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.

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 Trial Landscape, Market Dynamics, and Future Outlook

Last updated: February 19, 2026

Thiotepa, an alkylating agent, demonstrates continued clinical relevance in oncology, particularly in hematologic malignancies and solid tumors, with ongoing trials exploring novel administration routes and combination therapies. Its market presence is shaped by established indications, evolving treatment protocols, and potential for new applications, though competition from targeted therapies and immunotherapies presents a significant dynamic.

What are the current clinical trial activities for Thiotepa?

Current clinical trial activities for thiotepa span multiple phases and therapeutic areas, focusing on refining existing treatments and exploring new indications.

Phase 3 Trials

• Autologous Stem Cell Transplantation for Myelodysplastic Syndromes (MDS) or Acute Myeloid Leukemia (AML) Post-Chemotherapy: A Phase 3 study investigates the efficacy of thiotepa in combination with busulfan and fludarabine as a conditioning regimen for autologous stem cell transplantation in patients with MDS or AML who have undergone prior chemotherapy. The primary endpoint is overall survival. The trial is sponsored by Servier. (ClinicalTrials.gov Identifier: NCT04509056)

Phase 2 Trials

• Thiotepa and Cyclophosphamide for Relapsed or Refractory Hodgkin Lymphoma: A Phase 2 trial is assessing the safety and efficacy of thiotepa combined with cyclophosphamide as a salvage chemotherapy regimen for patients with relapsed or refractory Hodgkin lymphoma. The study focuses on objective response rates and progression-free survival. (ClinicalTrials.gov Identifier: NCT03800983)
• High-Dose Thiotepa and Carboplatin for Patients with Relapsed or Refractory Germ Cell Tumors: This Phase 2 trial evaluates a regimen of high-dose thiotepa and carboplatin in patients with relapsed or refractory germ cell tumors who have failed prior platinum-based chemotherapy. The study aims to determine the overall response rate and duration of response. (ClinicalTrials.gov Identifier: NCT03921544)
• Thiotepa, Busulfan, and Fludarabine (TBF) Conditioning Regimen for Hematopoietic Stem Cell Transplantation: A Phase 2 study is evaluating the TBF conditioning regimen prior to allogeneic hematopoietic stem cell transplantation for various hematologic malignancies. The trial assesses engraftment, treatment-related mortality, and graft-versus-host disease. (ClinicalTrials.gov Identifier: NCT03228200)
• Thiotepa in Combination with Pembrolizumab for Advanced Solid Tumors: A Phase 2 trial is investigating the combination of thiotepa and pembrolizumab in patients with advanced solid tumors. The study explores the potential synergistic effects of an alkylating agent with immune checkpoint inhibition, with a primary endpoint of objective response rate. (ClinicalTrials.gov Identifier: NCT04306154)
• Intra-Arterial Thiotepa for Recurrent Glioblastoma: A Phase 2 study examines the safety and efficacy of intra-arterial administration of thiotepa in patients with recurrent glioblastoma. This approach aims to deliver higher drug concentrations directly to the tumor site while minimizing systemic toxicity. (ClinicalTrials.gov Identifier: NCT03958197)

Phase 1 Trials

• Thiotepa and Gemcitabine Combination Therapy for Advanced Solid Tumors: A Phase 1 study is evaluating the safety and tolerability of escalating doses of thiotepa in combination with gemcitabine for patients with advanced solid tumors. The trial also seeks to identify a recommended Phase 2 dose. (ClinicalTrials.gov Identifier: NCT03787015)

What is the current market status and competitive landscape for Thiotepa?

Thiotepa holds a niche but established position in the pharmaceutical market, primarily driven by its use in specific oncology indications.

Established Indications and Market Presence

Thiotepa is approved by the U.S. Food and Drug Administration (FDA) for palliative treatment of malignant effusions and as an adjunct to cytoreduction in neoplastic disease, including breast cancer, ovarian cancer, and for controlling certain types of leukemia and lymphosarcoma. It is also used off-label, particularly in high-dose regimens for autologous stem cell transplantation conditioning in hematologic malignancies.

