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CLINICAL TRIALS PROFILE FOR DOPTELET

All Clinical Trials for doptelet

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT04312789 ↗	Avatrombopag for the Treatment of Thrombocytopenia After Donor Hematopoietic Stem Cell Transplant	Recruiting	Dova Pharmaceuticals	Phase 2	2020-08-10	This phase II trial studies the side effects and how well avatrombopag works for the treatment of thrombocytopenia after donor hematopoietic stem cell transplant. Thrombocytopenia is defined as abnormally low level of platelets in the blood. Avatrombopag is a small molecule thrombopoietin receptor agonist which stimulates thrombopoietin receptor leading to increase production of platelets.
NCT04312789 ↗	Avatrombopag for the Treatment of Thrombocytopenia After Donor Hematopoietic Stem Cell Transplant	Recruiting	National Cancer Institute (NCI)	Phase 2	2020-08-10	This phase II trial studies the side effects and how well avatrombopag works for the treatment of thrombocytopenia after donor hematopoietic stem cell transplant. Thrombocytopenia is defined as abnormally low level of platelets in the blood. Avatrombopag is a small molecule thrombopoietin receptor agonist which stimulates thrombopoietin receptor leading to increase production of platelets.
NCT04312789 ↗	Avatrombopag for the Treatment of Thrombocytopenia After Donor Hematopoietic Stem Cell Transplant	Recruiting	Basem William, MD	Phase 2	2020-08-10	This phase II trial studies the side effects and how well avatrombopag works for the treatment of thrombocytopenia after donor hematopoietic stem cell transplant. Thrombocytopenia is defined as abnormally low level of platelets in the blood. Avatrombopag is a small molecule thrombopoietin receptor agonist which stimulates thrombopoietin receptor leading to increase production of platelets.
NCT04312789 ↗	Avatrombopag for the Treatment of Thrombocytopenia After Donor Hematopoietic Stem Cell Transplant	Recruiting	Ohio State University Comprehensive Cancer Center	Phase 2	2020-08-10	This phase II trial studies the side effects and how well avatrombopag works for the treatment of thrombocytopenia after donor hematopoietic stem cell transplant. Thrombocytopenia is defined as abnormally low level of platelets in the blood. Avatrombopag is a small molecule thrombopoietin receptor agonist which stimulates thrombopoietin receptor leading to increase production of platelets.
NCT04516967 ↗	Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months	Recruiting	Dova Pharmaceuticals	Phase 3	2021-03-02	A Phase 3b Study to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects with Immune Thrombocytopenia for ≥6 Months
NCT04845659 ↗	Avatrombopaq In Patients With Cirrhosis	Not yet recruiting	Hvidovre University Hospital	Phase 4	2021-06-01	Thrombocytopenia is a frequent issue in patients with cirrhosis undergoing various types of procedures (e.g. liver biopsy, endoscopy and minor surgical interventions). Thrombocytopenia < 50*10^9/L increases the risk of perioperative and postoperative bleedning and might prevent patients with cirrhosis to undergo important procedures. Doptelet is a small molecular trombopoetin agonist, which results in proliferation and differentiation of megakaryocytes in the bone marrow resulting in increased levels of thrombocytes. It is given orally as a pill and is used to increase platelet count in patients with severe thrombocytopenia (< 50*10^9/L) and cirrhosis and thus not to normalize platelet count. This study investigates the safety and efficacy of Doptelet in patients with cirrhosis and thrombocytopenia (< 50*10^9/L) undergoing minor procedures like Transjugular Adjusted Liver Biopsy (TJALB) and gastroscopy.
NCT04915287 ↗	Efficacy of Avatrombopag in Thrombocytopenic Patients With Chronic Liver Disease Undergoing an Elective Procedure	Recruiting	Chinese PLA General Hospital	Phase 4	2021-06-06	In this study, investigators aimed to evaluate the efficacy of Avatrombopag in thrombocytopenic patients with chronic liver disease undergoing an elective invasive procedure through a prospective, non-randomized controlled, multicenter clinical trial. The patients were non-randomly assigned to the Avatrombopag group (119 patients) and the conventional treatment group (357 patients). The primary endpoint was the proportion of patients not requiring prophylactic platelet transfusion or rescue therapy due to bleeding from grouping up to 10 days post-procedure. Second endpoints included the proportion of patients achieving a platelet count of ≥50x10^9/L and the mean change in platelet count from baseline at the time before the procedure, the proportion of patients requiring platelet transfusion and the mean platelet transfusion units per capita, the incidence of bleeding events (WHO≥2 and requiring rescue therapy), the imaging evaluations of bleeding events, the incidence of adverse events, the changes in life quality between two groups before and after treatment, and the pharmacoeconomic index of two groups.

Clinical Trial Conditions for doptelet

Condition Name

Condition Name for doptelet
Intervention	Trials
Immune Thrombocytopenia	3
Thrombocytopenia; Drugs	2
Platelet Disorder	1
PLT After Allogenic Hematopoietic Stem Cell Transplantation	1

Condition MeSH

Condition MeSH for doptelet
Intervention	Trials
Thrombocytopenia	6
Purpura, Thrombocytopenic, Idiopathic	3
Liver Diseases	1
Liver Cirrhosis	1

Clinical Trial Locations for doptelet

Trials by Country

Trials by Country for doptelet
Location	Trials
United States	17
Turkey	4
France	2
Poland	2
Germany	2

Trials by US State

Trials by US State for doptelet
Location	Trials
Ohio	2
Pennsylvania	1
North Carolina	1
New Jersey	1
Nevada	1

Clinical Trial Progress for doptelet

Clinical Trial Phase

Clinical Trial Phase for doptelet
Clinical Trial Phase	Trials
Phase 4	2
Phase 3	3
Phase 2/Phase 3	1

Clinical Trial Status

Clinical Trial Status for doptelet
Clinical Trial Phase	Trials
Not yet recruiting	3
Recruiting	3
Withdrawn	1

Clinical Trial Sponsors for doptelet

Sponsor Name

Sponsor Name for doptelet
Sponsor	Trials
Dova Pharmaceuticals	3
National Cancer Institute (NCI)	1
Basem William, MD	1

Sponsor Type

Sponsor Type for doptelet
Sponsor	Trials
Other	5
Industry	4
NIH	1

Doptelet Market Analysis and Financial Projection

Doptelet (Avatrombopag): A Comprehensive Overview of Clinical Trials, Market Analysis, and Projections

Introduction

Doptelet, also known as avatrombopag, is a second-generation, once-daily, orally administered thrombopoietin (TPO) receptor agonist. It has been approved for the treatment of thrombocytopenia in adult patients with chronic liver disease (CLD) who are scheduled to undergo a procedure, as well as for the treatment of immune thrombocytopenia (ITP). Here, we delve into the clinical trials, market analysis, and future projections for Doptelet.

Clinical Trials Overview

Approval and Key Trials

Doptelet was approved by the FDA in 2019 for the treatment of ITP based on the results of a pivotal phase 3 clinical trial (NCT01438840)[1].

• Trial Design: The multicenter, randomized, double-blind, placebo-controlled study evaluated the efficacy and safety of Doptelet in adult patients with ITP over a 6-month period. Patients received Doptelet at a starting dose of 20 mg once daily, with dose adjustments allowed to a maximum of 40 mg or a minimum of 5 mg, according to the platelet count response[1].

• Primary Endpoint: The primary endpoint was the cumulative number of weeks of platelet response, defined as a platelet count ≥50×10^9/L without the need for rescue therapy for bleeding. Doptelet significantly outperformed the placebo, achieving a median of 12.4 weeks of platelet response versus 0 weeks with the placebo[1].

• Secondary Endpoints: Secondary endpoints included the platelet response rate at day 8 and reductions in the use of concomitant ITP medications. By day 8, Doptelet produced a markedly higher platelet response rate than the placebo (65.6% vs 0%)[1].

Long-Term Efficacy and Safety

The clinical trial included an optional open-label extension phase where all participants received Doptelet to assess longer-term safety and efficacy. During this phase, the observed platelet response rate was maintained up to week 36. A post hoc analysis revealed that 55.2% of patients treated with Doptelet in the core phase and 52.3% treated in both the core and extension phases consistently maintained their response without losing it[1].

Adverse Events

Treatment with Doptelet was generally well-tolerated, with a safety profile comparable to that of the placebo. The most common adverse event reported was headache. However, Doptelet has been associated with thrombotic and thromboembolic complications, such as portal vein thrombosis in patients with chronic liver disease and arterial or venous thromboembolic events in patients with ITP[1].

Market Analysis

Current Market Scenario

Doptelet is approved in several major markets, including the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan. The drug has been granted approval from the China National Medical Products Administration (NMPA) for the treatment of thrombocytopenia in adult patients with CLD who are scheduled to undergo a procedure[3].

Market Forecast

The market scenario for Doptelet is expected to change significantly due to extensive research in the treatment of indicated conditions and incremental healthcare spending worldwide. This is anticipated to expand the market size, enabling drug manufacturers to penetrate deeper into the market. Historical and forecasted sales data from 2017 to 2030 indicate a growing demand for Doptelet, driven by its efficacy and the increasing need for effective thrombocytopenia treatments[3][5].

Market Competitors and Emerging Therapies

Doptelet faces competition from other approved products for the treatment of thrombocytopenia and ITP. The launch of late-stage emerging therapies in the near future is expected to significantly impact the market. Companies and academics are working on novel approaches to treat and improve the disease condition, which could influence Doptelet's market dominance[3][5].

Regulatory Milestones and Development Activities

The regulatory milestones and development activities for Doptelet include detailed product descriptions, mechanism of action, dosage and administration, and pharmacological studies. The drug's market details across major markets, along with SWOT analysis and analyst views, provide a comprehensive picture of its market position[3][5].

Mechanism of Action and Dosage

Mechanism of Action

Doptelet is designed to mimic the effects of TPO, the primary regulator of normal platelet production. It acts as a TPO receptor agonist, stimulating the production of platelets in the bone marrow[2][5].

Dosage and Administration

Patients receive Doptelet at a starting dose of 20 mg once daily, with dose adjustments allowed to a maximum of 40 mg or a minimum of 5 mg, based on the platelet count response. This flexibility in dosing helps in achieving and maintaining the target platelet count range of 50 to 100×10^9/L[1].

Safety and Efficacy

Safety Profile

While Doptelet is generally well-tolerated, it has been associated with significant safety concerns, including thrombotic and thromboembolic complications. Patients with chronic liver disease should not be administered Doptelet to normalize platelet counts due to the risk of portal vein thrombosis[1][2].

Efficacy

The clinical trials have demonstrated Doptelet's efficacy in increasing and sustaining platelet counts. The drug has shown a quick onset of action, with a significantly higher platelet response rate compared to the placebo. Additionally, Doptelet treatment has resulted in a decrease in the use of other ITP medications in some patients[1].

Market Projections

Future Market Trends

The market for Doptelet is expected to grow due to increasing healthcare spending and the need for effective treatments for thrombocytopenia. The drug's approval in various regions and its inclusion in treatment guidelines will further drive its market penetration. However, the emergence of new therapies and competition from existing treatments will be key factors influencing its market position[3][5].

Sales Forecast

Historical and forecasted sales data indicate a steady growth in the sales of Doptelet from 2017 to 2030. This growth is supported by the drug's efficacy, safety profile, and the expanding market for thrombocytopenia treatments[3][5].

Key Takeaways

• Clinical Efficacy: Doptelet has demonstrated significant efficacy in clinical trials, particularly in increasing and sustaining platelet counts in patients with ITP and CLD.
• Market Growth: The market for Doptelet is projected to grow due to increasing healthcare spending and the need for effective thrombocytopenia treatments.
• Safety Concerns: Despite its efficacy, Doptelet is associated with thrombotic and thromboembolic complications, necessitating careful patient selection and monitoring.
• Regulatory Approvals: Doptelet has received approvals in several major markets, including the US, EU5, Japan, and China.
• Competitive Landscape: The drug faces competition from other approved products and emerging therapies, which will influence its market dominance.

FAQs

What is the primary mechanism of action of Doptelet?

Doptelet acts as a TPO receptor agonist, mimicking the effects of thrombopoietin to stimulate platelet production in the bone marrow[2][5].

What are the approved indications for Doptelet?

Doptelet is approved for the treatment of thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure and for the treatment of immune thrombocytopenia (ITP)[1][3].

What are the common adverse reactions associated with Doptelet?

The most common adverse event reported is headache. However, Doptelet is also associated with thrombotic and thromboembolic complications, such as portal vein thrombosis and arterial or venous thromboembolic events[1].

How is Doptelet administered?

Doptelet is administered orally once daily, with a starting dose of 20 mg that can be adjusted to a maximum of 40 mg or a minimum of 5 mg based on the platelet count response[1].

What is the expected market growth for Doptelet from 2017 to 2030?

The market for Doptelet is expected to grow due to increasing healthcare spending and the need for effective thrombocytopenia treatments. Historical and forecasted sales data indicate a steady growth in sales over this period[3][5].

References

1. DOPTELET® (avatrombopag). Highlights of prescribing information. AkaRx. Revised July 2024. Accessed November 28, 2024.
2. Global Doptelet (Avatrombopag) Industry to 2030 - Drug Insight and Market Forecast - ResearchAndMarkets.com. Business Wire, August 13, 2020.
3. Doptelet (Avatrombopag) - Drug Insight and Market Forecast - 2030. GlobeNewswire, August 26, 2020.
4. Avatrombopag (Doptelet) - Canada's Drug Agency. Canada's Drug Agency, 2024.
5. Doptelet (Avatrombopag): A 2nd Generation, Once Daily, Orally Administered TPO Receptor Agonist Approved for the Treatment of Thrombocytopenia - Global Drug Insight and Market Forecast - 2030. GlobeNewswire, December 30, 2020.