SXF2-8: 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

Sponsor
Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud (Other)
Overall Status
Completed
CT.gov ID
NCT02942498
Collaborator
Delos Clinical (Other)
34
1
2
28
1.2

Study Details

Study Description

Brief 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.

Condition or Disease Intervention/Treatment Phase
  • Drug: Vitamin C 10mg/Kg Vitamin E 10 mg/Kg
  • Drug: Placebo
Phase 3

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

Study Type:
Interventional
Actual Enrollment :
34 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Double (Participant, Investigator)
Primary Purpose:
Treatment
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
Study Start Date :
Jul 1, 2016
Actual Primary Completion Date :
Sep 25, 2017
Actual Study Completion Date :
Oct 31, 2018

Arms and Interventions

Arm Intervention/Treatment
Experimental: Vitamin C 10 mg/Kg + Vitamin E 10 mg/Kg

Vitamin C and Vitamin E supplementation 10 mg/kg/ day

Drug: Vitamin C 10mg/Kg Vitamin E 10 mg/Kg

Placebo Comparator: Placebo

Placebo solution

Drug: Placebo
Placebo

Outcome Measures

Primary Outcome Measures

  1. Autism Treatment Evaluation Checklist (ATEC). [32 weeks]

  2. Global Clinical Impression (GCI) [32 weeks]

  3. Peabody Picture Vocabulary Test (PiVT) [32 weeks]

  4. Battelle developmental inventory screening [32 weeks]

  5. Vineland Adaptive Behavior Scales [32 weeks]

  6. Adverse event reported [32 weeks]

  7. Quantitative Checklist for Autism in Toddlers (Q-Chat) test [32 weeks]

Secondary Outcome Measures

  1. Golberg scale GHQ-28 [32 weeks]

  2. Quality life SF36 test [32 weeks]

  3. Psychological General Well-Being Index [32 weeks]

  4. Sleep Disturbance Scale for Children [32 weeks]

Eligibility Criteria

Criteria

Ages Eligible for Study:
1 Year to 8 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion Criteria:
  1. Diagnosis of Fragile X syndrome by genetic testing of molecular biology, full mutation result methylation.

  2. Having an older age of 1 year and less than 9 years

  3. Having signed the informed consent document before starting their participation in the trial.

Exclusion Criteria:
  1. Any advanced, severe or unstable disease.

  2. Individuals with other psychiatric diagnosis as the first diagnosis.

  3. It have been suffered serious medical problems in the last 12 months.

  4. Be taking more than 100 mg of vitamin E or C a day in the last month.

  5. Having physical, mental or sensory impairments that prevent the assessment of effectiveness.

  6. Hypersensitivity to any component of the preparation.

  7. Liver failure or severe renal or previous history of kidney stones.

  8. Any treatment regimen, including treatment with psychotropic drugs and / or anticonvulsant therapy that has not been stable for a period ≥ 4 weeks before randomization.

  9. Current treatment with more than two psychoactive medications, excluding medication used specifically for the control of seizures.

  10. Hypoprothrombinemia secondary to vitamin K deficiency

  11. Sensitivity to any of the compounds of formula treatment.

  12. Patients diagnosed with congenital or idiopathic methemoglobinemia for diagnosis of glucose-6-phosphate dehydrogenase deficiency.

  13. Use of oral anticoagulants, iron or vitamin A.

  14. Forecast initiate or change pharmacological or no pharmacological interventions during the course of the study.

  15. Patients weighing less than 4.2 kg

Contacts and Locations

Locations

Site City State Country Postal Code
1 Hospital Regional de Málaga Malaga Málaga Spain 29009

Sponsors and Collaborators

  • Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud
  • Delos Clinical

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

Responsible Party:
Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud
ClinicalTrials.gov Identifier:
NCT02942498
Other Study ID Numbers:
  • SXF2-8
First Posted:
Oct 24, 2016
Last Update Posted:
Jul 22, 2022
Last Verified:
Sep 1, 2016
Keywords provided by Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud
Additional relevant MeSH terms:

Study Results

No Results Posted as of Jul 22, 2022