CLINICAL TRIALS PROFILE FOR THIOTEPA

All Clinical Trials for thiotepa

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00001498 ↗	A Pilot Trial of Sequential Chemotherapy With Antimetabolite Induction, High-Dose Alkylating Agent Consolidation With Peripheral Blood Progenitor Cell Support, and Intensification With Paclitaxel and Doxorubicin for Patients With High-Risk Breast Ca	Completed	National Cancer Institute (NCI)	Phase 2	1996-02-01	Stage III patients may begin therapy prior to or following surgery. Patients with undrainable significant third space fluid collection (e.g., pleural effusions, ascites) are entered directly on Consolidation. Patients receive induction chemotherapy with methotrexate and fluorouracil every 2 weeks for 4 courses. Patients then receive two 3-week courses of consolidation therapy with cyclophosphamide, followed by daily granulocyte colony-stimulating factor until completion of leukapheresis. Patients next receive myeloablative doses of thiotepa followed by stem cell rescue and granulocyte colony-stimulating factor. After hematopoietic reconstitution, patients receive 24-hour infusions of paclitaxel every 3 weeks for 4 doses, followed by doxorubicin or vinblastine every 3 weeks for 4 doses. Patients are then evaluated for additional therapy (surgery, radiotherapy, or hormonal therapy) as appropriate. Patients are followed every 3 months for 1 year, then every 6 months.
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.
NCT00002471 ↗	Combination Chemotherapy in Treating Patients With Acute B-Lymphoblastic Leukemia or Non-Hodgkin's Lymphoma	Completed	Memorial Sloan Kettering Cancer Center	Phase 2	1990-02-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining more than one drug may kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy in treating patients who have acute B-lymphoblastic leukemia or recurrent non-Hodgkin's lymphoma.
NCT00002508 ↗	Combination Chemotherapy Followed by Bone Marrow or Stem Cell Transplantation in Treating Patients With Relapsed or Refractory Germ Cell Tumors	Completed	Fox Chase Cancer Center	Phase 1/Phase 2	1990-11-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Peripheral stem cell transplantation may allow doctors to give higher doses of chemotherapy and kill more tumor cells. PURPOSE: Phase I/II trial to study the effectiveness of combination chemotherapy followed by peripheral stem cell transplantation or bone marrow transplantation in treating patients who have relapsed or recurrent germ cell cancer.
NCT00002508 ↗	Combination Chemotherapy Followed by Bone Marrow or Stem Cell Transplantation in Treating Patients With Relapsed or Refractory Germ Cell Tumors	Completed	Temple University	Phase 1/Phase 2	1990-11-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Peripheral stem cell transplantation may allow doctors to give higher doses of chemotherapy and kill more tumor cells. PURPOSE: Phase I/II trial to study the effectiveness of combination chemotherapy followed by peripheral stem cell transplantation or bone marrow transplantation in treating patients who have relapsed or recurrent germ cell cancer.
NCT00002515 ↗	Combination Chemotherapy Followed by Bone Marrow Transplantation in Treating Patients With Rare Cancer	Completed	Memorial Sloan Kettering Cancer Center	Phase 2	1992-10-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Bone marrow transplantation may allow doctors to give higher doses of chemotherapy and kill more tumor cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy with thiotepa, carboplatin, and topotecan followed by bone marrow transplantation in treating patients who have metastatic or progressive rare cancer.
NCT00002534 ↗	Bone Marrow Transplantation in Treating Patients With Acute Leukemia in First or Second Remission	Completed	National Cancer Institute (NCI)	Phase 3	1993-05-01	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: Randomized phase III trial to compare the effectiveness of bone marrow transplantation using untreated or treated bone marrow in treating patients with acute leukemia in first or second remission.

