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Last Updated: May 21, 2025

CLINICAL TRIALS PROFILE FOR MIDOSTAURIN


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All Clinical Trials for Midostaurin

Trial ID Title Status Sponsor Phase Start Date Summary
NCT00045942 ↗ PKC412 in Participants With Acute Myeloid Leukemia or With Myelodysplastic Syndrome (CPKC412A2104 Core); and PKC412 in Participants With Acute Myeloid Leukemia or With Myelodysplastic Syndrome With Either Wild Type or Mutated FMS-like Tyrosine Kinas Completed Dana-Farber Cancer Institute Phase 1/Phase 2 2002-01-30 CPKC412A2104 core had a 2 stage design. In stage 1, eight participants were treated. If at least one participant showed a clinical response, four more participants were recruited to stage 2. The trial was to be stopped if no participants showed a response in stage 1. POC was achieved if at least 2 participants out of 12 responded. In PKC412A2104E1, participants with AML or high risk MDS with wild-type or mutant FTL3 who had not previously received a FLT3 inhibitor were randomized to receive continuous twice daily oral doses of either 50 or 100 mg midostaurin in 1 28-day cycle regimen. Participants were to be treated until disease progression or the occurrence of unacceptable treatment-related toxicity. PKC412A2104 E2 contained 2 dosing regimens: 1) intra-participant midostaurin dose escalation and 2) midostaurin with itraconazole in participants with AML and high risk MDS irrespective of FLT3 status. Eligible participants were alternately assigned to the regimens. At the Investigator's discretion, intra-participant dose escalation was allowed for any previously enrolled CPKC412A2104E1 participant receiving midostaurin at the time of the approval of amendment 4. Participants were treated until the time of disease progression.
NCT00045942 ↗ PKC412 in Participants With Acute Myeloid Leukemia or With Myelodysplastic Syndrome (CPKC412A2104 Core); and PKC412 in Participants With Acute Myeloid Leukemia or With Myelodysplastic Syndrome With Either Wild Type or Mutated FMS-like Tyrosine Kinas Completed Memorial Sloan Kettering Cancer Center Phase 1/Phase 2 2002-01-30 CPKC412A2104 core had a 2 stage design. In stage 1, eight participants were treated. If at least one participant showed a clinical response, four more participants were recruited to stage 2. The trial was to be stopped if no participants showed a response in stage 1. POC was achieved if at least 2 participants out of 12 responded. In PKC412A2104E1, participants with AML or high risk MDS with wild-type or mutant FTL3 who had not previously received a FLT3 inhibitor were randomized to receive continuous twice daily oral doses of either 50 or 100 mg midostaurin in 1 28-day cycle regimen. Participants were to be treated until disease progression or the occurrence of unacceptable treatment-related toxicity. PKC412A2104 E2 contained 2 dosing regimens: 1) intra-participant midostaurin dose escalation and 2) midostaurin with itraconazole in participants with AML and high risk MDS irrespective of FLT3 status. Eligible participants were alternately assigned to the regimens. At the Investigator's discretion, intra-participant dose escalation was allowed for any previously enrolled CPKC412A2104E1 participant receiving midostaurin at the time of the approval of amendment 4. Participants were treated until the time of disease progression.
NCT00045942 ↗ PKC412 in Participants With Acute Myeloid Leukemia or With Myelodysplastic Syndrome (CPKC412A2104 Core); and PKC412 in Participants With Acute Myeloid Leukemia or With Myelodysplastic Syndrome With Either Wild Type or Mutated FMS-like Tyrosine Kinas Completed University of California, Los Angeles Phase 1/Phase 2 2002-01-30 CPKC412A2104 core had a 2 stage design. In stage 1, eight participants were treated. If at least one participant showed a clinical response, four more participants were recruited to stage 2. The trial was to be stopped if no participants showed a response in stage 1. POC was achieved if at least 2 participants out of 12 responded. In PKC412A2104E1, participants with AML or high risk MDS with wild-type or mutant FTL3 who had not previously received a FLT3 inhibitor were randomized to receive continuous twice daily oral doses of either 50 or 100 mg midostaurin in 1 28-day cycle regimen. Participants were to be treated until disease progression or the occurrence of unacceptable treatment-related toxicity. PKC412A2104 E2 contained 2 dosing regimens: 1) intra-participant midostaurin dose escalation and 2) midostaurin with itraconazole in participants with AML and high risk MDS irrespective of FLT3 status. Eligible participants were alternately assigned to the regimens. At the Investigator's discretion, intra-participant dose escalation was allowed for any previously enrolled CPKC412A2104E1 participant receiving midostaurin at the time of the approval of amendment 4. Participants were treated until the time of disease progression.
>Trial ID >Title >Status >Phase >Start Date >Summary

Clinical Trial Conditions for Midostaurin

Condition Name

Condition Name for Midostaurin
Intervention Trials
Acute Myeloid Leukemia 20
Myelodysplastic Syndrome 5
Untreated Adult Acute Myeloid Leukemia 4
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Condition MeSH

Condition MeSH for Midostaurin
Intervention Trials
Leukemia, Myeloid, Acute 37
Leukemia, Myeloid 34
Leukemia 34
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Clinical Trial Locations for Midostaurin

Trials by Country

Trials by Country for Midostaurin
Location Trials
United States 219
Italy 58
Japan 33
Germany 18
Spain 13
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Trials by US State

Trials by US State for Midostaurin
Location Trials
Massachusetts 15
California 14
Florida 9
Texas 9
Michigan 9
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Clinical Trial Progress for Midostaurin

Clinical Trial Phase

Clinical Trial Phase for Midostaurin
Clinical Trial Phase Trials
Phase 3 6
Phase 2/Phase 3 1
Phase 2 21
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Clinical Trial Status

Clinical Trial Status for Midostaurin
Clinical Trial Phase Trials
Completed 17
Recruiting 14
Active, not recruiting 6
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Clinical Trial Sponsors for Midostaurin

Sponsor Name

Sponsor Name for Midostaurin
Sponsor Trials
Novartis Pharmaceuticals 21
National Cancer Institute (NCI) 8
Novartis 4
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Sponsor Type

Sponsor Type for Midostaurin
Sponsor Trials
Other 47
Industry 34
NIH 8
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Midostaurin: Clinical Trials, Market Analysis, and Projections

Introduction to Midostaurin

Midostaurin, a tyrosine kinase inhibitor, has been a significant player in the treatment of acute myeloid leukemia (AML), particularly in patients with FLT3 mutations. Here, we will delve into the current clinical trials, market analysis, and future projections for this drug.

Clinical Trials Update

Ongoing Clinical Trials

One of the notable ongoing clinical trials is the global study evaluating the safety, efficacy, and pharmacokinetics of midostaurin in combination with standard chemotherapy in pediatric patients with newly diagnosed FLT3-mutated AML. This open-label, multi-center, single-arm study is divided into two parts:

  • Part 1: Focuses on defining the Recommended Phase 2 Dose (RP2D) of midostaurin. Patients are treated in cohorts, starting with a dose of 30mg/m² twice daily, and if well-tolerated, the dose is escalated to 60mg/m² twice daily[1].

  • Part 2: Evaluates the safety, tolerability, and efficacy of midostaurin at the confirmed RP2D. The treatment regimen includes two induction blocks, three consolidation blocks, followed by 12 cycles of post-consolidation therapy with midostaurin[1].

Previous Clinical Trials

Previous studies, such as the RATIFY trial and its follow-up, have shown promising results. The RATIFY study demonstrated that 59% of patients who received midostaurin achieved complete remission (CR)[4].

A subsequent phase 3b trial extended midostaurin treatment from 14 to 21 days and introduced variations in standard combination chemotherapy dosing. This study assessed the safety and efficacy of midostaurin plus chemotherapy in both young and older patients with FLT3-mutated AML. The results supported the efficacy of midostaurin in combination with chemotherapy regimens[4].

Market Analysis

Current Market Size

The midostaurin drugs market has seen significant growth in recent years. As of 2023, the global market size was valued at USD 1.2 billion[3].

Market Growth Projections

The market is expected to grow at a Compound Annual Growth Rate (CAGR) of 7.3% from 2024 to 2031, reaching a projected value of USD 1.9 billion by 2031. This growth is driven by several factors:

  • Growing Cancer Prevalence: The increasing frequency of FLT3-mutated AML and associated malignancies has heightened the demand for midostaurin[3].

  • Regulatory Approvals: Broader approvals and indications for midostaurin have expanded its market potential[3].

