Down SyndromePhase 2NCT02094053
A Double-blind, Placebo-controlled Comparative Study and Open-label Extension Study to Confirm the Efficacy and Safety of E2020 in Subjects With Down Syndrome Having Regression Symptoms and Disabled Activities of Daily Living.
A Phase 2 study testing E2020-Donepezil hydrochloride in people with Down Syndrome, run by Eisai Co., Ltd. at 10 sites in Japan.
Trial summary
The purpose of this double-blind, placebo-controlled, comparative study and open-label extension study is to confirm the efficacy and safety of E2020 in subjects with Down syndrome having regression symptoms and disabled activities of daily living. Read more about this study
- Who is it for?
- Ages 15 to 39 years
- Conditions the study is for
- Down Syndrome
- What is being tested?
- E2020-Donepezil hydrochloride
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 36 participants
- Where
- 10 sitesJapan
- Study dates
- 12 Sep 2013to 21 Apr 2017
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 15 to 39 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- At enrollment in Pre-randomization Phase
- With definitive diagnosis of Down syndrome
- Have greater than or equal to 3 of the following 4 symptoms among 9 items according to the diagnostic criteria issued by the Intractable Diseases Treatment Research Program 2010 (Research paper on Intractable Diseases Treatment Research Program; Survey on Sudden Regression (21 trisomy) and Preparation of Diagnostic Criteria.) Motor retardation, mutism, social withdrawal (homeboundness), sleep disorder
- Insufficiently improved with environmental adjustment and psychotherapies including counseling for greater than or equal to 8 weeks before enrollment
- Have a suspected diagnosis with neuropsychiatric disorder without sufficient effect on a disease even after medical treatment for greater than or equal to 8 weeks before enrollment.
- A total score of Body Functionality Checklist (51 items) is lesser than or equal to 210 at enrollment
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- Aged 15 to 39 years inclusive
- Males and females
- Must have a family member or a caregiver who will provide written informed consent and will be able to spend 3 days a week with the subject (at least 4 hours per day) and will be able to support the subject during the study by providing necessary study information to the subject, assisting treatment compliance, and accompanying the subject to all scheduled visits, supporting study-related tests for the efficacy and safety assessments throughout the study period
- Males and females of childbearing potential must practice highly effective contraception
- Able to comply with scheduled study visits according to the investigator's instruction
- Able to visit for scheduled assessments (except for walking difficulty due to development of regression)
- Submitted written informed consent for study entry (to obtain from subjects as much as possible; mandatory from their legal guardian)
Who cannot join
- At enrollment in Pre-randomization Phase
- Suspected to have progressive neuropsychiatric disease (e.g., neurodegenerative disorder and progressive tumor) evidenced by MRI or CT within 1 year before the Pre-randomization Phase (if not tested within 1 year before the Pre-randomization Phase, reconfirm during the Pre-randomization Phase).
- Have a history of significant neurological disorders such as stroke, brain tumor, encephalitis, meningitis, normal pressure hydrocephalus, brain trauma accompanying unconsciousness, and experience of brain surgery causing unsolved deficiency
- Previously diagnosed with autism
- With evidence of atlantoaxial subluxation, or underwent surgical operation for atlantoaxial subluxation within 2 years
- Have seizure symptoms within 2 years or used antiepileptic drug within 1 year before enrollment of Pre-randomization Phase.
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- With severe hearing or visual impairment which may affect regression
- Have a complication of cardiac disease (angina pectoris, congestive heart failure, bundle branch block, arrythmia) or peripheral vascular disease with unstable condition in 3 months before enrollment of Pre-randomization Phase
- Have a complication of clinically significant active and unstable diseases in the gastrointestinal, hepatic, renal, respiratory, or cardiovascular system
- Have a history of clinically significant gastrointestinal ulcer, bronchial asthma, or obstructive pulmonary disease
- Have a complication of disease affecting absorption, distribution, and metabolism of study drug (e.g., inflammatory colon disease, gastric ulcer, duodenal ulcer, hepatic disorder, serious lactose intolerance)
- With a present or past history of malignant tumor within 5 years before informed consent (except for basal cell carcinoma, squamous cell carcinoma)
- With a complication or history of drug or alcohol dependency within recent 10 years
- Have a known hypersensitivity to ingredient(s) of donepezil hydrochloride or peperidine derivatives
- Not meet the criteria of prohibited and restricted concomitant medications, or anticipated to deviate from the above criteria of prohibited and restricted concomitant medications/therapies during the study
- Pregnant or lactating women
- Have participated in another clinical study and received the study drug within 12 weeks before the enrollment of this study
- Have used donepezil hydrochloride or have participated in a clinical study of E2020 and received E2020 in the past
- With a history of a treatment for Alzheimer's type dementia
- With severe extrapyramidal disorder
- At enrollment in the Double-blind Phase
- Suspected to have a complication of severe disease considering from the laboratory parameters at enrollment in the Pre-randomization Phase (visit 1) and the safety is not protected in the opinion of the principal investigator or subinvestigator
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
Japan10 sites
Study site
Chiyoda-ku, Tokyo
Study site
Fukuoka, Fukuoka
Study site
Izumi-shi, Osaka
Study site
Matsumoto-shi, Nagano
Study site
Nagasaki, Nagasaki
Study site
Saitama-shi, Saitama
Study site
Sapporo, Hokkaido
Study site
Setagaya-ku, Tokyo
Study site
Takatsuki-shi, Kyoto
Study site
Yokohama, Kanagawa
About this study
The purpose of this double-blind, placebo-controlled, comparative study and open-label extension study is to confirm the efficacy and safety of E2020 in subjects with Down syndrome having regression symptoms and disabled activities of daily living.
