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.
NCT00004635 ↗	Thalidomide for the Treatment of Hormone-Dependent Prostate Cancer	Completed	University of Minnesota	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 III Multiple Myeloma	4
Prostate Cancer	4
Stage I Multiple Myeloma	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 and market projection: status, pipeline catalysts, and exclusivity risk

Last updated: May 22, 2026

What clinical trials update matters for Thalomid (thalidomide) in 2024–2026?

Answer: Thalomid’s clinical activity is concentrated in (1) maintenance or combination regimens for hematologic malignancies (notably multiple myeloma) and (2) label-relevant safety programs in teratogenic-risk compliance and pharmacovigilance. Most “new-signal” randomized efficacy studies in myeloma are now incremental or platform-based (IMiD combinations) rather than replacing the core regimen landscape.

What trial themes are currently active

1. Multiple myeloma combinations using IMiDs backbones
   • Thalidomide is still used in older and lower-cost regimens and as a comparator in some ongoing studies, particularly where resource constraints favor oral, off-the-shelf regimens.
2. Relapsed/refractory hematology studies using real-world operational endpoints
   • Ongoing protocols often include patient-reported outcomes, deep-response correlates, and real-world adherence components.
3. Safety, pregnancy prevention, and risk management compliance
   • Trials and postmarketing programs emphasize REMS adherence, controlled dispensing, and pregnancy testing workflows tied to the thalidomide REMS.

Key clinical-program datapoints that typically move the needle

• Response durability endpoints (PFS, time to progression) in combination arms.
• Safety management outcomes focused on neuropathy, thromboembolism, and cytopenias.
• Patient access and compliance metrics (dose interruptions, adherence under REMS).

How big is the Thalomid market today, and what segments drive demand?

Answer: Thalomid’s current market is driven by multiple myeloma use under existing standard-of-care options, with additional demand in settings where lower-cost oral regimens remain common. The dominant revenue pool is concentrated in mature markets with established myeloma treatment pathways and controlled distribution infrastructure.

Market demand segmentation

• Therapeutic indication concentration
  • Multiple myeloma remains the primary use driver. Thalidomide’s historical role in relapsed/refractory and maintenance strategies still anchors demand.
• Geography
  • Mature oncology markets account for most value due to reimbursement intensity and uptake of modern myeloma regimens that retain thalidomide as a component in specific lines.
• Channel dynamics
  • Specialty distribution and REMS-controlled dispensing are material to economics and patient flow.

Commercial reality for older oral oncology assets

• Thalomid competes against newer IMiD backbones and proteasome inhibitor-based regimens that have shifted prescribing patterns.
• Pricing pressure in mature markets is a consistent risk factor as generic thalidomide products enter or expand share (where permitted).

When does Thalomid lose exclusivity, and what does that mean for generic entry risk?

Answer: Thalomid’s exclusivity structure has already matured substantially in many jurisdictions because thalidomide is off-patent for the core active ingredient in most major markets. Market share is therefore most influenced by (1) formulation or method-of-use patent pockets (if any remain in specific regions) and (2) regulatory and REMS-related operational barriers rather than primary composition patent exclusivity.

Exclusivity and patent landscape logic for Thalomid

• Composition-of-matter: largely historical for thalidomide as an active ingredient.
• Formulation / method-of-use: can create “secondary” barriers in specific regions, but the practical effect depends on whether Orange Book listings or equivalent regulatory listings still exist and are enforceable for the marketed dosage form.
• Regulatory and REMS constraints: do not stop generic approvals by themselves, but they affect time-to-market, distribution readiness, and pharmacovigilance execution.

Generic entry risks

• The dominant generic risk for Thalomid is less “hard injunction” and more “share erosion” from accessible generics that meet regulatory requirements and can operate the REMS supply chain.
• Settlement agreements (where they exist) can shift entry timing by months or a year range, but the structural direction remains toward increased generic share.

What patents protect Thalomid (thalidomide) and what is the likely remaining enforcement posture?

Answer: Patent protection for thalidomide in the US has largely shifted away from active ingredient coverage toward secondary protections, including certain formulations, manufacturing approaches, and/or specific method-of-use claims, depending on jurisdiction and marketed dosage form. In practice, enforcement posture is often limited because thalidomide’s core compound patents expired years earlier.

How to interpret “patent estate strength” for an off-patent oral oncology asset

• If remaining listings exist: they typically relate to narrow formulation or dosing/administration methods that require close product-label and manufacturing comparison.
• If listings are absent: the generic entry clock is governed mainly by regulatory readiness and REMS operational timelines.

What is the Orange Book status of Thalomid, and which listings usually control launch timing?

Answer: Thalomid is marketed as an established thalidomide product with REMS controls. For launch-timing assessment, the controlling items in the US Orange Book are any listed patents tied to the marketed product and whether they have active Paragraph IV litigation or any settlement-driven entry delays. For thalidomide, the core active-ingredient claims are not typically the binding items.

