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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 (SXF2-8)

Primary Purpose

X Fragile Syndrome

Status

Completed

Phase

Phase 3

Locations

Spain

Study Type

Interventional

Intervention

Vitamin C 10mg/Kg Vitamin E 10 mg/Kg

Placebo

About this trial

This is an interventional treatment trial for X Fragile Syndrome focused on measuring behavior, cognitive

Eligibility Criteria

1 Year - 8 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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.

Exclusion Criteria:

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.
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

Sites / Locations

Hospital Regional de Málaga

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Vitamin C 10 mg/Kg + Vitamin E 10 mg/Kg

Placebo

Arm Description

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

Placebo solution

Outcomes

Primary Outcome Measures

Autism Treatment Evaluation Checklist (ATEC).

Global Clinical Impression (GCI)

Peabody Picture Vocabulary Test (PiVT)

Battelle developmental inventory screening

Vineland Adaptive Behavior Scales

Adverse event reported

Quantitative Checklist for Autism in Toddlers (Q-Chat) test

Secondary Outcome Measures

Golberg scale GHQ-28

Quality life SF36 test

Psychological General Well-Being Index

Sleep Disturbance Scale for Children

Full Information

NCT ID

NCT02942498

First Posted

October 7, 2016

Last Updated

July 21, 2022

Sponsor

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

Collaborators

Delos Clinical

1. Study Identification

Unique Protocol Identification Number

NCT02942498

Brief Title

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

Acronym

SXF2-8

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 Type

Interventional

2. Study Status

Record Verification Date

September 2016

Overall Recruitment Status

Completed

Study Start Date

July 2016 (undefined)

Primary Completion Date

September 25, 2017 (Actual)

Study Completion Date

October 31, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

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

Collaborators

Delos Clinical

4. Oversight

Data Monitoring Committee

5. 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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

X Fragile Syndrome

Keywords

behavior, cognitive

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Parallel Assignment

Masking

ParticipantInvestigator

Allocation

Randomized

Enrollment

34 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Vitamin C 10 mg/Kg + Vitamin E 10 mg/Kg

Arm Type

Experimental

Arm Description

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

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

Placebo solution

Intervention Type

Drug

Intervention Name(s)

Vitamin C 10mg/Kg Vitamin E 10 mg/Kg

Intervention Type

Drug

Intervention Name(s)

Placebo

Intervention Description

Placebo

Primary Outcome Measure Information:

Title

Autism Treatment Evaluation Checklist (ATEC).

Time Frame

32 weeks

Title

Global Clinical Impression (GCI)

Time Frame

32 weeks

Title

Peabody Picture Vocabulary Test (PiVT)

Time Frame

32 weeks

Title

Battelle developmental inventory screening

Time Frame

32 weeks

Title

Vineland Adaptive Behavior Scales

Time Frame

32 weeks

Title

Adverse event reported

Time Frame

32 weeks

Title

Quantitative Checklist for Autism in Toddlers (Q-Chat) test

Time Frame

32 weeks

Secondary Outcome Measure Information:

Title

Golberg scale GHQ-28

Time Frame

32 weeks

Title

Quality life SF36 test

Time Frame

32 weeks

Title

Psychological General Well-Being Index

Time Frame

32 weeks

Title

Sleep Disturbance Scale for Children

Time Frame

32 weeks

10. Eligibility

Sex

All

Minimum Age & Unit of Time

1 Year

Maximum Age & Unit of Time

8 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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. Exclusion Criteria: 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. 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

Facility Information:

Facility Name

Hospital Regional de Málaga

City

Malaga

State/Province

Málaga

ZIP/Postal Code

29009

Country

Spain

12. IPD Sharing Statement

Citations:

PubMed Identifier

14732624

Citation

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.

Results Reference

background

PubMed Identifier

18400731

Citation

Castilla 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.

Results Reference

background

PubMed Identifier

17403030

Citation

Barger 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.

Results Reference

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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

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