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Evaluation of the safety and effectiveness of oral propranolol in patients with von Hippel-Lindau disease and retinal hemangioblastomas: phase III clinical trial
  1. Beatriz González-Rodríguez1,
  2. Karina Villar Gómez de las Heras2,3,
  3. Daniel T Aguirre4,
  4. Luis Rodríguez-Padial5,
  5. Virginia Albiñana6,7,
  6. Lucía Recio-Poveda6,
  7. Angel M Cuesta6,7,
  8. Luisa Mª Botella6,7,
  9. Rosa María Jiménez-Escribano1
  1. 1Ophthalmology, Retina Service, Complejo Hospitalario de Toledo, Servicio de Salud de Castilla-La Mancha, Toledo, Spain
  2. 2DG Asistencia Sanitaria, Servicio de Salud de Castilla-La Mancha, Toledo, Spain
  3. 3Alianza VHL, Alianza de Familias de von Hippel-Lindau, Toledo, Spain
  4. 4Neurosurgery, Familial Neuro-Oncology Unit, Hospital Universitario Fundación Jiménez Díaz, Madrid, Spain
  5. 5Cardiology, Complejo Hospitalario de Toledo, Servicio de Salud de Castilla-La Mancha, Toledo, Spain
  6. 6Centro de Investigaciones Biológicas—CIB, Centro Superior de Investigaciones Científicas (CSIC), Madrid, Spain
  7. 7Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Madrid, Spain
  1. Correspondence to Dr Karina Villar Gómez de las Heras; kvillar{at}jccm.es

Footnotes

  • Presented at Two posters about this work were presented at the 9th European Conference on Rare Diseases & Orphan Products (ECRD 2018 Vienna) and at the 13th International VHL Medical/Research Symposium (MD Anderson Cancer Center, Houston, Texas, USA, 2018).

  • Contributors KV and RMJ planned the study. KV wrote the clinical trial protocol and carried out all the authorisation processes and contact with patients. LR participated in the selection of patients to assure they could take propranolol. He performed the cardiological evaluations and follow-up, from a cardiologic point of view. RMJ participated in the evaluation of patients to determine their eligibility according the inclusion criteria. She directed and coordinated the clinical visits, follow-up and complementary diagnostic tests. RMJ and BG performed the ophthalmological evaluations, some of them after the end of the clinical trial. DTA performed the neurological evaluations. LMB directed and coordinated the in vitro experiments, the design strategies and the experimental approach. VA and AMC carried out the experimental work, assisted by Lucia R. BG, RMJ and KV analysed the clinical results. BG wrote the first draft of the document and KV reviewed it and added content. KV, BG and AMC worked in the first review of the document. KV and BG worked in improving the final version of the manuscript. All authors read and approved the final manuscript.

  • Funding This clinical trial was supported by grants from Alianza VHL Spain, Fundación Iberdrola, Bodegas Protos and Fundación Port Aventura. Alianza VHL informed the families about the clinical trial for the recruitment of patients, performed the administrative procedures for authorisation and monitoring and financed the travel expenses of the patients for the visits.

  • Competing interests None declared.

  • Patient consent for publication Not required.

  • Ethics approval The protocol study and informed consent form were reviewed and approved by the Ethical Committee of the Complejo Hospitalario de Toledo. The study was classified by the Spanish Agency of Medicines and Medical Devices (AEMPS) as a phase III clinical trial, authorised, and registered in EU Clinical Trials Register (EMA).

  • Provenance and peer review Not commissioned; externally peer reviewed.

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Footnotes

  • Presented at Two posters about this work were presented at the 9th European Conference on Rare Diseases & Orphan Products (ECRD 2018 Vienna) and at the 13th International VHL Medical/Research Symposium (MD Anderson Cancer Center, Houston, Texas, USA, 2018).

  • Contributors KV and RMJ planned the study. KV wrote the clinical trial protocol and carried out all the authorisation processes and contact with patients. LR participated in the selection of patients to assure they could take propranolol. He performed the cardiological evaluations and follow-up, from a cardiologic point of view. RMJ participated in the evaluation of patients to determine their eligibility according the inclusion criteria. She directed and coordinated the clinical visits, follow-up and complementary diagnostic tests. RMJ and BG performed the ophthalmological evaluations, some of them after the end of the clinical trial. DTA performed the neurological evaluations. LMB directed and coordinated the in vitro experiments, the design strategies and the experimental approach. VA and AMC carried out the experimental work, assisted by Lucia R. BG, RMJ and KV analysed the clinical results. BG wrote the first draft of the document and KV reviewed it and added content. KV, BG and AMC worked in the first review of the document. KV and BG worked in improving the final version of the manuscript. All authors read and approved the final manuscript.

  • Funding This clinical trial was supported by grants from Alianza VHL Spain, Fundación Iberdrola, Bodegas Protos and Fundación Port Aventura. Alianza VHL informed the families about the clinical trial for the recruitment of patients, performed the administrative procedures for authorisation and monitoring and financed the travel expenses of the patients for the visits.

  • Competing interests None declared.

  • Patient consent for publication Not required.

  • Ethics approval The protocol study and informed consent form were reviewed and approved by the Ethical Committee of the Complejo Hospitalario de Toledo. The study was classified by the Spanish Agency of Medicines and Medical Devices (AEMPS) as a phase III clinical trial, authorised, and registered in EU Clinical Trials Register (EMA).

  • Provenance and peer review Not commissioned; externally peer reviewed.

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