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
Lymphoma	48

Clinical Trial Locations for THIOTEPA

Trials by Country

Trials by Country for THIOTEPA
Location	Trials
United States	800
Canada	59
China	25
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	15
PHASE1	3
Phase 4	1

Clinical Trial Status

Clinical Trial Status for THIOTEPA
Clinical Trial Phase	Trials
Completed	101
Recruiting	75
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	325
NIH	97
Industry	28

Clinical Trials Update, Market Analysis, and Projection for Thiotepa

Last updated: October 25, 2025

Introduction

Thiotepa (C4H8N3SSi), a pioneering alkylating agent used primarily in cancer therapy, has historically played a critical role in treating various malignancies, including bladder cancer, ovarian cancer, and certain hematological malignancies. As a chemotherapeutic agent with a unique mechanism of cross-linking DNA, thiotepa’s clinical development and regulatory landscape continue to evolve. This article provides an up-to-date overview of the latest clinical trials, market dynamics, and future projections for thiotepa, delivering essential insights for pharmaceutical companies, investors, and healthcare stakeholders.

Clinical Trials Update

Recent Developments and Ongoing Studies

Over the past two years, the clinical landscape for thiotepa has experienced noteworthy shifts, driven by advances in oncology research and evolving therapeutic strategies. While thiotepa’s traditional uses remain relevant, recent clinical trials have expanded into novel formulations and combinatorial regimens to improve efficacy and reduce toxicity.

1. New Clinical Trial Initiatives

Recent clinical trials registered on ClinicalTrials.gov highlight renewed interest in thiotepa. Notably, the following studies are in varying phases:

• Combination Therapies: Multiple Phase I/II studies are evaluating thiotepa’s synergistic effects when combined with immune checkpoint inhibitors (e.g., pembrolizumab) for metastatic bladder and ovarian cancers. For instance, a trial titled “Thiotepa Plus Pembrolizumab in Relapsed Ovarian and Bladder Carcinoma” (NCT04807130) is exploring safety profiles and preliminary efficacy data.

• Intravesical Use: Several ongoing studies assess intraurethral thiotepa for non-muscle invasive bladder cancer (NMIBC), aiming to reduce systemic toxicity.

2. Formulation Advances and Delivery Systems

Researchers are developing targeted delivery mechanisms, such as liposomal thiotepa and nanoparticle-based formulations, to optimize drug bioavailability and minimize adverse effects. These pipelines are still in early clinical or preclinical phases but hold potential for expanding thiotepa’s therapeutic window.

3. Regulatory and Patent Landscape

The U.S. Food and Drug Administration (FDA) has not recently approved new indications for thiotepa. However, orphan drug designations and patent extensions for specific formulations are under consideration, potentially incentivizing further clinical exploration.

Critical Challenges in Clinical Development

Despite these advancements, thiotepa’s clinical development faces hurdles, including:

• Toxicity Profile: Myelosuppression and neurotoxicity remain significant, necessitating careful dose optimization.

• Limited Innovation: Competition from targeted therapies and immunotherapies with better safety profiles restrict thiotepa’s next-generation development.

Market Analysis

Historical Market Performance

Thiotepa’s commercial success has historically been limited to niche indications, including conditioning regimens for hematopoietic stem cell transplantation (HSCT) and treatment of specific solid tumors. Its market peaked during the early 2000s, with several generic formulations available globally, especially in Europe and Asia.

Current Market Dynamics

The global anti-cancer drug market, projected to exceed USD 250 billion by 2027 [1], is increasingly driven by immunotherapy and targeted agents, which have overshadowed traditional chemotherapeutics like thiotepa in some areas.

However, niche segments retain demand:

• HSCT Conditioning: Thiotepa remains a key component in chemotherapy protocols for high-dose conditioning, particularly in Europe and Asia.

• Refractory or Rare Tumors: In rare cancers or cases where newer agents are unavailable or contraindicated, thiotepa still features prominently.

Market Drivers and Barriers

Drivers:

• Growing Need for Effective Conditioning Regimens: As HSCT expands, especially for hematological malignancies, thiotepa’s role persists.

• Emerging Combination Regimens: Synergistic protocols with immunotherapy may create new market opportunities.

Barriers:

• Adverse Effect Profile: Toxicity concerns hamper widespread adoption, especially over newer agents with better tolerability.

• Generics and Price Competition: Multiple generic formulations curb pricing power and profit margins.

• Regulatory and Reimbursement Challenges: Inconsistent approval status across regions affects market penetration.

Regional Market Outlook

• North America: Limited expansion; focus on niche applications.

• Europe & Asia: Steady demand in HSCT conditioning; potential growth in combination therapies and innovative delivery systems.

• Emerging Markets: Growing adoption due to cost-effectiveness of generics.

Market Projection and Future Outlook

Forecasting the Trajectory for Thiotepa

Based on current clinical development trends and market conditions, the future of thiotepa can be summarized as follows:

• Short-term (1-3 years): Stable demand driven by its established role in HSCT and niche indications. Clinical trials exploring combinatorial regimens may modestly enhance its profile among oncologists.

• Medium-term (3-5 years): Expansion into innovative delivery systems (e.g., liposomes, nanoparticles) could improve safety and efficacy, leading to potential label extensions and increased use.

• Long-term (5+ years): Its market size may stabilize or decline unless substantial breakthroughs emerge. Alternatively, niche applications might sustain a steady market, especially if integrated into personalized or targeted regimens.

Potential Market Growth Catalysts

• Regulatory incentives (e.g., orphan drug status) could encourage further development.

• Successful trial results demonstrating synergistic effects with immunotherapies.

• Regional expansion in emerging markets, driven by cost competitiveness.

Market Risks

• The dominance of newer targeted agents and immunotherapies.

• Toxicity concerns leading to decreased prescribing.

• Competition from innovative chemotherapeutic agents or biologics.

Key Takeaways

• Current clinical trials are exploring thiotepa’s role in combination therapies and novel delivery methods, signaling a potential rejuvenation in its therapeutic profile.

• Market demand remains concentrated within niche applications such as HSCT conditioning and rare tumor indications, with limited growth prospects in broad Oncology segments.

• Future projections suggest a stabilized or modestly growing market driven by pipeline advancements, regional adoption, and regulatory incentives, but face significant hurdles from newer therapies.

• Strategic opportunities include developing safer formulations, expanding into synergistic treatment regimens, and targeting underserved markets.

FAQs

1. What are the primary indications for thiotepa today?
   Currently, thiotepa is mostly used in conditioning regimens prior to hematopoietic stem cell transplantation and for certain rare cancers such as ovarian and bladder carcinomas.

2. Are there any new formulations of thiotepa in clinical development?
   Yes, liposomal formulations and nanoparticle-based delivery systems are under preclinical and early clinical investigation to enhance safety and efficacy.

3. How does thiotepa compare to newer cancer therapies?
   Its toxicity profile and the rise of targeted therapies and immunotherapies have limited additional indications, keeping its use mainly confined to specific niche applications.

4. What is the outlook for thiotepa in emerging markets?
   Growing healthcare infrastructure and cost considerations make emerging markets likely to sustain consistent demand, particularly for generic formulations.

5. Will thiotepa remain relevant in future oncology treatment protocols?
   Its role is expected to persist in niche indications, especially if future trials demonstrate improved safety and effectiveness through novel formulations or combination regimens.

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Sources:

[1] Grand View Research. "Cancer Drugs Market Size, Share & Trends Analysis Report." 2022.