Hemophilia A With Inhibitor Clinical Trial
Official title:
Phase III Multi-Center, Randomized, Controlled Inhibitor Eradication Trial, Comparing Eloctate Immune Tolerance Induction (ITI) Plus Emicizumab vs. Eloctate ITI Alone to Eradicate Inhibitor Formation in Severe Hemophilia A
This is a multi-center randomized phase III clinical trial, the Inhibitor Eradication Trial, in which Eloctate ITI plus Emicizumab will be compared with Eloctate ITI alone to eradicate inhibitors in severe hemophilia A.
This is a multi-center randomized phase III clinical trial, the Inhibitor Eradication Trial, in which Eloctate ITI plus Emicizumab will be compared with Eloctate ITI alone to eradicate inhibitors in patients with severe hemophilia A This adaptive design is necessary as randomized trials in rare diseases are often not possible. The INHIBIT Clinical Trials Platform includes two linked trials, the Inhibitor Prevention Trial (Prevention Trial) and the Inhibitor Eradication Trial (Eradication Trial) that will be conducted at up to 41 U.S. hemophilia treatment centers (HTCs) affiliated with universities. The Inhibitor Eradication Trial is a 48-week randomized phase III trial, in which 90 previously treated patients (PTPs) with severe hemophilia A and high-responding inhibitors (anti-VIII > 0.6 B.U.), will be enrolled. Subjects will include individuals with severe hemophilia A who develop inhibitors during the linked Inhibitor Prevention Trial and adults or children at the same HTCs refractory to or never undergoing immune tolerance induction (ITI). Once enrolled, subjects who meet all the inclusion and none of the exclusion criteria, will be randomized to weekly Eloctate ITI plus weekly Emicizumab vs. weekly Eloctate ITI alone to eradicate inhibitor formation, defined as anti-FVIII<0.6 B.U. Blood draws will be minimized to 6 timepoints, pre, 4, 12, 24, 36, and 48 weeks, and validated for small volumes, 3.8 cc (¾ tsp) each. The Inhibitor Eradication Trial is considered greater than minimal risk as study drug is given before the first bleed and special inhibitor studies are obtained. (NB: The Inhibitor Eradication Trial (PRO19070080) is linked to the Inhibitor Prevention Trial (PRO19040140), as part of the INHIBIT Clinical Trials Platform, and both trials will be conducted efficiently in the same hemophilia treatment centers (HTCs), with the same MDs, coordinators, visit frequency, blood sampling, and assays. ;
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT06320626 -
Pharmacokinetic-guided Dosing of Emicizumab
|
Phase 4 | |
Completed |
NCT04072237 -
Study of Coagulation Faction VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia
|
Phase 1 | |
Not yet recruiting |
NCT02554526 -
Basic Study of Combination Therapy Based on APCC-induced FVllI Activation in Hemophilia A Patients With Inhibitors
|
N/A | |
Completed |
NCT03818529 -
ATHN 8: Previously Untreated Patients (PUPs) Matter Study
|
||
Active, not recruiting |
NCT03619863 -
ATHN 7: Hemophilia Natural History Study
|
||
Recruiting |
NCT06010953 -
SS109 and NovoSeven ® PK / PD Profile, and Preliminary Efficacy and Safety of SS109 on Demand Treatment
|
Phase 1/Phase 2 | |
Completed |
NCT03951103 -
rFVIIIFc (Elocta®) ITI Chart Review in Patients With Haemophilia A
|
||
Recruiting |
NCT05888870 -
ITI Using SCT800 Alone or Combining Daratumumab in Hemophilia A Adolescents and Adults With High Titer Inhibitor
|
Phase 4 | |
Terminated |
NCT04489537 -
Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Hemophilia A or B
|
Phase 3 | |
Terminated |
NCT04548791 -
Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Inherited Bleeding Disorders
|
Phase 1/Phase 2 | |
Completed |
NCT06357572 -
Real-life Clinical FEIBA Samples Measured Using the Version A of the HemA EnzySystem
|
||
Recruiting |
NCT04647227 -
SEVENFACT® for Bleeding Events in Hemophilia With Inhibitors
|
Phase 4 | |
Completed |
NCT03372993 -
Prospective, Non-interventional Study to Evaluate Immunogenicity of AryoSeven
|
||
Terminated |
NCT03204539 -
INdividualized ITI Based on Fviii(ATE) Protection by VWF
|
Phase 4 | |
Active, not recruiting |
NCT04205175 -
A Study to Evaluate the Safest Dose Range for FEIBA in Hemophilia A Patients With Inhibitors on Emicizumab
|
Phase 4 | |
Recruiting |
NCT06312475 -
Efficacy and Safety of KN057 Prophylaxis in Patients With Haemophilia A or B With Inhibitors
|
Phase 3 | |
Recruiting |
NCT03598725 -
Low-dose ITI Strategy for Children in Hemophilia A With High-titer Inhibitor and Poor ITI Risk in China
|
Phase 4 | |
Completed |
NCT04723693 -
An Exploration of the Impact of Emicizumab on the Lives of People With Haemophilia and Inhibitors and Their Families
|
||
Completed |
NCT04789954 -
Study on the Dose-response Relationship of Pharmacodynamic Parameters in Patients With Hemophilia With Inhibitors
|
Early Phase 1 | |
Recruiting |
NCT04592692 -
A Pharmacokinetic and Clotting Activity Study of FVIII-PEGLip
|
Phase 2 |