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A Molecular and Functional Brain Imaging Study in Individuals With Down Syndrome and Healthy Controls Following Single Dose RG1662

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01667367
Recruitment Status : Completed
First Posted : August 17, 2012
Last Update Posted : September 2, 2015
Information provided by (Responsible Party):
Hoffmann-La Roche

Brief 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. 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.

Condition or disease Intervention/treatment Phase
Healthy Volunteer, Down Syndrome Drug: RG1662 Drug: placebo Phase 1

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 13 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Single (Participant)
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
Study Start Date : August 2012
Actual Primary Completion Date : October 2013
Actual Study Completion Date : October 2013

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Placebo Comparator: Placebo Drug: placebo
Single oral dose

Experimental: RG1662 Drug: RG1662
Single oral dose

Primary Outcome Measures :
  1. Brain GABAA receptor alpha5 subunit localization/density assessed by positron emission tomography (PET) [ Time Frame: approximately 1 day ]
  2. Brain GABAA receptor alpha5 subunit occupancy following single dose of RG1662 [ Time Frame: approximately 1 day ]
  3. Correlation of RG1662 plasma concentrations and GABAAalpha5 receptor occupancy [ Time Frame: approximately 12 months ]

Secondary Outcome Measures :
  1. Safety: Incidence of adverse events [ Time Frame: approximately 12 months ]
  2. 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 ]
  3. Functional brain connectivity of individuals with Down syndrome versus healthy controls receiving placebo [ Time Frame: approximately 12 months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 40 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes

Inclusion Criteria:

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

Exclusion Criteria:

  • 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
  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01667367

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United Kingdom
London, United Kingdom, NW10 7EW
Sponsors and Collaborators
Hoffmann-La Roche
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Study Director: Clinical Trials Hoffmann-La Roche

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Responsible Party: Hoffmann-La Roche Identifier: NCT01667367    
Other Study ID Numbers: BP25611
2012-001301-24 ( EudraCT Number )
First Posted: August 17, 2012    Key Record Dates
Last Update Posted: September 2, 2015
Last Verified: September 2015
Additional relevant MeSH terms:
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Down Syndrome
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn