Droxia

(Hydroxyurea)

ApprovedSmall molecule

Hydroxyurea is used to treat sickle cell disease by reducing painful episodes and other complications, and it's also used to treat certain types of cancer by slowing the growth of cancer cells.[2] Droxia is currently approved and in use across 26 indications[4].[1]

Droxia is one of 429 small molecule programs targeting Polycythemia Vera. It is currently in Approved.

Indications

161

Total Trials

Active Trials

428

Competitors

FDA Applications

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[4][1]

Indication

Polycythemia VeraTerminated

Beta-Thalassemia IntermediaApproved

StrokeRecruiting

Central Nervous System NeoplasmApproved

Desmoid-Type FibromatosisApproved

GliosarcomaApproved

FibromatosisApproved

MyelofibrosisApproved

Bone Marrow TransplantationRecruiting

HemochromatosisApproved

Polycythemia VeraApproved

Terminated

Beta-Thalassemia IntermediaApproved

Approved

StrokeApproved

Recruiting

Central Nervous System NeoplasmApproved

Approved

Desmoid-Type FibromatosisApproved

Approved

GliosarcomaApproved

Approved

FibromatosisApproved

Approved

MyelofibrosisApproved

Approved

Bone Marrow TransplantationPhase 2

Recruiting

HemochromatosisApproved

Approved

Showing 10 of 26 indications

Competition

Competitive Landscape

235 competitors in Acute Myeloid Leukemia

#DrugCompanyPhaseTrials

Novantrone

Pfizer

Phase 3

34 2

Xospata

Astellas

Phase 3

24 3

Fludara

Sanofi

Phase 3

18 4

Vepesid

AAvyxa

Phase 3

52 5

Mylotarg

WWyeth-Lederle Vaccines

Phase 3

38 6

Cerubidine

HHikma Pharmaceuticals LLC

Phase 3

42 7

Vanflyta

Daiichi Sankyo

Phase 3

16 8

Neupogen

EEastern Cooperative Oncology Group

Phase 3

30 9

Nexavar

Droxia

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2026

N/AActive, not recruiting

Trial

NCT02286154

Phase 2Active, not recruiting

Sickle Cell Anemia in Children

NCT03948867

Active, not recruiting

Myelofibrosis

NCT04217356

Q1 2027

Phase 1/2Active, not recruiting

Sickle Cell Disease

NCT05357482

Q3 2027

Phase 3Recruiting

Sickle Cell Anemia in Children

NCT05285917

Q4 2027

Recruiting

Sickle Cell Disease

NCT07543289

Clinical

Trials by Indication[1]

Sickle Cell Disease17 trials

Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia

Active, not recruiting·NNational Heart, Lung, and Blood Institute (NHLBI)·Phase 1/2·NCT05357482·40 enrolled

Hydroxyurea and Magnesium Pidolate to Treat People With Hemoglobin Sickle Cell Disease

Terminated·SSt. Jude Children's Research Hospital·Phase 2·NCT00532883·44 enrolled

Study of SANGUINATE™ Versus Hydroxyurea in Sickle Cell Disease (SCD) Patients

Completed·PProlong Pharmaceuticals·Phase 1·NCT01848925·24 enrolled

Show 14 more trials

Sickle Cell Disease - Stroke Prevention in Nigeria Trial

Completed·VVanderbilt University Medical Center·N/A·NCT01801423·29 enrolled

Assessment of Algorithm-Based Hydroxyurea Dosing on Fetal Hemoglobin Response, Acute Complications, and Organ Function in People With Sickle Cell Disease

Completed·NNational Heart, Lung, and Blood Institute (NHLBI)·Phase 1/2·NCT02225132·10 enrolled

Transplantation for Patients With Sickle Cell Disease From Mismatched Family Donors of Bone Marrow

Completed·EEmory University·Phase 2·NCT02757885·10 enrolled

A Trial for Prevention of Recurrent Ischemic Priapism in Men With Sickle Cell Disease: A Pilot Study

Completed·VVanderbilt University Medical Center·Phase 2·NCT05142254·64 enrolled

Hydroxyurea to Prevent Brain Injury in Sickle Cell Disease

Completed·JJohns Hopkins University·Phase 2·NCT01389024·28 enrolled

Risk Clinical Stratification of Sickle Cell Disease in Nigeria, Assessment of Efficacy/Safety of Hydroxyurea Treatment

Completed·LLoyola University·Phase 4·NCT02149537·53 enrolled

Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease

Active, not recruiting·NNational Heart, Lung, and Blood Institute (NHLBI)·Phase 1/2·NCT03077542·57 enrolled

Hydroxyurea and EPO in Sickle Cell Disease

Completed·JJulia Xu·Phase 1/2·NCT05451940·17 enrolled

Pharmacogenetics and Model-Informed Optimisation of Hydroxyurea Therapy in Sickle Cell Disease Patients of Nigerian Descent

Recruiting·OOchuko Orherhe··NCT07543289·100 enrolled

A Pilot Study of Hydroxyurea for the Treatment of Pulmonary Hypertension in Children and Young Adults With Sickle Cell Disease

Terminated·AAnn & Robert H Lurie Children's Hospital of Chicago·Preclinical·NCT00350844·6 enrolled

A Phase 2 Open-label Study to Evaluate the Activity of Etavopivat on Transcranial Doppler Velocities in Pediatric Patients With Sickle Cell Disease Who Are at Increased Risk for Primary Stroke

