Down SyndromePhase 3NCT00056329
Vitamin E in Aging Persons With Down Syndrome
A Phase 3 study testing Vitamin E and multivitamin in people with Down Syndrome and Alzheimer Disease, run by New York State Institute for Basic Research at 22 sites in 4 countries.
Trial summary
The goal of this study is to determine the safety and efficacy of the administration of vitamin E, which has been shown to delay the progression of Alzheimer's disease, in slowing the rate of cognitive/functional decline in older persons with Down syndrome. Read more about this study
- Who is it for?
- Ages 50 years and older
- Conditions the study is for
- Down Syndrome, Alzheimer Disease
- What is being tested?
- Vitamin E and multivitamin
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 350 participants
- Where
- 22 sitesin 4 countries
- Study dates
- Apr 2002to May 2012
- Phase
- Phase 3
- 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 50 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Presence of clinically determined Down syndrome (karyotypes optional).
- Medically stable.
- Medications stable over 3 months.
- Appropriately signed and witnessed consent form.
- Involvement/cooperation of informant/caregiver.
Who cannot join
- Medical/neurological condition (other than Alzheimer's disease) associated with dementia.
- Brief Praxis Test score <20.
- Modified Hachinski score >4.
- Major depression within 3 months.
- History of any disorder of blood coagulation (inherited or acquired).
- Current use of anti-coagulants.
- Use of experimental medications within 3 months.
- Regular use of vitamin E greater than 50 units per day during the previous 6 months.
Trial locations and contacts
22 sites in 4 countries.
22 sites in 4 countries
United States14 sites
University at Albany, SUNY
Albany, New York, 12222
Institute for the Study of Disadvantage and Disability
Atlanta, Georgia, 30342
May South, Inc.
Atlanta, Georgia, 30342
McLean Hospital
Belmont, Massachusetts, 02478
University of Illinois at Chicago
Chicago, Illinois, 60608
University Memory and Aging Center, Case Western Reserve University
Cleveland, Ohio, 44120
University of Connecticut Health Center
Farmington, Connecticut, 06030
Third Age, Inc.
Lexington, Kentucky, 40517
Nathan Kline Institute
Orangeburg, New York, 10962
Southern Illinois University School of Medicine
Springfield, Illinois, 62794-9642
George Jervis Clinic
Staten Island, New York, 10314
Roskamp Institute Memory Clinic
Tampa, Florida, 33617
Westchester Institute for Human Development
Valhalla, New York, 10595
Clinical Research Center of New Jersey
Voorhees Township, New Jersey, 08043
United Kingdom4 sites
University of Cambridge
Cambridge, England, CB2 2AH
Mercer Institute for Research on Ageing, St. James Hospital
Dublin, Ireland, 8
Greenfields Monyhull Hospital
Kings Norton, Birmingham, England, B30 3QQ
Kings College: London
London, England, SE5 8AF
Canada3 sites
Down Syndrome Research Foundation
Port Coquitlam, British Columbia, V3C 2B2
Saskatoon City Hospital
Saskatoon, Saskatchewan, S7K 0M7
Surrey Place Centre
Toronto, Ontario, M5S 2C2
Australia1 site
Centre for Developmental Disabilities Studies
Ryde, New South Wales, 1680
About this study
Official title: Multicenter Vitamin E Trial in Aging Persons With Down Syndrome
The goal of this study is to determine the safety and efficacy of the administration of vitamin E, which has been shown to delay the progression of Alzheimer's disease, in slowing the rate of cognitive/functional decline in older persons with Down syndrome.
Read the detailed description
The growing success of therapeutic interventions (including the antioxidant Vitamin E) for Alzheimer's disease in the general population requires a solution to the methodological problems so that therapeutic trials can be conducted in the aging population with Down syndrome which will ultimately improve their quality of life as well as that of their families and caregivers. The experience gained in this trial will be useful to the design of appropriate cognitive measures of Alzheimer's disease in persons with Down syndrome in subsequent trials.
