Study Results
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Basic Information
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ACTIVE_NOT_RECRUITING
PHASE2
51 participants
INTERVENTIONAL
2022-08-31
2026-12-31
Brief Summary
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Study of Emicizumab Prophylaxis in Participants With Hemophilia A With or Without Inhibitors Undergoing Minor Surgical Procedures
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NCT03315455
An Expanded Access Program of Emicizumab in Participants With Hemophilia A With Inhibitors
NCT03154437
Emicizumab in Acquired Hemophilia A
NCT04188639
A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants With Inhibitors
NCT02622321
Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Experimental-treatment
Treatment with emicizumab
emicizumab
This study design uses emicizumab as a prophylaxis treatment to prevent bleeding for all participants, bypassing agents (with the exception of aPCC) and treatment of concomitant diseases will be given as clinically indicated. All eligible subjects with AHA will receive the same study medication consisting of: two loading doses of the emicizumab on day 1 and 2 followed by once weekly subcutaneous emicizumab injections. Immunosuppressive therapy (IST) will be given concurrently as per investigator discretion.
Interventions
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emicizumab
This study design uses emicizumab as a prophylaxis treatment to prevent bleeding for all participants, bypassing agents (with the exception of aPCC) and treatment of concomitant diseases will be given as clinically indicated. All eligible subjects with AHA will receive the same study medication consisting of: two loading doses of the emicizumab on day 1 and 2 followed by once weekly subcutaneous emicizumab injections. Immunosuppressive therapy (IST) will be given concurrently as per investigator discretion.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Age ≥18 years at time of signing Informed Consent Form
* Ability to comply with the study protocol, in the investigator's judgment
* Diagnosis of AHA based on a reduced FVIII activity (\<50 %) and positive FVIII inhibitor (\>0.6 BU/ml) at screening (local laboratory)
* Current bleeding due to AHA at the time of screening
* Plan to be adherent to emicizumab prophylaxis during the study
* For women of childbearing potential who meet the following criteria:
* Refrain from heterosexual intercourse or use contraceptive methods that result in a failure rate of \<1% per year during the study period A woman with ≥ 12 continuous months of amenorrhea with no identified cause other than menopause and has not undergone surgical sterilization (removal of ovaries and/or uterus). use of combined oral or injected hormonal contraceptive, bilateral tubal ligation, male sterilization, hormone- releasing intrauterine devices, and copper intrauterine devices.
Exclusion Criteria
* Treatment with aPCC within the last 24 hours before first study treatment or planned treatment with aPCC during the course of the study
* Known positive lupus anticoagulant at the time of screening
* Severe uncontrolled infection at the time of screening
* Signs of active disseminated intravascular coagulation at the time of screening -
* Emicizumab ⎯ AHA Emi Version 1.0 20
* Current treatment for thromboembolic disease or signs of current thromboembolic disease at time of screening
* Patients who are at high risk for TMA (e.g., have a previous medical or family history of TMA), in the investigator's judgment
* Known severe congenital or acquired thrombophilia
* Life expectancy \<3 months at the time of screening
* Other conditions that substantially increase risk of bleeding or thrombosis by the discretion of the investigator
* Contraindications according to the Investigator's Brochure of emicizumab
* Current treatment with emicizumab at time of screening
* History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection by the discretion of the investigator
* Concurrent disease, treatment, or abnormality in clinical laboratory tests that could interfere with the conduct of the study, may pose additional risk, or would, in the opinion of the local investigator, preclude the patient's safe participation in and completion of the study
* Addiction or other diseases that preclude the patient from appropriately assessing the nature and scope as well as possible consequences of the clinical study by the discretion of the investigator
* Pregnant or breast-feeding women
* Would refuse treatment with blood or blood products, if necessary.
* Subject is in custody by order of an authority or a court of law
* Treatment with any of the following:
* An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration before Study Day 1
* A non-hemophilia-related investigational drug within the last 30 days or 5 half-lives- before Study Day 1, whichever is longer
* An investigational drug concurrently
* History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
18 Years
ALL
No
Sponsors
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Genentech, Inc.
INDUSTRY
University of Washington
OTHER
Responsible Party
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Rebecca Kruse-Jarres
Professor, School of Medicine: Hematology
Principal Investigators
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Rebecca Kruse-Jarres, MD, MPH
Role: PRINCIPAL_INVESTIGATOR
University of Washington
Locations
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UCSD Hemophilia and Thrombosis Treatment Center
San Diego, California, United States
Georgetown University
Washington D.C., District of Columbia, United States
Emory University
Atlanta, Georgia, United States
Bleeding and Clotting Disorders Institute
Peoria, Illinois, United States
Indiana Hemophilia and Thrombosis Center, Inc.
Indianapolis, Indiana, United States
Tulane University
New Orleans, Louisiana, United States
Mayo Clinic
Rochester, Minnesota, United States
Washington University
St Louis, Missouri, United States
University of North Carolina
Chapel Hill, North Carolina, United States
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, United States
Penn Blood Disorders Program, Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, United States
Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, United States
University of Vermont Medical Center
Burlington, Vermont, United States
UVA Comprehensive Cancer Center
Charlottesville, Virginia, United States
Washington Center for Bleeding Disorders
Seattle, Washington, United States
Versiti Inc.
Milwaukee, Wisconsin, United States
Countries
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References
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Shima M, Hanabusa H, Taki M, Matsushita T, Sato T, Fukutake K, Fukazawa N, Yoneyama K, Yoshida H, Nogami K. Factor VIII-Mimetic Function of Humanized Bispecific Antibody in Hemophilia A. N Engl J Med. 2016 May 26;374(21):2044-53. doi: 10.1056/NEJMoa1511769.
Devarajan P, Chen Z. Autoimmune effector memory T cells: the bad and the good. Immunol Res. 2013 Dec;57(1-3):12-22. doi: 10.1007/s12026-013-8448-1.
Mahlangu J. Emicizumab for the prevention of bleeds in hemophilia A. Expert Opin Biol Ther. 2019 Aug;19(8):753-761. doi: 10.1080/14712598.2019.1626370. Epub 2019 Jun 13.
Kessler CM, Knobl P. Acquired haemophilia: an overview for clinical practice. Eur J Haematol. 2015 Dec;95 Suppl 81:36-44. doi: 10.1111/ejh.12689.
Franchini M, Gandini G, Di Paolantonio T, Mariani G. Acquired hemophilia A: a concise review. Am J Hematol. 2005 Sep;80(1):55-63. doi: 10.1002/ajh.20390.
Kruse-Jarres R, Kempton CL, Baudo F, Collins PW, Knoebl P, Leissinger CA, Tiede A, Kessler CM. Acquired hemophilia A: Updated review of evidence and treatment guidance. Am J Hematol. 2017 Jul;92(7):695-705. doi: 10.1002/ajh.24777. Epub 2017 Jun 5.
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Tiede A, Klamroth R, Scharf RE, Trappe RU, Holstein K, Huth-Kuhne A, Gottstein S, Geisen U, Schenk J, Scholz U, Schilling K, Neumeister P, Miesbach W, Manner D, Greil R, von Auer C, Krause M, Leimkuhler K, Kalus U, Blumtritt JM, Werwitzke S, Budde E, Koch A, Knobl P. Prognostic factors for remission of and survival in acquired hemophilia A (AHA): results from the GTH-AH 01/2010 study. Blood. 2015 Feb 12;125(7):1091-7. doi: 10.1182/blood-2014-07-587089. Epub 2014 Dec 18.
Knoebl P, Thaler J, Jilma P, Quehenberger P, Gleixner K, Sperr WR. Emicizumab for the treatment of acquired hemophilia A. Blood. 2021 Jan 21;137(3):410-419. doi: 10.1182/blood.2020006315.
Al-Banaa K, Alhillan A, Hawa F, Mahmood R, Zaki A, El Abdallah M, Zimmerman J, Musa F. Emicizumab Use in Treatment of Acquired Hemophilia A: A Case Report. Am J Case Rep. 2019 Jul 18;20:1046-1048. doi: 10.12659/AJCR.916783.
Mohnle P, Pekrul I, Spannagl M, Sturm A, Singh D, Dechant C. Emicizumab in the Treatment of Acquired Haemophilia: A Case Report. Transfus Med Hemother. 2019 Apr;46(2):121-123. doi: 10.1159/000497287. Epub 2019 Mar 15.
