Fragile X SyndromePhase 1NCT01482143
Clinical Study to Assess the Pharmacokinetics, Safety and Tolerability of Single and Multiple Oral Doses of AFQ056 in Children With Fragile X Syndrome (FXS)
A Phase 1 study testing AFQ056 in people with Fragile X Syndrome, run by Novartis Pharmaceuticals at 4 sites in 2 countries.
Trial summary
The aim of this study is to characterize the pharmacokinetics and safety/tolerability of AFQ056 in children with Fragile X Syndrome(FXS) Read more about this study
- Who is it for?
- Ages 3 to 11 years
- Conditions the study is for
- Fragile X Syndrome
- What is being tested?
- AFQ056
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 4 sitesin 2 countries
- Study dates
- Mar 2012to Oct 2013
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 3 to 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Genetically confirmed diagnosis of FXS
- At Screening and first baseline, vital signs, body weight and body mass index (BMI) must be age-specific within normal ranges.
Who cannot join
- Use of any other investigational drug within 30 days or 5 half-lives (whichever is longer) of the investigational drug prior to screening until end of study visit.
- History of hypersensitivity to AFQ056 or any mGluR antagonist.
- Female patients who are confirmed or suspected to be sexually active.
- History or presence of any clinically significant disease of any major system organ class, within the past 2 years prior to screening including but not limited to psychiatric, neurological, cardiovascular, endocrine, metabolic, renal, or gastrointestinal disorders (except for typical features of FXS).
- Smokers.
- Loss of ≥10% of total blood volume within 8 weeks (or less if required for this age group and/or by local regulation) prior to dosing or longer if required for this age group and/or by local regulation.
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- Significant illness that did not completely resolve at least four weeks prior to the first baseline visit.
- Any abnormal laboratory values at screening or first baseline that are in the opinion of the investigator clinically significant and may jeopardize the safety of the study subject.
- Use of (or use within at least 5 half lives before dosing) concomitant medications that are strong/moderate inhibitors or inducers of CYP1A1/2, CYP2C9/19 or CYP3A4
- History or presence of Hepatitis B/C or HIV at screening
Trial locations and contacts
4 sites in 2 countries.
4 sites in 2 countries
United States3 sites
Novartis Investigative Site
Chicago, Illinois, 60612
Novartis Investigative Site
Nashville, Tennessee, 37232-7548
Novartis Investigative Site
Sacramento, California, 95817
About this study
Official title: Sequential, Two-period Study to Assess the Pharmacokinetics, Safety & Tolerability of Single and Multiple Oral Doses of AFQ056 in Patients With FXS (Fragile X Syndrome) Aged 5-11 Years (Cohort 1) and 3-4 Years (Cohort 2)
The aim of this study is to characterize the pharmacokinetics and safety/tolerability of AFQ056 in children with Fragile X Syndrome(FXS)
- Conditions
- Fragile X Syndrome
- Being tested
- AFQ056
- Keywords
- Fragile X Syndrome, pharmacokinetics, safety and tolerability
- Medical subject headings
- Fragile X Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AFQ056Drug
| Study group | Role | What they receive |
|---|---|---|
| All Study subjects | Experimental | Not described Receives: Drug: AFQ056 |
What is being measured
The primary outcome is the main question the study is designed to answer.
