EUCTR2011-002379-40-SE进行中（未招募）不适用
An open-label study to evaluate the long-term safety and tolerability of AFQ056 in adolescent patients with Fragile X Syndrome
适应症
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 180
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Group 1 patients:
- •- Must have completed study CAFQ056B2214 or another study of AFQ056 which included FXS patients below 18 years of age within one week of enrollment into the open-label study.
- •- Has a caregiver who spends, on average, at least 6 hours per day with the patient , who is willing to and capable of supervising treatment, providing input into efficacy and safety assessments, and accompanying the patient to study visits.
- •-Male or female, between 12 and 17 years of age, inclusive, at the time of inclusion in the CAFQ056B2214 study or at least 12 years of age at the time of entry into the current study when the patient participated in another study of AFQ056 which included FXS patients below 18 years of age;
- •-Have a caregiver or caregivers who are willing and capable of supervising treatment, providing input into efficacy and safety assessments, and accompanying the patient to study visits
- •Group 2 patients:
- •Must meet one of the following conditions:
- •o completed Study CAFQ056B2131
- •o completed Study CAFQ056B2214 or another study of AFQ056 which included FXS patients below 18 years of age but enrollment into the current study was delayed for more than a week
- •o discontinued prematurely from Study CAFQ056B2214 or another study of AFQ056 which included FXS patients below 18 years of age due to intolerability of the dosage in the patient's assigned treatment group
- •- Has a caregiver who spends, on average, at least 6 hours per day with the patient , who is willing to and capable of supervising treatment, providing input into efficacy and safety assessments, and accompanying the patient to study visits
- •-The caregiver/legally acceptable representative must be able to communicate well with the investigator and must understand and support the study requirements by providing written informed consent.
- •Where possible the patient should also provide his or her written assent
- •Other protocol-defined inclusion criteria may apply
- •-Male or female, between 12 and 17 years of age, inclusive, at the time of inclusion in Study CAFQ056B2214, or between 12 and 18 years of age, inclusive, at the time of inclusion in Study CAFQ056B2131, or at least 12 years of age at the time of entry into the current study when the patient participated in another study of AFQ056 which included FXS patients
- •below 18 years of age
- •-Have a caregiver or caregivers who are willing and capable of supervising treatment, providing input into efficacy and safety assessments, and accompanying the patient to study visits
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 160
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 20
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1) Discontinuation from study CAFQ056B2214 or CAFQ056B2131 or another study of AFQ056 which included FXS patients below 18 years of age due to safety reasons
- •2) Female patients who are sexually active at any time during the study
- •3) Any advanced, severe or unstable disease
- •4) past medical history of clinically significant ECG abnormalities or QTcF > 450 msec for males and > 470 msec for females during the screening period (Group 2 patients) or at the end of study visit in the previous study for Group 1 patients;
- •5) lab screening values that include AST, ALT, GGT, total bilirubin or creatinine = 1.5 X ULN (upper limit of normal) for the central laboratory
- •6)history of major surgery or major medical event that requires hospitalization or major surgery within the past 6 months, unless the patient recovered fully or is considered clinically stable by the study physician
- •7)donated or lost more than 2.4 mL of blood per kg of body weight within (4) weeks prior to Screening (V1) or longer if required by local regulation;
- •8) History and/or presence of schizophrenia, bipolar disease, psychosis, confusional states and/or repeated hallucinations as per DSM-IV criteria
- •9) History of suicidal behavior or considered a high suicidal risk
- •10) History of severe self-injurious behavior
- •11) History of uncontrolled seizure disorder or resistant to therapy within the past 2 years (Patients who are clinically stable under anticonvulsant therapy for the past 2 years are not excluded)
- •12) History of clinically significant allergies requiring hospitalization or non-inhaled corticosteroid therapy (asthma, anaphylaxis, etc.)
- •13) History of malignancy of any organ system (other than localized basal cell carcinoma of the skin), treated or untreated, within the past 5 years, regardless of whether or not there is evidence of local recurrence or metastases
- •14)pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG serum pregnancy test;
- •15) Using (or used within 6 weeks before randomization) concomitant medications that are potent inhibitors or inducers of CYP3A4, In addition, patients are to avoid drinking grapefruit juice during the study
- •16) patients who are using (or used within 6 weeks before baseline) digoxin or warfarin
- •17) Using glutamatergic agents (riluzole, memantine, etc.) or lithium within 6 weeks of baseline
- •18)use of investigational drugs (other than AFQ056) at the time of enrollment, or within 6 weeks or 5 half-lives of baseline, whichever is longer
- •19)participation in any pharmacological clinical investigation (other than observational studies or those including AFQ056) within 6 weeks prior to baseline or longer if required by local regulation
- •20)patients who, in the opinion of the investigator, are unsuitable in any other way to participate in this study, including being unable to comply with the requirements of the study or displaying bnormalities in safety assessments at baseline
- •21) patients who are unable to swallow study medication in a capsule formulation
- •Other protocol-defined exclusion criteria may apply
研究者
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