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

• ClinicalTrials.gov Identifier: NCT02024789
• Recruitment Status: Completed
• First Posted: December 31, 2013
• Last Update Posted: October 25, 2017
• Sponsor: Hoffmann-La Roche
• Information provided by (Responsible Party): Hoffmann-La Roche

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.

Condition or disease	Intervention/treatment	Phase
Down Syndrome	Drug: Placebo; Drug: RG1662	Phase 2

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 173 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Double (Participant, Investigator)
• Primary Purpose: Treatment
• 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)
• Actual Study Start Date: May 5, 2014
• Actual Primary Completion Date: May 4, 2016
• Actual Study Completion Date: May 4, 2016

Arms and Interventions

Arm	Intervention/treatment
Placebo Comparator: Placebo	Drug: Placebo; Orally twice daily, 26 weeks
Experimental: RG1662 120 mg bid	Drug: RG1662; 120 mg (80 mg for subjects 12 and 13 years of age) orally twice daily, 26 weeks
Experimental: RG1662 240 mg bid	Drug: RG1662; 240 mg (160 mg for subjects 12 and 13 years of age) orally twice daily, 26 weeks

Outcome Measures

Primary Outcome Measures :

1. Cognition as assessed by the Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) sub-tests [ Time Frame: 26 weeks ]
2. Adaptive behavior as assessed by the Vineland Adaptive Behavior Scales-II (VABS-II) standard scores [ Time Frame: 26 weeks ]
3. Clinical global impression as assessed by Clinician Rated Global Improvement (CGI-I) scale [ Time Frame: 26 weeks ]

Secondary Outcome Measures :

1. Incidence of abnormal ECG changes [ Time Frame: 26 weeks ]
2. Abnormal ECG changes in adolescents as compared to baseline [ Time Frame: from baseline to Week 26 ]
3. Safety: Incidence of adverse events [ Time Frame: approximately 32 weeks ]
4. Incidence of abnormal blood pressure [ Time Frame: 26 weeks ]
5. RG1662 plasma concentrations [ Time Frame: 26 weeks ]

Eligibility Criteria

• Ages Eligible for Study: 12 Years to 30 Years (Child, Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

  Show 37 Study Locations

Sponsors and Collaborators

Hoffmann-La Roche

Investigators

• Study Director: Clinical Trials; Hoffmann-La Roche

More Information

• Responsible Party: Hoffmann-La Roche
• ClinicalTrials.gov Identifier: NCT02024789 History of Changes
• Other Study ID Numbers: BP27832; 2013-001263-23 ( EudraCT Number )
• First Posted: December 31, 2013
• Last Update Posted: October 25, 2017
• Last Verified: October 2017

Additional relevant MeSH terms:

Down Syndrome; Syndrome; Disease; Pathologic Processes; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn