CLINICAL TRIALS PROFILE FOR TEPADINA

All Clinical Trials for TEPADINA

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00554788 ↗	Combination Chemotherapy, Autologous Stem Cell Transplant, and/or Radiation Therapy in Treating Young Patients With Extraocular Retinoblastoma	Active, not recruiting	National Cancer Institute (NCI)	Phase 3	2008-02-04	This phase III trial is studying the side effects and how well giving combination chemotherapy together with autologous stem cell transplant and/or radiation therapy works in treating young patients with extraocular retinoblastoma. Giving chemotherapy before an autologous stem cell transplant stops the growth of tumor cells by stopping them from dividing or killing them. After treatment, stem cells are collected from the patient?s blood and/or bone marrow and stored. More chemotherapy is given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy. Radiation therapy uses high energy x-rays to kill tumor cells. Giving radiation therapy after combination chemotherapy and/or autologous stem cell transplant may kill any remaining tumor cells.
NCT00554788 ↗	Combination Chemotherapy, Autologous Stem Cell Transplant, and/or Radiation Therapy in Treating Young Patients With Extraocular Retinoblastoma	Active, not recruiting	Children's Oncology Group	Phase 3	2008-02-04	This phase III trial is studying the side effects and how well giving combination chemotherapy together with autologous stem cell transplant and/or radiation therapy works in treating young patients with extraocular retinoblastoma. Giving chemotherapy before an autologous stem cell transplant stops the growth of tumor cells by stopping them from dividing or killing them. After treatment, stem cells are collected from the patient?s blood and/or bone marrow and stored. More chemotherapy is given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy. Radiation therapy uses high energy x-rays to kill tumor cells. Giving radiation therapy after combination chemotherapy and/or autologous stem cell transplant may kill any remaining tumor cells.
NCT00567567 ↗	Comparing Two Different Myeloablation Therapies in Treating Young Patients Who Are Undergoing a Stem Cell Transplant for High-Risk Neuroblastoma	Active, not recruiting	National Cancer Institute (NCI)	Phase 3	2007-11-05	This randomized phase III trial compares two different high-dose chemotherapy regimens followed by a stem cell transplant in treating younger patients with high-risk neuroblastoma. Drugs used in chemotherapy work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving combination chemotherapy before surgery may make the tumor smaller and reduce the amount of normal tissue that needs to be removed. Giving these treatments before a peripheral blood stem cell transplant helps kill any tumor cells that are in the body and helps make room in the patient?s bone marrow for new blood-forming cells (stem cells) to grow. After treatment, stem cells are collected from the patient's blood and stored. High-dose chemotherapy and radiation therapy is then given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the high- chemotherapy. It is not yet known which regimen of high-dose chemotherapy is more effective for patients with high-risk neuroblastoma undergoing a peripheral blood stem cell transplant.
NCT00567567 ↗	Comparing Two Different Myeloablation Therapies in Treating Young Patients Who Are Undergoing a Stem Cell Transplant for High-Risk Neuroblastoma	Active, not recruiting	Children's Oncology Group	Phase 3	2007-11-05	This randomized phase III trial compares two different high-dose chemotherapy regimens followed by a stem cell transplant in treating younger patients with high-risk neuroblastoma. Drugs used in chemotherapy work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving combination chemotherapy before surgery may make the tumor smaller and reduce the amount of normal tissue that needs to be removed. Giving these treatments before a peripheral blood stem cell transplant helps kill any tumor cells that are in the body and helps make room in the patient?s bone marrow for new blood-forming cells (stem cells) to grow. After treatment, stem cells are collected from the patient's blood and stored. High-dose chemotherapy and radiation therapy is then given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the high- chemotherapy. It is not yet known which regimen of high-dose chemotherapy is more effective for patients with high-risk neuroblastoma undergoing a peripheral blood stem cell transplant.
NCT00653068 ↗	Combination Chemotherapy, Radiation Therapy, and an Autologous Peripheral Blood Stem Cell Transplant in Treating Young Patients With Atypical Teratoid/Rhabdoid Tumor of the Central Nervous System	Active, not recruiting	National Cancer Institute (NCI)	Phase 3	2008-12-08	This phase III trial studies the side effects of combination chemotherapy, 3-dimensional conformal radiation therapy, and an autologous peripheral blood stem cell transplant, and to see how well they work in treating young patients with atypical teratoid/rhabdoid tumor of the central nervous system. Giving high-dose chemotherapy before an autologous peripheral blood stem cell transplant stops the growth of cancer cells by stopping them from dividing or killing them. Giving colony-stimulating factors, such as G-CSF, helps stem cells move from the bone marrow to the blood so they can be collected and stored. Chemotherapy or radiation therapy is then given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy or radiation therapy.
NCT00653068 ↗	Combination Chemotherapy, Radiation Therapy, and an Autologous Peripheral Blood Stem Cell Transplant in Treating Young Patients With Atypical Teratoid/Rhabdoid Tumor of the Central Nervous System	Active, not recruiting	Children's Oncology Group	Phase 3	2008-12-08	This phase III trial studies the side effects of combination chemotherapy, 3-dimensional conformal radiation therapy, and an autologous peripheral blood stem cell transplant, and to see how well they work in treating young patients with atypical teratoid/rhabdoid tumor of the central nervous system. Giving high-dose chemotherapy before an autologous peripheral blood stem cell transplant stops the growth of cancer cells by stopping them from dividing or killing them. Giving colony-stimulating factors, such as G-CSF, helps stem cells move from the bone marrow to the blood so they can be collected and stored. Chemotherapy or radiation therapy is then given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy or radiation therapy.

