Trial · NCT01791725

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

NCT01791725, Phase 2, Completed, OPKO Health, Inc.; results first posted 2016-01-29

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. A 4-Week Safety Study of Oral ELND005 in Young Adults With Down Syndrome Without Dementia. Trials, record NCT01791725. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01791725

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: A 4-Week Safety Study of Oral ELND005 in Young Adults With Down Syndrome Without Dementia
NCT number: NCT01791725
Status: Completed
Phases: Phase 2
Enrollment: 23 participants, actual
Lead sponsor: OPKO Health, Inc.
Conditions: Down Syndrome
Interventions: ELND005, Placebo
First posted: 2013-02-15
Last updated: 2019-11-04
Results first posted: 2016-01-29

Source ClinicalTrials.gov

Registered outcomes5

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Incidence of Adverse Events (TEAEs)	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	Changes From Baseline in Abnormal Neurological Examination Results	Baseline and 4 weeks	Subjects with Abnormal Neurological Examination Results
Other	Pharmacokinetic Assessment	Baseline and 4 Weeks	Mean Plasma ELND005 Concentrations- Cmax
Other	Cognitive Outcome (RADD Total Score)	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.
Other	Improvement in NPI Total Scores in Subjects With NPI Score ≥1 at Baseline Baseline	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.

Source ClinicalTrials.gov

Outcomes5

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Incidence of Adverse Events (TEAEs)	4 weeks		participants	NUMBER
OTHER_PRE_SPECIFIED	Changes From Baseline in Abnormal Neurological Examination Results	Baseline and 4 weeks		participants	NUMBER
OTHER_PRE_SPECIFIED	Pharmacokinetic Assessment	Baseline and 4 Weeks		μg/mL	MEAN
OTHER_PRE_SPECIFIED	Cognitive Outcome (RADD Total Score)	Baseline and 4 Weeks		units on a scale	MEAN
OTHER_PRE_SPECIFIED	Improvement in NPI Total Scores in Subjects With NPI Score ≥1 at Baseline Baseline	Baseline and 4 weeks	Subjects with NPI Score ≥1 at baseline	participants	NUMBER

Source ClinicalTrials.gov

Results by group24

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Incidence of Adverse Events (TEAEs)	ELND005 BID		5		participants
Incidence of Adverse Events (TEAEs)	ELND005 QD		2		participants
Incidence of Adverse Events (TEAEs)	Placebo		0		participants
Changes From Baseline in Abnormal Neurological Examination Results	ELND005 BID	Baseline	4		participants
Changes From Baseline in Abnormal Neurological Examination Results	ELND005 QD	Baseline	1		participants
Changes From Baseline in Abnormal Neurological Examination Results	Placebo	Baseline	3		participants
Changes From Baseline in Abnormal Neurological Examination Results	ELND005 BID	Week 4	4		participants
Changes From Baseline in Abnormal Neurological Examination Results	ELND005 QD	Week 4	1		participants
Changes From Baseline in Abnormal Neurological Examination Results	Placebo	Week 4	3		participants
Pharmacokinetic Assessment	ELND005 BID	Mean Cmax, First Dose, Day 0	1.68	Standard Deviation 0.90	μg/mL

1 to 10 of 24Source ClinicalTrials.gov

See all 24 results by group

Participant flow9

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	ELND005 BID	12
Overall Study	STARTED	ELND005 QD	5
Overall Study	STARTED	Placebo	6
Overall Study	COMPLETED	ELND005 BID	12
Overall Study	COMPLETED	ELND005 QD	4
Overall Study	COMPLETED	Placebo	6
Overall Study	NOT COMPLETED	ELND005 BID	0
Overall Study	NOT COMPLETED	ELND005 QD	1
Overall Study	NOT COMPLETED	Placebo	0

Source ClinicalTrials.gov

Adverse events33

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Bradycardia	Cardiac disorders	ELND005 BID	1	12
other	Bradycardia	Cardiac disorders	ELND005 QD	0	5
other	Bradycardia	Cardiac disorders	Placebo	0	6
other	Diarrhoea	Gastrointestinal disorders	ELND005 BID	1	12
other	Diarrhoea	Gastrointestinal disorders	ELND005 QD	0	5
other	Diarrhoea	Gastrointestinal disorders	Placebo	0	6
other	Catheter Site Pain	General disorders	ELND005 BID	0	12
other	Catheter Site Pain	General disorders	ELND005 QD	1	5
other	Catheter Site Pain	General disorders	Placebo	0	6
other	Seasonal Allergy	Immune system disorders	ELND005 BID	1	12

1 to 10 of 33Source ClinicalTrials.gov

See all 33 adverse events

Adverse event totals9

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	ELND005 BID
Total, all-cause mortality	ELND005 QD
Total, all-cause mortality	Placebo
Total, other adverse events	ELND005 BID	5	12
Total, other adverse events	ELND005 QD	2	5
Total, other adverse events	Placebo	0	6
Total, serious adverse events	ELND005 BID	0	12
Total, serious adverse events	ELND005 QD	0	5
Total, serious adverse events	Placebo	0	6

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
28453471	DERIVED

Source PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

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