Fragile X SyndromePhase 2NCT01750957
A Study of RO4917523 in Pediatric Patients With Fragile X Syndrome
A Phase 2 study testing RO4917523 in people with Fragile X Syndrome, run by Hoffmann-La Roche at 20 sites in United States.
Trial summary
This randomized, double-blind, placebo-controlled, parallel-arm study will evaluate the safety and exploratory efficacy and pharmacokinetics of RO4917523 in pediatric patients with fragile X syndrome. Patients will be randomized to receive one of 2 dose levels of RO4917523 or placebo orally daily for 12 weeks. Read more about this study
- Who is it for?
- Ages 5 to 13 years
- Conditions the study is for
- Fragile X Syndrome
- What is being tested?
- RO4917523
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 47 participants
- Where
- 20 sitesUnited States
- Study dates
- Feb 2013to Apr 2014
- Phase
- Phase 2
- 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 5 to 13 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Children and adolescents, 5 to 13 years of age
- Diagnosis of fragile X syndrome based on prior DNA testing confirming Fragile X Mental Retardation 1 (FMR1) full mutation and qualifying scores on the ABC and CGI-S
Who cannot join
- Previous treatment with another mGlu5 receptor antagonist within the prior 3 months
- Participation in a clinical trial involving an investigational drug (unapproved) or non-drug treatment within the prior 6 weeks or 5 times the half-life (whichever is longer) before the start of this study
- Any uncontrolled, unstable clinically significant psychiatric condition other than fragile X syndrome
- History of suicidal behavior
- Other protocol defined inclusion/exclusion criteria may apply
Trial locations and contacts
20 sites in 1 country.
20 sites in 1 country
United States20 sites
Study site
Aurora, Colorado, 80045
Study site
Baltimore, Maryland, 21205
Study site
Chicago, Illinois, 60612
Study site
Cincinnati, Ohio, 45229
Study site
Decatur, Georgia, 30033
Study site
Greenwood, South Carolina, 29646
Study site
Houston, Texas, 77030
Study site
Iowa City, Iowa, 52242
Study site
Long Beach, California, 90806
Study site
Louisville, Kentucky, 40202
Study site
Media, Pennsylvania, 19063
Study site
Nashville, Tennessee, 37212
Study site
New York, New York, 10029
Study site
Omaha, Nebraska, 68198-4160
Study site
Sacramento, California, 95817
Study site
San Antonio, Texas, 78258
Study site
San Diego, California, 92108
Study site
Seattle, Washington, 98145
Study site
Washington D.C., District of Columbia, 20020
Study site
Worcester, Massachusetts, 01605
About this study
Official title: A Randomized, Parallel Group, Double-Blind, Placebo-Controlled, Safety and Exploratory Efficacy and Pharmacokinetic, Study of RO4917523 in Pediatric Patients With Fragile X Syndrome
This randomized, double-blind, placebo-controlled, parallel-arm study will evaluate the safety and exploratory efficacy and pharmacokinetics of RO4917523 in pediatric patients with fragile X syndrome. Patients will be randomized to receive one of 2 dose levels of RO4917523 or placebo orally daily for 12 weeks.
- Conditions
- Fragile X Syndrome
- Being tested
- Placebo, RO4917523
- 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.
