Down SyndromePhase 2NCT02024789
A Study of RG1662 in Adults and Adolescents With Down Syndrome (CLEMATIS)
A Phase 2 study testing RG1662 in people with Down Syndrome, run by Hoffmann-La Roche at 37 sites in 10 countries.
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. Read more about this study
- Who is it for?
- Ages 12 to 30 years
- Conditions the study is for
- Down Syndrome
- What is being tested?
- RG1662
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 173 participants
- Where
- 37 sitesin 10 countries
- Study dates
- 5 May 2014to 4 May 2016
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
37 sites in 10 countries.
37 sites in 10 countries
United States9 sites
Johns Hopkins Hospital.
Baltimore, Maryland, 21287
Massachusette General Hospital; Medical Genetics
Boston, Massachusetts, 02114
Rush University Medical Center
Chicago, Illinois, 60612
Emory University School of Medicine; Department of Human Genetics & Pediatrics
Decatur, Georgia, 30033
Duke Clin Rsch Institute
Durham, North Carolina, 27710
Univ of CA San Diego; Neurosciences Comp.Alzheimer's
La Jolla, California, 92037
University of Wisconsin Madison, Waisman Center
Madison, Wisconsin, 53705
University of California DAVIS Medical Center; M.I.N.D. Institute, Section of Developmental Behavior
Sacramento, California, 95817
University of Utah School of Medicine; Department of Pediatrics
Salt Lake City, Utah, 84112
Spain6 sites
IMIM, Human Pharmacology and Clinical Neurosciences,
Barcelona, 08009
Hospital Universitario de la Princesa; Medicina Interna
Madrid, 28006
Hospital Infantil Universitario Niño Jesus; Pediatria Social
Madrid, 28009
Fundación Síndrome de Down; Fundación Síndrome de Down
Madrid, 28016
UVaMID Hospital Santa Caterina;; Servicio de Neurología
Salt, Girona, 17090
Complejo Hospitalario Universitario de Santiago (CHUS); Area Asistencial Integrada de Pediatría
Santiago de Compostela, La Coruña, 15706
France4 sites
Groupement Hospitalier Est-Hopital Femme Mere enfant/Hospice civils de lyon
Bron, 69003
CHU de Montpellier Hopital Arnaud de Villeneuve; de Génétique
Montpellier, 34295
Institut Jérôme Lejeune; Neuropsychology
Paris, 75015
CHU de Saint Etienne; Service de Génétique
Saint-Etienne, 42055
Mexico4 sites
Clínica Para la Atención del Neurodesarrollo
Aguascalientes, 20030
Hospital Dr. Angel Leaño; Pediatria
Guadalajara, Jalisco, 45200
Hospital Universitario Dr. Jose Eleuterio Gonzalez; Pediatria
Monterrey, 64460
Hospital Médica Tec 100
Querétaro, 76000
United Kingdom4 sites
Blackpool Teaching Hospitals NHS Foundation Trust; Child Development and Family Support Centre
Blackpool
Mental Health of Learning Disability, Kent & Medway NHS and Social Care Partnership Trust
Dartford, Kent, DA2 6PB
Doncaster and Bassetlaw Hospitals NHS Foundation Trust; Doncaster Royal Infirmary
Doncaster, DN2 5LT
Cornwall Partnership NHS Foundation Trust
Redruth, TR15 2SP
Italy3 sites
Ospedale Microcitemico; Clinica Pediatrica
Cagliari, Sardinia, 09121
Ospedale Pediatrico Bambino Gesù
Rome, Lazio, 00165
Policlinico Universitario "Agostino Gemelli";Dip. Tutela Salute Donna Bambino Adolescente
Rome, Lazio, 00168
New Zealand3 sites
Auckland Clinical Studies
Auckland, 1142
University of Otago; Psychological Medicine Department
Dunedin, 9016
Wellington Hospital Research Office
Wellington, 6021
Argentina2 sites
FLENI
CABA, C1428AQK
Instituto Neurologia Bs As
Ciudad Autonoma de Bs As, 1426
Canada1 site
True North Clinical Research Kentville
Kentville, Nova Scotia, B4N 4K9
Singapore1 site
KK Women's and Children's Hospital; Department of Neonatology
Singapore, 229899
About this study
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)
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.
