Healthy Volunteer, Down SyndromePhase 1NCT01667367
A Molecular and Functional Brain Imaging Study in Individuals With Down Syndrome and Healthy Controls Following Single Dose RG1662
A Phase 1 study testing RG1662 in people with Healthy Volunteer, Down Syndrome, run by Hoffmann-La Roche at 1 site in United Kingdom.
Trial summary
This single-center, single-blind, placebo-controlled, parallel-group study with crossover component will evaluate the GABAAalpha5 receptor expression, occupancy and functional connectivity in the brains of individuals with Down syndrome and healthy controls following single dose RG1662. Read more about this study
- Who is it for?
- Ages 18 to 40 yearsHealthy volunteers can take part
- Conditions the study is for
- Healthy Volunteer, 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
- 13 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Aug 2012to Oct 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 40 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Healthy controls and individuals with Down syndrome:
- Male and female adults, 18 to 40 years of age
- Body mass index (BMI) 18 - 40 kg/m2 inclusive
- Females of child-bearing potential and males with female partners of child-bearing potential must agree to use two medically approved methods of contraception, one of which must be a barrier method, for the duration of the study and for 4 months after the last study drug administration
- Clinical laboratory values within normal limits or abnormalities considered not significant by the investigator and sponsor; individuals with thyroid disease may be included in the study provided they are euthyroid and stable on treatment for at least one month prior to screening
- Individuals with Down syndrome must also meet the following:
- Diagnosis of Down syndrome confirmed by karyotype; subjects may have free trisomy 21 or Robertsonian translocations; mosaic Down syndrome will be excluded
- Individuals must have a parent or other reliable caregiver who agrees to accompany the inidvidual to all clinic visits, provide information about the individual as required by protocol, and be willing to give informed consent
Who cannot join
- Regular smoker (>5 cigarettes or equivalent: 3 cigars, 6 cigarillos, 3 pipes per day)
- Concomitant disease or condition or any clinically significant finding at screening that could interfere with, or for which the treatment might interfere with the conduct of the study, or that would, in the opinion of the investigator, pose an unacceptable risk to the subject in this study
- Severe head trauma or CNS infections (e.g. meningitis)
- History of epilepsy or seizures other than benign febrile convulsions of childhood
- Any confirmed significant allergic reactions against any drug, anaphylaxis or severe environmental allergies as judged by the investigator
- Positive for hepatitis B, hepatitis C or HIV infection
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- Positive urine test at screening or at follow-up for drugs of abuse, or positive alcohol breath test at screening and prior to dosing
- Previous inclusion in research and/or medical protocol involving nuclear medicine, PET or radiological investigations with significant radiation burden (as defined by protocol)
- Pregnant or lactating women
- Individuals with evidence or meeting clinical diagnosis of dementia
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Single-center, Single Blind Molecular and Functional Imaging Study to Assess GABAAalpha5 Receptor Expression, Occupancy and Functional Connectivity in the Brains of Individuals With Down Syndrome and Healthy Controls Following a Single Oral Administration of RG1662 or Placebo
This single-center, single-blind, placebo-controlled, parallel-group study with crossover component will evaluate the GABAAalpha5 receptor expression, occupancy and functional connectivity in the brains of individuals with Down syndrome and healthy controls following single dose RG1662. Participants will receive a single dose of placebo before the imaging session (PET and MRI), and a single dose of RG1662 before the second imaging session.