What to look for in Orange Book listings (launch-control factors)

• Drug product type and NDC linkage
• Listed patent types
  • Composition of matter
  • Method of use
  • Formulation/manufacturing
• Patent expiration and any unexpired pediatric exclusivity
• Any active court dockets tied to Paragraph IV certifications

How do biosimilar risk and biologics pathways apply to Thalomid?

Answer: Thalomid is a small molecule, not a biologic. Biosimilar risk is not applicable.

Which clinical trial or real-world endpoints most affect Thalomid prescribing decisions now?

Answer: Prescribers now weigh thalidomide primarily on clinical effectiveness in specific myeloma lines and on tolerability trade-offs, especially neuropathy and thromboembolism risk management.

Tolerability endpoints that drive continued use

• Peripheral neuropathy incidence and severity
• Thromboembolism rates under prophylaxis protocols
• Cytopenia frequency and grade distribution
• Treatment discontinuation rates due to adverse events

Effectiveness endpoints

• Overall response rate (ORR)
• Progression-free survival (PFS)
• Time to next treatment (TTNT) in real-world studies

What competitor products most influence Thalomid market share?

Answer: Thalomid’s market is challenged by newer myeloma regimen standards that use next-generation proteasome inhibitors, immunomodulatory combinations, and regimen simplification strategies. Thalidomide remains used where oral and cost-access pathways matter or where specific regimen logic is preferred.

Competitive set

• IMiD and combination regimens: lenalidomide- and pomalidomide-based strategies
• Proteasome inhibitor-based regimens: widely adopted in most lines
• Monoclonal antibody combinations: growing share in frontline and relapsed settings depending on country

What is the most likely 12–36 month market trajectory for Thalomid?

Answer: Over the next 12 to 36 months, Thalomid’s trajectory is most likely shaped by generic share expansion in jurisdictions where it is ongoing and by incremental label or regimen adoption dynamics in multiple myeloma.

Base-case projection framework

• Revenue direction: modest decline or flat-to-down as generic penetration increases and regimen preferences shift toward newer standards.
• Volume direction: stable to down depending on (1) generic availability and (2) physician shift away from older thalidomide regimens.
• Price/mix: negative pressure from lower-priced equivalents in markets with competitive generic supply.

Key market variables

• REMS operational capacity for new entrants
• Competitive pricing in mature markets
• Changes in reimbursement for older IMiD backbones
• Myeloma clinical guideline updates that shift line-of-therapy preferences

What patent litigation and settlements affect Thalomid generic entry timing?

Answer: For thalidomide, litigation tends to be concentrated around any remaining secondary listings (formulation or method-of-use) rather than core active-ingredient patents. The practical effect is usually timing-based rather than a permanent exclusion.

How to map litigation to commercial impact

• Identify whether any active Paragraph IV disputes exist for the specific NDC(s).
• Link docket status to:
  • tentative launch dates
  • court-ordered stay durations
  • settlement entry triggers

What manufacturing and IP barriers can slow generic competition for Thalomid?

Answer: The barriers are typically operational rather than technical: REMS-controlled distribution, quality system readiness, and ensuring product equivalence for the marketed dosage form under regulatory scrutiny.

Barriers that matter commercially

• REMS shipment and inventory management
• Pregnancy prevention workflow execution
• Pharmacovigilance and safety reporting readiness
• Batch release and stability qualification for the exact dosage form

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Key Takeaways

• Thalomid clinical activity centers on combination use in multiple myeloma and on compliance programs tied to thalidomide REMS.
• Thalomid market demand remains primarily myeloma-driven, but competitive pressure from newer regimen standards limits growth.
• Exclusivity is mostly a mature story; generic share expansion is the dominant near-term structural force in many markets.
• Patent enforcement, where present, usually reflects secondary listings rather than broad active-ingredient coverage.
• The most material “entry barrier” for generics is operational REMS execution and controlled distribution readiness.

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FAQs

1) What trial designs are most relevant when evaluating thalidomide in relapsed multiple myeloma today?

Randomized combination comparisons and real-world studies reporting neuropathy, discontinuation, and TTNT endpoints.

2) Does thalidomide have a biologics pathway or biosimilar equivalents?

No. Thalomid is a small molecule; biosimilar frameworks do not apply.

3) What REMS requirements most affect market access and prescribing workflows?

Pregnancy prevention program controls, controlled dispensing, pregnancy testing, and prescriber and patient enrollment workflows.

4) Are formulation patents more likely than composition patents to delay generic thalidomide launches?

Yes, where any remaining secondary listings exist for specific dosage forms, they can drive narrower launch timing disputes.

5) How does neuropathy risk change dosing and persistence with thalidomide regimens?

Neuropathy risk typically drives dose interruptions, discontinuation thresholds, and regimen selection toward lower-risk alternatives when clinically appropriate.

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References

1. FDA. Thalidomide REMS requirements and safety information. U.S. Food and Drug Administration.
2. U.S. FDA. Orange Book: Approved Drug Products with Therapeutic Equivalence Evaluations. Center for Drug Evaluation and Research.
3. ClinicalTrials.gov. Thalidomide (thalidomide) clinical studies records. U.S. National Institutes of Health.