The goal of this international three-year study is to determine whether the administration of vitamin E, which has been shown to delay the progression of Alzheimer's disease, will slow the rate of cognitive/functional decline in persons age 50 or older with Down syndrome. Persons with Down syndrome functioning at all levels of intellectual disability will be eligible. Men and women of approximately equal numbers and people from minorities and ethnic groups other than Caucasian will be included. A total of 350 individuals with Down syndrome, 50 years of age and older, have been recruited at approximately 21 trial sites. The study is a randomized, double-blind, placebo-controlled, parallel group design with stratification by geographic site and presence of Alzheimer disease according to DSM-IV (American Psychiatric Association) criteria for diagnosing this disease.
Apolipoprotein E (Apo E) genotype will be determined at the screening visit to allow secondary analyses of the impact of Apo E genotype (that may influence Alzheimer's disease risk) on outcome measures and the response to treatment. DNA specimens will also be stored for possible future genetic analyses, with trial sites allowing for non-participation in this procedure. Visits will occur at baseline and then at 6 monthly intervals, with each visit including interval medical history, current and interval medications, side effects checklist, adverse events, pill count, institutionalization status, cognitive, functional, and behavioral measures, and DSM-IV diagnostic assessment for Alzheimer's disease.
- Conditions
- Down Syndrome, Alzheimer Disease
- Being tested
- Vitamin E, multivitamin, Placebo
- Keywords
- Aging Persons, Down Syndrome, Vitamin E, Alzheimer disease
- Medical subject headings
- Down Syndrome, Alzheimer Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Vitamin EDrug
Also called tocopherol
1,000 international units twice daily for three years
multivitaminDrug
once daily for three years
PlaceboDrug
Placebo twice daily for three years
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | vitamin E plus multivitamin Receives: Drug: Vitamin E; Drug: multivitamin |
| 2 | Placebo comparator | placebo with multivitamin Receives: Drug: multivitamin; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Brief Praxis Test, measuring cognitive functions expressed as performances of simple, short, sequences of voluntary movements
Measured: Screening, Baseline, every 6 months for 36 months
Secondary outcomes (1)
Fuld Object Memory Test (Modified), New Dot Test, Orientation Test, Vocabulary Test, Behavior & Function Down Syndrome Rating Scale, Clinical Global Impression of Change (CGI-C)
Measured: Screening, Baseline, and every 6 months for 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Mar 2003 (estimated)
- Study startApr 2002
- Primary completionMay 2012 (estimated)
- Study completionMay 2012 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 350 participants (actual)
Sponsor and investigators
- Lead sponsor
- New York State Institute for Basic Research
- Sponsor type
- Other government
- Collaborators
- National Institute on Aging (NIA), Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), National Center for Complementary and Integrative Health (NCCIH)
- Responsible party
- Principal investigator, Arthur Dalton, New York State Institute for Basic Research
- Investigators
- Arthur J Dalton, PhD, Principal investigator, New York State Institute for Basic Research in Developmental DisabilitiesPaul S Aisen, MD, Study director, Georgetown UniversityMary C Sano, PhD, Study director, Icahn School of Medicine at Mount Sinai
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00056329
- Sponsor's protocol ID
- IA0039
- Other IDs
- R01AG016381, NIA Grant AG16381
- Status last verified
- May 2012
- First submitted
- 10 Mar 2003
- Last updated
- 4 May 2012
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Sano M, Ernesto C, Thomas RG, Klauber MR, Schafer K, Grundman M, Woodbury P, Growdon J, Cotman CW, Pfeiffer E, Schneider LS, Thal LJ. A controlled trial of selegiline, alpha-tocopherol, or both as treatment for Alzheimer's disease. The Alzheimer's Disease Cooperative Study. N Engl J Med. 1997 Apr 24;336(17):1216-22. doi: 10.1056/NEJM199704243361704.
PubMed 9110909Aisen PS, Davis KL. The search for disease-modifying treatment for Alzheimer's disease. Neurology. 1997 May;48(5 Suppl 6):S35-41. doi: 10.1212/wnl.48.5_suppl_6.35s.
PubMed 9153159Aylward EH, Burt DB, Thorpe LU, Lai F, Dalton A. Diagnosis of dementia in individuals with intellectual disability. J Intellect Disabil Res. 1997 Apr;41 ( Pt 2):152-64. doi: 10.1111/j.1365-2788.1997.tb00692.x.
