Hemophilia clinical trials at University of California Health
20 in progress, 7 open to eligible people
Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
open to eligible males ages 2-59
WP44714 is a Phase I/II, open-label, non-randomized, global, multicenter trial consisting of two parts: - Part 1 is a multiple-ascending dose (MAD) study in adult and adolescent male participants with severe or moderate hemophilia A with or without…
at UC Davis
Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B
open to eligible males ages 18 years and up
Participants in this study have a genetic mutation, specifically in the coagulation (blood clotting) Factor 9 gene that causes severe or moderately severe hemophilia B. This study is researching an experimental gene insertion therapy (the adding of…
at UCSF
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).
at UCSD
SEVENFACT® for Bleeding Events in Hemophilia With Inhibitors
open to eligible people ages 12-100
Phase IV multi-center, US-centric, open-label, safety study enrolling participants with Hemophilia A or B with inhibitors, 12 years of age and older, who are either on long term prophylactic treatment (e.g., emicizumab) at risk of experiencing a…
at UC Davis UCLA
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…
at UC Davis UCLA UCSD UCSF
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…
at UCSD
Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children Per Standard of Care (POPS)
open to eligible people ages 0-20
The study investigators are interested in learning more about how drugs, that are given to children by their health care provider, act in the bodies of children and young adults in hopes to find the most safe and effective dose for children. The…
at UCLA
Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Sorry, in progress, not accepting new patients
This study will evaluate the efficacy and safety of PF-06838435 (a gene therapy drug) in adult male participants with moderately severe to severe hemophilia B (participants that have a Factor IX circulating activity of 2% or less). The gene therapy…
at UCSF
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…
at UCSD
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…
at UCSD
Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B
Sorry, in progress, not accepting new patients
Long-term safety and efficacy follow-up for participants with Hemophilia B who were previously treated in the C0371005 (formerly SPK-9001-101) study, and a dose-escalation sub-study evaluating safety, tolerability, and kinetics of a higher dose with …
at UC Davis
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.
at UC Davis UCSD
Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively
Sorry, not currently recruiting here
A study to learn about the long-term safety and efficacy of giroctocogene fitelparvovec or fidanacogene elaparvovec in patients with hemophilia A or hemophilia B respectively, who have received treatment through prior participation in a…
at UC Davis UCSF
PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults
Sorry, in progress, not accepting new patients
C3731003 is a pivotal Phase 3 study to evaluate the clinical efficacy and safety of a single IV infusion of PF-07055480 / giroctocogene fitelparvovec (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in adult male participants with moderately…
at UCSF
Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A
Sorry, in progress, not accepting new patients
This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in combination with prophylactic corticosteroids in patients with severe hemophilia A.
at UC Davis
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…
at UC Davis
Compassionate Use of Concizumab if You Have Haemophilia
Sorry, not accepting new patients
The compassionate use programme will give participants concizumab for free, even though it is not yet approved by health authorities. This is because participants need this medicine to treat their haemophilia properly. The programme will check that…
at UCSF
Evaluating Effectiveness and Long Term Safety of Damoctocog Alfa Pegol in Patients, Who Have Been Diagnosed With Hemophilia A
Sorry, in progress, not accepting new patients
The aim of the HEM-POWR study is to understand better how Damoctocog alfa pegol (Jivi) is used to treat people with Hemophilia A in day-to-day life, how well the treatment is tolerated and how satisfied patients and physicians are with the…
at UC Davis
(LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX
Sorry, in progress, not accepting new patients
Long-term follow-up of subjects who received SB-318, SB-913, or SB-FIX in a previous trial and completed at least 52 weeks post-infusion follow-up in their primary protocol. Enrolled subjects will be followed for a total of up to 10 years following…
at UCSF
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).
at UC Davis UCSD
Our lead scientists for Hemophilia research studies include Annette Von Drygalski, MD Jaime Deville Alison Matsunaga, MD.
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