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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)

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ClinicalTrials.gov Identifier: NCT02942498
Recruitment Status : Unknown
Verified September 2016 by Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud.
Recruitment status was:  Recruiting
First Posted : October 24, 2016
Last Update Posted : October 24, 2016
Sponsor:
Collaborator:
Delos Clinical
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
X Fragile Syndrome 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.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 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 : July 2016
Estimated Primary Completion Date : October 2017
Estimated Study Completion Date : December 2017


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




Primary Outcome Measures :
  1. Autism Treatment Evaluation Checklist (ATEC). [ Time Frame: 32 weeks ]
  2. Global Clinical Impression (GCI) [ Time Frame: 32 weeks ]
  3. Peabody Picture Vocabulary Test (PiVT) [ Time Frame: 32 weeks ]
  4. Battelle developmental inventory screening [ Time Frame: 32 weeks ]
  5. Vineland Adaptive Behavior Scales [ Time Frame: 32 weeks ]
  6. Adverse event reported [ Time Frame: 32 weeks ]
  7. Quantitative Checklist for Autism in Toddlers (Q-Chat) test [ Time Frame: 32 weeks ]

Secondary Outcome Measures :
  1. Golberg scale GHQ-28 [ Time Frame: 32 weeks ]
  2. Quality life SF36 test [ Time Frame: 32 weeks ]
  3. Psychological General Well-Being Index [ Time Frame: 32 weeks ]
  4. Sleep Disturbance Scale for Children [ Time Frame: 32 weeks ]


Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year to 8 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02942498


Contacts
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Contact: Yolanda De Diego Otero, Ph D yolandadediego@ibima.eu

Locations
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Spain
Hospital Regional de Málaga Recruiting
Malaga, Málaga, Spain, 29009
Contact: Lucia Pérez Costilla, M.D PhD         
Sponsors and Collaborators
Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud
Delos Clinical

Publications:
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Responsible Party: Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud
ClinicalTrials.gov Identifier: NCT02942498     History of Changes
Other Study ID Numbers: SXF2-8
First Posted: October 24, 2016    Key Record Dates
Last Update Posted: October 24, 2016
Last Verified: September 2016
Keywords provided by Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud:
behavior, cognitive
Additional relevant MeSH terms:
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Fragile X Syndrome
Syndrome
Mental Disorders
Child Behavior Disorders
Disease
Pathologic Processes
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Sex Chromosome Disorders
Chromosome Disorders
Congenital Abnormalities
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System
Neurodevelopmental Disorders
Vitamins
Ascorbic Acid
Vitamin E
Tocopherols
Tocotrienols
alpha-Tocopherol
Micronutrients
Nutrients
Growth Substances
Physiological Effects of Drugs
Antioxidants
Molecular Mechanisms of Pharmacological Action