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

• ClinicalTrials.gov Identifier: NCT01791725
• Recruitment Status: Completed
• First Posted: February 15, 2013
• Results First Posted: January 29, 2016
• Last Update Posted: November 4, 2019
• Sponsor: OPKO Health, Inc.
• Collaborator: Elan Pharmaceuticals
• Information provided by (Responsible Party): OPKO Health, Inc.

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Down Syndrome	Drug: ELND005; Drug: Placebo	Phase 2

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 23 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Triple (Participant, Care Provider, Investigator)
• Primary Purpose: Treatment
• 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
• Study Start Date: September 2013
• Actual Primary Completion Date: June 2014
• Actual Study Completion Date: June 2014

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures :

1. Incidence of Adverse Events (TEAEs) [ Time Frame: 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.

Other Outcome Measures:

1. Changes From Baseline in Abnormal Neurological Examination Results [ Time Frame: Baseline and 4 weeks ]
   Subjects with Abnormal Neurological Examination Results
2. Pharmacokinetic Assessment [ Time Frame: Baseline and 4 Weeks ]
   Mean Plasma ELND005 Concentrations- Cmax
3. Cognitive Outcome (RADD Total Score) [ Time Frame: Baseline and 4 Weeks ]
   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.
4. Improvement in NPI Total Scores in Subjects With NPI Score ≥1 at Baseline Baseline [ Time Frame: Baseline and 4 weeks ]
   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.

Eligibility Criteria

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

Criteria

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

Contacts and Locations

Locations

United States, California

University of California, San Diego

La Jolla, California, United States, 92093-0949

University of California, Irvine

Orange, California, United States, 92868

United States, Massachusetts

Massachusetts General Hospital

Boston, Massachusetts, United States, 02114

Sponsors and Collaborators

OPKO Health, Inc.

Elan Pharmaceuticals

More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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.

• Responsible Party: OPKO Health, Inc.
• ClinicalTrials.gov Identifier: NCT01791725
• Other Study ID Numbers: ELND005-DS201
• First Posted: February 15, 2013
• Results First Posted: January 29, 2016
• Last Update Posted: November 4, 2019
• Last Verified: October 2019

Additional relevant MeSH terms:

Dementia; Down Syndrome; Syndrome; Disease; Pathologic Processes; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurocognitive Disorders; Mental Disorders; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Abnormalities, Multiple; Congenital Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn