CLINICAL TRIALS PROFILE FOR BUSULFAN

505(b)(2) Clinical Trials for Busulfan

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Combination	NCT01643668 ↗	Busulfan/Clofarabine + Allogeneic Stem Cell Transplantation	Completed	Massachusetts General Hospital	Phase 2	2012-07-01	This research is a phase II clinical trial. Phase II clinical trials test the effectiveness of an investigational intervention to learn whether it works in treating a specific cancer. "Investigational" means that the study intervention is still being studied and that research doctors are trying to find out more about it. It also means that the FDA has not yet approved this study intervention for your type of cancer. All participants on this study are treated in an identical manner. The investigators are doing this study because there continues to be a significant risk of relapse of disease after reduced intensity transplantation. In studies which have compared transplants using high-doses of chemotherapy and/or radiation versus reduced intensity transplants, patients undergoing reduced intensity transplants appear to have higher rates of relapse, but lower rates of toxicity and complication. This study attempts to utilize clofarabine, a newer chemotherapy agent shown to be quite active in AML, ALL, and MDS, to increase the anti-tumor effects of the conditioning regimen without accumulating unacceptable toxicity. The reduced intensity allogeneic stem cell transplantation procedure involves giving you chemotherapy in relatively less intense doses to suppress your immune system. This is followed by an infusion of healthy blood stem cells from a matched related donor or a matched unrelated volunteer donor. It is hoped that these donor cells can eventually then attack any cancer cells which remain. In this research study, the investigators are looking to see how well this new combination of busulfan and clofarabine works in reduced intensity allogeneic stem cell transplantation. By "works" the investigators mean to analyze safety, ability of donor cells to engraft (take hold), as well as measures of complications including toxicity, infections, graft-vs-host disease (GVHD), and relapse.

All Clinical Trials for Busulfan

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00001561 ↗	Active Immunization of Sibling Bone Marrow Transplant Donors Against Purified Myeloma Protein of the Recipient Undergoing Allogeneic Bone Marrow Transplantation	Completed	National Cancer Institute (NCI)	Phase 3	1996-11-01	Both patients and marrow donors are treated on Regimen A; patients then proceed to Regimen B. The following acronyms are used: ABM Allogeneic Bone Marrow BU Busulfan, NSC-750 CF Leucovorin calcium, NSC-3590 CTX Cyclophosphamide, NSC-26271 G-CSF Granulocyte Colony-Stimulating Factor (source not specified) GM-CSF Granulocyte-Macrophage Colony-Stimulating Factor (Hoechst/Immunex), NSC-613795 GVHD Graft-vs.-Host Disease Mesna Mercaptoethane sulfonate, NSC-113891 MTX Methotrexate, NSC-740 PP Unconjugated Myeloma Immunoglobulin plasma paraprotein, NSC-684150 PP-KLH Myeloma immunoglobulin plasma paraprotein vaccine, NSC-678327, with keyhole limpet hemocyanin TBI Total-Body Irradiation TSPA Thiotepa, NSC-6396 Regimen A (Donor and Patient): Vaccine Therapy with Immunoadjuvant. PP-KLH (individual myeloma immunoglobulin plasma paraprotein vaccine prepared from recipient's plasma paraprotein and conjugated with KLH); and PP; with GM-CSF. Regimen B (Patient): Myeloablative Radiotherapy and 2-Drug Combination Chemotherapy or 2-Drug Combination Myeloablative Chemotherapy followed by Hematopoietic Rescue with Growth Factor Support and GVHD Prophylaxis followed by Vaccine Therapy with Immunoadjuvant. TBI; and CTX/TSPA; or BU/CTX; followed by ABM; with G-CSF; and CYSP; MTX/CF; followed by PP-KLH; with GM-CSF.
NCT00002502 ↗	Combination Chemotherapy and Bone Marrow Transplantation in Treating Patients With Leukemia or Myelodysplastic Syndrome	Completed	National Cancer Institute (NCI)	Phase 2	1992-07-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with bone marrow transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy consisting of busulfan and cyclophosphamide followed by bone marrow transplantation in treating patients who have acute or chronic leukemia or myelodysplastic syndrome.
NCT00002502 ↗	Combination Chemotherapy and Bone Marrow Transplantation in Treating Patients With Leukemia or Myelodysplastic Syndrome	Completed	Memorial Sloan Kettering Cancer Center	Phase 2	1992-07-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with bone marrow transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy consisting of busulfan and cyclophosphamide followed by bone marrow transplantation in treating patients who have acute or chronic leukemia or myelodysplastic syndrome.
NCT00002547 ↗	Chemotherapy and Bone Marrow Transplantation in Treating Patients Acute Myeloid With Leukemia or Myelodysplastic Syndrome	Completed	National Cancer Institute (NCI)	Phase 2	1987-08-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with bone marrow transplantation may allow the doctor to give higher doses of chemotherapy and kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of bone marrow transplantation following combination chemotherapy in treating patients with acute myeloid leukemia or myelodysplastic syndrome .
NCT00002547 ↗	Chemotherapy and Bone Marrow Transplantation in Treating Patients Acute Myeloid With Leukemia or Myelodysplastic Syndrome	Completed	Barbara Ann Karmanos Cancer Institute	Phase 2	1987-08-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with bone marrow transplantation may allow the doctor to give higher doses of chemotherapy and kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of bone marrow transplantation following combination chemotherapy in treating patients with acute myeloid leukemia or myelodysplastic syndrome .

Clinical Trial Conditions for Busulfan

Condition Name

Condition Name for Busulfan
Intervention	Trials
Leukemia	142
Lymphoma	87
Myelodysplastic Syndromes	83
Myelodysplastic Syndrome	55

Condition MeSH

Condition MeSH for Busulfan
Intervention	Trials
Leukemia	264
Myelodysplastic Syndromes	174
Preleukemia	166
Leukemia, Myeloid	148

Clinical Trial Locations for Busulfan

Trials by Country

Trials by Country for Busulfan
Location	Trials
Canada	80
China	64
France	43
Italy	42
Germany	33

Trials by US State

Trials by US State for Busulfan
Location	Trials
Texas	89
New York	79
California	65
Maryland	63
Washington	55

Clinical Trial Progress for Busulfan

Clinical Trial Phase

Clinical Trial Phase for Busulfan
Clinical Trial Phase	Trials
Phase 4	8
Phase 3	66
Phase 2/Phase 3	27

Clinical Trial Status

Clinical Trial Status for Busulfan
Clinical Trial Phase	Trials
Completed	252
Recruiting	116
Unknown status	67

Clinical Trial Sponsors for Busulfan

Sponsor Name

Sponsor Name for Busulfan
Sponsor	Trials
National Cancer Institute (NCI)	164
M.D. Anderson Cancer Center	54
Fred Hutchinson Cancer Research Center	35

Sponsor Type

Sponsor Type for Busulfan
Sponsor	Trials
Other	813
NIH	201
Industry	91

Busulfan: Clinical Trials, Market Analysis, and Projections

Introduction to Busulfan

Busulfan is a chemotherapy agent that plays a critical role in the treatment of various hematological disorders, particularly in preparing patients for bone marrow transplants. It is widely used in the conditioning regimens for hematopoietic stem cell transplantation (HSCT) and in the treatment of chronic myeloid leukemia (CML) and other leukemias.

Clinical Trials Update

Current Studies and Objectives

One notable clinical trial involving busulfan is the study on the use of BIVV003 for autologous hematopoietic stem cell transplantation in patients with severe sickle cell disease. This Phase 1/2 study, sponsored by Sangamo Therapeutics, aims to evaluate the safety, tolerability, and efficacy of this treatment approach. The trial involves approximately eight adults and includes the use of busulfan as part of the conditioning regimen[1].

Patient Eligibility and Study Design

Participants in this study must be between 18 and 40 years old, have a confirmed diagnosis of sickle cell disease (HbSS or HbSβ0 genotype), and be clinically stable to undergo stem cell mobilization and myeloablative HSCT. They must also meet specific physiological function criteria, including acceptable cardiac, pulmonary, renal, and hepatic functions. The study excludes patients who have previously received an autologous or allogeneic HSCT, solid organ transplantation, or gene therapy, among other criteria[1].

Long-Term Follow-Up

Enrolled subjects will participate in a separate long-term follow-up study to monitor the safety and efficacy of BIVV003 treatment for up to 15 years post-transplant. This long-term monitoring is crucial for understanding the sustained effects and potential long-term side effects of the treatment[1].

Market Analysis

Market Size and Growth

The busulfan market is expected to grow steadily over the next few years, driven by several key factors. The increasing incidence of leukemia, growing adoption of busulfan in bone marrow and stem cell transplants, and escalating investments in research and development are primary drivers of this growth. The global busulfan market is forecasted to expand significantly by 2030, with market size growth rates varying by region and application[2][5].