Recruiting·FForma Therapeutics, Inc.·Phase 2·NCT05953584·27 enrolled

Haploidentical Donor Hematopoietic Cell Transplant for Sickle Cell Disease

Recruiting·SSt. Jude Children's Research Hospital·Phase 2·NCT07616154·45 enrolled

Reduced Intensity Conditioning for Haploidentical Bone Marrow Transplantation in Patients With Symptomatic Sickle Cell Disease. (BMTCTN1507)

Completed·MMedical College of Wisconsin·Phase 2·NCT03263559·95 enrolled

REALIZING EFFECTIVENESS ACROSS CONTINENTS WITH HYDROXYUREA (REACH): A PHASE I/II PILOT STUDY OF HYDROXYUREA FOR CHILDREN WITH SICKLE CELL ANEMIA

Active, not recruiting·CChildren's Hospital Medical Center, Cincinnati·Preclinical·NCT01966731·635 enrolled

Anemia15 trials

Hydroxyurea Treatment for Adult Sickle Cell Anemia Patients in Kinshasa

Completed·—·N/A·NCT05681598·166 enrolled

Hydroxyurea for the Treatment of Patients With Sickle Cell Anemia

Completed·NNational Heart, Lung, and Blood Institute (NHLBI)·Phase 2·NCT00001197·41 enrolled

SACRED A Prospective Research Study to Reduce Stroke in Children With Sickle Cell Anemia

Completed·—·N/A·NCT02769845·283 enrolled

Show 12 more trials

Pediatric Hydroxyurea in Sickle Cell Anemia (PED HUG)

Completed·NNational Heart, Lung, and Blood Institute (NHLBI)·Phase 2·NCT00000602

Pharmacokinetics (PK) of Liquid Hydroxyurea in Pediatric Patients With Sickle Cell Anemia

Completed·—·Phase 1·NCT01506544·39 enrolled

Risk-based Therapy for Sickle Cell Anemia: A Feasibility Study

Withdrawn·—·Phase 2·NCT02090296

Evaluation of Hydroxyurea Plus L-arginine or Sildenafil to Treat Sickle Cell Anemia

Completed·NNational Institutes of Health Clinical Center (CC)·Phase 1·NCT00056433·39 enrolled

Hydroxyurea to Prevent Stroke in Children With Sickle Cell Anemia and Elevated TCD Flow Velocity

Completed·—·Phase 2·NCT00402480·50 enrolled

Multicenter Study of Hydroxyurea in Patients With Sickle Cell Anemia (MSH)

Completed·NNational Heart, Lung, and Blood Institute (NHLBI)·Phase 3·NCT00000586

Hydroxyurea to Prevent Organ Damage in Children With Sickle Cell Anemia

Completed·NNational Heart, Lung, and Blood Institute (NHLBI)·Phase 3·NCT00006400·193 enrolled

Cerebrovascular Reserve and White Matter Disease in Patients with Chronic Anemia

Completed·—··NCT03715972·165 enrolled

Phase I/II Randomized Study of Hydroxyurea With or Without Clotrimazole in Patients With Sickle Cell Anemia

Completed·UUniversity of North Carolina·Phase 1/2·NCT00004492·20 enrolled

Long-Term Effects of Hydroxyurea in Children With Sickle Cell Anemia (The BABY HUG Follow-up Study)

Completed·NNational Heart, Lung, and Blood Institute (NHLBI)··NCT00890396·163 enrolled

Hydroxyurea Therapy for Neurological and Cognitive Protection in Pediatric Sickle Cell Anemia in Uganda ( BRAINSAFE-II )

Completed·GGlobal Health Uganda LTD·Phase 3·NCT04750707·270 enrolled

Hydroxyurea in Young Children With Sickle Cell Anemia

Completed·—·N/A·NCT00519701·14 enrolled

Effectiveness9 trials

The Safety and Effectiveness of Didanosine Plus Stavudine Plus Delavirdine Mesylate Plus MKC-442 in HIV-Infected Patients Who Have Not Had Success With Protease Inhibitors

Terminated· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 2·NCT00002420·25 enrolled

A Study to Compare the Effectiveness of Different Anti-HIV Drug Regimens in Keeping Levels of HIV in the Blood as Low as Possible

Completed·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 2·NCT00000939·150 enrolled

The Safety and Effectiveness of Hydroxyurea and ddI Used Individually or Together in HIV-Infected Patients

Completed·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 1·NCT00001074·140 enrolled

Show 6 more trials

Safety and Effectiveness of a Combination Anti-HIV Drug Treatment

Completed· GSK logo

GSK·Phase 4·NCT00005018·150 enrolled

A Study of the Safety and Effectiveness of Hydroxyurea in Patients on Potent Antiretroviral Therapy and Who Have Less Than 200 Copies/ml of HIV RNA in Their Blood

Completed·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 2·NCT00000916·399 enrolled

Realizing Effectiveness Across Continents With Hydroxyurea

Active, not recruiting·—·Phase 2·NCT06171217·810 enrolled

Safety and Effectiveness of Combining Hydroxyurea (HU) With Didanosine (ddI) and Stavudine (d4T) for Treatment of HIV-Infected Adults

Completed·—·Phase 1·NCT00002427·225 enrolled

Effectiveness of Early or Delayed Addition of Hydroxyurea to a Three-Drug Anti-HIV Drug Combination Including Didanosine, in Advanced HIV Patients Who Failed a First or Second Anti-HIV Triple-Drug Therapy

