A 4-Week Safety Study of Oral ELND005 in Young Adults With Down Syndrome Without Dementia (DS201)

OPKO Health

Status and phase: Completed; Phase 2

Conditions: Down Syndrome

Treatments: Drug: Placebo; Drug: ELND005

Study type: Interventional

Funder types: Industry

Identifiers

NCT01791725

ELND005-DS201

Details and patient eligibility

About

This is a prospective, randomized, double-blind, placebo-controlled, parallel-group, three-arm, multicenter study of the safety and PK of ELND005 administered orally for 4 weeks. This study will enroll Down Syndrome patients 18 to 45 years of age (inclusive) without dementia.

Enrollment: 23 patients

Sex: All

Ages: 18 to 45 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 18 - 45 years of age
• Has an IQ of > 40 (K-BIT)
• Able and willing to have a brain MRI

Exclusion criteria

• Symptoms of dementia or worsening cognition over the past year.
• Has a history of hepatitis B, hepatitis C, or HIV

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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23 participants in 3 patient groups, including a placebo group

ELND005 BID

Experimental group

Description:

ELND005 250 mg BID

Treatment:

Drug: ELND005

ELND005 QD

Experimental group

Description:

ELND005 250 mg QD

Treatment:

Drug: ELND005

Placebo

Placebo Comparator group

Description:

Placebo BID

Treatment:

Drug: Placebo