A Study of RG1662 in Adults and Adolescents With Down Syndrome (CLEMATIS)

Down Syndrome Clinical Trial

Official title:

A MULTI-CENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED PHASE 2 STUDY OF THE EFFICACY, SAFETY AND TOLERABILITY OF RG1662 IN ADULTS AND ADOLESCENTS WITH DOWN SYNDROME (CLEMATIS)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02024789
• Other study ID #: BP27832
• Secondary ID: 2013-001263-23
• Status: Completed
• Phase: Phase 2
• First received: December 27, 2013
• Last updated: October 24, 2017
• Start date: May 5, 2014
• Est. completion date: May 4, 2016

Study information

• Verified date: October 2017
• Source: Hoffmann-La Roche
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 173
• Est. completion date: May 4, 2016
• Est. primary completion date: May 4, 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 30 Years

Eligibility

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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

Related Conditions & MeSH terms

• Down Syndrome
• Syndrome

Intervention

Drug:
• Placebo
Orally twice daily, 26 weeks
• RG1662
120 mg (80 mg for subjects 12 and 13 years of age) orally twice daily, 26 weeks
• RG1662
240 mg (160 mg for subjects 12 and 13 years of age) orally twice daily, 26 weeks

Locations

Country	Name	City	State
Argentina	FLENI	Caba
Argentina	Instituto Neurologia Bs As	Ciudad Autonoma de Bs As
Canada	True North Clinical Research Kentville	Kentville	Nova Scotia
France	Groupement Hospitalier Est-Hopital Femme Mere enfant/Hospice civils de lyon	Bron
France	CHU de Montpellier Hopital Arnaud de Villeneuve; de Génétique	Montpellier
France	Institut Jérôme Lejeune; Neuropsychology	Paris
France	CHU de Saint Etienne; Service de Génétique	St Etienne
Italy	Ospedale Microcitemico; Clinica Pediatrica	Cagliari	Sardegna
Italy	Ospedale Pediatrico Bambino Gesù	Roma	Lazio
Italy	Policlinico Universitario "Agostino Gemelli";Dip. Tutela Salute Donna Bambino Adolescente	Roma	Lazio
Mexico	Clínica Para la Atención del Neurodesarrollo	Aguascalientes
Mexico	Hospital Dr. Angel Leaño; Pediatria	Guadalajara	Jalisco
Mexico	Hospital Universitario Dr. Jose Eleuterio Gonzalez; Pediatria	Monterrey
Mexico	Hospital Médica Tec 100	Queretaro
New Zealand	Auckland Clinical Studies	Auckland
New Zealand	University of Otago; Psychological Medicine Department	Dunedin
New Zealand	Wellington Hospital Research Office	Wellington
Singapore	KK Women's and Children's Hospital; Department of Neonatology	Singapore
Spain	IMIM, Human Pharmacology and Clinical Neurosciences,	Barcelona
Spain	Fundación Síndrome de Down; Fundación Síndrome de Down	Madrid
Spain	Hospital Infantil Universitario Niño Jesus; Pediatria Social	Madrid
Spain	Hospital Universitario de la Princesa; Medicina Interna	Madrid
Spain	UVaMID Hospital Santa Caterina;; Servicio de Neurología	Salt	Girona
Spain	Complejo Hospitalario Universitario de Santiago (CHUS); Area Asistencial Integrada de Pediatría	Santiago de Compostela	La Coruña
United Kingdom	Blackpool Teaching Hospitals NHS Foundation Trust; Child Development and Family Support Centre	Blackpool
United Kingdom	Mental Health of Learning Disability, Kent & Medway NHS and Social Care Partnership Trust	Dartford, Kent
United Kingdom	Doncaster and Bassetlaw Hospitals NHS Foundation Trust; Doncaster Royal Infirmary	Doncaster
United Kingdom	Cornwall Partnership NHS Foundation Trust	Redruth
United States	Johns Hopkins Hospital.	Baltimore	Maryland
United States	Massachusette General Hospital; Medical Genetics	Boston	Massachusetts
United States	Rush University Medical Center	Chicago	Illinois
United States	Emory University School of Medicine; Department of Human Genetics & Pediatrics	Decatur	Georgia
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	University of Wisconsin Madison, Waisman Center	Madison	Wisconsin
United States	University of California DAVIS Medical Center; M.I.N.D. Institute, Section of Developmental Behavior	Sacramento	California
United States	University of Utah School of Medicine; Department of Pediatrics	Salt Lake City	Utah

Sponsors (1)

Lead Sponsor	Collaborator
Hoffmann-La Roche

Countries where clinical trial is conducted

United States,  Argentina,  Canada,  France,  Italy,  Mexico,  New Zealand,  Singapore,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Cognition as assessed by the Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) sub-tests		26 weeks
Primary	Adaptive behavior as assessed by the Vineland Adaptive Behavior Scales-II (VABS-II) standard scores		26 weeks
Primary	Clinical global impression as assessed by Clinician Rated Global Improvement (CGI-I) scale		26 weeks
Secondary	Incidence of abnormal ECG changes		26 weeks
Secondary	Abnormal ECG changes in adolescents as compared to baseline		from baseline to Week 26
Secondary	Safety: Incidence of adverse events		approximately 32 weeks
Secondary	Incidence of abnormal blood pressure		26 weeks
Secondary	RG1662 plasma concentrations		26 weeks