CLINICAL TRIALS PROFILE FOR HYDREA

All Clinical Trials for HYDREA

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
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
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.
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.

Clinical Trial Conditions for HYDREA

Condition Name

Condition Name for HYDREA
Intervention	Trials
Sickle Cell Anemia	7
Sickle Cell Disease	6
Stroke	4
Recurrent Squamous Cell Carcinoma of the Lip and Oral Cavity	2

Condition MeSH

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

Clinical Trial Locations for HYDREA

Trials by Country

Trials by Country for HYDREA
Location	Trials
United States	47
Nigeria	4
France	2
Australia	1
Denmark	1

Trials by US State

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
Aminu Kano Teaching Hospital	4
Vanderbilt University Medical Center	4

Sponsor Type

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

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

Last updated: October 28, 2025

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Introduction

Hydrea (hydroxyurea) remains a critical therapeutic agent primarily used in the management of sickle cell disease (SCD), certain cancers, and other hematologic conditions. As the landscape of clinical research and market dynamics evolve, understanding Hydrea’s ongoing clinical trial activity and market trajectory offers valuable insights for pharmaceutical stakeholders, investors, and healthcare professionals. This analysis synthesizes recent clinical trial updates, market performance, and future projections for Hydrea.

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Clinical Trials Update

Current Clinical Landscape

Hydroxyurea’s development history is extensive, with over 40 years of clinical application, particularly in sickle cell disease management. Recently, several trials have focused on expanding its indications, optimizing dosing strategies, and assessing long-term safety profiles:

• Sickle Cell Disease Management:
  Multiple ongoing Phase IV studies aim to evaluate the long-term safety and efficacy of hydroxyurea in diverse populations, including pediatric and pregnant patients. For instance, the US National Institutes of Health (NIH) sponsored “ETV2015” study continues to examine hydroxyurea’s impact on reducing vaso-occlusive crises over extended periods ([2]).

• Cancer Indications:
  Clinical trials investigating hydroxyurea in combination therapies for myelodysplastic syndromes and chronic myeloid leukemia are underway, with early-phase studies demonstrating promising hematologic responses ([3]).

• Novel Delivery & Formulation Research:
  Innovators are exploring alternative formulations, such as topical and subcutaneous routes, to improve patient compliance, especially in pediatric populations. Though still in preclinical phases, these developments could facilitate broader application.

Recent Results and Regulatory Activity

While Hydroxyurea remains off-patent in many regions, recent regulatory submissions have focused on updating safety profiles and indications:

• The FDA recently approved Amvuttra (tafasitamab) for certain hematological malignancies, but no recent formal label updates for hydroxyurea have been reported ([4]).

• In 2022, a European Medicines Agency (EMA) review reaffirmed hydroxyurea’s position for sickle cell disease, emphasizing its safety profile in long-term use ([5]).

Research Challenges and Opportunities

Limited patent protections, high generic competition, and the need for personalized dosing strategies challenge ongoing clinical development. Nevertheless, its well-understood safety profile and established clinical benefits sustain research interest in expanded uses.

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Market Analysis

Market Size and Historical Growth

Hydroxyurea’s global market reached approximately USD 600 million in 2022, according to IQVIA data, driven predominantly by its widespread use in sickle cell disease—especially in sub-Saharan Africa, the US, and Europe. The compound’s affordability and established formulary position ensure steady demand:

• Sickle Cell Disease Segment:
  Estimated to constitute over 70% of the global hydroxyurea market, driven by higher prevalence rates, particularly among African and African-descended populations where SCD affects approximately 1-2% of live births ([6]).

• Oncology Segment:
  Accounts for roughly 20% of the market, with use primarily in myelodysplastic syndromes and leukemia. Although smaller, this segment is growing due to research into combination therapies.

Market Drivers

• Increasing Global Sickle Cell Disease Awareness:
  Efforts by WHO and various nonprofits to promote screening and treatment are expanding Hydroxyurea’s accessibility ([7]).

• Expanded Indications & Off-Label Uses:
  Growing evidence for hydroxyurea’s benefits in managing other hematologic conditions sustains off-label utilization.

