X Fragile SyndromePhase 3SXF2-8NCT02942498
Clinical Trial for Asses the Safety and Efficacy of Vitamin C and Vitamin E in Combination Versus Placebo for Treating Cognitive and Behavior Disorder in Children With Fragile X Syndrome
A Phase 3 study testing Vitamin C 10mg/Kg Vitamin E 10 mg/Kg in people with X Fragile Syndrome, run by Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud at 1 site in Spain.
Trial summary
The purpose of this study is to determine vitamin C and vitamin E in combination are effective in the treatment of cognitive and behavior disorder in children with fragile X syndrome. Read more about this study
- Who is it for?
- Ages 1 year to 8 years
- Conditions the study is for
- X Fragile Syndrome
- What is being tested?
- Vitamin C 10mg/Kg Vitamin E 10 mg/Kg
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 34 participants
- Where
- 1 siteSpain
- Study dates
- Jul 2016to 31 Oct 2018
- 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 1 year to 8 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Diagnosis of Fragile X syndrome by genetic testing of molecular biology, full mutation result methylation.
- Having an older age of 1 year and less than 9 years
- Having signed the informed consent document before starting their participation in the trial.
Who cannot join
- Any advanced, severe or unstable disease.
- Individuals with other psychiatric diagnosis as the first diagnosis.
- It have been suffered serious medical problems in the last 12 months.
- Be taking more than 100 mg of vitamin E or C a day in the last month.
- Having physical, mental or sensory impairments that prevent the assessment of effectiveness.
- Hypersensitivity to any component of the preparation.
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- Liver failure or severe renal or previous history of kidney stones.
- Any treatment regimen, including treatment with psychotropic drugs and / or anticonvulsant therapy that has not been stable for a period ≥ 4 weeks before randomization.
- Current treatment with more than two psychoactive medications, excluding medication used specifically for the control of seizures.
- Hypoprothrombinemia secondary to vitamin K deficiency
- Sensitivity to any of the compounds of formula treatment.
- Patients diagnosed with congenital or idiopathic methemoglobinemia for diagnosis of glucose-6-phosphate dehydrogenase deficiency.
- Use of oral anticoagulants, iron or vitamin A.
- Forecast initiate or change pharmacological or no pharmacological interventions during the course of the study.
- Patients weighing less than 4.2 kg
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Clinical Trials Phase III, Double Blind, Crossover to Asses the Safety and Efficacy of Vitamin C and Vitamin E in Combination Versus Placebo for Treating Cognitive and Behavior Disorder in Children With Fragile X Syndrome
The purpose of this study is to determine vitamin C and vitamin E in combination are effective in the treatment of cognitive and behavior disorder in children with fragile X syndrome.
Read the detailed description
The combination of vitamin E and vitamin C supplementation has been associated with a lower prevalence (-78%) and incidence (-64%) of Alzheimer's disease in the elderly population. It has recently been shown that dietary vitamin E supplementation reduces the production of free radicals inhibiting NADPH oxidase activity in circulating neutrophils. Another work describes the inhibition of glutamate release by activated microglia in cell cultures incubated with vitamin E, effect that can prevent excitotoxicity.
The investigators propose to evaluate the effectiveness of treatment in neurodevelopmental disorders affected by fragile X syndrome (FXS) with lipophilic compounds antioxidants such as tocopherol and hydrophilic compounds antioxidants such as ascorbic acid, which regulate oxidative stress and improve learning and behavioral mouse model and humans.
Our group has positive results in the use of this combination of antioxidants as a treatment for fragile X syndrome in adolescents. This disease has developed previous clinical trials with EUDRACT codes: 2009-017837-23 and 2013-004276-35.
The use of the combination of vitamin C and E in the treatment of cognitive and behavioral disorder in FXS, is patented PCT-050 187 with reference number 2011070875
This combination will be administered as a single oral dose with a total dose of 10mg / kg / day for each of the vitamins. This dose is maintained within the therapeutic range of both antioxidants.
- Conditions
- X Fragile Syndrome
- Being tested
- Vitamin C 10mg/Kg Vitamin E 10 mg/Kg, Placebo
- Keywords
- behavior, cognitive
- Medical subject headings
- Fragile X Syndrome, Behavior
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Vitamin C 10mg/Kg Vitamin E 10 mg/KgDrug
PlaceboDrug
Placebo
| Study group | Role | What they receive |
|---|---|---|
| Vitamin C 10 mg/Kg + Vitamin E 10 mg/Kg | Experimental | Vitamin C and Vitamin E supplementation 10 mg/kg/ day Receives: Drug: Vitamin C 10mg/Kg Vitamin E 10 mg/Kg |
| Placebo | Placebo comparator | Placebo solution Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Autism Treatment Evaluation Checklist (ATEC).
