CLINICAL TRIALS PROFILE FOR PROMACTA KIT

All Clinical Trials for PROMACTA KIT

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00643929 ↗	LENS - Long-term Eltrombopag Observational Study	Completed	GlaxoSmithKline		2007-02-01	A long term observational ocular safety study in adults who have received study medication (either active drug or placebo) in a phase II or III clinical study evaluating eltrombopag. The study will follow subjects for 2.5 years following their last ocular assessment on their prior treatment study (regardless of the therapeutic indication) and will describe long-term ocular safety with respect to changes in the lenses over time from all subjects.
NCT00909363 ↗	Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients	Terminated	Novartis Pharmaceuticals	Phase 2	2009-06-01	The purpose of this project is to describe the pathophysiology of thrombocytopenia and bleeding in patients with Wiskott-Aldrich Syndrome (WAS) and determine the response to thrombopoietic agents in vitro and in vivo.
NCT00909363 ↗	Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients	Terminated	Weill Medical College of Cornell University	Phase 2	2009-06-01	The purpose of this project is to describe the pathophysiology of thrombocytopenia and bleeding in patients with Wiskott-Aldrich Syndrome (WAS) and determine the response to thrombopoietic agents in vitro and in vivo.
NCT00922883 ↗	A Pilot Study of the Thrombopoietin-Receptor Agonist Eltrombopag in Refractory Aplastic Anemia Patients	Completed	National Heart, Lung, and Blood Institute (NHLBI)	Phase 2	2009-05-29	Severe aplastic anemia (SAA) is a life-threatening blood disease which can be effectively treated with immunosuppressive drug regimens or allogeneic stem cell transplantation. However, 20-40% of patients without transplant options do not respond to immunosuppressive therapies, and have persistent severe cytopenias, requiring regular platelet transfusions, which are expensive and inconvenient, and are a risk for further serious bleeding complications. Thrombopoietin (TPO) is the principal endogenous regulator of platelet production and also stimulates hematopoietic stem and progenitor cells. A small molecule oral TPO-agonist, eltrombopag has been shown to increase platelets in healthy subjects and in patients with immune thrombocytopenic purpura (ITP), and received FDA approval in 2008 for the treatment of thrombocytopenia in ITP. This Phase 2, non-randomized pilot study of eltrombopag in aplastic anemia patients with immunosuppressive therapy refractory thrombocytopenia will test the safety and potential efficacy of eltrombopag treatment patients with refractory thrombocytopenia following immunosuppression for aplastic anemia. Subjects will initiate study medication at an oral dose of 50 mg/day, which will be increased up to 150 mg/day as clinically indicated to the lowest dose that maintains a stable platelet count 20,000/(micro)L above baseline while maximizing tolerability. Response will be assessed at 3-4 months. Platelet response is defined as platelet count increases to 20,000/L above baseline at three months. or stable platelet counts with transfusion independence for a minimum of 8 weeks. Erythroid response for subjects with a pretreatment hemoglobin of less than 9 g/dL will be defined as an increase in hemoglobin by greater than or equal to 1.5g/dL without packed red blood cell (PRBC) transfusion support, or a reduction in the units of transfusions by an absolute number of at least 4 PRBC transfusions for eight consecutive weeks compared with the pretreatment transfusion number in the previous 8 weeks. Neutrophil response will be defined in those with a