EUCTR2010-022638-96-FR进行中（未招募）1 期
A randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy and safety 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
- •Patients eligible for inclusion in this study have to fulfill all of the following criteria at Screening (V1), unless another time point prior to randomization is specified:
- •1. the caregiver/legal guardian 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 (in accordance with local ethical/regulatory requirements);
- •2. male or female, between 12 and 17 years of age, inclusive;
- •3. have a previous diagnosis of FXS based upon documented genetic testing results (full mutation >200 CGG repeats). The diagnosis will need to be confirmed by genetic testing prior to the patient entering the Placebo Run-in Period;
- •4. have a Clinical Global Impression Severity Score (CGI-S) of = 4 (moderately ill);
- •5. have a score of > 20 in the ABC-C total scale;
- •6. have an IQ < 70 as measured by the Leiter International Performance Scale – Revised (Leiter-R);
- •7. have a caregiver who spends, on average, at least six hours per day with the patient, who is willing and capable of supervising treatment, providing input into efficacy and safety assessments, and accompanying the patient to all study visits;
- •8. have a caregiver who, in the opinion of the investigator, is a reliable source of information regarding the patient and the severity of his/her disease.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 180
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Patients fulfilling any of the following criteria prior to randomization are not eligible
- •for inclusion in this study :
- •1. female patients who are sexually active at any time during the study. Sexual activity should be carefully evaluated by the Investigator and if sexual activity is
- •confirmed or suspected, the female participant should not be enrolled into the study.
- •Furthermore, the Investigator should continue to evaluate the possibility of sexual activity throughout the study and the caregiver/legal guardian should be instructed to immediately inform the Investigator if the female participant becomes or is suspected to have become sexually active during the study. In these cases, appropriate actions should be taken by the Investigator and the patient must be discontinued from the study;
- •2. any advanced, severe or unstable disease that may interfere with the primary or secondary study outcome evaluations or put the patient at special risk;
- •3. past medical history of clinically significant ECG abnormalities or QTcF > 450 msec for males and > 470 msec for females at screening or baseline;
- •4. 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;
- •5. have history of major surgery or major medical event that require hospitalization or major surgery within the past 6 months, unless they recovered fully or are considered clinically stable;
- •6. 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;
- •7. 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;
- •8. history of suicidal behavior or considered a high suicidal risk;
- •9. history of severe self-injurious behavior;
- •10. 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 allowed);
- •11. history of clinically significant allergies requiring hospitalization or non-inhaled
- •corticosteroid therapy (asthma, anaphylaxis, etc.);
- •12. 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;
- •13. pregnant or nursing (lactating) females, 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;
- •14. patients on any treatment regimen, including psychotropic and/or current anticonvulsant therapy that has not been stable for = 6 weeks prior to randomization;
- •15. current treatment with more than two psychoactive medications, excluding anti-epileptics;
- •16. patients who are using (or used within 6 weeks before randomization) 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);
- •17. patients who are using (or used within 6 weeks before randomization) digoxin or warfarin;
研究者
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