CLINICAL TRIALS PROFILE FOR DROXIA

All Clinical Trials for DROXIA

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01389024 ↗	Hydroxyurea to Prevent Brain Injury in Sickle Cell Disease	Active, not recruiting	Children's Hospital of Philadelphia	Phase 2	2011-10-01	This is a pilot study of hydroxyurea versus placebo to reduce central nervous system complications (abnormally fast blood flow to the brain, silent cerebral infarct or stroke) in young children with sickle cell disease. The investigators plan to identify children 12 to 48 months old without central nervous system complications and randomly assign 20 to treatment with hydroxyurea and 20 to treatment with placebo for 36 months. Neither the study doctors nor the participants will know which treatment they are receiving.
NCT01389024 ↗	Hydroxyurea to Prevent Brain Injury in Sickle Cell Disease	Active, not recruiting	Children's Mercy Hospital Kansas City	Phase 2	2011-10-01	This is a pilot study of hydroxyurea versus placebo to reduce central nervous system complications (abnormally fast blood flow to the brain, silent cerebral infarct or stroke) in young children with sickle cell disease. The investigators plan to identify children 12 to 48 months old without central nervous system complications and randomly assign 20 to treatment with hydroxyurea and 20 to treatment with placebo for 36 months. Neither the study doctors nor the participants will know which treatment they are receiving.
NCT00354913 ↗	Imatinib Mesylate and Hydroxyurea in Treating Patients With Recurrent or Progressive Meningioma	Completed	National Cancer Institute (NCI)	Phase 2	2005-05-01	RATIONALE: Imatinib mesylate may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as hydroxyurea, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving imatinib mesylate together with hydroxyurea may kill more tumor cells. PURPOSE: This phase II trial is studying how well giving imatinib mesylate together with hydroxyurea works in treating patients with recurrent or progressive meningioma.
NCT00354913 ↗	Imatinib Mesylate and Hydroxyurea in Treating Patients With Recurrent or Progressive Meningioma	Completed	Novartis Pharmaceuticals	Phase 2	2005-05-01	RATIONALE: Imatinib mesylate may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as hydroxyurea, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving imatinib mesylate together with hydroxyurea may kill more tumor cells. PURPOSE: This phase II trial is studying how well giving imatinib mesylate together with hydroxyurea works in treating patients with recurrent or progressive meningioma.
NCT00354913 ↗	Imatinib Mesylate and Hydroxyurea in Treating Patients With Recurrent or Progressive Meningioma	Completed	Duke University	Phase 2	2005-05-01	RATIONALE: Imatinib mesylate may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as hydroxyurea, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving imatinib mesylate together with hydroxyurea may kill more tumor cells. PURPOSE: This phase II trial is studying how well giving imatinib mesylate together with hydroxyurea works in treating patients with recurrent or progressive meningioma.

Clinical Trial Conditions for DROXIA

Condition Name

Condition Name for DROXIA
Intervention	Trials
Sickle Cell Disease	5
Sickle Cell Anemia	3
Head and Neck Squamous Cell Carcinoma	2
Stroke	2

Condition MeSH

Condition MeSH for DROXIA
Intervention	Trials
Anemia, Sickle Cell	7
Anemia	3
Squamous Cell Carcinoma of Head and Neck	2
Head and Neck Neoplasms	2

Clinical Trial Locations for DROXIA

Trials by Country

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Trials by Country for DROXIA
Location	Trials
United States	33
Nigeria	1
Congo, The Democratic Republic of the	1
Brazil	1
Congo	1

Trials by US State

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−

Trials by US State for DROXIA
Location	Trials
North Carolina	3
Tennessee	3
Missouri	3
Alabama	2
Texas	2

Clinical Trial Progress for DROXIA

Clinical Trial Phase

Clinical Trial Phase for DROXIA
Clinical Trial Phase	Trials
Phase 3	2
Phase 2	7
Phase 1/Phase 2	2

Clinical Trial Status

Clinical Trial Status for DROXIA
Clinical Trial Phase	Trials
Recruiting	3
Active, not recruiting	3
Terminated	3

Clinical Trial Sponsors for DROXIA

Sponsor Name

Sponsor Name for DROXIA
Sponsor	Trials
National Cancer Institute (NCI)	4
National Heart, Lung, and Blood Institute (NHLBI)	3
St. Jude Children's Research Hospital	3

Sponsor Type

Sponsor Type for DROXIA
Sponsor	Trials
Other	59
NIH	10
Industry	2

Clinical Trials, Market Analysis, and Projections for DROXIA (Hydroxyurea) in Sickle Cell Disease Treatment

Introduction

DROXIA, the brand name for hydroxyurea, is a crucial medication in the management of sickle cell disease (SCD), particularly for reducing the frequency of painful crises and the need for blood transfusions. Here, we will delve into the latest clinical trials, market analysis, and future projections for DROXIA.

Clinical Trials Update

PIVOT Clinical Trial

A significant recent development is the PIVOT clinical trial, which aimed to evaluate the benefits of hydroxyurea in patients with the HbSC variant of sickle cell disease. The trial showed promising results, with participants receiving hydroxyurea experiencing improvements in several laboratory measures of SCD severity. These improvements included higher volumes of larger red blood cells, higher fetal hemoglobin levels, and lower leukocyte and neutrophil counts. Additionally, the trial reported reductions in the rate of vaso-occlusive crises and hospitalizations compared to the placebo group[1].

Safety and Efficacy

While the PIVOT trial highlighted the efficacy of hydroxyurea, it also noted a number of mild and transient side effects that exceeded the goal related to dose-limiting side effects. However, researchers emphasized that these side effects should not prevent the progression to a larger Phase III study[1].

Market Analysis

Current Market Size and Growth

The global sickle cell disease treatment market, which includes pharmacotherapy with drugs like DROXIA, has been growing rapidly. In 2023, the market size was approximately $2.85 billion and is expected to reach $7.84 billion by 2030, growing at a CAGR of 15.51%[2].

U.S. Market

In the U.S., the sickle cell disease treatment market was valued at $1.44 billion in 2022 and is projected to grow at a CAGR of 17.3% during the forecast period. The pharmacotherapy segment, which includes hydroxyurea, is expected to grow at the fastest CAGR due to the approval and commercialization of new pipeline drugs[5].

Treatment Modalities

The market is segmented into bone marrow transplant, blood transfusion, and pharmacotherapy. The pharmacotherapy segment, particularly hydroxyurea and other branded products like Adakveo and Oxbryta, is driving the market growth. Hydroxyurea remains a cornerstone in the pharmacotherapy segment due to its established efficacy in reducing painful crises and the need for blood transfusions[5].

Market Projections

Future Growth Drivers

The growth of the sickle cell disease treatment market, including the demand for DROXIA, is driven by several factors:

• Technological Advancements: Advances in genetic research and a deeper understanding of the disease pathophysiology are leading to more effective treatments.
• Government and Non-Government Funding: Increased funding for R&D initiatives is crucial for developing new therapies.
• Emerging Therapies: Gene therapy solutions and personalized medicine approaches are expected to provide durable cures rather than just symptomatic relief[2].

Challenges and Opportunities

Despite the positive outlook, there are challenges such as the high treatment cost, which can limit the adoption of these drugs. However, the approval and commercialization of new pipeline drugs, such as Global Blood Therapeutics’ Inclacumab, are expected to fuel the market growth in the coming years[5].

Key Attributes of DROXIA

Indications and Usage

DROXIA is indicated to reduce the frequency of painful crises and the need for blood transfusions in patients with sickle cell anemia with recurrent moderate to severe painful crises[4].

Dosage and Administration

The initial dose of DROXIA is 15 mg/kg once daily, with the dose adjusted based on blood count monitoring. The maximum tolerated dose is 35 mg/kg/day, and the dose should be reduced or discontinued if toxic blood counts are observed[4].

Safety Considerations

DROXIA can cause hemolytic anemia, malignancies, and fetal harm. Patients must be monitored for blood counts throughout treatment, and effective contraception is advised for patients of reproductive potential[4].

Expert Insights

"It’s exciting that we have four terrific studies designed to benefit people with SCD, including treatments that have been available but haven’t been used in certain populations, and potential new therapies," said Charles Abrams, MD, a prominent hematologist from the University of Pennsylvania[1].

Key Takeaways

• Clinical Trials: The PIVOT trial demonstrated the efficacy of hydroxyurea in reducing SCD severity and vaso-occlusive crises.
• Market Growth: The global and U.S. sickle cell disease treatment markets are expected to grow significantly, driven by technological advancements and new drug approvals.
• Treatment Modalities: Pharmacotherapy, particularly hydroxyurea, remains a key segment driving market growth.
• Challenges: High treatment costs and side effects are challenges that need to be addressed.
• Future Outlook: Emerging therapies, including gene therapy and personalized medicine, are expected to enhance long-term treatment outcomes.

FAQs

What is the primary indication for DROXIA in sickle cell disease treatment?

DROXIA is indicated to reduce the frequency of painful crises and the need for blood transfusions in patients with sickle cell anemia with recurrent moderate to severe painful crises[4].

What are the key findings from the PIVOT clinical trial on hydroxyurea?

The PIVOT trial showed significant improvements in laboratory measures of SCD severity and reductions in vaso-occlusive crises and hospitalizations among participants receiving hydroxyurea[1].

How is the global sickle cell disease treatment market expected to grow?

The global sickle cell disease treatment market is expected to grow at a CAGR of 15.51%, reaching $7.84 billion by 2030 from $2.85 billion in 2023[2].

What are the main drivers of the sickle cell disease treatment market growth?

The growth is driven by technological advancements in genetic research, a deeper understanding of disease pathophysiology, and increased governmental and non-governmental funding for R&D initiatives[2].

What are the potential challenges in the adoption of DROXIA and other pharmacotherapy drugs?

High treatment costs and potential side effects are significant challenges that can limit the adoption of these drugs[5].

Are there any emerging therapies that could impact the market for DROXIA?

Yes, emerging therapies such as gene therapy solutions and personalized medicine approaches are expected to provide durable cures and enhance long-term treatment outcomes for sickle cell disease[2].

Sources

1. Hydroxyurea May Help People With HbSC Form of Sickle Cell Disease - ScienceBlog, Cincinnati Children's.
2. Sickle Cell Disease Treatment Market Forecast Report 2025-2030 - BusinessWire.
3. Clinical Trials Market Size, Share, Trends and Revenue Forecast - MarketsandMarkets.
4. DROXIA U.S. Prescribing Information - Bristol Myers Squibb.
5. U.S. Sickle Cell Disease Treatment Market Analysis [2030] - Fortune Business Insights.