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
Verified date | January 2023 |
Source | University of Pittsburgh |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Terminated |
Enrollment | 1 |
Est. completion date | June 27, 2022 |
Est. primary completion date | June 27, 2022 |
Accepts healthy volunteers | No |
Gender | Male |
Age group | N/A and older |
Eligibility | Inclusion Criteria: 1. Male adults or children > 4 months of age. 2. Severe hemophilia A (FVIII < 0.01 U/ml). 3. Current or past high-responding inhibitor, anti-FVIII >= 5.0 B.U., ITI-refractory or ITI-naive. Exclusion Criteria: 1. Acquired hemophilia or any bleeding disorder other than hemophilia A. 2. Current use of Emicizumab, or if used, > 8 weeks since last treatment. 3. Use of an experimental drug(s). 4. Surgery anticipated in the next 48 weeks. 5. Life expectancy less than 5 years. 6. Patient/parent/caretaker unable or unwilling to keep a personal diary of bleeding frequency and study drug treatment, make monthly visits and blood draws at weeks 4, 8, 12, 24, 36, and 48. 7. Other illness, condition, or reason in the opinion of the investigator that would make the patient unsuitable for the trial. |
Country | Name | City | State |
---|---|---|---|
United States | Hemophilia Center of Western PA | Pittsburgh | Pennsylvania |
United States | University of Pittsburgh and Hemophilia Center Western PA | Pittsburgh | Pennsylvania |
Lead Sponsor | Collaborator |
---|---|
Margaret Ragni | Health Resources and Services Administration (HRSA) |
United States,
Bertolet M, Brooks MM, Ragni MV. The design of a Bayesian platform trial to prevent and eradicate inhibitors in patients with hemophilia. Blood Adv. 2020 Nov 10;4(21):5433-5441. doi: 10.1182/bloodadvances.2020002789. — View Citation
Ebbert PT, Xavier F, Malec LM, Seaman CD, Ragni MV. Observational study of recombinant factor VIII-Fc, rFVIIIFc, in hemophilia A. Thromb Res. 2020 Nov;195:51-54. doi: 10.1016/j.thromres.2020.07.004. Epub 2020 Jul 5. — View Citation
Ragni MV, George LA; Members of Working Group 1, the NHLBI State of the Science Workshop on factor VIII inhibitors: Generating a national blueprint for future research. The national blueprint for future factor VIII inhibitor clinical trials: NHLBI State of the Science (SOS) Workshop on factor VIII inhibitors. Haemophilia. 2019 Jul;25(4):581-589. doi: 10.1111/hae.13717. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Inhibitor Eradication | The proportion eradicating anti-FVIII inhibitors | 48 weeks | |
Secondary | Number of Bleeding Events | The number of bleeding events: hematoma, joint, central nervous system, other bleeds. | 48 weeks | |
Secondary | FVIII Trough Level | The FVIII trough activity by chromogenic assay. | 48 weeks | |
Secondary | Human Leukocyte Antigen (HLA) Haplotype | The number of HLA haplotype variants. | 48 weeks | |
Secondary | FVIII Mutation | The number of FVIII mutation variants. | 48 weeks |
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