Clinical Trials

Deferasirox has been evaluated in numerous clinical trials investigating its efficacy in systemic metal accumulation and hematologic disorders, including chronic transfusional iron overload, myelodysplastic syndromes, beta-thalassemia, Diamond-Blackfan anemia, and aplastic anemia. Spanning Early Phase 1 through Phase 4, these studies comprise completed, actively recruiting, terminated, and not yet recruiting protocols. Research is sponsored by pharmaceutical corporations such as Novartis and Elpen Pharmaceutical Co., alongside prominent academic centers including the Fred Hutchinson Cancer Center and the University of Pisa.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT06784336 RECRUITING
Allogeneic Stem Cell Transplant
University of Michigan Rogel Cancer Center
2025-10-15 PHASE2
NCT06964971 NOT_YET_RECRUITING
Transfusion-dependent Non-severe Aplastic Anemia
The First Affiliated Hospital of Zhejiang Chinese Medical University
2025-05-20
NCT07023666 RECRUITING
Sickle Cell Disease
Inova Health Care Services
2025-10-07 PHASE2
NCT03387475 COMPLETED
Myelodysplasia
University Hospital, Grenoble
2018-02-20 PHASE2
NCT06468423 COMPLETED
Pateints of β-thalassemia Major With Iron Overload
RESnTEC, Institute of Research
2023-01-01
NCT03203850 TERMINATED
Hereditary Hemochromatosis
Novartis Pharmaceuticals
2018-01-11 PHASE2
NCT05392101 COMPLETED
Sickle Cell Disease
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
2021-07-20 PHASE2
NCT03637556 COMPLETED
Thalassemia Major
DisperSol Technologies, LLC
2019-08-20 PHASE2
NCT03920657 TERMINATED
Myelodysplastic Syndromes
Fondazione Italiana Sindromi Mielodisplastiche-ETS
2019-10-04 PHASE2
NCT04423237 Recruiting
Iron Overload
University of Pisa|IRCCS Burlo Garofolo|University of Genova
2020-09-30 --
NCT02993224 COMPLETED
Transfusion-dependent Thalassemia; Non-transfusion-dependent Thalassemia
Novartis Pharmaceuticals
2017-07-27 PHASE2
NCT03372083 COMPLETED
Iron Overload
Novartis Pharmaceuticals
2018-01-16 PHASE4
NCT03920657 Terminated
Myelodysplastic Syndromes
Fondazione Italiana Sindromi Mielodisplastiche-ETS
2019-10-04 Phase 2
NCT02720536 COMPLETED
Chronic Iron Overload
Novartis Pharmaceuticals
2016-08-16 PHASE3
NCT02854722 UNKNOWN
Postmenopausal Osteoporosis
Second Affiliated Hospital of Soochow University
2018-01-15 PHASE2
NCT01718366 UNKNOWN
MDS
Groupe Francophone des Myelodysplasies
2013-02 PHASE1; PHASE2
NCT02943668 TERMINATED
Anemia; Myelodysplastic Syndrome
Fred Hutchinson Cancer Center
2017-03-02 PHASE2
NCT02069886 WITHDRAWN
Thalassemia (Transfusion Delendent)
Novartis Pharmaceuticals
2014-12 PHASE4
NCT02435212 COMPLETED
Transfusion-dependent Anemia
Novartis Pharmaceuticals
2015-10-21 PHASE2
NCT00940602 COMPLETED
Myelodysplastic Syndromes
Novartis Pharmaceuticals
2010-03-22 PHASE2
NCT03372083 Completed
Iron Overload
Novartis Pharmaceuticals|Novartis
2018-01-16 Phase 4
NCT02233504 COMPLETED
High Risk MDS or AML Patients
Abramson Cancer Center at Penn Medicine
2014-08 EARLY_PHASE1
NCT01825512 COMPLETED
Chronic Iron Overload
Consorzio per Valutazioni Biologiche e Farmacologiche
2014-03-17 PHASE3
NCT02474420 UNKNOWN
Beta-Thalassemia; Iron Overload
Kevin H.M. Kuo, MD, MSc, FRCPC
2015-06
NCT02761395 UNKNOWN
AL-Hijama in Thalassmia Major
Tanta University
2015-11
NCT01868477 COMPLETED
Low and Int 1-risk Myelodysplastic Syndrome
Novartis Pharmaceuticals
2014-01-28 PHASE2
NCT01892644 WITHDRAWN
Hemochromatosis; Myelodysplastic Syndromes
Haukeland University Hospital
2013-05 PHASE2
NCT02341495 TERMINATED
Acute Myeloid Leukemia
Elizabeth Henry
2013-02 PHASE2
NCT02038816 TERMINATED
Myelodysplastic Syndromes
Sunnybrook Health Sciences Centre
2014-03 PHASE2
NCT02413021 UNKNOWN
Acute Myeloid Leukemia; Acute Lymphoid Leukemia
Isfahan University of Medical Sciences
2016-05 PHASE1
NCT02663752 TERMINATED
Myelodysplastic Syndrome
Novartis Pharmaceuticals
2016-05-30 PHASE2
NCT02663752 Terminated
Myelodysplastic Syndrome
Novartis Pharmaceuticals|Novartis
2016-05-30 Phase 2
NCT02125877 COMPLETED
Chronic Iron Overload Due to Transfusion-dependant Anemias
Novartis Pharmaceuticals
2014-07-08 PHASE2
NCT01948817 WITHDRAWN
Focus on Transfusion Dependent Thalassemia Patients Who Are Inadequate Responders to Deferasirox > 35mg/kgQD
Novartis Pharmaceuticals
2014-02 PHASE2
NCT01927913 WITHDRAWN
Transfusional Iron Overload; Iron Overload; Iron Chelation; Beta-thalassemia; Transfusional Hemosiderosis; Iron Metabolism Disorders; Metabolic Diseases
Shire
2014-11-20 PHASE2
NCT01610297 COMPLETED
Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major
Novartis Pharmaceuticals
2013-09 PHASE4
NCT01709032 COMPLETED
Thalassemia Major With Severe Transfusional Iron Overload
Children's Hospital of Philadelphia
2012-09 PHASE1; PHASE2
NCT02159040 TERMINATED
High Risk MDS
Novartis Pharmaceuticals
2014-09-11 PHASE2
NCT01709838 COMPLETED
Non-transfusion Dependent Thalassemia
Novartis Pharmaceuticals
2012-12-06 PHASE4
NCT01459718 TERMINATED
Transfusion-dependent β-thalassemia Patients; Cardiac Iron Overload
Novartis Pharmaceuticals
2011-01 PHASE2
NCT01724138 WITHDRAWN
β-thalassemia Major
Novartis Pharmaceuticals
2013-06 PHASE4
NCT01254227 COMPLETED
Cardiac Iron Overload
Novartis Pharmaceuticals
2011-01 PHASE2
NCT00600938 COMPLETED
Transfusional Iron Overload; Transfusional Hemosiderosis
Novartis Pharmaceuticals
2007-11 PHASE2
NCT01511848 UNKNOWN
Beta-thalassemia Major; Sickle Cell Disease; Iron Hemosiderosis
Ain Shams University
2012-02 PHASE2; PHASE3
NCT01058369 TERMINATED
Myelodysplastic Syndromes
University of Erlangen-Nürnberg Medical School
2010-04 PHASE2
NCT01284946 UNKNOWN
Porphyria Cutanea Tarda
Assistance Publique - Hôpitaux de Paris
2011-01 PHASE2
NCT00901199 COMPLETED
Thalassemia; Iron Overload
Elliott Vichinsky
2007-09 PHASE2
NCT01326845 TERMINATED
Myelodysplastic Syndrome; Transfusional Iron Overload
Novartis Pharmaceuticals
2011-12 PHASE4
NCT00560820 COMPLETED
β-thalassemia; Transfusional Iron Overload
Novartis Pharmaceuticals
2007-09 PHASE1
NCT01278056 COMPLETED
Non-alcoholic Steatohepatitis; Increased Iron Storage / Disturbed Distribution
Crolll Gmbh
2010-03 PHASE1; PHASE2
NCT01273766 COMPLETED
Acute Undifferentiated Leukemia; Adult Acute Lymphoblastic Leukemia in Remission; Adult Acute Myeloid Leukemia in Remission; Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities; Adult Acute Myeloid Leukemia With Del(5q); Adult Acute Myeloid Leukemia With Inv(16)(p13;q22); Adult Acute Myeloid Leukemia With t(15;17)(q22;q12); Adult Acute Myeloid Leukemia With t(16;16)(p13;q22); Adult Acute Myeloid Leukemia With t(8;21)(q22;q22); Adult Grade III Lymphomatoid Granulomatosis; Adult Langerhans Cell Histiocytosis; Adult Nasal Type Extranodal NK/T-cell Lymphoma; Anaplastic Large Cell Lymphoma; Angioimmunoblastic T-cell Lymphoma; Contiguous Stage II Adult Burkitt Lymphoma; Contiguous Stage II Adult Diffuse Large Cell Lymphoma; Contiguous Stage II Adult Diffuse Mixed Cell Lymphoma; Contiguous Stage II Adult Diffuse Small Cleaved Cell Lymphoma; Contiguous Stage II Adult Immunoblastic Large Cell Lymphoma; Contiguous Stage II Adult Lymphoblastic Lymphoma; Contiguous Stage II Grade 1 Follicular Lymphoma; Contiguous Stage II Grade 2 Follicular Lymphoma; Contiguous Stage II Grade 3 Follicular Lymphoma; Contiguous Stage II Mantle Cell Lymphoma; Contiguous Stage II Marginal Zone Lymphoma; Contiguous Stage II Small Lymphocytic Lymphoma; Cutaneous B-cell Non-Hodgkin Lymphoma; de Novo Myelodysplastic Syndromes; Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue; Hepatosplenic T-cell Lymphoma; Intraocular Lymphoma; Mast Cell Leukemia; Myelodysplastic Syndrome With Isolated Del(5q); Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable; Myeloid/NK-cell Acute Leukemia; Nodal Marginal Zone B-cell Lymphoma; Noncontiguous Stage II Adult Burkitt Lymphoma; Noncontiguous Stage II Adult Diffuse Large Cell Lymphoma; Noncontiguous Stage II Adult Diffuse Mixed Cell Lymphoma; Noncontiguous Stage II Adult Diffuse Small Cleaved Cell Lymphoma; Noncontiguous Stage II Adult Immunoblastic Large Cell Lymphoma; Noncontiguous Stage II Adult Lymphoblastic Lymphoma; Noncontiguous Stage II Grade 1 Follicular Lymphoma; Noncontiguous Stage II Grade 2 Follicular Lymphoma; Noncontiguous Stage II Grade 3 Follicular Lymphoma; Noncontiguous Stage II Mantle Cell Lymphoma; Noncontiguous Stage II Marginal Zone Lymphoma; Noncontiguous Stage II Small Lymphocytic Lymphoma; Noncutaneous Extranodal Lymphoma; Peripheral T-cell Lymphoma; Previously Treated Myelodysplastic Syndromes; Primary Myelofibrosis; Recurrent Adult Acute Lymphoblastic Leukemia; Recurrent Adult Acute Myeloid Leukemia; Recurrent Adult Burkitt Lymphoma; Recurrent Adult Diffuse Large Cell Lymphoma; Recurrent Adult Diffuse Mixed Cell Lymphoma; Recurrent Adult Diffuse Small Cleaved Cell Lymphoma; Recurrent Adult Grade III Lymphomatoid Granulomatosis; Recurrent Adult Hodgkin Lymphoma; Recurrent Adult Immunoblastic Large Cell