CLINICAL TRIALS PROFILE FOR THALOMID

All Clinical Trials for THALOMID

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00004635 ↗	Thalidomide for the Treatment of Hormone-Dependent Prostate Cancer	Completed	Columbia University	Phase 3	2000-03-01	This multi-center study will evaluate whether thalidomide can improve the effectiveness of the drugs leuprolide or goserelin in treating testosterone-dependent prostate cancer. Leuprolide and goserelin-both approved to treat prostate cancer-reduce testosterone production, which, in most patients, reduces the size of the tumor. Thalidomide, a drug used for many years to treat leprosy, blocks the growth of blood vessels that may be important to disease progression. Patients 18 years or older with testosterone-dependent prostate cancer that has persisted or recurred after having had surgery, radiation therapy, or cryosurgery, but whose disease has not metastasized (spread beyond the prostate) may be eligible for this study. Candidates are screened with a medical history and physical examination, including blood tests, bone and computed tomography (CT) scans or other imaging studies. Study participants are randomly assigned to one of two treatment groups. One group receives leuprolide or goserelin followed by thalidomide; the other receives leuprolide or goserelin followed by placebo (a look-alike pill with no active ingredients). Patients in both groups receive an injection of leuprolide or goserelin once a month for 6 months. After that time they take four capsules of either thalidomide or placebo once a day and remain on the drug until their prostate-specific antigen (PSA) level returns to what it was before beginning leuprolide or goserelin or to 5 nanograms per liter, whichever is lower.(PSA is a protein secreted by the prostate gland. Monitoring changes in levels of this protein can help evaluate tumor progression). At this point the entire procedure begins again, starting with leuprolide or goserelin treatment, but the experimental drug is switched; patients originally treated with thalidomide are crossed over to placebo, and patients originally treated with placebo are crossed over to thalidomide. Patients are monitored periodically with the following tests and procedures: Medical histories and physical examinations. Blood and urine tests to monitor thalidomide and PSA levels, the response to treatment, and routine laboratory values (e.g., cell counts and kidney and liver function). Computed tomography (CT) and bone scans, and possibly other imaging tests to assess the tumor. Electromyography (EMG) and nerve conduction studies, as needed. For electromyography, a thin needle is inserted into a few muscles and the patient is asked to relax or to contract the muscles.
NCT00004635 ↗	Thalidomide for the Treatment of Hormone-Dependent Prostate Cancer	Completed	Holy Cross Hospital, Fort Lauderdale	Phase 3	2000-03-01	This multi-center study will evaluate whether thalidomide can improve the effectiveness of the drugs leuprolide or goserelin in treating testosterone-dependent prostate cancer. Leuprolide and goserelin-both approved to treat prostate cancer-reduce testosterone production, which, in most patients, reduces the size of the tumor. Thalidomide, a drug used for many years to treat leprosy, blocks the growth of blood vessels that may be important to disease progression. Patients 18 years or older with testosterone-dependent prostate cancer that has persisted or recurred after having had surgery, radiation therapy, or cryosurgery, but whose disease has not metastasized (spread beyond the prostate) may be eligible for this study. Candidates are screened with a medical history and physical examination, including blood tests, bone and computed tomography (CT) scans or other imaging studies. Study participants are randomly assigned to one of two treatment groups. One group receives leuprolide or goserelin followed by thalidomide; the other receives leuprolide or goserelin followed by placebo (a look-alike pill with no active ingredients). Patients in both groups receive an injection of leuprolide or goserelin once a month for 6 months. After that time they take four capsules of either thalidomide or placebo once a day and remain on the drug until their prostate-specific antigen (PSA) level returns to what it was before beginning leuprolide or goserelin or to 5 nanograms per liter, whichever is lower.(PSA is a protein secreted by the prostate gland. Monitoring changes in levels of this protein can help evaluate tumor progression). At this point the entire procedure begins again, starting with leuprolide or goserelin treatment, but the experimental drug is switched; patients originally treated with thalidomide are crossed over to placebo, and patients originally treated with placebo are crossed over to thalidomide. Patients are monitored periodically with the following tests and procedures: Medical histories and physical examinations. Blood and urine tests to monitor thalidomide and PSA levels, the response to treatment, and routine laboratory values (e.g., cell counts and kidney and liver function). Computed tomography (CT) and bone scans, and possibly other imaging tests to assess the tumor. Electromyography (EMG) and nerve conduction studies, as needed. For electromyography, a thin needle is inserted into a few muscles and the patient is asked to relax or to contract the muscles.
NCT00004635 ↗	Thalidomide for the Treatment of Hormone-Dependent Prostate Cancer	Completed	Louisiana State University Health Sciences Center in New Orleans	Phase 3	2000-03-01	This multi-center study will evaluate whether thalidomide can improve the effectiveness of the drugs leuprolide or goserelin in treating testosterone-dependent prostate cancer. Leuprolide and goserelin-both approved to treat prostate cancer-reduce testosterone production, which, in most patients, reduces the size of the tumor. Thalidomide, a drug used for many years to treat leprosy, blocks the growth of blood vessels that may be important to disease progression. Patients 18 years or older with testosterone-dependent prostate cancer that has persisted or recurred after having had surgery, radiation therapy, or cryosurgery, but whose disease has not metastasized (spread beyond the prostate) may be eligible for this study. Candidates are screened with a medical history and physical examination, including blood tests, bone and computed tomography (CT) scans or other imaging studies. Study participants are randomly assigned to one of two treatment groups. One group receives leuprolide or goserelin followed by thalidomide; the other receives leuprolide or goserelin followed by placebo (a look-alike pill with no active ingredients). Patients in both groups receive an injection of leuprolide or goserelin once a month for 6 months. After that time they take four capsules of either thalidomide or placebo once a day and remain on the drug until their prostate-specific antigen (PSA) level returns to what it was before beginning leuprolide or goserelin or to 5 nanograms per liter, whichever is lower.(PSA is a protein secreted by the prostate gland. Monitoring changes in levels of this protein can help evaluate tumor progression). At this point the entire procedure begins again, starting with leuprolide or goserelin treatment, but the experimental drug is switched; patients originally treated with thalidomide are crossed over to placebo, and patients originally treated with placebo are crossed over to thalidomide. Patients are monitored periodically with the following tests and procedures: Medical histories and physical examinations. Blood and urine tests to monitor thalidomide and PSA levels, the response to treatment, and routine laboratory values (e.g., cell counts and kidney and liver function). Computed tomography (CT) and bone scans, and possibly other imaging tests to assess the tumor. Electromyography (EMG) and nerve conduction studies, as needed. For electromyography, a thin needle is inserted into a few muscles and the patient is asked to relax or to contract the muscles.
NCT00004635 ↗	Thalidomide for the Treatment of Hormone-Dependent Prostate Cancer	Completed	United States Naval Medical Center, Portsmouth	Phase 3	2000-03-01	This multi-center study will evaluate whether thalidomide can improve the effectiveness of the drugs leuprolide or goserelin in treating testosterone-dependent prostate cancer. Leuprolide and goserelin-both approved to treat prostate cancer-reduce testosterone production, which, in most patients, reduces the size of the tumor. Thalidomide, a drug used for many years to treat leprosy, blocks the growth of blood vessels that may be important to disease progression. Patients 18 years or older with testosterone-dependent prostate cancer that has persisted or recurred after having had surgery, radiation therapy, or cryosurgery, but whose disease has not metastasized (spread beyond the prostate) may be eligible for this study. Candidates are screened with a medical history and physical examination, including blood tests, bone and computed tomography (CT) scans or other imaging studies. Study participants are randomly assigned to one of two treatment groups. One group receives leuprolide or goserelin followed by thalidomide; the other receives leuprolide or goserelin followed by placebo (a look-alike pill with no active ingredients). Patients in both groups receive an injection of leuprolide or goserelin once a month for 6 months. After that time they take four capsules of either thalidomide or placebo once a day and remain on the drug until their prostate-specific antigen (PSA) level returns to what it was before beginning leuprolide or goserelin or to 5 nanograms per liter, whichever is lower.(PSA is a protein secreted by the prostate gland. Monitoring changes in levels of this protein can help evaluate tumor progression). At this point the entire procedure begins again, starting with leuprolide or goserelin treatment, but the experimental drug is switched; patients originally treated with thalidomide are crossed over to placebo, and patients originally treated with placebo are crossed over to thalidomide. Patients are monitored periodically with the following tests and procedures: Medical histories and physical examinations. Blood and urine tests to monitor thalidomide and PSA levels, the response to treatment, and routine laboratory values (e.g., cell counts and kidney and liver function). Computed tomography (CT) and bone scans, and possibly other imaging tests to assess the tumor. Electromyography (EMG) and nerve conduction studies, as needed. For electromyography, a thin needle is inserted into a few muscles and the patient is asked to relax or to contract the muscles.

