Hydroxyurea

Generic Name: Hydroxyurea

Brand Names: Droxia, Hydrea, Siklos, Xromi

Drug Type: Small Molecule

Chemical Formula: CH_₄N_₂O_₂

CAS Number: 127-07-1

Unique Ingredient Identifier: X6Q56QN5QC

Background: An antineoplastic agent that inhibits DNA synthesis through the inhibition of ribonucleoside diphosphate reductase.

Indication: Hydroxyurea is indicated to reduce the frequency of painful crises and to reduce the need for blood transfusions in adult and pediatric patients, 2 years of age and older, with sickle cell anemia with recurrent moderate to severe painful crises.

Associated Conditions: Chronic Myelogenous Leukemia (CML), Essential Thrombocythemia (ET), Head and Neck Primary Squamous Cell Carcinoma, Hypereosinophilic Syndrome (HES), Locally Advanced Squamous Cell Carcinomas of the Head and Neck (SCCHN), Meningiomas, Polycythemia Vera (PV), Sickle Cell Crisis, Vaso-occlusive Crisis

Associated Therapies: Chemoradiotherapy, Radiation Therapy

Clinical Trials

Ziftomenib for the Treatment of Patients With NPM1 Mutated or KMT2A Rearranged Acute Myeloid Leukemia Not Eligible for Standard Therapy

Phase 2

Not yet recruiting

Conditions: Acute Myeloid Leukemia

Interventions: Procedure: Biospecimen Collection
Procedure: Bone Marrow Aspiration
Procedure: Bone Marrow Biopsy
Drug: Cytarabine
Procedure: Echocardiography Test
Drug: Hydroxyurea
Procedure: Leukapheresis
Procedure: Multigated Acquisition Scan
Other: Questionnaire Administration
Drug: Ziftomenib

First Posted Date: 2025-04-16

Last Posted Date: 2025-04-16

Lead Sponsor: Uma Borate

Target Recruit Count: 70

Registration Number: NCT06930352

Locations: 🇺🇸
Ohio State University Comprehensive Cancer Center, Columbus, Ohio, United States

PASS of Xromi Comparing Safety and Effectiveness in Children Under 2 Years With Sickle Cell Disease [PRECISE PASS]

Recruiting

Conditions: Sickle Cell Disease

Interventions: Drug: Xromi

First Posted Date: 2025-04-11

Last Posted Date: 2025-05-16

Lead Sponsor: Nova Laboratories Limited

Target Recruit Count: 180

Registration Number: NCT06923111

Locations: 🇬🇧
Basildon University Hospital, Basildon, Essex, United Kingdom
🇬🇧
Noah's Ark Children's Hospital for Wales, Cardiff, Leicestershire, United Kingdom
🇬🇧
University College London Hospital, London, North London, United Kingdom

and more 2 locations

Genetically Engineered Cells (CD83 CAR T Cells) for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia

Phase 1

Not yet recruiting

Conditions: Recurrent Acute Myeloid Leukemia
Refractory Acute Myeloid Leukemia

Interventions: Drug: Autologous Anti-CD83 CAR T-cells
Procedure: Biospecimen Collection
Procedure: Chest Radiography
Procedure: Computed Tomography
Drug: Cyclophosphamide
Procedure: Echocardiography
Drug: Fludarabine Phosphate
Drug: Hydroxyurea
Procedure: Leukapheresis
Procedure: Lumbar Puncture
Procedure: Positron Emission Tomography
Other: Questionnaire Administration

First Posted Date: 2025-03-12

Last Posted Date: 2025-05-04

Lead Sponsor: Roswell Park Cancer Institute

Target Recruit Count: 26

Registration Number: NCT06871410

Locations: 🇺🇸
Roswell Park Cancer Institute, Buffalo, New York, United States

Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children With Sickle Cell Disease

Phase 2

Recruiting

Conditions: Sickle Cell Disease

Interventions: Drug: Hydroxycarbamid

First Posted Date: 2024-08-29

Last Posted Date: 2025-03-13

Lead Sponsor: Theravia

Target Recruit Count: 50

Registration Number: NCT06578507

Locations: 🇫🇷
GHEF- Site de Marne-la-Vallée, Jossigny, France
🇫🇷
Institut d'Hématologie et d'oncologie pédiatrique - IHOPe, Lyon, France
🇫🇷
Centre Hospitalier Intercommunal Créteil, Créteil, France

and more 3 locations

Stroke Prevention in Nigeria 2 Trial

Phase 4

Recruiting

Conditions: Sickle Cell Disease
Stroke

Interventions: Drug: Hydroxyurea

First Posted Date: 2024-07-29

Last Posted Date: 2025-02-25

Lead Sponsor: Vanderbilt University Medical Center

Target Recruit Count: 220

Registration Number: NCT06526117

Locations: 🇳🇬
Aminu Kano Teaching Hospital, Kano, Nigeria
🇳🇬
Murtala Muhammad Specialist Hospital, Kano, Nigeria

Bomedemstat vs Hydroxyurea for Essential Thrombocythemia (MK-3543-007)

Phase 3

Recruiting

Conditions: Essential Thrombocythemia

Interventions: Drug: Bomedemstat
Drug: Hydroxyurea
Drug: Hydroxyurea placebo
Drug: Bomedemstat placebo

First Posted Date: 2024-06-13

Last Posted Date: 2025-05-16

Lead Sponsor: Merck Sharp & Dohme LLC

Target Recruit Count: 300

Registration Number: NCT06456346

Locations: 🇪🇸
Clinica Universidad de Navarra-Hematology Department ( Site 0485), Madrid, Madrid, Comunidad De, Spain
🇪🇸
Hospital Universitario Ramón y Cajal-Hematology ( Site 0484), Madrid, Madrid, Comunidad De, Spain
🇪🇸
Hospital Costa del Sol-Hematology Service ( Site 0493), Marbella, Malaga, Spain

and more 111 locations

Comparison Of Efficacy And Safety Of Thalidomide Vs Hydroxyurea In Thalassemia Patients: A Single-Centre Pilot Study.

Phase 2

Completed

Conditions: Beta-Thalassemia

Interventions: Drug: Thalidomide 100mg
Drug: Hydroxyurea

First Posted Date: 2024-02-02

Last Posted Date: 2024-03-12

Lead Sponsor: National Institute of Blood and Marrow Transplant (NIBMT), Pakistan

Target Recruit Count: 24

Registration Number: NCT06239389

Locations: 🇵🇰
National Institute of Blood Diseases and Bone Marrow Transplantation, Karachi, Sindh, Pakistan

A Study to Investigate Treatment of HU and VPA, or 6-MP and VPA in Unfit AML/HR-MDS Patients

Phase 1

Not yet recruiting

Conditions: Myelodysplastic Syndromes, Adult
Acute Myeloid Leukemia, Adult

Interventions: Drug: Hydroxyurea, Hydroxycarbamide
Drug: Valproic acid
Drug: 6-Mercaptopurine (6-MP)

First Posted Date: 2024-01-10

Last Posted Date: 2024-01-10

Lead Sponsor: Haukeland University Hospital

Target Recruit Count: 48

Registration Number: NCT06199557

Locations: 🇳🇴
Haukeland University Hospital, Bergen, Norway

Realizing Effectiveness Across Continents With Hydroxyurea

Phase 2

Active, not recruiting

Conditions: Children
Sickle Cell Disease

Interventions: Drug: Hydroxyurea

First Posted Date: 2023-12-14

Last Posted Date: 2025-05-14

Lead Sponsor: Children's Hospital Medical Center, Cincinnati

Target Recruit Count: 810

Registration Number: NCT06171217

Locations: 🇦🇴
Hospital Pediátrico David Bernardino, Luanda, Angola
🇨🇩
Centre Hospitalier Monkole, Kinshasa, Congo, The Democratic Republic of the
🇰🇪
KEMRI/Wellcome Trust Research, Kilifi, Kenya

and more 1 locations

Thalidomide and Hydroxyurea Combination in β-Thalassemia Patients

Phase 2

Completed

Conditions: Thalassemia, Beta

Interventions: Drug: Hydroxyurea and Thalidomide

First Posted Date: 2023-12-01

Last Posted Date: 2023-12-01

Lead Sponsor: Children's Hospital Karachi

Target Recruit Count: 603

Registration Number: NCT06153784

Locations: 🇵🇰
Children's Hospital Karachi, Karachi, Sindh, Pakistan

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