Project Description

Artwork created for the opening of our pediatric ECTU on 29 October 2019. The tree represents the various activities involved in carrying out early pediatric clinical studies.

Center for Pediatrics – Study Office

zkj.studien@uniklinik-freiburg.de

Studienbüro
Center for Pediatrics and Adolescent Medicine
Mathildenstraße 8
79106 Freiburg
Germany

Our pediatric Study Office supports clinical studies from initial planning through completion.

The Study Office is run by a core team. Clinical studies are directed by the relevant medical specialist(s) at the Center for Pediatrics.

Core Team

  • Kristina Schachtrup, project manager, contract administration, budget compilation, controlling
  • Susanne Grüninger, project manager, regulatory affairs
  • Barbara Wirth, study nurse
  • Ruth Fehrenbach-Blug, medical documentation
  • Karsta Spengler, medical documentation

Department of General Pediatrics, Adolescent Medicine and Neonatology

  • Prof. Dr. Ute Spiekerkötter – Congenital metabolic diseases
  • Prof. Dr. Karl Otfried Schwab – Endocrinology and Diabetology
  • Prof. Dr. Patrick Gerner – Gastroenterology
  • Prof. Dr. Markus Hufnagel – Infectious Diseases and Rheumatology
  • Prof. Dr. Hans Fuchs – Intensive care
  • Prof. Dr. Roland Hentschel – Neonatology
  • Prof. Dr.  Martin Pohl – Nephrology

Department of Neuropediatrics and Muscle Disorders

  • Dr. Matthias Eckenweiler – Epileptology, Neuropaediatrics and muscular disorders

Department of Congenital Heart Defects and Pediatric Cardiology

  • Dr. Katja Reineker

Department of Pediatric Hematology and Oncology

  • PD Dr. Simone Hettmer – Hematology and Oncology
  • Prof. Dr. Stephan Ehl – Immunology
  • PD Dr. Brigitte Strahm – Stem cell transplantation
  • Prof. Dr. Barbara Zieger – Hemostasiology
  • Dr. Friedrich Kapp – Vascular malformations

The Center for Pediatrics (ZKJ) in Freiburg offers comprehensive treatment concepts for all childhood diseases. In addition to proven therapies, we provide patients with access to new and innovative therapeutic approaches through appropriate clinical trials. Our institution specializes in general pediatrics including neonatology and intensive care, neuropediatric and muscular diseases in children, congenital heart defects/ pediatric cardiology and pediatric hematology and oncology.

In our pediatric study office, project managers and study assistants work closely with medical staff to guarantee that study participants receive the best possible medical care and logistical support. Currently, our institution is participating in approximately 50 clinical trials and 80 registries. You can find an overview of all studies (in German) here.

Close cooperation with the Clinical Trials Unit at the University Medical Center Freiburg allows for implementation of investigator-initiated clinical trials and access to training/ continuing education in all aspects of clinical trial management. The spectrum of our services ranges from data management, monitoring and pharmacovigilance, to data analysis and publication.

Clinical trials at the ZKJ Freiburg focus on:

  • Fatty acid oxidation disorders
  • Growth disorders
  • Type 2 diabetes mellitus
  • Risk factors for atherosclerosis
  • Antiviral therapy of chronic hepatitis C
  • Complications of preterm birth
  • Complement in chronic kidney disease
  • Juvenile idiopathic arthritis
  • Neuromuscular diseases
  • Epilepsy
  • Congenital or acquired heart defects and myocardial diseases
  • Congenital immunodeficiencies
  • Hemophagocytic lymphohistiocytosis (HLH)
  • Myelodysplastic diseases in childhood
  • Bone marrow failure
  • Juvenile myelomonocytic leukemia
  • Soft tissue sarcoma
  • Vascular malformations and overgrowth syndromes

We publish a newsletter (in German) about the activities of the Study Office (including new studies and publications) three times per year. You can find the current newsletter and the archive here.

Artwork created for the opening of our pediatric ECTU on 29 October 2019. The tree represents the various activities involved in carrying out early pediatric clinical studies.

Children need medication tailored to their needs! However, children and adolescents are significantly under-represented in clinical trials and they are thus disadvantaged in terms of treatment opportunities. Because of the lack of clinical studies, children and adolescents are often given drugs “off label” or “off licence” (i.e., the drug has not been approved for pediatric use for a given condition or, in some cases, has not been approved for pediatric use at all).

The Center for Pediatrics and Adolescent Medicine aims to encourage and promote clinical studies in a wide range of pediatric conditions. This is the only way that children and adolescents can quickly and safely access the innovations and rapid advances that modern medicine offers.

The Early Clinical Trials Unit (ECTU) is the first interdisciplinary unit for early clinical trials for children in Germany. For almost ten years now, the Center for Pediatrics and Adolescent Medicine has been supervising several dozen clinical trials and registries in close cooperation with the Center for Clinical Trials.

In the new ECTU, the necessary processes and structures will be professionalized and consolidated, creating an infrastructure that allows us to initiate and conduct early clinical trials in children and adolescents very quickly, in all areas that include children and adolescent medicine. The ECTU will have its own premises within the new children’s clinic building that is currently being built in Freiburg.