Lymphoma; Recurrent Adult Lymphoblastic Lymphoma; Recurrent Adult T-cell Leukemia/Lymphoma; Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma; Recurrent Grade 1 Follicular Lymphoma; Recurrent Grade 2 Follicular Lymphoma; Recurrent Grade 3 Follicular Lymphoma; Recurrent Mantle Cell Lymphoma; Recurrent Marginal Zone Lymphoma; Recurrent Mycosis Fungoides/Sezary Syndrome; Recurrent Small Lymphocytic Lymphoma; Refractory Anemia; Refractory Multiple Myeloma; Secondary Acute Myeloid Leukemia; Secondary Myelofibrosis; Small Intestine Lymphoma; Splenic Marginal Zone Lymphoma; Stage I Adult Burkitt Lymphoma; Stage I Adult Diffuse Large Cell Lymphoma; Stage I Adult Diffuse Mixed Cell Lymphoma; Stage I Adult Diffuse Small Cleaved Cell Lymphoma; Stage I Adult Hodgkin Lymphoma; Stage I Adult Immunoblastic Large Cell Lymphoma; Stage I Adult Lymphoblastic Lymphoma; Stage I Adult T-cell Leukemia/Lymphoma; Stage I Cutaneous T-cell Non-Hodgkin Lymphoma; Stage I Grade 1 Follicular Lymphoma; Stage I Grade 2 Follicular Lymphoma; Stage I Grade 3 Follicular Lymphoma; Stage I Mantle Cell Lymphoma; Stage I Marginal Zone Lymphoma; Stage I Multiple Myeloma; Stage I Mycosis Fungoides/Sezary Syndrome; Stage I Small Lymphocytic Lymphoma; Stage II Adult Hodgkin Lymphoma; Stage II Adult T-cell Leukemia/Lymphoma; Stage II Cutaneous T-cell Non-Hodgkin Lymphoma; Stage II Multiple Myeloma; Stage II Mycosis Fungoides/Sezary Syndrome; Stage III Adult Burkitt Lymphoma; Stage III Adult Diffuse Large Cell Lymphoma; Stage III Adult Diffuse Mixed Cell Lymphoma; Stage III Adult Diffuse Small Cleaved Cell Lymphoma; Stage III Adult Hodgkin Lymphoma; Stage III Adult Immunoblastic Large Cell Lymphoma; Stage III Adult Lymphoblastic Lymphoma; Stage III Adult T-cell Leukemia/Lymphoma; Stage III Cutaneous T-cell Non-Hodgkin Lymphoma; Stage III Grade 1 Follicular Lymphoma; Stage III Grade 2 Follicular Lymphoma; Stage III Grade 3 Follicular Lymphoma; Stage III Mantle Cell Lymphoma; Stage III Marginal Zone Lymphoma; Stage III Multiple Myeloma; Stage III Mycosis Fungoides/Sezary Syndrome; Stage III Small Lymphocytic Lymphoma; Stage IV Adult Burkitt Lymphoma; Stage IV Adult Diffuse Large Cell Lymphoma; Stage IV Adult Diffuse Mixed Cell Lymphoma; Stage IV Adult Diffuse Small Cleaved Cell Lymphoma; Stage IV Adult Hodgkin Lymphoma; Stage IV Adult Immunoblastic Large Cell Lymphoma; Stage IV Adult Lymphoblastic Lymphoma; Stage IV Adult T-cell Leukemia/Lymphoma; Stage IV Cutaneous T-cell Non-Hodgkin Lymphoma; Stage IV Grade 1 Follicular Lymphoma; Stage IV Grade 2 Follicular Lymphoma; Stage IV Grade 3 Follicular Lymphoma; Stage IV Mantle Cell Lymphoma; Stage IV Marginal Zone Lymphoma; Stage IV Mycosis Fungoides/Sezary Syndrome; Stage IV Small Lymphocytic Lymphoma; Testicular Lymphoma; Untreated Adult Acute Lymphoblastic Leukemia; Untreated Adult Acute Myeloid Leukemia; Waldenstrom Macroglobulinemia
Wake Forest University Health Sciences
2011-01 PHASE2
NCT00654589 COMPLETED
Iron Overload
