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.
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.
NCT01000051 ↗	Eltrombopag for Post Transplant Thrombocytopenia	Completed	Novartis Pharmaceuticals	Phase 2	2010-02-17	The goal of this clinical research study is to learn if eltrombopag can help to improve platelet counts in patients with low platelets after they have had a stem cell transplant. The safety of this drug will also be studied.

Clinical Trial Conditions for promacta

Condition Name

Condition Name for promacta
Intervention	Trials
Thrombocytopenia	9
Leukemia	4
Hepatitis C	3
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities	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
Canada	10
Germany	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	5

Sponsor Type

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

Last updated: April 27, 2026

Promacta (eltrombopag): Clinical Trial Update, Market Analysis, and 2025-2030 Projection

What is Promacta and what claims does the current evidence support?

Promacta is eltrombopag, an oral thrombopoietin receptor agonist (TPO-RA) developed for thrombocytopenia in multiple clinical settings. Commercially, Promacta is positioned around durable platelet response, reduced need for transfusion in select patient groups, and established long-term safety in practice.

Core approved use areas (US label framework)

• Chronic immune thrombocytopenia (ITP) in adults and pediatric patients aged 1 year and older (with persistent or chronic disease).
• Severe aplastic anemia (SAA) in patients who have had an insufficient response to immunosuppressive therapy (ESA).
• Chronic hepatitis C (CHC)–associated thrombocytopenia to allow initiation and maintenance of antiviral therapy (historically key; more constrained now due to curative DAAs).

Mechanism and differentiation Eltrombopag activates the TPO receptor (c-Mpl), driving megakaryocyte maturation and platelet production. As a class, TPO-RAs compete primarily on response durability, dose titration burden, thromboembolic risk mitigation, and switching patterns after inadequate response.

Commercial implication The market is increasingly shaped by: (1) chronic ITP growth and treatment algorithm selection, (2) TPO-RA switching and sequencing, (3) shrinking CHC-era demand due to DAAs, and (4) payer preference for established products with predictable platelet response patterns.

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What does the clinical trial landscape show right now for Promacta?

A complete, current “last-trial-update” view requires trial-level timelines across multiple registries. Under the operational constraints here, only the items with durable public identification and alignment to the current evidence base can be stated as confirmed clinical positioning.

Clinical development theme: durability across hematologic thrombocytopenias Across Promacta’s broad indication set, the clinical evidence that has historically supported use in practice is anchored to:

• Chronic ITP: response rates and durable platelet maintenance under long-term dosing; dose titration protocols aimed at avoiding excessive platelet counts.
• SAA: response in patients inadequately responsive to prior immunosuppression, where eltrombopag is used to stimulate hematopoietic recovery.
• CHC: enabling thrombocytopenia management to support antiviral therapy initiation and maintenance (now structurally less important post-DAA scale-up).

How this shows up in trial endpoints and regulatory framing Trial endpoints repeatedly emphasize:

• Platelet response (threshold-based and duration-based).
• Time to stable response and the feasibility of individualized dose titration.
• Safety metrics tied to thrombosis risk, liver enzyme monitoring, and bleeding/thrombotic events.

Practical “current” interpretation

• For chronic ITP, Promacta remains clinically validated in treatment algorithms where patients need reliable platelet control, including after prior therapies.
• For SAA, it remains clinically positioned as an ESA-like adjunct in selected refractory patients, where durable platelet and hematologic recovery matter.
• For CHC, demand is no longer trial-driven in the same way due to reduced need for DAA-era antiviral thrombocytopenia management.

Key evidence anchors Promacta’s modern label and clinical credibility rest on pivotal randomized and long-term studies in chronic ITP and refractory SAA and on earlier CHC studies. The U.S. prescribing information provides the regulatory backbone for efficacy and safety positioning. [1]

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Where does Promacta sit in the competitive market for thrombocytopenia care?

Promacta competes in multiple overlapping markets:

• Chronic ITP: TPO-RAs are the main class competitors (eltrombopag vs avatrombopag vs romiplostim). Brand positioning emphasizes dose titration control, platelet response stability, and clinician familiarity.
• Refractory SAA: competition is indirect against other hematologic interventions and supportive strategies; TPO-RA adoption depends on patient selection and response durability.
• CHC-era thrombocytopenia: competition is largely irrelevant now due to DAA adoption. Where residual demand exists, it is driven by historical treatment patterns rather than active new patient cohorts.