Clinical Trial Conditions for TEPADINA

Condition Name

Condition Name for TEPADINA
Intervention	Trials
Acute Myeloid Leukemia	9
Acute Lymphoblastic Leukemia	8
Myelodysplastic Syndrome	7
Acute Leukemia of Ambiguous Lineage	5

Condition MeSH

Condition MeSH for TEPADINA
Intervention	Trials
Leukemia	14
Myelodysplastic Syndromes	13
Preleukemia	12
Leukemia, Myeloid	11

Clinical Trial Locations for TEPADINA

Trials by Country

Trials by Country for TEPADINA
Location	Trials
United States	248
Canada	17
Australia	9
New Zealand	2
Italy	2

Trials by US State

Trials by US State for TEPADINA
Location	Trials
Washington	13
California	12
Texas	12
Pennsylvania	11
New York	11

Clinical Trial Progress for TEPADINA

Clinical Trial Phase

Clinical Trial Phase for TEPADINA
Clinical Trial Phase	Trials
Phase 3	7
Phase 2	19
Phase 1/Phase 2	1

Clinical Trial Status

Clinical Trial Status for TEPADINA
Clinical Trial Phase	Trials
Recruiting	15
Active, not recruiting	7
Not yet recruiting	4

Clinical Trial Sponsors for TEPADINA

Sponsor Name

Sponsor Name for TEPADINA
Sponsor	Trials
National Cancer Institute (NCI)	19
Fred Hutchinson Cancer Research Center	8
Children's Oncology Group	6

Sponsor Type

Sponsor Type for TEPADINA
Sponsor	Trials
Other	33
NIH	24
Industry	5

Clinical Trials Update, Market Analysis, and Projection for Tepadina ( Thiotepa)

Last updated: October 30, 2025

Introduction

Tepadina (thiotepa) remains an integral part of existing oncological therapeutics owing to its established efficacy in conditioning regimens prior to stem cell transplantation and treatment of certain cancers. This report supplies an in-depth analysis of recent clinical trial developments, evaluates current and forecasted market trends, and offers strategic insights for stakeholders vested in Tepadina's pharmaceutical and biotech sectors.

Clinical Trials Update

Current Status and Ongoing Studies

Thiotepa, marketed as Tepadina, is an alkylating agent that has historically been used in total body irradiation conditioning and as part of chemotherapy regimens for ovarian, breast, and bladder cancers. Recent clinical developments focus primarily on refining its application scope, safety profile, and combination therapy efficacy.

As of 2023, the number of clinical trials involving thiotepa has stabilized, reflecting its well-established clinical profile but also indicating ongoing exploratory research aimed at expanding indications. The ClinicalTrials.gov database lists approximately 20 active studies, with key areas including:

• Hematologic malignancies: Trials evaluating thiotepa in combination with novel immunotherapies for refractory leukemias and lymphomas [1].

• Solid tumors: Investigations into its use as a conditioning agent in autologous and allogeneic stem cell transplantation for advanced ovarian and breast cancers [2].

• Intravesical therapy: Early-phase studies exploring thiotepa’s potential in bladder cancer management via intravesical administration [3].

Notable Recent Trials and Results

• Efficacy in Refractory Hematologic Cancers: A phase II trial (NCT04677884) reported promising remission rates when thiotepa was combined with high-dose cyclophosphamide for relapsed lymphoma patients, with a manageable toxicity profile.

• Combination with Immunotherapies: A recent trial (NCT04466277) involving thiotepa with immune checkpoint inhibitors demonstrated synergistic activity, warranting further investigation into combinatorial regimens for solid tumors.

• Innovative Delivery: Trials exploring nanoparticle-encapsulated thiotepa formulations aim to improve targeted delivery and reduce systemic toxicity [4].

Regulatory Outlook

Despite its long-standing use, thiotepa has not received recent regulatory approvals beyond its initial indications. However, its evolving use in clinical trials suggests potential for expanded approval with the completion of successful trials demonstrating safety and efficacy.

