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

Roche

Status and phase: Terminated; Phase 2

Conditions: Down Syndrome

Treatments: Drug: RO5186582; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT02484703

WP28760

Details and patient eligibility

About

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.

Enrollment: 45 patients

Sex: All

Ages: 6 to 11 years old

Volunteers: No Healthy Volunteers

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)

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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45 participants in 4 patient groups, including a placebo group

Placebo

Placebo Comparator group

Description:

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

Treatment:

Drug: Placebo

RO5186582 120 mg BID

Experimental group

Description:

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

Treatment:

Drug: RO5186582

RO5186582 40 mg BID

Experimental group

Description:

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

Treatment:

Drug: RO5186582

RO5186582 60 mg BID

Experimental group

Description:

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

Treatment:

Drug: RO5186582