DS201

CompletedPhase 2Results posted

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

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

Lead sponsor

OPKO Health, Inc.

Asset

ELND005

Listed sites

Recruiting sites

Enrollment

actual

Study population

Mixed / unspecified dementia

Key I/E criterion

•Age 18-45

Primary endpoint

•Adverse Events (TEAEs)

Footprint

Where this trial recruits

Site locations as reported to ClinicalTrials.gov. Site count is not enrollment count; per-site enrollment is not available from source.

Identifiers

Registered as

Org study IDELND005-DS201

NCT IDNCT01791725

Timeline

Milestones

Study first posted2013-02-15estimated

Study start2013-09 (month precision)

Primary completion2014-06actual (month precision)

Study completion2014-06actual (month precision)

Results first posted2016-01-29estimated

Last update posted2019-11-04actual

Assets

Drug assets

ELND005

Study populations

Who this study enrolls

Mixed / unspecified dementia

Eligibility

Who can enroll

Minimum age18 Years

Maximum age45 Years

SexAll

Healthy volunteersNot accepted

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

Endpoints (10)

What's being measured

Protocol endpoints and posted registry outcome measures, grouped into outcome categories. Composite endpoints show their component event types. Standard codes (LOINC, SNOMED CT) are shown where available.

Coverage by outcome category

Safety / tolerability / PK

Global cognition

Behavior / neuropsychiatric

Other (unclassified)

Global cognition

2 endpoints

Other/protocol endpoint

Cognitive Outcome (RADD Total Score)

Time frame:Baseline and 4 Weeks

ratio, descriptive

Other_pre_specified/registry result

Cognitive Outcome (RADD Total Score)

Time frame:Baseline and 4 Weeks

ratio, descriptive

Posted result

Group	Value (mean), units on a scale	Standard deviation
ELND005 BIDDay 0n=12 Participants	58.7	10.2
Week 4n=12 Participants	59.1	11.5
ELND005 QDDay 0n=4 Participants	58.0	15.6
Week 4n=4 Participants	64.3	13.5
PlaceboDay 0n=6 Participants	62.2	9.8
Week 4n=6 Participants	62.8	10.3

Behavior / neuropsychiatric

2 endpoints

Other/protocol endpoint

Improvement in NPI Total Scores in Subjects With NPI Score ≥1 at Baseline Baseline

Time frame:Baseline and 4 weeks

Neuropsychiatric Inventory (NPI)

change from baseline, improvement

Other_pre_specified/registry result

Improvement in NPI Total Scores in Subjects With NPI Score ≥1 at Baseline Baseline

Time frame:Baseline and 4 weeks

Neuropsychiatric Inventory (NPI)

change from baseline, improvement

Posted result

Group	Value (number), participants	Reported bounds
ELND005 BIDn=8 Participants	7	-
ELND005 QDn=4 Participants	0	-
Placebon=3 Participants	1	-

Safety / tolerability / PK

4 endpoints

Primary/protocol endpoint

Incidence of Adverse Events (TEAEs)

Time frame:4 weeks

event count, event

Primary/registry result

Incidence of Adverse Events (TEAEs)

Time frame:4 weeks

event count, event

Posted result

Group	Value (number), participants	Reported bounds
ELND005 BIDn=12 Participants	5	-
ELND005 QDn=5 Participants	2	-
Placebon=6 Participants	0	-

Other/protocol endpoint

Pharmacokinetic Assessment

Time frame:Baseline and 4 Weeks

concentration, descriptive

Other_pre_specified/registry result

Pharmacokinetic Assessment

Time frame:Baseline and 4 Weeks

concentration, descriptive

Posted result

Group	Value (mean), μg/mL	Standard deviation
ELND005 BIDMean Cmax, First Dose, Day 0n=12 Participants	1.68	0.90
Mean Cmax, Last Dose, Day 28n=12 Participants	6.33	1.95
ELND005 QDMean Cmax, First Dose, Day 0n=4 Participants	2.61	0.62
Mean Cmax, Last Dose, Day 28n=4 Participants	4.48	1.13
PlaceboMean Cmax, First Dose, Day 0n=6 Participants	0	0
Mean Cmax, Last Dose, Day 28n=6 Participants	0	0

Other (unclassified)

2 endpoints

Other/protocol endpoint/low confidence

Changes From Baseline in Abnormal Neurological Examination Results

Time frame:Baseline and 4 weeks

descriptive

Other_pre_specified/registry result/low confidence

Changes From Baseline in Abnormal Neurological Examination Results

Time frame:Baseline and 4 weeks

descriptive

Posted result

Group	Value (number), participants	Reported bounds
ELND005 BIDBaselinen=12 Participants	4	-
Week 4n=12 Participants	4	-
ELND005 QDBaselinen=4 Participants	1	-
Week 4n=4 Participants	1	-
PlaceboBaselinen=6 Participants	3	-
Week 4n=6 Participants	3	-

Publications (1)

Bibliography

Records linked to this trial through ClinicalTrials.gov references, PubMed NCT search, and curated study seeds. 'Canonical' marks design/result papers; others are registry references or candidates.

Registry references + supporting bibliography

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.
PMID28453471via DERIVED

Provenance

Sources

Trial identity, design, statusClinicalTrials.gov API v2

Snapshot dateJuly 21, 2026

Endpoint classificationDelfa ADRD endpoint taxonomy

Results tableClinicalTrials.gov results section

Trial facts come from public ClinicalTrials.gov records. Endpoint categories are Delfa's classification of those records, not a ClinicalTrials.gov field. All figures reflect the July 21, 2026 snapshot.