Reviewed by Michael Gill, B. Sc.
25 Hemophilia Clinical Trials Near Me
Top Hospitals for Hemophilia Clinical Trials
Image of Rush University Medical Center in Illinois.
Rush University Medical Center
Chicago
5Active Trials
14All Time Trials for Hemophilia
2006First Hemophilia Trial
Image of Arkansas Children's Hospital in Arkansas.
Arkansas Children's Hospital
Little Rock
5Active Trials
9All Time Trials for Hemophilia
2011First Hemophilia Trial
Top Cities for Hemophilia Clinical Trials
Most Recent Hemophilia Clinical Trials
Clinical Trial
Began Recruiting Date
Phase
Top Treatments for Hemophilia Clinical Trials
Treatment Name
Active Hemophilia Clinical Trials
All Time Trials for Hemophilia
First Recorded Hemophilia Trial
Emicizumab
4
18
2015
PF-06741086
2
5
2017
efanesoctocog alfa (BIVV001)
2
3
2019
valoctocogene roxaparvovec
2
3
2015
Fitusiran
2
3
2018
Recently Completed Studies with FDA Approved Treatments for Hemophilia
Treatment
Year
Sponsor
efanesoctocog alfa (BIVV001)
2019
Bioverativ, a Sanofi company
Fitusiran
2018
Genzyme, a Sanofi Company
Emicizumab
2017
Hoffmann-La Roche
Emicizumab
2016
Hoffmann-La Roche

About The Author

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 21st, 2021

Last Reviewed: November 13th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.

References1 Oldenburg J, Mahlangu JN, Kim B, Schmitt C, Callaghan MU, Young G, Santagostino E, Kruse-Jarres R, Negrier C, Kessler C, Valente N, Asikanius E, Levy GG, Windyga J, Shima M. Emicizumab Prophylaxis in Hemophilia A with Inhibitors. N Engl J Med. 2017 Aug 31;377(9):809-818. doi: 10.1056/NEJMoa1703068. Epub 2017 Jul 10. https://pubmed.ncbi.nlm.nih.gov/286915572 Blanchette VS, Key NS, Ljung LR, Manco-Johnson MJ, van den Berg HM, Srivastava A; Subcommittee on Factor VIII, Factor IX and Rare Coagulation Disorders of the Scientific and Standardization Committee of the International Society on Thrombosis and Hemostasis. Definitions in hemophilia: communication from the SSC of the ISTH. J Thromb Haemost. 2014 Nov;12(11):1935-9. doi: 10.1111/jth.12672. Epub 2014 Sep 3. https://pubmed.ncbi.nlm.nih.gov/250592853 Shima M, Hanabusa H, Taki M, Matsushita T, Sato T, Fukutake K, Fukazawa N, Yoneyama K, Yoshida H, Nogami K. Factor VIII-Mimetic Function of Humanized Bispecific Antibody in Hemophilia A. N Engl J Med. 2016 May 26;374(21):2044-53. doi: 10.1056/NEJMoa1511769. https://pubmed.ncbi.nlm.nih.gov/272231464 Gringeri A, Leissinger C, Cortesi PA, Jo H, Fusco F, Riva S, Antmen B, Berntorp E, Biasoli C, Carpenter S, Kavakli K, Morfini M, Négrier C, Rocino A, Schramm W, Windyga J, Zülfikar B, Mantovani LG. Health-related quality of life in patients with haemophilia and inhibitors on prophylaxis with anti-inhibitor complex concentrate: results from the Pro-FEIBA study. Haemophilia. 2013 Sep;19(5):736-43. doi: 10.1111/hae.12178. Epub 2013 Jun 4. https://pubmed.ncbi.nlm.nih.gov/237312465 Quinn J, Delaney KA, Wong WY, Miesbach W, Bullinger M. Psychometric Validation of the Haemo-QOL-A in Participants with Hemophilia A Treated with Gene Therapy. Patient Relat Outcome Meas. 2022 Jul 18;13:169-180. doi: 10.2147/PROM.S357555. eCollection 2022. https://pubmed.ncbi.nlm.nih.gov/358799316 Rosen S, Tiefenbacher S, Robinson M, Huang M, Srimani J, Mackenzie D, Christianson T, Pasi KJ, Rangarajan S, Symington E, Giermasz A, Pierce GF, Kim B, Zoog SJ, Vettermann C. Activity of transgene-produced B-domain-deleted factor VIII in human plasma following AAV5 gene therapy. Blood. 2020 Nov 26;136(22):2524-2534. doi: 10.1182/blood.2020005683. https://pubmed.ncbi.nlm.nih.gov/329159507 Levy GG, Asikanius E, Kuebler P, Benchikh El Fegoun S, Esbjerg S, Seremetis S. Safety analysis of rFVIIa with emicizumab dosing in congenital hemophilia A with inhibitors: Experience from the HAVEN clinical program. J Thromb Haemost. 2019 Sep;17(9):1470-1477. doi: 10.1111/jth.14491. Epub 2019 Jun 17. https://pubmed.ncbi.nlm.nih.gov/311242728 Kumar R, Dunn A, Schneiderman JE, Gonzales A, Bouskill V, Widener P, Stanek J, Pluthero FG, Waller A, Tarango C, Ahuja S, Kerlin BA, Kahr WH, Rand ML, Lillicrap D, Carcao M. Moderate-intensity aerobic exercise versus desmopressin in adolescent males with mild hemophilia A: a randomized trial. Blood. 2022 Jul 15. pii: blood.2022016146. doi: 10.1182/blood.2022016146. [Epub ahead of print] https://pubmed.ncbi.nlm.nih.gov/358394509 Young G, Liesner R, Chang T, Sidonio R, Oldenburg J, Jiménez-Yuste V, Mahlangu J, Kruse-Jarres R, Wang M, Uguen M, Doral MY, Wright LY, Schmitt C, Levy GG, Shima M, Mancuso ME. A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors. Blood. 2019 Dec 12;134(24):2127-2138. doi: 10.1182/blood.2019001869. https://pubmed.ncbi.nlm.nih.gov/3169780110 George LA, Monahan PE, Eyster ME, Sullivan SK, Ragni MV, Croteau SE, Rasko JEJ, Recht M, Samelson-Jones BJ, MacDougall A, Jaworski K, Noble R, Curran M, Kuranda K, Mingozzi F, Chang T, Reape KZ, Anguela XM, High KA. Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A. N Engl J Med. 2021 Nov 18;385(21):1961-1973. doi: 10.1056/NEJMoa2104205. https://pubmed.ncbi.nlm.nih.gov/34788507