- Conditions
- Down Syndrome
- Being tested
- Placebo, RG1662
- Medical subject headings
- Down Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlaceboDrug
Orally twice daily, 26 weeks
RG1662Drug
120 mg (80 mg for subjects 12 and 13 years of age) orally twice daily, 26 weeks
RG1662Drug
240 mg (160 mg for subjects 12 and 13 years of age) orally twice daily, 26 weeks
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
| RG1662 120 mg bid | Experimental | Not described Receives: Drug: RG1662 |
| RG1662 240 mg bid | Experimental | Not described Receives: Drug: RG1662 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cognition as assessed by the Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) sub-tests
Measured: 26 weeks
Adaptive behavior as assessed by the Vineland Adaptive Behavior Scales-II (VABS-II) standard scores
Measured: 26 weeks
Clinical global impression as assessed by Clinician Rated Global Improvement (CGI-I) scale
Measured: 26 weeks
Secondary outcomes (5)
Incidence of abnormal ECG changes
Measured: 26 weeks
Abnormal ECG changes in adolescents as compared to baseline
Measured: from baseline to Week 26
Safety: Incidence of adverse events
Measured: approximately 32 weeks
Incidence of abnormal blood pressure
Measured: 26 weeks
RG1662 plasma concentrations
Measured: 26 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Dec 2013 (estimated)
- Study start5 May 2014
- Primary completion4 May 2016
- Study completion4 May 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Down SyndromeStudies of the same condition, open and under way.
- More trials run by Hoffmann-La RocheThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 173 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02024789
- Sponsor's protocol ID
- BP27832
- Other IDs
- 2013-001263-23
- Status last verified
- Oct 2017
- First submitted
- 27 Dec 2013
- Last updated
- 25 Oct 2017
- Results posted
- No
Publications
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.
PubMed 35123401
Study at a glance
From the registry record, last updated by the sponsor on 25 Oct 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT02024789
- Acronym / sponsor ID
- BP27832
- Conditions
- Down Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 173 participants (actual)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Study start
- 5 May 2014 (actual)
- Primary completion
- 4 May 2016 (actual)
- Study completion
- 4 May 2016 (actual)
- First posted
- 31 Dec 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: Placebo |
| RG1662 120 mg bid | Experimental | Drug: RG1662 |
| RG1662 240 mg bid | Experimental | Drug: RG1662 |
Who may qualify
- Ages
- 12 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PlaceboOrally twice daily, 26 weeks | Drug | Placebo |
| RG1662120 mg (80 mg for subjects 12 and 13 years of age) orally twice daily, 26 weeks | Drug | RG1662 120 mg bid |
| RG1662240 mg (160 mg for subjects 12 and 13 years of age) orally twice daily, 26 weeks | Drug | RG1662 240 mg bid |
Primary outcome
Cognition as assessed by the Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) sub-tests
Time frame: 26 weeks
Adaptive behavior as assessed by the Vineland Adaptive Behavior Scales-II (VABS-II) standard scores
Time frame: 26 weeks
Clinical global impression as assessed by Clinician Rated Global Improvement (CGI-I) scale
Time frame: 26 weeks
5 secondary outcomes
Incidence of abnormal ECG changes
Time frame: 26 weeks
Abnormal ECG changes in adolescents as compared to baseline
Time frame: from baseline to Week 26
Safety: Incidence of adverse events
Time frame: approximately 32 weeks
Incidence of abnormal blood pressure
Time frame: 26 weeks
RG1662 plasma concentrations
Time frame: 26 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Oct 2017.
- Derived from this study: PubMed 35123401Goeldner 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.
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Down Syndrome
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trials study director
Hoffmann-La Roche
37 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing RG1662 in people with Down Syndrome, run by Hoffmann-La Roche at 37 sites in 10 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Oct 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 37 sites in United States, Spain, France, Mexico, United Kingdom and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cognition as assessed by the Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) sub-tests (time frame: 26 weeks). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02024789, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Oct 2017.