A Screening Protocol to Assess Adults and Adolescents With Down Syndrome for Eligibility For Upcoming Study of RG1662 (Study BP27832)

A Screening Protocol to Assess Adult and Adolescent Individuals With Down Syndrome for Eligibility to Participate in an Upcoming Study to Evaluate the Efficacy, Safety and Tolerability of RG1662 (Study BP27832)

This is a single visit study in which people with Down syndrome aged 12-30 will attend the clinical center to undergo assessments to evaluate their eligibility for potential enrollment in the future BP27832 Phase II clinical study which will evaluate the efficacy, safety and tolerability of RG1662. Study participants may withdraw from this study at any time and for any reason.

Study Overview

• Status: Completed
• Conditions: Down Syndrome

• Study Type: Observational
• Enrollment (Actual): 135

Contacts and Locations

Study Locations

• Argentina
  • Capital Federal, Argentina, C1428AQK
    • Instituto de Investigaciones Neurologicas Raul Carrea FLENI
  • Ciudad Autonoma de Bs As, Argentina, 1426
    • Instituto Neurologia Bs As
• Canada
  • Nova Scotia
    • Kentville, Nova Scotia, Canada, B4N 4K9
      • True North Clinical Research Kentville
• Mexico
  • Aguascalientes, Mexico, 20030
    • Clínica Para la Atención del Neurodesarrollo
  • Queretaro, Mexico, 76000
    • Hospital Médica Tec 100
  • Jalisco
    • Guadalajara, Jalisco, Mexico, 45200
      • Hospital Dr. Angel Leaño; Pediatria
• New Zealand
  • Auckland, New Zealand, 1142
    • Auckland Clinical Studies
  • Dunedin, New Zealand, 9016
    • University of Otago; Psychological Medicine Department
  • Wellington, New Zealand, 6021
    • Wellington Hospital Research Office
• Singapore
  • Singapore, Singapore, 229899
    • KK Women's and Children's Hospital; Department of Neonatology
• Spain
  • Barcelona, Spain, 08009
    • IMIM, Human Pharmacology and Clinical Neurosciences,
  • Madrid, Spain, 28006
    • Hospital Universitario de la Princesa; Medicina Interna
  • Girona
    • Salt, Girona, Spain, 17090
      • UVaMID Hospital Santa Caterina;; Servicio de Neurología
• United States
  • California
    • La Jolla, California, United States, 92037
      • Univ of CA San Diego; Neurosciences Comp.Alzheimer's
  • Georgia
    • Decatur, Georgia, United States, 30033
      • Emory University School of Medicine; Department of Human Genetics & Pediatrics
  • Illinois
    • Chicago, Illinois, United States, 60612
      • Rush University Medical Center
  • Maryland
    • Baltimore, Maryland, United States, 21287
      • Johns Hopkins Hospital.
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusette General Hospital; Medical Genetics
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Duke Clin Rsch Institute
  • Texas
    • Dallas, Texas, United States, 75390-8813
      • UT Southwestern Medical Center
  • Utah
    • Salt Lake City, Utah, United States, 84112
      • University of Utah School of Medicine; Department of Pediatrics
  • Wisconsin
    • Madison, Wisconsin, United States, 53705
      • University of Wisconsin Madison, Waisman Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years to 30 years (ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

People with Down syndrome

Description

Inclusion Criteria:

Study participants will be assessed on the following criteria that they will be required to meet in order to participate in the future Phase II study BP27832:

• Males and females 12-30 years of age (18-30 in the US)
• Clinical diagnosis of Down syndrome (trisomy 21) documented by chromosomal analysis (karyotyping)
• Body-mass Index (BMI) 18-42 and 15-32 kg/m2 inclusive for adults and adolescents respectively
• Ability to complete the Clinical Evaluation of Language Fundamentals (CELF)-preschool 2 word classes task (i.e., ≥ 7 for the adults or ≥ 4 for the adolescents in the expressive raw score).
• Study participant willing and assenting or consenting to participate
• Parent or guardian willing to give written informed consent
• Study participants must have a parent, or other reliable caregiver who will agree to accompany the study participant to clinic visits during the treatment study, BP27832
• The parent or caregiver must be a constant and reliable informant with sufficient contact with the study participant to have detailed knowledge of the study participant's adaptive functioning in order to be able to complete assessments accurately
• Study participants must be verbal and able to be understood most of the time and must not use other forms of communication, signs, symbol boards or devices as their primary form of communication
• Study participants must have sufficient vision and hearing to participate in study evaluations
• Study participants on anti-epileptic treatment must be on stable doses for 4 weeks prior to enrollment in the treatment protocol

Exclusion Criteria:

In the future Phase II study, study participants must not meet any of the following criteria :

• Study participants with severe lactose intolerance
• Study participants with moderate or severe Obstructive Sleep Apnea (OSA) as defined by Apnea-Hypopnea Index (AHI) (>15 events per hour not well controlled by positive airway pressure therapy with stable settings) for 6 weeks prior to the screening visit
• Study participants with history of malignancy if not considered likely to be cured
• Personal history of infantile spasms, of epilepsy, of severe head trauma or Central Nervous System (CNS) infections (e.g. meningitis), with the exception of a single isolated febrile seizure
• Study participants with history of epilepsy within the last 2 years.
• Evidence of active, clinically significant, and unstable gastrointestinal, renal, hepatic, endocrine or cardiovascular system disease
• Study participants with a current Diagnostic and Statistical Manual of Mental Disorders (DSM-5) diagnosis of any primary psychiatric diagnosis (including autism spectrum disorder). Diagnoses that are secondary, such as intellectual disability, attention deficit hyperactivity disorder, depression and conduct disorder are allowed as long as they are considered stable and to not interfere with conduct of a future treatment study
• Study participants with a history of suicide attempt or deliberate self-harm due to suicidal ideation will not be included. Suicidal ideation (even in the absence of suicide attempt or deliberate self-harm) during the 6 months prior to screening
• Concomitant use of excluded approved or unapproved medications
• Personal or family history of congenital long QT syndrome
• History of hepatitis C or known Human Immunodeficiency Virus (HIV) infection
• Pregnant or breast feeding

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
People with Down syndrome

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Identification of people with Down syndrome aged 12-30 eligible for upcoming BP27832 study of RG1662	1 day

Collaborators and Investigators

• Sponsor: Hoffmann-La Roche

Study record dates

Study Major Dates

• Study Start (ACTUAL): October 31, 2013
• Primary Completion (ACTUAL): January 31, 2015
• Study Completion (ACTUAL): January 31, 2015

Study Registration Dates

• First Submitted: August 8, 2013
• First Submitted That Met QC Criteria: August 8, 2013
• First Posted (ESTIMATE): August 12, 2013

Study Record Updates

• Last Update Posted (ACTUAL): July 19, 2017
• Last Update Submitted That Met QC Criteria: July 17, 2017
• Last Verified: July 1, 2017

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Congenital Abnormalities; Genetic Diseases, Inborn; Intellectual Disability; Abnormalities, Multiple; Chromosome Disorders; Syndrome; Down Syndrome
• Other Study ID Numbers: BP28947; 2013-001264-33 (EUDRACT_NUMBER)