Withdrawn·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 2·NCT00008866

The Safety and Effectiveness of Didanosine Plus Stavudine Plus Nevirapine Combined With MKC-442 in HIV-Infected Patients Who Have Not Had Success With Protease Inhibitors

Terminated· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 2·NCT00002418·25 enrolled

Chronic Myelogenous Leukemia7 trials

Imatinib Mesylate With or Without Interferon Alfa or Cytarabine Compared With Interferon Alfa Followed by Donor Stem Cell Transplant in Treating Patients With Newly Diagnosed Chronic Phase Chronic Myelogenous Leukemia

Completed·HHeidelberg University·Phase 3·NCT00055874·1,551 enrolled

Homoharringtonine Compared With Hydroxyurea for Chronic Myelogenous Leukemia That Has Not Responded to Interferon Alfa

Terminated·AAlliance for Clinical Trials in Oncology·Phase 3·NCT00004933·5 enrolled

Interferon Alfa in Treating Patients With Newly Diagnosed Chronic Myelogenous Leukemia

Unknown status·MMedical Research Council·Phase 3·NCT00002869·800 enrolled

Show 4 more trials

Combination Chemotherapy Followed by Peripheral Stem Cell Transplantation in Treating Patients With Chronic Myelogenous Leukemia

Completed·FFred Hutchinson Cancer Center·Phase 2·NCT00002674·30 enrolled

Interferon-alfa With or Without Chemotherapy and Peripheral Stem Cell Transplantation in Treating Patients With Newly Diagnosed Chronic Myelogenous Leukemia

Completed·EEastern Cooperative Oncology Group·Phase 3·NCT00002868·744 enrolled

Chemotherapy and Biological Therapy With or Without Bone Marrow or Peripheral Stem Cell Transplant in Treating Patients With Chronic Myelogenous Leukemia

Unknown status·IIII. Medizinische Klinik Mannheim·Phase 3·NCT00025402·1,000 enrolled

Chemotherapy, Interferon, and Bone Marrow Transplantation in Treating Patients With Chronic Myelogenous Leukemia

Unknown status·GGerman CML Study Group·Phase 3·NCT00002771·750 enrolled

HIV infection5 trials

Combination Drug Treatment of Pediatric HIV Infection

Completed·NNational Cancer Institute (NCI)·Phase 1·NCT00001818·50 enrolled

A Study to Evaluate Various Combinations of Anti-HIV Medications to Treat Early HIV Infection

Completed·NNational Institute of Allergy and Infectious Diseases (NIAID)·N/A·NCT00000919·900 enrolled

Safety and Effectiveness of an Anti-HIV Drug Combination With and Without Hydroxyurea in Patients With Early HIV Infection

Withdrawn·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 2·NCT00006339

Show 2 more trials

A Pilot Open Label Trial of HIV Therapy With d4T (Stavudine), ddI (Didanosine), Nelfinavir and Hydroxyurea in Subjects With Recent HIV Infection

Completed· Bristol-Myers Squibb logo

Bristol-Myers Squibb·N/A·NCT00002177

A Pilot Open Label Trial of HIV Therapy With d4T (Stavudine), ddI (Didanosine), Nelfinavir and Hydroxyurea in Subjects With Early Asymptomatic HIV Infection

Completed· Bristol-Myers Squibb logo

Bristol-Myers Squibb·N/A·NCT00002176

67 more indications

Meningioma5 trials

Combination of Hydroxyurea and Verapamil for Refractory Meningiomas

Completed·UUniversity of Utah·Phase 2·NCT00706810·8 enrolled

S9811 Hydroxyurea in Treating Patients With Unresectable Benign Meningioma

Completed·SSWOG Cancer Research Network·Phase 2·NCT00003590·29 enrolled

Hydroxyurea With or Without Imatinib Mesylate in Treating Patients With Recurrent or Progressive Meningioma

Unknown status·IIstituto Scientifico H. San Raffaele·Phase 2·NCT00904735·76 enrolled

Show 2 more trials

Hydroxyurea in Treating Patients With Recurrent and/or Unresectable Meningioma

Completed·UUNICANCER·Phase 2·NCT00006119

Imatinib Mesylate and Hydroxyurea in Treating Patients With Recurrent or Progressive Meningioma

Completed·—·Phase 2·NCT00354913·21 enrolled

Polycythemia Vera5 trials

Study on Efficacy and Safety of Givinostat Versus Hydroxyurea in Patients With Polycythemia Vera

Recruiting· Italfarmaco logo

Italfarmaco·Phase 3·NCT06093672·220 enrolled

Large-scale Trial Testing the Intensity of CYTOreductive Therapy in Polycythemia Vera (PV)

Terminated·CConsorzio Mario Negri Sud·Phase 3·NCT01645124·365 enrolled

Pegylated Interferon Alpha-2b Versus Hydroxyurea in Polycythemia Vera

Completed·AAOP Orphan Pharmaceuticals AG·Phase 3·NCT01949805·257 enrolled

Show 2 more trials

Randomized Switch Study From Hydroxyurea to Ruxolitinib for RELIEF of Polycythemia Vera Symptoms: The Relief Study

Completed· Incyte logo

Incyte·Phase 3·NCT01632904·110 enrolled

HU-F-AIM - A Prospective, Interventional Study to Evaluate HU-resistance in Polycythemia Vera Patients Who Meet Predictive Parameters Identified in the Machine Learning Project PV-AIM

Terminated· Novartis logo

Novartis·Approved·NCT05853458·76 enrolled

Beta-Thalassemia4 trials

Effects of Hydroxyurea and Metformin in Transfusion Dependent Beta-Thalassemia

Recruiting·—·Phase 4·NCT06606886·96 enrolled

Hydroxyurea to Treat Beta-Thalassemia (Cooley's Anemia)

Completed·NNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)·Phase 2·NCT00001958·100 enrolled

The Efficacy and Safety of HYDROXYUREA in Management of Beta Thalassemia Patients in Karachi Pakistan

Unknown status·DDr.Saqib Hussain Ansari·Phase 2·NCT03183375·100 enrolled

Show 1 more trial

Evaluating the Safety of G-CSF Mobilization in Individuals With Beta Thalassemia Major

Completed·UUniversity of Washington·N/A·NCT00336362·26 enrolled

Stroke4 trials

Stroke Prevention in Nigeria 2 Trial

Recruiting·VVanderbilt University Medical Center·Phase 4·NCT06526117·220 enrolled

Stroke With Transfusions Changing to Hydroxyurea

Terminated·SSt. Jude Children's Research Hospital·Phase 3·NCT00122980·134 enrolled

Primary Prevention of Stroke in Children With Sickle Cell Anaemia in Nigeria in the Community

Unknown status·—·N/A·NCT05434000·217 enrolled

Show 1 more trial

Primary Prevention of Stroke in Children With SCD in Sub-Saharan Africa II

Completed·VVanderbilt University Medical Center·Phase 3·NCT02560935·440 enrolled

Head and Neck Cancer4 trials

Chemotherapy and Locoregional Therapy Trial (Surgery or Radiation) for Patients With Head and Neck Cancer

Completed·UUniversity of Chicago·Phase 2·NCT03107182·72 enrolled

Dosing Study of Radiation Combined With Tislelizumab and Pamiparib in Patients With Previously Treated Head and Neck Cancer

Recruiting·UUniversity of Chicago·Phase 1·NCT05526924·30 enrolled

Combination Chemotherapy With or Without Veliparib in Treating Patients With Stage IV Head and Neck Cancer

Completed·NNational Cancer Institute (NCI)·Phase 1/2·NCT01711541·24 enrolled

Show 1 more trial

Chemotherapy Plus Radiation Therapy in Treating Patients With Head and Neck Cancer

Completed·UUniversity of Chicago·Phase 2·NCT00002951·53 enrolled

Spinal Muscular Atrophy3 trials

A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients

Completed·SStanford University·Phase 1/2·NCT00568698·29 enrolled

A Trial of Hydroxyurea in Spinal Muscular Atrophy

Completed·KKaohsiung Medical University Chung-Ho Memorial Hospital·Phase 2/3·NCT00485511

A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients

Completed·SStanford University·Phase 1/2·NCT00568802·27 enrolled

Advanced Head and Neck Cancer3 trials

Fluorouracil, Hydroxyurea, Cetuximab and Twice-daily Intensity Radiation Therapy for Advanced Head and Neck Cancer

Completed·JJohnny Kao·Phase 2·NCT00462735·33 enrolled

A Multicenter Study of Intensive Concomitant Chemoradiotherapy With Filgrastim (GCSF) for Patients With Locoregionally Advanced Head and Neck Cancer

Completed·UUniversity of Chicago·Phase 2·NCT01693718·44 enrolled

Bevacizumab, Fluorouracil, and Hydroxyurea Plus Radiation Therapy in Treating Patients With Advanced Head and Neck Cancer

Completed·NNational Cancer Institute (NCI)·Phase 1·NCT00023959·39 enrolled

Glioblastoma3 trials

Hydroxy-urea and Temozolomide in Patients With a Recurrent Malignant Brain Tumor (Glioblastoma)

Unknown status·MM.E. van Linde·Phase 1·NCT03463733·54 enrolled

Radiation Therapy in Treating Patients With Glioblastoma

Completed·NNational Cancer Institute (NCI)·Phase 1·NCT00019058·34 enrolled

Study of Imatinib Mesylate in Combination With Hydroxyurea Versus Hydroxyurea Alone as an Oral Therapy in Patients With Temozolomide Resistant Progressive Glioblastoma

Completed· Novartis logo

Novartis·Phase 3·NCT00154375·240 enrolled

Treatment3 trials

Novel Dose Escalation to Predict Treatment With Hydroxyurea

Completed·—·Phase 1·NCT02042222·70 enrolled

Treatment of CML Patients With Imatinib and Hydroxyurea (CML2004)

Completed·UUniversity of Leipzig·Phase 1/2·NCT02480608·113 enrolled

EXTEND EXpanding Treatment for Existing Neurological Disease

Completed·—·Phase 2·NCT02556099·43 enrolled

Essential Thrombocythemia3 trials

A Study of Anagrelide and Hydroxyurea in High-Risk Essential Thrombocythemia Patients

Completed· Takeda logo

Takeda·Phase 4·NCT00202644·150 enrolled

Randomized Trial of Pegylated Interferon Alfa-2a Versus Hydroxyurea in Polycythemia Vera (PV) and Essential Thrombocythemia (ET)

Completed·RRonald Hoffman·Phase 3·NCT01259856·168 enrolled

A Phase 3, Randomized, Double-blind, Active-Comparator-Controlled Clinical Study to Evaluate the Efficacy and Safety of Bomedemstat (MK-3543) Versus Hydroxyurea in Cytoreductive Therapy Naïve Essential Thrombocythemia Participants

Active, not recruiting· Merck & Co. logo

Merck & Co.·Phase 3·NCT06456346·300 enrolled

Malaria2 trials

Novel Use Of Hydroxyurea in an African Region With Malaria

Completed·IIndiana University·Phase 3·NCT01976416·208 enrolled

Optimizing Hydroxyurea Therapy in Children With SCA In Malaria Endemic Areas

Completed·IIndiana University·Phase 3·NCT03128515·187 enrolled

Thalassemia2 trials

Combination Therapy of Hydroxyurea With L-Carnitine and Magnesium Chloride in Thalassemia Intermedia

Completed·SShiraz University of Medical Sciences·Phase 2·NCT00809042·120 enrolled

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

Completed·NNational Institute of Blood and Marrow Transplant (NIBMT), Pakistan·Phase 2·NCT06239389·24 enrolled

Trial2 trials

Primary Thrombocythaemia 1 Trial

Completed·UUniversity of Cambridge·N/A·NCT00175838·1,398 enrolled

Therapeutic Response Evaluation and Adherence Trial (TREAT)

Active, not recruiting·—·N/A·NCT02286154·150 enrolled

Hemoglobin SC Disease2 trials

Treatment of Hemoglobin SC Disease With Hydroxyurea

Terminated·—·Phase 2·NCT02336373·32 enrolled

Treatment of Adult Patients With Hemoglobin SC Disease (SCYTHE)

Terminated·—·Phase 2·NCT02640573·1 enrolled

Radiation Therapy2 trials

Combination Chemotherapy Plus Radiation Therapy in Treating Patients With Advanced Mouth Cancer

Completed·—·Phase 1/2·NCT00004901

Combination Chemotherapy Plus Radiation Therapy in Treating Patients With Previously Untreated Advanced Cancer of the Mouth, Pharynx, or Larynx

Unknown status·NNational Cancer Institute (NCI)·Phase 2·NCT00004094

Lymphoma2 trials

Hydroxyurea in Treating Patients With Epstein-Barr Virus-Associated Primary CNS Lymphoma and AIDS

Completed·SSt. Jude Children's Research Hospital·Phase 2·NCT00002940

Chemotherapy for Relapsed Epstein Barr Virus Associated Lymphoma

Terminated·UUniversity of Miami·Phase 2·NCT01964755·6 enrolled

Treatment-Refractory Solid Tumors2 trials

A Phase 1 Study of OTS-412 (Recombinant Oncolytic Vaccinia Virus) in Combination With Hydroxyurea or Hydroxyurea/Atezolizumab in Treatment-refractory Solid Tumor Patients

Recruiting·BBionoxx Inc.·Phase 1·NCT07190833·21 enrolled

A Phase 1 Study of OTS-412 (Recombinant Oncolytic Vaccinia Virus) in Combination With Hydroxyurea or Hydroxyurea/Atezolizumab in Treatment-refractory Solid Tumor Patients

Not yet recruiting·BBionoxx Inc.·Phase 1·NCT07170592·24 enrolled

Sickle Cell Anemia in Children2 trials

Stroke Prevention With Hydroxyurea Enabled Through Research and Education (SPHERE): A Prospective Trial to Reduce Primary Stroke in Children With Sickle Cell Anaemia

Active, not recruiting·CChildren's Hospital Medical Center, Cincinnati·Phase 2·NCT03948867·202 enrolled

BrUOG 419 - Promoting Utilization and Safety of Hydroxyurea Using Precision in Africa (PUSHUP)

Recruiting·BBrown University·Phase 3·NCT05285917·400 enrolled

Acute Myeloid Leukemia2 trials

A Phase II, Multicentre, Open Label Clinical Trial Evaluating the Efficacy and Safety of Oral Decitabine Plus Ivosidenib in Adult Patients With Newly Diagnosed Acute Myeloid Leukemia Older Than 60 Years Old and/or Who Are Ineligible for Standard Induction Chemotherapy

Recruiting·PPETHEMA Foundation·Phase 2·NCT07507760·50 enrolled

Frontline Ziftomenib in NPM1-Mutated or KMT2A-Rearranged Acute Myeloid Leukemia in Patients Not Eligible for Intensive Induction or Other Therapy

Recruiting·UUma Borate·Phase 2·NCT06930352·70 enrolled

Pediatric1 trial

Hydroxyurea Therapy: Optimizing Access in Pediatric Populations Everywhere

Terminated·SSt. Jude Children's Research Hospital·Phase 2·NCT03825341·1 enrolled

Locally Advanced Head and Neck Cancer1 trial

Cetuximab (ERBITUX®) Added to Two Concurrent Chemoradiotherapy Platforms in Locally Advanced Head and Neck Cancer

Completed·UUniversity of Chicago·Phase 2·NCT00468169·110 enrolled

Oropharyngeal Cancer1 trial

Nab-paclitaxel and Carboplatin Followed by Response-Based Local Therapy in Treating Patients With Stage III or IV HPV-Related Oropharyngeal Cancer

Completed·UUniversity of Chicago·Phase 2·NCT02258659·62 enrolled

CML1 trial

Evaluation of the Therapeutic Effect of HU Pulse Therapy for CML Patients

Unknown status·FFirst Affiliated Hospital of Harbin Medical University·Phase 3·NCT03515018·60 enrolled

Acute Lymphoblastic Leukemia1 trial

Isatuximab in Combination With Chemotherapy in Pediatric Patients With Relapsed/Refractory Acute Lymphoblastic Leukemia or Acute Myeloid Leukemia

