Clinical Trials


NEW: Roche initiates new multi-national RG1662 Phase II clinical trial in individuals ages 12-30 years old with Down syndrome

Status: Ongoing — currently recruiting

Roche, a leader in innovative research-focused healthcare and the world’s largest biotech company, has initiated a new multi-national Phase II clinical trial to evaluate the efficacy and safety of RG1662 in adolescents and adults, ages 12-30 years old, with Down syndrome. Following the successful completion of the previous groundbreaking Phase I clinical trial with this new drug that is being developed to address the cognitive and behavioral deficits in individuals with Down syndrome, this new significantly larger Phase II trial is a randomized, double-blind, placebo-controlled clinical study in which participants will be randomized to receive RG1662 either at low or high dose or placebo orally twice daily for 26 weeks.

This new trial, “A Study of RG1662 in Adults and Adolescents with Down Syndrome (CLEMATIS)”, will be conducted at multiple sites across the US, including La Jolla, CA, Sacramento, CA, Decatur, GA, Chicago, IL, Baltimore, MD, Boston, MA, Durham, NC, Dallas, TX, Salt Lake City, UT, and Madison, WI. In addition, the trial will be conducted at multiple international sites, including UK (4), France (4), Spain (3), Singapore (1), New Zealand (3), Canada (1), Mexico (5) and Argentina (2).

Learn more about this important new study and information on US recruitment and participation by calling 1-888-662-6728 and/or clicking here, and for international recruitment and participation here. 

Elan (now Transition Therapeutics) Announces Study of ELND005

Status: Completed

Biotechnology company Elan Corporation, plc (now Transition Therapeutics), has announced the initiation of a Phase 2A clinical trial of ELND005 (scyllo-inositol) in young adults, 18-45 years of age, with Down syndrome. ELND005 is a molecule that may hold the potential to improve cognition in Ds by reducing aggregation of beta-amyloid, which is a product of the amyloid precursor protein (APP) encoded by a gene on chromosome 21 — thus preventing the intraneuronal buildup of beta-amyloid plaques that most people with Ds develop by their 40s — and addressing a metabolic abnormality in Ds that correlates with the severity of cognitive dysfunction. The study now underway will primarily evaluate the safety and pharmokinetics of the molecule, and will include select cognitive and behavioral measures.

There are currently three clinical trial sites in the US: University of California, San Diego in La Jolla, CA; University of California, Irvine in Orange, CA; and Massachusetts General Hospital in Boston, MA. Learn more about this clinical study, including details on participation.


Balance Therapeutics’ COMPOSE clinical study for BTD-001

Status: Ongoing — currently recruiting

Balance Therapeutics is currently conducting a Phase Ib clinical trial at multiple sites in Australia to assess the safety and potential efficacy of a compound that could enhance cognitive function in people with Down syndrome age 13 to 35.  The Compose study (Cognition and Memory in People with Down Syndrome) is evaluating the potential of a compound called BTD-001 to improve memory, language, and learning. BTD-001 is well understood and had been used over decades to treat a variety of conditions. The clinical study is based on original research at Stanford University which was supported by the Foundation. The National Institutes of Health has also provided funding grants to support additional development.

Learn more about this clinical study opportunity, including details about participation.

Study on Spoken Language Development
Status: Ongoing — currently recruiting

Individuals with Down syndrome between the ages of 6 and 23 are invited to participate in a major new multi-site research study funded by the NIH Eunice Kennedy Shriver National Institute of Child Health and Human Development. The study will examine different methods of sampling expressive language in children, adolescents, and young adults with DS to determine whether such measures could serve as useful indicators of efficacy in drug studies. This is a behavioral study and does not involve any use of medication. Learn more about this study and how to participate.

Roche's Clinical Trial of an Investigational Drug, RG1662

Status: Completed

In 2011, Roche initiated an early-stage clinical trial, to test an investigational drug in individuals with Down syndrome. The goal is to evaluate its tolerability and obtain an early reading of its effect on the cognitive and behavioral deficits associated with this condition.

The first stage of this clinical trial involved individuals with Trisomy 21 between the ages of 18-30 residing near one of the eight trial locations around the country. Designed to take place over approximately 16 weeks, the study sought to test the tolerability of the investigational drug. During the study, the heart rate, EEG, and laboratory parameters of safety of the participants were monitored very closely, and parents were involved with tracking physical and any emotional/cognitive changes.

Information about this trial is archived on

Roche's Clinical Study to Evaluate Neurocognition Measures

Status: Completed

Following its 2011 initiation of the study of RG1662, Roche launched a second new Down syndrome clinical study, “A Non-Drug Study of The Suitability of Neurocognitive Tests and Functioning Scales for the Measurement of Cognitive and Functioning Changes in Individuals with Down Syndrome." The goal of this study is to evaluate the suitability of neurocognitive tests and functioning scales for the measurement of cognitive and functioning changes in individuals with Down syndrome.

The primary objectives of this clinical study include assessing the performance and reliability of specific scales and measures of cognitive function, memory, adaptive behavior, activities of daily living and impact on caregivers.  This is a non-drug study; participants will not be required to take any study medication. Study participants will be administered various neurocognitive tests and their caregiver will be asked to complete questionnaires. The study, with duration up to 27 weeks for participants involving approximately three visits, was designed to involve 90 individuals, ages 12-30 years, with Down syndrome.

There are currently multiple clinical study sites internationally, including two in the US. The University of Arizona in Tucson, AZ and Duke University in Durham, NC represent the US clinical study sites.  Dr. Jamie Edgin, Co-Principal Investigator of the Down Syndrome Research Group and LuMind-supported researcher, is the study lead at the University of Arizona.

You can learn more about this clinical study at and the Roche Trials Database.

Roche’s Clinical Trial on Molecular and Functional Imaging

Status: Completed

In August 2012, Roche initiated a third Down syndrome clinical trial, “A Molecular and Functional Brain Imaging Study in Individuals with Down Syndrome and Healthy Controls Following Single Dose RG1662”. The goal of this new study was to use MRI and PET imaging scans to evaluate the GABA-A alpha5 receptor expression, occupancy and functional connectivity in the brains of individuals with Down syndrome and healthy controls, ages 18-30 years, following single-dose RG1662. This study was designed to provide additional clinical information to support RG1662 clinical trials in individuals with Down syndrome.

You can learn more about this study and associated information at

LuMind Research Down Syndrome Foundation

225 Cedar Hill Street, Suite 200  Marlborough, MA  01752
Phone: 508.630.2177

© 2015 LuMind Foundation. All Rights Reserved.