Hemophilia A Clinical Trial
Official title:
Emicizumab PUPs and Nuwiq ITI Study
This study prospectively investigates the safety, FVIII immunogenicity, and hemostatic efficacy of prophylactic HEMLIBRA® given with a concomitant low dose recombinant factor VIII (rFVIII) known as NUWIQ®, in HA infants and children <3 years old who have had little to no previous exposure to FVIII. In addition, the study investigates the safety and efficacy of a novel FVIII ITI regimen in children <21 with existing low and high titer inhibitors (LTI and HTI).
Hemophilia A (HA) is a congenital bleeding disorder caused by deficient or dysfunctional factor VIII (FVIII) which leads to bleeding correlated with severity. Management is focused on FVIII replacement in reaction to a bleed or preventive as prophylaxis. Effective treatment is complicated by the: (1) difficulty to administer standard replacement therapy via intravenous injection especially in infants and young children; and (2) development of inhibitors (FVIII neutralizing antibodies). Inhibitors can increase morbidity and mortality and exponentially raise the cost of health care. Although inherited and environmental risk factors for inhibitor formation have been identified, there is no effective strategy to prevent inhibitors from developing. Emicizumab (HEMLIBRA®) was recently approved by the Food and Drug Administration (FDA) in infants, children, and adults with congenital hemophilia A, with and without inhibitors, and offers hemostatic efficacy while reducing the burden of administration since it is given weekly, biweekly (every 2 weeks), or monthly via subcutaneous (SQ) route compared to the intravenous (IV) route of FVIII. This study prospectively investigates the safety, FVIII immunogenicity, and hemostatic efficacy of prophylactic HEMLIBRA® given with a concomitant low dose recombinant factor VIII (rFVIII) known as NUWIQ®, in HA infants and children <3 years old who have had little to no previous exposure to FVIII. In addition, the study investigates the safety and efficacy of a novel FVIII ITI regimen in children <21 with existing low and high titer inhibitors (LTI and HTI). ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT03834727 -
Characterizing the Impact and Treatment of Reproductive Tract Bleeding on Women and Post-menarchal Girls With Bleeding Disorders
|
||
| Completed |
NCT03191799 -
A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors
|
Phase 3 | |
| Completed |
NCT01599819 -
BAX 855 Dose-Escalation Safety Study
|
Phase 1 | |
| Terminated |
NCT04541628 -
Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A
|
Phase 1/Phase 2 | |
| Completed |
NCT02847637 -
A Clinical Trial to Evaluate Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors
|
Phase 3 | |
| Completed |
NCT04072237 -
Study of Coagulation Faction VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia
|
Phase 1 | |
| Completed |
NCT04085458 -
Study to Gain More Information on How Safe and Effective Jivi Works in Patients With Severe Hemophilia A (Post-marketing Investigation)
|
Phase 4 | |
| Completed |
NCT04565236 -
A Post Approval Commitment Study to Gain More Information on How Safe and Effective KOVALTRY is in Chinese Children, Adolescents /Adults With Severe Hemophilia A
|
Phase 4 | |
| Recruiting |
NCT05987449 -
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
|
Phase 1/Phase 2 | |
| Active, not recruiting |
NCT04621916 -
Preventing Inhibitor Recurrence Indefinitely
|
Phase 4 | |
| Not yet recruiting |
NCT02888223 -
Pharmacokinetic Study of SCT800 in Previously Treated Patients With Hemophilia A
|
Phase 1 | |
| Completed |
NCT02528968 -
National Study of a Pharmacokinetic-Focused Educational Package for Patients With Severe Haemophilia A
|
N/A | |
| Completed |
NCT02225483 -
Phenotypic Heterogeneity in Hemophilia A: An Investigation of the Role of Platelet Function
|
N/A | |
| Completed |
NCT02199717 -
An Institutional Pilot Study to Investigate Physical Activity Patterns in Boys With Hemophilia
|
N/A | |
| Completed |
NCT01217255 -
Comparing the Burden of Illness of Hemophilia in the Developing and the Developed World
|
||
| Terminated |
NCT00995046 -
Individually Tailored Prophylaxis in Patients With Severe Hemophilia A
|
N/A | |
| Completed |
NCT00969319 -
Effekt-2 - Efficacy and Safety of Long-term Treatment With KOGENATE® FS in Latin America
|
N/A | |
| Completed |
NCT00868530 -
Study Evaluating On-Demand Treatment Of Xyntha In Chinese Subjects
|
Phase 3 | |
| Completed |
NCT00839202 -
Activity and Content of Factor VIII (FVIII) in Human Plasma: The Assessment of a Novel Immunoassay
|
N/A | |
| Completed |
NCT00629837 -
Pharmacokinetics and Safety of a Single Intravenous Infusion of BAY 79-4980
|
Phase 1 |