Competitive “switching logic” Real-world switching typically follows:

• Inadequate platelet response at tolerable doses
• Loss of response over time
• Safety signals that push clinicians toward alternative agents or schedule adjustments

Promacta’s established safety monitoring profile (platelet targets, liver enzymes, thrombotic precautions) supports continued use where prescribers seek predictable management frameworks. [1]

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How large is the addressable market and what is driving it?

Addressable market components

1. Chronic ITP patient base
   • Driven by prevalence and incidence of autoimmune thrombocytopenia requiring second-line and later management.
2. Refractory SAA
   • Smaller cohort size than ITP but clinically high-urgency where hematologic recovery is critical.
3. CHC-associated thrombocytopenia
   • Structurally smaller now due to DAA dominance.

Demand drivers

• Sustained chronic ITP need across years and treatment lines.
• Algorithm entrenchment of TPO-RAs as a core therapy after steroids/IVIG/other immunomodulators.
• Long-term tolerability frameworks that reduce real-world friction to continuation.
• Payer and guideline adoption favoring agents with extensive post-approval experience.

Demand headwinds

• CHC cohort contraction due to DAA era.
• Class competition and incremental substitution among TPO-RAs.
• Dose titration intensity and monitoring burden that affect uptake at payers and in practice settings.

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What is the projected market trajectory for Promacta (2025-2030)?

A hard projection requires a defined baseline (current revenue, patient counts, geography, and payer mix). Under the constraints here, only a structured directional projection can be produced without inventing numbers.

Projected trajectory (directional)

• 2025-2027: stable to modest growth driven by chronic ITP maintenance, with incremental substitution inside the TPO-RA class.
• 2028-2030: mature demand profile with growth limited by saturation of treated chronic ITP populations and continued class competition. Any growth tends to come from penetration in refractory segments and incremental SAA uptake.

Assumption set implicit in the projection

• CHC-driven demand remains subdued.
• Promacta retains core share within chronic ITP where clinician familiarity and dosing manageability matter.
• Competition shifts from “access expansion” to “share capture” among TPO-RAs.

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What are the key commercialization risks and levers?

Levers

• Chronic ITP durability narrative supported by long-term experience and label-based monitoring protocols. [1]
• SAA positioning as an option after insufficient response to prior immunosuppression. [1]
• Stable manufacturing and established supply chain for a mature oncology-adjacent hematology brand.

Risks

• TPO-RA class substitution if payers favor lower-cost alternatives or if response profiles differ by patient subtype.
• Safety-driven switching: thrombosis risk management and platelet over-shoots can drive dose changes and discontinuations. [1]
• Regulatory or labeling changes not visible in this constrained update set could alter patient eligibility.

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Key Takeaways

• Promacta (eltrombopag) is a mature TPO-RA with confirmed clinical and regulatory positioning across chronic ITP, refractory SAA, and historically CHC-associated thrombocytopenia. [1]
• The market growth engine is chronic ITP, with CHC demand structurally constrained by DAA adoption, leaving TPO-RA class competition as the main share-shaping force.
• 2025-2030 trajectory is directional: stable to modest growth through chronic ITP penetration and maintenance, with limited upside as the category matures and substitution persists.

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FAQs

1. Is Promacta still relevant for CHC-associated thrombocytopenia?
   Its role is smaller in the DAAs era; the label exists, but the clinical population needing CHC-era thrombocytopenia management is reduced.

2. What patient groups drive Promacta demand today?
   The largest driver is chronic ITP requiring sustained platelet control, with refractory SAA as a secondary driver.

3. How does Promacta compete versus other TPO-RAs?
   It competes on long-term dosing management, platelet response durability, and clinician familiarity within chronic ITP treatment algorithms.

4. What are the main safety considerations affecting use?
   Platelet count overshoot and thrombotic risk mitigation, with ongoing monitoring requirements described in the prescribing information. [1]

5. What is the main limitation on Promacta growth through 2030?
   Category maturity and ongoing substitution within TPO-RAs, plus reduced CHC-era demand.

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References

[1] U.S. Food and Drug Administration. (n.d.). PROMACTA (eltrombopag) prescribing information. FDA. https://www.accessdata.fda.gov/