Terminated· Sanofi logo

Sanofi·Phase 2·NCT03860844·67 enrolled

Induction Chemotherapy1 trial

Docetaxel Based Chemotherapy Plus or Minus Induction Chemotherapy to Decrease Events in Head and Neck Cancer (DeCIDE)

Completed·UUniversity of Chicago·Phase 3·NCT00117572·285 enrolled

Drug1 trial

A Study of MKC-442 in Combination With Other Anti-HIV Drugs

Completed·TTriangle Pharmaceuticals·Phase 2·NCT00002412

Tenofovir1 trial

Safety and Durability ofTenofovir and a Cell Cycle Agent for Viral Suppression

Completed·UUniversity of Maryland, Baltimore·N/A·NCT00344981·9 enrolled

Cancer of the Esophagus1 trial

Combination Chemotherapy Combined With Radiation Therapy in Treating Patients Who Have Stage II or Stage III Cancer of the Esophagus

Unknown status·GGERCOR - Multidisciplinary Oncology Cooperative Group·Phase 1/2·NCT00008047

Myelodysplastic/Myeloproliferative Neoplasm1 trial

Luspatercept With or Without Hydroxyurea for the Treatment of Myelodysplastic/Myeloproliferative Neoplasms With Ring Sideroblasts and Thrombocytosis or Unclassifiable With Ring Sideroblasts

Terminated·MMayo Clinic·Phase 2·NCT05005182·3 enrolled

Thyroid Cancer1 trial

Chemotherapy Plus Radiation Therapy in Treating Patients With Previously Untreated Thyroid Cancer

Completed·NNational Cancer Institute (NCI)·Phase 2·NCT00004089

Esophageal Cancer1 trial

Combination Chemotherapy and Interferon Alfa Followed by Surgery and/or Radiation Therapy in Treating Patients With Stage I, Stage II, or Stage III Esophageal Cancer

Terminated·NNorthwestern University·Phase 2·NCT00004897

Older Patients1 trial

Selinexor (KPT-330) in Older Patients With Relapsed AML

Completed·KKaryopharm Therapeutics Inc·Phase 2·NCT02088541·317 enrolled

Acute Promyelocytic Leukemia1 trial

Treatment of Non-high-risk Acute Promyelocytic Leukemia (APL) With Realgar-Indigo Naturalis Formula (RIF)

Unknown status·FFirst Affiliated Hospital Xi'an Jiaotong University·Phase 3·NCT02899169·110 enrolled

Efficacy1 trial

Anagrelide vs. Hydroxyurea - Efficacy and Tolerability Study in Patients With Essential Thrombocythaemia

Completed·AAOP Orphan Pharmaceuticals AG·Phase 3·NCT01065038

Stomach Cancer1 trial

Comparison of Combination Chemotherapy Regimens in Treating Patients With Advanced Stomach Cancer

Completed·EEastern Cooperative Oncology Group·Phase 2·NCT00003172

Oxidative Stress1 trial

Hydroxy Urea, Omega 3, Nigella Sativa,Honey on Oxidative Stress and Iron Chelation in Pediatric Major Thalassemia

Completed·—·Phase 2/3·NCT04292314·350 enrolled

Gastrointestinal1 trial

Combination Chemotherapy Plus Interferon Alfa Followed by Filgrastim in Treating Patients With Gastrointestinal Tract Cancer

Completed·MMontefiore Medical Center·Phase 2·NCT00019474·60 enrolled

Desmoid Tumor1 trial

Effect of Hydroxyurea as Treatment for Primary Desmoid Tumors

Withdrawn·—·Phase 2·NCT00978146

Leukemia1 trial

VNP40101M in Treating Patients With Acute Myelogenous Leukemia or High-Risk Myelodysplasia

Completed·VVion Pharmaceuticals·Phase 2·NCT00083187·230 enrolled

Myelodysplastic Syndrome1 trial

Intensive Compared With Nonintensive Chemotherapy in Treating Older Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome

Completed·LLeukemia Research Fund·Phase 3·NCT00005823·2,000 enrolled

Recurrent Head and Neck Cancer1 trial

Paclitaxel Albumin-Stabilized Nanoparticle Formulation and Carboplatin Followed By Chemoradiation in Treating Patients With Recurrent Head and Neck Cancer

Completed·UUniversity of Chicago·Phase 1·NCT01847326·48 enrolled

Safety and Efficacy1 trial

A Randomized, Placebo-Controlled Study of the Safety and Efficacy of Efavirenz, Didanosine, and Stavudine in Combination With or Without Hydroxyurea in Antiretroviral Naive or Experienced HIV-Infected Patients

Completed·NNorthwestern University·Phase 3·NCT00002230·100 enrolled

Advanced or Metastatic Solid Tumors1 trial

A Study of onCARlytics (CF33-CD19) in Combination With Blinatumomab in Adults With Advanced or Metastatic Solid Tumors (OASIS)

Terminated·IImugene Limited·Phase 1·NCT06063317·25 enrolled

Non-Small Cell Lung Cancer1 trial

Hydroxyurea Plus Combination Chemotherapy in Patients With Non-small Cell Lung Cancer

Unknown status·OOttawa Regional Cancer Centre·Phase 1·NCT00002887·30 enrolled

Randomized1 trial

Randomized Phase III Study of Decitabine +/- Hydroxyurea (HY) Versus HY in Advanced Proliferative CMML

Completed·GGroupe Francophone des Myelodysplasies·Phase 3·NCT02214407·170 enrolled

Observational Study1 trial

Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG) Follow-up Observational Study II Protocol

