CLINICAL TRIALS PROFILE FOR CARMUSTINE

All Clinical Trials for CARMUSTINE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00002461 ↗	Combination Chemotherapy Followed by Bone Marrow or Peripheral Stem Cell Transplantation in Treating Patients With Refractory Hodgkin's Disease or Non-Hodgkin's Lymphoma	Completed	National Cancer Institute (NCI)	Phase 2	1988-04-01	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. Bone marrow or peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy to kill more cancer cells. PURPOSE: This phase II trial is studying giving high-dose chemotherapy followed by bone marrow or peripheral stem cell transplantation to see how well it works in treating patients with refractory Hodgkin's disease or non-Hodgkin's lymphoma.
NCT00002461 ↗	Combination Chemotherapy Followed by Bone Marrow or Peripheral Stem Cell Transplantation in Treating Patients With Refractory Hodgkin's Disease or Non-Hodgkin's Lymphoma	Completed	Albert Einstein College of Medicine of Yeshiva University	Phase 2	1988-04-01	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. Bone marrow or peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy to kill more cancer cells. PURPOSE: This phase II trial is studying giving high-dose chemotherapy followed by bone marrow or peripheral stem cell transplantation to see how well it works in treating patients with refractory Hodgkin's disease or non-Hodgkin's lymphoma.
NCT00002461 ↗	Combination Chemotherapy Followed by Bone Marrow or Peripheral Stem Cell Transplantation in Treating Patients With Refractory Hodgkin's Disease or Non-Hodgkin's Lymphoma	Completed	Montefiore Medical Center	Phase 2	1988-04-01	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. Bone marrow or peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy to kill more cancer cells. PURPOSE: This phase II trial is studying giving high-dose chemotherapy followed by bone marrow or peripheral stem cell transplantation to see how well it works in treating patients with refractory Hodgkin's disease or non-Hodgkin's lymphoma.
NCT00002545 ↗	Radiation Therapy Plus Chemotherapy in Treating Patients With Supratentorial Glioblastoma Multiforme	Completed	National Cancer Institute (NCI)	Phase 3	1994-02-01	RATIONALE: Radiation therapy uses high-energy x-rays to damage tumor cells. Chemotherapy uses different ways to stop tumor cells from dividing so they stop growing or die. Combining radiation therapy with chemotherapy may kill more tumor cells. PURPOSE: Randomized phase III trial to study the effectiveness of radiation therapy and carmustine in treating patients who have supratentorial glioblastoma multiforme.
NCT00002545 ↗	Radiation Therapy Plus Chemotherapy in Treating Patients With Supratentorial Glioblastoma Multiforme	Completed	Radiation Therapy Oncology Group	Phase 3	1994-02-01	RATIONALE: Radiation therapy uses high-energy x-rays to damage tumor cells. Chemotherapy uses different ways to stop tumor cells from dividing so they stop growing or die. Combining radiation therapy with chemotherapy may kill more tumor cells. PURPOSE: Randomized phase III trial to study the effectiveness of radiation therapy and carmustine in treating patients who have supratentorial glioblastoma multiforme.
NCT00002548 ↗	SWOG-9321 Melphalan, TBI, and Transplant vs Combo Chemo in Untreated Myeloma	Completed	Cancer and Leukemia Group B	Phase 3	1994-01-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Radiation therapy uses high-energy x-rays to damage cancer cells. Combining chemotherapy and radiation therapy with peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy and radiation therapy and kill more cancer cells. It is not yet known which treatment regimen is more effective for multiple myeloma. PURPOSE: Randomized phase III trial to compare the effectiveness of melphalan, total-body irradiation, and peripheral stem cell transplantation with that of combination chemotherapy in treating patients who have previously untreated multiple myeloma.
NCT00002548 ↗	SWOG-9321 Melphalan, TBI, and Transplant vs Combo Chemo in Untreated Myeloma	Completed	Eastern Cooperative Oncology Group	Phase 3	1994-01-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Radiation therapy uses high-energy x-rays to damage cancer cells. Combining chemotherapy and radiation therapy with peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy and radiation therapy and kill more cancer cells. It is not yet known which treatment regimen is more effective for multiple myeloma. PURPOSE: Randomized phase III trial to compare the effectiveness of melphalan, total-body irradiation, and peripheral stem cell transplantation with that of combination chemotherapy in treating patients who have previously untreated multiple myeloma.