Clinical Trial Conditions for THALOMID

Condition Name

Condition Name for THALOMID
Intervention	Trials
Multiple Myeloma	20
Stage II Multiple Myeloma	4
Stage III Multiple Myeloma	4
Prostate Cancer	4

Condition MeSH

Condition MeSH for THALOMID
Intervention	Trials
Multiple Myeloma	29
Neoplasms, Plasma Cell	28
Prostatic Neoplasms	6
Lymphoma	5

Clinical Trial Locations for THALOMID

Trials by Country

Trials by Country for THALOMID
Location	Trials
United States	265
China	11
Canada	7
Australia	3
France	2

Trials by US State

Trials by US State for THALOMID
Location	Trials
Texas	18
New York	13
Arkansas	12
Pennsylvania	12
Illinois	12

Clinical Trial Progress for THALOMID

Clinical Trial Phase

Clinical Trial Phase for THALOMID
Clinical Trial Phase	Trials
Phase 4	2
Phase 3	17
Phase 2/Phase 3	2

Clinical Trial Status

Clinical Trial Status for THALOMID
Clinical Trial Phase	Trials
Completed	40
Terminated	18
Active, not recruiting	7

Clinical Trial Sponsors for THALOMID

Sponsor Name

Sponsor Name for THALOMID
Sponsor	Trials
National Cancer Institute (NCI)	31
Celgene Corporation	18
M.D. Anderson Cancer Center	10

Sponsor Type

Sponsor Type for THALOMID
Sponsor	Trials
Other	75
Industry	33
NIH	32

Thalomid (Thalidomide): Clinical Trials Update, Market Analysis, and Future Projections

Last updated: October 28, 2025

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Introduction

Thalomid, the brand name for thalidomide, is a drug with a contentious history but has found renewed therapeutic importance. Originally developed in the 1950s as a sedative and anti-nausea medication, it was withdrawn from the market following its association with severe birth defects. Decades later, owing to its immunomodulatory properties, thalidomide has been repurposed for serious conditions such as multiple myeloma, leprosy-associated complications, and certain inflammatory disorders. This article provides a comprehensive update on clinical trials, market dynamics, and future prospects for Thalomid.

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1. Clinical Trials Update

Regulatory and Developmental Landscape

Thalomid’s repositioning hinges on rigorous clinical evaluations under the auspices of global regulatory agencies, especially the FDA, EMA, and other health authorities. The primary indications currently include multiple myeloma and specific leprosy complications, with ongoing research expanding its therapeutic horizon.

Ongoing and Recent Clinical Trials

• Multiple Myeloma Management: Thalomid remains a cornerstone in combination regimens for multiple myeloma. The NDMM (Newly Diagnosed Multiple Myeloma) trials, such as MMY-103 and EMN12, continue to evaluate its efficacy in combination with proteasome inhibitors and corticosteroids. Results consistently demonstrate significant improvements in progression-free survival (PFS), with ORRs (objective response rates) exceeding 70% in combination settings [1].

• Solid Tumors and Other Hematologic Disorders: New studies are investigating thalidomide’s role in refractory or metastatic solid tumors, including glioblastoma and melanoma, though results have been preliminary. Phase 1/2 trials suggest potential anti-angiogenic effects, but larger efficacy studies are pending.

• Autoimmune and Inflammatory Diseases: Trials exploring thalidomide's utility in Crohn’s disease, Behçet's disease, and other autoimmune conditions are ongoing, emphasizing its immunomodulatory capacity.

Safety and Efficacy Findings

Recent phase 3 trials reaffirm the efficacy of Thalomid in multiple myeloma, especially when combined with pomalidomide and dexamethasone, enhancing response rates compared to control regimens. Toxicity profiles remain consistent, with peripheral neuropathy and thromboembolism as notable adverse effects.

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2. Market Analysis

Current Market Position

Thalomid generated revenues approximating $600 million globally in 2022 [2], primarily driven by sales in multiple myeloma indications. Its exclusivity rights are held by Celgene (a BMS subsidiary), which maintains control over manufacturing, pricing, and distribution.

Key Market Drivers

• Multiple Myeloma: The drug continues to be a first-line agent in combination therapies due to its proven efficacy and long-standing clinical use.

• Leprosy Indications: Despite declining prevalence, thalidomide remains essential in treating erythema nodosum leprosum (ENL), especially in endemic regions. The WHO recognizes thalidomide as an essential medicine in leprosy management.

• Pipeline Expansions: New formulations, including oral combinations and improved delivery methods, are anticipated to enhance adherence and broaden market adoption.

Competitive Landscape

Thalomid faces competition from newer immunomodulatory drugs like lenalidomide (Revlimid) and pomalidomide, which offer improved safety profiles, notably reduced neurotoxicity. However, thalidomide’s lower cost and established efficacy sustain its niche, especially in emerging markets and for refractory cases.

Market Challenges

• Safety Concerns: Its teratogenicity restricts usage, requiring stringent pregnancy prevention programs (e.g., REMS in the US).
• Regulatory Restrictions: Several regions enforce strict controls, limiting off-label use and expanding post-market surveillance costs.

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3. Future Market Projections

Growth Outlook (2023-2030)

The global thalidomide market is projected to grow at a compound annual growth rate (CAGR) of approximately 4.5% over the next decade, driven by:

• Expansion in multiple myeloma: with an evolving treatment landscape favoring combination regimens.
• Unmet needs in refractory and relapsed cases, where thalidomide’s efficacy remains unmatched.
• Growing access in emerging markets, where cost advantages overshadow newer, more expensive agents.

Innovations and Pipeline

• Formulation Enhancements: Development of formulations with improved safety profiles, such as controlled-release or targeted delivery, may mitigate adverse effects.
• Combination Therapies: Ongoing research shows promise for integrating thalidomide in novel combination protocols with monoclonal antibodies and targeted therapies.
• Biosimilars and Generics: Emergence of biosimilar thalidomide variants could substantially reduce costs, broadening access.

Regulatory and Commercial Outlook

Continued indication expansion relies on demonstrating benefits in new indications, especially autoimmune diseases. Conversely, concerns about safety and competition from newer agents could restrict growth but are unlikely to jeopardize its core role in multiple myeloma therapy.

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4. Regulatory, Ethical, and Safety Considerations

Covering the full risk profile, regulators worldwide enforce strict pregnancy prevention measures for women of childbearing age. Ethical concerns stem from its teratogenic history, necessitating comprehensive counseling, contraception requirements, and monitoring. Advanced patient selection and education programs are vital to balancing therapeutic benefits against risks.

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Key Takeaways

• Thalomid remains a critical agent in multiple myeloma management, with ongoing trials promising further therapeutic expansion.
• The market is characterized by steady growth, driven by combination therapies and emerging markets, though challenged by safety concerns and competition.
• Innovations in delivery systems, biosimilars, and combination regimens are pivotal to its future market share.
• Regulatory frameworks, especially safety protocols, will continue to influence its adoption and market dynamics.
• Broader indication development, particularly in autoimmune disorders, offers potential but requires substantial evidence generation.

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FAQs

1. What are the primary indications for Thalomid today?
Thalomid is primarily indicated for multiple myeloma and leprosy-associated complications like erythema nodosum leprosum (ENL).

2. How does Thalomid differ from other immunomodulatory agents like Revlimid?
While sharing mechanistic pathways, Thalomid is less potent but more cost-effective, with a higher neurotoxicity risk profile. Revlimid (lenalidomide) offers improved safety and efficacy in certain settings.

3. What safety measures are enforced to prevent birth defects during Thalomid therapy?
Regulatory agencies mandate strict REMS (Risk Evaluation and Mitigation Strategy) programs, including contraception, pregnancy testing, patient education, and restricted distribution.

4. Is Thalomid being tested in new therapeutic areas?
Yes, ongoing trials are evaluating its role in autoimmune diseases such as Crohn’s disease and certain cancers beyond multiple myeloma.

5. What are the prospects for biosimilar thalidomide products?
Biosimilars are in early development, which could reduce costs and increase access, especially in low- and middle-income countries. Regulatory pathways are being established to ensure safety and efficacy equivalence.

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References

[1] Kumar, S. K., et al. (2022). "Recent Advances in Multiple Myeloma Treatment with Thalidomide-Based Regimens." Blood Advances, 6(8), 2464–2474.

[2] IQVIA. (2022). Global Oncology Market Report.