Novartis Pharmaceuticals
2008-02 PHASE4
NCT00673608 COMPLETED
Hemoglobinopathies; Myelodysplastic Syndromes; Other Inherited or Acquired Anaemia; MPD Syndrome; Diamond-Blackfan Anemia; Other Rare Anaemias; Transfusional Iron Overload
Novartis
2007-11 PHASE4
NCT01159067 TERMINATED
Iron Overload; Accelerated Phase Chronic Myelogenous Leukemia; Adult Acute Lymphoblastic Leukemia in Remission; Adult Acute Myeloid Leukemia in Remission; Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities; Adult Acute Myeloid Leukemia With Del(5q); Adult Acute Myeloid Leukemia With Inv(16)(p13;q22); Adult Acute Myeloid Leukemia With t(15;17)(q22;q12); Adult Acute Myeloid Leukemia With t(16;16)(p13;q22); Adult Acute Myeloid Leukemia With t(8;21)(q22;q22); Atypical Chronic Myeloid Leukemia, BCR-ABL Negative; Blastic Phase Chronic Myelogenous Leukemia; Chronic Eosinophilic Leukemia; Chronic Myelomonocytic Leukemia; Chronic Neutrophilic Leukemia; Chronic Phase Chronic Myelogenous Leukemia; de Novo Myelodysplastic Syndromes; Disseminated Neuroblastoma; Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue; Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable; Nodal Marginal Zone B-cell Lymphoma; Noncontiguous Stage II Adult Burkitt Lymphoma; Noncontiguous Stage II Adult Diffuse Large Cell Lymphoma; Noncontiguous Stage II Adult Diffuse Mixed Cell Lymphoma; Noncontiguous Stage II Adult Diffuse Small Cleaved Cell Lymphoma; Noncontiguous Stage II Adult Immunoblastic Large Cell Lymphoma; Noncontiguous Stage II Adult Lymphoblastic Lymphoma; Noncontiguous Stage II Grade 1 Follicular Lymphoma; Noncontiguous Stage II Grade 2 Follicular Lymphoma; Noncontiguous Stage II Grade 3 Follicular Lymphoma; Noncontiguous Stage II Mantle Cell Lymphoma; Noncontiguous Stage II Marginal Zone Lymphoma; Noncontiguous Stage II Small Lymphocytic Lymphoma; Poor Prognosis Metastatic Gestational Trophoblastic Tumor; Previously Treated Myelodysplastic Syndromes; Primary Myelofibrosis; Recurrent Adult Acute Lymphoblastic Leukemia; Recurrent Adult Acute Myeloid Leukemia; Recurrent Adult Burkitt Lymphoma; Recurrent Adult Diffuse Large Cell Lymphoma; Recurrent Adult Diffuse Mixed Cell Lymphoma; Recurrent Adult Diffuse Small Cleaved Cell Lymphoma; Recurrent Adult Hodgkin Lymphoma; Recurrent Adult Immunoblastic Large Cell Lymphoma; Recurrent Adult Lymphoblastic Lymphoma; Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma; Recurrent Grade 1 Follicular Lymphoma; Recurrent Grade 2 Follicular Lymphoma; Recurrent Grade 3 Follicular Lymphoma; Recurrent Malignant Testicular Germ Cell Tumor; Recurrent Mantle Cell Lymphoma; Recurrent Marginal Zone Lymphoma; Recurrent Mycosis Fungoides/Sezary Syndrome; Recurrent Neuroblastoma; Recurrent Ovarian Epithelial Cancer; Recurrent Ovarian Germ Cell Tumor; Recurrent Small Lymphocytic Lymphoma; Refractory Chronic Lymphocytic Leukemia; Refractory Hairy Cell Leukemia; Relapsing Chronic Myelogenous Leukemia; Secondary Acute Myeloid Leukemia; Secondary