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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01791725
Recruitment Status : Completed
First Posted : February 15, 2013
Results First Posted : January 29, 2016
Last Update Posted : November 4, 2019
Elan Pharmaceuticals
Information provided by (Responsible Party):
OPKO Health, Inc.

Tracking Information
First Submitted Date  ICMJE February 6, 2013
First Posted Date  ICMJE February 15, 2013
Results First Submitted Date  ICMJE September 3, 2015
Results First Posted Date  ICMJE January 29, 2016
Last Update Posted Date November 4, 2019
Study Start Date  ICMJE September 2013
Actual Primary Completion Date June 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 22, 2015)
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.
Original Primary Outcome Measures  ICMJE
 (submitted: February 12, 2013)
  • Incidence of treatment-emergent adverse events (TEAEs), [ Time Frame: 4 weeks ]
  • Changes from Baseline in neurological examinations [ Time Frame: 4 weeks ]
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE
 (submitted: February 12, 2013)
  • Plasma ELND005 concentrations [ Time Frame: 4 Weeks ]
    ELND005 PK profile to optimize PK model including patient population
  • Changes from baseline in vital signs [ Time Frame: 4 weeks ]
Current Other Pre-specified Outcome Measures
 (submitted: December 22, 2015)
  • Changes From Baseline in Abnormal Neurological Examination Results [ Time Frame: Baseline and 4 weeks ]
    Subjects with Abnormal Neurological Examination Results
  • Pharmacokinetic Assessment [ Time Frame: Baseline and 4 Weeks ]
    Mean Plasma ELND005 Concentrations- Cmax
  • 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.
  • 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.
Original Other Pre-specified Outcome Measures
 (submitted: February 12, 2013)
  • Cognitive Outcome [ Time Frame: 4 Weeks ]
    Rapid Assessment for Development Disabilities (RADD)
  • Functional Outcomes [ Time Frame: 4 Weeks ]
    Vineland Adaptive Behavior Scale, Second Edition (VABS-II)
Descriptive Information
Brief Title  ICMJE A 4-Week Safety Study of Oral ELND005 in Young Adults With Down Syndrome Without Dementia
Official Title  ICMJE 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
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 Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Down Syndrome
Intervention  ICMJE
  • Drug: ELND005
  • Drug: Placebo
Study Arms  ICMJE
  • Experimental: ELND005 BID
    ELND005 250 mg BID
    Intervention: Drug: ELND005
  • Experimental: ELND005 QD
    ELND005 250 mg QD
    Intervention: Drug: ELND005
  • Placebo Comparator: Placebo
    Placebo BID
    Intervention: Drug: Placebo
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.

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: December 1, 2014)
Original Estimated Enrollment  ICMJE
 (submitted: February 12, 2013)
Actual Study Completion Date  ICMJE June 2014
Actual Primary Completion Date June 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 45 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT01791725
Other Study ID Numbers  ICMJE ELND005-DS201
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party OPKO Health, Inc.
Original Responsible Party Elan Pharmaceuticals
Current Study Sponsor  ICMJE OPKO Health, Inc.
Original Study Sponsor  ICMJE Elan Pharmaceuticals
Collaborators  ICMJE Elan Pharmaceuticals
Investigators  ICMJE Not Provided
PRS Account OPKO Health, Inc.
Verification Date October 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP