Fragile X SyndromePhase 2NCT01015430
A Study With RO4917523 in Patients With Fragile X Syndrome
A Phase 2 study testing RO4917523 in people with Fragile X Syndrome, run by Hoffmann-La Roche at 5 sites in United States.
Trial summary
This randomized, double-blind multiple ascending dose study will evaluate the safety and tolerability, pharmacokinetics and efficacy of RO4917523 in patients with Fragile X Syndrome. The patients will be randomized to receive either active drug or placebo. The anticipated time on study treatment is 6 weeks. Read more about this study
- Who is it for?
- Ages 18 to 50 years
- Conditions the study is for
- Fragile X Syndrome
- What is being tested?
- RO4917523
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 5 sitesUnited States
- Study dates
- Nov 2009to Jan 2011
- 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 18 to 50 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Adult patients, 18 to 50 years of age
- Fragile X Syndrome
- IQ less than 75
- Reliable caregiver
Who cannot join
- Current psychosis or presumption of psychosis
- History of suicidal behavior or considered a high suicidal risk
- Severe self-injurious behavior
- Any unstable medical condition other than Fragile X Syndrome (e.g. hypertension, diabetes)
- Current seizure disorder
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Study site
Chicago, Illinois, 60612
Study site
Decatur, Georgia, 30033
Study site
Indianapolis, Indiana, 46202
Study site
Nashville, Tennessee, 37203
Study site
Sacramento, California, 95817
About this study
Official title: A Randomized, Double-blind, Placebo-controlled, Pharmacokinetic, Safety and Tolerability, and Exploratory Efficacy and Pharmacodynamic Effects Study of RO4917523 in Adult Patients With Fragile X Syndrome.
This randomized, double-blind multiple ascending dose study will evaluate the safety and tolerability, pharmacokinetics and efficacy of RO4917523 in patients with Fragile X Syndrome. The patients will be randomized to receive either active drug or placebo. The anticipated time on study treatment is 6 weeks. The target sample size is <100 patients.
- Conditions
- Fragile X Syndrome
- Being tested
- Placebo (for RO4917523 ascending doses), Placebo (for RO4917523 fixed dose), 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.
Placebo (for RO4917523 ascending doses)Drug
Placebo daily po
Placebo (for RO4917523 fixed dose)Drug
Placebo daily po
RO4917523Drug
RO4917523 ascending doses daily po
RO4917523Drug
RO4917523 fixed dose daily po
| Study group | Role | What they receive |
|---|---|---|
| Placebo (for RO4917523 ascending doses) | Placebo comparator | Not described Receives: Drug: Placebo (for RO4917523 ascending doses); Drug: Placebo (for RO4917523 fixed dose) |
| Placebo (for RO4917523 fixed dose) | Placebo comparator | Not described Receives: Drug: Placebo (for RO4917523 ascending doses); Drug: Placebo (for RO4917523 fixed dose) |
| RO4917523 ascending doses | Experimental | Not described Receives: Drug: RO4917523 |
| RO4917523 fixed dose | Experimental | Not described Receives: Drug: RO4917523 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety, Tolerability and Pharmacokinetics: AEs, laboratory parameters
Measured: AEs throughout study; laboratory assessments: days 1, 8, 15, 29, 43
Secondary outcomes (1)
Efficacy: Behavior and cognition assessments
Measured: Every 2 weeks throughout study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2009 (estimated)
- Study startNov 2009
- Primary completionJan 2011
- Study completionJan 2011
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
- 40 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
- NCT01015430
- Sponsor's protocol ID
- NP22578
- Status last verified
- Nov 2016
- First submitted
- 17 Nov 2009
- Last updated
- 2 Nov 2016
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Nov 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Placebo-controlled, Pharmacokinetic, Safety and Tolerability, and Exploratory Efficacy and Pharmacodynamic Effects Study of RO4917523 in Adult Patients With Fragile X Syndrome.
- ClinicalTrials.gov ID
- NCT01015430
- Acronym / sponsor ID
- NP22578
- Conditions
- Fragile X Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Study start
- Nov 2009
- Primary completion
- Jan 2011 (actual)
- Study completion
- Jan 2011 (actual)
- First posted
- 18 Nov 2009 (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 multiple ascending dose study will evaluate the safety and tolerability, pharmacokinetics and efficacy of RO4917523 in patients with Fragile X Syndrome. The patients will be randomized to receive either active drug or placebo. The anticipated time on study treatment is 6 weeks. The target sample size is <100 patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Placebo (for RO4917523 ascending doses) | Placebo comparator | Drug: Placebo (for RO4917523 ascending doses), Drug: Placebo (for RO4917523 fixed dose) |
| Placebo (for RO4917523 fixed dose) | Placebo comparator | Drug: Placebo (for RO4917523 ascending doses), Drug: Placebo (for RO4917523 fixed dose) |
| RO4917523 ascending doses | Experimental | Drug: RO4917523 |
| RO4917523 fixed dose | Experimental | Drug: RO4917523 |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (4 listed)
- Adult patients, 18 to 50 years of age
- Fragile X Syndrome
- IQ less than 75
- Reliable caregiver
Key exclusion criteria (5 listed)
- Current psychosis or presumption of psychosis
- History of suicidal behavior or considered a high suicidal risk
- Severe self-injurious behavior
- Any unstable medical condition other than Fragile X Syndrome (e.g. hypertension, diabetes)
- Current seizure disorder
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 |
|---|---|---|
| Placebo (for RO4917523 ascending doses)Placebo daily po | Drug | Placebo (for RO4917523 ascending doses), Placebo (for RO4917523 fixed dose) |
| Placebo (for RO4917523 fixed dose)Placebo daily po | Drug | Placebo (for RO4917523 ascending doses), Placebo (for RO4917523 fixed dose) |
| RO4917523RO4917523 ascending doses daily po | Drug | RO4917523 ascending doses |
| RO4917523RO4917523 fixed dose daily po | Drug | RO4917523 fixed dose |
Primary outcome
Safety, Tolerability and Pharmacokinetics: AEs, laboratory parameters
Time frame: AEs throughout study; laboratory assessments: days 1, 8, 15, 29, 43
1 secondary outcome
Efficacy: Behavior and cognition assessments
Time frame: Every 2 weeks throughout study
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 2 Nov 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
5 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 5 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 4 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 2 Nov 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 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, Tolerability and Pharmacokinetics: AEs, laboratory parameters (time frame: AEs throughout study; laboratory assessments: days 1, 8, 15, 29, 43). 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 2 Nov 2016.