Trial record 2 of 6 for:    ELND005

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

This study has been completed.
Sponsor:
Collaborator:
Elan Pharmaceuticals
Information provided by (Responsible Party):
Transition Therapeutics Ireland Limited
ClinicalTrials.gov Identifier:
NCT01791725
First received: February 6, 2013
Last updated: December 22, 2015
Last verified: December 2015
  Purpose
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 Intervention Phase
Down Syndrome
Drug: ELND005
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, 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

Resource links provided by NLM:


Further study details as provided by Transition Therapeutics Ireland Limited:

Primary Outcome Measures:
  • Incidence of Adverse Events (TEAEs) [ Time Frame: 4 weeks ] [ Designated as safety issue: Yes ]
    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:
  • Changes From Baseline in Abnormal Neurological Examination Results [ Time Frame: Baseline and 4 weeks ] [ Designated as safety issue: No ]
    Subjects with Abnormal Neurological Examination Results

  • Pharmacokinetic Assessment [ Time Frame: Baseline and 4 Weeks ] [ Designated as safety issue: No ]
    Mean Plasma ELND005 Concentrations- Cmax

  • Cognitive Outcome (RADD Total Score) [ Time Frame: Baseline and 4 Weeks ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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.


Enrollment: 23
Study Start Date: September 2013
Study Completion Date: June 2014
Primary Completion Date: June 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  Eligibility

Ages Eligible for Study:   18 Years to 45 Years
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01791725

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
Transition Therapeutics Ireland Limited
Elan Pharmaceuticals
  More Information

No publications provided

Responsible Party: Transition Therapeutics Ireland Limited
ClinicalTrials.gov Identifier: NCT01791725     History of Changes
Other Study ID Numbers: ELND005-DS201 
Study First Received: February 6, 2013
Results First Received: September 3, 2015
Last Updated: December 22, 2015
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Down Syndrome
Syndrome
Abnormalities, Multiple
Chromosome Disorders
Congenital Abnormalities
Disease
Genetic Diseases, Inborn
Intellectual Disability
Nervous System Diseases
Neurobehavioral Manifestations
Neurologic Manifestations
Pathologic Processes

ClinicalTrials.gov processed this record on February 11, 2016