Completed·NNational Heart, Lung, and Blood Institute (NHLBI)··NCT01783990·150 enrolled

Non-malignant Disorders1 trial

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

Recruiting·PPaul Szabolcs·Phase 2·NCT01962415·100 enrolled

Advanced Malignant Solid Tumors1 trial

A Dose Escalation Study of Hydroxyurea in Combination With SCH900776 in Advanced Malignant Solid Tumors

Withdrawn·—·Phase 1·NCT01521299

Newly Diagnosed1 trial

Vinblastine for Leukoreduction in Newly Diagnosed AML and Hyperleukocytosis

Unknown status·HHospital Universitario Dr. Jose E. Gonzalez·Phase 2·NCT05062278·46 enrolled

Oral1 trial

Pharmacokinetics of Oral Hydroxyurea Solution

Completed·NNova Laboratories Limited·Phase 1/2·NCT03763656·33 enrolled

Glioblastoma Multiforme1 trial

Efficacy and Safety of Imatinib Mesylate Plus Hydroxyurea (HU) in Patients With Recurrent Glioblastoma Multiforme (GBM)

Terminated· Novartis logo

Novartis·Phase 2·NCT00290771·231 enrolled

Malnutrition1 trial

Management of Severe Acute Malnutrition in SCD, in Northern Nigeria

Completed·VVanderbilt University Medical Center·Phase 2·NCT03634488·132 enrolled

Age1 trial

Hydroxyurea Management in Kids: Intensive Versus Stable Dosage Strategies

Completed·SSt. Jude Children's Research Hospital·Phase 2·NCT03020615·58 enrolled

Malignant Glioma1 trial

Imatinib Mesylate, Vatalanib, and Hydroxyurea in Treating Patients With Recurrent or Relapsed Malignant Glioma

Completed·—·Phase 1·NCT00387933·37 enrolled

Primary Progressive Multiple Sclerosis1 trial

Hydroxyurea in Primary Progressive Multiple Sclerosis

Terminated·SS. Andrea Hospital·Phase 2/3·NCT01103583·33 enrolled

Sickle Cell Crisis1 trial

Hydroxyurea in the Emergency Room to Lessen Pain in Sickle Cell Crisis

Unknown status·IInstituto Estadual de Hematologia Arthur de Siqueira Cavalcanti·Phase 2·NCT03062501·30 enrolled

Undifferentiated Carcinoma1 trial

PHASE III TRIAL COMPARING, NEOADJUVANT CHEMOTHERAPY FOLLOWED BY STANDARD RADIOTHERAPY VERSUS THE SAME NEOADJUVANT CHEMOTHERAPY FOLLOWED BY STANDARD RADIOTHERAPY ASSOCIATED WITH DAILY HYDROXYUREA IN THE TREATMENT OF LOCALLY ADVANCED UNDIFFERENTIATED CARCINOMA NASOPHARYNGEAL TYPE.

Unknown status·GGustave Roussy, Cancer Campus, Grand Paris·Phase 3·NCT00180973·520 enrolled

Pulmonary Arterial Hypertension1 trial

Hydroxyurea in Pulmonary Arterial Hypertension

Withdrawn·NNational Heart, Lung, and Blood Institute (NHLBI)·Phase 1·NCT01950585

Bone Marrow Transplantation1 trial

Lung and Bone Marrow Transplantation for Lung and Bone Marrow Failure

Recruiting·PPaul Szabolcs·Phase 1/2·NCT03500731·8 enrolled

High Risk1 trial

HSCT for High Risk Inherited Inborn Errors

Completed·MMasonic Cancer Center, University of Minnesota·Phase 2·NCT00383448·38 enrolled

Prevention1 trial

Alternative Dosing And Prevention of Transfusions (ADAPT)

Active, not recruiting·—·Phase 1·NCT05662098·100 enrolled

Recurrent Acute Myeloid Leukemia1 trial

CD83 (RPCAR01) CAR T in Relapsed or Refractory Acute Myeloid Leukemia (AML): A Phase I Trial

Recruiting·RRoswell Park Cancer Institute·Phase 1·NCT06871410·26 enrolled

Leukemia, Myelomonocytic, Chronic1 trial

A Randomized Phase 2 Study of Pacritinib vs. Hydroxyurea in Patients With Advanced Proliferative Chronic Myelomonocytic Leukemia

Recruiting·TTheradex·Phase 2·NCT07033598·66 enrolled

Sickle Cell Anemia1 trial

T-Cell Depleted, Alternative Donor Transplant in Pediatric and Adult Patients With Severe Sickle Cell Disease (SCD) and Other Transfusion-Dependent Anemias

Recruiting·PPaul Szabolcs·Preclinical·NCT03653338·5 enrolled

Lactating Women on Select DOI1 trial

Pharmacokinetics and Safety of Commonly Used Drugs in Lactating Women and Breastfed Infants

Recruiting·DDuke University··NCT03511118·1,600 enrolled

Myelofibrosis1 trial

A Patient Preferences-Controlled Study of Allogeneic Hematopoietic Cell Transplantation Versus Best Available Non-Transplant Therapies in Patients With High-Risk Myelofibrosis (ALLO-BAT Study)

Active, not recruiting·UUniversity Health Network, Toronto··NCT04217356·90 enrolled

