CLINICAL TRIALS PROFILE FOR HYDREA

All Clinical Trials for HYDREA

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00336362 ↗	Evaluating the Safety of G-CSF Mobilization in Individuals With Beta Thalassemia Major	Completed	George Papanicolaou Hospital	N/A	2006-07-01	Beta thalassemia major is a serious genetic disease of the blood. Treatments are limited, and although a bone marrow transplant from a compatible donor can be curative, only a limited percentage of individuals with this disease have a matched donor available. A long-term goal of study researchers is to develop a gene transfer process as a method of curing beta thalassemia major. Gene transfer involves obtaining blood stem cells from an individual, adding a normal globin gene to the stem cells, and putting the cells back into the individual. Before gene transfer methods can be attempted in individuals with beta thalassemia major, a safe method of obtaining blood stem cells needs to be developed. The purpose of this study is to investigate the safety and feasibility of collecting peripheral blood stem cells (PBSC) from individuals with beta thalassemia major. Research participants will be given G-CSF (filgrastim) for several days to increase the number of stem cells in the blood, a process called "mobilization." After mobilization, participants will undergo a procedure called apheresis to remove the white blood cells. Researchers in the laboratory will purify the stem cells from the mixture and test methods of putting a normal globin gene into the stem cells. Half of the participants will receive hydroxyurea (HU) prior to G-CSF mobilization. HU is used in splenectomized patients to attempt to reduce the risk of clotting during mobilization. In non-splenectomized patients, HU is given in an attempt to decrease the size of the spleen.
NCT00336362 ↗	Evaluating the Safety of G-CSF Mobilization in Individuals With Beta Thalassemia Major	Completed	National Heart, Lung, and Blood Institute (NHLBI)	N/A	2006-07-01	Beta thalassemia major is a serious genetic disease of the blood. Treatments are limited, and although a bone marrow transplant from a compatible donor can be curative, only a limited percentage of individuals with this disease have a matched donor available. A long-term goal of study researchers is to develop a gene transfer process as a method of curing beta thalassemia major. Gene transfer involves obtaining blood stem cells from an individual, adding a normal globin gene to the stem cells, and putting the cells back into the individual. Before gene transfer methods can be attempted in individuals with beta thalassemia major, a safe method of obtaining blood stem cells needs to be developed. The purpose of this study is to investigate the safety and feasibility of collecting peripheral blood stem cells (PBSC) from individuals with beta thalassemia major. Research participants will be given G-CSF (filgrastim) for several days to increase the number of stem cells in the blood, a process called "mobilization." After mobilization, participants will undergo a procedure called apheresis to remove the white blood cells. Researchers in the laboratory will purify the stem cells from the mixture and test methods of putting a normal globin gene into the stem cells. Half of the participants will receive hydroxyurea (HU) prior to G-CSF mobilization. HU is used in splenectomized patients to attempt to reduce the risk of clotting during mobilization. In non-splenectomized patients, HU is given in an attempt to decrease the size of the spleen.
NCT00336362 ↗	Evaluating the Safety of G-CSF Mobilization in Individuals With Beta Thalassemia Major	Completed	University of Washington	N/A	2006-07-01	Beta thalassemia major is a serious genetic disease of the blood. Treatments are limited, and although a bone marrow transplant from a compatible donor can be curative, only a limited percentage of individuals with this disease have a matched donor available. A long-term goal of study researchers is to develop a gene transfer process as a method of curing beta thalassemia major. Gene transfer involves obtaining blood stem cells from an individual, adding a normal globin gene to the stem cells, and putting the cells back into the individual. Before gene transfer methods can be attempted in individuals with beta thalassemia major, a safe method of obtaining blood stem cells needs to be developed. The purpose of this study is to investigate the safety and feasibility of collecting peripheral blood stem cells (PBSC) from individuals with beta thalassemia major. Research participants will be given G-CSF (filgrastim) for several days to increase the number of stem cells in the blood, a process called "mobilization." After mobilization, participants will undergo a procedure called apheresis to remove the white blood cells. Researchers in the laboratory will purify the stem cells from the mixture and test methods of putting a normal globin gene into the stem cells. Half of the participants will receive hydroxyurea (HU) prior to G-CSF mobilization. HU is used in splenectomized patients to attempt to reduce the risk of clotting during mobilization. In non-splenectomized patients, HU is given in an attempt to decrease the size of the spleen.
NCT00127972 ↗	2NN & CHARM Long-Term Follow-up Study	Completed	Boehringer Ingelheim	Phase 4	2004-02-01	It is desirable to obtain extended follow up data on subjects who participated in the 2NN study and the CHARM study in order to see if the beneficial effect of using nevirapine continues up to 144 weeks of treatment.
NCT00127972 ↗	2NN & CHARM Long-Term Follow-up Study	Completed	International Antiviral Therapy Evaluation Center	Phase 4	2004-02-01	It is desirable to obtain extended follow up data on subjects who participated in the 2NN study and the CHARM study in order to see if the beneficial effect of using nevirapine continues up to 144 weeks of treatment.
NCT00023959 ↗	Bevacizumab, Fluorouracil, and Hydroxyurea Plus Radiation Therapy in Treating Patients With Advanced Head and Neck Cancer	Completed	National Cancer Institute (NCI)	Phase 1	2001-07-01	Monoclonal antibodies, such as bevacizumab, can block cancer growth in different ways. Some block the ability of cancer cells to grow and spread. Others find cancer cells and help kill them or deliver cancer-killing substances to them. Drugs used in chemotherapy work in different ways to stop tumor cells from dividing so they stop growing or die. Radiation therapy uses high-energy x-rays to damage tumor cells. Combining monoclonal antibody therapy with chemotherapy and radiation therapy may be an effective treatment for head and neck cancer. This phase I trial is to see if combining bevacizumab, fluorouracil, and hydroxyurea with radiation therapy works in treating patients who have advanced head and neck cancer

Clinical Trial Conditions for HYDREA

Condition Name

Condition Name for HYDREA
Intervention	Trials
Sickle Cell Anemia	7
Sickle Cell Disease	6
Stroke	4
Recurrent Salivary Gland Cancer	2

Condition MeSH

Condition MeSH for HYDREA
Intervention	Trials
Anemia, Sickle Cell	11
Carcinoma, Squamous Cell	5
Anemia	5
Carcinoma	5

Clinical Trial Locations for HYDREA

Trials by Country

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Trials by Country for HYDREA
Location	Trials
United States	47
Nigeria	4
France	2
Denmark	1
Congo, The Democratic Republic of the	1

Trials by US State

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Trials by US State for HYDREA
Location	Trials
North Carolina	6
Illinois	6
Tennessee	4
Texas	3
New York	3

Clinical Trial Progress for HYDREA

Clinical Trial Phase

Clinical Trial Phase for HYDREA
Clinical Trial Phase	Trials
Phase 4	1
Phase 3	5
Phase 2	11

Clinical Trial Status

Clinical Trial Status for HYDREA
Clinical Trial Phase	Trials
Completed	9
Active, not recruiting	6
Terminated	4

Clinical Trial Sponsors for HYDREA

Sponsor Name

Sponsor Name for HYDREA
Sponsor	Trials
National Cancer Institute (NCI)	8
National Heart, Lung, and Blood Institute (NHLBI)	4
Aminu Kano Teaching Hospital	4

Sponsor Type

Sponsor Type for HYDREA
Sponsor	Trials
Other	83
NIH	17
Industry	4

Hydrea (Hydroxyurea): Clinical Trials, Market Analysis, and Projections

Introduction to Hydrea (Hydroxyurea)

Hydrea, known chemically as hydroxyurea, is a medication that has been widely used in the treatment of various medical conditions, including sickle cell disease and certain types of cancer. Here, we will delve into the latest clinical trials, market analysis, and projections for this versatile drug.

Clinical Trials Update

PIVOT Clinical Trial for HbSC Sickle Cell Disease

A significant recent development in the clinical use of hydroxyurea is the PIVOT clinical trial. This Phase II trial, conducted in Ghana, aimed to evaluate the benefits of hydroxyurea for individuals with the hemoglobin SC (HbSC) variant of sickle cell disease. The trial showed promising results, with participants receiving hydroxyurea experiencing improvements in several laboratory measures of disease 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 vaso-occlusive crises and hospitalizations among both child and adult participants compared to those on placebo[1].

H-PRIME Trial in Uganda

Another ongoing trial is the H-PRIME study in Uganda, which is investigating the best way to treat sickle cell disease in children. This trial compares high and low doses of hydroxyurea, given pragmatically without extensive laboratory monitoring, which is often impractical in low-income settings. The trial also includes comparisons of different antimalarial and antimicrobial regimens to address common causes of early death among children with sickle cell disease in sub-Saharan Africa. The H-PRIME trial aims to recruit 1,800 children and will follow up participants until January 2028[4].

Market Analysis

Market Size and Forecast

The hydroxyurea market has been growing steadily and is projected to continue this trend. As of 2023, the market size was valued at USD 1.2 billion and is expected to reach USD 2 billion by 2031, growing at a Compound Annual Growth Rate (CAGR) of 5% from 2024 to 2031. This growth is driven by several factors, including the rising prevalence of sickle cell disease and cancer, regulatory approvals for new indications, increased awareness among healthcare providers and patients, and the availability of generic versions[2].

Regional Market Dynamics

The hydroxyurea market is segmented into various geographical regions, each with its unique dynamics. North America, particularly the United States, holds a prominent position due to the high incidence of sickle cell disease and certain cancers, along with advanced healthcare infrastructure and significant research and development investments. Europe, especially countries like Germany and France, also represents a critical market due to sophisticated healthcare policies and growing awareness of rare diseases. The Asia-Pacific region is emerging as a lucrative market, driven by the rise in incidence rates of hematological conditions and increased government funding for healthcare initiatives. Other regions, such as the Middle East and Africa, and Latin America, present both challenges and opportunities, with a focus on improving access to treatment amidst varied economic conditions[2].

Key Players

The hydroxyurea market is dominated by several major pharmaceutical companies, including Bristol-Myers Squibb Company, Teva Pharmaceutical Industries Ltd., Novartis AG, Pfizer Inc., Mylan N.V., Hikma Pharmaceuticals PLC, Amgen Inc., Merck & Co., Inc., Apotex Inc., and Fresenius Kabi AG. These companies are driving market growth through ongoing research and development, regulatory approvals, and strategic marketing efforts[2].

Drivers of Market Growth

Rising Prevalence of Sickle Cell Disease

The increasing global incidence of sickle cell disease is a significant driver of the hydroxyurea market. In the United States, about 25% to 30% of people with sickle cell disease have the HbSC variant, while in Ghana, this figure is closer to 50%. The need for effective management of this condition is driving demand for hydroxyurea[1].

Cancer Treatment Applications

Hydroxyurea is also used in the treatment of certain cancers, particularly myeloproliferative disorders. The growing incidence of cancer globally is boosting the market demand for hydroxyurea. For example, Bristol-Myers Squibb has been focusing on enhancing the clinical application of hydroxyurea in combination with newer targeted therapies and immunotherapies for advanced head and neck cancer[2][3].

Regulatory Approvals and Awareness

Regulatory approvals for new indications or formulations of hydroxyurea can expand its market presence. Increased awareness among healthcare providers and patients about the benefits of hydroxyurea is also leading to higher adoption rates. Generic availability of the drug makes it more accessible to various patient populations, further driving market growth[2].

Economic and Healthcare Infrastructure Factors

The cost-effectiveness of hydroxyurea compared to other treatments is a significant driver, especially in resource-limited settings. Advanced healthcare infrastructure in regions like North America and Europe also supports the market growth by facilitating research, development, and distribution of the drug[2].

Challenges and Opportunities

Access to Treatment in Low-Income Settings

One of the challenges in the hydroxyurea market is ensuring access to treatment in low-income settings. Trials like H-PRIME are addressing this issue by evaluating pragmatic dosing strategies that do not require extensive laboratory monitoring. This approach can make hydroxyurea more feasible in resource-limited areas[4].

Ongoing Research and Development

Ongoing clinical trials and research into additional therapeutic uses of hydroxyurea are expected to enhance market growth. The drug's potential in treating other conditions, beyond sickle cell disease and cancer, is being explored, which could further expand its market presence[2].

Key Takeaways

• Clinical Trials: Recent trials like PIVOT and H-PRIME are showing promising results for the use of hydroxyurea in managing sickle cell disease, particularly the HbSC variant.
• Market Growth: The hydroxyurea market is projected to grow from USD 1.2 billion in 2023 to USD 2 billion by 2031, driven by rising disease prevalence, regulatory approvals, and increased awareness.
• Regional Dynamics: North America, Europe, and the Asia-Pacific region are key markets, with varying dynamics influenced by local healthcare systems and disease prevalence.
• Drivers: The market is driven by the rising prevalence of sickle cell disease and cancer, regulatory approvals, awareness, generic availability, and economic factors.
• Challenges and Opportunities: Ensuring access to treatment in low-income settings and ongoing research into new therapeutic uses are critical for market growth.

FAQs

Q: What is the primary use of hydroxyurea in medical treatment?

A: Hydroxyurea is primarily used to manage sickle cell disease and certain types of cancer, including myeloproliferative disorders.

Q: What are the key findings of the PIVOT clinical trial?

A: The PIVOT trial showed that hydroxyurea significantly improved laboratory measures of SCD severity and reduced vaso-occlusive crises and hospitalizations in participants with the HbSC variant of sickle cell disease.

Q: How is the H-PRIME trial different from other hydroxyurea trials?

A: The H-PRIME trial is unique because it evaluates the use of hydroxyurea in children with sickle cell disease in a low-income setting, using pragmatic dosing strategies without extensive laboratory monitoring.

Q: What is the projected market size of hydroxyurea by 2031?

A: The hydroxyurea market is projected to reach USD 2 billion by 2031, growing at a CAGR of 5% from 2024 to 2031.

Q: Which regions are expected to drive the growth of the hydroxyurea market?

A: North America, Europe, and the Asia-Pacific region are expected to be key drivers of the hydroxyurea market, due to their advanced healthcare infrastructure and high incidence of relevant diseases.

Sources

1. ScienceBlog Cincinnati Children's: "Hydroxyurea May Help People With HbSC Form of Sickle Cell Disease"
2. Verified Market Research: "Hydroxyurea Market Trends, Size, & Forecast"
3. BioSpace: "Head and Neck Cancer Market Estimated to Reach USD 7,279.8 Million by 2034"
4. MRC Clinical Trials Unit at UCL: "New trial for sickle cell disease opens in Uganda"
5. Cognitive Market Research: "Hydroxyurea Market Report 2025 (Global Edition)"