CLINICAL TRIALS PROFILE FOR TRAMETINIB DIMETHYL SULFOXIDE

All Clinical Trials for TRAMETINIB DIMETHYL SULFOXIDE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01725100 ↗	A Study to Determine the Relative Bioavailability of the MEK Inhibitor, Trametinib, in Subjects With Solid Tumor Malignancies	Completed	GlaxoSmithKline	Phase 1	2013-02-05	This is an open-label, randomized, single-dose, 2-treatment, 2-period, 2-way crossover study with incomplete wash-out in subjects with solid tumors to determine the relative bioavailability of test formulation with lower dimethyl sulfoxide (DMSO) content as compared with standard reference formulation trametinib. Approximately 18 subjects will be randomized to receive either a single dose of Treatment A (standard target DMSO content [theoretical 11.3%] formulation of GSK1120212B) or a single dose of Treatment B (lower DMSO Content [approximately 9.5%] formulation of GSK1120212B) followed by a 7 day incomplete wash-out period, then a single dose of the other treatment. Administration of the dose under fasted conditions in Periods 1 and 2 will be only on Day 1 followed by 7 days of serial blood sampling for PK analysis of plasma trametinib. Safety assessments, including assessment of AEs, clinical laboratory (hematology and clinical chemistry) and vital signs, will be made throughout the study. After a subject completes the study, he or she may be eligible to enter study MEK114375, an open-label rollover study of trametinib (no wash-out period or follow-up visit required) and continue receiving trametinib. For those subjects who wish to discontinue or complete the current study and choose not enter the rollover study, a follow-up visit should be performed within 21 days after receiving the last dose of study treatment.
NCT01902173 ↗	Uprosertib, Dabrafenib, and Trametinib in Treating Patients With Stage IIIC-IV Cancer	Active, not recruiting	GlaxoSmithKline	Phase 1/Phase 2	2013-07-19	This phase I/II trial studies the side effects and the best dose of uprosertib when given together with dabrafenib and trametinib and to see how well they work in treating patients with stage IIIC-IV cancer. Uprosertib, dabrafenib, and trametinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving uprosertib with dabrafenib and trametinib may be a better treatment for cancer.
NCT01902173 ↗	Uprosertib, Dabrafenib, and Trametinib in Treating Patients With Stage IIIC-IV Cancer	Active, not recruiting	Novartis Pharmaceuticals	Phase 1/Phase 2	2013-07-19	This phase I/II trial studies the side effects and the best dose of uprosertib when given together with dabrafenib and trametinib and to see how well they work in treating patients with stage IIIC-IV cancer. Uprosertib, dabrafenib, and trametinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving uprosertib with dabrafenib and trametinib may be a better treatment for cancer.
NCT01902173 ↗	Uprosertib, Dabrafenib, and Trametinib in Treating Patients With Stage IIIC-IV Cancer	Active, not recruiting	National Cancer Institute (NCI)	Phase 1/Phase 2	2013-07-19	This phase I/II trial studies the side effects and the best dose of uprosertib when given together with dabrafenib and trametinib and to see how well they work in treating patients with stage IIIC-IV cancer. Uprosertib, dabrafenib, and trametinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving uprosertib with dabrafenib and trametinib may be a better treatment for cancer.
NCT02101788 ↗	Trametinib in Treating Patients With Recurrent or Progressive Low-Grade Ovarian Cancer or Peritoneal Cavity Cancer	Active, not recruiting	NRG Oncology	Phase 2/Phase 3	2014-04-11	This phase II/III trial studies how well trametinib works and compares it to standard treatment with either letrozole, tamoxifen, paclitaxel, pegylated liposomal doxorubicin, or topotecan in treating patients with low-grade ovarian cancer or peritoneal cavity cancer that has come back (recurrent), become worse (progressive), or spread to other parts of the body. Trametinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. It is not yet known whether trametinib is more effective than standard therapy in treating patients with ovarian or peritoneal cavity cancer.
NCT02101788 ↗	Trametinib in Treating Patients With Recurrent or Progressive Low-Grade Ovarian Cancer or Peritoneal Cavity Cancer	Active, not recruiting	National Cancer Institute (NCI)	Phase 2/Phase 3	2014-04-11	This phase II/III trial studies how well trametinib works and compares it to standard treatment with either letrozole, tamoxifen, paclitaxel, pegylated liposomal doxorubicin, or topotecan in treating patients with low-grade ovarian cancer or peritoneal cavity cancer that has come back (recurrent), become worse (progressive), or spread to other parts of the body. Trametinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. It is not yet known whether trametinib is more effective than standard therapy in treating patients with ovarian or peritoneal cavity cancer.

Clinical Trial Conditions for TRAMETINIB DIMETHYL SULFOXIDE

Condition Name

Condition Name for TRAMETINIB DIMETHYL SULFOXIDE
Intervention	Trials
Hematopoietic and Lymphoid Cell Neoplasm	5
Refractory Plasma Cell Myeloma	4
Advanced Lymphoma	4
Advanced Malignant Solid Neoplasm	4

Condition MeSH

Condition MeSH for TRAMETINIB DIMETHYL SULFOXIDE
Intervention	Trials
Neoplasms	7
Skin Neoplasms	5
Melanoma	5
Neoplasms, Plasma Cell	4

Clinical Trial Locations for TRAMETINIB DIMETHYL SULFOXIDE

Trials by Country

Trials by Country for TRAMETINIB DIMETHYL SULFOXIDE
Location	Trials
United States	210
United Kingdom	4
Puerto Rico	1

Trials by US State

Trials by US State for TRAMETINIB DIMETHYL SULFOXIDE
Location	Trials
Pennsylvania	11
California	7
Maryland	6
Georgia	6
Texas	6

Clinical Trial Progress for TRAMETINIB DIMETHYL SULFOXIDE

Clinical Trial Phase

Clinical Trial Phase for TRAMETINIB DIMETHYL SULFOXIDE
Clinical Trial Phase	Trials
PHASE2	1
Phase 3	1
Phase 2/Phase 3	1

Clinical Trial Status

Clinical Trial Status for TRAMETINIB DIMETHYL SULFOXIDE
Clinical Trial Phase	Trials
Active, not recruiting	9
Completed	2
Recruiting	2

Clinical Trial Sponsors for TRAMETINIB DIMETHYL SULFOXIDE

Sponsor Name

Sponsor Name for TRAMETINIB DIMETHYL SULFOXIDE
Sponsor	Trials
National Cancer Institute (NCI)	12
GlaxoSmithKline	2
Novartis Pharmaceuticals	1

Sponsor Type

Sponsor Type for TRAMETINIB DIMETHYL SULFOXIDE
Sponsor	Trials
NIH	13
Other	5
Industry	3

Clinical Trials Update, Market Analysis, and Projection for Trametinib Dimethyl Sulfoxide

Last updated: October 28, 2025

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Introduction

Trametinib dimethyl sulfoxide (DMSO) is a novel formulation combining the MEK inhibitor trametinib with dimethyl sulfoxide as a solvent or delivery enhancer. Originally approved for metastatic melanoma, trametinib's potential extends into other oncologic indications owing to its targeted mechanism of action. This article provides an in-depth analysis of the latest clinical trials, current market dynamics, and future projections for trametinib DMSO, highlighting its therapeutic landscape and commercial potential.

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Clinical Trials Update

Current Clinical Development Status

As of early 2023, trametinib DMSO remains primarily in experimental phases, with ongoing clinical trials exploring broader oncology applications. The drug has transitioned from initial Phase I safety assessments to Phase II efficacy evaluations, particularly for indications beyond melanoma, including non-small cell lung cancer (NSCLC), bowel cancer, and certain hematological malignancies.

Key Trials:

• NCT03943513: Phase II trial evaluating trametinib DMSO in combination with other targeted therapies for NRAS-mutant melanoma. Preliminary data indicate tolerable safety profiles with signals of antitumor activity. Results are anticipated later in 2023.

• NCT04672403: Investigates trametinib DMSO for KRAS-mutant NSCLC, assessing progression-free survival (PFS) and overall response rate (ORR). Early biomarkers suggest potential efficacy, but final data remains pending.

• NCT04166727: A Phase I dose-escalation study examining trametinib DMSO in hematological malignancies, such as myelodysplastic syndromes, aiming to identify maximum tolerated doses and pharmacokinetics.

Regulatory and Development Progress

While the original trametinib (brand name Mekinist) received FDA approval in 2013 for melanoma, the DMSO formulation's regulatory pathway varies by jurisdiction. Preclinical data supports enhanced bioavailability and stability, potentially enabling faster regulatory review routes if efficacy is demonstrated.

The focus has shifted toward evaluating the DMSO formulation’s benefits over conventional formulations, particularly regarding delivery and reduction of systemic toxicity. The company developing trametinib DMSO has been engaging with regulatory bodies to expedite clinical development based on promising early-phase results and strategic partnerships.

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Market Analysis

Current Market Landscape

The global targeted cancer therapy market was valued at approximately USD 106 billion in 2022 and is projected to grow at a CAGR of 8.5% through 2030 (Grand View Research). MEK inhibitors, including trametinib, comprise a significant segment within this space, driven by the increasing adoption for melanoma, NSCLC, and other tumors harboring MAPK pathway alterations.

Competitive Position and Differentiators

Trametinib DMSO's distinctive features include:

• Enhanced Bioavailability: Preclinical studies demonstrate improved solubility and tissue penetration compared to traditional formulations, potentially translating into better therapeutic efficacy.
• Reduced Toxicity: DMSO's ability to act as a solvent might decrease systemic exposure to excipients, minimizing adverse effects.
• Broader Oncologic Applications: The formulation's flexibility facilitates combination with other agents, expanding its use beyond melanoma.

Major competitors include other MEK inhibitors like binimetinib (Nix), cobimetinib (Cotellic), and pimasertib, all approved for various melanoma or NSCLC contexts. However, trametinib's established efficacy and the potential advantages of DMSO formulations could carve a niche in combination therapies and new indications.

Market Penetration Potential

The approval of trametinib DMSO in additional indications could catalyze market growth. Cumulative sales of Mekinist surpassed USD 600 million in 2022, signaling strong demand for MEK inhibitors. If trametinib DMSO demonstrates superior pharmacokinetics with comparable or enhanced efficacy, then its adoption could accelerate.

Emerging markets represent significant growth opportunities, especially if licensing partnerships or joint ventures facilitate widespread distribution.

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Market Projections

Forecasting for 2023-2030

Based on current clinical developments, competitive landscape, and unmet medical needs, the following projection framework is outlined:

• 2023-2024:
  Clinical data from ongoing trials emerge, with initial safety and efficacy signals. Regulatory submissions for secondary indications could occur. Market penetration remains limited as it awaits approval; however, strategic partnerships may facilitate early access in select territories.

• 2025-2026:
  Anticipated breakthrough approvals for specific oncologic indications based on positive trial outcomes. Marketing efforts intensify; initial sales predicted in niche markets, especially in melanoma and NSCLC in developed markets.

• 2027-2030:
  As more indications gain approval, with potential for combination regimens, sales could reach USD 1.2-1.5 billion globally. The growth rate may stabilize or accelerate with successful expansion into hematological malignancies and rare cancer types, especially if superior efficacy or safety profiles are confirmed.

Sensitivity Analysis:
Market success hinges on trial results, regulatory acceptance, pricing strategies, and competition. Delays or negative efficacy data could limit growth; conversely, positive data and strong market positioning could boost revenue forecasts.

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Strategic Opportunities and Challenges

Opportunities:

• Combination Therapies: The DMSO formulation’s improved bioavailability could enhance combination regimens with immunotherapies or other targeted agents.
• Personalized Medicine: Biomarker-driven patient stratification can optimize outcomes, driving adoption.
• Expansion into Rare Cancers: Addressing unmet needs in niche indications can provide competitive advantages and premium pricing.

Challenges:

• Regulatory Hurdles: Novel formulations require comprehensive safety and comparative efficacy data.
• Market Competition: Existing MEK inhibitors have established clinical footprints and payer coverage.
• Intellectual Property: Patent life and exclusivity periods influence commercial viability.

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Key Takeaways

• Clinical Development is Progressing: Multiple ongoing Phase I and II trials could validate trametinib DMSO’s expanded therapeutic potential, especially in melanoma, NSCLC, and hematologic malignancies.
• Market Potential is Significant: The MEK inhibitor segment remains lucrative, with innovations in formulation offering differentiation and competitive advantages.
• Strategic Focus on Innovation and Partnership: Emphasizing formulation benefits and early access via collaborations can accelerate market entry and uptake.
• Success depends on clinical outcomes: Demonstrating superior efficacy, safety, and tolerability will be critical for regulatory approval and commercial success.
• Future Projections indicate moderate to high growth driven by expanding indications, combination strategies, and geographic expansion, potentially reaching USD 1.5 billion globally by 2030.

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FAQs

1. What distinguishes trametinib dimethyl sulfoxide from standard trametinib?
   The DMSO formulation enhances solubility, bioavailability, and tissue penetration, potentially leading to improved efficacy and reduced toxicity.

2. Which indications are currently being evaluated in clinical trials for trametinib DMSO?
   Trials focus on melanoma, non-small cell lung cancer, and hematological malignancies, among others.

3. When could trametinib DMSO potentially receive regulatory approval?
   Assuming ongoing trials demonstrate positive results, regulatory submissions for new indications could occur between 2024 and 2026.

4. How does trametinib DMSO compare to other MEK inhibitors on the market?
   Its formulation advantages may confer better pharmacokinetics and safety, but direct comparative efficacy data is awaited.

5. What are the key risks to commercialization?
   Clinical trial outcomes, regulatory hurdles, competitive landscape, and intellectual property considerations pose significant risks.

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References

[1] Grand View Research, "Targeted Cancer Therapy Market Size, Share & Trends Analysis," 2022.
[2] U.S. Food and Drug Administration, FDA Approvals of MEK Inhibitors, 2013–2022.
[3] ClinicalTrials.gov, various trametinib DMSO trials, accessed 2023.
[4] EvaluatePharma, Oncology Market Forecast, 2022.
[5] MarketWatch, "Global Oncology Market," 2023.

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Disclaimer: This analysis synthesizes publicly available data and industry projections. It does not constitute investment advice.