ong-term, safety and tolerability study of AFQ056 in adolescent patients with Fragile X Syndrome (Open–label)
- Conditions
- Fragile X syndromeMedDRA version: 14.1 Level: PT Classification code 10017324 Term: Fragile X syndrome System Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2011-002379-40-GB
- Lead Sponsor
- ovartis Pharma Services AG
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 119
Group 1 patients:
• Must have completed the 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 particiapted 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
1) Discontinuation from CAFQ056B2214 or CAFQ056B2131 or another study at 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 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 clinially 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 anti-convulsant 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 randiomization) concomitant medications that are potent inhibitors or inducers or 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 that 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 abnormalities in safety assessments at baseline
21) Patients who are unable to swallow study medication in a capsule formulation
Other pro
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method