Fragile X SyndromePhase 2NCT01517698
A Study of RO4917523 in Patients With Fragile X Syndrome
A Phase 2 study testing RO4917523 0.5 mg and RO4917523 1.5 mg in people with Fragile X Syndrome, run by Hoffmann-La Roche at 51 sites in 10 countries.
Trial summary
This multi-center, randomized, double-blind, placebo-controlled, parallel-group study will investigate the efficacy and safety of RO4917523 in adolescent and adult patients with fragile X syndrome. Patients will be randomized to receive oral doses of 0.5 mg or 1.5 mg of RO4917523, or matching placebo once daily. Read more about this study
- Who is it for?
- Ages 14 to 50 years
- Conditions the study is for
- Fragile X Syndrome
- What is being tested?
- RO4917523 0.5 mg and RO4917523 1.5 mg
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 185 participants
- Where
- 51 sitesin 10 countries
- Study dates
- May 2012to 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 14 to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Adult and adolescent patients, 14-50 years of age
- Diagnosis of fragile X syndrome with a confirmed fragile X mental retardation 1 (FMR1) full mutation and qualifying scores on the Aberrant Behavior Checklist (ABC) and CGI-S
- Patients must agree to either remain completely abstinent or to use two effective contraceptive methods during and 3 weeks after the study
Who cannot join
- Previous treatment with another metabotropic glutamate (mGLU) receptor antagonist within 18 months or with RO4917523
- Participation in a clinical trial involving an investigational (unapproved) drug within 3 months or 5 times the half-life (whichever is longer) before start of this study
- Any uncontrolled, unstable clinically significant psychiatric condition other than fragile X syndrome
- History of suicidal behavior
Trial locations and contacts
51 sites in 10 countries.
51 sites in 10 countries
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
Durham, North Carolina, 27710
Study site
Houston, Texas, 77030
Study site
Iowa City, Iowa, 52242-1083
Study site
Long Beach, California, 90806
Study site
Louisville, Kentucky, 40202
Study site
Media, Pennsylvania, 19063
Study site
Nashville, Tennessee, 37203
Study site
New York, New York, 10029
Study site
Phoenix, Arizona, 85006
Study site
Sacramento, California, 95817
Study site
San Antonio, Texas, 78258
Study site
Seattle, Washington, 98145
Study site
Staten Island, New York, 10314
Study site
Washington D.C., District of Columbia, 20010
Study site
Worcester, Massachusetts, 01655
France7 sites
Study site
Bordeaux, 33076
Study site
Bron, 69003
Study site
Dijon, 21079
Study site
Marseille, 13385
Study site
Montpellier, 34295
Study site
Paris, 75013
Study site
Tours, 37044
Spain6 sites
Study site
Donostia / San Sebastian, Guipuzcoa, 20009
Study site
El Palmar. Murcia, Murcia, 30120
Study site
Madrid, Madrid, 28040
Study site
Madrid, Madrid, 28046
Study site
San Cugat Del Valles, Barcelona, 08195
Study site
Torremolinos, Malaga, 29620
United Kingdom6 sites
Study site
Belfast, BT12 6AB
Study site
Dundee, DD1 9SY
Study site
Edinburgh, EH10 5HF
Study site
Glasgow, GS 8SJ
Study site
London, W1W 7EJ
Study site
Manchester, M13 9WL
Argentina3 sites
Study site
Bahía Blanca, 8000
Study site
Caba, 1425
Study site
Caba, C1126AAB
Canada2 sites
Study site
Brampton, Ontario, L6Y 1M5
Study site
Sherbrooke, Quebec, J1H 5N4
About this study
Official title: A Randomized, Double-blind, 12-week, Parallel Group, Placebo-controlled Study of Efficacy and Safety of RO4917523 in Patients With Fragile X Syndrome.
This multi-center, randomized, double-blind, placebo-controlled, parallel-group study will investigate the efficacy and safety of RO4917523 in adolescent and adult patients with fragile X syndrome. Patients will be randomized to receive oral doses of 0.5 mg or 1.5 mg of RO4917523, or matching placebo once daily. The anticipated time on study treatment is 12 weeks.
