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| Name | Class |
|---|---|
| Elan Pharmaceuticals | INDUSTRY |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ELND005 BID | Experimental | ELND005 250 mg BID |
|
| ELND005 QD | Experimental | ELND005 250 mg QD |
|
| Placebo | Placebo Comparator | Placebo BID |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ELND005 | Drug |
| ||
| Placebo |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
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| Measure | Description | Time Frame |
|---|---|---|
| Changes From Baseline in Abnormal Neurological Examination Results | Subjects with Abnormal Neurological Examination Results | Baseline and 4 weeks |
| Pharmacokinetic Assessment | Mean Plasma ELND005 Concentrations- Cmax |
Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California, San Diego | La Jolla | California | 92093-0949 | United States | ||
| University of California, Irvine |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 28453471 | Derived | 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. |
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| ID | Title | Description |
|---|---|---|
| FG000 | ELND005 BID | ELND005 250 mg BID ELND005 |
| FG001 | ELND005 QD | ELND005 250 mg QD ELND005 |
| FG002 | Placebo | Placebo BID Placebo |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | ELND005 BID | ELND005 250 mg BID ELND005 |
| BG001 | ELND005 QD | ELND005 250 mg QD ELND005 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Posted | Number | participants | 4 weeks |
|
Adverse events were recorded for each patient, starting from the time the consent form was signed until completion of the study.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | ELND005 BID | ELND005 250 mg BID ELND005 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Bradycardia | Cardiac disorders | MedDRA (16.1) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Aleksandra Pastrak,MD, PhD, VP of Clinical Development and Medical Officer | Transition Therapeutics Ireland Limited | +1 416 263 1227 | apastrak@transitiontherapeutics.com |
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| ID | Term |
|---|---|
| D004314 | Down Syndrome |
| ID | Term |
|---|---|
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
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| ID | Term |
|---|---|
| C009217 | scyllitol |
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|
| Baseline and 4 Weeks |
| Cognitive Outcome (RADD Total Score) | 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. | Baseline and 4 Weeks |
| Improvement in NPI Total Scores in Subjects With NPI Score ≥1 at Baseline Baseline | 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. | Baseline and 4 weeks |
| Orange |
| California |
| 92868 |
| United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| BG002 |
| Placebo |
Placebo BID Placebo |
| BG003 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| IQ Score | IQ was measured by the Kaufman Brief Intelligence Test (KBIT) | Mean | Standard Deviation | units on a scale |
|
Placebo BID Placebo |
|
|
| Other Pre-specified | Changes From Baseline in Abnormal Neurological Examination Results | Subjects with Abnormal Neurological Examination Results | Posted | Number | participants | Baseline and 4 weeks |
|
|
|
| Other Pre-specified | Pharmacokinetic Assessment | Mean Plasma ELND005 Concentrations- Cmax | Posted | Mean | Standard Deviation | μg/mL | Baseline and 4 Weeks |
|
|
|
| Other Pre-specified | Cognitive Outcome (RADD Total Score) | 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. | Posted | Mean | Standard Deviation | units on a scale | Baseline and 4 Weeks |
|
|
|
| Other Pre-specified | Improvement in NPI Total Scores in Subjects With NPI Score ≥1 at Baseline Baseline | 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. | Subjects with NPI Score ≥1 at baseline | Posted | Number | participants | Baseline and 4 weeks |
|
|
|
| 0 |
| 12 |
| 5 |
| 12 |
| EG001 | ELND005 QD | ELND005 250 mg QD ELND005 | 0 | 5 | 2 | 5 |
| EG002 | Placebo | Placebo BID Placebo | 0 | 6 | 0 | 6 |
| Diarrhoea | Gastrointestinal disorders | MedDRA (16.1) | Systematic Assessment |
|
| Catheter Site Pain | General disorders | MedDRA (16.1) | Systematic Assessment |
|
| Seasonal Allergy | Immune system disorders | MedDRA (16.1) | Systematic Assessment |
|
| Muscle Strain | Injury, poisoning and procedural complications | MedDRA (16.1) | Systematic Assessment |
|
| Electrocardiogram abnormal | Investigations | MedDRA (16.1) | Systematic Assessment |
|
| Resting Tremor | Nervous system disorders | MedDRA (16.1) | Systematic Assessment |
|
| Anger | Psychiatric disorders | MedDRA (16.1) | Systematic Assessment |
|
| Pseudofolliculitis barbae | Skin and subcutaneous tissue disorders | MedDRA (16.1) | Systematic Assessment |
|
| Oral Disorder | Gastrointestinal disorders | MedDRA (16.1) | Systematic Assessment |
|
| Restless leg syndrome | Nervous system disorders | MedDRA (16.1) | Systematic Assessment |
|
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| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D025063 | Chromosome Disorders |
| D030342 | Genetic Diseases, Inborn |
| Title | Measurements |
|---|---|
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