A combination of ascorbic acid and α-tocopherol to test the effectiveness and safety in the fragile X syndrome: study protocol for a phase II, randomized, placebo-controlled trial
| Autor: | Lourdes Sánchez-Salido, Francisco Garcia-Guirado, Yolanda de Diego-Otero, Ignacio del Arco-Herrera, Rafaela Caballero-Andaluz, Teresa Ferrando-Lucas, Carolina Quintero-Navarro, Isabel Fernandez-Carvajal, Lucía Pérez-Costillas, R. Calvo-Medina |
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| Přispěvatelé: | [de Diego-Otero,Y, Quintero-Navarro,C, Sánchez-Salido,L, García-Guirado,F, Pérez-Costilla,L] Unidad de Gestión Clínica de Salud Mental, Hospital Regional Universitario de Málaga. Instituto de Investigación Biomédica de Málaga (IBIMA), Málaga, Spain. [Calvo-Medina,R] Unidad de Gestión Clínica de Pediatría, Hospital Regional Universitario de Málaga, Málaga, Spain. [del Arco-Herrera, I ] Infobiotic, Málaga, Spain. [Fernández-Carvajal,I] Unidad de Genética Molecular de la Enfermedad, Instituto de Biología y Genética Molecular (IBGM)-CSIC-Universidad de Valladolid, Valladolid, Spain. [Ferrando-Lucas,T] Servicio de Neuropediatría, Hospital Quirón, Madrid, Spain. [Caballero-Andaluz,R] Departamento de Psiquiatría, Facultad de Medicina, Universidad de Sevilla, Sevilla, Spain. [Pérez-Costilla,L] Departamento de Psiquiatría, Facultad de Medicina, Universidad de Málaga, Málaga, Spain. [[Diego-Otero, Y de] Unidad de Gestión Clínica de Salud Mental, Hospital Regional Universitario de Málaga, Laboratorio de Investigación, Málaga, Spain., the Spanish Ministry of Health, Research Funds from FEDER-EU (TRA152, EC10-191 and EC11-434), the Health Department of the Andalusian Regional Government (PI09-0507), the Economic Innovation and Science Regional Government (CTS546 and P10-CTS-05704) and the Jerome Lejeune Foundation (Paris, France)., Ministerio de Sanidad, Servicios Sociales e Igualdad (España), European Commission, Junta de Andalucía, Jérôme Lejeune Foundation |
| Rok vydání: | 2014 |
| Předmět: |
Male
Pediatrics Síndrome del cromosoma X frágil Time Factors Phenomena and Processes::Physical Phenomena::Time::Time Factors [Medical Subject Headings] España alpha-Tocopherol Placebo-controlled study Medicine (miscellaneous) Analytical Diagnostic and Therapeutic Techniques and Equipment::Investigative Techniques::Methods::Research Design [Medical Subject Headings] Child Behavior ComputingMilieux_LEGALASPECTSOFCOMPUTING Pilot Projects Ascorbic Acid Alfa-tocoferol Antioxidants Trial law.invention Organisms::Eukaryota::Animals::Chordata::Vertebrates::Mammals::Primates::Haplorhini::Catarrhini::Hominidae::Humans [Medical Subject Headings] Chemicals and Drugs::Chemical Actions and Uses::Pharmacologic Actions::Molecular Mechanisms of Pharmacological Action::Antioxidants [Medical Subject Headings] Study Protocol Child Development Cognition Randomized controlled trial Clinical Protocols law Chemicals and Drugs::Pharmaceutical Preparations::Drug Combinations [Medical Subject Headings] Pharmacology (medical) Analytical Diagnostic and Therapeutic Techniques and Equipment::Therapeutics::Clinical Protocols [Medical Subject Headings] Masculino Child GeneralLiterature_REFERENCE(e.g. dictionaries encyclopedias glossaries) Geographicals::Geographic Locations::Europe::Spain [Medical Subject Headings] Wechsler Scales Wechsler Adult Intelligence Scale Experimental treatment Checklist Fragile X syndrome Drug Combinations Treatment Outcome Research Design Named Groups::Persons::Age Groups::Adolescent [Medical Subject Headings] Psychiatry and Psychology::Behavior and Behavior Mechanisms::Behavior::Child Behavior [Medical Subject Headings] Ácido ascórbico Clinical psychology medicine.medical_specialty Adolescent Phenomena and Processes::Physiological Phenomena::Physiological Processes::Growth and Development::Human Development::Child Development [Medical Subject Headings] Check Tags::Male [Medical Subject Headings] Double-Blind Method Rating scale medicine Chemicals and Drugs::Biological Factors::Biological Markers [Medical Subject Headings] Diseases::Congenital Hereditary and Neonatal Diseases and Abnormalities::Congenital Abnormalities::Chromosome Disorders::Sex Chromosome Disorders::Fragile X Syndrome [Medical Subject Headings] Humans Health Care::Health Care Quality Access and Evaluation::Quality of Health Care::Health Care Evaluation Mechanisms::Data Collection::Checklist [Medical Subject Headings] Psychiatry and Psychology::Behavior