Measured: 32 weeks
Global Clinical Impression (GCI)
Measured: 32 weeks
Peabody Picture Vocabulary Test (PiVT)
Measured: 32 weeks
Battelle developmental inventory screening
Measured: 32 weeks
Vineland Adaptive Behavior Scales
Measured: 32 weeks
Adverse event reported
Measured: 32 weeks
Quantitative Checklist for Autism in Toddlers (Q-Chat) test
Measured: 32 weeks
Secondary outcomes (4)
Golberg scale GHQ-28
Measured: 32 weeks
Quality life SF36 test
Measured: 32 weeks
Psychological General Well-Being Index
Measured: 32 weeks
Sleep Disturbance Scale for Children
Measured: 32 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Oct 2016 (estimated)
- Study startJul 2016
- Primary completion25 Sep 2017
- Study completion31 Oct 2018
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 X Fragile 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
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 34 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud
- Sponsor type
- University, hospital or other
- Collaborators
- Delos Clinical
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02942498
- Sponsor's protocol ID
- SXF2-8
- Acronym
- SXF2-8
- Status last verified
- Sep 2016
- First submitted
- 7 Oct 2016
- Last updated
- 22 Jul 2022
- Results posted
- No
- Data monitoring committee
- No
Publications
Zandi PP, Anthony JC, Khachaturian AS, Stone SV, Gustafson D, Tschanz JT, Norton MC, Welsh-Bohmer KA, Breitner JC; Cache County Study Group. Reduced risk of Alzheimer disease in users of antioxidant vitamin supplements: the Cache County Study. Arch Neurol. 2004 Jan;61(1):82-8. doi: 10.1001/archneur.61.1.82.
PubMed 14732624Castilla P, Davalos A, Teruel JL, Cerrato F, Fernandez-Lucas M, Merino JL, Sanchez-Martin CC, Ortuno J, Lasuncion MA. Comparative effects of dietary supplementation with red grape juice and vitamin E on production of superoxide by circulating neutrophil NADPH oxidase in hemodialysis patients. Am J Clin Nutr. 2008 Apr;87(4):1053-61. doi: 10.1093/ajcn/87.4.1053.
PubMed 18400731Barger SW, Goodwin ME, Porter MM, Beggs ML. Glutamate release from activated microglia requires the oxidative burst and lipid peroxidation. J Neurochem. 2007 Jun;101(5):1205-13. doi: 10.1111/j.1471-4159.2007.04487.x. Epub 2007 Mar 30.
PubMed 17403030
Study at a glance
From the registry record, last updated by the sponsor on 22 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Trials Phase III, Double Blind, Crossover to Asses the Safety and Efficacy of Vitamin C and Vitamin E in Combination Versus Placebo for Treating Cognitive and Behavior Disorder in Children With Fragile X Syndrome
- ClinicalTrials.gov ID
- NCT02942498
- Acronym / sponsor ID
- SXF2-8 / SXF2-8
- Conditions
- X Fragile Syndrome
- Keywords
- behavior, cognitive
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 34 participants (actual)
- Lead sponsor
- Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y SaludUniversity, hospital or other
- Collaborators
- Delos Clinical
- Study start
- Jul 2016
- Primary completion
- 25 Sep 2017 (actual)
- Study completion
- 31 Oct 2018 (actual)
- First posted
- 24 Oct 2016 (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 vitamin C and vitamin E in combination are effective in the treatment of cognitive and behavior disorder in children with fragile X syndrome.
Detailed description
The combination of vitamin E and vitamin C supplementation has been associated with a lower prevalence (-78%) and incidence (-64%) of Alzheimer's disease in the elderly population. It has recently been shown that dietary vitamin E supplementation reduces the production of free radicals inhibiting NADPH oxidase activity in circulating neutrophils. Another work describes the inhibition of glutamate release by activated microglia in cell cultures incubated with vitamin E, effect that can prevent excitotoxicity.