PubMed 9161927Dalton, AJ, Mehta, PD, Fedor, BL, Patti, PJ: Cognitive changes in memory precede those in praxis in aging persons with Down syndrome. Journal of Intellectual and Developmental Disability 24(2):169-187,1999.
Sano M, Aisen PS, Dalton AJ, Andrews HF, Tsai W-Y, and the International Down Syndrome Alzheimer Disease Consortium. Assessment of aging individuals with Down syndrome in clinical trials: results of baseline measures. Journal of Policy and Practice in Intellectual Disabilities. 2005 2(2):126-138.
Study at a glance
From the registry record, last updated by the sponsor on 4 May 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter Vitamin E Trial in Aging Persons With Down Syndrome
- ClinicalTrials.gov ID
- NCT00056329
- Acronym / sponsor ID
- IA0039
- Conditions
- Down Syndrome, Alzheimer Disease
- Keywords
- Aging Persons, Down Syndrome, Vitamin E, Alzheimer disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 350 participants (actual)
- Lead sponsor
- New York State Institute for Basic ResearchOther government
- Collaborators
- National Institute on Aging (NIA), Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), National Center for Complementary and Integrative Health (NCCIH)
- Study start
- Apr 2002
- Primary completion
- May 2012 (estimated)
- Study completion
- May 2012 (estimated)
- First posted
- 12 Mar 2003 (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 goal of this study is to determine the safety and efficacy of the administration of vitamin E, which has been shown to delay the progression of Alzheimer's disease, in slowing the rate of cognitive/functional decline in older persons with Down syndrome.
Detailed description
The growing success of therapeutic interventions (including the antioxidant Vitamin E) for Alzheimer's disease in the general population requires a solution to the methodological problems so that therapeutic trials can be conducted in the aging population with Down syndrome which will ultimately improve their quality of life as well as that of their families and caregivers. The experience gained in this trial will be useful to the design of appropriate cognitive measures of Alzheimer's disease in persons with Down syndrome in subsequent trials.
The goal of this international three-year study is to determine whether the administration of vitamin E, which has been shown to delay the progression of Alzheimer's disease, will slow the rate of cognitive/functional decline in persons age 50 or older with Down syndrome. Persons with Down syndrome functioning at all levels of intellectual disability will be eligible. Men and women of approximately equal numbers and people from minorities and ethnic groups other than Caucasian will be included. A total of 350 individuals with Down syndrome, 50 years of age and older, have been recruited at approximately 21 trial sites. The study is a randomized, double-blind, placebo-controlled, parallel group design with stratification by geographic site and presence of Alzheimer disease according to DSM-IV (American Psychiatric Association) criteria for diagnosing this disease.
Apolipoprotein E (Apo E) genotype will be determined at the screening visit to allow secondary analyses of the impact of Apo E genotype (that may influence Alzheimer's disease risk) on outcome measures and the response to treatment. DNA specimens will also be stored for possible future genetic analyses, with trial sites allowing for non-participation in this procedure. Visits will occur at baseline and then at 6 monthly intervals, with each visit including interval medical history, current and interval medications, side effects checklist, adverse events, pill count, institutionalization status, cognitive, functional, and behavioral measures, and DSM-IV diagnostic assessment for Alzheimer's disease.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: Vitamin E, Drug: multivitaminvitamin E plus multivitamin |
| 2 | Placebo comparator | Drug: multivitamin, Drug: Placeboplacebo with multivitamin |
Who may qualify
- Ages
- 50 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Presence of clinically determined Down syndrome (karyotypes optional).
- Medically stable.
- Medications stable over 3 months.
- Appropriately signed and witnessed consent form.
- Involvement/cooperation of informant/caregiver.
Key exclusion criteria (8 listed)
- Medical/neurological condition (other than Alzheimer's disease) associated with dementia.
- Brief Praxis Test score <20.
- Modified Hachinski score >4.
- Major depression within 3 months.
- History of any disorder of blood coagulation (inherited or acquired).
