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

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

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.

Study Overview

• Status: Completed
• Conditions: Down Syndrome
• Intervention / Treatment: Drug: Placebo; Drug: ELND005

• Study Type: Interventional
• Enrollment (Actual): 23
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • La Jolla, California, United States, 92093-0949
      • University of California, San Diego
    • Orange, California, United States, 92868
      • University of California, Irvine
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts General Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 45 years (Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: ELND005 BID; ELND005 250 mg BID	Drug: ELND005
Experimental: ELND005 QD; ELND005 250 mg QD	Drug: ELND005
Placebo Comparator: Placebo; Placebo BID	Drug: Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes From Baseline in Abnormal Neurological Examination Results	Subjects with Abnormal Neurological Examination Results	Baseline and 4 weeks
Pharmacokinetic Assessment	Mean Plasma ELND005 Concentrations- Cmax	Baseline and 4 Weeks
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
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

Collaborators and Investigators

• Sponsor: OPKO Health, Inc.
• Collaborators: Elan Pharmaceuticals

Publications and helpful links

General Publications

• Rafii MS, Skotko BG, McDonough ME, Pulsifer M, Evans C, Doran E, Muranevici G, Kesslak P, Abushakra S, Lott IT; ELND005-DS Study Group. A Randomized, Double-Blind, Placebo-Controlled, Phase II Study of Oral ELND005 (scyllo-Inositol) in Young Adults with Down Syndrome without Dementia. J Alzheimers Dis. 2017;58(2):401-411. doi: 10.3233/JAD-160965.

Study record dates

Study Major Dates

• Study Start: September 1, 2013
• Primary Completion (Actual): June 1, 2014
• Study Completion (Actual): June 1, 2014

Study Registration Dates

• First Submitted: February 6, 2013
• First Submitted That Met QC Criteria: February 12, 2013
• First Posted (Estimate): February 15, 2013

Study Record Updates

• Last Update Posted (Actual): November 4, 2019
• Last Update Submitted That Met QC Criteria: October 17, 2019
• Last Verified: October 1, 2019

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Pathologic Processes; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Neurocognitive Disorders; Disease; Congenital Abnormalities; Genetic Diseases, Inborn; Intellectual Disability; Abnormalities, Multiple; Chromosome Disorders; Syndrome; Dementia; Down Syndrome
• Other Study ID Numbers: ELND005-DS201