Down SyndromePhase 2/3NCT00294593
Efficacy Study of Folinic Acid to Improve Mental Development of Children With Down Syndrome
A Phase 2/3 study testing folinic acid in people with Down Syndrome, run by Institut Jerome Lejeune at 1 site in France.
Trial summary
The purpose of this study is to determine whether folinic acid can improve developmental quotient of young Down syndrome patients, given that these present signs of folate deficiency which are known to cause reversible neurological, psychiatric and cognitive disorders. Read more about this study
- Who is it for?
- Ages 3 to 30 months
- Conditions the study is for
- Down Syndrome
- What is being tested?
- folinic acid
- How many people
- 120 participants
- Where
- 1 siteFrance
- Study dates
- Oct 2000to Dec 2003
- Phase
- Phase 2/3
- 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 3 to 30 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Down syndrome without mosaicism
- age 3 to 30 months
- weight over 4 kg
- possible assessment by the revised Brunet-Lezine scale
Who cannot join
- history of leukemia
- West syndrome or non-stable epilepsy
- non-stable thyroxin treatment
Trial locations and contacts
1 site in 1 country.
About this study
Official title: One-year Double-blind Placebo-controlled Phase 2-3 Study to Evaluate the Effect of Oral Folinic Acid Treatment (1mg/kg/d) on the Psychomotor Development of Young Down Syndrome Patients
The purpose of this study is to determine whether folinic acid can improve developmental quotient of young Down syndrome patients, given that these present signs of folate deficiency which are known to cause reversible neurological, psychiatric and cognitive disorders.
- Conditions
- Down Syndrome
- Being tested
- folinic acid
- Keywords
- Down syndrome, folinic acid therapy, cognitive functions
- Medical subject headings
- Down Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
folinic acidDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
Score on a psychomotor development scale after a 6 and 12 months treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Feb 2006 (estimated)
- Study startOct 2000
- Study completionDec 2003
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Enrolment
- 120 participants
Sponsor and investigators
- Lead sponsor
- Institut Jerome Lejeune
- Sponsor type
- University, hospital or other
- Collaborators
- Fondation Jérôme Lejeune
- Investigators
- Henri Blehaut, M.D., Study director, Institut Jerome LejeuneClotilde Mircher, MD, Principal investigator, Institut Jerome Lejeune
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00294593
- Sponsor's protocol ID
- IJL-FA-TH01
- Status last verified
- Dec 2009
- First submitted
- 21 Feb 2006
- Last updated
- 17 Dec 2009
- Results posted
- No
Publications
Botez MI, Botez T, Leveille J, Bielmann P, Cadotte M. Neuropsychological correlates of folic acid deficiency: facts and hypotheses. In: Botez MI, Reynolds EH, eds. Folic acid in neurology, psychiatry and internal medicine. New York, Raven Press, 1979:435-461.
Lejeune J. [Pathogenesis of mental impairment in trisomy 21]. Ann Genet. 1991;34(2):55-64. French.
PubMed 1836122Blehaut H, Mircher C, Ravel A, Conte M, de Portzamparc V, Poret G, de Kermadec FH, Rethore MO, Sturtz FG. Effect of leucovorin (folinic acid) on the developmental quotient of children with Down's syndrome (trisomy 21) and influence of thyroid status. PLoS One. 2010 Jan 11;5(1):e8394. doi: 10.1371/journal.pone.0008394.
PubMed 20084109
Study at a glance
From the registry record, last updated by the sponsor on 17 Dec 2009. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- One-year Double-blind Placebo-controlled Phase 2-3 Study to Evaluate the Effect of Oral Folinic Acid Treatment (1mg/kg/d) on the Psychomotor Development of Young Down Syndrome Patients
- ClinicalTrials.gov ID
- NCT00294593
- Acronym / sponsor ID
- IJL-FA-TH01
- Conditions
- Down Syndrome
- Keywords
- Down syndrome, folinic acid therapy, cognitive functions
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 120 participants
- Lead sponsor
- Institut Jerome LejeuneUniversity, hospital or other
- Collaborators
- Fondation Jérôme Lejeune
- Study start
- Oct 2000
- Study completion
- Dec 2003
- First posted
- 22 Feb 2006 (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 study is to determine whether folinic acid can improve developmental quotient of young Down syndrome patients, given that these present signs of folate deficiency which are known to cause reversible neurological, psychiatric and cognitive disorders.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
Who may qualify
- Ages
- 3 to 30 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Down syndrome without mosaicism
- age 3 to 30 months
- weight over 4 kg
- possible assessment by the revised Brunet-Lezine scale
Key exclusion criteria (3 listed)
- history of leukemia
- West syndrome or non-stable epilepsy
- non-stable thyroxin treatment
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 |
|---|---|---|
| folinic acid | Drug | – |
Primary outcome
Score on a psychomotor development scale after a 6 and 12 months treatment
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 17 Dec 2009.
- BackgroundBotez MI, Botez T, Leveille J, Bielmann P, Cadotte M. Neuropsychological correlates of folic acid deficiency: facts and hypotheses. In: Botez MI, Reynolds EH, eds. Folic acid in neurology, psychiatry and internal medicine. New York, Raven Press, 1979:435-461.
- Background: PubMed 1836122Lejeune J. [Pathogenesis of mental impairment in trisomy 21]. Ann Genet. 1991;34(2):55-64. French.
- Derived from this study: PubMed 20084109Blehaut H, Mircher C, Ravel A, Conte M, de Portzamparc V, Poret G, de Kermadec FH, Rethore MO, Sturtz FG. Effect of leucovorin (folinic acid) on the developmental quotient of children with Down's syndrome (trisomy 21) and influence of thyroid status. PLoS One. 2010 Jan 11;5(1):e8394. doi: 10.1371/journal.pone.0008394.
Related trials for Down Syndrome
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Down Syndrome
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.
Henri Blehaut, M.D. study director
Institut Jerome Lejeune
Clotilde Mircher, MD principal investigator
Institut Jerome Lejeune
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing folinic acid in people with Down Syndrome, run by Institut Jerome Lejeune at 1 site in France.
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 4 inclusion and 3 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 17 Dec 2009. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined 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.
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?
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?
Score on a psychomotor development scale after a 6 and 12 months treatment. 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 NCT00294593, which also lists 3 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 17 Dec 2009.