Unspecified6 trials

Transcranial Doppler (TCD) With Transfusions Changing to Hydroxyurea

Terminated·—·Phase 3·NCT01425307·159 enrolled

Hydroxyurea Optimization Through Precision Study

Completed·LLifespan·Phase 3·NCT03789591·104 enrolled

Study of Clotrimazole and Hydroxyurea in Patients With Sickle Cell Syndromes

Completed·—·N/A·NCT00004404·20 enrolled

Show 3 more trials

Mobile-Directly Observed Therapy on Adherence to Hydroxyurea

Completed·MMuhimbili University of Health and Allied Sciences·Phase 2·NCT02844673·98 enrolled

Hydroxyurea and Transfusion

Completed·—·Phase 2·NCT03644953·14 enrolled

Sparing Conversion to Abnormal TCD (Transcranial Doppler) Elevation (SCATE)

Terminated·—·Phase 3·NCT01531387·38 enrolled

Regulatory

FDA Approval Timeline[5]

Dec 7, 1967

DROXIA, HYDREA

NDA NDA016295

May 3, 1977

DROXIA, HYDREA

NDA NDA016295

Feb 19, 1980

DROXIA, HYDREA

NDA NDA016295

Jul 16, 1985

DROXIA, HYDREA

NDA NDA016295

Mar 19, 1987

DROXIA, HYDREA

NDA NDA016295

Mar 19, 1987

DROXIA, HYDREA

NDA NDA016295

May 10, 1988

DROXIA, HYDREA

NDA NDA016295

Aug 25, 1988

DROXIA, HYDREA

NDA NDA016295

Jul 31, 1992

DROXIA, HYDREA

NDA NDA016295

Mar 9, 1993

DROXIA, HYDREA

NDA NDA016295

Sep 5, 1995

DROXIA, HYDREA

NDA NDA016295

Sep 24, 1996

DROXIA, HYDREA

NDA NDA016295

Sep 24, 1996

DROXIA, HYDREA

NDA NDA016295

Oct 15, 1996

DROXIA, HYDREA

NDA NDA016295

Nov 25, 1996

DROXIA, HYDREA

NDA NDA016295

Apr 7, 1997

DROXIA, HYDREA

NDA NDA016295

May 23, 1997

DROXIA, HYDREA

NDA NDA016295

Feb 25, 1998

DROXIA, HYDREA

NDA NDA016295

Feb 25, 1998

DROXIA, HYDREA

NDA NDA016295

Feb 25, 1998

DROXIA

NDA NDA016295

Nov 10, 1998

DROXIA, HYDREA

NDA NDA016295

Aug 11, 1999

DROXIA, HYDREA

NDA NDA016295

Jan 12, 2000

DROXIA, HYDREA

NDA NDA016295

Feb 20, 2001

DROXIA, HYDREA

NDA NDA016295

Apr 4, 2001

DROXIA, HYDREA

NDA NDA016295

Jun 26, 2003

DROXIA, HYDREA

NDA NDA016295

Feb 19, 2004

DROXIA, HYDREA

NDA NDA016295

Sep 19, 2006

DROXIA, HYDREA

NDA NDA016295

May 7, 2010

DROXIA, HYDREA

NDA NDA016295

Jan 26, 2012

DROXIA, HYDREA

NDA NDA016295

Jan 26, 2012

DROXIA, HYDREA

NDA NDA016295

Jul 16, 2015

DROXIA, HYDREA

NDA NDA016295

Jul 16, 2015

DROXIA, HYDREA

NDA NDA016295

Mar 23, 2016

DROXIA, HYDREA

NDA NDA016295

Mar 23, 2016

DROXIA, HYDREA

NDA NDA016295

Dec 18, 2017

DROXIA, HYDREA

NDA NDA016295

Dec 18, 2017

DROXIA, HYDREA

NDA NDA016295

Dec 21, 2017

SIKLOS

NDA NDA208843

Jul 22, 2019

DROXIA, HYDREA

NDA NDA016295

Dec 18, 2019

DROXIA, HYDREA

NDA NDA016295

Feb 9, 2021

DROXIA, HYDREA

NDA NDA016295

Aug 5, 2021

DROXIA, HYDREA

NDA NDA016295

Jan 13, 2022

DROXIA, HYDREA

NDA NDA016295

Jun 7, 2023

DROXIA, HYDREA

NDA NDA016295

Nov 28, 2023

DROXIA, HYDREA

NDA NDA016295

Apr 4, 2024

XROMI

NDA NDA216593

Jun 20, 2024

DROXIA, HYDREA

NDA NDA016295

Sources

References

[1]
ClinicalTrials.gov — 161 trial records for Hydroxyurea. U.S. National Library of Medicine, National Institutes of Health.
[2]
MedlinePlus Drug Information — Droxia drug information. U.S. National Library of Medicine, National Institutes of Health.
[3]
ChEMBL — Hydroxyurea compound record. European Bioinformatics Institute (EMBL-EBI).
[4]
Open Targets Platform — Drug–disease associations for Hydroxyurea. EMBL-EBI & Wellcome Sanger Institute.
[5]
OpenFDA — FDA approval records (NDA NDA016295). U.S. Food & Drug Administration.
[6]
FDA Orange Book — Patent and exclusivity data for Droxia. U.S. Food & Drug Administration.
[7]
SEC EDGAR — Theravia company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: Ribonucleoside-Diphosphate Reductase RR1 Inhibitor[3]
Modality: Small molecule[3]
Highest Phase: Approved[1]
Company: TTheravia

Patent Landscape[6]

Patent Cliff

Dec 23, 2041

Earliest patent expiration — generic entry possible after this date

Patent #Expires

12409156Dec 23, 2041