A Study of RG1662 in Adults and Adolescents With Down Syndrome (CLEMATIS)
- Conditions
- Down Syndrome
- Interventions
- Drug: PlaceboDrug: RG1662
- Registration Number
- NCT02024789
- Lead Sponsor
- Hoffmann-La Roche
- Brief Summary
This multi-center, randomized, double-blind, 3-arm, parallel-group, placebo-controlled study will evaluate the efficacy and safety of RG1662 in adults and adolescents with Down syndrome. Subjects will be randomized to receive RG1662 either at low or high dose or placebo orally twice daily for 26 weeks.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 173
- Individuals aged 12-30 years of age inclusive
- Clinical diagnosis of Down syndrome (trisomy 21) confirmed by chromosomal analysis (karyotyping)
- Males, or non-pregnant, non-lactating females. For females of childbearing potential, strict contraceptive prevention is required.
- Body-mass Index (BMI) 18-42 and 15-30 kg/m2 inclusive for adults and adolescents respectively
- Ability to complete the Clinical Evaluation of Language Fundamentals (CELF)-preschool 2 word classes task
- Subjects must have a parent, or other reliable caregiver who agrees to accompany the subject to all clinic visits, provide information about the subject as required by the protocol, and ensure compliance with the medication schedule
- Study participants must have sufficient language, vision and hearing to participate in study evaluations, as judged clinically by investigator
- Subjects with a current DSM 5 diagnosis of any primary psychiatric diagnosis (including ASD or MDD)
- 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)
- Subjects with a known or suspected clinical seizure event of any type within 24 months prior to screening
- Clinically relevant ECG abnormalities at screening or baseline; QTcF above 450 ms; personal or family history (first degree relatives) of congenital long QT syndrome
- Inadequate renal or hepatic function
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Placebo - RG1662 120 mg bid RG1662 - RG1662 240 mg bid RG1662 -
- Primary Outcome Measures
Name Time Method Cognition as assessed by the Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) sub-tests 26 weeks Adaptive behavior as assessed by the Vineland Adaptive Behavior Scales-II (VABS-II) standard scores 26 weeks Clinical global impression as assessed by Clinician Rated Global Improvement (CGI-I) scale 26 weeks
- Secondary Outcome Measures
Name Time Method Abnormal ECG changes in adolescents as compared to baseline from baseline to Week 26 Safety: Incidence of adverse events approximately 32 weeks RG1662 plasma concentrations 26 weeks Incidence of abnormal blood pressure 26 weeks Incidence of abnormal ECG changes 26 weeks
Trial Locations
- Locations (37)
Policlinico Universitario "Agostino Gemelli";Dip. Tutela Salute Donna Bambino Adolescente
🇮🇹Roma, Lazio, Italy
ClÃnica Para la Atención del Neurodesarrollo
🇲🇽Aguascalientes, Mexico
Hospital Universitario Dr. Jose Eleuterio Gonzalez; Pediatria
🇲🇽Monterrey, Mexico
KK Women's and Children's Hospital; Department of Neonatology
🇸🇬Singapore, Singapore
Hospital Médica Tec 100
🇲🇽Queretaro, Mexico
Instituto Neurologia Bs As
🇦🇷Ciudad Autonoma de Bs As, Argentina
University of California DAVIS Medical Center; M.I.N.D. Institute, Section of Developmental Behavior
🇺🇸Sacramento, California, United States
CHU de Saint Etienne; Service de Génétique
🇫🇷St Etienne, France
Groupement Hospitalier Est-Hopital Femme Mere enfant/Hospice civils de lyon
🇫🇷Bron, France
Hospital Universitario de la Princesa; Medicina Interna
🇪🇸Madrid, Spain
Auckland Clinical Studies
🇳🇿Auckland, New Zealand
University of Otago; Psychological Medicine Department
🇳🇿Dunedin, New Zealand
Fundación SÃndrome de Down; Fundación SÃndrome de Down
🇪🇸Madrid, Spain
UVaMID Hospital Santa Caterina;; Servicio de NeurologÃa
🇪🇸Salt, Girona, Spain
Hospital Infantil Universitario Niño Jesus; Pediatria Social
🇪🇸Madrid, Spain
FLENI
🇦🇷Caba, Argentina
University of Wisconsin Madison, Waisman Center
🇺🇸Madison, Wisconsin, United States
Ospedale Pediatrico Bambino Gesù
🇮🇹Roma, Lazio, Italy
IMIM, Human Pharmacology and Clinical Neurosciences,
🇪🇸Barcelona, Spain
Ospedale Microcitemico; Clinica Pediatrica
🇮🇹Cagliari, Sardegna, Italy
Cornwall Partnership NHS Foundation Trust
🇬🇧Redruth, United Kingdom
Mental Health of Learning Disability, Kent & Medway NHS and Social Care Partnership Trust
🇬🇧Dartford, Kent, United Kingdom
Complejo Hospitalario Universitario de Santiago (CHUS); Area Asistencial Integrada de PediatrÃa
🇪🇸Santiago de Compostela, La Coruña, Spain
Blackpool Teaching Hospitals NHS Foundation Trust; Child Development and Family Support Centre
🇬🇧Blackpool, United Kingdom
Doncaster and Bassetlaw Hospitals NHS Foundation Trust; Doncaster Royal Infirmary
🇬🇧Doncaster, United Kingdom
Wellington Hospital Research Office
🇳🇿Wellington, New Zealand
Emory University School of Medicine; Department of Human Genetics & Pediatrics
🇺🇸Decatur, Georgia, United States
University of Utah School of Medicine; Department of Pediatrics
🇺🇸Salt Lake City, Utah, United States
Johns Hopkins Hospital.
🇺🇸Baltimore, Maryland, United States
Massachusette General Hospital; Medical Genetics
🇺🇸Boston, Massachusetts, United States
Duke Clin Rsch Institute
🇺🇸Durham, North Carolina, United States
Univ of CA San Diego; Neurosciences Comp.Alzheimer's
🇺🇸La Jolla, California, United States
CHU de Montpellier Hopital Arnaud de Villeneuve; de Génétique
🇫🇷Montpellier, France
Hospital Dr. Angel Leaño; Pediatria
🇲🇽Guadalajara, Jalisco, Mexico
Rush University Medical Center
🇺🇸Chicago, Illinois, United States
True North Clinical Research Kentville
🇨🇦Kentville, Nova Scotia, Canada
Institut Jérôme Lejeune; Neuropsychology
🇫🇷Paris, France