Severe Hemophilia A Clinical Trial
Official title:
Phase III INHIBIT Platform: Prevention Trial, Eloctate vs Emicizumab to Prevent Inhibitors; Eradication Trial: Eloctate Immune Tolerance (ITI) Plus Emicizumab vs vs Eloctate ITI Alone to Eradicate Inhibitors in Severe Hemophilia A
This study will evaluate if Eloctate is superior to Emicizumab in reducing inhibitors in children with severe hemophilia when given before the first bleed (preemptive) and continued weekly to prevent bleeds (prophylaxis); and whether Eloctate immune tolerance induction (ITI) plus emicizumab is superior to Eloctate ITI alone in eradicating inhibitor formation in children and adults with severe hemophilia A.
This is a multi-center, randomized Phase III Clinical Trials Platform (INHIBIT) in which hemostatic agents will be compared using adaptive design to prevent and eradicate inhibitors in patients with severe hemophilia A. This adaptive design is necessary as randomized trials in rare diseases are otherwise not possible. The INHIBIT Trial Platform includes one Inhibitor Prevention Trial and one Inhibitor Eradication Trial that will be conducted at up to 41 U.S. hemophilia treatment centers (HTCs) affiliated with universities. The Inhibitor Prevention Trial is a 48-week randomized phase III trial in which 66 previously untreated patients (PUPs) (children < 6 yr) with severe hemophilia A will be enrolled and randomized to preemptive weekly Eloctate vs. Emicizumab to prevent inhibitor formation, defined as anti-FVIII > 5.0 BU. 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 >5.0 B.U.), including subjects developing inhibitors during the Prevention Trials and adults or children of any age at the same HTCs refractory to or never previously tolerated, will be enrolled and randomized to Eloctate ITI god plus weekly Emicizumab vs. 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 Inhibit Trials Platform is considered greater than minimal risk as study drug is given before the first bleed and special inhibitor studies are obtained. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Withdrawn |
NCT01051544 -
Study of First TIME Immunotolerance Induction in Severe Hemophilia A Patients With Inhibitor at High Risk of Failure: Comparison With FVIII Concentrates With or Without Von Willebrand Factor - RES.I.S.T. Naive
|
N/A | |
| Completed |
NCT02172950 -
An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A
|
Phase 3 | |
| Completed |
NCT01051076 -
Rescue Immunotolerance Study in Induction of Immune Tolerance (ITI)-Experienced Patients (RES.I.S.T. Experienced)
|
N/A | |
| Completed |
NCT03376516 -
Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Paediatric Patients With Severe Haemophilia A
|
Phase 3 | |
| Completed |
NCT01181128 -
Study to Evaluate the Safety, Pharmacokinetics and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Subjects With Severe Hemophilia A
|
Phase 3 | |
| Not yet recruiting |
NCT06136507 -
Study of Efficacy and Safety of FRSW107 in Pediatric Patients With Severe Hemophilia A
|
Phase 3 | |
| Completed |
NCT02083965 -
Pharmacokinetics of rFVIIIFc at Two Vial Strengths
|
Phase 1 | |
| Completed |
NCT01085344 -
Canadian Hemophilia Prophylaxis Study
|
Phase 4 | |
| Completed |
NCT04864743 -
A Study to Evaluate the Pharmacokinetics，Safety and Tolerability of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection
|
Phase 1 | |
| Completed |
NCT01341912 -
Study to Investigate the Long-term Efficacy and Safety of Human-cl rhFVIII in Previously Treated Patients (PTPs)
|
Phase 3 | |
| Completed |
NCT01125813 -
Efficacy and Safety Study of Human-cl rhFVIII in PTPs With Severe Hemophilia A
|
Phase 3 | |
| Active, not recruiting |
NCT05181618 -
A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis
|
Phase 4 | |
| Terminated |
NCT04046848 -
Safety and Pharmacokinetics of Subcutaneous Injection of OCTA101 in Adult Patients With Severe Hemophilia A
|
Phase 1/Phase 2 | |
| Completed |
NCT06137092 -
rFVIII-Fc (Produced by AryoGen Pharmed Co.) Pharmacokinetic Study
|
Phase 3 | |
| Terminated |
NCT01405742 -
Hemophilia Adult Prophylaxis Study
|
Phase 3 | |
| Active, not recruiting |
NCT04431726 -
A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors
|
Phase 3 | |
| Recruiting |
NCT05935358 -
Nuwiq for Perioperative Management Of Patients With Haemophilia A on Emicizumab Regular Prophylaxis Study
|
Phase 4 | |
| Completed |
NCT01712438 -
Human Cell Line-derived Recombinant Factor VIII (Human-cl-rhFVIII) in Previously Untreated Patients
|
Phase 3 | |
| Recruiting |
NCT06142552 -
Phase 3 Clinical Project of Pegylated Recombinant Human Coagulation Factor VIII-Fc Fusion Protein
|
Phase 3 | |
| Completed |
NCT02954575 -
Clinical Study to Investigate the PK, Efficacy, and Safety of Wilate in Patients With Severe Hemophilia A
|
Phase 3 |