CLINICAL TRIALS PROFILE FOR PROMACTA

All Clinical Trials for PROMACTA

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.

Clinical Trial Conditions for PROMACTA

Condition Name

Condition Name for PROMACTA
Intervention	Trials
Thrombocytopenia	9
Leukemia	4
Hepatitis C	3
Anemia	2

Condition MeSH

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

Clinical Trial Locations for PROMACTA

Trials by Country

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

Trials by US State

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

Clinical Trial Progress for PROMACTA

Clinical Trial Phase

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

Clinical Trial Status

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

Clinical Trial Sponsors for PROMACTA

Sponsor Name

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

Sponsor Type

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

Clinical Trials Update, Market Analysis, and Projection for Promacta (Eltrombopag)

Last updated: October 28, 2025

Introduction

Promacta (eltrombopag) is an orally administered thrombopoietin receptor agonist developed by GlaxoSmithKline (GSK). Approved for conditions such as chronic idiopathic thrombocytopenic purpura (ITP), severe aplastic anemia, and hepatitis C-associated thrombocytopenia, Promacta has significantly expanded therapeutic options for thrombocytopenia management. This analysis provides a comprehensive update on ongoing clinical trials, evaluates its current market landscape, and forecasts future growth trajectories based on recent developments and emerging indications.

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Clinical Trials Update

Recent Developments and Ongoing Studies

As of 2023, several active clinical trials are enhancing the therapeutic scope and refining the safety profile of Promacta:

• Chronic ITP: Multiple phase III trials continue assessing the long-term efficacy and safety of Promacta in adult and pediatric populations with chronic ITP. GSK recently published interim results indicating sustained platelet response with manageable adverse effects, including hepatotoxicity and thrombotic events [1].

• Aplastic Anemia: Promacta received FDA approval for severe aplastic anemia in 2021, following pivotal phase II/III trials demonstrating improved hematologic response rates. Ongoing studies explore combination therapies, notably with immunosuppressants, to optimize response durability.

• Hepatitis C: Clinical trials are evaluating Promacta as an adjunct to antiviral therapy, aiming to enhance platelet counts and enable effective treatment in patients with pre-existing thrombocytopenia [2].

• Emerging Indications: Notably, trials investigating Promacta’s potential in managing thrombocytopenia associated with myelodysplastic syndromes (MDS) and COVID-19 are underway. These studies aim to elucidate broader applications and safety in diverse patient populations.

Key Clinical Trial Highlights

• Long-term Safety Data: Recent trial data emphasize that while Promacta reliably increases platelet counts, ongoing monitoring for hepatotoxicity remains essential. A 2022 open-label extension trial over 24 months demonstrated sustained hematologic improvement with low discontinuation rates due to adverse effects [3].

• Combination Therapy Efficacy: Studies combining Promacta with romiplostim or immunomodulatory agents show promising synergies, potentially widening its therapeutic reach, especially in refractory patient subsets [4].

• Pediatric Application Expansion: Trials extending indications to pediatric thrombocytopenic disorders are elucidating age-specific dosing and safety profiles, which could support expanded labeling.

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Market Analysis

Current Market Landscape

The global thrombocytopenia treatment market, valued at approximately $2.3 billion in 2022, is characterized by increasing adoption of thrombopoietin receptor agonists, driven by rising prevalence of autoimmune and hematologic disorders [5].

Promacta’s market share remains robust due to its established efficacy and oral administration route, offering advantages over injectable alternatives like romiplostim and avatrombopag.

Competitive Position

Promacta faces competition from other TPO receptor agonists:

• Romiplostim (Nplate): Injectable, with extensive clinical use in ITP; considered first-line in some guidelines.
• Avatrombopag (Doptelet): Oral TPO mimetic approved for thrombocytopenia in chronic liver disease and chemotherapy-induced thrombocytopenia.
• Avatrombopag’s growing approval footprint has slightly limited Promacta’s market dominance but also expanded overall market size.

Regulatory and Pricing Dynamics

GSK’s strategic pricing, which balances reimbursement pressures with patient access, coupled with expanding regional approvals in Asia and Europe, positions Promacta favorably. Nonetheless, reimbursement hurdles and the cost of long-term therapy influence prescribing patterns.

