Clinical trial NCT01436955: A Study of RG1662 in Individuals With Down Syndrome
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Down Syndrome
Lead sponsor: Hoffmann-La Roche
Sponsor class: INDUSTRY
First posted: 2011-09-20
Enrollment: 35
Sex: ALL
Minimum age (years): 18
Maximum age (years): 30
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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.


Eligibility criteria: 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

Primary outcomes:
measure: Safety: Incidence of adverse events
timeFrame: 16 weeks
Secondary outcomes:
measure: Neurocognitive assessment: CANTAB (Computerized battery for assessing motor practice, reaction time and memory)/standard cognitive tests
timeFrame: 5-6 weeks
measure: Pharmacokinetics: Serum concentration/area under the concentration-time curve (AUC)
timeFrame: 5-6 weeks
Other outcomes:
Locations:
city: Phoenix
country: United States
state: Arizona
city: Little Rock
country: United States
state: Arkansas
city: La Jolla
country: United States
state: California
city: Aurora
country: United States
state: Colorado
city: Decatur
country: United States
state: Georgia
city: Chicago
country: United States
state: Illinois
city: Baltimore
country: United States
state: Maryland
city: Durham
country: United States
state: North Carolina
city: London
country: United Kingdom
Study officials:
affiliation: Hoffmann-La Roche
name: Clinical Trials
role: STUDY_DIRECTOR
References: