Clinical trials: Busulfan, completed
Beta-Thalassemia Major, Sickle Cell Disease
A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease
This is a Phase 1/2, open label, safety, and efficacy study of the administration of LentiGlobin BB305 Drug Product to participants with either transfusion dependent beta-thalassemia (TDT) or sickle cell disease (SCD).
- Trial name
- NCT02151526
- Testing
- LentiGlobin BB305 Drug Product
- Where
- France
- Volunteers
- 7 people
- Sponsor
- Genetix Biotherapeutics Inc.
Infant Nutrition
A New Adaptive Feeding Plan for Newborns
The purpose of this clinical trial is to identify the infant formula(s) for which the investigators get the closest gut maturation index compared to the one they get with breastfed babies.
- Trial name
- NCT00984230
- Testing
- BSF, BSF + Lactoferrin + Probiotics, BSF + Lactoferrin + Probiotics + OS
- Where
- 6 sites in Austria, France and 1 more
- Volunteers
- 186 people, healthy volunteers welcome
- Sponsor
- Société des Produits Nestlé (SPN)
Beta-Thalassemia, Thalassemia
A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participans with transfusion-dependent β-thalassemia (TDT).
- Conditions
- Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases
- Trial name
- NCT03655678
- Testing
- CTX001
- Where
- 14 sites in United States, Canada and 3 more
- Volunteers
- 59 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Hematologic Diseases
A Study Evaluating the Safety and Efficacy of the BD211 Drug Product in β-Thalassemia Major Participants
This is a Phase 1,open label,safety,and efficacy study in subjects with non-β0/β0 TDT β-thalassemia Major by transplanting BD211 drug product which is for autologous use only,via a single IV administration.
- Trial name
- NCT05015920
- Testing
- BD211 Drug Product
- Where
- China
- Volunteers
- 2 people
- Sponsor
- Shanghai BDgene Co., Ltd.
Transfusion Dependent Beta-thalassemia
A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)
This is a single-arm, multi-site, single-dose, Phase 1/2 study to assess ST-400 in 6 subjects with transfusion-dependent β-thalassemia (TDT) who are ≥18 and ≤40 years of age.
- Trial name
- NCT03432364
- Testing
- ST-400 Investigational product
- Where
- 6 sites in United States
- Volunteers
- 5 people
- Sponsor
- Sangamo Therapeutics
Sickle Cell Disease, Hematological Diseases
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participants with severe sickle cell disease (SCD).
- Conditions
- Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies
- Trial name
- NCT03745287
- Testing
- Exa-cel
- Where
- 17 sites in United States, Belgium and 5 more
- Volunteers
- 63 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Primary Immunodeficiencies, Inborn Errors of Metabolism
Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases
The aim of the trial is to describe the safety and efficacy of intravenous (i.v.) Treosulfan compared to the conventional (myeloablative) dose of i.v.
- Conditions
- Primary Immunodeficiencies, Inborn Errors of Metabolism, Haemoglobinopathies, Bone Marrow Failure Syndromes
- Trial name
- NCT02349906
- Testing
- Treosulfan, Busilvex
- Where
- 21 sites in Czechia, Germany and 2 more
- Volunteers
- 106 people
- Sponsor
- medac GmbH
Graft Versus Host Disease, Hematologic Malignancies
Trial to Evaluate Palifermin in the Reduction of Acute Graft Versus Host Disease in Patients With Hematologic Malignancies Undergoing Allogeneic Marrow/Peripheral Blood Progenitor Cell (PBPC) Transplantation
The main purpose of this study is to evaluate the effect of palifermin versus placebo in the reduction of severe acute graft versus host disease (GVHD) and severe oral mucositis.
- Trial name
- NCT00189488
- Testing
- Palifermin, Conditioning Regimen, Allogeneic stem cell transplant
- Volunteers
- 155 people
- Sponsor
- Swedish Orphan Biovitrum
Cerebral Adrenoleukodystrophy (CALD)
A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
The purpose of this study is to evaluate the efficacy and safety of Lenti-D Drug Product (also known as elivaldogene autotemcel or Skysona, hereafter referred to as eli-cel) after myeloablative conditioning with busulfan and…
- Trial name
- NCT03852498
- Testing
- Lenti-D
- Where
- 8 sites in United States, France and 4 more
- Volunteers
- 35 people
- Sponsor
- Genetix Biotherapeutics Inc.
MPN (Myeloproliferative Neoplasms)
Prospective Observational Study Of Patients With Polycythemia Vera In US Clinical Practices (REVEAL)
This is a Phase IV, multicenter, non-interventional, non-randomized, prospective, observational study in an adult population (patients >18 years old) of men and women who have been diagnosed with clinically overt PV and are being…
- Trial name
- NCT02252159
- Where
- 266 sites in United States, Puerto Rico
- Volunteers
- 2,544 people
- Sponsor
- Incyte Corporation
Human Immunodeficiency Virus
Safety Study of a Dual Anti-HIV Gene Transfer Construct to Treat HIV-1 Infection
This is an early phase research study looking at whether an experimental gene transfer, LVsh5/C46 (also known as Cal-1), is safe and if it can protect the immune system from the effects of HIV without the use of antiretroviral…
- Trial name
- NCT01734850
- Testing
- Busulfan, Cal-1 modified HSPC, Cal-1 modified CD4+ T lymphocytes
- Where
- 2 sites in United States
- Volunteers
- 13 people
- Sponsor
- Calimmune, Inc.
