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Drugs in development · updated 30 October 2026

Hydroxyurea: clinical trials and development status

Other names and codes: Hydroxycarbamide, Siklos, Xromi, Droxia, Hydrea

NLM verified identityApproved in the US (FDA)Authorised in the EU (EMA) since 29 June 2007

As of 30 October 2026 Hydroxyurea is in 28 active clinical trials (recruiting or ongoing, phases I–III) for 44 diseases. The most advanced phase is III.

28Active trials
IIIMost advanced phase
44Diseases studied

Summary updated every week · data as of 30 October 2026. In Phuturdex you can follow Hydroxyurea and get an email at every change or safety alert.

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Diseases studied

For each disease: the drug’s most advanced phase and active trials, with the reference trial on ClinicalTrials.gov. Showing 30 of 44 diseases.

DiseaseMost advanced phaseActive trialsReference trial
Sickle-cell diseaseIII13NCT05285917
Polycythemia veraIII2NCT04116502
Thrombocythemia, EssentialIII1NCT06456346
Beta thalassemiaII4NCT01962415
Acute myeloid leukemiaII3NCT06930352
Diamond-Blackfan anemiaII2NCT01962415
AdrenoleukodystrophyII1NCT01962415
Alpha-mannosidosisII1NCT01962415
AnemiaI–II1NCT03653338
Arthritis, JuvenileII1NCT01962415
Autoimmune lymphoproliferative syndromeII1NCT01962415
Chediak-Higashi syndromeII1NCT01962415
Chronic granulomatous diseaseII1NCT01962415
Chronic myelomonocytic leukemiaII1NCT07033598
Chronic obstructive pulmonary diseaseI–II1NCT03500731
Congenital amegakaryocytic thrombocytopeniaII1NCT01962415
Congenital bone marrow failure syndromesII1NCT01962415
Crohn's diseaseII1NCT01962415
Dyskeratosis congenitaII1NCT01962415
EmphysemaI–II1NCT03500731
Gaucher's diseaseII1NCT01962415
Graft-versus-host diseaseI–II1NCT03077542
HemoglobinopathiesII1NCT06490601
Hemophagocytic lymphohistiocytosisII1NCT01962415
Hereditary diffuse leukoencephalopathy with spheroidsII1NCT01962415
Idiopathic pulmonary fibrosisI–II1NCT03500731
Immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndromeII1NCT01962415
Inflammatory bowel diseasesII1NCT01962415
Leukocyte adhesion deficiency type 1II1NCT01962415
Leukodystrophy, Globoid CellII1NCT01962415

Everything on Hydroxyurea

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Source: Phuturdex analysis of ClinicalTrials.gov (U.S. National Library of Medicine), FDA and EMA data, updated 30 October 2026. Active trials = recruiting or ongoing, phases I–III. Diseases are those named by each trial (NLM MeSH terms). The estimated launch, when shown, is a Phuturdex calculation described on the Method page, not official data.

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