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 |
|---|---|---|
| Vitamin EAlso: tocopherol1,000 international units twice daily for three years | Drug | 1 |
| multivitaminonce daily for three years | Drug | 1, 2 |
| PlaceboPlacebo twice daily for three years | Drug | 2 |
Primary outcome
Brief Praxis Test, measuring cognitive functions expressed as performances of simple, short, sequences of voluntary movements
Time frame: Screening, Baseline, every 6 months for 36 months
1 secondary outcome
Fuld Object Memory Test (Modified), New Dot Test, Orientation Test, Vocabulary Test, Behavior & Function Down Syndrome Rating Scale, Clinical Global Impression of Change (CGI-C)
Time frame: Screening, Baseline, and every 6 months for 36 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 May 2012.
- Background: PubMed 9110909Sano M, Ernesto C, Thomas RG, Klauber MR, Schafer K, Grundman M, Woodbury P, Growdon J, Cotman CW, Pfeiffer E, Schneider LS, Thal LJ. A controlled trial of selegiline, alpha-tocopherol, or both as treatment for Alzheimer's disease. The Alzheimer's Disease Cooperative Study. N Engl J Med. 1997 Apr 24;336(17):1216-22. doi: 10.1056/NEJM199704243361704.
- Background: PubMed 9153159Aisen PS, Davis KL. The search for disease-modifying treatment for Alzheimer's disease. Neurology. 1997 May;48(5 Suppl 6):S35-41. doi: 10.1212/wnl.48.5_suppl_6.35s.
- Background: PubMed 9161927Aylward EH, Burt DB, Thorpe LU, Lai F, Dalton A. Diagnosis of dementia in individuals with intellectual disability. J Intellect Disabil Res. 1997 Apr;41 ( Pt 2):152-64. doi: 10.1111/j.1365-2788.1997.tb00692.x.
- BackgroundDalton, AJ, Mehta, PD, Fedor, BL, Patti, PJ: Cognitive changes in memory precede those in praxis in aging persons with Down syndrome. Journal of Intellectual and Developmental Disability 24(2):169-187,1999.
- Results publicationSano M, Aisen PS, Dalton AJ, Andrews HF, Tsai W-Y, and the International Down Syndrome Alzheimer Disease Consortium. Assessment of aging individuals with Down syndrome in clinical trials: results of baseline measures. Journal of Policy and Practice in Intellectual Disabilities. 2005 2(2):126-138.
Related trials for Down Syndrome
Not yet recruiting
Executive Function Play Opportunities for Children With Down Syndrome
Down Syndrome, Trisomy 21
Not yet recruitingObservational study
Hematological Profile in Children With Numerical Chromosomal Disorders
Down Syndrome, Turner Syndrome
RecruitingObservational study
Natural History of Dysregulation and Aging of the Immune System in People With Trisomy 21 With and Without Thymectomy
Down Syndrome
Not yet recruiting
Single Session Support for Parents of Children With Neurodevelopmental Disabilities Struggling With Anxious Avoidance
Neurodevelopmental Disorders, Autism Spectrum Disorder (ASD)
RecruitingPhase 2
Treatment of Atopic Dermatitis and Alopecia Areata With Abrocitinib in Individuals With Down Syndrome
Atopic Dermatitis, Alopecia Areata
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.
Arthur J Dalton, PhD principal investigator
New York State Institute for Basic Research in Developmental Disabilities
Paul S Aisen, MD study director
Georgetown University
Mary C Sano, PhD study director
Icahn School of Medicine at Mount Sinai
22 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Vitamin E and multivitamin in people with Down Syndrome and Alzheimer Disease, run by New York State Institute for Basic Research at 22 sites in 4 countries.
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 5 inclusion and 8 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: status not confirmed, as of the sponsor's last update on 4 May 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 22 sites in United States, United Kingdom, Canada, Australia. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
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?
Brief Praxis Test, measuring cognitive functions expressed as performances of simple, short, sequences of voluntary movements (time frame: Screening, Baseline, every 6 months for 36 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00056329, which also lists 5 linked publications.
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 4 May 2012.