Fragile x syndrome MedDRA version: 19.0 Level: PT Classification code 10017324 Term: Fragile X syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: Vitamin C
Pharmaceutical Form: Oral solution
INN or Proposed INN: Vitamin C
Other descriptive name: ACIDUM ASCORBICUM D6
Concentration unit: mg/ml milligram(s)/millilitre
Concentration t
Sponsors
Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. Diagnosis of Fragile X syndrome by genetic testing of molecular biology, with full mutation result methylation. 2. Patients older than 2 years and under 9 years. 3. Signed the informed consent document before starting their participation in the trial. Are the trial subjects under 18? yes Number of subjects for this age range: 30 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Any advanced, severe or unstable disease. 2. Individuals with other psychiatric diagnosed as the first diagnosis. 3. Suffer serious medical problems in the last 12 months. 4. Previous treatment with 100 mg of vitamin E or C a day in the last month. 5. Any 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 prior to randomization. 9. Current treatment with more than two psychoactive drugs, excluding medication used specifically for seizure control. 10. Hypoprothrombinemia secondary to vitamin K deficiency. 11. Sensitivity to any of the compounds of formula treatment. 12. Deficiency of G-6-PD. 13. Use of oral anticoagulants. 14. Suspicion of change the pharmacological or non-pharmacological interventions during the course of the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To Evaluate the safety, tolerability and efficacy of the combination of two antioxidants ascorbic acid and tocopherol, for the treatment of cognitive and behavioral disorders in children diagnosed of fragile X syndrome.;Secondary Objective: 1. To evaluate the safety and tolerability of Vitamin C 10 mg / kg / day and vitamin E 10 mg / kg / day in the treatment of subjects in the pediatric population diagnosed with Fragile X Syndrome. 2.To evaluate the reduction of the overall clinical severity of pediatric subjects diagnosed with Fragile X Syndrome. 3. To evaluate the improvement of cognitive symptoms associated with pediatric subjects with a diagnosis of fragile X syndrome. 4. To evaluate the overall improvement of sleep in pediatric subjects with a diagnosis of fragile X syndrome. 5. To evaluate the improvement of language in pediatric subjects diagnosed with Fragile X Syndrome. 6. To evaluate the effectiveness of treatment to 32 weeks of pediatric subjects with a diagnosis of fragile X syndrome. 7. To evaluate the improvement of quality of life and health of primary caregivers of pediatric subjects with a diagnosis of fragile X syndrome.;Primary end point(s): ICG scale (Guy 1976);Timepoint(s) of evaluation of this end point: 32 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - McCarthy test - TVIP - ATEC - Vineland scale - IDEA - SDSC - SF36 - IBP - GHQ-28 - Nutritional questionnaire - DBC-P;Timepoint(s) of evaluation of this end point: 40 weeks | — |
Countries
Spain
Contacts
Public ContactClinical Trial Assistant
Delos Clinical
Outcome results
None listed