Long-acting recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in children: Results of a phase 3 trial
The result's identifiers
Result code in IS VaVaI
<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00064203%3A_____%2F16%3A10333367" target="_blank" >RIV/00064203:_____/16:10333367 - isvavai.cz</a>
Alternative codes found
RIV/00216208:11130/16:10333367 RIV/00843989:_____/16:E0106485
Result on the web
<a href="http://dx.doi.org/10.1160/TH16-03-0179" target="_blank" >http://dx.doi.org/10.1160/TH16-03-0179</a>
DOI - Digital Object Identifier
<a href="http://dx.doi.org/10.1160/TH16-03-0179" target="_blank" >10.1160/TH16-03-0179</a>
Alternative languages
Result language
angličtina
Original language name
Long-acting recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in children: Results of a phase 3 trial
Original language description
A global phase 3 study evaluated the pharmacokinetics, efficacy and safety of a recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in 27 previously treated male children (1-11 years) with severe and moderately severe haemophilia B (factor IX [FIX] activity LESS-THAN OR EQUAL TO2 IU/dl). All patients received routine prophylaxis once every seven days for up to 77 weeks, and treated any bleeding episodes on-demand. The mean terminal half-life of rIX-FP was 91.4 hours (h), 4.3-fold longer than previous FIX treatment and clearance was 1.11 ml/h/kg, 6.4-fold slower than previous FIX treatment. The median (Q1, Q3) annualised spontaneous bleeding rate was 0.00 (0.00, 0.91) and was similar between the <6 years and GREATER-THAN OR EQUAL TO6 years age groups, with a weekly median prophylactic dose of 46 IU/kg. In addition, patients maintained a median trough level of 13.4 IU/dl FIX activity on weekly prophylaxis. Overall, 97.2 % of bleeding episodes were successfully treated with one or two injections of rIX-FP (95 % CI: 92 % to 99 %), 88.7 % with one injection, and 96 % of the treatments were rated effective (excellent or good) by the Investigator. No patient developed FIX inhibitors and no safety concerns were identified. These results indicate that rIX-FP is safe and effective for preventing and treating bleeding episodes in children with haemophilia B with weekly prophylaxis. Routine prophylaxis with rIX-FP at treatment intervals of up to 14 days are currently being investigated in children with severe and moderately severe haemophilia B. Clinicaltrials.gov (NCT01662531). (C) Schattauer 2016.
Czech name
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Czech description
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Classification
Type
J<sub>x</sub> - Unclassified - Peer-reviewed scientific article (Jimp, Jsc and Jost)
CEP classification
FD - Oncology and haematology
OECD FORD branch
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Result continuities
Project
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Continuities
I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace
Others
Publication year
2016
Confidentiality
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů
Data specific for result type
Name of the periodical
Thrombosis and Haemostasis
ISSN
0340-6245
e-ISSN
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Volume of the periodical
116
Issue of the periodical within the volume
4
Country of publishing house
DE - GERMANY
Number of pages
10
Pages from-to
659-668
UT code for WoS article
000384964200012
EID of the result in the Scopus database
2-s2.0-84989158710