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University of Bergen
Pediatric Follow-up Group

Innovative Therapies for Children

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The Pediatric Clinical Trials Unit at Haukeland University Hospital was established in 2007 as the first of its kind in Northern Europe and has a large track record on running pediatric clinical trials (Phase I-IV). At Stavanger University Hospital the Pediatric Clinical Trials Unit was established in 2018. There is a broad collaboration both with the industry and international academic milieus. Several of the trials are funded from EU: Incentive and Vaccelerate.

NorPedMed is a national network to increase clinical drug trials for children and has been funded by Norwegian Government since 2013. NorPedMed is partner in the EU-funded PedCRIN “The Paediatric Clinical Research Infrastructure Network” and Conect4Children “Collaborative Network for European Clinical Trials for Children” as well as part of NordicPedMed funded by Nordic Council since 2015.

NorCRIN was established as the Norwegian branch of ECRIN in 2012 and has been funded from Norwegian Research Counsel since 2015. The secretariat of NorCRIN and NorPedMed are both placed in Bergen and collaborate well to increase clinical studies in Norway.

 

Peer review publications

  1. Wallace EL, Goker-Alpan O, Wilcox WR, Holida M, Bernat J, Longo N, Linhart A, Hughes DA, Hopkin RJ, Tøndel C, Langeveld M, Giraldo P, Pisani A, Germain DP, Mehta A, Deegan PB, Molnar MJ, Ortiz D, Jovanovic A, Muriello M, Barshop BA, Kimonis V, Vujkovac B, Nowak A, Geberhiwot T, Kantola I, Knoll J, Waldek S, Nedd K, Karaa A, Brill-Almon E, Alon S, Chertkoff R, Rocco R, Sakov A, Warnock DG. Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study. J Med Genet. 2024 May 21;61(6):520-530. doi: 10.1136/jmg-2023-109445. PMID: 37940383; PMCID: PMC11137442.
  2. Linhart A, Dostálová G, Nicholls K, West ML, Tøndel C, Jovanovic A, Giraldo P, Vujkovac B, Geberhiwot T, Brill-Almon E, Alon S, Chertkoff R, Rocco R, Hughes D. Safety and efficacy of pegunigalsidase alfa in patients with Fabry disease who were previously treated with agalsidase alfa: results from BRIDGE, a phase 3 open-label study. Orphanet J Rare Dis. 2023 Oct 21;18(1):332. doi: 10.1186/s13023-023-02937-6. PMID: 37865771.
  3. Bradley JS, Makieieva N, Tøndel C, Roilides E, Kelly MS, Patel M, Vaddady P, Maniar A, Zhang Y, Paschke A, Chen LF. Pharmacokinetics, Safety, and Tolerability of Imipenem/Cilastatin/Relebactam in Children with Confirmed or Suspected Gram-Negative Bacterial Infections: A Phase 1b, Open-Label, Single-Dose Clinical Trial. J Clin Pharmacol. 2023 Aug 10. doi: 10.1002/jcph.2334. Epub ahead of print. PMID: 37562063.
  4. Lynggaard LS, Vaitkeviciene G, Langenskiöld C, Lehmann AK, Lähteenmäki PM, Lepik K, El Hariry I, Schmiegelow K, Albertsen BK. Asparaginase encapsulated in erythrocytes as second-line treatment in hypersensitive patients with acute lymphoblastic leukaemia. Br J Haematol. 2022 Jun;197(6):745-754. doi: 10.1111/bjh.18152. Epub 2022 Mar 28. PMID: 35344210.
  5. van der Veen SJ, Körver S, Hirsch A, Hollak CEM, Wijburg FA, Brands MM, Tøndel C, van Kuilenburg ABP, Langeveld M. Early start of enzyme replacement therapy in pediatric male patients with classical Fabry disease is associated with attenuated disease progression. Mol Genet Metab. 2022 Feb;135(2):163-169. doi: 10.1016/j.ymgme.2021.12.004. Epub 2021 Dec 17. PMID: 35033446.
