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

Primary Purpose

Down Syndrome

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Placebo
RG1662
RG1662
Sponsored by
Hoffmann-La Roche
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Down Syndrome

Eligibility Criteria

12 Years - 30 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Univ of CA San Diego; Neurosciences Comp.Alzheimer's
  • University of California DAVIS Medical Center; M.I.N.D. Institute, Section of Developmental Behavior
  • Emory University School of Medicine; Department of Human Genetics & Pediatrics
  • Rush University Medical Center
  • Johns Hopkins Hospital.
  • Massachusette General Hospital; Medical Genetics
  • Duke Clin Rsch Institute
  • University of Utah School of Medicine; Department of Pediatrics
  • University of Wisconsin Madison, Waisman Center
  • FLENI
  • Instituto Neurologia Bs As
  • True North Clinical Research Kentville
  • Groupement Hospitalier Est-Hopital Femme Mere enfant/Hospice civils de lyon
  • CHU de Montpellier Hopital Arnaud de Villeneuve; de Génétique
  • Institut Jérôme Lejeune; Neuropsychology
  • CHU de Saint Etienne; Service de Génétique
  • Ospedale Pediatrico Bambino Gesù
  • Policlinico Universitario "Agostino Gemelli";Dip. Tutela Salute Donna Bambino Adolescente
  • Ospedale Microcitemico; Clinica Pediatrica
  • Hospital Dr. Angel Leaño; Pediatria
  • Clínica Para la Atención del Neurodesarrollo
  • Hospital Universitario Dr. Jose Eleuterio Gonzalez; Pediatria
  • Hospital Médica Tec 100
  • Auckland Clinical Studies
  • University of Otago; Psychological Medicine Department
  • Wellington Hospital Research Office
  • KK Women's and Children's Hospital; Department of Neonatology
  • UVaMID Hospital Santa Caterina;; Servicio de Neurología
  • Complejo Hospitalario Universitario de Santiago (CHUS); Area Asistencial Integrada de Pediatría
  • IMIM, Human Pharmacology and Clinical Neurosciences,
  • Hospital Universitario de la Princesa; Medicina Interna
  • Hospital Infantil Universitario Niño Jesus; Pediatria Social
  • Fundación Síndrome de Down; Fundación Síndrome de Down
  • Blackpool Teaching Hospitals NHS Foundation Trust; Child Development and Family Support Centre
  • Mental Health of Learning Disability, Kent & Medway NHS and Social Care Partnership Trust
  • Doncaster and Bassetlaw Hospitals NHS Foundation Trust; Doncaster Royal Infirmary
  • Cornwall Partnership NHS Foundation Trust

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Placebo Comparator

Experimental

Experimental

Arm Label

Placebo

RG1662 120 mg bid

RG1662 240 mg bid

Arm Description

Outcomes

Primary Outcome Measures

Cognition as assessed by the Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) sub-tests
Adaptive behavior as assessed by the Vineland Adaptive Behavior Scales-II (VABS-II) standard scores
Clinical global impression as assessed by Clinician Rated Global Improvement (CGI-I) scale

Secondary Outcome Measures

Incidence of abnormal ECG changes
Abnormal ECG changes in adolescents as compared to baseline
Safety: Incidence of adverse events
Incidence of abnormal blood pressure
RG1662 plasma concentrations

Full Information

First Posted
December 27, 2013
Last Updated
October 24, 2017
Sponsor
Hoffmann-La Roche
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1. Study Identification

Unique Protocol Identification Number
NCT02024789
Brief Title
A Study of RG1662 in Adults and Adolescents With Down Syndrome (CLEMATIS)
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)
Study Type
Interventional

2. Study Status

Record Verification Date
October 2017
Overall Recruitment Status
Completed
Study Start Date
May 5, 2014 (Actual)
Primary Completion Date
May 4, 2016 (Actual)
Study Completion Date
May 4, 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Hoffmann-La Roche

