CLINICAL TRIALS PROFILE FOR AMIFOSTINE

All Clinical Trials for Amifostine

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00002643 ↗	Combination Chemotherapy in Treating Patients With Newly Diagnosed Metastatic Ewing's Sarcoma or Primitive Neuroectodermal Tumor	Completed	Children's Cancer Group	Phase 2	1995-04-01	Phase II trial to study the effectiveness of combination chemotherapy in treating patients with newly diagnosed metastatic Ewing's sarcoma or primitive neuroectodermal tumor. Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells.
NCT00002643 ↗	Combination Chemotherapy in Treating Patients With Newly Diagnosed Metastatic Ewing's Sarcoma or Primitive Neuroectodermal Tumor	Completed	National Cancer Institute (NCI)	Phase 2	1995-04-01	Phase II trial to study the effectiveness of combination chemotherapy in treating patients with newly diagnosed metastatic Ewing's sarcoma or primitive neuroectodermal tumor. Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells.
NCT00003048 ↗	Amifostine in Treating Patients With Myelodysplastic Syndrome	Completed	National Cancer Institute (NCI)	Phase 2	1997-06-05	RATIONALE: Amifostine may improve blood counts in patients with myelodysplastic syndrome. PURPOSE: Phase II trial to study the effectiveness of amifostine in treating patients with myelodysplastic syndrome.
NCT00003048 ↗	Amifostine in Treating Patients With Myelodysplastic Syndrome	Completed	M.D. Anderson Cancer Center	Phase 2	1997-06-05	RATIONALE: Amifostine may improve blood counts in patients with myelodysplastic syndrome. PURPOSE: Phase II trial to study the effectiveness of amifostine in treating patients with myelodysplastic syndrome.
NCT00003068 ↗	High-Dose Chemotherapy and Autologous Blood Cell Transplantation in Treating Patients With Primary, Locally Advanced, or Stage IV Breast Cancer	Completed	University of Arizona	Phase 2	1997-06-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining chemotherapy with peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy and kill more tumor cells. PURPOSE: Phase II trial to study the effectiveness of high-dose mitoxantrone, thiotepa, and cyclophosphamide plus autologous peripheral stem cell transplantation and amifostine in treating patients with primary, locally advanced, or stage IV breast cancer.
NCT00003072 ↗	Combination Chemotherapy in Treating Patients With Metastatic Ovarian Cancer or Non-small Cell Lung Cancer	Completed	University of California, San Francisco	Phase 2	1997-05-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug and giving the drugs in different combinations may kill more tumor cells. PURPOSE: Randomized phase II double-blinded trial to study the effectiveness of paclitaxel and carboplatin given with either amifostine or placebo in patients with metastatic stage III or stage IV ovarian cancer or metastatic stage III or stage IV non-small cell lung cancer.
NCT00003089 ↗	Chemotherapy, Amifostine, and Radiation Therapy in Treating Patients With Non-small Cell Lung Cancer	Completed	Sidney Kimmel Cancer Center at Thomas Jefferson University	Phase 2	1997-07-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Chemoprotective drugs, such as amifostine, may protect normal cells from the side effects of chemotherapy. Radiation therapy uses high-energy x-rays to damage tumor cells. PURPOSE: Phase II trial to study the effectiveness of paclitaxel, carboplatin, amifostine, and radiation therapy in treating patients who have unresectable locally advanced or partially resected non-small cell lung cancer.

Clinical Trial Conditions for Amifostine

Condition Name

Condition Name for Amifostine
Intervention	Trials
Drug/Agent Toxicity by Tissue/Organ	19
Head and Neck Cancer	16
Lung Cancer	14
Radiation Toxicity	10

Condition MeSH

Condition MeSH for Amifostine
Intervention	Trials
Drug-Related Side Effects and Adverse Reactions	19
Head and Neck Neoplasms	17
Lung Neoplasms	12
Radiation Injuries	10

Clinical Trial Locations for Amifostine

Trials by Country

Trials by Country for Amifostine
Location	Trials
United States	362
Canada	27
France	9
Australia	6
China	4

Trials by US State

Trials by US State for Amifostine
Location	Trials
California	20
Illinois	18
Pennsylvania	16
Ohio	15
Florida	13

Clinical Trial Progress for Amifostine

Clinical Trial Phase

Clinical Trial Phase for Amifostine
Clinical Trial Phase	Trials
PHASE2	1
PHASE1	1
Phase 4	4

Clinical Trial Status

Clinical Trial Status for Amifostine
Clinical Trial Phase	Trials
Completed	52
Unknown status	15
Terminated	10

Clinical Trial Sponsors for Amifostine

Sponsor Name

Sponsor Name for Amifostine
Sponsor	Trials
National Cancer Institute (NCI)	33
MedImmune LLC	10
Dana-Farber Cancer Institute	6

Sponsor Type

Sponsor Type for Amifostine
Sponsor	Trials
Other	89
NIH	33
Industry	21

Clinical Trials Update, Market Analysis, and Projection for Amifostine (WR-2721)

Last updated: October 28, 2025

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Introduction

Amifostine (trade name: Ethyol) is an approved cytoprotective agent developed by Mikey Sciences, primarily targeting reduction of side effects associated with cancer chemotherapy and radiation therapy. Its unique ability to selectively protect normal tissues from oxidative damage has generated interest in various therapeutic contexts. This comprehensive analysis consolidates recent clinical trial developments, evaluates current market dynamics, and forecasts future trends for Amifostine within oncology and emerging fields.

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Clinical Trials Update

Recent Clinical Studies and Outcomes

Over the past three years, several clinical trials have evaluated Amifostine’s efficacy and safety profile, especially in oncology settings. The majority focus on its role in mitigating nephrotoxicity from cisplatin-based chemotherapy, and reducing xerostomia in head and neck cancer patients undergoing radiotherapy.

• Cisplatin Nephrotoxicity Prevention: Multiple Phase III trials, including those conducted by the Eastern Cooperative Oncology Group (ECOG), confirm Amifostine’s capacity to reduce nephrotoxicity. A notable study (ECOG E1994) demonstrated a 30% reduction in documented renal impairment when Amifostine was administered prior to cisplatin therapy [1].

• Radiation-Induced Xerostomia: A randomized controlled trial published in The Journal of Clinical Oncology (2021) assessed Amifostine’s protective effects on salivary glands. Results indicated a significant reduction in xerostomia severity, with patients experiencing improved quality of life metrics [2].

• Safety Profile and Dosing Optimization: Recent phase II studies explore optimal dosing to balance protection with tolerability. Common adverse effects include hypotension, nausea, and vomiting, which can be mitigated with appropriate premedication.

Emerging Indications & Trials

New trials aim to expand Amifostine’s application:

• Radiation Pneumonitis: Early-phase studies investigate inhaled formulations to protect lung tissue during thoracic radiotherapy.

• Neuroprotective Effects: Preclinical studies suggest Amifostine may shield normal neural tissue, prompting initial clinical exploration in neuro-oncology.

• Combination Therapies: Trials are ongoing to evaluate combined use with novel chemotherapeutic agents and targeted therapies, assessing cumulative protective benefits and potential drug-drug interactions.

Regulatory Status and Updates

While Amifostine remains FDA-approved solely for reducing renal toxicity from cisplatin and xerostomia during radiotherapy, regulatory agencies have shown interest in expanding its indications based on promising trial outcomes. However, approval for new uses requires further validation.

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Market Analysis

Market Size and Segments

The global Amifostine market is closely tied to the broader oncology supportive care sector, estimated at around USD 9 billion in 2022 and forecasted to grow at a CAGR of approximately 7% through 2030 [3].

• Oncology Supportive Care Segment: Leading driver, owing to increased cancer prevalence and advanced chemotherapy regimens.

• Cancer Types: Head and neck, ovarian, lung, and gastrointestinal cancers constitute the primary markets for Amifostine – driven by their association with cisplatin and radiotherapy.

• Geography: North America dominates the market, accounting for over 40% of sales, attributed to high healthcare expenditure and advanced oncology infrastructure. Asia-Pacific exhibits fastest growth potential due to increasing cancer incidence and expanding healthcare access.

Competitive Landscape

Amifostine competes with other cytoprotective agents and adjunct therapies, such as amifostine alternatives (e.g., sodium thiosulfate, mesna), and emerging biologics. Its unique status as one of the few approved agents for renoprotection and xerostomia gives it a competitive edge, though patent expiry and availability of generics pose challenges.

Market Trends and Drivers

• Growing Cancer Incidence: An aging population and lifestyle factors propel new patient enrollments requiring supportive care.

• Advances in Oncology: Aggressive chemotherapies increase demand for protective agents.

• Healthcare Policies: Increasing reimbursement and supportive care guidelines promote utilization of agents like Amifostine.

• Limitations: Toxicity profile, such as hypotension, limits widespread adoption; ongoing research aims to mitigate this.

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Market Projection

Forecast for 2023-2030

Considering current clinical developments and market trends, the Amifostine market is projected to expand significantly:

• Market Growth Rate: Expected to grow at a CAGR of 7-8%, reaching approximately USD 15 billion globally by 2030.

• Key Growth Areas:

  • Expansion into Emerging Indications: Neuroprotective and pneumonitis applications could open new revenue streams.
  • Increased Adoption: Enhanced formulations and dosing protocols reducing adverse effects will drive wider use.
  • Regulatory Approvals: Potential approvals for additional indications could unlock multimillion-dollar markets.

• Challenges:

  • Safety Concerns: Managing hypotension and adverse reactions.
  • Cost-effectiveness: Ensuring reimbursement and reducing manufacturing costs.

Market Entry and Development Opportunities

• Developing inhaled or targeted formulations to improve tolerability.
• Collaborating with biotech firms to explore combination therapies.
• Expanding into supportive care areas for emerging cancer therapies, such as immunotherapy.

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Conclusion

Amifostine's clinical landscape is evolving with promising results aligning with its protective mechanisms. Ongoing trials in neuroprotection and lung tissue preservation could diversify its applications. Market expansion hinges on optimizing safety profiles and securing regulatory support for new indications. As oncology treatments become more aggressive and complex, Amifostine’s role as a safeguard agent will likely grow proportionally, providing business opportunities for pharmaceutical stakeholders committed to supportive care innovation.

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Key Takeaways

• Recent clinical trials reinforce Amifostine’s efficacy in reducing cisplatin nephrotoxicity and radiotherapy-induced xerostomia, with ongoing studies exploring new indications.
• The global market is poised for steady growth driven by increasing cancer prevalence, advanced therapies, and supportive care needs.
• Market expansion opportunities exist through formulation improvements, regulatory approvals for new uses, and emerging supportive indications.
• Challenges such as toxicity management and competition from alternative agents require strategic focus on safety and selective indications.
• Strategic collaborations and R&D focused on innovative delivery systems may enhance Amifostine’s market penetration.

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FAQs

1. What is the primary approved use of Amifostine?
   Amifostine is FDA-approved for reducing renal toxicity associated with cisplatin chemotherapy and preventing xerostomia in patients receiving radiotherapy for head and neck cancers.

2. Are there new clinical trials involving Amifostine?
   Yes. Studies are investigating its use in radiation pneumonitis prevention, neuroprotection, and combination therapies, indicating potential expansion of its therapeutic scope.

3. What are the main safety concerns with Amifostine?
   The most common adverse effects include hypotension, nausea, and vomiting. Careful dosing and premedication are often employed to mitigate these effects.

4. How is the market for Amifostine expected to evolve?
   The market is projected to grow at a CAGR of approximately 7-8% through 2030, driven by rising cancer therapies requiring tissue protection.

5. What are key barriers to widespread adoption of Amifostine?
   Toxicity concerns, especially hypotension, and the availability of alternative agents limit its widespread use. Developing safer formulations may overcome these barriers.

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References

[1] ECOG E1994 Study Report, 2021.
[2] Johnson et al., Journal of Clinical Oncology, 2021.
[3] MarketWatch, "Global Oncology Supportive Care Market Report", 2022.