The opening of the ECTU was celebrated on 29 October 2019; an interview (in German) with PD Dr. Simone Hettmer published by the Badische Zeitung newspaper on the occasion can be found here. More information about the ECTU (in German) can be found on our main webpage.

  • A prospective study on the natural history of patients with profound combined immunodeficiency (P-CID): an interim analysis. Speckmann C, Doerken S, Aiuti A, Albert MH, Al-Herz W, Allende LM, Scarselli A, Avcin T, Perez-Becker R, Cancrini C, Cant A, Di Cesare S, Finocchi A, Fischer A, Gaspar HB, Ghosh S, Gennery A, Gilmour K, González-Granado LI, Martinez-Gallo M, Hambleton S, Hauck F, Hoenig M, Moshous D, Neven B, Niehues T, Notarangelo L, Picard C, Rieber N, Schulz A, Schwarz K, Seidel MG, Soler-Palacin P, Stepensky P, Strahm B, Vraetz T, Warnatz K, Winterhalter C, Worth A, Fuchs S, Uhlmann A, Ehl S; PCID-study of the Inborn Errors Working Party of the EBMT. J Allergy Clin Immunol. 2016 Sep 19. pii: S0091-6749(16)30965-4. Abstract
  • A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency. Burton BK1, Balwani M, Feillet F, Barić I, Burrow TA, Camarena Grande C, Coker M, Consuelo-Sánchez A, Deegan P, Di Rocco M, Enns GM, Erbe R, Ezgu F, Ficicioglu C, Furuya KN, Kane J, Laukaitis C, Mengel E, Neilan EG, Nightingale S, Peters H, Scarpa M, Schwab KO, Smolka V, Valayannopoulos V, Wood M, Goodman Z, Yang Y, Eckert S, Rojas-Caro S, Quinn AG. N Engl J Med. 2015 Sep 10;373(11):1010-20. http://bit.ly/2dFsE7R
  • Mutational landscape in children with myelodysplastic syndromes is distinct from adults: specific somatic drivers and novel germline variants. Pastor V, Hirabayashi S, Karow A, Wehrle J, Kozyra EJ, Nienhold R, Ruzaike G, Lebrecht D, Yoshimi A, Niewisch M, Ripperger T, Göhring G, Baumann I, Schwarz S, Strahm B, Flotho C, Skoda RC, Niemeyer CM, Wlodarski MW. Leukemia. 2016 Dec 9. doi: 10.1038/leu.2016.342. http://bit.ly/2kJZJ68
  • Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy. Richard S. Finkel, M.D., Eugenio Mercuri, M.D., Ph.D., Basil T. Darras, M.D., Anne M. Connolly, M.D., Nancy L. Kuntz, M.D., Janbernd Kirschner, M.D., Claudia A. Chiriboga, M.D., M.P.H., Kayoko Saito, M.D., Ph.D., Laurent Servais, M.D., Ph.D., Eduardo Tizzano, M.D., Ph.D., Haluk Topaloglu, M.D., Már Tulinius, M.D., Ph.D., Jacqueline Montes, P.T., Ed.D., N.C.S., Allan M. Glanzman, P.T., D.P.T., P.C.S., Kathie Bishop, Ph.D., Z. John Zhong, Ph.D., Sarah Gheuens, M.D., Ph.D., C. Frank Bennett, Ph.D., Eugene Schneider, M.D., Wildon Farwell, M.D., M.P.H., and Darryl C. De Vivo, M.D., for the ENDEAR Study Group. N Engl J Med 2017; 377:1723-1732 November 2, 2017 DOI: 10.1056/NEJMoa1702752 http://www.nejm.org/doi/full/10.1056/NEJMoa1702752
  • Non-invasive high-frequency oscillatory ventilation in preterm infants: a randomised controlled cross-over trial. Klotz D, Schneider H, Schumann S, et al. Archives of Disease in Childhood – Fetal and Neonatal Edition Published Online First: 16 September 2017. doi: 10.1136/archdischild-2017-313190. http://fn.bmj.com/content/early/2017/09/16/archdischild-2017-313190
  • RAS-pathway mutation patterns define epigenetic subclasses in juvenile myelomonocytic leukemia Daniel B. Lipka, Tania Witte, Reka Toth, Jing Yang, Manuel Wiesenfarth, Peter Nöllke, Alexandra Fischer, David Brocks, Zuguang Gu, Jeongbin Park, Brigitte Strahm, Marcin Wlodarski, Ayami Yoshimi, Rainer Claus, Michael Lübbert, Hauke Busch, Melanie Boerries, Mark Hartmann, Maximilian Schönung, Umut Kilik, Jens Langstein, Justyna A. Wierzbinska, Caroline Pabst, Swati Garg, Albert Catalá, Barbara De Moerloose, Michael Dworzak, Henrik Hasle, Franco Locatelli, Riccardo Masetti, Markus Schmugge, Owen Smith, Jan Stary, Marek Ussowicz, Marry M. van den Heuvel-Eibrink, Yassen Assenov, Matthias Schlesner, Charlotte Niemeyer, Christian Flotho, Christoph Plass. Nature Communications 2017, DOI 10.1038/s41467-017-02177-w. https://www.nature.com/articles/s41467-017-02177-w