- Conditions
- Fragile X Syndrome
- Being tested
- Placebo, RO4917523 0.5 mg, RO4917523 1.5 mg
- 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
placebo to RO4917523 orally once a day for 12 weeks
RO4917523 0.5 mgDrug
0.5 mg orally once a day for 12 weeks
RO4917523 1.5 mgDrug
1.5 mg orally once a day for 12 weeks
| Study group | Role | What they receive |
|---|---|---|
| RO4917523 0.5 mg | Experimental | Not described Receives: Drug: RO4917523 0.5 mg |
| RO4917523 1.5 mg | Experimental | Not described Receives: Drug: RO4917523 1.5 mg |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Anxiety Depression and Mood Scale (ADAMS) total score
Measured: 12 weeks
Safety (incidence of adverse events)
Measured: 12 weeks
Secondary outcomes (6)
Change in Social Responsiveness Scale (SRS)
Measured: 12 weeks
Change in Clinical Global Impressions Scale - Improvement (CGI-I)
Measured: 12 weeks
Change in Clinical Global Impressions Scale - Severity of Illness (CGI-S)
Measured: 12 weeks
Change in Aberrant Behavior Checklist total score
Measured: 12 weeks
Change in Aberrant Behavior Checklist factor scores
Measured: 12 weeks
Change in Anxiety Depression and Mood Scale (ADAMS) factor scores
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jan 2012 (estimated)
- Study startMay 2012
- 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
- 185 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
- NCT01517698
- Sponsor's protocol ID
- NP27936
- Status last verified
- Jul 2016
- First submitted
- 20 Jan 2012
- Last updated
- 11 Jul 2016
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Jul 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, 12-week, Parallel Group, Placebo-controlled Study of Efficacy and Safety of RO4917523 in Patients With Fragile X Syndrome.
- ClinicalTrials.gov ID
- NCT01517698
- Acronym / sponsor ID
- NP27936
- Conditions
- Fragile X Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 185 participants (actual)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Study start
- May 2012
- Primary completion
- Apr 2014 (actual)
- Study completion
- Apr 2014 (actual)
- First posted
- 25 Jan 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 multi-center, randomized, double-blind, placebo-controlled, parallel-group study will investigate the efficacy and safety of RO4917523 in adolescent and adult patients with fragile X syndrome. Patients will be randomized to receive oral doses of 0.5 mg or 1.5 mg of RO4917523, or matching placebo once daily. The anticipated time on study treatment is 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 |
|---|---|---|
| RO4917523 0.5 mg | Experimental | Drug: RO4917523 0.5 mg |
| RO4917523 1.5 mg | Experimental | Drug: RO4917523 1.5 mg |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 14 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Adult and adolescent patients, 14-50 years of age
- Diagnosis of fragile X syndrome with a confirmed fragile X mental retardation 1 (FMR1) full mutation and qualifying scores on the Aberrant Behavior Checklist (ABC) and CGI-S
- Patients must agree to either remain completely abstinent or to use two effective contraceptive methods during and 3 weeks after the study
Key exclusion criteria (4 listed)
- Previous treatment with another metabotropic glutamate (mGLU) receptor antagonist within 18 months or with RO4917523
- Participation in a clinical trial involving an investigational (unapproved) drug within 3 months or 5 times the half-life (whichever is longer) before start of this study
- Any uncontrolled, unstable clinically significant psychiatric condition other than fragile X syndrome
- History of suicidal behavior
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 |
|---|---|---|
| Placeboplacebo to RO4917523 orally once a day for 12 weeks | Drug | Placebo |
| RO4917523 0.5 mg0.5 mg orally once a day for 12 weeks | Drug | RO4917523 0.5 mg |
| RO4917523 1.5 mg1.5 mg orally once a day for 12 weeks | Drug | RO4917523 1.5 mg |
Primary outcome
Change in Anxiety Depression and Mood Scale (ADAMS) total score
Time frame: 12 weeks
Safety (incidence of adverse events)
Time frame: 12 weeks
6 secondary outcomes
Change in Social Responsiveness Scale (SRS)
Time frame: 12 weeks
Change in Clinical Global Impressions Scale - Improvement (CGI-I)
Time frame: 12 weeks
Change in Clinical Global Impressions Scale - Severity of Illness (CGI-S)
Time frame: 12 weeks
Change in Aberrant Behavior Checklist total score
Time frame: 12 weeks
Change in Aberrant Behavior Checklist factor scores
Time frame: 12 weeks
Change in Anxiety Depression and Mood Scale (ADAMS) factor scores
Time frame: 12 weeks
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 Jul 2016.
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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.
Clinical Trials study director
Hoffmann-La Roche
51 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 RO4917523 0.5 mg and RO4917523 1.5 mg in people with Fragile X Syndrome, run by Hoffmann-La Roche at 51 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 3 inclusion and 4 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 Jul 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 51 sites in United States, France, Spain, United Kingdom, Argentina 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. 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?
Change in Anxiety Depression and Mood Scale (ADAMS) total score (time frame: 12 weeks). The study lists 2 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 11 Jul 2016.