Clinical Trial Conditions for thiotepa

Condition Name

Condition Name for thiotepa
Intervention	Trials
Leukemia	33
Lymphoma	29
Breast Cancer	28
Myelodysplastic Syndromes	22

Condition MeSH

Condition MeSH for thiotepa
Intervention	Trials
Leukemia	78
Myelodysplastic Syndromes	54
Preleukemia	49
Precursor Cell Lymphoblastic Leukemia-Lymphoma	48

Clinical Trial Locations for thiotepa

Trials by Country

Trials by Country for thiotepa
Location	Trials
United States	801
Canada	59
China	26
Australia	24
United Kingdom	17

Trials by US State

Trials by US State for thiotepa
Location	Trials
New York	67
Texas	40
Pennsylvania	38
California	35
Washington	34

Clinical Trial Progress for thiotepa

Clinical Trial Phase

Clinical Trial Phase for thiotepa
Clinical Trial Phase	Trials
PHASE2	18
PHASE1	4
Phase 4	1

Clinical Trial Status

Clinical Trial Status for thiotepa
Clinical Trial Phase	Trials
Completed	101
Recruiting	76
Terminated	26

Clinical Trial Sponsors for thiotepa

Sponsor Name

Sponsor Name for thiotepa
Sponsor	Trials
National Cancer Institute (NCI)	86
Fred Hutchinson Cancer Research Center	21
Memorial Sloan Kettering Cancer Center	20

Sponsor Type

Sponsor Type for thiotepa
Sponsor	Trials
Other	329
NIH	97
Industry	28

Thiotepa clinical trials update, market analysis, and forecast (2026–2036)

Last updated: May 20, 2026

Thiotepa is an older, off-patent alkylating chemotherapy with current use concentrated in high-acuity oncology settings, including conditioning regimens for hematopoietic stem cell transplantation (HSCT) and select bladder cancer protocols. Commercial availability is largely driven by supply continuity and hospital/institutional purchasing rather than brand-led life-cycle management. The near-term market outlook hinges on (1) continued institutional demand for transplant conditioning, (2) uptake in bladder cancer and other salvage-adjacent indications where thiotepa is incorporated, and (3) whether reformulations or combination regimens generate incremental clinical adoption.

Clinical development and “update” reality check No credible basis exists here to produce a complete, accurate, current “clinical trials update” for thiotepa without using specific trial identifiers (NCT/EudraCT), sponsor datasets, or up-to-date registry extracts. The same constraint applies to building a defensible forecast tied to specific active interventional studies, because thiotepa’s market is typically influenced by regimen inclusion and regional procurement more than by a single late-stage pivotal program.

Given these constraints, a complete and accurate clinical-trials update and projection cannot be produced.

What is thiotepa’s current clinical use and how is it positioned in treatment regimens?

Thiotepa is used as an alkylating agent in conditioning regimens for HSCT and in certain uro-oncology settings. Commercially, this typically maps to:

• HSCT conditioning: high-acuity, protocol-driven administration in transplant centers.
• Urothelial/bladder cancer: thiotepa appears in specific intravesical or salvage protocols depending on jurisdiction and local clinical practice.

Which diseases drive thiotepa demand in oncology?

Demand centers are usually:

• Hematologic malignancies requiring HSCT conditioning.
• Selected solid-tumor and urothelial salvage pathways where thiotepa is incorporated as part of combination or device-free chemotherapy protocols.

What delivery formats and dosing patterns matter commercially?

Market access and utilization generally depend on:

• Hospital supply reliability (dose form, packaging, and availability).
• Protocol inclusion at transplant centers and oncology networks.
• Compatibility with conditioning schedules and supportive care infrastructure.

How big is the thiotepa market today and what are the key value drivers?

A complete market size, by geography and formulation, cannot be stated accurately without access to up-to-date sales datasets, payer and procurement disclosures, or regulator-linked utilization reporting.

What can be stated at a decision-useful level:

• Thiotepa is structurally an “older chemo” product category where volumes are affected more by transplant/oncology procedure volumes than by sustained brand price power.
• Pricing is typically constrained by competition and generic availability.
• Value is more sensitive to supply continuity and dosing outcomes in institutional procurement than to incremental product differentiation.

What drives procurement in transplant conditioning?

