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
Myelodysplastic Syndromes	89
Lymphoma	87
Acute Myeloid Leukemia	61

Condition MeSH

Condition MeSH for busulfan
Intervention	Trials
Leukemia	264
Myelodysplastic Syndromes	181
Preleukemia	166
Leukemia, Myeloid, Acute	157

Clinical Trial Locations for busulfan

Trials by Country

Trials by Country for busulfan
Location	Trials
Canada	80
China	76
France	44
Italy	42
Germany	33

Trials by US State

Trials by US State for busulfan
Location	Trials
Texas	91
New York	79
Maryland	67
California	67
Washington	56

Clinical Trial Progress for busulfan

Clinical Trial Phase

Clinical Trial Phase for busulfan
Clinical Trial Phase	Trials
PHASE3	2
PHASE2	20
PHASE1	8

Clinical Trial Status

Clinical Trial Status for busulfan
Clinical Trial Phase	Trials
Completed	252
Recruiting	135
Unknown status	67

Clinical Trial Sponsors for busulfan

Sponsor Name

Sponsor Name for busulfan
Sponsor	Trials
National Cancer Institute (NCI)	166
M.D. Anderson Cancer Center	56
Fred Hutchinson Cancer Research Center	35

Sponsor Type

Sponsor Type for busulfan
Sponsor	Trials
Other	853
NIH	207
Industry	95

Last updated: July 26, 2026

Busulfan clinical trials update, market analysis, and market projections (global brands, generics, and pipeline risks)

Busulfan is an established chemotherapy agent used in hematologic malignancies and as a myeloablative conditioning regimen before hematopoietic stem cell transplant (HSCT). Clinical development activity in busulfan is structurally different from late-stage “new drug” programs: much of the market is driven by (1) formulation and dosing-regimen studies, (2) transplant conditioning protocols, and (3) biosimilar-like competitive dynamics are not applicable because busulfan is a small molecule with largely generic competition. The practical investment and competitive question is not “will busulfan be approved,” but how quickly different formulations and administration pathways (oral vs IV), plus conditioning regimen protocol changes, affect utilization and pricing.

No sufficient, drug-specific, source-verifiable clinical trial status and market metric set is provided in the prompt to produce a complete and accurate “clinical trials update,” “market analysis,” and “projection” without risking fabricated claims.

What patents protect busulfan and its formulations?

No source-verifiable patent estate details were provided.

When does busulfan lose exclusivity?

No source-verifiable exclusivity or expiration dates were provided.

What generic entry risks exist for busulfan?

No source-verifiable Orange Book status, FDA approval history, or pending Paragraph IV litigation were provided.

What is the Orange Book status of busulfan products?

No source-verifiable Orange Book listing data (application numbers, reference listed drug, patents, exclusivity codes) was provided.

What formulations are protected by busulfan patents (oral vs IV)?

No source-verifiable formulation patent details were provided.

What patent litigation affects busulfan?

No source-verifiable litigation dockets, settlements, or injunction timelines were provided.

How does busulfan compare with conditioning alternatives (BEAM, melphalan, treosulfan, fludarabine/cyclophosphamide)?

No source-verifiable comparative utilization, trial endpoints, or guideline adoption data was provided.

What clinical trials update is available for busulfan right now?

No source-verifiable trial registry updates (ClinicalTrials.gov NCT numbers, status, enrollment changes, results timing) were provided.

How big is the busulfan market and how fast does it grow?

No source-verifiable market size, prescription volumes, pricing, or regional breakdowns were provided.

How much busulfan revenue exposure do generics create?

No source-verifiable revenue attribution or WAC/AWP-to-net pricing data was provided.

What market projections are realistic for busulfan through 2030?

No source-verifiable base-year market, scenario drivers, or dataset-backed forecasting approach was provided.

Key Takeaways

• Busulfan is used primarily in HSCT conditioning, where utilization is protocol-driven rather than pipeline-driven.
• Competitive dynamics are dominated by generic availability and regimen adoption, not by biologics-style competition.
• A credible “clinical trials update” and numeric “market projection” require source-verifiable trial and market datasets that were not provided in the prompt.

FAQs

1. How are IV vs oral busulfan dosing protocols changing HSCT conditioning practice?
2. Which transplant regimens are substituting away from busulfan in specific indications?
3. What is the impact of therapeutic drug monitoring (TDM) on busulfan exposure targets?
4. How do generic launches affect busulfan net pricing in US and EU markets?
5. What endpoints in conditioning trials most influence guideline updates for busulfan use?

References

No sources were provided to cite.