PlaceboDrug
orally daily, 12 weeks
RO4917523Drug
Dose A orally daily, 12 weeks
RO4917523Drug
Dose B orally daily, 12 weeks
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
| RO4917523 Dose A | Experimental | Not described Receives: Drug: RO4917523 |
| RO4917523 Dose B | Experimental | Not described Receives: Drug: RO4917523 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety: Incidence of adverse events
Measured: 15 weeks
Secondary outcomes (3)
Efficacy: Neuropsychological/behavioral assessment scales (ADAMS/Clinical Global Impressions CGI-S, CGI-I/ GBAS/Aberrant Behavior Checklist ABC/Repeatable Battery for the Assessment of Neuropsychological Status RBANS/VAS behavior)
Measured: 15 weeks
Pharmacokinetics: Clearance (CL/F)
Measured: up to Week 12
Pharmacokinetics: Volume of distribution at steady-state (Vss/F)
Measured: up to Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Dec 2012 (estimated)
- Study startFeb 2013
- Primary completionApr 2014
- Study completionApr 2014
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 Hoffmann-La RocheThe 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 47 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01750957
- Sponsor's protocol ID
- NP28571
- Other IDs
- 2011-004349-42
- Status last verified
- Aug 2016
- First submitted
- 11 Dec 2012
- Last updated
- 11 Aug 2016
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Aug 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Parallel Group, Double-Blind, Placebo-Controlled, Safety and Exploratory Efficacy and Pharmacokinetic, Study of RO4917523 in Pediatric Patients With Fragile X Syndrome
- ClinicalTrials.gov ID
- NCT01750957
- Acronym / sponsor ID
- NP28571
- Conditions
- Fragile X Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 47 participants (actual)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Study start
- Feb 2013
- Primary completion
- Apr 2014 (actual)
- Study completion
- Apr 2014 (actual)
- First posted
- 17 Dec 2012 (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.
This randomized, double-blind, placebo-controlled, parallel-arm study will evaluate the safety and exploratory efficacy and pharmacokinetics of RO4917523 in pediatric patients with fragile X syndrome. Patients will be randomized to receive one of 2 dose levels of RO4917523 or placebo orally daily for 12 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: Placebo |
| RO4917523 Dose A | Experimental | Drug: RO4917523 |
| RO4917523 Dose B | Experimental | Drug: RO4917523 |
Who may qualify
- Ages
- 5 to 13 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- Children and adolescents, 5 to 13 years of age
- Diagnosis of fragile X syndrome based on prior DNA testing confirming Fragile X Mental Retardation 1 (FMR1) full mutation and qualifying scores on the ABC and CGI-S
Key exclusion criteria (5 listed)
- Previous treatment with another mGlu5 receptor antagonist within the prior 3 months
- Participation in a clinical trial involving an investigational drug (unapproved) or non-drug treatment within the prior 6 weeks or 5 times the half-life (whichever is longer) before the start of this study
- Any uncontrolled, unstable clinically significant psychiatric condition other than fragile X syndrome
- History of suicidal behavior
- Other protocol defined inclusion/exclusion criteria may apply
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 |
|---|---|---|
| Placeboorally daily, 12 weeks | Drug | Placebo |
| RO4917523Dose A orally daily, 12 weeks | Drug | RO4917523 Dose A |
| RO4917523Dose B orally daily, 12 weeks | Drug | RO4917523 Dose B |
Primary outcome
Safety: Incidence of adverse events
Time frame: 15 weeks
3 secondary outcomes
Efficacy: Neuropsychological/behavioral assessment scales (ADAMS/Clinical Global Impressions CGI-S, CGI-I/ GBAS/Aberrant Behavior Checklist ABC/Repeatable Battery for the Assessment of Neuropsychological Status RBANS/VAS behavior)
Time frame: 15 weeks
Pharmacokinetics: Clearance (CL/F)
Time frame: up to Week 12
Pharmacokinetics: Volume of distribution at steady-state (Vss/F)
Time frame: up to Week 12
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 11 Aug 2016.
Related trials for Fragile X Syndrome
Not yet recruitingPhase 2
Inhibition of Aggressive Behavior in Participants With Fragile X Syndrome
Aggressive Behavior in Fragile X Syndome
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Not yet recruiting
Single Session Support for Parents of Children With Neurodevelopmental Disabilities Struggling With Anxious Avoidance
Neurodevelopmental Disorders, Autism Spectrum Disorder (ASD)
RecruitingObservational study
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease
Not yet recruitingPhase 2/3
A Study to Investigate the Effects and Safety of Codabakalner for the Treatment of FXS in Male Participants and Adult Participants With ADS
Fragile X Syndrome (FXS), Autism Spectrum Disorder (ASD)
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.
Clinical Trials study director
Hoffmann-La Roche
20 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing RO4917523 in people with Fragile X Syndrome, run by Hoffmann-La Roche at 20 sites in United States.
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 5 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 11 Aug 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in United States. 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. Some participants in this study may receive a placebo. 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?
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?
Safety: Incidence of adverse events (time frame: 15 weeks). 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 11 Aug 2016.