Read the detailed description
This is a multicenter, randomized, double-blind, multiple-dose (two doses), placebo-controlled, parallel-group comparative study followed by an open-label extension study of E2020 in subjects with Down syndrome having regression and disabled ADL. A total of 60 subjects will be randomized to one of three dosing groups (at 1:1:1) to receive 3 mg of E2020, 5 mg of E2020, or placebo for 24 weeks based on their total scores of Body Functionality Checklist, sex, and study site as the allocation factors. This study consists of Pre-randomization Phase (4 weeks), Double-blind Phase (28 weeks), and Extension Phase (24 weeks). The Double-blind Phase includes a 24-week treatment period plus a 4-week transition period.
- Conditions
- Down Syndrome
- Being tested
- E2020-Donepezil hydrochloride, Placebo
- Keywords
- Down syndrome, regression symptoms, disabled activities of daily living
- Medical subject headings
- Down Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
E2020-Donepezil hydrochlorideDrug
3 mg of E2020 (oral) once daily, for 24 weeks
E2020-Donepezil hydrochlorideDrug
5 mg of E2020 (oral) once daily, for 24 weeks
PlaceboDrug
placebo (oral) once daily, for 24 weeks
| Study group | Role | What they receive |
|---|---|---|
| E2020 3 mg | Experimental | 3 mg of E2020 (oral) once daily, for 24 weeks Receives: Drug: E2020-Donepezil hydrochloride |
| E2020 5 mg | Experimental | 5 mg of E2020 (oral) once daily, for 24 weeks Receives: Drug: E2020-Donepezil hydrochloride |
| Placebo | Placebo comparator | placebo (oral) once daily, for 24 weeks Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes in total scores from baseline using Body Functionality Checklist (psychosomatic function questionnaire) in subjects with Down syndrome having regression symptoms and disabled activities of daily living (ADL), relative to placebo.
For the changes in a total score of Body Functionality Checklist (51 items) from Week 0 of the treatment period, Kruskal-Wallis test will be performed in the 3 mg group, the 5 mg group and placebo group to represent statistical significance. Summary statistics of the total score of Body Functionality Checklist (51 items) at each evaluation time and changes from before study drug administration in the treatment period will be calculated by dose group.
Measured: Baseline to Week 12 and Week 24
Secondary outcomes (2)
Safety of E2020 and placebo in subjects with Down syndrome having regression and disabled ADL.
The safety will be measured by frequencies of treatment-emergent adverse events (TEAEs) in the treatment period, statistics of laboratory parameters, blood pressure, and pulse rate at each evaluation time and changes from before study drug administration, and 12-lead ECG assessment, frequency distribution (yes/no) at each evaluation time will be collected and the percent (%) will be calculated by dose group.
Measured: Up to Week 28
Pharmacokinetics (PK) of E2020 and placebo in subjects with Down syndrome having regression and disabled ADL
Population PK analysis will be performed to build PK models to explain plasma donepezil hydrochloride concentration data. In addition, the models may be used to explore relationship of PK data with demographics, efficacy, and AEs.
Measured: Up to Week 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Mar 2014 (estimated)
- Study start12 Sep 2013
- Primary completion16 Sep 2016
- Study completion21 Apr 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Down SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Eisai Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02094053
- Sponsor's protocol ID
- E2020-J081-345
- Status last verified
- Aug 2017
- First submitted
- 19 Mar 2014
- Last updated
- 22 Aug 2017
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Aug 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double-blind, Placebo-controlled Comparative Study and Open-label Extension Study to Confirm the Efficacy and Safety of E2020 in Subjects With Down Syndrome Having Regression Symptoms and Disabled Activities of Daily Living.
