EUCTR2011-001952-12-GB进行中（未招募）1 期
An open-label study to evaluate the long-term safety, tolerability and efficacy of AFQ056 in adult 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
- 148
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
入选标准
- •The study will be conducted in centers participating in studies CAFQ056A2212 or CAFQ056A2204 and other studies of AFQ056 which
- •included adult patients with FXS.
- •Group 1 patients
- •1. Must have completed the CAFQ056A2212 study or another study of
- •AFQ056 which included adult FXS patients within one week of
- •enrollment into the open-label study.
- •2. Written informed consent must be obtained from the patient's legally acceptable representative before any assessment is performed;
- •3. Male or female, at least 18 years of age at the time of screening in the previous study of AFQ056;
- •4. Have a caregiver or caregivers who spend(s), on average, at least six
- •hours per day with the patient, who is/are willing and capable of
- •supervising treatment, providing input into efficacy and safety
- •assessments, and accompanying the patient to study visits.
- •Group 2 patients
- •1. Must meet one of the following conditions:
- •a. completed Study CAFQ056A2204
- •b. completed Study CAFQ056A2212 or another study of AFQ056 which
- •included adult patients with FXS but enrollment into the current study
- •was delayed for more than a week
- •c. discontinued prematurely from Study CAFQ056A2212 or another study of AFQ056 which included adult patients with FXS due to intolerability of the dosage in the patient's assigned treatment group (Patients who discontinue prematurely from their previous study due to intolerability of their assigned dose Other protocol-defined inclusion criteria may apply level may enter the current study, but at a date no sooner than their originallyscheduled completion of the prior study)
- •2. Written informed consent must be obtained from the patient's legally acceptable representative before any assessment is performed;
- •3. Male or female, at least 18 years of age at the time of screening in the previous study of AFQ056
- •4. Have a caregiver or caregivers who spend(s), on average, at least six
- •hours per day with the patient, who is/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? no
- •Number of subjects for this age range: 0
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 200
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range 0
排除标准
- •1. Any advanced, severe or unstable disease
- •2. past medical history of clinically significant ECG abnormalities or QTcF
- •> 450 msec for males and > 470 msec for females at screening (Group
- •2) or at the end of study visit in the previous trial for Group 1 patients
- •3. lab screening values that include AST, ALT, GGT, total bilirubin or
- •creatinine = 1.5 X ULN (upper limit of normal) for the central laboratory; total or unconjugated (indirect) bilirubin levels up to 3 X ULN for the central laboratory are allowed if the patient has a diagnosis of Gilbert's syndrome;
- •4. 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;
- •5. history and/or presence of schizophrenia, bipolar disease, psychosis,
- •confusional states and/or repeated hallucinations as per Diagnostic and
- •Statistical Manual of Mental Disorders (DSM-IV) criteria;
- •6. history of suicidal behavior or considered a high suicidal risk;
- •7. History of severe self-injurious behavior
- •8. History of uncontrolled seizure disorder or resistant to therapy within
- •the past 2 years (Patients who are clinically stable under anti-convulsant therapy for the past 2 years are not excluded)
- •9. History of clinically significant allergies requiring hospitalization or
- •non-inhaled corticosteroid therapy (asthma, anaphylaxis, etc.)
- •10. 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;
- •11. 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;
- •12. patients who are using (or used within 6 weeks before baseline)
- •concomitant medications that are potent inhibitors or inducers of CYP3A4 (e.g., ketoconazole, ritonavir, carbamazepine, etc.) (Refer to Appendix 2 for a list of medications). In addition, patients are to avoid drinking grapefruit juice during the study;
- •13. patients who are using (or used within 6 weeks before baseline)
- •digoxin or warfarin; 14. patients using glutamatergic agents (riluzole,
- •memantine, etc.) or lithium within 6 weeks of baseline (Refer to
- •Appendix 2 for a list of medications);
- •15. use of investigational drugs (other than AFQ056) at the time of
- •enrollment, or within 6 weeks or 5 half-lives of baseline, whichever is
- •16. 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;
- •17. patients who, in the opinion of the investigator, are unsuitable in any other way to participate in this study, including being unable to
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