Down SyndromePhase 2NCT02484703
A Study of RO5186582 in Down Syndrome Among Children 6 to 11 Years of Age
A Phase 2 study testing RO5186582 in people with Down Syndrome, run by Hoffmann-La Roche at 9 sites in United States.
Trial summary
This study will evaluate the safety, tolerability, efficacy, and pharmacokinetic and pharmacodynamic activity of 3 different dosages of RO5186582 compared with placebo. A total of approximately 46 participants will be enrolled, in order to have at least 32 evaluable, and will be randomly assigned to 1 of 4 treatments… Read more about this study
- Who is it for?
- Ages 6 to 11 years
- Conditions the study is for
- Down Syndrome
- What is being tested?
- RO5186582
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 45 participants
- Where
- 9 sitesUnited States
- Study dates
- 28 Oct 2015to 3 Aug 2016
- 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 6 to 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Diagnosis of Down syndrome, except for mosaic Down syndrome
- Available parent or caregiver to attend clinic visits and provide information about the participant's behavior and symptoms
Who cannot join
- Any primary psychiatric comorbid disorder
- History of infantile spasms, West syndrome, Lennox-Gastaut syndrome, early infantile epileptic encephalopathy, treatment-refractory epilepsy with cognitive/developmental regression, severe head trauma, or central nervous system (CNS) infection
- Seizure event of any type within 12 months prior to Screening or relevant changes in anti-epileptic drugs 6 weeks prior to enrollment
- Significant sleep disruption
- Significant gastrointestinal, renal, hepatic, endocrine, or cardiovascular disease
- New-onset or ongoing hematologic/oncologic disorder
- Severe lactose intolerance
- Participation in another clinical study within 1 month or 6 half-lives prior to first dose, or any extent of participation in Study BP29589 (NCT02451657)
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
United States9 sites
Massachusette General Hospital; Medical Genetics
Boston, Massachusetts, 02114
Boston Children's Hospital, Department of Neurology
Boston, Massachusetts, 02115
Rush University Medical Center
Chicago, Illinois, 60612
Emory University School of Medicine; Department of Human Genetics & Pediatrics
Decatur, Georgia, 30033
Duke Clin Rsch Institute
Durham, North Carolina, 27710
University of Wisconsin Hospital and Clinics
Madison, Wisconsin, 53792
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Southwest Autism Research & Resource Center
Phoenix, Arizona, 85006
UPMC Western Psychiatric Institute and Clinic
Pittsburgh, Pennsylvania, 15203 1101
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled, Parallel Group 26-Week Dose-Investigating Study to Explore the Pharmacokinetics, Pharmacodynamic Effects, Efficacy, Safety and Tolerability of RO5186582 in Children With Down Syndrome Aged 6-11 Years
This study will evaluate the safety, tolerability, efficacy, and pharmacokinetic and pharmacodynamic activity of 3 different dosages of RO5186582 compared with placebo. A total of approximately 46 participants will be enrolled, in order to have at least 32 evaluable, and will be randomly assigned to 1 of 4 treatments in a 1:1:1:1 ratio, with 9 children per treatment arm. The target ratio between 6-8 years and 9-11 years age groups is approximately 1:1 in each treatment arm, with a minimum of 3 children per age group in each treatment arm.
- Conditions
- Down Syndrome
- Being tested
- Placebo, RO5186582
- Medical subject headings
- Down Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlaceboDrug
Participants will receive matching placebo PO BID. Study medication will first be administered on Day 1, and only the morning dose will be given on the last day of treatment (Week 26).
RO5186582Drug
Participants will receive 1 of 3 dosages of RO5186582 PO BID, including 40 mg, 60 mg, or 120 mg. Study medication will first be administered on Day 1, and only the morning dose will be given on the last day of treatment (Week 26).
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Participants will receive matching placebo by mouth (PO) twice daily (BID) for up to 26 weeks. Receives: Drug: Placebo |
| RO5186582 120 mg BID | Experimental | Participants will receive RO5186582 at a dosage of 120 milligrams (mg) PO BID for up to 26 weeks. Receives: Drug: RO5186582 |
| RO5186582 40 mg BID | Experimental | Participants will receive RO5186582 at a dosage of 40 mg PO BID for up to 26 weeks. Receives: Drug: RO5186582 |
| RO5186582 60 mg BID | Experimental | Participants will receive RO5186582 at a dosage of 60 mg PO BID for up to 26 weeks. Receives: Drug: RO5186582 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants With Adverse Events (AEs)
Measured: Baseline up to Week 6
Percentage of Participants With Epileptiform Abnormalities as Assessed Using Electroencephalogram (EEG) Analysis
Measured: Baseline up to Week 6
Percentage of Participants by Suicidality Classification as Assessed Using an Adapted Form of the Columbia Classification Algorithm for Suicide Assessment (C-CASA)
Measured: Baseline up to Week 6
Anxiety, Depression, and Mood Scale (ADAMS) Score
Measured: Baseline up to Week 6
Hyperactivity and Impulsivity as Assessed by the Short Version of Conners Third Edition Parent Short-Form (Conners-3) Score
Measured: Baseline up to Week 6
Sleep Disturbances as Assessed by the Children's Sleep Habits Questionnaire (CSHQ) Score
Measured: Baseline up to Week 6
Gamma Power at Posterior Electrodes as Assessed Using EEG Analysis
Measured: Baseline up to Week 6
Theta Power at Posterior Electrodes as Assessed Using EEG Analysis
Measured: Baseline up to Week 6
Cognition as Assessed by the Children's Memory Scale (CMS) Subtests Score
Measured: Baseline up to Week 6
Secondary outcomes (14)
Percentage of Participants With AEs
Measured: Baseline up to Week 26
Percentage of Participants With Epileptiform Abnormalities as Assessed Using EEG Analysis
Measured: Baseline up to Week 26
Percentage of Participants by Suicidality Classification as Assessed Using an Adapted Form of the C-CASA
Measured: Baseline up to Week 26
ADAMS Score
Measured: Baseline up to Week 26
Hyperactivity and Impulsivity as Assessed by the Short Version of Conners-3 Score
Measured: Baseline up to Week 26
Sleep Disturbances as Assessed by the CSHQ Score
Measured: Baseline up to Week 26
Gamma Power at Posterior Electrodes as Assessed Using EEG Analysis
Measured: Baseline up to Week 26
Theta Power at Posterior Electrodes as Assessed Using EEG Analysis
Measured: Baseline up to Week 26
Cognition as Assessed by the CMS Subtests Score
Measured: Baseline up to Week 26
Adaptive Behavior as Assessed by the Vineland Adaptive Behavior Scales-II (VABS-II) Score
Measured: Baseline up to Week 26
Clinical Global Impression-Improvement (CGI-I) Scale Score
Measured: Baseline up to Week 26
Daily Functional Memory as Assessed by the Observer Memory Questionnaire-Parent Form (OMQ-PF) Score
Measured: Baseline up to Week 26
Intellectual Quotient (IQ) as Assessed by the Leiter 3
Measured: Baseline up to Week 26
Plasma Concentration of RO5186582
Measured: Predose (2 predose samples separated by at least 1 hour) at Weeks 2 and 6; and predose or postdose (as convenient) during Weeks 10, 17, and 26
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jun 2015 (estimated)
- Study start28 Oct 2015
- Primary completion3 Aug 2016
- Study completion3 Aug 2016
Why it stopped: Study was terminated due to data from another study showing no efficacy. There were no safety issues of concern.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Down 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
- 45 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
- NCT02484703
- Sponsor's protocol ID
- WP28760
- Status last verified
- Jul 2017
- First submitted
- 16 Jun 2015
- Last updated
- 6 Jul 2017
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled, Parallel Group 26-Week Dose-Investigating Study to Explore the Pharmacokinetics, Pharmacodynamic Effects, Efficacy, Safety and Tolerability of RO5186582 in Children With Down Syndrome Aged 6-11 Years
- ClinicalTrials.gov ID
- NCT02484703
- Acronym / sponsor ID
- WP28760
- Conditions
- Down Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study was terminated due to data from another study showing no efficacy. There were no safety issues of concern.
