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

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ClinicalTrials.gov Identifier: NCT01791725
Recruitment Status : Completed
First Posted : February 15, 2013
Results First Posted : January 29, 2016
Last Update Posted : January 29, 2016
Elan Pharmaceuticals
Information provided by (Responsible Party):
Transition Therapeutics Ireland Limited

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 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
Primary Completion Date : June 2014
Study Completion Date : June 2014

Resource links provided by the National Library of Medicine

U.S. FDA Resources

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

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.

Information from the National Library of Medicine

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

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01791725

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
Transition Therapeutics Ireland Limited
Elan Pharmaceuticals

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Transition Therapeutics Ireland Limited
ClinicalTrials.gov Identifier: NCT01791725     History of Changes
Other Study ID Numbers: ELND005-DS201
First Posted: February 15, 2013    Key Record Dates
Results First Posted: January 29, 2016
Last Update Posted: January 29, 2016
Last Verified: December 2015

Additional relevant MeSH terms:
Down Syndrome
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