Clinical trials: Busulfan, open to volunteers
- Drug or intervention Busulfan
- Status Open to volunteers
- Funder type Industry (pharma, biotech, devices)
Subjects Treated With Busulfan
Busulfan Sample Collection Study
Busulfan (Busulfex®) is an alkylating drug used as a conditioning regimen prior to allogeneic hematopoietic stem cell transplantation.
- Trial name
- NCT05711732
- Testing
- MyCare Oncology Busulfan Assay Kit
- Where
- 3 sites in United States
- Volunteers
- 150 people
- Sponsor
- Saladax Biomedical, Inc.
Hemoglobinopathy, Sickle Cell Disease
A Long-term Follow-up Study in Patients Who Received BEAM-101
This is a Long-term Follow-up (LTFU) study in patients who received BEAM-101 in the parent study (Study BTX-AUT-001). Eligible patients who received BEAM-101 will be asked to participate in this LTFU study prior to completing…
- Trial name
- NCT07373639
- Testing
- Long-Term Follow-up Study of patients who received BEAM-101
- Where
- 17 sites in United States
- Volunteers
- 50 people
- Sponsor
- Beam Therapeutics Inc.
Beta-Thalassemia, Thalassemia
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD).
- Conditions
- Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn
- Trial name
- NCT05477563
- Testing
- CTX001
- Where
- 6 sites in United States, Germany and 2 more
- Volunteers
- 26 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Hemophilia A
Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A
This is a first-in-human, non-randomized, open label, single treatment, Phase 1 study in approximately 7 patients with severe hemophilia A.
- Trial name
- NCT04418414
- Testing
- Gene therapy, Biological
- Volunteers
- 7 people
- Sponsor
- Expression Therapeutics, LLC
Chronic Granulomatous Disease, Granulomatous Disease, Chronic
A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )
This is an open-label, single-arm, multicenter Phase 1/2 study evaluating the safety and efficacy of gene therapy by transplantation of Prime Edited autologous CD34+ stem cells modified ex vivo (PM359) in participants with…
- Trial name
- NCT06559176
- Testing
- PM359
- Where
- 5 sites in United States, Canada and 1 more
- Volunteers
- 12 people
- Sponsor
- Prime Medicine, Inc.
Transfusion-dependent Beta-Thalassemia
β-Thalassemia Treatment With KL003 Cell Injection
This is a non-randomized, open label, single-dose study in up to 41 participants with β-thalassemia major. The goal of this clinical trial is to evaluate the safety and efficacy of KL003 cell injection in subjects with…
- Trial name
- NCT06280378
- Testing
- KL003 Cell Injection Drug Product
- Where
- 2 sites in China
- Volunteers
- 41 people
- Sponsor
- Kanglin Biotechnology (Hangzhou) Co., Ltd.