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

This study is currently recruiting participants.
Verified June 2013 by Hoffmann-La Roche
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche
ClinicalTrials.gov Identifier:
NCT01667367
First received: August 14, 2012
Last updated: June 11, 2013
Last verified: June 2013
History of Changes
  Purpose

This single-center, double-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. On two separate visits, no less than 96 hours and no more than 6 weeks apart, subjects will receive a single oral dose of either RG1662 or placebo followed by positron emission tomography (PET) and magnetic resonance imaging (MRI) scans.


Condition Intervention Phase
Down Syndrome, Healthy Volunteer
 Drug: RG1662
Drug: placebo
 Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Pharmacodynamics Study
Intervention Model: Crossover Assignment
Masking: Double-Blind
Official Title: A Single-center, Double 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

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Hoffmann-La Roche:

Primary Outcome Measures:
  • Brain GABAA receptor alpha5 subunit localization/density assessed by positron emission tomography (PET) [ Time Frame: approximately 1 day ] [ Designated as safety issue: No ]
  • Brain GABAA receptor alpha5 subunit occupancy following single dose of RG1662 [ Time Frame: approximately 1 day ] [ Designated as safety issue: No ]
  • Correlation of RG1662 plasma concentrations and GABAAalpha5 receptor occupancy [ Time Frame: approximately 12 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Safety: Incidence of adverse events [ Time Frame: approximately 12 months ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • Functional brain connectivity of individuals with Down syndrome versus healthy controls receiving placebo [ Time Frame: approximately 12 months ] [ Designated as safety issue: No ]

Estimated Enrollment: 17
Study Start Date: August 2012
Estimated Study Completion Date: September 2013
Estimated Primary Completion Date: September 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: RG1662 Drug: RG1662
Single oral dose
Placebo Comparator: Placebo Drug: placebo
Single oral dose

  Eligibility

Ages Eligible for Study:   18 Years to 30 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Healthy controls and individuals with Down syndrome:

  • Male and female adults, 18 to 30 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; subjects 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
  • Subjects must have a parent or other reliable caregiver who agrees to accompany the subject to all clinic visits, provide information about the subject 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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01667367

Contacts
Contact: Reference Study ID Number: BP25611 www.roche.com/about_roche/roche_worldwide.htm 888-662-6728 (U.S. Only) global.rochegenentechtrials@roche.com

Locations
United Kingdom
Recruiting
London, United Kingdom, NW10 7EW
Sponsors and Collaborators
Hoffmann-La Roche
Investigators
Study Director: Clinical Trials Hoffmann-La Roche
  More Information

No publications provided

Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT01667367     History of Changes
Other Study ID Numbers: BP25611, 2012-001301-24
Study First Received: August 14, 2012
Last Updated: June 11, 2013
Health Authority: United Kingdom: Medicines and Healthcare Products Regulatory Agency

Additional relevant MeSH terms:
Down Syndrome
Mental Retardation
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
 Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on June 13, 2013