EUCTR2011-002379-40-BE进行中（未招募）1 期
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
- 1 期
- 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
- •-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
- •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
- •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
- •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
- 另有 2 项未显示
研究者
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