The area under the plasma (or serum or blood) concentration-time curve from time zero to infinity [mass x time / volume] (AUCinf)
Measured: Time Frame: Day 1 (period 1): 0.5, 2, 4, 8, 12, 24 hours post-dose; Day 7 (period 2): pre-dose; 0.5, 2, 4, 8 hours post dose
The area under the plasma (or serum or blood) concentration-time curve from time zero to the time of the last quantifiable concentration [mass x time / volume] (AUClast)
Measured: Time Frame: Day 1 (period 1): 0.5, 2, 4, 8, 12, 24 hours post-dose; Day 7 (period 2): pre-dose; 0.5, 2, 4, 8 hours post dose
Maximum observed plasma concentration (Cmax)
Measured: Time Frame: Day 1 (period 1): 0.5, 2, 4, 8, 12, 24 hours post-dose; Day 7 (period 2): pre-dose; 0.5, 2, 4, 8 hours post dose
Secondary outcomes (8)
Physical examination
Measured: Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7
Vital signs and body measurements
Measured: Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7
Electrocardiograms
Measured: Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7
hematology
Measured: Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7
blood chemistry
Measured: Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7
neurological examination
Measured: Screening: once anytime between Day -30 and Day -1; once on Day 7
Adverse events (AE) monitoring
Measured: During the study (total of approximately 32 days) and 3 days after study completion
Serious adverse events (SAE) monitoring
Measured: During the study (total of approximately 32 days) and 30 days after study completion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Nov 2011 (estimated)
- Study startMar 2012
- Primary completionOct 2013
- Study completionOct 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 1
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 21 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
- NCT01482143
- Sponsor's protocol ID
- CAFQ056B2154
- Other IDs
- 2011-004867-65
- Status last verified
- Sep 2014
- First submitted
- 21 Nov 2011
- Last updated
- 8 Dec 2020
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Dec 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Sequential, Two-period Study to Assess the Pharmacokinetics, Safety & Tolerability of Single and Multiple Oral Doses of AFQ056 in Patients With FXS (Fragile X Syndrome) Aged 5-11 Years (Cohort 1) and 3-4 Years (Cohort 2)
- ClinicalTrials.gov ID
- NCT01482143
- Acronym / sponsor ID
- CAFQ056B2154
- Conditions
- Fragile X Syndrome
- Keywords
- Fragile X Syndrome, pharmacokinetics, safety and tolerability
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Mar 2012
- Primary completion
- Oct 2013 (actual)
- Study completion
- Oct 2013 (actual)
- First posted
- 30 Nov 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The aim of this study is to characterize the pharmacokinetics and safety/tolerability of AFQ056 in children with Fragile X Syndrome(FXS)
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| All Study subjects | Experimental | Drug: AFQ056 |
Who may qualify
- Ages
- 3 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- Genetically confirmed diagnosis of FXS
- At Screening and first baseline, vital signs, body weight and body mass index (BMI) must be age-specific within normal ranges.
Key exclusion criteria (10 listed)
- Use of any other investigational drug within 30 days or 5 half-lives (whichever is longer) of the investigational drug prior to screening until end of study visit.
- History of hypersensitivity to AFQ056 or any mGluR antagonist.
- Female patients who are confirmed or suspected to be sexually active.
- History or presence of any clinically significant disease of any major system organ class, within the past 2 years prior to screening including but not limited to psychiatric, neurological, cardiovascular, endocrine, metabolic, renal, or gastrointestinal disorders (except for typical features of FXS).
- Smokers.
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 |
|---|---|---|
| AFQ056 | Drug | All Study subjects |
Primary outcome
The area under the plasma (or serum or blood) concentration-time curve from time zero to infinity [mass x time / volume] (AUCinf)
Time frame: Time Frame: Day 1 (period 1): 0.5, 2, 4, 8, 12, 24 hours post-dose; Day 7 (period 2): pre-dose; 0.5, 2, 4, 8 hours post dose
The area under the plasma (or serum or blood) concentration-time curve from time zero to the time of the last quantifiable concentration [mass x time / volume] (AUClast)
Time frame: Time Frame: Day 1 (period 1): 0.5, 2, 4, 8, 12, 24 hours post-dose; Day 7 (period 2): pre-dose; 0.5, 2, 4, 8 hours post dose
Maximum observed plasma concentration (Cmax)
Time frame: Time Frame: Day 1 (period 1): 0.5, 2, 4, 8, 12, 24 hours post-dose; Day 7 (period 2): pre-dose; 0.5, 2, 4, 8 hours post dose
8 secondary outcomes
Physical examination
Time frame: Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7
Vital signs and body measurements
Time frame: Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7
Electrocardiograms
Time frame: Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7
hematology
Time frame: Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7
blood chemistry
Time frame: Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7
neurological examination
Time frame: Screening: once anytime between Day -30 and Day -1; once on Day 7
Adverse events (AE) monitoring
Time frame: During the study (total of approximately 32 days) and 3 days after study completion
Serious adverse events (SAE) monitoring
Time frame: During the study (total of approximately 32 days) and 30 days after study completion
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Dec 2020.
- Results for CAFQ056B2154 can be found on the Novartis Clinical Trial Results WebsiteLink provided by the sponsor.
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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
4 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing AFQ056 in people with Fragile X Syndrome, run by Novartis Pharmaceuticals at 4 sites in 2 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 2 inclusion and 10 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: finished, as of the sponsor's last update on 8 Dec 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States, Spain. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
The area under the plasma (or serum or blood) concentration-time curve from time zero to infinity [mass x time / volume] (AUCinf) (time frame: Time Frame: Day 1 (period 1): 0.5, 2, 4, 8, 12, 24 hours post-dose; Day 7 (period 2): pre-dose; 0.5, 2, 4, 8 hours post dose). The study lists 3 primary outcomes in all. 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 8 Dec 2020.