The investigators propose to evaluate the effectiveness of treatment in neurodevelopmental disorders affected by fragile X syndrome (FXS) with lipophilic compounds antioxidants such as tocopherol and hydrophilic compounds antioxidants such as ascorbic acid, which regulate oxidative stress and improve learning and behavioral mouse model and humans.
Our group has positive results in the use of this combination of antioxidants as a treatment for fragile X syndrome in adolescents. This disease has developed previous clinical trials with EUDRACT codes: 2009-017837-23 and 2013-004276-35.
The use of the combination of vitamin C and E in the treatment of cognitive and behavioral disorder in FXS, is patented PCT-050 187 with reference number 2011070875
This combination will be administered as a single oral dose with a total dose of 10mg / kg / day for each of the vitamins. This dose is maintained within the therapeutic range of both antioxidants.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Vitamin C 10 mg/Kg + Vitamin E 10 mg/Kg | Experimental | Drug: Vitamin C 10mg/Kg Vitamin E 10 mg/KgVitamin C and Vitamin E supplementation 10 mg/kg/ day |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo solution |
Who may qualify
- Ages
- 1 year to 8 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Diagnosis of Fragile X syndrome by genetic testing of molecular biology, full mutation result methylation.
- Having an older age of 1 year and less than 9 years
- Having signed the informed consent document before starting their participation in the trial.
Key exclusion criteria (15 listed)
- Any advanced, severe or unstable disease.
- Individuals with other psychiatric diagnosis as the first diagnosis.
- It have been suffered serious medical problems in the last 12 months.
- Be taking more than 100 mg of vitamin E or C a day in the last month.
- Having physical, mental or sensory impairments that prevent the assessment of effectiveness.
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 C 10mg/Kg Vitamin E 10 mg/Kg | Drug | Vitamin C 10 mg/Kg + Vitamin E 10 mg/Kg |
| PlaceboPlacebo | Drug | Placebo |
Primary outcome
Autism Treatment Evaluation Checklist (ATEC).
Time frame: 32 weeks
Global Clinical Impression (GCI)
Time frame: 32 weeks
Peabody Picture Vocabulary Test (PiVT)
Time frame: 32 weeks
Battelle developmental inventory screening
Time frame: 32 weeks
Vineland Adaptive Behavior Scales
Time frame: 32 weeks
Adverse event reported
Time frame: 32 weeks
Quantitative Checklist for Autism in Toddlers (Q-Chat) test
Time frame: 32 weeks
4 secondary outcomes
Golberg scale GHQ-28
Time frame: 32 weeks
Quality life SF36 test
Time frame: 32 weeks
Psychological General Well-Being Index
Time frame: 32 weeks
Sleep Disturbance Scale for Children
Time frame: 32 weeks
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 Jul 2022.
- Background: PubMed 14732624Zandi PP, Anthony JC, Khachaturian AS, Stone SV, Gustafson D, Tschanz JT, Norton MC, Welsh-Bohmer KA, Breitner JC; Cache County Study Group. Reduced risk of Alzheimer disease in users of antioxidant vitamin supplements: the Cache County Study. Arch Neurol. 2004 Jan;61(1):82-8. doi: 10.1001/archneur.61.1.82.
- Background: PubMed 18400731Castilla P, Davalos A, Teruel JL, Cerrato F, Fernandez-Lucas M, Merino JL, Sanchez-Martin CC, Ortuno J, Lasuncion MA. Comparative effects of dietary supplementation with red grape juice and vitamin E on production of superoxide by circulating neutrophil NADPH oxidase in hemodialysis patients. Am J Clin Nutr. 2008 Apr;87(4):1053-61. doi: 10.1093/ajcn/87.4.1053.
- Background: PubMed 17403030Barger SW, Goodwin ME, Porter MM, Beggs ML. Glutamate release from activated microglia requires the oxidative burst and lipid peroxidation. J Neurochem. 2007 Jun;101(5):1205-13. doi: 10.1111/j.1471-4159.2007.04487.x. Epub 2007 Mar 30.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Vitamin C 10mg/Kg Vitamin E 10 mg/Kg in people with X Fragile Syndrome, run by Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud at 1 site in Spain.
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 3 inclusion and 15 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 Jul 2022. 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 Spain. 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?
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?
Autism Treatment Evaluation Checklist (ATEC). (time frame: 32 weeks). The study lists 7 primary outcomes in all. 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 NCT02942498, 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 22 Jul 2022.