- Enrolment
- 45 participants (actual)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Study start
- 28 Oct 2015 (actual)
- Primary completion
- 3 Aug 2016 (actual)
- Study completion
- 3 Aug 2016 (actual)
- First posted
- 30 Jun 2015 (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 study will evaluate the safety, tolerability, efficacy, and pharmacokinetic and pharmacodynamic activity of 3 different dosages of RO5186582 compared with placebo. A total of approximately 46 participants will be enrolled, in order to have at least 32 evaluable, and will be randomly assigned to 1 of 4 treatments in a 1:1:1:1 ratio, with 9 children per treatment arm. The target ratio between 6-8 years and 9-11 years age groups is approximately 1:1 in each treatment arm, with a minimum of 3 children per age group in each treatment arm.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: PlaceboParticipants will receive matching placebo by mouth (PO) twice daily (BID) for up to 26 weeks. |
| RO5186582 120 mg BID | Experimental | Drug: RO5186582Participants will receive RO5186582 at a dosage of 120 milligrams (mg) PO BID for up to 26 weeks. |
| RO5186582 40 mg BID | Experimental | Drug: RO5186582Participants will receive RO5186582 at a dosage of 40 mg PO BID for up to 26 weeks. |
| RO5186582 60 mg BID | Experimental | Drug: RO5186582Participants will receive RO5186582 at a dosage of 60 mg PO BID for up to 26 weeks. |
Who may qualify
- Ages
- 6 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- Diagnosis of Down syndrome, except for mosaic Down syndrome
- Available parent or caregiver to attend clinic visits and provide information about the participant's behavior and symptoms
Key exclusion criteria (8 listed)
- Any primary psychiatric comorbid disorder
- History of infantile spasms, West syndrome, Lennox-Gastaut syndrome, early infantile epileptic encephalopathy, treatment-refractory epilepsy with cognitive/developmental regression, severe head trauma, or central nervous system (CNS) infection
- Seizure event of any type within 12 months prior to Screening or relevant changes in anti-epileptic drugs 6 weeks prior to enrollment
- Significant sleep disruption
- Significant gastrointestinal, renal, hepatic, endocrine, or cardiovascular disease
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 |
|---|---|---|
| PlaceboParticipants will receive matching placebo PO BID. Study medication will first be administered on Day 1, and only the morning dose will be given on the last day of treatment (Week 26). | Drug | Placebo |
| RO5186582Participants will receive 1 of 3 dosages of RO5186582 PO BID, including 40 mg, 60 mg, or 120 mg. Study medication will first be administered on Day 1, and only the morning dose will be given on the last day of treatment (Week 26). | Drug | RO5186582 120 mg BID, RO5186582 40 mg BID, RO5186582 60 mg BID |
Primary outcome
Percentage of Participants With Adverse Events (AEs)
Time frame: Baseline up to Week 6
Percentage of Participants With Epileptiform Abnormalities as Assessed Using Electroencephalogram (EEG) Analysis
Time frame: Baseline up to Week 6
Percentage of Participants by Suicidality Classification as Assessed Using an Adapted Form of the Columbia Classification Algorithm for Suicide Assessment (C-CASA)
Time frame: Baseline up to Week 6
Anxiety, Depression, and Mood Scale (ADAMS) Score
Time frame: Baseline up to Week 6
Hyperactivity and Impulsivity as Assessed by the Short Version of Conners Third Edition Parent Short-Form (Conners-3) Score
Time frame: Baseline up to Week 6
Sleep Disturbances as Assessed by the Children's Sleep Habits Questionnaire (CSHQ) Score
Time frame: Baseline up to Week 6
Gamma Power at Posterior Electrodes as Assessed Using EEG Analysis
Time frame: Baseline up to Week 6
Theta Power at Posterior Electrodes as Assessed Using EEG Analysis
Time frame: Baseline up to Week 6
Cognition as Assessed by the Children's Memory Scale (CMS) Subtests Score
Time frame: Baseline up to Week 6
14 secondary outcomes
Percentage of Participants With AEs
Time frame: Baseline up to Week 26
Percentage of Participants With Epileptiform Abnormalities as Assessed Using EEG Analysis
Time frame: Baseline up to Week 26
Percentage of Participants by Suicidality Classification as Assessed Using an Adapted Form of the C-CASA
Time frame: Baseline up to Week 26
ADAMS Score
Time frame: Baseline up to Week 26
Hyperactivity and Impulsivity as Assessed by the Short Version of Conners-3 Score
Time frame: Baseline up to Week 26
Sleep Disturbances as Assessed by the CSHQ Score
Time frame: Baseline up to Week 26
Gamma Power at Posterior Electrodes as Assessed Using EEG Analysis
Time frame: Baseline up to Week 26
Theta Power at Posterior Electrodes as Assessed Using EEG Analysis
Time frame: Baseline up to Week 26
Cognition as Assessed by the CMS Subtests Score
Time frame: Baseline up to Week 26
Adaptive Behavior as Assessed by the Vineland Adaptive Behavior Scales-II (VABS-II) Score
Time frame: Baseline up to Week 26
Clinical Global Impression-Improvement (CGI-I) Scale Score
Time frame: Baseline up to Week 26
Daily Functional Memory as Assessed by the Observer Memory Questionnaire-Parent Form (OMQ-PF) Score
Time frame: Baseline up to Week 26
Intellectual Quotient (IQ) as Assessed by the Leiter 3
Time frame: Baseline up to Week 26
Plasma Concentration of RO5186582
Time frame: Predose (2 predose samples separated by at least 1 hour) at Weeks 2 and 6; and predose or postdose (as convenient) during Weeks 10, 17, and 26
Results and safety
The study was stopped early (reason given: Study was terminated due to data from another study showing no efficacy. There were no safety issues of concern.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jul 2017.
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Down Syndrome
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
9 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 RO5186582 in people with Down Syndrome, run by Hoffmann-La Roche at 9 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 8 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 6 Jul 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 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 was stopped early (reason given: Study was terminated due to data from another study showing no efficacy. There were no safety issues of concern.). Results are not available in the source record.
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?
Percentage of Participants With Adverse Events (AEs) (time frame: Baseline up to Week 6). The study lists 9 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 6 Jul 2017.