Hemophilia clinical trials at UCSD
8 in progress, 3 open to eligible people
Emicizumab in Patients With Acquired Hemophilia A
open to eligible people ages 18 years and up
This is a phase II multicenter open-label, single-arm prospective study to evaluate the efficacy of prophylactic emicizumab administered on a scheduled basis to prevent bleeds in patients with acquired hemophilia A (AHA).
San Diego, California and other locations
ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
open to all eligible people
In parallel with the growth of American Thrombosis and Hemostasis Network's (ATHN) clinical studies, the number of new therapies for all congenital and acquired hematologic conditions, not just those for bleeding and clotting disorders, is…
San Diego, California and other locations
Emicizumab vs. Factor VIII Prophylaxis on Joint and Bone Health in Severe Hemophilia A
open to eligible males ages 16 years and up
The investigators propose to study longitudinal joint and bone density changes in patients with severe Hemophilia A. Per current standard of care, most patients are on prophylactic FVIII replacement therapy intravenously several times weekly with a…
San Diego, California and other locations
Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia
Sorry, in progress, not accepting new patients
This is a multicenter, multinational, open-label, one-way cross-over, Phase 3, single-arm study for treatment of hemophilia. The purpose of this study is to measure the frequency of treated bleeding episodes with fitusiran in male adult and…
San Diego, California and other locations
HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients
Sorry, in progress, not accepting new patients
This is an open-label, single-dose, multi-center, multinational trial to demonstrate the efficacy of AMT-061 and to further describe its safety profile. The study drug is identified as AAV5-hFIXco-Padua (AMT- 061). AMT-061 is a recombinant…
San Diego, California and other locations
Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A
Sorry, in progress, not accepting new patients
Primary Objective: - To evaluate the long-term safety of BIVV001 in previously treated subjects with hemophilia A Secondary Objectives: - To evaluate the efficacy of BIVV001 as a prophylaxis treatment. - To evaluate the efficacy of BIVV001 in the…
San Diego, California and other locations
Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B
Sorry, in progress, not accepting new patients
The purpose of this study is to evaluate the safety of single ascending IV doses of a Factor IX (FIX) Gene Therapy in up to 16 Adults with Hemophilia B.
San Diego, California and other locations
Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)
Sorry, accepting new patients by invitation only
The primary purpose of this study is to assess the long-term safety and efficacy in male adults with hemophilia B who were treated with CSL222 (CSL222) in parent studies CSL222_2001 (NCT03489291) or CSL222_3001 (NCT03569891).
San Diego, California and other locations
Our lead scientists for Hemophilia research studies include Annette Von Drygalski, MD.
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