A Study of RO5186582 in Down Syndrome Among Children 6 to 11 Years of Age

NCT ID: NCT02484703

Last Updated: 2017-07-06

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 45 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-10-28
• Study Completion Date: 2016-08-03

Brief Summary

This study will evaluate the safety, tolerability, efficacy, and pharmacokinetic and pharmacodynamic activity of 3 different dosages of RO5186582 compared with placebo. A total of approximately 46 participants will be enrolled, in order to have at least 32 evaluable, and will be randomly assigned to 1 of 4 treatments in a 1:1:1:1 ratio, with 9 children per treatment arm. The target ratio between 6-8 years and 9-11 years age groups is approximately 1:1 in each treatment arm, with a minimum of 3 children per age group in each treatment arm.

Conditions

Down Syndrome

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

Placebo

Participants will receive matching placebo by mouth (PO) twice daily (BID) for up to 26 weeks.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Participants will receive matching placebo PO BID. Study medication will first be administered on Day 1, and only the morning dose will be given on the last day of treatment (Week 26).

RO5186582 120 mg BID

Participants will receive RO5186582 at a dosage of 120 milligrams (mg) PO BID for up to 26 weeks.

Group Type: EXPERIMENTAL

RO5186582

Intervention Type: DRUG

Participants will receive 1 of 3 dosages of RO5186582 PO BID, including 40 mg, 60 mg, or 120 mg. Study medication will first be administered on Day 1, and only the morning dose will be given on the last day of treatment (Week 26).

RO5186582 40 mg BID

Participants will receive RO5186582 at a dosage of 40 mg PO BID for up to 26 weeks.

Group Type: EXPERIMENTAL

RO5186582

Intervention Type: DRUG

Participants will receive 1 of 3 dosages of RO5186582 PO BID, including 40 mg, 60 mg, or 120 mg. Study medication will first be administered on Day 1, and only the morning dose will be given on the last day of treatment (Week 26).

RO5186582 60 mg BID

Participants will receive RO5186582 at a dosage of 60 mg PO BID for up to 26 weeks.

Group Type: EXPERIMENTAL

RO5186582

Intervention Type: DRUG

Participants will receive 1 of 3 dosages of RO5186582 PO BID, including 40 mg, 60 mg, or 120 mg. Study medication will first be administered on Day 1, and only the morning dose will be given on the last day of treatment (Week 26).

Interventions

Placebo

Participants will receive matching placebo PO BID. Study medication will first be administered on Day 1, and only the morning dose will be given on the last day of treatment (Week 26).

Intervention Type: DRUG

RO5186582

Participants will receive 1 of 3 dosages of RO5186582 PO BID, including 40 mg, 60 mg, or 120 mg. Study medication will first be administered on Day 1, and only the morning dose will be given on the last day of treatment (Week 26).

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Diagnosis of Down syndrome, except for mosaic Down syndrome
• Available parent or caregiver to attend clinic visits and provide information about the participant's behavior and symptoms

Exclusion Criteria

• Any primary psychiatric comorbid disorder
• History of infantile spasms, West syndrome, Lennox-Gastaut syndrome, early infantile epileptic encephalopathy, treatment-refractory epilepsy with cognitive/developmental regression, severe head trauma, or central nervous system (CNS) infection
• Seizure event of any type within 12 months prior to Screening or relevant changes in anti-epileptic drugs 6 weeks prior to enrollment
• Significant sleep disruption
• Significant gastrointestinal, renal, hepatic, endocrine, or cardiovascular disease
• New-onset or ongoing hematologic/oncologic disorder
• Severe lactose intolerance
• Participation in another clinical study within 1 month or 6 half-lives prior to first dose, or any extent of participation in Study BP29589 (NCT02451657)

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 11 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Hoffmann-La Roche

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Trials

Role: STUDY_DIRECTOR

Hoffmann-La Roche

Locations

Southwest Autism Research & Resource Center

Phoenix, Arizona, United States

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

Decatur, Georgia, United States

Rush University Medical Center

Chicago, Illinois, United States

Massachusette General Hospital; Medical Genetics

Boston, Massachusetts, United States

Boston Children's Hospital, Department of Neurology

Boston, Massachusetts, United States

Duke Clin Rsch Institute

Durham, North Carolina, United States

UPMC Western Psychiatric Institute and Clinic

Pittsburgh, Pennsylvania, United States

Vanderbilt University Medical Center

Nashville, Tennessee, United States

University of Wisconsin Hospital and Clinics

Madison, Wisconsin, United States

Countries

United States

Other Identifiers

WP28760

Identifier Type: -

Identifier Source: org_study_id