  6. Casabona G, Habib MA, Povey M, Riise Bergsaker MA, Flodmark CE, Espnes KA, Tøndel C, Silfverdal SA. Randomised controlled trial showed long-term efficacy, immunogenicity and safety of varicella vaccines in Norwegian and Swedish children. Acta Paediatr. 2022 Feb;111(2):391-400. doi: 10.1111/apa.16136. Epub 2021 Oct 28. PMID: 34606114; PMCID: PMC9297935.
  7. van der Zanden TM, Mooij MG, Vet NJ, Neubert A, Rascher W, Lagler FB, Male C, Grytli H, Halvorsen T, de Hoog M, de Wildt SN. Benefit-Risk Assessment of Off-Label Drug Use in Children: The Bravo Framework. Clin Pharmacol Ther. 2021 Oct;110(4):952-965. doi: 10.1002/cpt.2336. Epub 2021 Jul 29. PMID: 34145575; PMCID: PMC8518427.
  8. Baka-Ostrowska M, Bolong DT, Persu C, Tøndel C, Steup A, Lademacher C, Martin N. Efficacy and safety of mirabegron in children and adolescents with neurogenic detrusor overactivity: An open-label, phase 3, dose-titration study. Neurourol Urodyn. 2021 Aug;40(6):1490-1499. doi: 10.1002/nau.24657. Epub 2021 May 31. PMID: 34058027; PMCID: PMC8361978.
  9. Arrieta AC, Ang JY, Espinosa C, Fofanov O, Tøndel C, Chou MZ, De Anda CS, Kim JY, Li D, Sabato P, Sears PS, Bradley JS. Pharmacokinetics and Safety of Single-dose Tedizolid Phosphate in Children 2 to <12 Years of Age. Pediatr Infect Dis J. 2021 Apr 1;40(4):317-323. doi: 10.1097/INF.0000000000003030. PMID: 33710976.
  10. Moreno-Martinez D, Aguiar P, Auray-Blais C, Beck M, Bichet DG, Burlina A, Cole D, Elliott P, Feldt-Rasmussen U, Feriozzi S, Fletcher J, Giugliani R, Jovanovic A, Kampmann C, Langeveld M, Lidove O, Linhart A, Mauer M, Moon JC, Muir A, Nowak A, Oliveira JP, Ortiz A, Pintos-Morell G, Politei J, Rozenfeld P, Schiffmann R, Svarstad E, Talbot AS, Thomas M, Tøndel C, Warnock D, West ML, Hughes DA. Standardising clinical outcomes measures for adult clinical trials in Fabry disease: A global Delphi consensus. Mol Genet Metab. 2021 Apr;132(4):234-243. doi: 10.1016/j.ymgme.2021.02.001. Epub 2021 Feb 20. PMID: 33642210.
  11. Halton J, Brandão LR, Luciani M, Bomgaars L, Chalmers E, Mitchell LG, Nurmeev I, Sharathkumar A, Svirin P, Gorbatikov K, Tartakovsky I, Simetzberger M, Huang F, Sun Z, Kreuzer J, Gropper S, Reilly P, Brueckmann M, Albisetti M; DIVERSITY Trial Investigators. Dabigatran etexilate for the treatment of acute venous thromboembolism in children (DIVERSITY): a randomised, controlled, open-label, phase 2b/3, non-inferiority trial. Lancet Haematol. 2021 Jan;8(1):e22-e33. doi: 10.1016/S2352-3026(20)30368-9. Epub 2020 Dec 5. PMID: 33290737.
  12. Santos RD, Ruzza A, Hovingh GK, Wiegman A, Mach F, Kurtz CE, Hamer A, Bridges I, Bartuli A, Bergeron J, Szamosi T, Santra S, Stefanutti C, Descamps OS, Greber-Platzer S, Luirink I, Kastelein JJP, Gaudet D; HAUSER-RCT Investigators. Evolocumab in Pediatric Heterozygous Familial Hypercholesterolemia. N Engl J Med. 2020 Oct 1;383(14):1317-1327. doi: 10.1056/NEJMoa2019910. Epub 2020 Aug 29. PMID: 32865373.
  13. Mohn KG, Brokstad KA, Islam S, Oftung F, Tøndel C, Aarstad HJ, Cox RJ. Early Induction of Cross-Reactive CD8+ T-Cell Responses in Tonsils After Live-Attenuated Influenza Vaccination in Children. J Infect Dis. 2020 Apr 7;221(9):1528-1537. doi: 10.1093/infdis/jiz583. PMID: 32255493; PMCID: PMC7137893.
  14. Rittig S, Baka-Ostrowska M, Tøndel C, Walle JV, Kjaeer B, Passier P, Bosman B, Stroosma O, Tannenbaum S. The pharmacokinetics, safety, and tolerability of mirabegron in children and adolescents with neurogenic detrusor overactivity or idiopathic overactive bladder and development of a population pharmacokinetic model-based pediatric dose estimation. J Pediatr Urol. 2020 Feb;16(1):31.e1-31.e10. doi: 10.1016/j.jpurol.2019.10.009. Epub 2019 Oct 22. PMID: 31787582.
