ong-term, safety and tolerability study of AFQ056 in adolescent patients with Fragile X Syndrome (Open–label)

Phase 1

• Conditions: Fragile X syndrome; MedDRA version: 14.1Level: PTClassification code 10017324Term: Fragile X syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2011-002379-40-BE
• Lead Sponsor: ovartis Pharma Services AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-10-24
• Last Update Posted: 21 August 2017

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 180

Inclusion Criteria

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

Exclusion Criteria

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified