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

NCT01791725 | Completed Phase 2 Results

• 1 other identifier: ELND005-DS201
• Study Type: interventional
• Target: 23
• Locations: 1 country
• Sites: 3

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.

Conditions

Down Syndrome

Outcome Measures

Primary Outcomes (1)

• Incidence of Adverse Events (TEAEs)

  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.

  4 weeks

Other Outcomes (4)

• Changes From Baseline in Abnormal Neurological Examination Results

  Baseline and 4 weeks

• Pharmacokinetic Assessment

  Baseline and 4 Weeks

• Cognitive Outcome (RADD Total Score)

  Baseline and 4 Weeks

Study Arms (3)

ELND005 BID

EXPERIMENTAL

ELND005 250 mg BID

Drug: ELND005

ELND005 QD

EXPERIMENTAL

ELND005 250 mg QD

Drug: ELND005

Placebo

PLACEBO COMPARATOR

Placebo BID

Drug: Placebo

Interventions

ELND005 DRUG

ELND005 BID; ELND005 QD

Placebo DRUG

Placebo

Eligibility Criteria

• Age: 18 Years - 45 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• years of age
• Has an IQ of \> 40 (K-BIT)
• Able and willing to have a brain MRI

You may not qualify if:

• Symptoms of dementia or worsening cognition over the past year.
• Has a history of hepatitis B, hepatitis C, or HIV

Sponsors & Collaborators

• OPKO Health, Inc.: lead
• Elan Pharmaceuticals: collaborator

Study Sites (3)

University of California, San Diego

La Jolla, California, 92093-0949, United States

Location

University of California, Irvine

Orange, California, 92868, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Related Publications (1)

• 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.

  PMID: 28453471 DERIVED

MeSH Terms

Conditions

Down Syndrome

Interventions

scyllitol

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn

Results Point of Contact

• Title: Aleksandra Pastrak,MD, PhD, VP of Clinical Development and Medical Officer
• Organization: Transition Therapeutics Ireland Limited

apastrak@transitiontherapeutics.com

+1 416 263 1227

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 6, 2013
• First Posted: February 15, 2013
• Study Start: September 1, 2013
• Primary Completion: June 1, 2014
• Study Completion: June 1, 2014
• Last Updated: November 4, 2019
• Results First Posted: January 29, 2016

Record last verified: 2019-10

Locations

US (3)