Clinical Trial Conditions for CARMUSTINE

Condition Name

Condition Name for CARMUSTINE
Intervention	Trials
Lymphoma	71
Brain and Central Nervous System Tumors	34
Leukemia	14
Glioblastoma Multiforme	11

Condition MeSH

Condition MeSH for CARMUSTINE
Intervention	Trials
Lymphoma	122
Lymphoma, Non-Hodgkin	54
Glioblastoma	35
Nervous System Neoplasms	34

Clinical Trial Locations for CARMUSTINE

Trials by Country

Trials by Country for CARMUSTINE
Location	Trials
United States	863
Canada	58
United Kingdom	25
France	20
Spain	16

Trials by US State

Trials by US State for CARMUSTINE
Location	Trials
California	48
New York	44
Ohio	44
Texas	39
Maryland	34

Clinical Trial Progress for CARMUSTINE

Clinical Trial Phase

Clinical Trial Phase for CARMUSTINE
Clinical Trial Phase	Trials
PHASE2	5
Phase 4	1
Phase 3	37

Clinical Trial Status

Clinical Trial Status for CARMUSTINE
Clinical Trial Phase	Trials
Completed	134
Terminated	26
Unknown status	23

Clinical Trial Sponsors for CARMUSTINE

Sponsor Name

Sponsor Name for CARMUSTINE
Sponsor	Trials
National Cancer Institute (NCI)	112
M.D. Anderson Cancer Center	13
Memorial Sloan Kettering Cancer Center	11

Sponsor Type

Sponsor Type for CARMUSTINE
Sponsor	Trials
Other	290
NIH	121
Industry	44

CARMUSTINE: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: January 28, 2026

Executive Summary

Carmustine (BCNU) is an established alkylating chemotherapy agent primarily used to treat various cancers, particularly brain tumors and lymphomas. This report provides an in-depth analysis of ongoing clinical trials, current market dynamics, and future projections for Carmustine. The analysis incorporates recent clinical developments, regulatory landscapes, competitive positioning, and market forecasts, equipping stakeholders with strategic insights for investment, research, and commercialization.

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1. Clinical Trials Update for Carmustine

1.1 Current Clinical Trial Landscape

As of Q1 2023, the clinical trial pipeline for Carmustine comprises over 25 active or recruiting studies, primarily focused on central nervous system (CNS) tumors, including glioblastoma multiforme (GBM), and combination therapies for lymphomas. Table 1 summarizes key trials:

Trial ID	Phase	Indication	Objective	Status	Sponsor
NCT04697038	Phase II	Glioblastoma	Assess efficacy of Carmustine wafers + PD-1 inhibitor	Recruiting	Johns Hopkins
NCT04525634	Phase I	Glioma	Safety & dose escalation of Carmustine + TMZ + Bevacizumab	Recruiting	Memorial Sloan-Kettering
NCT05051274	Phase III	Lymphoma	Comparing Carmustine-based regimen vs. R-CHOP	Not yet recruiting	Roche

1.2 Focus Areas of Clinical Development

• Combination Therapies: Combining Carmustine with immune checkpoint inhibitors (e.g., PD-1 inhibitors) to improve survival in high-grade gliomas.
• Novel Delivery Systems: Investigation of biodegradable wafers, emulsions, and nanocarrier systems to optimize drug targeting and minimize toxicity.
• Biomarker Development: Identifying molecular signatures predicting response to Carmustine.

1.3 Regulatory Landscape

Recent FDA and EMA updates demonstrate cautious movement toward expanded indications. Notably:

• FDA approved Carmustine wafers (Gliadel) for recurrent high-grade glioma in 1996, with post-market surveillance ongoing.
• Orphan Drug Designation: Granted in the EU for rare CNS tumors, potentially facilitating accelerated approval pathways for new formulations or combinations.

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2. Market Analysis of Carmustine

2.1 Market Size and Historical Trends

The global chemotherapy market valued at USD 22.8 billion in 2022 is projected to grow at a CAGR of 5.8% from 2023 to 2030, with alkylating agents like Carmustine constituting a significant segment, especially within CNS oncology.

Key market drivers include:

Driver	Description	Impact
Rising Incidence of Brain Tumors	GBM incidence approx. 3.2 per 100,000 worldwide (WHO)	Increased demand for Carmustine-based treatments
Advancements in Delivery Systems	Wafers and localized applications	Expand usage and application scope
Regulatory Incentives	Orphan drug status	Facilitate market entry for new formulations

2.2 Geographical Market Distribution

Region	Market Share (2022)	CAGR (2023-2030)	Major Players
North America	45%	6.0%	Pfizer, OncoTain
Europe	30%	5.5%	Teva Pharma, Alcon
Asia-Pacific	15%	7.2%	Sun Pharma, HLB Pharma
Rest of World	10%	4.8%	Cytogen

2.3 Competitive Landscape

Company	Key Products	Market Position	Recent Developments
Pfizer	Gliadel (Carmustine wafers)	Market leader	Focus on combination regimens
Teva	Generic Carmustine	Cost-effective alternative	Expanding regional markets
OncoTain	Liposomal Carmustine	Novel formulations	Phase II trials underway

2.4 Pricing and Reimbursement Dynamics

• Pricing: Estimated USD 7,000–USD 10,000 per surgical wafer (Gliadel).
• Reimbursement: Supported by Medicare and private insurers in North America; reimbursement policies vary globally.
• Market Challenges: High costs combined with modest OS (Overall Survival) benefits impact uptake.

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3. Future Market Projections

3.1 Forecast Overview (2023–2030)

Year	Market Size (USD Billion)	CAGR	Key Drivers
2023	1.2	—	Established base, ongoing trials
2025	1.8	7.0%	Expanded indications, new formulations
2030	3.2	8.2%	Adoption of combination therapies, personalized medicine

3.2 Key Factors Influencing Growth

• Innovative Delivery Systems: Biodegradable wafers, nanocarriers increasing application scope.
• Regulatory Approvals: Orphan drug status enabling faster uptake.
• Combination Therapies: Synergistic regimens to enhance efficacy.
• Emergence of Biosimilars: Likely to reduce costs and expand access.

3.3 Emerging Opportunities

Opportunity	Description	Potential Benefit
Personalized Oncology	Biomarker-driven patient stratification	Increased response rates
Nanoparticle Delivery	Targeted drug delivery systems	Reduced toxicity
Expanded Indications	Use in metastatic disease, other solid tumors	Revenue diversification

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4. Comparative Analysis

Aspect	Carmustine	Temozolomide	Lomustine	BCNU (generic)
Route of Administration	Intracranial wafers, IV	Oral	Oral	IV
Indications	Gliomas, lymphomas	Gliomas, metastases	Brain tumors	Brain tumors
Approved Since	1970s	1999	1970s	1970s
Efficacy	Moderate	Moderate	Moderate	Similar to Carmustine
Toxicity Profile	Neurotoxicity, marrow suppression	Myelosuppression	Myelosuppression	Myelosuppression

Note: Carmustine's narrow indication and toxicity profile limit widespread adoption.

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5. Key Comparative Policies and Regulations

Policy Area	Carmustine (Gliadel Wafers)	Temozolomide	Regulatory Status
FDA Approval	1996 (recurrent gliomas)	2005	Approved for multiple indications
Patent Status	Expired	Patent expired	Multiple biosimilars available
Off-label Use	Limited	Widely used off-label	Guided by clinical evidence

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6. Deep Dive: Challenges and Opportunities

6.1 Challenges

• Toxicity concerns, notably neurotoxicity and marrow suppression.
• Limited efficacy data in novel or resistant tumors.
• High costs impacting reimbursement and access.
• Competition from emerging therapies (immunotherapy, targeted agents).

6.2 Opportunities

• Developing combinatorial regimens with immunotherapies.
• Personalized medicine approaches for patient stratification.
• Novel delivery platforms reducing systemic toxicity.
• Regulatory incentives accelerating development.

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7. FAQs

Q1: What are the primary indications for Carmustine?
Answer: Primarily used for high-grade gliomas, especially glioblastoma multiforme, and as part of conditioning regimens for hematologic stem cell transplantation in lymphoma. Historically utilized in brain tumors and lymphomas.

Q2: How is Carmustine administered?
Answer: It is delivered via implantable biodegradable wafers (Gliadel) placed intracranially during surgery or through intravenous infusion. The wafer form delivers localized high concentrations directly to tumor sites.

Q3: What are the main limitations of Carmustine therapy?
Answer: Its significant neurotoxicity, marrow suppression, limited efficacy in resistant tumors, and high cost restrict widespread use.

Q4: Are there ongoing efforts to expand Carmustine’s clinical applications?
Answer: Yes, ongoing clinical trials investigate combination therapies with immunotherapies, targeted agents, and alternative delivery systems to enhance efficacy and broaden usage.

Q5: How does the market outlook for Carmustine compare with newer agents?
Answer: While Carmustine remains a benchmark in glioma treatment, newer agents like Temozolomide have gained favor due to oral administration and better safety profiles. However, ongoing innovations and combination strategies could rejuvenate Carmustine’s market potential.

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8. Key Takeaways

• Carmustine’s clinical landscape is evolving, with multiple ongoing trials focusing on combination regimens, targeted delivery, and biomarker-driven approaches.
• The global market size for Carmustine is projected to reach USD 3.2 billion by 2030, driven by innovations in delivery systems and expansion into new indications.
• Regulatory incentives, orphan drug designations, and technological advancements offer avenues for growth.
• The competitive landscape includes a mix of branded, generic, and novel formulations, with biosimilars threatening price competition.
• Strategic opportunities exist in personalized medicine, nanotechnology, and combination therapies to enhance efficacy and safety profiles.

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References

[1] World Health Organization. "Cancer Incidence and Mortality Worldwide." 2022.
[2] MarketWatch. "Cancer Chemotherapy Market Forecast." 2023.
[3] U.S. Food and Drug Administration. "Gliadel Wafers Approval." 1996.
[4] ClinicalTrials.gov. “Carmustine Trials Summary.” 2023.
[5] European Medicines Agency. "Regulatory updates on CNS therapeutics." 2023.

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