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Down Syndrome

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
173 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Title
RG1662 120 mg bid
Arm Type
Experimental
Arm Title
RG1662 240 mg bid
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Orally twice daily, 26 weeks
Intervention Type
Drug
Intervention Name(s)
RG1662
Intervention Description
120 mg (80 mg for subjects 12 and 13 years of age) orally twice daily, 26 weeks
Intervention Type
Drug
Intervention Name(s)
RG1662
Intervention Description
240 mg (160 mg for subjects 12 and 13 years of age) orally twice daily, 26 weeks
Primary Outcome Measure Information:
Title
Cognition as assessed by the Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) sub-tests
Time Frame
26 weeks
Title
Adaptive behavior as assessed by the Vineland Adaptive Behavior Scales-II (VABS-II) standard scores
Time Frame
26 weeks
Title
Clinical global impression as assessed by Clinician Rated Global Improvement (CGI-I) scale
Time Frame
26 weeks
Secondary Outcome Measure Information:
Title
Incidence of abnormal ECG changes
Time Frame
26 weeks
Title
Abnormal ECG changes in adolescents as compared to baseline
Time Frame
from baseline to Week 26
Title
Safety: Incidence of adverse events
Time Frame
approximately 32 weeks
Title
Incidence of abnormal blood pressure
Time Frame
26 weeks
Title
RG1662 plasma concentrations
Time Frame
26 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Maximum Age & Unit of Time
30 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Trials
Organizational Affiliation
Hoffmann-La Roche
Official's Role
Study Director
Facility Information:
Facility Name
Univ of CA San Diego; Neurosciences Comp.Alzheimer's
City
La Jolla
State/Province
California
ZIP/Postal Code
92037
Country
United States
Facility Name
University of California DAVIS Medical Center; M.I.N.D. Institute, Section of Developmental Behavior
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
Emory University School of Medicine; Department of Human Genetics & Pediatrics
City
Decatur
State/Province
Georgia
ZIP/Postal Code
30033
Country
United States
Facility Name
Rush University Medical Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Johns Hopkins Hospital.
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
Massachusette General Hospital; Medical Genetics
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Duke Clin Rsch Institute
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
University of Utah School of Medicine; Department of Pediatrics
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84112
Country
United States
Facility Name
University of Wisconsin Madison, Waisman Center
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53705
Country
United States
Facility Name
FLENI
City
Caba
ZIP/Postal Code
C1428AQK
Country
Argentina
Facility Name
Instituto Neurologia Bs As
City
Ciudad Autonoma de Bs As
ZIP/Postal Code
1426
Country
Argentina
Facility Name
True North Clinical Research Kentville
City
Kentville
State/Province
Nova Scotia
ZIP/Postal Code
B4N 4K9
Country
Canada
Facility Name
Groupement Hospitalier Est-Hopital Femme Mere enfant/Hospice civils de lyon
City
Bron
ZIP/Postal Code
69003
Country
France
Facility Name
CHU de Montpellier Hopital Arnaud de Villeneuve; de Génétique
City
Montpellier
ZIP/Postal Code
34295
Country
France
Facility Name
Institut Jérôme Lejeune; Neuropsychology
City
Paris
ZIP/Postal Code
75015
Country
France
Facility Name
CHU de Saint Etienne; Service de Génétique
City
St Etienne
ZIP/Postal Code
42055
Country
France
Facility Name
Ospedale Pediatrico Bambino Gesù
City
Roma
State/Province
Lazio
ZIP/Postal Code
00165
Country
Italy
Facility Name
Policlinico Universitario "Agostino Gemelli";Dip. Tutela Salute Donna Bambino Adolescente
City
Roma
State/Province
Lazio
ZIP/Postal Code
00168
Country
Italy
Facility Name
Ospedale Microcitemico; Clinica Pediatrica
City
Cagliari
State/Province
Sardegna
ZIP/Postal Code
09121
Country
Italy
Facility Name
Hospital Dr. Angel Leaño; Pediatria
City
Guadalajara
State/Province
Jalisco
ZIP/Postal Code
45200
Country
Mexico
Facility Name
Clínica Para la Atención del Neurodesarrollo
City
Aguascalientes
ZIP/Postal Code
20030
Country
Mexico
Facility Name
Hospital Universitario Dr. Jose Eleuterio Gonzalez; Pediatria
City
Monterrey
ZIP/Postal Code
64460
Country
Mexico
Facility Name
Hospital Médica Tec 100
City
Queretaro
ZIP/Postal Code
76000
Country
Mexico
Facility Name
Auckland Clinical Studies
City
Auckland
ZIP/Postal Code
1142
Country
New Zealand
Facility Name
University of Otago; Psychological Medicine Department
City
Dunedin
ZIP/Postal Code
9016
Country
New Zealand
Facility Name
Wellington Hospital Research Office
City
Wellington
ZIP/Postal Code
6021
Country
New Zealand
Facility Name
KK Women's and Children's Hospital; Department of Neonatology
City
Singapore
ZIP/Postal Code
229899
Country
Singapore
Facility Name
UVaMID Hospital Santa Caterina;; Servicio de Neurología
City
Salt
State/Province
Girona
ZIP/Postal Code
17090
Country
Spain
Facility Name
Complejo Hospitalario Universitario de Santiago (CHUS); Area Asistencial Integrada de Pediatría
City
Santiago de Compostela
State/Province
La Coruña
ZIP/Postal Code
15706
Country
Spain
Facility Name
IMIM, Human Pharmacology and Clinical Neurosciences,
City
Barcelona
ZIP/Postal Code
08009
Country
Spain
Facility Name
Hospital Universitario de la Princesa; Medicina Interna
City
Madrid
ZIP/Postal Code
28006
Country
Spain
Facility Name
Hospital Infantil Universitario Niño Jesus; Pediatria Social
City
Madrid
ZIP/Postal Code
28009
Country
Spain
Facility Name
Fundación Síndrome de Down; Fundación Síndrome de Down
City
Madrid
ZIP/Postal Code
28016
Country
Spain
Facility Name
Blackpool Teaching Hospitals NHS Foundation Trust; Child Development and Family Support Centre
City
Blackpool
Country
United Kingdom
Facility Name
Mental Health of Learning Disability, Kent & Medway NHS and Social Care Partnership Trust
City
Dartford, Kent
ZIP/Postal Code
DA2 6PB
Country
United Kingdom
Facility Name
Doncaster and Bassetlaw Hospitals NHS Foundation Trust; Doncaster Royal Infirmary
City
Doncaster
ZIP/Postal Code
DN2 5LT
Country
United Kingdom
Facility Name
Cornwall Partnership NHS Foundation Trust
City
Redruth
ZIP/Postal Code
TR15 2SP
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
35123401
Citation
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.
Results Reference
derived

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

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