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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT01433354




Registration number
NCT01433354
Ethics application status
Date submitted
31/08/2011
Date registered
13/09/2011
Date last updated
24/03/2016

Titles & IDs
Public title
Long-term, Safety and Tolerability Study of AFQ056 in Adolescent Patients With Fragile X Syndrome (Open-label)
Scientific title
An Open-label Study to Evaluate the Long-term Safety and Tolerability of AFQ056 in Adolescent Patients With Fragile X Syndrome
Secondary ID [1] 0 0
2011-002379-40
Secondary ID [2] 0 0
CAFQ056B2278
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Fragile X Syndrome 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Mental Health 0 0 0 0
Learning disabilities
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - AFQ056

Experimental: AFQ056 Treatment - All patients will initiate treatment with AFQ056 at a starting dose of 25 mg b.i.d. The dose will be titrated from 25 mg b.i.d to 50 mg b.i.d., 75 mg b.i.d. and 100 mg b.i.d. at weekly intervals. Dose adjustments (up- and down-titrations) will be permitted as needed to manage any tolerability issues and to ensure that patients reach their highest tolerated dose, not to exceed 100 mg b.i.d.


Treatment: Drugs: AFQ056
The investigational drug, AFQ056, will be provided as hard gelatin capsules. Two different oral dosage strengths,25mg and 100 mg, identical in appearance, will be used.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs) - Adverse events were summarized for the open-label treatment period, where the open-label treatment period is defined based on how AEs were collected and reported according to the manner in which participants entered the current study and which treatment (AFQ056 or placebo) they were receiving in the previous study.
AEs which were continuing from the core study or that started after the end of core study but prior to first dose of open-label study medication in the extension study for Category 1 participants are shown under 'Prior to Ext. first dose'.
AEs which started during the open-label treatment period are presented based on the last AFQ056 dose taken on or before the onset date of the AE (25 mg bid; 50 mg bid; 75 mg bid; or 100 mg bid). No efficacy data presented as study was terminated.
Timepoint [1] 0 0
Prior to first dose in extension study, Baseline (start of study treatment in extension study) to End of trial

Eligibility
Key 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 or caregivers who spend, 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.

- Group 2 patients:

- Must meet one of the following conditions:

- Completed Study CAFQ056B2131

- 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.

- 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 or caregivers who spend, 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.
Minimum age
12 Years
Maximum age
18 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Discontinuation from Study CAFQ056B2214 or CAFQ056B2131 or another study of AFQ056
which included FXS patients below 18 years of age due to safety reasons

- Female patients who are sexually active at any time during the study

- Any advanced, severe or unstable disease

- History and/or presence of schizophrenia, bipolar disease, psychosis, confusional
states and/or repeated hallucinations as per DSM-IV criteria

- History of suicidal behavior or considered a high suicidal risk

- History of severe self-injurious behavior

- 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)

- History of clinically significant allergies requiring hospitalization or non-inhaled
corticosteroid therapy (asthma, anaphylaxis, etc.)

- 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

- Patients who are using (or used within 6 weeks before baseline) digoxin or warfarin

- Using (or used within 6 weeks before baseline) concomitant medications that are potent
inhibitors or inducers of CYP3A4

- Using glutamatergic agents (riluzole, memantine, etc.) or lithium within 6 weeks of
baseline

Other protocol-defined inclusion/exclusion criteria may apply.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2/Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Terminated
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Novartis Investigative Site - Westmead
Recruitment hospital [2] 0 0
Novartis Investigative Site - Parkville
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Georgia
Country [3] 0 0
United States of America
State/province [3] 0 0
Illinois
Country [4] 0 0
United States of America
State/province [4] 0 0
Massachusetts
Country [5] 0 0
United States of America
State/province [5] 0 0
Nebraska
Country [6] 0 0
United States of America
State/province [6] 0 0
New York
Country [7] 0 0
United States of America
State/province [7] 0 0
Tennessee
Country [8] 0 0
Belgium
State/province [8] 0 0
Bruxelles
Country [9] 0 0
Belgium
State/province [9] 0 0
Leuven
Country [10] 0 0
Denmark
State/province [10] 0 0
Glostrup
Country [11] 0 0
France
State/province [11] 0 0
Bron Cedex
Country [12] 0 0
Germany
State/province [12] 0 0
Mainz
Country [13] 0 0
Germany
State/province [13] 0 0
München
Country [14] 0 0
Germany
State/province [14] 0 0
Tübingen
Country [15] 0 0
Germany
State/province [15] 0 0
Würzburg
Country [16] 0 0
Israel
State/province [16] 0 0
Ramat Gan
Country [17] 0 0
Italy
State/province [17] 0 0
GE
Country [18] 0 0
Italy
State/province [18] 0 0
Padova
Country [19] 0 0
Netherlands
State/province [19] 0 0
Rotterdam
Country [20] 0 0
Spain
State/province [20] 0 0
Andalucia
Country [21] 0 0
Spain
State/province [21] 0 0
Barcelona
Country [22] 0 0
Spain
State/province [22] 0 0
Cataluña
Country [23] 0 0
Sweden
State/province [23] 0 0
Spånga
Country [24] 0 0
Switzerland
State/province [24] 0 0
Lausanne
Country [25] 0 0
Switzerland
State/province [25] 0 0
Zurich
Country [26] 0 0
United Kingdom
State/province [26] 0 0
Edinburgh

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Novartis Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to generate long-term safety, tolerability and efficacy data for
AFQ056 in eligible adolescent patients with FXS who have participated in the CAFQ056B2214
study, the PK study CAFQ056B2131, or another study of AFQ056 which included FXS patients
below 18 years of age provided the patient is at least 12 years of age at the time of entry
into the current study.
Trial website
https://clinicaltrials.gov/show/NCT01433354
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Novartis Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT01433354