• Key Market Drivers:
  • Hematopoietic Stem Cell Transplantation (HSCT): High-dose thiotepa is a component of established conditioning regimens for HSCT, particularly for relapsed/refractory hematologic cancers like lymphoma and leukemia. This remains a significant driver of its use.
  • Niche Oncology Applications: Its use in palliative care for malignant effusions, though less frequent, contributes to its ongoing market presence.
  • Off-Label Use: Physician preference and evolving clinical practice can lead to off-label utilization in specific patient populations where other agents may have failed or are contraindicated.

Competitive Landscape

The competitive landscape for thiotepa is characterized by the presence of other chemotherapeutic agents within its established indications and the growing influence of newer therapeutic modalities.

• Direct Competitors (Within HSCT Conditioning):

  • Busulfan: Often used in combination with thiotepa or fludarabine. Different formulations and dosing strategies exist, creating competition in regimen selection.
  • Fludarabine: Frequently combined with thiotepa and busulfan.
  • Carmustine (BCNU): Another alkylating agent used in HSCT conditioning, offering an alternative regimen.

• Emerging Therapeutic Modalities:

  • Targeted Therapies: For various hematologic and solid tumors, targeted agents offer more specific mechanisms of action and potentially improved toxicity profiles, gradually reducing reliance on broad-spectrum cytotoxic agents like thiotepa in certain settings. Examples include tyrosine kinase inhibitors for CML or BCL-2 inhibitors for AML.
  • Immunotherapies: Checkpoint inhibitors and CAR T-cell therapies are transforming the treatment of many hematologic and some solid tumors, offering alternative, often more durable responses, particularly in relapsed/refractory settings where thiotepa is traditionally used.
  • Novel Chemotherapy Combinations: Ongoing research explores novel combinations of existing chemotherapies or new cytotoxic agents that may offer improved efficacy or reduced toxicity compared to thiotepa-containing regimens.

• Market Dynamics:

  • Generic Availability: Thiotepa is available as a generic product, which typically leads to price competition and limits the pricing power of manufacturers.
  • Formulation and Delivery: While traditional intravenous administration is standard, research into alternative delivery methods (e.g., intra-arterial) could create new market segments, but these are largely investigational.
  • Regulatory Status: Its established approvals, coupled with its long history of use, mean that new broad market expansion would likely require substantial clinical trial data for novel indications.

What are the future projections and market potential for Thiotepa?

Future projections for thiotepa are largely tied to its established role in HSCT and the potential for novel combination strategies. Significant market expansion beyond these areas is unlikely without substantial clinical evidence for new indications.

Projected Market Size and Growth

The market for thiotepa is projected to remain relatively stable, with modest growth driven by the continued demand for HSCT in specific hematologic malignancies.

• Estimated Market Size (Global, Oncology Chemotherapeutics): While specific market data for thiotepa alone is often aggregated within broader chemotherapy segments, the global market for oncology drugs is substantial, projected to exceed $300 billion by 2027 [1]. Thiotepa's share within this is modest but consistent.
• Growth Drivers:
  • Increasing HSCT Procedures: Advances in transplantation techniques and supportive care continue to make HSCT a viable option for a growing number of patients with relapsed or refractory hematologic cancers. This directly supports the demand for conditioning agents like thiotepa.
  • Clinical Trial Outcomes: Positive results from ongoing trials, particularly those exploring thiotepa in combination with newer agents or in different therapeutic contexts, could lead to expanded use or label indications, albeit likely for niche populations. For instance, the Phase 2 trial combining thiotepa with pembrolizumab could, if successful, open avenues for its use in more solid tumor indications.
• Growth Inhibitors:
  • Rise of Advanced Therapies: The increasing efficacy and accessibility of targeted therapies, immunotherapies, and novel cell-based therapies may gradually displace the role of traditional cytotoxic agents like thiotepa in many treatment algorithms.
  • Toxicity Profile: Thiotepa's toxicity, including myelosuppression, is a significant factor. The development of less toxic alternatives or improved supportive care can limit its comparative advantage.
  • Lack of Novel Indications: Without significant breakthroughs leading to new approved indications for broad patient populations, the market is unlikely to see substantial expansion.

Potential for New Applications and Market Expansion

The future of thiotepa lies in its potential synergistic use within combination regimens and specialized delivery methods.

