CLINICAL TRIALS PROFILE FOR BUSULFEX

All Clinical Trials for Busulfex

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00002831 ↗	Chemotherapy Plus Peripheral Stem Cell Transplantation in Treating Patients With Chronic Myelogenous or Acute Leukemia	Completed	National Cancer Institute (NCI)	Phase 1/Phase 2	1995-08-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells. PURPOSE: Phase I/II trial to study the effectiveness of high-dose chemotherapy plus peripheral stem cell transplantation in treating patients with chronic myelogenous or acute leukemia.
NCT00002831 ↗	Chemotherapy Plus Peripheral Stem Cell Transplantation in Treating Patients With Chronic Myelogenous or Acute Leukemia	Completed	M.D. Anderson Cancer Center	Phase 1/Phase 2	1995-08-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells. PURPOSE: Phase I/II trial to study the effectiveness of high-dose chemotherapy plus peripheral stem cell transplantation in treating patients with chronic myelogenous or acute leukemia.
NCT00003926 ↗	Amifostine to Protect From Side Effects of PSCT in Treating Patients With Solid Tumors	Terminated	Masonic Cancer Center, University of Minnesota	Phase 1	1998-11-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining chemotherapy with peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more tumor cells. Chemoprotective drugs such as amifostine may protect normal cells from the side effects of high-dose chemotherapy. PURPOSE: Phase I trial to study the effectiveness of amifostine in protecting from the side effects of peripheral stem cell transplantation in treating patients who have high-risk or relapsed solid tumors.
NCT00004088 ↗	Combination Chemo, Peripheral Stem Cell Transplant, Biological Therapy, Pamidronate and Thalidomide for Multiple Myeloma	Completed	National Cancer Institute (NCI)	Phase 2	1999-04-13	RATIONALE: Drugs used in chemotherapy work in different ways to stop cancer cells from dividing so they stop growing or die. Peripheral stem cell transplantation may allow doctors to give higher doses of chemotherapy drugs and kill more cancer cells. Biological therapies, such as interferon alfa, use different ways to stimulate the immune system and stop cancer cells from growing. Thalidomide may stop the growth of cancer cells by stopping blood flow to the tumor. Pamidronate may help to reduce the side effects of treatment for multiple myeloma. PURPOSE: This phase II trial is studying combination chemotherapy, peripheral stem cell transplantation, biological therapy, pamidronate, and thalidomide to see how well they work in treating patients with stage I, stage II, or stage III multiple myeloma.
NCT00004088 ↗	Combination Chemo, Peripheral Stem Cell Transplant, Biological Therapy, Pamidronate and Thalidomide for Multiple Myeloma	Completed	City of Hope Medical Center	Phase 2	1999-04-13	RATIONALE: Drugs used in chemotherapy work in different ways to stop cancer cells from dividing so they stop growing or die. Peripheral stem cell transplantation may allow doctors to give higher doses of chemotherapy drugs and kill more cancer cells. Biological therapies, such as interferon alfa, use different ways to stimulate the immune system and stop cancer cells from growing. Thalidomide may stop the growth of cancer cells by stopping blood flow to the tumor. Pamidronate may help to reduce the side effects of treatment for multiple myeloma. PURPOSE: This phase II trial is studying combination chemotherapy, peripheral stem cell transplantation, biological therapy, pamidronate, and thalidomide to see how well they work in treating patients with stage I, stage II, or stage III multiple myeloma.
NCT00014469 ↗	Combination Chemotherapy Followed by Bone Marrow Transplantation in Treating Patients With Advanced Hematologic Cancer	Completed	National Cancer Institute (NCI)	Phase 2	2000-12-01	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 busulfan and melphalan followed by donor bone marrow transplantation in treating patients who have advanced hematologic cancer.

Clinical Trial Conditions for Busulfex

Condition Name

Condition Name for Busulfex
Intervention	Trials
Leukemia	33
Myelodysplastic Syndrome	25
Lymphoma	21
Acute Myeloid Leukemia	18

Condition MeSH

Condition MeSH for Busulfex
Intervention	Trials
Leukemia	62
Myelodysplastic Syndromes	41
Preleukemia	40
Leukemia, Myeloid	39

Clinical Trial Locations for Busulfex

Trials by Country

Trials by Country for Busulfex
Location	Trials
United States	311
Canada	19
Korea, Republic of	4
Israel	2
China	2

Trials by US State

Trials by US State for Busulfex
Location	Trials
Texas	54
New York	17
Minnesota	17
Massachusetts	15
California	13

Clinical Trial Progress for Busulfex

Clinical Trial Phase

Clinical Trial Phase for Busulfex
Clinical Trial Phase	Trials
Phase 3	8
Phase 2/Phase 3	5
Phase 2	79

Clinical Trial Status

Clinical Trial Status for Busulfex
Clinical Trial Phase	Trials
Completed	57
Recruiting	29
Terminated	24

Clinical Trial Sponsors for Busulfex

Sponsor Name

Sponsor Name for Busulfex
Sponsor	Trials
M.D. Anderson Cancer Center	47
National Cancer Institute (NCI)	42
Masonic Cancer Center, University of Minnesota	14

Sponsor Type

Sponsor Type for Busulfex
Sponsor	Trials
Other	177
NIH	50
Industry	25

Busulfan: Clinical Trials, Market Analysis, and Projections

Introduction to Busulfan

Busulfan is a chemotherapeutic agent used primarily in the treatment of chronic myelogenous leukemia (CML) and other hematologic malignancies. It is often employed as part of a conditioning regimen for allogeneic hematopoietic stem cell transplantation.