  • Research Advancements: Ongoing clinical trials and studies confirming the effectiveness of midostaurin continue to fuel its market growth[3].

  • Better Diagnostics: Improved diagnostic techniques have made it easier to identify appropriate patients, thereby increasing the use of midostaurin[3].

Regional and Segment Analysis

The midostaurin market is analyzed at the global, regional, and country levels. The market report includes segment analysis by type, application, and region, providing a comprehensive overview of the market dynamics[2][5].

Competitive Landscape

Novartis is a key player in the midostaurin market, given its role in developing and marketing the drug. The competitive landscape also includes other pharmaceutical companies involved in the development of tyrosine kinase inhibitors[5].

Market Projections

Forecasted Market Size

Despite some reports suggesting a decline in market size to USD 103.3 million by 2030 with a CAGR of -3.0% from 2024 to 2030, the more robust and widely accepted projection indicates a growth trajectory. The market is expected to reach USD 1.9 billion by 2031, driven by the factors mentioned above[3][5].

Market Trends and Drivers

The market is driven by several trends and drivers, including:

  • Technological Trends: Advances in diagnostic techniques and personalized medicine are enhancing the use of midostaurin[2].

  • ESG Analysis: Environmental, social, and governance factors are increasingly influencing market dynamics and investor decisions[2].

  • Consumer Behavior: Growing awareness and positive patient outcomes are contributing to the drug's popularity[3].

"Growing Cancer Prevalence: There is a greater need for midostaurin due to the increased frequency of FLT3-mutated AML and associated malignancies."[3]

Challenges and Opportunities

Challenges

Despite the positive outlook, the midostaurin market faces several challenges, including:

  • Regulatory Hurdles: Strict regulatory environments can impact the approval and marketing of the drug[2].

  • Competition: The presence of other tyrosine kinase inhibitors in the market can pose competition to midostaurin[5].

Opportunities

The market also presents several opportunities:

  • Expanding Indications: Potential for midostaurin to be approved for other malignancies beyond FLT3-mutated AML[3].

  • Emerging Markets: Growing healthcare infrastructure in emerging markets offers new avenues for market expansion[2].

Key Takeaways

  • Clinical Trials: Ongoing trials are focusing on defining the optimal dose and evaluating the safety and efficacy of midostaurin in pediatric patients with FLT3-mutated AML.
  • Market Growth: The midostaurin market is projected to grow significantly, reaching USD 1.9 billion by 2031, driven by growing cancer prevalence, regulatory approvals, and research advancements.
  • Market Drivers: Technological trends, ESG factors, and positive consumer behavior are key drivers of the market.
  • Challenges and Opportunities: The market faces regulatory hurdles and competition but also offers opportunities for expanding indications and entering emerging markets.

FAQs

What is midostaurin used for?

Midostaurin is primarily used to treat acute myeloid leukemia (AML) in patients with FLT3 mutations.

What are the current clinical trials for midostaurin?

Current clinical trials include a global study evaluating the safety, efficacy, and pharmacokinetics of midostaurin in combination with standard chemotherapy in pediatric patients with newly diagnosed FLT3-mutated AML.

What is the projected market size for midostaurin by 2031?

The midostaurin market is expected to reach USD 1.9 billion by 2031, growing at a CAGR of 7.3% from 2024 to 2031.

What are the key drivers of the midostaurin market?

Key drivers include growing cancer prevalence, regulatory approvals, research advancements, and better diagnostic techniques.

Who is the main player in the midostaurin market?

Novartis is a key player in the development and marketing of midostaurin.

Sources

  1. Novartis Clinical Trials: "A Global Study of Midostaurin in Combination With Chemotherapy to Evaluate the Safety, Efficacy and Pharmacokinetics in Pediatrics Patients With Newly Diagnosed FLT3-Mutated Acute Myeloid Leukemia."
  2. Cognitive Market Research: "Midostaurin Market Report 2024 (Global Edition)."
  3. Market Research Intellect: "Midostaurin Drugs Market Size, Share & Trends [2031]."
  4. Blood Advances: "Midostaurin plus daunorubicin or idarubicin for young and older patients with FLT3-mutated acute myeloid leukemia."
  5. Valuates Reports: "Midostaurin Drugs - Market, Report Size, Worth, Revenue, Growth."
Last updated: 2025-01-01

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