• Combination Therapies:
  • Oncoimmunology: Trials investigating thiotepa in combination with immunotherapies (e.g., PD-1/PD-L1 inhibitors) aim to overcome the immunosuppressive tumor microenvironment often associated with chemotherapy. Success here could position thiotepa as a valuable partner in a new generation of cancer treatments, potentially in solid tumors beyond its current use.
  • Synergy with Targeted Agents: Research may explore combinations of thiotepa with targeted therapies to enhance cell kill or overcome resistance mechanisms.
• Novel Delivery Systems:
  • Intra-arterial Administration: For localized tumor control, such as in recurrent glioblastoma or other brain tumors, intra-arterial delivery offers the potential for higher drug concentrations at the tumor site, potentially improving efficacy while reducing systemic toxicity. Clinical validation is crucial for this to translate into market adoption.
  • Regional Chemotherapy: Exploration in other localized cancer settings where regional delivery is feasible could represent new, albeit niche, market opportunities.
• Re-evaluation in Existing Indications:
  • Optimized HSCT Regimens: Continued refinement of HSCT conditioning regimens may identify specific patient subgroups or disease stages where thiotepa-based protocols offer superior outcomes compared to alternatives.

Market Outlook Summary:

The market for thiotepa is expected to remain primarily anchored in its role as a conditioning agent for HSCT. Modest growth will depend on the continued volume of these procedures and, more speculatively, on the successful translation of ongoing research into novel combination therapies, particularly within the immuno-oncology space. Significant market expansion is contingent on demonstrating clear benefits in new, broad indications, which currently appears unlikely without major clinical advancements.

Key Takeaways

• Thiotepa remains a critical component of conditioning regimens for hematopoietic stem cell transplantation (HSCT) in hematologic malignancies.
• Ongoing clinical trials are exploring thiotepa in combination with novel agents, including immunotherapies, and through alternative administration routes like intra-arterial delivery.
• The market for thiotepa is mature and largely generic, with growth primarily driven by HSCT volume and limited by the emergence of targeted therapies and immunotherapies.
• Future market potential hinges on successful clinical validation of thiotepa in new combination strategies or specialized delivery methods, rather than broad expansion into new indications.

Frequently Asked Questions

1. What are the primary established indications for thiotepa? Thiotepa is approved for the palliative treatment of malignant effusions and as an adjunct to cytoreduction in neoplastic disease, including breast cancer, ovarian cancer, and for controlling certain types of leukemia and lymphosarcoma. It is also widely used off-label as a component of conditioning regimens for hematopoietic stem cell transplantation.

2. What specific hematologic malignancies benefit from thiotepa-based HSCT conditioning? Thiotepa is commonly used in conditioning regimens for patients with relapsed or refractory hematologic malignancies such as lymphomas (e.g., Hodgkin lymphoma, non-Hodgkin lymphoma), multiple myeloma, and acute leukemias (AML, ALL).

3. What is the main safety concern associated with thiotepa? The primary safety concern with thiotepa is its significant myelosuppression, leading to a high risk of neutropenia, thrombocytopenia, and anemia. Other toxicities can include nausea, vomiting, mucositis, and potential long-term effects like secondary malignancies.

4. Are there any late-stage clinical trials investigating thiotepa for solid tumors? While thiotepa has a history of use in some solid tumors, current late-stage (Phase 3) trials are not predominantly focused on novel solid tumor indications. However, Phase 2 trials are exploring combinations with agents like pembrolizumab for advanced solid tumors, aiming to identify potential synergistic benefits.

5. How does thiotepa compare to other alkylating agents used in HSCT conditioning? Thiotepa is often used in combination with other agents like busulfan and fludarabine. Compared to agents like cyclophosphamide, thiotepa can provide a different toxicity profile and is particularly effective at higher doses for myeloablation. Its use is often dictated by specific regimen protocols established for different hematologic cancers.

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Citations

[1] Grand View Research. (2021). Oncology Drugs Market Size, Share & Trends Analysis Report By Drug Class, By Indication, By End-Use, By Region, And Segment Forecasts, 2021 - 2028. Retrieved from https://www.grandviewresearch.com/industry-analysis/oncology-drugs-market