Clinical Trials and Efficacy

Conditioning Regimens for HCT

A significant clinical trial compared two myeloablative conditioning regimens for allogeneic hematopoietic cell transplantation (HCT): the BuCy regimen (busulfan plus cyclophosphamide) and the BuFlu regimen (busulfan plus fludarabine)[1].

• BuCy vs. BuFlu Regimen: The trial involved 126 patients, with 64 receiving the BuCy regimen and 62 receiving the BuFlu regimen. The results indicated that the BuCy regimen had better overall survival (OS), relapse-free survival (RFS), and event-free survival (EFS) compared to the BuFlu regimen. Specifically, the OS at 2 years was 67.4% for BuCy versus 41.4% for BuFlu[1].

Prospective Clinical Trial of Busulfan Injection

A prospective, single-arm, open-label study involving 61 patients evaluated the safety and efficacy of Busulfan Injection as part of a conditioning regimen for allogeneic hematopoietic stem cell transplantation. Patients received busulfan followed by cyclophosphamide (BuCy2 regimen). The study demonstrated effective myeloablation, with all patients receiving the full scheduled dose of busulfan[4].

Literature Review and Historical Studies

Historical studies, including those by Clift, Devergie, Ringden, and Blume, have also evaluated the efficacy of busulfan-containing conditioning regimens. These studies compared busulfan/cyclophosphamide (BU/CY) with cyclophosphamide/total body irradiation (CY/TBI) and showed favorable outcomes for the BU/CY regimen in patients with CML and other hematologic malignancies[4].

Market Analysis

Global Market Size and Growth

The global busulfan market is expected to grow significantly over the next few years. As of 2023, the market size was valued at a substantial amount, with a projected compound annual growth rate (CAGR) from 2024 to 2031[2][5].

• Market Trends: The market growth is driven by factors such as the increasing prevalence of hematologic malignancies, advancements in transplantation technologies, and the globalization of clinical trials. The COVID-19 pandemic also accelerated the development and investment in novel drug treatments, including those involving busulfan[3][5].

Regional Analysis

The busulfan market is segmented by region, with North America, Europe, Asia, and other regions contributing to the global market size.

• North America and Europe: These regions are expected to maintain significant market shares due to advanced healthcare infrastructure and high adoption rates of new treatments.
• Asia: This region is anticipated to show rapid growth due to increasing healthcare spending and a large patient population[5].

Competitive Landscape

The busulfan market is competitive, with several key players involved in the production and distribution of busulfan in various forms, including injections and tablets.

• Key Players: Companies such as Pfizer and others are prominent in the market, with a focus on improving formulations and delivery methods to enhance patient outcomes[5].

Market Projections

Forecasted Market Size

The global busulfan market is projected to grow substantially from 2025 to 2030, driven by increasing demand and technological advancements.

• CAGR: The market is expected to grow at a CAGR of around XX% from 2024 to 2031, indicating a robust growth trajectory[2][5].

Segment Analysis

The market is segmented by type (injection and tablet) and application (various hematologic malignancies).

• Type: The injection form is expected to dominate the market due to its ease of administration and precise dosing.
• Application: The use of busulfan in conditioning regimens for HCT is the primary application, with growing demand in treating CML, acute myelogenous leukemia (AML), and other conditions[5].

Challenges and Opportunities

Challenges

• Adverse Events: Clinical trials have highlighted the potential for severe adverse events, such as infections and gastrointestinal issues, associated with busulfan-containing regimens[1][4].
• Regulatory Hurdles: The development and approval of new formulations or regimens involving busulfan can be complex and time-consuming.

Opportunities

• Technological Advancements: Improvements in drug delivery systems and personalized medicine approaches could enhance the efficacy and safety of busulfan treatments.
• Expanding Indications: Research into new indications for busulfan could expand its market potential beyond current uses.

Key Takeaways

• Clinical Efficacy: Busulfan, particularly in the BuCy regimen, has demonstrated superior outcomes in clinical trials for HCT conditioning.
• Market Growth: The global busulfan market is expected to grow significantly, driven by increasing demand and technological advancements.
• Regional Variations: The market is segmented by region, with North America and Europe leading and Asia showing rapid growth.
• Challenges and Opportunities: While adverse events and regulatory hurdles exist, opportunities for technological advancements and expanding indications are promising.

FAQs

What is the primary use of busulfan in clinical settings?

Busulfan is primarily used as part of a conditioning regimen for allogeneic hematopoietic stem cell transplantation, particularly in the treatment of chronic myelogenous leukemia (CML) and other hematologic malignancies.

Which conditioning regimen is more effective: BuCy or BuFlu?

The BuCy regimen (busulfan plus cyclophosphamide) has been shown to be more effective than the BuFlu regimen (busulfan plus fludarabine) in terms of overall survival, relapse-free survival, and event-free survival[1].

What are the common adverse events associated with busulfan?

Common adverse events include severe infections, gastrointestinal issues, and hepatic adverse events, although the frequencies of these events can vary between different regimens[1][4].

How is the global busulfan market expected to grow?

The global busulfan market is projected to grow at a significant CAGR from 2024 to 2031, driven by increasing demand, technological advancements, and the globalization of clinical trials[2][5].

What are the key regions driving the busulfan market growth?

North America and Europe are currently the leading regions, but Asia is expected to show rapid growth due to increasing healthcare spending and a large patient population[5].

Sources

1. Randomized trial of myeloablative conditioning regimens: busulfan ... - PubMed
2. Busulfan Market Report 2024 (Global Edition) - Cognitive Market Research
3. Clinical Trials Market Size, Share And Growth Report, 2030 - Grand View Research
4. BUSULFAN INJECTION Clinical Studies - Pfizer Medical Information
5. Global Busulfan Market Insights, Forecast to 2030 - GII Research