Kruse-Jarres R, St-Louis J, Greist A, Shapiro A, Smith H, Chowdary P, Drebes A, Gomperts E, Bourgeois C, Mo M, Novack A, Farin H, Ewenstein B. Efficacy and safety of OBI-1, an antihaemophilic factor VIII (recombinant), porcine sequence, in subjects with acquired haemophilia A. Haemophilia. 2015 Mar;21(2):162-170. doi: 10.1111/hae.12627. Epub 2015 Jan 27.
Tiede A, Scharf RE, Dobbelstein C, Werwitzke S. Management of acquired haemophilia A. Hamostaseologie. 2015;35(4):311-8. doi: 10.5482/HAMO-14-11-0064. Epub 2015 Jan 7.
Langer AL, Etra A, Aledort L. Evaluating the safety of emicizumab in patients with hemophilia A. Expert Opin Drug Saf. 2018 Dec;17(12):1233-1237. doi: 10.1080/14740338.2019.1551356. Epub 2018 Nov 28.
Muto A, Yoshihashi K, Takeda M, Kitazawa T, Soeda T, Igawa T, Sampei Z, Kuramochi T, Sakamoto A, Haraya K, Adachi K, Kawabe Y, Nogami K, Shima M, Hattori K. Anti-factor IXa/X bispecific antibody ACE910 prevents joint bleeds in a long-term primate model of acquired hemophilia A. Blood. 2014 Nov 13;124(20):3165-71. doi: 10.1182/blood-2014-07-585737. Epub 2014 Oct 1.
Rodriguez-Merchan EC, Valentino LA. Emicizumab: Review of the literature and critical appraisal. Haemophilia. 2019 Jan;25(1):11-20. doi: 10.1111/hae.13641. Epub 2018 Nov 15.
Levy GG, Asikanius E, Kuebler P, Benchikh El Fegoun S, Esbjerg S, Seremetis S. Safety analysis of rFVIIa with emicizumab dosing in congenital hemophilia A with inhibitors: Experience from the HAVEN clinical program. J Thromb Haemost. 2019 Sep;17(9):1470-1477. doi: 10.1111/jth.14491. Epub 2019 Jun 17.
Pipe SW, Shima M, Lehle M, Shapiro A, Chebon S, Fukutake K, Key NS, Portron A, Schmitt C, Podolak-Dawidziak M, Selak Bienz N, Hermans C, Campinha-Bacote A, Kiialainen A, Peerlinck K, Levy GG, Jimenez-Yuste V. Efficacy, safety, and pharmacokinetics of emicizumab prophylaxis given every 4 weeks in people with haemophilia A (HAVEN 4): a multicentre, open-label, non-randomised phase 3 study. Lancet Haematol. 2019 Jun;6(6):e295-e305. doi: 10.1016/S2352-3026(19)30054-7. Epub 2019 Apr 16.
Takeyama M, Nogami K, Matsumoto T, Noguchi-Sasaki M, Kitazawa T, Shima M. An anti-factor IXa/factor X bispecific antibody, emicizumab, improves ex vivo coagulant potentials in plasma from patients with acquired hemophilia A. J Thromb Haemost. 2020 Apr;18(4):825-833. doi: 10.1111/jth.14746. Epub 2020 Feb 26.
Young G, Callaghan M, Dunn A, Kruse-Jarres R, Pipe S. Emicizumab for hemophilia A with factor VIII inhibitors. Expert Rev Hematol. 2018 Nov;11(11):835-846. doi: 10.1080/17474086.2018.1531701. Epub 2018 Oct 10.
Collins PW, Liesner R, Makris M, Talks K, Chowdary P, Chalmers E, Hall G, Riddell A, Percy CL, Hay CR, Hart DP. Treatment of bleeding episodes in haemophilia A complicated by a factor VIII inhibitor in patients receiving Emicizumab. Interim guidance from UKHCDO Inhibitor Working Party and Executive Committee. Haemophilia. 2018 May;24(3):344-347. doi: 10.1111/hae.13495.
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Holling TM, Schooten E, van Den Elsen PJ. Function and regulation of MHC class II molecules in T-lymphocytes: of mice and men. Hum Immunol. 2004 Apr;65(4):282-90. doi: 10.1016/j.humimm.2004.01.005.
Other Identifiers
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STUDY00013920
Identifier Type: -
Identifier Source: org_study_id
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