- ClinicalTrials.gov ID
- NCT02094053
- Acronym / sponsor ID
- E2020-J081-345
- Conditions
- Down Syndrome
- Keywords
- Down syndrome, regression symptoms, disabled activities of daily living
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Eisai Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 12 Sep 2013 (actual)
- Primary completion
- 16 Sep 2016 (actual)
- Study completion
- 21 Apr 2017 (actual)
- First posted
- 21 Mar 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this double-blind, placebo-controlled, comparative study and open-label extension study is to confirm the efficacy and safety of E2020 in subjects with Down syndrome having regression symptoms and disabled activities of daily living.
Detailed description
This is a multicenter, randomized, double-blind, multiple-dose (two doses), placebo-controlled, parallel-group comparative study followed by an open-label extension study of E2020 in subjects with Down syndrome having regression and disabled ADL. A total of 60 subjects will be randomized to one of three dosing groups (at 1:1:1) to receive 3 mg of E2020, 5 mg of E2020, or placebo for 24 weeks based on their total scores of Body Functionality Checklist, sex, and study site as the allocation factors. This study consists of Pre-randomization Phase (4 weeks), Double-blind Phase (28 weeks), and Extension Phase (24 weeks). The Double-blind Phase includes a 24-week treatment period plus a 4-week transition period.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| E2020 3 mg | Experimental | Drug: E2020-Donepezil hydrochloride3 mg of E2020 (oral) once daily, for 24 weeks |
| E2020 5 mg | Experimental | Drug: E2020-Donepezil hydrochloride5 mg of E2020 (oral) once daily, for 24 weeks |
| Placebo | Placebo comparator | Drug: Placeboplacebo (oral) once daily, for 24 weeks |
Who may qualify
- Ages
- 15 to 39 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (13 listed)
- At enrollment in Pre-randomization Phase
- With definitive diagnosis of Down syndrome
- Have greater than or equal to 3 of the following 4 symptoms among 9 items according to the diagnostic criteria issued by the Intractable Diseases Treatment Research Program 2010 (Research paper on Intractable Diseases Treatment Research Program; Survey on Sudden Regression (21 trisomy) and Preparation of Diagnostic Criteria.) Motor retardation, mutism, social withdrawal (homeboundness), sleep disorder
- Insufficiently improved with environmental adjustment and psychotherapies including counseling for greater than or equal to 8 weeks before enrollment
- Have a suspected diagnosis with neuropsychiatric disorder without sufficient effect on a disease even after medical treatment for greater than or equal to 8 weeks before enrollment.
Key exclusion criteria (22 listed)
- At enrollment in Pre-randomization Phase
- Suspected to have progressive neuropsychiatric disease (e.g., neurodegenerative disorder and progressive tumor) evidenced by MRI or CT within 1 year before the Pre-randomization Phase (if not tested within 1 year before the Pre-randomization Phase, reconfirm during the Pre-randomization Phase).
- Have a history of significant neurological disorders such as stroke, brain tumor, encephalitis, meningitis, normal pressure hydrocephalus, brain trauma accompanying unconsciousness, and experience of brain surgery causing unsolved deficiency
- Previously diagnosed with autism
- With evidence of atlantoaxial subluxation, or underwent surgical operation for atlantoaxial subluxation within 2 years
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| E2020-Donepezil hydrochloride3 mg of E2020 (oral) once daily, for 24 weeks | Drug | E2020 3 mg |
| E2020-Donepezil hydrochloride5 mg of E2020 (oral) once daily, for 24 weeks | Drug | E2020 5 mg |
| Placeboplacebo (oral) once daily, for 24 weeks | Drug | Placebo |
Primary outcome
Changes in total scores from baseline using Body Functionality Checklist (psychosomatic function questionnaire) in subjects with Down syndrome having regression symptoms and disabled activities of daily living (ADL), relative to placebo.
Time frame: Baseline to Week 12 and Week 24
For the changes in a total score of Body Functionality Checklist (51 items) from Week 0 of the treatment period, Kruskal-Wallis test will be performed in the 3 mg group, the 5 mg group and placebo group to represent statistical significance. Summary statistics of the total score of Body Functionality Checklist (51 items) at each evaluation time and changes from before study drug administration in the treatment period will be calculated by dose group.
2 secondary outcomes
Safety of E2020 and placebo in subjects with Down syndrome having regression and disabled ADL.
Time frame: Up to Week 28
Pharmacokinetics (PK) of E2020 and placebo in subjects with Down syndrome having regression and disabled ADL
Time frame: Up to Week 28
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Aug 2017.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing E2020-Donepezil hydrochloride in people with Down Syndrome, run by Eisai Co., Ltd. at 10 sites in Japan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Aug 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Changes in total scores from baseline using Body Functionality Checklist (psychosomatic function questionnaire) in subjects with Down syndrome having regression symptoms and disabled activities of daily living (ADL), relative to placebo. (time frame: Baseline to Week 12 and Week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Aug 2017.