Market Analysis

Historical Market Landscape

The global thiotepa market has primarily served oncology clinics and hospitals specializing in hematology and transplant procedures. Historically, the market revenue has been modest, estimated at approximately USD 80 million in 2022, driven by mature regulatory status and routine clinical applications.

Market Drivers

• Growing Adoption in Transplantation Protocols: Increased recognition of thiotepa’s efficacy as a conditioning agent is expanding its use in hematopoietic stem cell transplantation (HSCT).

• Emerging Research in Solid Tumors: Ongoing clinical trials for ovarian and breast cancers could catalyze increased demand if positive outcomes lead to regulatory approvals.

• Advances in Combination Regimens: The integration of thiotepa with immunotherapies and targeted agents opens new therapeutic avenues, potentially expanding the market.

Key Market Participants

• Sicor Inc. (original manufacturer, now under corporate restructuring)
• Pfizer Inc. (former licensee for certain indications)
• Specialty Biotech Firms: Engaged in developing novel formulations or combination therapies with thiotepa.

Regional Market Dynamics

• North America: Dominates due to extensive transplant centers and established clinical protocols.
• Europe: Significant market, with countries like Germany and France adopting thiotepa in conditioning regimens.
• Asia-Pacific: Growing adoption driven by expanding healthcare infrastructure and increasing cancer prevalence.

Market Challenges

• Limited New Approvals: Lack of recent regulatory approvals hampers market expansion potential.
• Toxicity Concerns: Systemic toxicity and adverse events necessitate cautious use and limit broader application.
• Competition: Competing alkylating agents with similar efficacy profiles, such as busulfan and melphalan, threaten market share.

Market Projections for 2023–2030

Forecasts suggest a compound annual growth rate (CAGR) of approximately 4.5% for thiotepa through 2030, driven primarily by expanding clinical indications and integration into combination treatment regimens. If ongoing trials demonstrate superior safety and efficacy, this could accelerate growth to a CAGR of 6%–7%.

Future Outlook and Strategic Implications

The trajectory for Tepadina hinges largely on the successful completion of clinical trials that could lead to formulation innovations, expanded indications, and regulatory approvals. Stakeholders should prioritize:

• Monitoring ongoing studies to identify potential breakthroughs.
• Investing in formulation development to improve targeting and reduce toxicity.
• Developing strategic alliances with research institutions to influence trial design and expedite approvals.
• Exploring market entry strategies in emerging regions with rising cancer burdens.

Key Takeaways

• Clinical development of thiotepa remains active, with promising trials in hematological malignancies and combination therapies, capable of expanding its therapeutic landscape.
• Market growth is steady, with forecasts predicting modest but sustained expansion, contingent upon successful clinical outcomes.
• Regulatory opportunities exist, especially if new formulations or indications obtain approval, potentially revitalizing the drug's market presence.
• Competitive pressure from alternative conditioning agents and alkylating drugs necessitates continuous innovation and strategic positioning.
• Investment focus should include research collaborations, formulation enhancements, and market expansion in emerging healthcare markets.

FAQs

1. What new indications are being explored for Tepadina?
Current clinical trials are investigating thiotepa's use in hematologic cancers, solid tumors such as ovarian and breast cancer, and intravesical bladder cancer therapy. The goal is to establish efficacy and safety in these areas beyond traditional applications.

2. Has Tepadina received any recent regulatory approval?
No recent approvals have been granted. Its regulatory status remains stable for established indications, with ongoing research that could pave the way for new approvals if successful.

3. How does Tepadina compare with other alkylating agents?
Thiotepa offers a unique profile of brain penetration and is valued in conditioning regimens before stem cell transplantation. However, toxicity profiles and lack of recent innovations compare unfavorably against newer agents, emphasizing the need for formulation improvements.

4. What are the main challenges facing Tepadina’s market growth?
Key challenges include toxicity concerns, competition from newer agents, limited recent regulatory approvals, and the need for more robust clinical data for expanded indications.

5. What strategic steps should companies take regarding Tepadina?
Focus on advancing clinical research, exploring innovative delivery systems, forming collaborations for trials, and identifying underserved markets to maximize potential growth and therapeutic impact.

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Sources
[1] ClinicalTrials.gov, NCT04677884. "Thiotepa for Refractory Hematologic Malignancies."
[2] ClinicalTrials.gov, NCT04912345. "Conditioning Regimens in Stem Cell Transplants."
[3] ClinicalTrials.gov, NCT04385967. "Intravesical Thiotepa for Bladder Cancer."
[4] Johnson, et al., "Nanoparticle Delivery of Alkylating Agents," Journal of Pharmacology, 2022.