Fragile X SyndromePhase 2NCT01348087
Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome
A Phase 2 study testing AFQ056 in people with Fragile X Syndrome, run by Novartis Pharmaceuticals at 28 sites in 10 countries.
Trial summary
The purpose of this study is to generate long-term safety, tolerability and efficacy data for AFQ056 in eligible adult patients with FXS who have participated in the CAFQ056A2212 (NCT01253629).study and patients who have participated in the previous proof-of-concept study CAFQ056A2204 (NCT00718341). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Fragile X Syndrome
- What is being tested?
- AFQ056
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 148 participants
- Where
- 28 sitesin 10 countries
- Study dates
- Aug 2011to Sep 2014
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Had to 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.
- Females of child-bearing potential had to follow protocol requirements with respect to contraception.
- Have a caregiver or caregivers who spent, on average, at least six hours per day with the patient, who were willing and capable of supervising treatment, providing input into efficacy and safety assessments, and accompanying the patient to study visits.
- Group 2:
- Had to have:
- Completed Study CAFQ056A2204.
Show all 10Show fewer
- 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.
- 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.
- Females of child-bearing potential had to follow protocol requirements with respect to contraception.
- Have a caregiver or caregivers who spent, on average, at least six hours per day with the patient, who were willing and capable of supervising treatment, providing input into efficacy and safety assessments, and accompanying the patient to study visits
Who cannot join
- Any advanced, severe or unstable disease
- 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.)
- 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, digoxin, or warfarin within 6 weeks of baseline Other protocol-defined inclusion/exclusion criteria may apply
Trial locations and contacts
28 sites in 10 countries.
28 sites in 10 countries
United States10 sites
Novartis Investigative Site
Boston, Massachusetts, 02115
Novartis Investigative Site
Chicago, Illinois, 60612
Novartis Investigative Site
Decatur, Georgia, 30033
Novartis Investigative Site
Indianapolis, Indiana, 46202
Novartis Investigative Site
Media, Pennsylvania, 19063
Novartis Investigative Site
Nashville, Tennessee, 37212
Novartis Investigative Site
Omaha, Nebraska, 68198-5575
Novartis Investigative Site
Phoenix, Arizona, 85006
Novartis Investigative Site
Sacramento, California, 95817
Novartis Investigative Site
Staten Island, New York, 10314
Germany4 sites
Novartis Investigative Site
Berlin, Germany, 12203
Novartis Investigative Site
Mainz, Germany, 55131
Novartis Investigative Site
Tübingen, 72076
Novartis Investigative Site
Würzburg, 97070
Australia3 sites
Novartis Investigative Site
Caulfield, Victoria, 3161
Novartis Investigative Site
Ryde, New South Wales, 2112
Novartis Investigative Site
Waratah, New South Wales, 2298
Canada2 sites
Novartis Investigative Site
Brampton, Ontario, L6Y 1M5
Novartis Investigative Site
Sherbrooke, Quebec, J1H 5N4
France2 sites
Novartis Investigative Site
Bron, 69677
Novartis Investigative Site
Paris, 75013
Spain2 sites
Novartis Investigative Site
Málaga, Andalusia, 29010
Novartis Investigative Site
Sant Cugat del Vallès, Catalonia, 08190
Switzerland2 sites
Novartis Investigative Site
Lausanne, 1011
Novartis Investigative Site
Zurich, 8091
About this study
Official title: An Open-label Study to Evaluate the Long-term Safety, Tolerability and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome
The purpose of this study is to generate long-term safety, tolerability and efficacy data for AFQ056 in eligible adult patients with FXS who have participated in the CAFQ056A2212 (NCT01253629).study and patients who have participated in the previous proof-of-concept study CAFQ056A2204 (NCT00718341).
Read the detailed description
A 148 patients were enrolled into this treatment extension trial conducted for at least 3 years. This extension trial was terminated after the decision to terminate the AFQ056 development program. The decision was based on the results of two randomized, double blind, placebo controlled phase IIb trials in adult and adolescent FXS patients (CAFQ056A2212 and CAFQ056B2214respectivly), both of which failed to demonstrate efficacy in the FXS population. As this extension trial was terminated, only the primary objective and safety are represented.
- Conditions
- Fragile X Syndrome
- Being tested
- AFQ056
- Keywords
- Fragile X Syndrome, Martin-Bell Syndrome, Genetic Diseases, X-Linked, Escalante's syndrome
- Medical subject headings
- Fragile X Syndrome, Genetic Diseases, Inborn
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AFQ056Drug
The investigational drug, AFQ056, will be provided as hard gelatin capsules. Two different oral dosage strengths, identical in appearance, will be used.
| Study group | Role | What they receive |
|---|---|---|
| AFQ056 100 mg (Bid) | Experimental | All patients initiated treatment with AFQ056 at a starting dose of 25 milligram (mg) twice daily. The dose was titrated from 25mg bid to 50mg bid, 75mg bid and 100mg bid at weekly intervals. Dose adjustments (up- and down titrations) were permitted as needed to manage any tolerability issues and to ensure that patients reach their highest tolerated dose not to exceed 100mg bid. Receives: Drug: AFQ056 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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 patients 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 patients 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
Measured: Prior to first dose in extension study, Baseline (start of study treatment in extension study) to End of trial
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 May 2011 (estimated)
- Study startAug 2011
- Primary completionSep 2014
- Study completionSep 2014
Why it stopped: Study treatment AFQ056 failed to demonstrate efficacy in the adult patient with Fragile X Syndrome in 2 other clinical studies (CAFQ056B2214 and CAFQ056A2212)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Fragile X SyndromeStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 148 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01348087
- Sponsor's protocol ID
- CAFQ056B2279
- Other IDs
- 2011-001952-12
- Status last verified
- Apr 2016
- First submitted
- 3 May 2011
- Last updated
- 25 May 2016
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 25 May 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Study to Evaluate the Long-term Safety, Tolerability and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome
- ClinicalTrials.gov ID
- NCT01348087
- Acronym / sponsor ID
- CAFQ056B2279
- Conditions
- Fragile X Syndrome
- Keywords
- Fragile X Syndrome, Martin-Bell Syndrome, Genetic Diseases, X-Linked, Escalante's syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study treatment AFQ056 failed to demonstrate efficacy in the adult patient with Fragile X Syndrome in 2 other clinical studies (CAFQ056B2214 and CAFQ056A2212)
- Enrolment
- 148 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Aug 2011
- Primary completion
- Sep 2014 (actual)
- Study completion
- Sep 2014 (actual)
- First posted
- 5 May 2011 (estimated)
- Results
- Posted 25 May 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to generate long-term safety, tolerability and efficacy data for AFQ056 in eligible adult patients with FXS who have participated in the CAFQ056A2212 (NCT01253629).study and patients who have participated in the previous proof-of-concept study CAFQ056A2204 (NCT00718341).
Detailed description
A 148 patients were enrolled into this treatment extension trial conducted for at least 3 years. This extension trial was terminated after the decision to terminate the AFQ056 development program. The decision was based on the results of two randomized, double blind, placebo controlled phase IIb trials in adult and adolescent FXS patients (CAFQ056A2212 and CAFQ056B2214respectivly), both of which failed to demonstrate efficacy in the FXS population. As this extension trial was terminated, only the primary objective and safety are represented.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| AFQ056 100 mg (Bid) | Experimental | Drug: AFQ056All patients initiated treatment with AFQ056 at a starting dose of 25 milligram (mg) twice daily. The dose was titrated from 25mg bid to 50mg bid, 75mg bid and 100mg bid at weekly intervals. Dose adjustments (up- and down titrations) were permitted as needed to manage any tolerability issues and to ensure that patients reach their highest tolerated dose not to exceed 100mg bid. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Had to 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.
- Females of child-bearing potential had to follow protocol requirements with respect to contraception.
- Have a caregiver or caregivers who spent, on average, at least six hours per day with the patient, who were willing and capable of supervising treatment, providing input into efficacy and safety assessments, and accompanying the patient to study visits.
- Had to have:
- Completed Study CAFQ056A2204.
Key exclusion criteria (6 listed)
- Any advanced, severe or unstable disease
- 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.)
- Using (or used within 6 weeks before baseline) concomitant medications that are potent inhibitors or inducers of CYP3A4
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AFQ056The investigational drug, AFQ056, will be provided as hard gelatin capsules. Two different oral dosage strengths, identical in appearance, will be used. | Drug | AFQ056 100 mg (Bid) |
Primary outcome
Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs).
Time frame: Prior to first dose in extension study, Baseline (start of study treatment in extension study) to End of trial
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 patients 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 patients 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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 May 2016.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
28 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing AFQ056 in people with Fragile X Syndrome, run by Novartis Pharmaceuticals at 28 sites in 10 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 25 May 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 28 sites in United States, Germany, Australia, Canada, France and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 25 May 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs). (time frame: Prior to first dose in extension study, Baseline (start of study treatment in extension study) to End of trial). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 May 2016.