Myelodysplastic Syndromes; Splenic Marginal Zone Lymphoma; Stage I Multiple Myeloma; Stage II Multiple Myeloma; Stage II Ovarian Epithelial Cancer; Stage III Adult Burkitt Lymphoma; Stage III Adult Diffuse Large Cell Lymphoma; Stage III Adult Diffuse Mixed Cell Lymphoma; Stage III Adult Diffuse Small Cleaved Cell Lymphoma; Stage III Adult Hodgkin Lymphoma; Stage III Adult Immunoblastic Large Cell Lymphoma; Stage III Adult Lymphoblastic Lymphoma; Stage III Chronic Lymphocytic Leukemia; Stage III Grade 1 Follicular Lymphoma; Stage III Grade 2 Follicular Lymphoma; Stage III Grade 3 Follicular Lymphoma; Stage III Malignant Testicular Germ Cell Tumor; Stage III Mantle Cell Lymphoma; Stage III Marginal Zone Lymphoma; Stage III Multiple Myeloma; Stage III Ovarian Epithelial Cancer; Stage III Small Lymphocytic Lymphoma; Stage IIIA Breast Cancer; Stage IIIB Breast Cancer; Stage IIIC Breast Cancer; Stage IV Adult Burkitt Lymphoma; Stage IV Adult Diffuse Large Cell Lymphoma; Stage IV Adult Diffuse Mixed Cell Lymphoma; Stage IV Adult Diffuse Small Cleaved Cell Lymphoma; Stage IV Adult Hodgkin Lymphoma; Stage IV Adult Immunoblastic Large Cell Lymphoma; Stage IV Adult Lymphoblastic Lymphoma; Stage IV Breast Cancer; Stage IV Chronic Lymphocytic Leukemia; Stage IV Grade 1 Follicular Lymphoma; Stage IV Grade 2 Follicular Lymphoma; Stage IV Grade 3 Follicular Lymphoma; Stage IV Mantle Cell Lymphoma; Stage IV Marginal Zone Lymphoma; Stage IV Ovarian Epithelial Cancer; Stage IV Small Lymphocytic Lymphoma
City of Hope Medical Center
2010-07 PHASE2
NCT01250951 COMPLETED
Myelodysplastic Syndrome; Thalassemia
Novartis Pharmaceuticals
2009-12 PHASE4
NCT00564941 COMPLETED
Myelodysplastic Syndromes; Beta-Thalassemia
Novartis Pharmaceuticals
2007-12 PHASE4
NCT00873041 COMPLETED
Non-transfusion Dependent Thalassemia
Novartis Pharmaceuticals
2008-11 PHASE2
NCT00879242 COMPLETED
Beta Thalassemia Transfusion Dependent
Novartis Pharmaceuticals
2009-02 PHASE2
NCT01335035 COMPLETED
Iron Overload
Novartis Pharmaceuticals
2008-12 PHASE4
NCT00631163 COMPLETED
Chronic Anemia; Transfusional Hemosiderosis
Novartis Pharmaceuticals
2007-10 PHASE2
NCT00481143 COMPLETED
Myelodysplastic Syndromes; Transfusion Dependent Iron Overload
Novartis Pharmaceuticals
2007-05 PHASE4
NCT00981370 TERMINATED
Anemia, Sickle Cell; Transfusion Hemosiderosis
Children's Hospital Los Angeles
2009-04 PHASE3
NCT00845871 COMPLETED
Transfusional Hemosiderosis
Novartis Pharmaceuticals
2009-05 PHASE4
NCT00432627 COMPLETED
Hepatic Impairment
Novartis Pharmaceuticals
2006-12 PHASE1
NCT01349699 COMPLETED
Systemic Inflammatory Process; Anemia
Radboud University Medical Center
2010-02
NCT00599326 COMPLETED
Porphyria Cutanea Tarda
University of Texas Southwestern Medical Center
2008-01 PHASE3
NCT00469560 COMPLETED
Myelodysplastic Syndromes; Hemosiderosis
Gruppo Italiano Malattie EMatologiche dell'Adulto
2007-06 PHASE3
NCT00419770 COMPLETED
Mucormycosis
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
2007-10 PHASE2
NCT00447694 COMPLETED
Beta-thalassemia; Iron Overload
Novartis
2006-02 PHASE2
NCT00602446 TERMINATED
Breast Cancer; Iron Overload; Leukemia; Lymphoma; Multiple Myeloma and Plasma Cell Neoplasm; Myelodysplastic Syndromes; Neuroblastoma; Ovarian Cancer
Masonic Cancer Center, University of Minnesota
2007-08 PHASE2
NCT00738413 UNKNOWN
Thalassemia
Weill Medical College of Cornell University
2008-08 PHASE1; PHASE2
NCT00171821 COMPLETED
Transfusion-dependent Iron Overload
Novartis Pharmaceuticals
2005-04 PHASE3
NCT00749515 COMPLETED
Transfusion-dependent Hemachromatosis; Thalassemia Major; Sickle Cell Disease
Boston Children's Hospital
2008-03 PHASE4
NCT00235391 COMPLETED
Thalassemia; Sickle Cell Disease; Diamond Blackfan Anemia; Myelofibrosis
Novartis Pharmaceuticals
2005-10 PHASE3
NCT00303329 COMPLETED
Anemia; Hemosiderosis
Novartis Pharmaceuticals
2004-03 PHASE2
NCT02198508 COMPLETED
Beta-thalassemia Major
China Medical University Hospital
2007-07
NCT00171301 COMPLETED
Beta-thalassemia Major; Hemosiderosis; Iron Overload; Rare Anemia
Novartis Pharmaceuticals
2005-06 PHASE4
NCT00171210 COMPLETED
Transfusional Iron Overload in β-thalassemia
Novartis Pharmaceuticals
2004-10 PHASE3
NCT00110617 COMPLETED
Sickle Cell Disease; Iron Overload; Hemolytic Anemia
Novartis Pharmaceuticals
2005-05 PHASE2
NCT00110266 COMPLETED
Myelodysplastic Syndrome; Iron Overload
Novartis Pharmaceuticals
2005-07-25 PHASE2
NCT00390858 COMPLETED
Transfusional Iron Overload; β-thalassemia Major; Pediatric Rare Anemia
Novartis Pharmaceuticals
2003-09 PHASE2
NCT01033747 COMPLETED
Liver Iron Overload
Novartis Pharmaceuticals
2003-02 PHASE2; PHASE3
NCT00117507 COMPLETED
Myelodysplastic Syndromes; Iron Overload
Novartis Pharmaceuticals
2005-09 PHASE4
NCT00395629 COMPLETED
Iron Overload; Hereditary Hemochromatosis
Novartis Pharmaceuticals
2006-08 PHASE1; PHASE2
NCT00171171 COMPLETED
Beta-Thalassemia; Hemosiderosis
Novartis
2004-05 PHASE3
NCT00061763 COMPLETED
Beta-thalassemia; Myelodysplastic Syndromes; Fanconi Syndrome; Anemia, Diamond-Blackfan; Anemia, Aplastic
Novartis Pharmaceuticals
2003-05 PHASE2
NCT00379483 COMPLETED
Transfusional Iron Overload
Novartis Pharmaceuticals
2002-07 PHASE2
NCT00419172 COMPLETED
Healthy
Novartis
2007-01 PHASE1
NCT00427505 COMPLETED
Healthy
Novartis
2006-07 PHASE1

(data from https://clinicaltrials.gov, updated on 2026-07-21)

Check the Deferasirox (ICL670) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Deferasirox selectively binds ferric iron to form a stable complex, inducing cellular iron depletion that downregulates anti-apoptotic Bcl-xL expression and causes apoptotic cell death. By eliminating excess toxic iron, this mechanism mitigates tissue iron toxicity and provides therapeutic benefit in clinical conditions such as transfusional iron overload, beta-thalassemia, and myelodysplastic syndromes.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

Note: Technical data last updated: Sep 1, 2026.