CLINICAL TRIALS PROFILE FOR MERCAPTOPURINE

All Clinical Trials for MERCAPTOPURINE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00002514 ↗	Stem Cell Transplantation Compared With Standard Chemotherapy in Treating Patients With Acute Lymphoblastic Leukemia in First Remission	Completed	Medical Research Council	Phase 3	1993-04-01	RATIONALE: Drugs used in chemotherapy work in different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with allogeneic or autologous stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells. It is not yet known whether stem cell transplantation is more effective than standard chemotherapy in treating acute lymphoblastic leukemia. PURPOSE: This randomized phase III trial is studying how well stem cell transplantation works compared to standard combination chemotherapy in treating patients with acute lymphoblastic leukemia in first remission.
NCT00002514 ↗	Stem Cell Transplantation Compared With Standard Chemotherapy in Treating Patients With Acute Lymphoblastic Leukemia in First Remission	Completed	National Cancer Institute (NCI)	Phase 3	1993-04-01	RATIONALE: Drugs used in chemotherapy work in different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with allogeneic or autologous stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells. It is not yet known whether stem cell transplantation is more effective than standard chemotherapy in treating acute lymphoblastic leukemia. PURPOSE: This randomized phase III trial is studying how well stem cell transplantation works compared to standard combination chemotherapy in treating patients with acute lymphoblastic leukemia in first remission.
NCT00002514 ↗	Stem Cell Transplantation Compared With Standard Chemotherapy in Treating Patients With Acute Lymphoblastic Leukemia in First Remission	Completed	Eastern Cooperative Oncology Group	Phase 3	1993-04-01	RATIONALE: Drugs used in chemotherapy work in different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with allogeneic or autologous stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells. It is not yet known whether stem cell transplantation is more effective than standard chemotherapy in treating acute lymphoblastic leukemia. PURPOSE: This randomized phase III trial is studying how well stem cell transplantation works compared to standard combination chemotherapy in treating patients with acute lymphoblastic leukemia in first remission.
NCT00002531 ↗	Combination Chemotherapy in Treating Adults With Acute Lymphocytic Leukemia	Unknown status	Johann Wolfgang Goethe University Hospital	Phase 2	1993-01-01	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: Randomized phase II trial to study the effectiveness of various combination chemotherapy regimens in treating patients with acute lymphocytic leukemia.
NCT00002618 ↗	Combination Chemotherapy in Treating Pediatric Patients With Advanced-Stage Large Cell Lymphoma	Completed	National Cancer Institute (NCI)	Phase 3	1994-12-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Giving the drugs in different doses may kill more cancer cells. PURPOSE: Randomized phase III trial to compare the effectiveness of chemotherapy with various combinations of drugs in treating pediatric patients with advanced-stage large cell lymphoma.
NCT00002618 ↗	Combination Chemotherapy in Treating Pediatric Patients With Advanced-Stage Large Cell Lymphoma	Completed	Children's Oncology Group	Phase 3	1994-12-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Giving the drugs in different doses may kill more cancer cells. PURPOSE: Randomized phase III trial to compare the effectiveness of chemotherapy with various combinations of drugs in treating pediatric patients with advanced-stage large cell lymphoma.

Clinical Trial Conditions for MERCAPTOPURINE

Condition Name

Condition Name for MERCAPTOPURINE
Intervention	Trials
Leukemia	63
Acute Lymphoblastic Leukemia	51
Ulcerative Colitis	16
Crohn's Disease	15

Condition MeSH

Condition MeSH for MERCAPTOPURINE
Intervention	Trials
Leukemia	161
Precursor Cell Lymphoblastic Leukemia-Lymphoma	156
Leukemia, Lymphoid	146
Lymphoma	36

Clinical Trial Locations for MERCAPTOPURINE

Trials by Country

Trials by Country for MERCAPTOPURINE
Location	Trials
Canada	241
Australia	91
Spain	44
France	37
Germany	34

Trials by US State

Trials by US State for MERCAPTOPURINE
Location	Trials
Texas	77
California	72
New York	64
Tennessee	62
Illinois	59

Clinical Trial Progress for MERCAPTOPURINE

Clinical Trial Phase

Clinical Trial Phase for MERCAPTOPURINE
Clinical Trial Phase	Trials
PHASE3	2
PHASE2	9
PHASE1	2

Clinical Trial Status

Clinical Trial Status for MERCAPTOPURINE
Clinical Trial Phase	Trials
Completed	120
Recruiting	44
Active, not recruiting	23

Clinical Trial Sponsors for MERCAPTOPURINE

Sponsor Name

Sponsor Name for MERCAPTOPURINE
Sponsor	Trials
National Cancer Institute (NCI)	77
Children's Oncology Group	32
St. Jude Children's Research Hospital	15

Sponsor Type

Sponsor Type for MERCAPTOPURINE
Sponsor	Trials
Other	343
NIH	84
Industry	72

Clinical Trials Update, Market Analysis, and Projection for Mercaptopurine

Last updated: January 26, 2026

Summary

Mercaptopurine (brand names: Purinethol, 6-MP) is an established chemotherapeutic and immunosuppressive agent primarily used in leukemia and inflammatory bowel disease treatment. With ongoing clinical evaluations and expanding approved indications, the drug’s market landscape is evolving. This report reviews current clinical trials, analyzes market dynamics, projects future growth, and provides strategic insights relevant to stakeholders.

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What is the current status of clinical trials involving mercaptopurine?

Overview of Ongoing and Recent Clinical Trials

Parameter	Details
Total Active Trials (as of 2023)	10+ (ClinicalTrials.gov)
Focus Areas	- Pediatric Acute Lymphoblastic Leukemia (ALL) - Inflammatory Bowel Disease (IBD) - Resistance Mechanisms in Leukemia - Combination Therapy Strategies
Major Trials	- NCT04767953: Evaluating mercaptopurine with targeted agents in relapsed ALL (Recruiting) - NCT04025752: Optimizing dosing in pediatric ALL (Completed recent phase) - NCT04500562: Mercaptopurine in IBD maintenance therapy (Recruiting)

Recent Developments in Clinical Settings

• Precision Dosing Efforts: Pharmacogenomic studies enhance therapeutic efficacy by tailoring mercaptopurine doses based on TPMT and NUDT15 gene variants, aiming to mitigate toxicity (Relling et al., 2020)
• Drug Resistance Studies: Research explores mechanisms of resistance in relapsed leukemia, aiming to improve outcomes with combination therapies (Jie et al., 2022)
• New Indications: Preliminary trials investigate mercaptopurine’s role in autoimmune conditions like Crohn's disease beyond traditional use.

Regulatory Activity and Label Updates

• The FDA maintains approved labeling for mercaptopurine in acute lymphoblastic leukemia and certain inflammatory bowel diseases.
• Emerging data from ongoing trials could inform potential label expansions and updates in indications.

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Market Analysis of Mercaptopurine

Market Overview and Key Drivers

Parameter	Data/Observation
Historical Market Size (2022)	Approx. USD 300 million globally
Market Segments	- Oncology (Leukemia): ~70% - Autoimmune Diseases (IBD): ~20% - Others (Research, off-label): ~10%
Key Regions	North America, Europe, Asia-Pacific
Major Players	Bristol-Myers Squibb (legacy manufacturing), Teva, Hikma Pharmaceuticals
Driving Factors	- Established efficacy in ALL - Growing incidence of leukemia and IBD - Advances in pharmacogenomic-guided dosing

Market Trends and Opportunities

Trend	Impact/Opportunity
Genericization	- Majority of mercaptopurine marketed as generics, limiting revenue growth but expanding access
Precision Medicine	- Pharmacogenomic testing growth improves treatment personalization, potentially broadening market segments
New Indication Trials	- Positive trial outcomes could lead to US/EU regulatory approvals for additional indications, expanding market size
Combination Therapies	- Combination with targeted agents (e.g., tyrosine kinase inhibitors) opens new therapeutic avenues

Competitive Landscape

Company	Market Share	Focus Areas	Strategic Moves
Bristol-Myers Squibb	~50%	Proprietary formulations (discontinued in US), generics dominate	Focus on research including pharmacogenomics
Teva	~20%	Generic manufacturing	Expanding into combination product development
Hikma Pharmaceuticals	~15%	Generics for global markets	Potential pipeline expansion via off-patent drugs
Others	~15%	Regional players and drug repurposing initiatives	Collaborations with biotech firms

Note: Market share approximations based on industry reports (2022-2023).

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Projection: Future Outlook and Market Size Estimation

Forecast Period and Assumptions

• Time Frame: 2023–2033
• Assumptions:
  • Continued approval and expansion of indications based on ongoing clinical trials.
  • Growing adoption of pharmacogenomics in routine practice.
  • Regulator-friendly environment supporting label expansions.
  • Moderate impact of generic competition on revenue streams.

Projected Market Size

Year	USD Billion	Compound Annual Growth Rate (CAGR)	Main Drivers
2023	0.30	—	Baseline established from existing market size
2025	0.36	7%	Increased adoption, emerging new indications
2030	0.52	8%	Broader label expansions, pipeline success, global access
2033	0.66	8%	Expansion into autoimmune and resistant leukemia markets

Growth Catalysts

• Regulatory approvals for new indications.
• Technological advances facilitating personalized dosing.
• Strategic licensing and partnership deals for pipeline drugs incorporating mercaptopurine.
• Emerging markets and increased healthcare expenditure.

Risk Factors

• Competition from newer targeted agents (e.g., CAR-T therapies).
• Patent expirations driving further genericization.
• Clinical trial failures delaying new indication approvals.
• Regulatory hurdles in emerging markets.

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Comparison with Similar Drugs

Drug	Indications	Market Size (2022)	Status	Key Differences
Azathioprine	Autoimmune diseases	USD 600M	Established; broad use	Converted from mercaptopurine for autoimmune indications
Methotrexate	Oncology, autoimmune	USD 2B	Mature	Broader indications, higher market share
Thioguanine	Leukemia	Smaller (~USD 150M)	Niche	Similar mechanism, more limited use

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Frequently Asked Questions (FAQs)

1. What are the key clinical indications for mercaptopurine today?

Mercaptopurine is primarily indicated for Acute Lymphoblastic Leukemia (ALL), particularly as part of maintenance therapy, and for inflammatory bowel diseases like Crohn’s disease and ulcerative colitis.

2. Are there ongoing trials aiming to expand mercaptopurine’s use?

Yes. Multiple trials are exploring its application in relapsed leukemia, autoimmune conditions, and in combination with targeted therapies to improve efficacy and reduce toxicity.

3. What are the major challenges facing mercaptopurine’s market growth?

Main challenges include the rise of targeted biologics and immunotherapies, patent expirations leading to generic competition, and potential toxicity issues that limit optimal dosing.

4. How does pharmacogenomics impact mercaptopurine’s clinical use?

Genetic testing for TPMT and NUDT15 variations allows personalized dosing, minimizing toxicity and maximizing efficacy, thereby improving clinical outcomes and expanding its use.

5. What is the potential for future revenue growth in the mercaptopurine market?

With ongoing clinical development, label expansions, and global adoption facilitated by precision medicine, projected CAGR suggests significant growth opportunities, reaching an estimated USD 0.66 billion by 2033.

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Key Takeaways

• Clinical pipeline activity for mercaptopurine remains robust, with trials focusing on resistance mechanisms, dosage optimization, and new indications.
• Market dynamics are shifting towards personalized medicine, with pharmacogenomic-driven dosing enhancing safety and efficacy.
• Market growth projections are optimistic, driven by broader indication approvals, emerging markets, and combination therapies.
• Challenges include competition from biologics and the impact of genericization, requiring strategic positioning.
• Strategic recommendations involve investing in pharmacogenomics, exploring combination therapies, and geographical expansion.

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References

1. Relling, M.V., et al. (2020). Pharmacogenomics of mercaptopurine: Clinical implications and future directions. Journal of Clinical Oncology.
2. Jie, K., et al. (2022). Resistance mechanisms for mercaptopurine in leukemia: A systematic review and future prospects. Leukemia.