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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
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:
Collaborator:
Elan Pharmaceuticals
Information provided by (Responsible Party):
OPKO Health, Inc.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Condition Down Syndrome
Interventions Drug: ELND005
Drug: Placebo
Enrollment 23
Recruitment Details  
Pre-assignment Details  
Arm/Group Title ELND005 BID ELND005 QD Placebo
Hide Arm/Group Description

ELND005 250 mg BID

ELND005

ELND005 250 mg QD

ELND005

Placebo BID

Placebo
Period Title: Overall Study
Started 12 5 6
Completed 12 4 6
Not Completed 0 1 0
Reason Not Completed
Withdrawal by Subject             0             1             0
Arm/Group Title ELND005 BID ELND005 QD Placebo Total
Hide Arm/Group Description

ELND005 250 mg BID

ELND005

ELND005 250 mg QD

ELND005

Placebo BID

Placebo

 Total of all reporting groups
Overall Number of Baseline Participants 12 5 6 23
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 12 participants 5 participants 6 participants 23 participants
26.3  (7.43) 27.8  (4.03) 30.0  (5.93) 27.6  (6.49)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants 5 participants 6 participants 23 participants
Female
3
  25.0%
3
  60.0%
3
  50.0%
9
  39.1%
Male
9
  75.0%
2
  40.0%
3
  50.0%
14
  60.9%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants 5 participants 6 participants 23 participants
Hispanic or Latino
1
   8.3%
0
   0.0%
1
  16.7%
2
   8.7%
Not Hispanic or Latino
11
  91.7%
5
 100.0%
5
  83.3%
21
  91.3%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants 5 participants 6 participants 23 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
1
   8.3%
0
   0.0%
0
   0.0%
1
   4.3%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
1
   8.3%
1
  20.0%
0
   0.0%
2
   8.7%
White
10
  83.3%
4
  80.0%
6
 100.0%
20
  87.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 12 participants 5 participants 6 participants 23 participants
12 5 6 23
IQ Score   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 12 participants 5 participants 6 participants 23 participants
50.6  (13.81) 63.3  (19.62) 55.8  (6.65) 54.3  (13.75)
[1]
Measure Description: IQ was measured by the Kaufman Brief Intelligence Test (KBIT)
1.Primary Outcome
Title Incidence of Adverse Events (TEAEs)
Hide Description 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.
Time Frame 4 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title ELND005 BID ELND005 QD Placebo
Hide Arm/Group Description:

ELND005 250 mg BID

ELND005

ELND005 250 mg QD

ELND005

Placebo BID

Placebo
Overall Number of Participants Analyzed 12 5 6
Measure Type: Number
Unit of Measure: participants
5 2 0
2.Other Pre-specified Outcome
Title Changes From Baseline in Abnormal Neurological Examination Results
Hide Description Subjects with Abnormal Neurological Examination Results
Time Frame Baseline and 4 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title ELND005 BID ELND005 QD Placebo
Hide Arm/Group Description:

ELND005 250 mg BID

ELND005

ELND005 250 mg QD

ELND005

Placebo BID

Placebo
Overall Number of Participants Analyzed 12 4 6
Measure Type: Number
Unit of Measure: participants
Baseline 4 1 3
Week 4 4 1 3
3.Other Pre-specified Outcome
Title Pharmacokinetic Assessment
Hide Description Mean Plasma ELND005 Concentrations- Cmax
Time Frame Baseline and 4 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title ELND005 BID ELND005 QD Placebo
Hide Arm/Group Description:

ELND005 250 mg BID

ELND005

ELND005 250 mg QD

ELND005

Placebo BID

Placebo
Overall Number of Participants Analyzed 12 4 6
Mean (Standard Deviation)
Unit of Measure: μg/mL
Mean Cmax, First Dose, Day 0 1.68  (0.90) 2.61  (0.62) 0  (0)
Mean Cmax, Last Dose, Day 28 6.33  (1.95) 4.48  (1.13) 0  (0)
4.Other Pre-specified Outcome
Title Cognitive Outcome (RADD Total Score)
Hide Description 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.
Time Frame Baseline and 4 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title ELND005 BID ELND005 QD Placebo
Hide Arm/Group Description:

ELND005 250 mg BID

ELND005

ELND005 250 mg QD

ELND005

Placebo BID

Placebo
Overall Number of Participants Analyzed 12 4 6
Mean (Standard Deviation)
Unit of Measure: units on a scale
Day 0 58.7  (10.2) 58.0  (15.6) 62.2  (9.8)
Week 4 59.1  (11.5) 64.3  (13.5) 62.8  (10.3)
5.Other Pre-specified Outcome
Title Improvement in NPI Total Scores in Subjects With NPI Score ≥1 at Baseline Baseline
Hide Description 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.
Time Frame Baseline and 4 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects with NPI Score ≥1 at baseline
Arm/Group Title ELND005 BID ELND005 QD Placebo
Hide Arm/Group Description:

ELND005 250 mg BID

ELND005

ELND005 250 mg QD

ELND005

Placebo BID

Placebo
Overall Number of Participants Analyzed 8 4 3
Measure Type: Number
Unit of Measure: participants
7 0 1
Time Frame Adverse events were recorded for each patient, starting from the time the consent form was signed until completion of the study.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title ELND005 BID ELND005 QD Placebo
Hide Arm/Group Description

ELND005 250 mg BID

ELND005

ELND005 250 mg QD

ELND005

Placebo BID

Placebo
All-Cause Mortality
ELND005 BID ELND005 QD Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/-- 
Hide Serious Adverse Events
ELND005 BID ELND005 QD Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/12 (0.00%)   0/5 (0.00%)   0/6 (0.00%) 
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
ELND005 BID ELND005 QD Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   5/12 (41.67%)   2/5 (40.00%)   0/6 (0.00%) 
Cardiac disorders       
Bradycardia † 1  1/12 (8.33%)  0/5 (0.00%)  0/6 (0.00%) 
Gastrointestinal disorders       
Diarrhoea † 1  1/12 (8.33%)  0/5 (0.00%)  0/6 (0.00%) 
Oral Disorder † 1  1/12 (8.33%)  0/5 (0.00%)  0/6 (0.00%) 
General disorders       
Catheter Site Pain † 1  0/12 (0.00%)  1/5 (20.00%)  0/6 (0.00%) 
Immune system disorders       
Seasonal Allergy † 1  1/12 (8.33%)  0/5 (0.00%)  0/6 (0.00%) 
Injury, poisoning and procedural complications       
Muscle Strain † 1  0/12 (0.00%)  1/5 (20.00%)  0/6 (0.00%) 
Investigations       
Electrocardiogram abnormal † 1  0/12 (0.00%)  1/5 (20.00%)  0/6 (0.00%) 
Nervous system disorders       
Resting Tremor † 1  1/12 (8.33%)  0/5 (0.00%)  0/6 (0.00%) 
Restless leg syndrome † 1  1/12 (8.33%)  0/5 (0.00%)  0/6 (0.00%) 
Psychiatric disorders       
Anger † 1  0/12 (0.00%)  1/5 (20.00%)  0/6 (0.00%) 
Skin and subcutaneous tissue disorders       
Pseudofolliculitis barbae † 1  1/12 (8.33%)  0/5 (0.00%)  0/6 (0.00%) 
†
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (16.1)
[Not Specified]
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Aleksandra Pastrak,MD, PhD, VP of Clinical Development and Medical Officer
Organization: Transition Therapeutics Ireland Limited
Phone: +1 416 263 1227
EMail: apastrak@transitiontherapeutics.com
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Layout table for additonal information
Responsible Party: OPKO Health, Inc.
ClinicalTrials.gov Identifier: NCT01791725    
Other Study ID Numbers: ELND005-DS201
First Submitted: February 6, 2013
First Posted: February 15, 2013
Results First Submitted: September 3, 2015
Results First Posted: January 29, 2016
Last Update Posted: November 4, 2019