  15. Brandão LR, Albisetti M, Halton J, Bomgaars L, Chalmers E, Mitchell LG, Nurmeev I, Svirin P, Kuhn T, Zapletal O, Tartakovsky I, Simetzberger M, Huang F, Sun Z, Kreuzer J, Gropper S, Brueckmann M, Luciani M; DIVERSITY Study Investigators. Safety of dabigatran etexilate for the secondary prevention of venous thromboembolism in children. Blood. 2020 Feb 13;135(7):491-504. doi: 10.1182/blood.2019000998. Erratum in: Blood. 2020 May 7;135(19):1720. PMID: 31805182; PMCID: PMC7019192.
  16. Naumburg E, Rane A, Halvorsen T, Glosli H, Henriksen TB, Haraldsson À, Kallio J, Lepola P. Tardy development of safe medicines for children: a Nordic network offers new platform to reduce this inequity. Acta Paediatr. 2019 Jun;108(6):992-993. doi: 10.1111/apa.14775. Epub 2019 Apr 7. PMID: 30844088.
  17. Hem IR, Gjerstad C, Berg A. Bruk av insentiver til deltagere i medisinsk og helsefaglig forskning i Norge [Use of incentives for participants in medical and health research in Norway]. Tidsskr Nor Laegeforen. 2019 Feb 11;139(3). Norwegian. doi: 10.4045/tidsskr.17.0958. PMID: 30754941.
  18. Ramaswami U, Bichet DG, Clarke LA, Dostalova G, Fainboim A, Fellgiebel A, Forcelini CM, An Haack K, Hopkin RJ, Mauer M, Najafian B, Scott CR, Shankar SP, Thurberg BL, Tøndel C, Tylki-Szymanska A, Bénichou B, Wijburg FA. Low-dose agalsidase beta treatment in male pediatric patients with Fabry disease: A 5-year randomized controlled trial. Mol Genet Metab. 2019 May;127(1):86-94. doi: 10.1016/j.ymgme.2019.03.010. Epub 2019 Apr 3. PMID: 30987917.
  19. Mikalsen IB, Karlstad Ø, Furu K, Øymar K. Prescribing of asthma drugs for children 2004-2015. Tidsskr Nor Laegeforen. 2018 Feb 19;138(4). English, Norwegian. doi: 10.4045/tidsskr.17.0227. PMID: 29460600.
  20. Berg A, Arntzen E. Ny og bedre spesialistutdanning for legene [New and better specialist training for the physicians]. Tidsskr Nor Laegeforen. 2017 Sep 5;137(17). Norwegian. doi: 10.4045/tidsskr.17.0603. PMID: 28925194.
  21. Mohn KG, Brokstad KA, Pathirana RD, Bredholt G, Jul-Larsen Å, Trieu MC, Lartey SL, Montomoli E, Tøndel C, Aarstad HJ, Cox RJ. Live Attenuated Influenza Vaccine in Children Induces B-Cell Responses in Tonsils. J Infect Dis. 2016 Sep 1;214(5):722-31. doi: 10.1093/infdis/jiw230. Epub 2016 May 30. Erratum in: J Infect Dis. 2016 Oct 3;: PMID: 27247344; PMCID: PMC4978372.
  22. Berg A, Gjerstad C, Ruyter KW. Å skille diagnostikk og forskning [Distinguishing diagnostics from research]. Tidsskr Nor Laegeforen. 2016 Feb 23;136(4):302. Norwegian. doi: 10.4045/tidsskr.15.1352. PMID: 26905843.
  23. Wijburg FA, Bénichou B, Bichet DG, Clarke LA, Dostalova G, Fainboim A, Fellgiebel A, Forcelini C, An Haack K, Hopkin RJ, Mauer M, Najafian B, Scott CR, Shankar SP, Thurberg BL, Tøndel C, Tylki-Szymańska A, Ramaswami U. Characterization of early disease status in treatment-naive male paediatric patients with Fabry disease enrolled in a randomized clinical trial. PLoS One. 2015 May 8;10(5):e0124987. doi: 10.1371/journal.pone.0124987. PMID: 25955246; PMCID: PMC4425695.
  24. Mohn KG, Bredholt G, Brokstad KA, Pathirana RD, Aarstad HJ, Tøndel C, Cox RJ. Longevity of B-cell and T-cell responses after live attenuated influenza vaccination in children. J Infect Dis. 2015 May 15;211(10):1541-9. doi: 10.1093/infdis/jiu654. Epub 2014 Nov 25. PMID: 25425696; PMCID: PMC4407761.

 

Related persons 
Camilla Tøndel's picture

Camilla Tøndel

Professor, Head of Teaching

Department of Clinical Science

  • +47 924 68 362

camilla.tondel@uib.no

Thomas Halvorsen's picture

Thomas Halvorsen

Professor

Department of Clinical Science

thomas.halvorsen@uib.no

Ingvild Bruun Mikalsen's picture

Ingvild Bruun Mikalsen

Associate Professor

Department of Clinical Science

ingvild.mikalsen@uib.no

12.06.2024