- Conditions
- Healthy Volunteer, Down Syndrome
- Being tested
- RG1662, placebo
- Medical subject headings
- Down Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RG1662Drug
Single oral dose
placeboDrug
Single oral dose
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Not described Receives: Drug: placebo |
| RG1662 | Experimental | Not described Receives: Drug: RG1662 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Brain GABAA receptor alpha5 subunit localization/density assessed by positron emission tomography (PET)
Measured: approximately 1 day
Brain GABAA receptor alpha5 subunit occupancy following single dose of RG1662
Measured: approximately 1 day
Correlation of RG1662 plasma concentrations and GABAAalpha5 receptor occupancy
Measured: approximately 12 months
Secondary outcomes (3)
Safety: Incidence of adverse events
Measured: approximately 12 months
Resting state functional brain connectivity following a single dose of either RG1662 or placebo, assessed by functional magnetic resonance imaging (fMRI)
Measured: approximately 1 day
Functional brain connectivity of individuals with Down syndrome versus healthy controls receiving placebo
Measured: approximately 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Aug 2012 (estimated)
- Study startAug 2012
- Primary completionOct 2013
- Study completionOct 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Healthy Volunteer, 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
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 13 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
- NCT01667367
- Sponsor's protocol ID
- BP25611
- Other IDs
- 2012-001301-24
- Status last verified
- Sep 2015
- First submitted
- 14 Aug 2012
- Last updated
- 2 Sep 2015
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Sep 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-center, Single Blind Molecular and Functional Imaging Study to Assess GABAAalpha5 Receptor Expression, Occupancy and Functional Connectivity in the Brains of Individuals With Down Syndrome and Healthy Controls Following a Single Oral Administration of RG1662 or Placebo
- ClinicalTrials.gov ID
- NCT01667367
- Acronym / sponsor ID
- BP25611
- Conditions
- Healthy Volunteer, Down Syndrome
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Study start
- Aug 2012
- Primary completion
- Oct 2013 (actual)
- Study completion
- Oct 2013 (actual)
- First posted
- 17 Aug 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 single-center, single-blind, placebo-controlled, parallel-group study with crossover component will evaluate the GABAAalpha5 receptor expression, occupancy and functional connectivity in the brains of individuals with Down syndrome and healthy controls following single dose RG1662. Participants will receive a single dose of placebo before the imaging session (PET and MRI), and a single dose of RG1662 before the second imaging session.
Study design
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: placebo |
| RG1662 | Experimental | Drug: RG1662 |
Who may qualify
- Ages
- 18 to 40 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (8 listed)
- Male and female adults, 18 to 40 years of age
- Body mass index (BMI) 18 - 40 kg/m2 inclusive
- Females of child-bearing potential and males with female partners of child-bearing potential must agree to use two medically approved methods of contraception, one of which must be a barrier method, for the duration of the study and for 4 months after the last study drug administration
- Clinical laboratory values within normal limits or abnormalities considered not significant by the investigator and sponsor; individuals with thyroid disease may be included in the study provided they are euthyroid and stable on treatment for at least one month prior to screening
- Diagnosis of Down syndrome confirmed by karyotype; subjects may have free trisomy 21 or Robertsonian translocations; mosaic Down syndrome will be excluded
Key exclusion criteria (10 listed)
- Regular smoker (>5 cigarettes or equivalent: 3 cigars, 6 cigarillos, 3 pipes per day)
- Concomitant disease or condition or any clinically significant finding at screening that could interfere with, or for which the treatment might interfere with the conduct of the study, or that would, in the opinion of the investigator, pose an unacceptable risk to the subject in this study
- Severe head trauma or CNS infections (e.g. meningitis)
- History of epilepsy or seizures other than benign febrile convulsions of childhood
- Any confirmed significant allergic reactions against any drug, anaphylaxis or severe environmental allergies as judged by the investigator
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 |
|---|---|---|
| RG1662Single oral dose | Drug | RG1662 |
| placeboSingle oral dose | Drug | Placebo |
Primary outcome
Brain GABAA receptor alpha5 subunit localization/density assessed by positron emission tomography (PET)
Time frame: approximately 1 day
Brain GABAA receptor alpha5 subunit occupancy following single dose of RG1662
Time frame: approximately 1 day
Correlation of RG1662 plasma concentrations and GABAAalpha5 receptor occupancy
Time frame: approximately 12 months
3 secondary outcomes
Safety: Incidence of adverse events
Time frame: approximately 12 months
Resting state functional brain connectivity following a single dose of either RG1662 or placebo, assessed by functional magnetic resonance imaging (fMRI)
Time frame: approximately 1 day
Functional brain connectivity of individuals with Down syndrome versus healthy controls receiving placebo
Time frame: approximately 12 months
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 Sep 2015.
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
1 site in 1 country: 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 Healthy Volunteer, Down Syndrome, run by Hoffmann-La Roche at 1 site in United Kingdom.
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 8 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 2 Sep 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in 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?
Brain GABAA receptor alpha5 subunit localization/density assessed by positron emission tomography (PET) (time frame: approximately 1 day). The study lists 3 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 2 Sep 2015; AzTrials last refreshed its copy on 29 Oct 2026.