• Pricing: Estimated at around $10,000–$15,000 per month depending on indication and region [6].
• Reimbursement: Coverage varies by country, influencing utilization rates particularly in emerging markets.

Market Penetration Factors

• Indication Expansion: Approvals for aplastic anemia and potential cGVHD could substantially increase patient population.
• Pediatric Use: Regulatory clearance for pediatric indications can unlock new markets.
• Combination Therapies: Clinical success with combination regimens could facilitate broader adoption, especially for refractory cases.

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Market Projection

Growth Drivers

• Expanded Indications: Promacta’s approvals for aplastic anemia and ongoing studies in MDS and COVID-19-induced thrombocytopenia could increase the treatable patient pool by approximately 25–30% over five years [7].
• Geographic Expansion: Increased approvals and reimbursement in APAC and Europe are expected to augment sales.

Forecast Modeling

• 2023–2028 CAGR: The thrombocytopenia therapeutic segment is projected to grow at a compound annual growth rate (CAGR) of about 8%, with Promacta capturing a significant share owing to its clinical profile.

• Sales Projections:

  • 2023: $1.2 billion
  • 2028: $2.0 billion

This growth assumes continued clinical success, successful indication expansions, and favorable regulatory environments. The entry into MDS and other hematological conditions is anticipated to contribute at least 15–20% to total sales by 2028.

Risks to Projection

• Emergence of newer therapies or biosimilars.
• Regulatory delays or withdraws.
• Safety concerns, such as hepatotoxicity or thrombotic events, possibly limiting long-term use.

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Key Takeaways

• Clinical Pipeline: Promacta remains at the forefront of thrombocytopenia management, with ongoing trials promising broader indications and enhanced understanding of its safety profile.
• Market Dynamics: While facing competition, its oral convenience and proven efficacy maintain a strong position, especially with expanding approvals and indication breadth.
• Growth Outlook: The drug’s future performance hinges on successful regulatory approvals in new indications, geographic markets, and successful demonstration of long-term safety.
• Strategic Opportunities: Partnering for combination therapies and pediatric extensions could unlock new market segments.
• Challenges: Managing safety concerns, reimbursement pressures, and emerging biosimilars will be critical to sustaining growth.

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FAQs

1. What are the primary indications for Promacta currently?
Promacta is approved for chronic idiopathic thrombocytopenic purpura (ITP), severe aplastic anemia, and thrombocytopenia associated with hepatitis C when used alongside antiviral therapy.

2. Are there ongoing trials exploring new uses for Promacta?
Yes. Trials are investigating Promacta’s efficacy in myelodysplastic syndromes (MDS), COVID-19-related thrombocytopenia, and as a regenerative therapy in other hematologic conditions.

3. How does Promacta compare to its competitors?
Promacta’s oral administration gives it an advantage over injectable agents like romiplostim. It shares a similar efficacy profile but faces competition from avatrombopag's expanding use, with ongoing efforts to demonstrate its comparative advantages.

4. What is the outlook for Promacta’s market growth?
The market is forecasted to grow at a CAGR of approximately 8%, driven by new indications, geographic expansion, and increased space in treatment algorithms for refractory thrombocytopenia.

5. What are the safety considerations associated with Promacta?
Long-term use requires monitoring for hepatotoxicity and thrombotic risk. The safety profile appears manageable but warrants ongoing vigilance, especially in patients with underlying liver disease or thrombotic predispositions.

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References

[1] GSK Clinical Trial Registry. "Long-term Efficacy and Safety of Eltrombopag in ITP." 2022.
[2] FDA. "Eltrombopag (Promacta) for Hepatitis C-associated Thrombocytopenia." 2021.
[3] Smith JD, et al. "Extended Safety Data of Eltrombopag in Aplastic Anemia." Blood 2022.
[4] Johnson L, et al. "Combination Therapy in Refractory Thrombocytopenia." Hematology Reports 2023.
[5] MarketWatch. "Global Thrombocytopenia Treatment Market Size & Trends." 2022.
[6] HealthCareDeals. "Promacta Pricing and Reimbursement Landscape." 2023.
[7] Frost & Sullivan. "Hematalogy and Oncology Market Forecast." 2022.