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.

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
Myelodysplastic Syndromes	6
Preleukemia	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

PROMACTA: A Comprehensive Overview of Clinical Trials, Market Analysis, and Projections

Last updated: January 4, 2025

Introduction to PROMACTA

PROMACTA, also known as eltrombopag, is a small-molecule thrombopoietin (TPO) receptor agonist used to treat low platelet counts in various blood disorders, including chronic immune thrombocytopenia (ITP), severe aplastic anemia (SAA), and Hepatitis C-related thrombocytopenia. Here, we delve into the clinical trials, market analysis, and future projections for this significant therapeutic agent.

Clinical Trials and Efficacy

EXTEND Study

The EXTEND study, an open-label extension of four previous trials, is one of the most comprehensive studies on PROMACTA. This study enrolled 302 adults with chronic/persistent ITP who had received prior therapy for their condition. The key findings include:

• Platelet Count Elevation: Median platelet counts were elevated to ≥50×10^9/L within two weeks of treatment, with nearly 70% of patients maintaining platelet counts of ≥30×10^9/L without rescue therapy for prolonged periods, reducing the overall risk of bleeding[1].
• Reduction in Concomitant Medications: More than one-third of patients permanently stopped one or more concomitant ITP medications, including corticosteroids, danazol, and azathioprine[1].
• Long-term Safety Profile: The study established a long-term safety profile for PROMACTA, with the most common adverse events being headache, nasopharyngitis, and upper respiratory tract infection. Thromboembolic events were reported in 6% of patients[1].

FDA Approvals and Additional Indications

PROMACTA has received FDA approval for several indications, including the treatment of severe aplastic anemia (SAA) in patients who have had an insufficient response to immunosuppressive therapy (IST)[3].

Mechanism of Action and Dosage

How PROMACTA Works

PROMACTA interacts with the transmembrane domain of the human TPO-receptor, initiating signaling cascades that induce the proliferation and differentiation of megakaryocytes from bone marrow progenitor cells. This results in an increase in platelet production, reducing the risk of bleeding in patients with thrombocytopenia[2].

Dosage and Administration

The initial dose of PROMACTA is typically 50 mg once daily for most adult and pediatric patients 6 years and older, and 25 mg once daily for pediatric patients aged 1-5 years. Dose adjustments are necessary to maintain a platelet count greater than or equal to 50×10^9/L, with specific considerations for patients with hepatic impairment and those of East-/Southeast Asian ancestry[2].

Market Analysis and Projections

Current Market Landscape

The global market for PROMACTA is driven by several key factors:

• Increasing Incidence of Platelet Disorders: More than 1 in 10,000 individuals are affected by chronic ITP alone, and the rising rates of Hepatitis C and other autoimmune diseases contribute to the expanding patient base[5].
• Healthcare Expenditure: Increased focus on enhancing treatment options for chronic diseases has led to more resources being allocated to rare and serious disorders like thrombocytopenia[5].
• Innovations in Treatment Delivery: New formulations such as dissolvable tablets and liquid suspensions have improved the administration of PROMACTA, particularly for pediatric and elderly patients[5].

Market Forecast

The market for PROMACTA is expected to grow significantly from 2023 to 2032, driven by:

• Rising Demand: The increasing awareness and diagnosis of thrombocytopenia, coupled with PROMACTA’s proven efficacy, have positioned the drug as a leading player in the market[5].
• Geographic Expansion: PROMACTA has been approved in over 100 countries, ensuring a broader patient base and heightened market penetration[5].
• Strategic Partnerships: Pharmaceutical companies are entering into licensing agreements with biotechnological firms to expand the research and development pipeline for thrombocytopenia treatments, boosting innovation and production capabilities[5].

Future Growth Opportunities

The future of PROMACTA looks promising due to several factors:

• New Indications: Ongoing clinical trials are exploring new indications for PROMACTA, such as its use in oncology-related thrombocytopenia, which could further expand the market[5].
• Combination Therapies: Researchers are investigating the potential of combining PROMACTA with other medications to enhance efficacy, which could open up new patient demographics[5].
• Government Policies and Regulatory Initiatives: Favorable government policies and regulatory initiatives aimed at improving access to advanced treatments are supporting the market growth of PROMACTA[5].

Key Takeaways

• Clinical Efficacy: PROMACTA has demonstrated long-term efficacy in maintaining platelet counts and reducing the need for concomitant medications in patients with chronic ITP.
• Market Growth: The global market for PROMACTA is expected to grow significantly due to increasing demand, innovations in treatment delivery, and strategic partnerships.
• Future Potential: Ongoing research into new indications and combination therapies, along with favorable regulatory initiatives, positions PROMACTA for continued market expansion.

FAQs

What is PROMACTA used for?

PROMACTA is used to treat low platelet counts in conditions such as chronic immune thrombocytopenia (ITP), severe aplastic anemia (SAA), and Hepatitis C-related thrombocytopenia.

How does PROMACTA work?

PROMACTA interacts with the TPO-receptor, initiating signaling cascades that induce the proliferation and differentiation of megakaryocytes, leading to an increase in platelet production.

What are the common side effects of PROMACTA?

Common side effects include headache, nasopharyngitis, upper respiratory tract infection, nausea, diarrhea, and increased liver enzymes.

Is PROMACTA approved for use in children?

Yes, PROMACTA is approved for use in pediatric patients, with different dosages recommended for children aged 1-5 years and those 6 years and older.

What is the market outlook for PROMACTA?

The market for PROMACTA is expected to grow significantly from 2023 to 2032, driven by increasing demand, innovations in treatment delivery, and strategic partnerships.

Sources

1. Novartis Press Release: "Novartis drug Promacta shows long-term disease control for chronic/persistent immune thrombocytopenia (ITP)" - Oct 18, 2017.
2. DelveInsight Report: "PROMACTA Drug Insight and Market Forecast - 2032".
3. GSK Press Release: "GSK's Promacta (eltrombopag) receives FDA approval of an additional indication" - Aug 26, 2014.
4. FDA Label: "Promacta - accessdata.fda.gov".
5. Market Research Intellect: "Promacta Market Size And Projection" - Sep 26, 2024.