Hemophilia clinical trials at UCSD
8 in progress, 2 open to eligible people
CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)
open to eligible people ages 18 years and up
The purpose of this study is to assess the risk of bleeding due to failure of expected pharmacological action of CSL222 in adults with severe or moderately severe hemophilia B with detectable pretreatment AAV5 Nabs.
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 ATHN's clinical studies, the number of new therapies for all blood disorders is increasing significantly. Some of the recently FDA-approved therapies for congenital and acquired hematologic conditions have not yet demonstrated long-term safety and effectiveness beyond the pivotal trials that led to their approval. In addition, results from well controlled, pivotal studies often cannot be replicated once a therapy has been approved for general use.2,3,4,5 In 2019 alone, the FDA has issued approvals for 24 new therapies for congenital and acquired hematologic conditions.6 In addition, almost 10,000 new studies for hematologic diseases are currently registered on www.clinicaltrials.gov.7 With this increase in potential new therapies possible, it is imperative that clinicians and clinical researchers in the field of non-neoplastic hematology have a uniform, secure, unbiased, and enduring method to collect long-term safety and efficacy data. As emphasized in a recently published review, accurate, uniform and quality national data collection is critical in clinical research, particularly for longitudinal cohort studies covering a lifetime of biologic risk.8
San Diego, California and other locations
Emicizumab in Patients With Acquired Hemophilia A
Sorry, in progress, not accepting new patients
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
Learn About How Changing Therapy From Emicizumab to Marstacimab Affects People With the Severe Hemophilia A.
Sorry, not currently recruiting here
The purpose of the study is to learn about safety, how the body processes marstacimab and how it works in patients with severe hemophilia A without inhibitors. Hemophilia A is rare bleeding disorder where the blood doesn't clot normally. This causes a person to bleed a lot, even from a small cut. These patients who are on emicizumab medicine for routine prophylaxis for at least 6 months, and desire to switch to marstacimab medicine. Inhibitors are antibodies that the immune system develops because it sees the infused clotting factor as a foreign substance that needs to be destroyed. Antibodies are proteins that eat up the activated factor before it has time to stop the bleeding. Prophylaxis are preventive medicines. This study is seeking for participants: - with severe Hemophilia A withouth inhibitors who are on emicizumab treatment for at least 6 months. - must be 12 to less than 75 years old - must have a body weight of at least 35 kilograms. The results from this study will serve as a guide to doctors and their hemophilia A patients who will change their medicines in the real-world clinical setting. Patients who can take part in the study will receive marstacimab medicine as weekly injections under the skin of 150 milligrams for 4 months. Study treatment with marstacimab will be initiated no earlier than 14 days after last dose of emicizumab. The study can last up to 6 months. The sponsor will provide marstacimab. Patients will continue their usual treatment with the infused clotting factor for their bleeds when taking part in the study. Roll-over into an optional study treatment extension period will be available to participants who wish to continue prophylaxis with marstacimab in countries where it is not commercially available.
La Jolla, 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 treatment of bleeding episodes. - To evaluate BIVV001 consumption for prevention and treatment of bleeding episodes. - To evaluate the effect of BIVV001 prophylaxis on joint health outcomes. - To evaluate the effect of BIVV001 prophylaxis on Quality of Life (QoL) outcomes. - To evaluate the safety and tolerability of BIVV001 treatment. - To assess the PK of BIVV001 based on the one stage activated partial thromboplastin time (aPTT) and two-stage chromogenic FVIII activity assays (only applicable to Arm B). - To evaluate the efficacy of BIVV001 for perioperative management
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
Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)
Sorry, accepting new patients by invitation only
The BMN 270 clinical development program consists of multiple interventional studies designed to assess the safety and efficacy of a single infusion of BMN 270 for at least 5 years post-infusion. This long-term follow-up study is needed to help further understand the long-term safety of BMN 270 beyond 5 years and to assess the durability of efficacy.
San Diego, California and other locations
Our lead scientists for Hemophilia research studies include Annette Von Drygalski, MD.
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