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

Phase 2
Completed
Conditions
Down Syndrome
Interventions
Drug: Placebo
Registration Number
NCT01791725
Lead Sponsor
OPKO Health, Inc.
Brief Summary

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.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
23
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

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
ELND005 QDELND005ELND005 250 mg QD
PlaceboPlaceboPlacebo BID
ELND005 BIDELND005ELND005 250 mg BID
Primary Outcome Measures
NameTimeMethod
Incidence of Adverse Events (TEAEs)4 weeks

For all AE summaries, if a patient had more than one AE within a preferred term, the patient was counted only once, at the maximum severity and with the closest relationship to study drug. If a patient had more than one AE within a SOC, the subject was similarly counted only once when reporting results for that SOC.

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (3)

Massachusetts General Hospital

🇺🇸

Boston, Massachusetts, United States

University of California, Irvine

🇺🇸

Orange, California, United States

University of California, San Diego

🇺🇸

La Jolla, California, United States

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