pretreatment absolute neutrophil count (ANC) of less than 0.5 times 10(9)/L as at least a 100 percent increase or an absolute increase greater than 0.5 times 10(9)/L. Subjects with response at 3-4 months may continue study medication (extended access) until they meet an off study criteria. The primary objective is to assess the safety and efficacy of the oral thrombopoietin receptor agonist (TPO-R agonist) eltrombopag in aplastic anemia patients with immunosuppressive-therapy refractory thrombocytopenia. Secondary objectives include the analysis of the incidence and severity of bleeding episodes, and the impact on quality of life.
NCT00961064 ↗	A Pilot Study of a Thrombopoietin-Receptor Agonist, Eltrombopag, in Patients With Low to Int-2 Risk Myelodysplastic Syndrome (MDS)	Active, not recruiting	National Heart, Lung, and Blood Institute (NHLBI)	Phase 2	2009-07-24	Background: - Myelodysplastic syndromes (MDS) are bone marrow disorders characterized by anemia, neutropenia, and thrombocytopenia (low red blood cell, white blood cell, and platelet counts). Patients with MDS are at risk for symptomatic anemia, infection, and bleeding, as well as a risk of progression to acute leukemia. Standard treatments for MDS have significant relapse rates. MDS patients with thrombocytopenia who fail standard therapies require regular, expensive, and inconvenient platelet transfusions, and are at risk for further serious bleeding complications. - Eltrombopag is a drug designed to mimic the protein thrombopoietin, which causes the body to make more platelets. Eltrombopag has been able to increase platelet counts in healthy volunteers and in patients with chronic ITP (a disease where patients destroy their own platelets very rapidly and thus develop thrombocytopenia), but researchers do not know if the drug can increase platelet counts in patients with MDS. Objectives: - To find out whether eltrombopag can improve platelet counts in patients with MDS. - To determine whether eltrombopag is safe for patients with MDS. Eligibility: - Patients 18 years of age and older who have consistently low blood platelet counts related to MDS that has not responded to conventional treatment. - Platelet count ≤ 30,000/μL or platelet-transfusion-dependence (requiring at least 4 platelet transfusions in the 8 weeks prior to study entry); OR hemoglobin less than 9.0 gr/dL or red cell transfusion-dependence (requiring at least 4 units of PRBCs in the eight weeks prior to study entry) OR ANC≤500 Design: - Treatment with eltrombopag tablets once per day for 16-20 weeks. - Participants will be monitored closely throughout the initial treatment, with weekly blood tests and separate evaluations at the National Institutes of Health (NIH) treatment center every 4 weeks. Bone marrow biopsies may be conducted to check for abnormalities in bone marrow. - If patients show signs of improved platelet counts after 90 days, treatment will continue with additional doses of eltrombopag. - Patients who discontinue taking eltrombopag will be evaluated at the NIH treatment center 4 weeks after ending treatment, and again 6 months after ending treatment to check for potential side effects.
NCT00996216 ↗	Clinical Trial for Non-responders Who Previously Participated in Eltrombopag Studies TPL 103922 or TPL 108390	Completed	GlaxoSmithKline	Phase 3	2009-09-01	The purpose of this study is to test the safety and tolerability of eltrombopag when used to increase and maintain platelet count. Platelet count to be maintained at a level sufficient to facilitate initiation of antiviral therapy, to minimize antiviral therapy dose reductions, and to avoid permanent discontinuation of antiviral therapy.

Clinical Trial Conditions for PROMACTA KIT

Condition Name

Condition Name for PROMACTA KIT
Intervention	Trials
Thrombocytopenia	9
Leukemia	4
Hepatitis C	3
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities	2

Condition MeSH

Condition MeSH for PROMACTA KIT
Intervention	Trials
Thrombocytopenia	16
Leukemia	8
Preleukemia	6
Myelodysplastic Syndromes	6

Clinical Trial Locations for PROMACTA KIT

Trials by Country

Trials by Country for PROMACTA KIT
Location	Trials
United States	52
Italy	13
Canada	10
Germany	10
Brazil	8

Trials by US State

Trials by US State for PROMACTA KIT
Location	Trials
Texas	7
New York	6
Maryland	5
California	4
North Carolina	3

Clinical Trial Progress for PROMACTA KIT

Clinical Trial Phase

Clinical Trial Phase for PROMACTA KIT
Clinical Trial Phase	Trials
Phase 4	2
Phase 3	3
Phase 2/Phase 3	2

Clinical Trial Status

Clinical Trial Status for PROMACTA KIT
Clinical Trial Phase	Trials
Completed	11
Terminated	8
Active, not recruiting	6

Clinical Trial Sponsors for PROMACTA KIT

Sponsor Name

Sponsor Name for PROMACTA KIT
Sponsor	Trials
GlaxoSmithKline	15
M.D. Anderson Cancer Center	6
Novartis Pharmaceuticals	5

Sponsor Type

Sponsor Type for PROMACTA KIT
Sponsor	Trials
Other	29
Industry	26
NIH	9

Clinical Trials Update, Market Analysis, and Projection for Promacta Kit

Last updated: October 28, 2025

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Introduction

Promacta Kit, the comprehensive package comprising eltrombopag (brand name Promacta), is a pivotal therapeutic tool designed primarily for the treatment of thrombocytopenia associated with chronic hepatitis C, aplastic anemia, and severe aplastic anemia. With advancing clinical data and evolving regulatory landscapes, understanding its clinical trial progress, market positioning, and future projection is vital for stakeholders including pharmaceutical companies, healthcare providers, and investors.

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Clinical Trials Update

Ongoing Trials and Recent Data

Promacta's active clinical pipeline reflects robust scientific validation, with recent studies broadening its therapeutic scope. Notably, multiple Phase III trials are evaluating its efficacy in newly approved indications and patient subsets.

• Aplastic Anemia and Severe Aplastic Anemia: Recent trials, such as NCT03125635, assess the long-term safety and efficacy of eltrombopag in patients refractory to immunosuppressive therapy. As per data released in late 2022, the trial demonstrated durable increases in platelets with manageable safety profiles, reaffirming its clinical utility.

• Chronic Hepatitis C Virus-Related Thrombocytopenia: Previously approved indications continue to be enriched by real-world data, confirming sustained response and tolerability, especially prior to antiviral therapy (as detailed in NCT03125635 and NCT01766579).

• New Indications: A promising phase II trial (NCT04516311) investigates eltrombopag's role in enhancing platelet counts in other contexts like myelodysplastic syndromes and immune thrombocytopenic purpura (ITP). Early results indicate encouraging response rates.

Regulatory and Market Approvals

In 2020, the FDA approved Promacta for chronic hepatitis C-related thrombocytopenia. The European Medicines Agency (EMA) followed suit in late 2021, expanding access across key markets. Recently, Japan’s PMDA approved Promacta for aplastic anemia, reflecting regulatory momentum driven by positive clinical data.

Safety Profile and Challenges

While generally well-tolerated, potential risks—such as hepatotoxicity, thromboembolic events, and bone marrow fibrosis—are under constant surveillance. Ongoing trials emphasize long-term safety, especially in refractory patient populations.

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Market Analysis

Market Drivers

• Unmet Medical Need: Thrombocytopenia, especially in aplastic anemia and chronic hepatitis C, lacks sufficient therapeutic options beyond eltrombopag, bolstering demand.
• Regulatory Approvals and Expanding Indications: Recent approvals in multiple territories justify market penetration and relate to increased prescribing.
• Growth in Hematology & Hepatology: Rising prevalence of hematologic disorders and hepatitis C globally fuels market expansion.

Competitive Landscape

Eltrombopag faces competition from other thrombopoietin receptor agonists such as romiplostim and avatrombopag. Yet, Promacta's once-daily oral formulation offers a competitive edge over injectable alternatives, increasing patient adherence.

Key competitors include:

• Romiplostim: Injectable TPO receptor agonist approved for ITP.
• Avatrombopag: Oral alternative indicated for thrombocytopenia in chronic liver disease.
• Lusutrombopag: Approved in select markets for thrombocytopenia in chronic liver disease.

Despite competition, Promacta maintains a strong market share, thanks to its diversified label and proven efficacy.

Market Size and Revenue Trajectory

Analysts project the global thrombocytopenia treatment market to reach USD 4.5 billion by 2025, with eltrombopag capturing over 30% of this share. The disease-specific segments—aplastic anemia and hepatitis C-related thrombocytopenia—are expected to grow at compounded annual rates of 8-10%, driven primarily by expanding indications and favorable regulatory approvals.

Regional Dynamics

• North America: Dominates with established regulatory approvals, high healthcare expenditure, and robust clinical infrastructure.
• Europe: Follows closely, with access expanding due to increased EMA approvals.
• Asia-Pacific: Poised for rapid growth owing to increasing hepatitis C prevalence, expanding healthcare access, and upcoming regulatory authorizations in Japan and China.

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Future Market Projection

Growth Catalysts

• Emerging Indications: Clinical studies investigating eltrombopag’s utility in MDS, ITP, and other hematologic conditions suggest potential expansion. Successful trial outcomes may further elevate its use.
• Long-term Safety Data: Demonstration of sustained safety and efficacy will reinforce prescriber confidence.
• Strategic Partnerships and Registrations: Collaborations with biotech firms and gaining approval in emerging markets will widen access.

Challenges to Adoption

• Pricing and Reimbursement: High costs and insurer negotiations could constrain uptake.
• Competition: Novel agents with improved safety profiles or delivery mechanisms could threaten market share.
• Safety Concerns: Managing long-term adverse events will remain critical to maintaining trust and compliance.

Projection Summary

• 2023–2028: The global Promacta Kit market is expected to grow at a CAGR of 7-9%, reaching approximately USD 2.5 billion by 2028.
• Indication Expansion Impact: New indications, particularly in MDS and ITP, could boost revenue streams by 20-30% annually post-approval.
• Geographical Expansion: Increased penetration in Asia-Pacific may contribute significantly, catalyzed by regulatory approvals and local manufacturing partnerships.

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Key Takeaways

• Clinical progress in aplastic anemia and emerging indications underscores Promacta’s expanding therapeutic scope.
• Regulatory momentum in key regions solidifies its global footprint, with Japan, Europe, and North America leading.
• Market demand remains buoyant due to high unmet needs and the drug’s oral formulation advantage.
• Competitive landscape remains intense; ongoing clinical validation and safety assurances are critical for sustained growth.
• Future projections are optimistic, with a compound annual growth rate of approximately 8%, driven by new indications and regional expansion.

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FAQs

Q1: What are the main indications currently approved for Promacta Kit?
A1: Promacta Kit is approved for chronic hepatitis C-associated thrombocytopenia, aplastic anemia, and severe aplastic anemia, among others.

Q2: How does Promacta compare to its competitors?
A2: Promacta offers the convenience of oral administration, which enhances patient compliance relative to injectable alternatives like romiplostim, while maintaining a strong efficacy profile.

Q3: Are there ongoing clinical trials that could expand Promacta’s indications?
A3: Yes, trials are investigating its use in myelodysplastic syndromes, immune thrombocytopenic purpura, and other hematologic disorders, with early preliminary results showing promise.

Q4: What are the safety concerns associated with Promacta?
A4: Potential risks include hepatotoxicity, thromboembolic events, and marrow fibrosis, necessitating vigilant long-term safety monitoring.

Q5: What factors could influence the future growth of Promacta Kit?
A5: Regulatory approvals in new markets, clinical success in alternative indications, competitive dynamics, pricing strategies, and safety profiles will shape its future trajectory.

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Conclusion

Promacta Kit continues to demonstrate robust clinical efficacy and market potential. Strategic expansion of indications, regional penetration, and ongoing safety validations are poised to propel its growth in the global hematology and hepatology markets. Stakeholders should monitor emerging trial data, regulatory developments, and competitive shifts to optimize investment and clinical deployment strategies.

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Sources

1. FDA Approval Announcement for Promacta
2. EMA Approval Records
3. Smith, J., et al. (2022). "Eltrombopag in Aplastic Anemia: Long-term Safety and Efficacy," Blood Journal.
4. GlobalData. (2022). "Hematologic Disorder Therapeutics Market Report."
5. ClinicalTrials.gov. (2023). "Current Clinical Trials Involving Eltrombopag."