Clinical Trials
Down Syndrome
Title: A 10-WEEK, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF DONEPEZIL HYDROCHLORIDE (ARICEPTâ) IN THE TREATMENT OF THE COGNITIVE DYSFUNCTION EXHIBITED BY CHILDREN WITH DOWN SYNDROME
Purpose of the study
The objective of this study is to evaluate the efficacy and safety of donepezil hydrochloride in the treatment of the cognitive dysfunction exhibited by children with Down syndrome as assessed in the domains of communication, daily living skills, and socialization.
Who is eligible?
Subjects (male or female) will be residing in the community, 10 to 17 years of age, weighing >20 kg, ambulatory or ambulatory-aided, with a diagnosis of Down syndrome (trisomy 21) documented by chromosomal analysis and staging of mild or moderate mental retardation as indicated by a Vineland-II composite adaptive behavior standard score of >55 at screen. Prior cholinesterase inhibitor use is allowed as long as use has been discontinued for greater than or equal to 3 months prior to study entry and has not been discontinued for intolerance or lack of efficacy or solely for study entry.
Down Syndrome
Title: A 12-WEEK DOUBLE BLIND PLACEBO CONTROLLED CLINICAL TRIAL FOLLOWED BY A 36 WEEK TREATMENT EXTENSION TO EVALUATE THE SAFETY AND EFFICACY OF RIVASTIGMINE IN CHILDREN WITH DOWN SYNDROME
Purpose of the study
This study is looking at the effects of a cholinergic medication (rivastigmine tartrate) on healthy adolescents with Down syndrome to see if it improves memory, attention, language, or activities of daily living.
Who is eligible?
Children ages 10 to 18 with Down syndrome (trisomy 21, translocation, or mosaic) with no health problems that contraindicate use of rivastigmine tartrate. Subjects must be able to communicate independently with examiners without the use of sign or assistive devices.
Glycogen Storage Disease Type II (Pompe Disease)
For information on current research studies and clinical trials on GSD type II (Pompe Disease):
Contact: Joanne MacKay, Nurse Practitioner, at 919-681-1945, macke003@mc.duke.edu, or Stephanie DeArmey, Physician Assistant, at 919-681-1946, dearm001@mc.duke.edu.
Duchenne and Becker Muscular Dystrophy
This study is investigating PTC 124 as a treatment in patients with Duchenne and Becker Muscular Dystrophy who have nonsense mutations.
Please note: This study is closed to enrollment
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