Application Areas

Busulfan is primarily used in chemotherapy protocols, particularly for treating chronic myeloid leukemia and other hematological disorders. Its application in HSCT conditioning regimens is a major contributor to its market demand. The drug is also used off-label in various other medical conditions, further broadening its market scope[2].

End Users

The primary end users of busulfan include hospitals, oncology clinics, and research and academic institutions. These healthcare institutions are key consumers due to the critical role busulfan plays in stem cell transplantation and cancer treatment[2].

Market Challenges and Opportunities

Despite the promising growth trajectory, the busulfan market faces several challenges. These include stringent regulatory requirements, high treatment costs, and potential severe side effects such as cytopenia and organ toxicity. However, opportunities lie in emerging markets with improving healthcare infrastructure and the potential for innovative formulations with better safety profiles. Strategic partnerships between pharmaceutical companies and research institutions are also crucial for staying at the forefront of developments and enhancing the therapeutic landscape of busulfan applications[2][5].

Market Projections

Global Forecast

The global busulfan market is projected to grow significantly from 2025 to 2030. This growth is anticipated across various regions, including North America, Europe, Asia, and the Middle East, Africa, and Latin America. The market size is expected to increase due to the rising demand for busulfan in cancer treatment and the expanding use in stem cell transplants[5].

Regional Growth

The market growth rate varies by region, with Asia and North America expected to be key drivers. The increasing incidence of leukemia and other hematological disorders in these regions, coupled with advancements in healthcare infrastructure, will contribute to the market expansion[5].

Competitive Landscape

The busulfan market is competitive, with several key players including Accord Healthcare Ltd., Amneal Pharmaceuticals, Inc., Aurobindo Pharma Ltd., Bristol-Myers Squibb Company, and others. These companies are investing in research and development to improve busulfan formulations and expand their market share. Strategic partnerships and innovations in drug delivery systems are also critical for maintaining a competitive edge[2][5].

Pharmacokinetics and Metabolism

Population Pharmacokinetics

Recent studies have focused on the population pharmacokinetics of busulfan and its metabolite sulfolane. A study involving patients with myelofibrosis showed that total body weight and a single-nucleotide polymorphism of glutathione-S-transferase A1 (GSTA1 SNP) significantly impact the volume of distribution and metabolite clearance, respectively. This understanding is crucial for optimizing busulfan dosing and minimizing side effects[3].

Key Takeaways

• Clinical Trials: Busulfan is being evaluated in clinical trials for its use in conditioning regimens for HSCT, particularly in patients with severe sickle cell disease.
• Market Growth: The busulfan market is expected to grow driven by increasing leukemia incidence, adoption in stem cell transplants, and R&D investments.
• Application Areas: Busulfan is primarily used in chemotherapy protocols and HSCT conditioning regimens.
• Challenges: The market faces challenges such as regulatory hurdles, high treatment costs, and potential side effects.
• Opportunities: Emerging markets and innovative formulations offer growth opportunities.
• Pharmacokinetics: Understanding the pharmacokinetics of busulfan is crucial for optimizing dosing and minimizing side effects.

FAQs

What is the primary use of busulfan in medical treatment?

Busulfan is primarily used as a chemotherapy agent to prepare patients for bone marrow transplants and in the treatment of chronic myeloid leukemia and other hematological disorders.

Which regions are expected to drive the growth of the busulfan market?

The busulfan market is expected to grow significantly in regions such as North America, Europe, and Asia, driven by the increasing incidence of leukemia and advancements in healthcare infrastructure.

What are the key challenges facing the busulfan market?

The busulfan market faces challenges including stringent regulatory requirements, high treatment costs, and potential severe side effects such as cytopenia and organ toxicity.

How is busulfan used in the context of hematopoietic stem cell transplantation?

Busulfan is used as part of the conditioning regimen in HSCT to suppress bone marrow function before the introduction of new stem cells.

What are the potential opportunities for growth in the busulfan market?

Opportunities for growth include emerging markets with improving healthcare infrastructure, innovative formulations with better safety profiles, and strategic partnerships between pharmaceutical companies and research institutions.

Sources

1. ClinicalTrials.gov: A Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease.
2. 360iResearch: Busulfan Market Size & Share 2025-2030.
3. MDPI: Population Pharmacokinetics of Busulfan and Its Metabolite Sulfolane in Patients with Myelofibrosis.
4. National Cancer Institute: Clinical Trials Using Busulfan.
5. GII Research: Global Busulfan Market Insights, Forecast to 2030.