Beta-Thalassemia
A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia
This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 18 participants less than or equal to (<=) 50 years of age with transfusion-dependent β-thalassemia (TDT), who have a β0/β0, β0/IVS-I-110, or…
- Trial name
- NCT03207009
- Testing
- LentiGlobin BB305 Drug Product
- Where
- 9 sites in United States, France and 4 more
- Volunteers
- 19 people
- Sponsor
- Genetix Biotherapeutics Inc.
Multiple Myeloma
IV Busulfan Plus Bortezomib Conditioning Regimen for Second Autologous Stem Cell Transplant in Multiple Myeloma Patients
Study for the outcome and safety of individualized busulfan dosing with bortezomib for patients preparing for a second stem cell transplant to treat multiple myeloma.
- Trial name
- NCT01009840
- Testing
- IV busulfan, bortezomib, Autologous Hematopoietic Stem Cell Transplant (HSCT)
- Where
- 13 sites in United States, Canada
- Volunteers
- 30 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Lysosomal Storage Disease, Metachromatic Leukodystrophy
A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD)
OTL-200 is autologous CD34+ cells transduced with lentiviral vector containing human arylsulfatase A (ARSA) complementary deoxyribonucleic acid (cDNA) used for the treatment of MLD.
- Trial name
- NCT03392987
- Testing
- OTL-200
- Where
- Italy
- Volunteers
- 10 people
- Sponsor
- Orchard Therapeutics
High Risk Myeloid Malignancies, Cord Blood Transplant
US Study of ECT-001-CB in Pediatric and Young Adult Patients With High-Risk Myeloid Malignancies
Cord blood (CB) transplants are an option for patients lacking an HLA identical donor but are hampered by low cell dose, prolonged aplasia and high transplant related mortality.
- Trial name
- NCT04990323
- Testing
- ECT-001-CB (UM171-Expanded Cord Blood Transplant)
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- ExCellThera inc.
Acute Myeloid Leukemia, Myelodysplastic Syndrome
Clinical Phase III Trial Treosulfan-based Conditioning Versus Reduced-intensity Conditioning (RIC)
This randomized allogeneic transplantation protocol compares i.v. Treosulfan-based conditioning therapy with reduced intensity i.v.
- Trial name
- NCT00822393
- Testing
- Busulfan, Treosulfan
- Where
- 33 sites in Finland, France and 4 more
- Volunteers
- 570 people
- Sponsor
- medac GmbH
Sickle Cell Disease
A Study Evaluating the Safety and Efficacy of Lovo-cel in Severe Sickle Cell Disease
This is a non-randomized, open label, multi-site, single dose, Phase 1/2 study in approximately 50 adults and adolescents with severe SCD.
- Trial name
- NCT02140554
- Testing
- lovo-cel
- Where
- 11 sites in United States
- Volunteers
- 54 people
- Sponsor
- Genetix Biotherapeutics Inc.
Sickle Cell Disease
A Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease
This is an open label, multicenter, Phase 1/2 study in approximately eight adults with severe Sickle Cell Disease (SCD). The study will evaluate the safety, tolerability, and efficacy of autologous hematopoietic stem cell…
- Trial name
- NCT03653247
- Testing
- Plerixafor, Busulfan, BIVV003
- Where
- 5 sites in United States
- Volunteers
- 7 people
- Sponsor
- Sangamo Therapeutics
Lymphoma
PK-directed Dose Adjustment of IV Busulfan Conditioning Regimen for Autologous Stem Cell Transplant in Lymphoma Patients
This is a study for the outcome and safety of individualized busulfan dosing with cyclophosphamide and etoposide for patients preparing for a stem cell transplant to treat Non-Hodgkin or Hodgkin's Lymphoma.
- Trial name
- NCT00948090
- Testing
- IV Busulfan, Cyclophosphamide and Etoposide (BuCyE Regimen)
- Where
- 42 sites in United States, Canada
- Volunteers
- 207 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Hematologic Malignancies, Acute Myeloid Leukemia
Study of ProTmune for Allogeneic HCT in Adult Patients With Hematologic Malignancies
This study is a Phase 1, non-randomized, open-label/Phase 2 randomized, blinded study of ProTmune (ex vivo programmed mobilized peripheral blood cells) versus non-programmed mobilized peripheral blood cells for allogeneic…
- Conditions
- Hematologic Malignancies, Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndromes
- Trial name
- NCT02743351
- Testing
- ProTmune, Control Arm
- Where
- 15 sites in United States
- Volunteers
- 96 people
- Sponsor
- Fate Therapeutics