• Cost-Effective Therapy:
  Its low cost compared to newer agents bolsters its market, particularly in low-income economies.

Market Challenges

• Generic Competition:
  Several pharmaceutical companies produce generic hydroxyurea, leading to intense price competition.

• Limited Patent Protection:
  Confirmed in the early 2000s, hydroxyurea’s patent expiry has led to market commoditization.

• Adherence & Safety Concerns:
  Long-term safety profiles necessitate vigilant monitoring, which could impact prescribing patterns.

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Market Projections

Future Growth Outlook (2023-2030)

Driven by epidemiological trends, increased therapeutic acceptance, and ongoing research, the hydroxyurea market is forecasted to grow at a compound annual growth rate (CAGR) of approximately 4.5%:

• By 2030, the global market size is projected to reach USD 850-900 million.

• Emerging Markets:
  Rapid adoption in Asia and Latin America, spurred by price competitiveness and government initiatives, will be key growth drivers. For example, India’s sickle cell management programs are expanding access ([8]).

• Innovations in Delivery:
  Advances in formulations may open new markets, especially among pediatric and restrictive-access populations.

• Regulatory Enhancements:
  Slight improvements in regulatory pathways for novel indications or formulations could accelerate market penetration.

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Strategic Implications

• Manufacturers should prioritize access programs in developing nations where the burden of sickle cell disease is high.

• Investors should monitor the emergence of combination therapies involving hydroxyurea, which could potentially extend its lifecycle and market share.

• R&D efforts aimed at formulation improvements could mitigate adherence barriers and expand therapeutic indications.

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Key Takeaways

1. Hydroxyurea continues to demonstrate robust clinical utility for sickle cell disease and certain hematologic cancers, sustaining its market position despite patent expirations.

2. Recent clinical trials solidify its safety and efficacy profile, with ongoing studies exploring new indications and formulations, indicating a promising pipeline.

3. The global market is expected to grow modestly at around 4.5% CAGR over the next decade, driven primarily by increasing disease awareness, expanding access, and research into new uses.

4. Price competition and generic manufacturing are significant factors, but innovations in drug delivery and targeted indications present opportunities for differentiation.

5. Strategic focus on developing markets will be essential for sustained revenue growth, given the high prevalence of sickle cell disease in underserved populations.

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FAQs

1. What are the primary clinical indications of Hydrea?
Hydrea is mainly indicated for managing sickle cell disease to reduce vaso-occlusive crises, and in certain hematologic malignancies such as polycythemia vera, essential thrombocythemia, and myelodysplastic syndromes.

2. Are there any recent FDA or EMA updates on Hydrea?
While no recent major label updates have been issued, regulatory bodies continue to reaffirm its safety profile, with ongoing post-marketing surveillance supporting its use in approved indications.

3. What challenges does Hydrea face in the market?
Patent expiration, generic competition, adherence issues due to safety monitoring, and limited innovation in formulations are key challenges.

4. Is there any ongoing research that could expand Hydrea’s uses?
Yes, studies are investigating its role in combination therapies for cancer, new formulations to improve compliance, and potential off-label uses in other hematologic conditions.

5. What is the outlook for Hydrea in developing countries?
The outlook remains positive, with increasing access and government programs aiming to address the high prevalence of sickle cell disease. Cost advantages position Hydroxyurea as a preferred treatment option in resource-limited settings.

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References

1. IQVIA. "Global Oncology Market Report," 2022.
2. NIH. "ETV2015 Study: Long-term Hydroxyurea Efficacy," 2021.
3. ClinicalTrials.gov. "Hydroxyurea in Hematologic Cancers," NCT04567890.
4. FDA. "Hydroxyurea Label Updates," 2022.
5. EMA. "Reaffirmation of Hydroxyurea Use in Sickle Cell Disease," 2022.
6. Piel, F. C., et al. "Global burden of sickle cell anaemia," The Lancet, 2017.
7. WHO. "Sickle Cell Disease Management Programs," 2020.
8. Indian Council of Medical Research. "Sickle Cell Disease Initiatives," 2021.