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

Down Syndrome Clinical Trial

— DS201  

Official title:

A 4-Week Randomized, Double-Blind, Placebo-Controlled, Phase 2a Safety and PK Study of Oral ELND005 in Young Adults With Down Syndrome Without Dementia

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01791725
• Other study ID #: ELND005-DS201
• Status: Completed
• Phase: Phase 2
• Start date: September 2013
• Est. completion date: June 2014

Study information

• Verified date: October 2019
• Source: OPKO Health, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 23
• Est. completion date: June 2014
• Est. primary completion date: June 2014
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 45 Years

Eligibility

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

Related Conditions & MeSH terms

• Dementia
• Down Syndrome
• Syndrome

Intervention

Drug:
• ELND005

• Placebo

Locations

Country	Name	City	State
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	University of California, San Diego	La Jolla	California
United States	University of California, Irvine	Orange	California

Sponsors (2)

Lead Sponsor	Collaborator
OPKO Health, Inc.	Elan Pharmaceuticals

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Changes From Baseline in Abnormal Neurological Examination Results	Subjects with Abnormal Neurological Examination Results	Baseline and 4 weeks
Other	Pharmacokinetic Assessment	Mean Plasma ELND005 Concentrations- Cmax	Baseline and 4 Weeks
Other	Cognitive Outcome (RADD Total Score)	Rapid Assessment for Development Disabilities (RADD) The RADD test was developed from the low-difficulty items from published intelligence tests (Walsh et al 2007). It was specifically developed for evaluation of individuals with intellectual disabilities and developmental disabilities. It is a validated and reliable cognitive screening instrument that can be rapidly administered. The RADD is composed of 76 items. Each item is scored as 0 (incorrect) or 1 (correct).The test assesses a wide range of functional abilities including receptive and expressive language, orientation, registration, recall, attention, self identification, motor skills, imitation, abstract reasoning, number skills, comprehension and short-term memory to give a total score. Scores are from 0 to 76. A higher total score is correlated with a higher Cognitive Impairment level.	Baseline and 4 Weeks
Other	Improvement in NPI Total Scores in Subjects With NPI Score =1 at Baseline Baseline	The Neuropsychiatric Inventory(NPI) (Cummings et al 1994) is a behavioral measure that assesses psychopathology in dementia patients. The NPI was administered at the Baseline Visit (Day 1) and at Day 28 (EOS) or ET. A decrease in score shows an improvement in symptoms.	Baseline and 4 weeks
Primary	Incidence of Adverse Events (TEAEs)	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.	4 weeks