A Study of RG1662 in Individuals With Down Syndrome

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.

Study Overview

• Status: Completed
• Conditions: Down Syndrome
• Intervention / Treatment: Drug: RG1662; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 35
• Phase: Phase 1

Contacts and Locations

Study Locations

• United Kingdom
  • London, United Kingdom, NW10 7EW
• United States
  • Arizona
    • Phoenix, Arizona, United States, 85013
  • Arkansas
    • Little Rock, Arkansas, United States, 72204
  • California
    • La Jolla, California, United States, 92037
  • Colorado
    • Aurora, Colorado, United States, 80045
  • Georgia
    • Decatur, Georgia, United States, 30033
  • Illinois
    • Chicago, Illinois, United States, 60612
  • Maryland
    • Baltimore, Maryland, United States, 21287
  • North Carolina
    • Durham, North Carolina, United States, 27710

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 30 years (Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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)
• 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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: A	Drug: RG1662; Cohorts receiving multiple oral doses
Placebo Comparator: B	Drug: Placebo; multiple oral doses

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Safety: Incidence of adverse events	16 weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
Neurocognitive assessment: CANTAB (Computerized battery for assessing motor practice, reaction time and memory)/standard cognitive tests	5-6 weeks
Pharmacokinetics: Serum concentration/area under the concentration-time curve (AUC)	5-6 weeks

Collaborators and Investigators

• Sponsor: Hoffmann-La Roche

Study record dates

Study Major Dates

• Study Start: November 1, 2011
• Primary Completion (Actual): September 1, 2013
• Study Completion (Actual): September 1, 2013

Study Registration Dates

• First Submitted: August 18, 2011
• First Submitted That Met QC Criteria: September 16, 2011
• First Posted (Estimate): September 20, 2011

Study Record Updates

• Last Update Posted (Estimate): March 3, 2015
• Last Update Submitted That Met QC Criteria: March 2, 2015
• Last Verified: March 1, 2015

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Congenital Abnormalities; Genetic Diseases, Inborn; Intellectual Disability; Abnormalities, Multiple; Chromosome Disorders; Syndrome; Down Syndrome
• Other Study ID Numbers: BP25543