and Behavior Mechanisms::Human Development::Adolescent Development [Medical Subject Headings] Named Groups::Persons::Age Groups::Child [Medical Subject Headings] Analytical Diagnostic and Therapeutic Techniques and Equipment::Diagnosis::Prognosis::Treatment Outcome [Medical Subject Headings] Psychiatry and Psychology::Psychological Phenomena and Processes::Mental Processes::Cognition [Medical Subject Headings] Analytical Diagnostic and Therapeutic Techniques and Equipment::Investigative Techniques::Epidemiologic Methods::Epidemiologic Study Characteristics as Topic::Pilot Projects [Medical Subject Headings] business.industry Psychiatry and Psychology::Behavior and Behavior Mechanisms::Behavior::Adolescent Behavior [Medical Subject Headings] Adolescent Development medicine.disease Ascorbic acid Clinical trial Adolescent Behavior Spain Oxidative stress Data_GENERAL Chemicals and Drugs::Heterocyclic Compounds::Heterocyclic Compounds 2-Ring::Benzopyrans::Vitamin E::Tocopherols::alpha-Tocopherol [Medical Subject Headings] Fragile X Syndrome Autism Psychiatry and Psychology::Behavioral Disciplines and Activities::Psychological Tests::Aptitude Tests::Intelligence Tests::Wechsler Scales [Medical Subject Headings] Resultado del tratamiento business Biomarkers |
| Zdroj: | Trials Digital.CSIC. Repositorio Institucional del CSIC instname |
| ISSN: | 1745-6215 |
| Popis: | This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0). [Background]: Fragile X syndrome (FXS) is an inherited neurodevelopmental condition characterised by behavioural, learning disabilities, phisical and neurological symptoms. In addition, an important degree of comorbidity with autism is also present. Considered a rare disorder affecting both genders, it first becomes apparent during childhood with displays of language delay and behavioural symptoms.Main aim: To show whether the combination of 10 mg/kg/day of ascorbic acid (vitamin C) and 10 mg/kg/day of α-tocopherol (vitamin E) reduces FXS symptoms among male patients ages 6 to 18 years compared to placebo treatment, as measured on the standardized rating scales at baseline, and after 12 and 24 weeks of treatment. Secondary aims: To assess the safety of the treatment. To describe behavioural and cognitive changes revealed by the Developmental Behaviour Checklist Short Form (DBC-P24) and the Wechsler Intelligence Scale for Children-Revised. To describe metabolic changes revealed by blood analysis. To measure treatment impact at home and in an academic environment. [Methods/Design]: A phase II randomized, double-blind pilot clinical trial. Scope: male children and adolescents diagnosed with FXS, in accordance with a standardized molecular biology test, who met all the inclusion criteria and none of the exclusion criteria. Instrumentation: clinical data, blood analysis, Wechsler Intelligence Scale for Children-Revised, Conners parent and teacher rating scale scores and the DBC-P24 results will be obtained at the baseline (t0). Follow up examinations will take place at 12 weeks (t1) and 24 weeks (t2) of treatment. [Discussion]: A limited number of clinical trials have been carried out on children with FXS, but more are necessary as current treatment possibilities are insufficient and often provoke side effects. In the present study, we sought to overcome possible methodological problems by conducting a phase II pilot study in order to calculate the relevant statistical parameters and determine the safety of the proposed treatment. The results will provide evidence to improve hyperactivity control and reduce behavioural and learning problems using ascorbic acid (vitamin C) and α-tocopherol (vitamin E). The study protocol was approved by the Regional Government Committee for Clinical Trials in Andalusia and the Spanish agency for drugs and health products. [Trial registration]: ClinicalTrials.gov Identifier: NCT01329770 (29 March 2011). The trial protocol is approved and funded by the Spanish Ministry of Health, Research Funds from FEDER-EU (TRA152, EC10-191 and EC11-434), the Health Department of the Andalusian Regional Government (PI09-0507), the Economic Innovation and Science Regional Government (CTS546 and P10-CTS-05704) and the Jerome Lejeune Foundation (Paris, France). YDDO is the recipient of a Nicolas Monarde contract from the Servicio Andaluz de Salud. Consejería de Salud. Junta de Andalucía. |
| Databáze: | OpenAIRE |
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