Safety Efficacy Study in 12-30y old Down Syndrome subjects

Phase 1

• Conditions: Down Syndrome; MedDRA version: 17.0Level: PTClassification code 10044688Term: Trisomy 21System Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2013-001263-23-GB
• Lead Sponsor: F. Hoffmann-La Roche Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-12-17
• Last Update Posted: 3 April 2017

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 180

Inclusion Criteria

1. Individuals aged 12-30 years of age inclusive.

2. Clinical diagnosis of Down syndrome (trisomy 21) confirmed by chromosomal analysis (karyotyping). Subjects may have standard trisomy 21, Robertsonian translocation, isochromosome 21 (so called 21q21q Robertsonian) translocation), Down syndrome with reciprocal translocation or mosaicism.

3. Males, or nonpregnant, nonlactating females. For females of childbearing potential, strict contraceptive prevention is required: continuation of hormonal contraception, or intra uterine device. True abstinence is acceptable provided that participants are under the supervision of a caregiver attesting that participants are not sexually active and the results of pregnancy tests are negative before onset of treatment.

4. Body-mass Index (BMI) 18-42 and 15-32 kg/m2 inclusive for adults and adolescents respectively

5. Ability to complete the Clinical Evaluation of Language Fundamentals (CELF)-preschool 2 word classes task (i.e., = 7 for adults or = 4 for adolescents in the expressive raw score).
Are the trial subjects under 18? yes
Number of subjects for this age range: 90
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 90
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Subjects with a current Diagnostic and Statistical Manual of Mental Disorders (DSM 5) diagnosis of any primary psychiatric diagnosis (including autism spectrum disorder or major depressive 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 to not interfere with study conduct and the subject is on stable treatment for 3 months preceding enrollment.

2. Subjects with a history of infantile spasms, of West syndrome, Lennox-Gastaut syndrome, Early Infantile Epileptic Encephalopathy or any treatment-refractory epilepsy associated with cognitive or developmental regression, of severe head trauma or CNS infections (e.g. meningitis).

3. Subjects with a known or suspected clinical seizure event of any type within 24 months prior to screening.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified