NCT03273998 - Clinical Trial References

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

Citation(s)

Bolton-Maggs PH
The rare inherited coagulation disorders. Pediatr Blood Cancer. 2013;60 Suppl 1:S37-40. doi: 10.1002/pbc.24336. Epub 2012 Oct 25. Review.
Ehrenforth S, Klarmann D, Zabel B, Scharrer I, Kreuz W
Severe factor V deficiency presenting as subdural haematoma in the newborn. Eur J Pediatr. 1998 Dec;157(12):1032.
KENDALL AG, LOWENSTEIN L
Alterations in blood coagulation and hemostasis during extracorporeal circulation. II. Can Med Assoc J. 1962 Oct 20;87:859-64.
Kulkarni R, Soucie JM
Pediatric hemophilia: a review. Semin Thromb Hemost. 2011 Oct;37(7):737-44. doi: 10.1055/s-0031-1297164. Epub 2011 Dec 20. Review.
Mannucci PM, Duga S, Peyvandi F
Recessively inherited coagulation disorders. Blood. 2004 Sep 1;104(5):1243-52. Epub 2004 May 11. Review.
Peyvandi F, Mannucci PM
Rare coagulation disorders. Thromb Haemost. 1999 Oct;82(4):1207-14. Review.
von der Lippe C, Frich JC, Harris A, Solbraekke KN
Treatment of hemophilia: A qualitative study of mothers' perspectives. Pediatr Blood Cancer. 2017 Jan;64(1):121-127. doi: 10.1002/pbc.26167. Epub 2016 Jul 29.

Cross-sectional Study on Prevalence of Coagulation Factors Deficiency in Children Attending Assiut University Children Hospital ( a One Year Study)

Clinical trial NCT03273998 - References