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

Phase 3

Completed

• Conditions: X Fragile Syndrome
• Interventions: Drug: Vitamin C 10mg/Kg Vitamin E 10 mg/Kg; Drug: Placebo

• Registration Number: NCT02942498
• Lead Sponsor: Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud

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.

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 Timeline

• Study Start: 2016-07
• Status Verified: 2016-09
• Study First Submitted: 2016-10-07
• Study First Submitted QC: 2016-10-21
• Study First Posted: 2016-10-24
• Primary Completion: 2017-09-25
• Study Completion: 2018-10-31
• Last Update Submitted: 2022-07-21
• Last Update Posted: 2022-07-22

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 34

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Vitamin C 10 mg/Kg + Vitamin E 10 mg/Kg	Vitamin C 10mg/Kg Vitamin E 10 mg/Kg	Vitamin C and Vitamin E supplementation 10 mg/kg/ day
Placebo	Placebo	Placebo solution

Primary Outcome Measures

Name	Time	Method
Autism Treatment Evaluation Checklist (ATEC).	32 weeks
Global Clinical Impression (GCI)	32 weeks
Battelle developmental inventory screening	32 weeks
Vineland Adaptive Behavior Scales	32 weeks
Adverse event reported	32 weeks
Peabody Picture Vocabulary Test (PiVT)	32 weeks
Quantitative Checklist for Autism in Toddlers (Q-Chat) test	32 weeks

Secondary Outcome Measures

Name	Time	Method
Golberg scale GHQ-28	32 weeks
Quality life SF36 test	32 weeks
Psychological General Well-Being Index	32 weeks
Sleep Disturbance Scale for Children	32 weeks

Trial Locations

Locations (1)

Hospital Regional de Málaga; 🇪🇸 Malaga, Málaga, Spain