or CBD-containing product (aside from ZYN002) within three months of Screening Visit or during the study.
- •6.Patient has a positive drug screen, including ethanol, cocaine, THC, barbiturates, amphetamines (unless prescribed), benzodiazepines, and opiates.
- •7.Patient is using the following AEDs: clobazam, phenobarbital, ethosuximide, felbamate, or vigabatrin.
- •8.Patient is using any strong inhibitor/inducer of CYP3A4 or sensitive substrate for CYP3A4
- •9.Patients may not be taking minocycline for 30 days prior to Screening or throughout the study.
- •10.Patients may not be taking any benzodiazepines at screening or throughout the study.
- •11.Patient has an advanced, severe, or unstable disease that may interfere with the study outcome evaluations.
- •12.Patient is expected to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study.
- •13.Patient has an acute or progressive neurological disease, psychosis, schizophrenia, or any psychiatric disorder or severe mental abnormalities (other than Fragile X Syndrome) that are likely to require changes in drug therapy or interfere with the objectives of the study or the ability to adhere to protocol requirements.
- •14.Patient has a positive result for the presence of Hepatitis B Surface antigen (HBsAg), Hepatitis C virus antibodies (HCV), or human immunodeficiency virus (HIV) antibodies.
- •15.Has suspected or confirmed cardiovascular disease, advanced arteriosclerosis, structural cardiac abnormality, cardiomyopathy, serious heart rhythm abnormalities, coronary artery disease, cardiac conduction problems, exercise-related cardiac events including syncope and pre-syncope, risk factors for Torsades de pointes (e.g. heart failure, hypokalemia, family history of Long QT Syndrome), or other serious cardiac problems .
- •16.Any clinically significant condition or abnormal findings at the Screening Visit that would, in the opinion of the Investigator, preclude study participation or interfere with the evaluation of the study medication.
- •17.Any skin disease or condition, including eczema, psoriasis, melanoma, acne, contact dermatitis, scarring, imperfections, lesions, tattoos, or discoloration, that may affect treatment application, application site assessments, or absorption of the trial drug.
- •18.History of treatment for, or evidence of, drug abuse within the past year.
- •19.Previous participation in a ZYN002 study.
- •20.Patient responds yes” to Question 4 or 5 on the C-SSRS (Children) during Screening or at any time on study
研究者
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