Current Clinical Trials Underway

Currently, three human clinical trials are underway. The trials are being conducted by Roche Laboratories, Balance Therapeutics and Elan Pharmaceuticals. The Roche and Balance trials are investigating compounds that are shown to restore more balanced neuro-transmission, and improved learning, in mouse models. The Elan trial is testing a compound that may reduce beta-amyloid aggregation. This aggregation is a contributor to plaque formation observed in persons with Alzheimer's disease.

Roche Laboratories
A multi-national screening protocol clinical study for anticipated RG1662 Phase II clinical trial in individuals with Down syndrome

Trial sites listed on this site include:

United Kingdom


United States

Little Rock, AR
Phoenix, AZ
La Jolla, CA
Orange, CA
Aurora, CO
Decatur, GA
Chicago, IL
Baltimore, MD
Durham, NC

Balance Therapeutics
BTD-001 DS102: A Phase 1b, Double-blind, Randomized, Placebo-Controlled, Parallel Group Study of the Safety, Tolerability, Preliminary Efficacy and Pharmacodynamics of BTD-001 in Young Adults and Adolescents with Down Syndrome. 

A 4-Week Safety Study of Oral ELND005 in Young Adults With Down Syndrome Without Dementia (DS201). Learn more here.

A National Down Syndrome Patient Registry
The NIH announced on October 20 that the contract has been awarded to establish a new Down Syndrome Patient Registry. The registry will allow people with Down syndrome or their family members to enter contact information and health history into a secure, on-line database. This will facilitate connections between individuals with Down syndrome and researchers. Details of the registry may be found here