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.

Tracking Information

• First Submitted Date: February 6, 2013
• First Posted Date: February 15, 2013
• Results First Submitted Date: September 3, 2015
• Results First Posted Date: January 29, 2016
• Last Update Posted Date: November 4, 2019
• Study Start Date: September 2013
• Actual Primary Completion Date: June 2014 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (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 (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 ]

• Current Secondary Outcome Measures: Not Provided

Original Secondary Outcome Measures (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: A 4-Week Safety Study of Oral ELND005 in Young Adults With Down Syndrome Without Dementia
• 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
• 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: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Triple (Participant, Care Provider, Investigator); Primary Purpose: Treatment
• Condition: Down Syndrome

Intervention

• Drug: ELND005
• Drug: Placebo

Study Arms

• 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.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: December 1, 2014): 23
• Original Estimated Enrollment (submitted: February 12, 2013): 24
• Actual Study Completion Date: June 2014
• Actual Primary Completion Date: June 2014 (Final data collection date for primary outcome measure)

Eligibility 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 45 Years (Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT01791725
• Other Study ID Numbers: ELND005-DS201
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: OPKO Health, Inc.
• Original Responsible Party: Elan Pharmaceuticals
• Current Study Sponsor: OPKO Health, Inc.
• Original Study Sponsor: Elan Pharmaceuticals
• Collaborators: Elan Pharmaceuticals
• Investigators: Not Provided
• PRS Account: OPKO Health, Inc.
• Verification Date: October 2019