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

NCT ID: NCT02024789

Last Updated: 2017-10-25

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

173 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-05-05

Study Completion Date

2016-05-04

Brief Summary

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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

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Conditions

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Down Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Orally twice daily, 26 weeks

RG1662 120 mg bid

Group Type EXPERIMENTAL

RG1662

Intervention Type DRUG

120 mg (80 mg for subjects 12 and 13 years of age) orally twice daily, 26 weeks

RG1662 240 mg bid

Group Type EXPERIMENTAL

RG1662

Intervention Type DRUG

240 mg (160 mg for subjects 12 and 13 years of age) orally twice daily, 26 weeks

Interventions

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Placebo

Orally twice daily, 26 weeks

Intervention Type DRUG

RG1662

120 mg (80 mg for subjects 12 and 13 years of age) orally twice daily, 26 weeks

Intervention Type DRUG

RG1662

240 mg (160 mg for subjects 12 and 13 years of age) orally twice daily, 26 weeks

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

12 Years

Maximum Eligible Age

30 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Hoffmann-La Roche

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Trials

Role: STUDY_DIRECTOR

Hoffmann-La Roche

Locations

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Univ of CA San Diego; Neurosciences Comp.Alzheimer's

La Jolla, California, United States

Site Status

University of California DAVIS Medical Center; M.I.N.D. Institute, Section of Developmental Behavior

Sacramento, California, United States

Site Status

Emory University School of Medicine; Department of Human Genetics & Pediatrics

Decatur, Georgia, United States

Site Status

Rush University Medical Center

Chicago, Illinois, United States

Site Status

Johns Hopkins Hospital.

Baltimore, Maryland, United States

Site Status

Massachusette General Hospital; Medical Genetics

Boston, Massachusetts, United States

Site Status

Duke Clin Rsch Institute

Durham, North Carolina, United States

Site Status

University of Utah School of Medicine; Department of Pediatrics

Salt Lake City, Utah, United States

Site Status

University of Wisconsin Madison, Waisman Center

Madison, Wisconsin, United States

Site Status

FLENI

CABA, , Argentina

Site Status

Instituto Neurologia Bs As

Ciudad Autonoma de Bs As, , Argentina

Site Status

True North Clinical Research Kentville

Kentville, Nova Scotia, Canada

Site Status

Groupement Hospitalier Est-Hopital Femme Mere enfant/Hospice civils de lyon

Bron, , France

Site Status

CHU de Montpellier Hopital Arnaud de Villeneuve; de Génétique

Montpellier, , France

Site Status

Institut Jérôme Lejeune; Neuropsychology

Paris, , France

Site Status

CHU de Saint Etienne; Service de Génétique

Saint-Etienne, , France

Site Status

Ospedale Pediatrico Bambino Gesù

Rome, Lazio, Italy

Site Status

Policlinico Universitario "Agostino Gemelli";Dip. Tutela Salute Donna Bambino Adolescente

Rome, Lazio, Italy

Site Status

Ospedale Microcitemico; Clinica Pediatrica

Cagliari, Sardinia, Italy

Site Status

Hospital Dr. Angel Leaño; Pediatria

Guadalajara, Jalisco, Mexico

Site Status

Clínica Para la Atención del Neurodesarrollo

Aguascalientes, , Mexico

Site Status

Hospital Universitario Dr. Jose Eleuterio Gonzalez; Pediatria

Monterrey, , Mexico

Site Status

Hospital Médica Tec 100

Querétaro, , Mexico

Site Status

Auckland Clinical Studies

Auckland, , New Zealand

Site Status

University of Otago; Psychological Medicine Department

Dunedin, , New Zealand

Site Status

Wellington Hospital Research Office

Wellington, , New Zealand

Site Status

KK Women's and Children's Hospital; Department of Neonatology

Singapore, , Singapore

Site Status

UVaMID Hospital Santa Caterina;; Servicio de Neurología

Salt, Girona, Spain

Site Status

Complejo Hospitalario Universitario de Santiago (CHUS); Area Asistencial Integrada de Pediatría

Santiago de Compostela, La Coruña, Spain

Site Status

IMIM, Human Pharmacology and Clinical Neurosciences,

Barcelona, , Spain

Site Status

Hospital Universitario de la Princesa; Medicina Interna

Madrid, , Spain

Site Status

Hospital Infantil Universitario Niño Jesus; Pediatria Social

Madrid, , Spain

Site Status

Fundación Síndrome de Down; Fundación Síndrome de Down

Madrid, , Spain

Site Status

Blackpool Teaching Hospitals NHS Foundation Trust; Child Development and Family Support Centre

Blackpool, , United Kingdom

Site Status

Mental Health of Learning Disability, Kent & Medway NHS and Social Care Partnership Trust

Dartford, Kent, , United Kingdom

Site Status

Doncaster and Bassetlaw Hospitals NHS Foundation Trust; Doncaster Royal Infirmary

Doncaster, , United Kingdom

Site Status

Cornwall Partnership NHS Foundation Trust

Redruth, , United Kingdom

Site Status

Countries

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

References

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Goeldner C, Kishnani PS, Skotko BG, Casero JL, Hipp JF, Derks M, Hernandez MC, Khwaja O, Lennon-Chrimes S, Noeldeke J, Pellicer S, Squassante L, Visootsak J, Wandel C, Fontoura P, d'Ardhuy XL; Clematis Study Group. A randomized, double-blind, placebo-controlled phase II trial to explore the effects of a GABAA-alpha5 NAM (basmisanil) on intellectual disability associated with Down syndrome. J Neurodev Disord. 2022 Feb 5;14(1):10. doi: 10.1186/s11689-022-09418-0.

Reference Type DERIVED
PMID: 35123401 (View on PubMed)

Other Identifiers

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2013-001263-23

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

BP27832

Identifier Type: -

Identifier Source: org_study_id