Clinical trial NCT01084135: Rivastigmine Study in Adolescents With Down Syndrome
Official title: A 20-Week Double-Blind Placebo Controlled Clinical Trial to Evaluate the Safety and Efficacy of Rivastigmine in Children (Ages 10-18) With Down Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Down Syndrome
Keywords: Clinical trial
Down syndrome
adolescents
cognitive
rivastigmine
Lead sponsor: Duke University
Sponsor class: OTHER
Collaborators: name: Taishoff Family Foundation
name: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
First posted: 2010-03-10
Enrollment: 42
Sex: ALL
Minimum age (years): 10
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to determine if short term use of rivastigmine can improve functional abilities (for example, language, memory, and executive function) in adolescents with Down syndrome.

Detailed description: This 24 week, double-blind, placebo controlled trial will be completed at the Clinical Research Unit of Duke University Medical Center and at the Kennedy Krieger Institute (KKI). Sixteen evaluable subjects will be enrolled at Duke and 24 evaluable subjects will be enrolled at KKI. The study consists of four visits, a screening visit (-4 weeks), a baseline visit (week 0); a safety visit at week 10, and a final/termination visit at week 20.

The specific aims of this study are to: a) investigate efficacy of rivastigmine tartrate treatment; b) build upon our open-label treatment results of overall function and language improvement in adolescents with Down syndrome (DS) in a double-blind, placebo-controlled clinical trial; and c) investigate other specific cognitive domains that may selectively respond to rivastigmine tartrate treatment.

The original IRB-approved protocol included the Parent/Caregiver Rating Form of the Vineland Adaptive Behavior Scales- Second Edition (VABS-II) . The protocol was amended to replace the Parent/Caregiver Rating Form of the Vineland Adaptive Behavior Scales- Second Edition (VABS-II) with the Vineland Adaptive Behavior Scales, Second Edition, Survey Interview Form. The protocol was also amended to extend the trial from 12 weeks to 20 weeks. Due to the changes in the amended protocol the subject enrolled prior to the IRB amendment will not be included in the data analysis section.

Eligibility criteria: Inclusion Criteria:

1. Correct VERBAL responses for 7/9 of the Expressive One Word Picture Vocabulary Test items.
2. Subject able to put at least 2-3 words together in conversational speech.
3. Subject's speech is understandable to the examiner for the majority of the time.
4. Subjects are in good health and medically stable

Exclusion Criteria:

1. Subject uses sign language as a primary means of communication
2. Subject has a medical history that contraindicate the use of rivastigmine (For example, patients with active seizure disorders, asthma, celiac disease, heart disease or heart rhythm disorders).

Primary outcomes:
measure: Vineland Adaptive Behavior Scales, Second Edition (Survey Interview Form)
timeFrame: Baseline & Study termination (Week 20)
Secondary outcomes:
measure: Behavior Rating Inventory of Executive Function-Preschool (BRIEF-P)
timeFrame: Baseline and Final (Week 20) visit
Other outcomes:
Locations:
city: Baltimore
country: United States
facility: Kennedy Krieger Institute
state: Maryland
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
Study officials:
affiliation: Duke University
name: Priya Kishnani, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Kennedy Krieger Institute/Johns Hopkins
name: George Capone, MD
role: PRINCIPAL_INVESTIGATOR
References: