Down SyndromePhase 1NCT01436955
A Study of RG1662 in Individuals With Down Syndrome
A Phase 1 study testing RG1662 in people with Down Syndrome, run by Hoffmann-La Roche at 9 sites in 2 countries.
Trial summary
This multi-center, randomized double-blind, placebo-controlled study will assess the safety and tolerability of RG1662 in individuals with Down Syndrome. Eligible subjects will be randomized in cohorts to receive either multiple oral doses of RG1662 or placebo. Anticipated time on study treatment is 38 days. Read more about this study
- Who is it for?
- Ages 18 to 30 years
- Conditions the study is for
- Down Syndrome
- What is being tested?
- RG1662
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 35 participants
- Where
- 9 sitesin 2 countries
- Study dates
- Nov 2011to Sep 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 18 to 30 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Adult individuals, 18 to 30 years of age, with diagnosis of Down Syndrome (confirmed by karyotype)
- Males and non-pregnant non-lactating females
- Parent or legal guardian/representative and caregiver willing to give written informed consent
- Subject willing and assenting or consenting to participate
Who cannot join
- Major depressive disorder not adequately controlled by a selective serotonin reuptake inhibitor or a serotonin norepinephrine reuptake inhibitor
- Subjects meeting clinical diagnostic criteria for autistic spectrum disorder, attention deficit or hyperactivity disorder unlikely to cooperate and take part successfully in the study assessments
- Subjects with other primary psychiatric diagnosis
- Subjects with evidence or meeting clinical diagnosis of dementia
- Subjects with personal history of cardiac abnormalities; patients that have previously undergone congenital heart surgery can be enrolled provided they have no residual complication requiring intervention (e.g. pace maker)
- Subjects with clinically significant obstructive pulmonary disease or asthma that is not adequately treated (oral steroids are not allowed)
Show all 10Show fewer
- Subjects with thyroid dysfunction that is not adequately controlled and stabilized on treatment for at least 8 weeks
- Subjects with sustained elevated supine blood pressure (SBP/DBP) at rest exceeding 140/90 mmHg or notable resting tachycardia (mean HR > 60 bpm) or blood pressure below 90/40 mmHg
- Subjects who have taken any other investigational medications within 3 months
- Body mass index (BMI) > 40 kg/m2
Trial locations and contacts
9 sites in 2 countries.
9 sites in 2 countries
United States8 sites
Study site
Aurora, Colorado, 80045
Study site
Baltimore, Maryland, 21287
Study site
Chicago, Illinois, 60612
Study site
Decatur, Georgia, 30033
Study site
Durham, North Carolina, 27710
Study site
La Jolla, California, 92037
Study site
Little Rock, Arkansas, 72204
Study site
Phoenix, Arizona, 85013
About this study
Official title: A Multi-center, Randomized, Double-blind, Placebo-controlled, Multiple Dose Study to Investigate Safety and Tolerability of RG1662 in Individuals With Down Syndrome
This multi-center, randomized double-blind, placebo-controlled study will assess the safety and tolerability of RG1662 in individuals with Down Syndrome. Eligible subjects will be randomized in cohorts to receive either multiple oral doses of RG1662 or placebo. Anticipated time on study treatment is 38 days.
- Conditions
- Down Syndrome
- Being tested
- Placebo, RG1662
- 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
multiple oral doses
RG1662Drug
Cohorts receiving multiple oral doses
| Study group | Role | What they receive |
|---|---|---|
| A | Experimental | Not described Receives: Drug: RG1662 |
| B | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety: Incidence of adverse events
Measured: 16 weeks
Secondary outcomes (2)
Neurocognitive assessment: CANTAB (Computerized battery for assessing motor practice, reaction time and memory)/standard cognitive tests
Measured: 5-6 weeks
Pharmacokinetics: Serum concentration/area under the concentration-time curve (AUC)
Measured: 5-6 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Sep 2011 (estimated)
- Study startNov 2011
- Primary completionSep 2013
- Study completionSep 2013
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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 35 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
- NCT01436955
- Sponsor's protocol ID
- BP25543
- Status last verified
- Mar 2015
- First submitted
- 18 Aug 2011
- Last updated
- 3 Mar 2015
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-center, Randomized, Double-blind, Placebo-controlled, Multiple Dose Study to Investigate Safety and Tolerability of RG1662 in Individuals With Down Syndrome
- ClinicalTrials.gov ID
- NCT01436955
- Acronym / sponsor ID
- BP25543
- Conditions
- Down Syndrome
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 35 participants (actual)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Study start
- Nov 2011
- Primary completion
- Sep 2013 (actual)
- Study completion
- Sep 2013 (actual)
- First posted
- 20 Sep 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.
This multi-center, randomized double-blind, placebo-controlled study will assess the safety and tolerability of RG1662 in individuals with Down Syndrome. Eligible subjects will be randomized in cohorts to receive either multiple oral doses of RG1662 or placebo. Anticipated time on study treatment is 38 days.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| A | Experimental | Drug: RG1662 |
| B | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 18 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (4 listed)
- Adult individuals, 18 to 30 years of age, with diagnosis of Down Syndrome (confirmed by karyotype)
- Males and non-pregnant non-lactating females
- Parent or legal guardian/representative and caregiver willing to give written informed consent
- Subject willing and assenting or consenting to participate
Key exclusion criteria (10 listed)
- Major depressive disorder not adequately controlled by a selective serotonin reuptake inhibitor or a serotonin norepinephrine reuptake inhibitor
- Subjects meeting clinical diagnostic criteria for autistic spectrum disorder, attention deficit or hyperactivity disorder unlikely to cooperate and take part successfully in the study assessments
- Subjects with other primary psychiatric diagnosis
- Subjects with evidence or meeting clinical diagnosis of dementia
- Subjects with personal history of cardiac abnormalities; patients that have previously undergone congenital heart surgery can be enrolled provided they have no residual complication requiring intervention (e.g. pace maker)
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 |
|---|---|---|
| Placebomultiple oral doses | Drug | B |
| RG1662Cohorts receiving multiple oral doses | Drug | A |
Primary outcome
Safety: Incidence of adverse events
Time frame: 16 weeks
2 secondary outcomes
Neurocognitive assessment: CANTAB (Computerized battery for assessing motor practice, reaction time and memory)/standard cognitive tests
Time frame: 5-6 weeks
Pharmacokinetics: Serum concentration/area under the concentration-time curve (AUC)
Time frame: 5-6 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 3 Mar 2015.
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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 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing RG1662 in people with Down Syndrome, run by Hoffmann-La Roche at 9 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 4 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 3 Mar 2015. 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, United Kingdom. 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 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?
Safety: Incidence of adverse events (time frame: 16 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 3 Mar 2015.