Key drivers:

• Number of HSCT procedures and patient eligibility.
• Institutional protocol preferences (conditioning regimen selection).
• Formulary decisions and logistics across hospital networks.

What drives procurement in uro-oncology?

Key drivers:

• Adoption of thiotepa-containing salvage or bladder treatment protocols.
• Availability of alternatives and intravesical regimen preference patterns.

When does thiotepa face generic or competitive pressure and what does that do to pricing?

Thiotepa is widely characterized as off-patent; therefore, competitive pressure is generally ongoing. The practical impact on commercialization is:

• Lower unit pricing versus origin-controlled eras.
• Profitability depends on manufacturing cost, supply reliability, and contract pricing with hospital groups.

What competitive landscape risks matter most?

• Supply interruptions for injectable oncology agents.
• Manufacturing scale constraints for older cytotoxics.
• Regional sourcing dependencies.

What clinical trial signals would change thiotepa’s adoption rate?

For thiotepa, adoption changes typically come from:

• Prospective evidence supporting regimen efficacy/safety in transplant conditioning or salvage settings.
• Demonstration of reduced toxicity or improved disease control through combination strategies.
• Expansion into new lines of therapy where thiotepa becomes a protocol-standard component.

How should investors interpret trial outcomes for thiotepa?

Because thiotepa’s use is protocol-driven, the market impact of trial results tends to be high when:

• Evidence supports a regimen shift across major transplant or oncology networks.
• Results are practice-changing rather than incremental, especially where dosing can be integrated without major workflow disruption.

How does thiotepa compare with alternative conditioning agents and bladder salvage drugs?

Thiotepa competes in the therapeutic “function” sense rather than through direct one-to-one substitution:

• In HSCT conditioning, it competes with other alkylators and conditioning combinations depending on disease risk and transplant protocols.
• In bladder cancer, it competes with other intravesical and systemic salvage regimens.

Where thiotepa tends to fit in regimen selection

• When protocols call for thiotepa-containing intensity or specific regimen architecture.
• When patient and disease characteristics align with thiotepa’s historical evidence base.

What is the regulatory status of thiotepa in the US and EU?

A complete, accurate regulatory status summary requires current labeling and marketing authorization details by jurisdiction, including any changes to indications, warnings, and dosing regimens.

Without up-to-date primary-source checks (FDA labeling, EMA product information, and local SmPC changes), the status cannot be stated precisely.

What is the revenue outlook for thiotepa under base, bull, and bear scenarios?

A defensible scenario forecast requires:

• Current baseline revenue and unit volume.
• Active clinical pipeline inputs tied to adoption curves.
• Pricing and competitive erosion assumptions by geography.

No such dataset is available in this response, so a quantified forecast would be speculative.

What patent and exclusivity factors affect thiotepa commercialization?

Thiotepa is an older active ingredient, and the commercial market generally behaves as generic-led. A litigation- or exclusivity-driven forecast requires Orange Book and jurisdictional patent estate mapping by drug product and strength, which is not provided here.

Key Takeaways

• Thiotepa’s commercialization is primarily protocol-driven in HSCT conditioning and select uro-oncology use, with pricing and volumes shaped by competition and institutional procurement.
• A “clinical trials update” and a quantified market forecast cannot be produced accurately without current trial registry extracts and up-to-date market/pricing baselines.
• The adoption hinge points are practice-changing evidence in conditioning or salvage regimens and supply continuity that sustains hospital contracting.

FAQs

1. Is thiotepa used in transplant conditioning for all hematologic malignancies?
2. What factors influence hospital formulary adoption of thiotepa injectable products?
3. How does thiotepa’s competitive positioning differ in HSCT conditioning versus bladder salvage?
4. What types of thiotepa clinical trial endpoints would most affect real-world adoption?
5. How do supply disruptions for cytotoxic injectables typically impact thiotepa availability and revenue?

References (APA)

1. (No primary sources were cited because current thiotepa-specific clinical registry, label, and sales data were not provided in the prompt.)