Clinical trial NCT01920633: A Screening Protocol to Assess Adults and Adolescents With Down Syndrome for Eligibility For Upcoming Study of RG1662 (Study BP27832)
Official title: 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)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Down Syndrome
Lead sponsor: Hoffmann-La Roche
Sponsor class: INDUSTRY
First posted: 2013-08-12
Enrollment: 135
Sex: ALL
Minimum age (years): 12
Maximum age (years): 30
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: People with Down syndrome

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Identification of people with Down syndrome aged 12-30 eligible for upcoming BP27832 study of RG1662
timeFrame: 1 day
Secondary outcomes:
Other outcomes:
Locations:
city: La Jolla
country: United States
facility: Univ of CA San Diego; Neurosciences Comp.Alzheimer's
state: California
city: Decatur
country: United States
facility: Emory University School of Medicine; Department of Human Genetics & Pediatrics
state: Georgia
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Baltimore
country: United States
facility: Johns Hopkins Hospital.
state: Maryland
city: Boston
country: United States
facility: Massachusette General Hospital; Medical Genetics
state: Massachusetts
city: Durham
country: United States
facility: Duke Clin Rsch Institute
state: North Carolina
city: Dallas
country: United States
facility: UT Southwestern Medical Center
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah School of Medicine; Department of Pediatrics
state: Utah
city: Madison
country: United States
facility: University of Wisconsin Madison, Waisman Center
state: Wisconsin
city: Capital Federal
country: Argentina
facility: Instituto de Investigaciones Neurologicas Raul Carrea FLENI
city: Ciudad Autonoma de Bs As
country: Argentina
facility: Instituto Neurologia Bs As
city: Kentville
country: Canada
facility: True North Clinical Research Kentville
state: Nova Scotia
city: Guadalajara
country: Mexico
facility: Hospital Dr. Angel Leaño; Pediatria
state: Jalisco
city: Aguascalientes
country: Mexico
facility: Clínica Para la Atención del Neurodesarrollo
city: Queretaro
country: Mexico
facility: Hospital Médica Tec 100
city: Auckland
country: New Zealand
facility: Auckland Clinical Studies
city: Dunedin
country: New Zealand
facility: University of Otago; Psychological Medicine Department
city: Wellington
country: New Zealand
facility: Wellington Hospital Research Office
city: Singapore
country: Singapore
facility: KK Women's and Children's Hospital; Department of Neonatology
city: Salt
country: Spain
facility: UVaMID Hospital Santa Caterina;; Servicio de Neurología
state: Girona
city: Barcelona
country: Spain
facility: IMIM, Human Pharmacology and Clinical Neurosciences,
city: Madrid
country: Spain
facility: Hospital Universitario de la Princesa; Medicina Interna
Study officials:
affiliation: Hoffmann-La Roche
name: Clinical Trials
role: STUDY_DIRECTOR
References: