thalassemia
Information
- Disease name
- thalassemia
- Disease ID
- DOID:10241
- Description
- "A microcytic anemia characterized by decreased synthesis of one or more hemoglobin polypeptide chains." [url:https\://www.genome.gov/Genetic-Disorders/Thalassemia, url:https\://www.ncbi.nlm.nih.gov/pubmed/11283697]
Disease area statistics
[No Data.]
Chromosome band
[No Data.]
Annotation
| Genes | Mutation | Description | Source | Links |
|---|
| NCT ID | Status | Phase | Summary | Start date | Completion date |
|---|---|---|---|---|---|
| NCT03249831 | Active, not recruiting | Phase 1 | A Blood Stem Cell Transplant for Sickle Cell Disease | January 4, 2019 | November 17, 2024 |
| NCT03655678 | Active, not recruiting | Phase 2/Phase 3 | A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia | September 14, 2018 | August 2024 |
| NCT02165007 | Active, not recruiting | Phase 1 | Haploidentical Hematopoietic Stem Cell Transplantation | January 2015 | March 2026 |
| NCT03333486 | Active, not recruiting | Phase 2 | Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer | December 7, 2017 | August 28, 2024 |
| NCT03692052 | Active, not recruiting | Phase 2 | A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia | March 20, 2019 | September 30, 2030 |
| NCT00125788 | Completed | Phase 2 | L-Glutamine Therapy for Sickle Cell Anemia and Sickle ß0 Thalassemia | March 2004 | July 2008 |
| NCT00153985 | Completed | Phase 2 | Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies | March 2004 | July 2009 |
| NCT00176852 | Completed | Phase 2/Phase 3 | Stem Cell Transplant for Hemoglobinopathy | June 2002 | January 2020 |
| NCT00235391 | Completed | Phase 3 | Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload | October 2005 | October 2008 |
| NCT00346242 | Completed | Phase 4 | Evaluation of Efficacy of Zoledronic Acid in Patients With Haemoglobin Syndromes (Thalassemia and Sicle Cell Anaemia) and Risk of Skeletal Events | March 2004 | |
| NCT00427661 | Completed | N/A | A Pilot Study of HSCT for Patients With High-risk Hemoglobinopathy Using a Nonmyeloablative Preparative Regimen | June 2002 | May 2014 |
| NCT00456443 | Completed | Invasive Infections in Children With Hemoglobinopathies | January 2006 | June 2006 | |
| NCT00456690 | Completed | N/A | Endocrine and Nutritional Assessment in B Thalassemia Major | May 2007 | December 2014 |
| NCT00459732 | Completed | N/A | Zinc & Bone Health in Thalassemia: The Think Zinc Study | April 2006 | February 2011 |
| NCT00502788 | Completed | Phase 2 | Evaluating the Safety of Two Medications to Treat Hepatitis C in People With Thalassemia (The HepC Study) | May 2003 | August 2006 |
| NCT00520559 | Completed | T2* in Transfusion Dependant Anemia, MI, LVF, Normal Patients | January 2007 | August 2007 | |
| NCT00735540 | Completed | Clinical Diagnosis of Teenagers Admitted to Pediatric Departments | May 2008 | May 2010 | |
| NCT00000595 | Completed | Phase 2 | Evaluation of Subcutaneous Desferrioxamine as Treatment for Transfusional Hemochromatosis | January 1978 | September 1994 |
| NCT00000623 | Completed | Thalassemia (Cooley's Anemia) Clinical Research Network (TCRN) | July 2000 | July 2006 | |
| NCT00001203 | Completed | Deferoxamine for the Treatment of Hemochromatosis | April 22, 1985 | November 9, 2015 | |
| NCT00004982 | Completed | Phase 1 | Combination Iron Chelation Therapy | December 1998 | November 2002 |
| NCT00872170 | Completed | Phase 2/Phase 3 | Sildenafil to Improve Exercise Capacity in People With Thalassemia and Pulmonary Hypertension | March 2009 | November 2010 |
| NCT00872339 | Completed | Assessment of Pain in People With Thalassemia | March 2009 | December 2010 | |
| NCT00872833 | Completed | Assessment of Pain in People With Thalassemia Who Are Treated With Regular Blood Transfusions | July 2009 | October 2010 | |
| NCT00901199 | Completed | Phase 2 | Combined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload | September 2007 | October 2012 |
| NCT00957931 | Completed | Phase 2 | Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs | March 2009 | August 2013 |
| NCT00971698 | Completed | The Spleen in Sickle Cell Anemia and Sickle Cell Thalassemia | February 2009 | August 2010 | |
| NCT00971880 | Completed | Blood Transfusions in Thalassemia Patients, Complications and Adverse Effects | May 2009 | November 2010 | |
| NCT00972231 | Completed | Iron Overload and Growth Velocity in Thalassemia and Sickle Cell Anemia | January 2009 | December 2010 | |
| NCT01048905 | Completed | Phase 2 | Glutamine Therapy for Hemolysis-Associated Pulmonary Hypertension | March 2009 | March 2014 |
| NCT01114776 | Completed | Multi-Center Study of Iron Overload: Pilot Study | November 1, 2009 | September 30, 2013 | |
| NCT01158794 | Completed | Genes Influencing Iron Overload State | September 21, 2010 | April 17, 2019 | |
| NCT01250951 | Completed | Phase 4 | This Study Will Evaluate Efficacy and Safety of Deferasirox in Patients With Myelodysplastic Syndromes (MDS), Thalassemia and Rare Anemia Types Having Transfusion-induced Iron Overload. | December 2009 | September 2011 |
| NCT01316549 | Completed | Study of Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation | January 1, 2011 | April 30, 2016 | |
| NCT01369719 | Completed | N/A | Efficacy and Safety of Desferal Versus Osveral in Transfusional Iron Overload | February 2010 | May 2011 |
| NCT01395199 | Completed | Phase 3 | Amlodipine in the Prevention and Treatment of Iron Overload in Patients With Thalassemia Major | August 2012 | February 2015 |
| NCT01443312 | Completed | Demographic, Clinical, Laboratory and Genetical Characteristics of Patients With Beta Thalassemia Intermedia | October 2011 | December 2014 | |
| NCT01549080 | Completed | Study of Effects of YisuiShengxueGranules on Thalassemia | July 2011 | January 2012 | |
| NCT01597765 | Completed | N/A | Effect of Antioxidant Cocktail in Beta-thalassemia/Hb E Patients | June 2009 | June 2011 |
| NCT01772680 | Completed | N/A | Zinc and Diabetes in Patients With Thalassemia: a Pilot Study | November 2012 | May 2015 |
| NCT01905774 | Completed | Renal Function Among Thalassemia Patients Treated by a Oral Chelator Deferasirox | March 2011 | August 2015 | |
| NCT01911871 | Completed | Multicenter Observational Study on Myocardial Iron Overload in 3 Multitransfused Populations | March 2012 | March 2015 | |
| NCT01913548 | Completed | Multi-Center Study of Iron Overload: Survey Study (MCSIO) | March 31, 2010 | October 31, 2013 | |
| NCT02065492 | Completed | Phase 2/Phase 3 | Amlodipine for Myocardial Iron in Thalassemia | February 2014 | November 2015 |
| NCT02133560 | Completed | N/A | Use of Mobile Technology for Intensive Training in Medication Management | May 2014 | August 1, 2016 |
| NCT02904252 | Completed | The Prevalence and Severity of HCV Infection in Thalassemia Major and Thalassemia Intermedia in Siriraj Hospital | January 1, 2016 | October 31, 2016 | |
| NCT03032666 | Completed | Phase 4 | Sofosbuvir/Ledipasvir for Hepatitis C Genotype 1-6 in Patients With Transfusion-Dependent Thalassemia: An Open Label Trial | May 1, 2017 | August 6, 2018 |
| NCT03095326 | Completed | Phase 4 | Pneumococcal Vaccination for Splenectomised Thalassemia Major Patients in Indonesia | September 1, 2013 | February 1, 2014 |
| NCT03117192 | Completed | Phase 4 | Zinc Supplementation on Cellular Immunity in Thalassemia Major | September 1, 2013 | February 1, 2014 |
| NCT03165864 | Completed | Phase 1 | Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Healthy Volunteers | May 9, 2017 | September 25, 2018 |
| NCT03170245 | Completed | Adiponectin, IL-6 and hsC-RP in Relation to Carotid Intima-media Thickness in B-thalassemia Patients | August 1, 2018 | February 15, 2021 | |
| NCT03342404 | Completed | Phase 2 | A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia | February 5, 2018 | November 28, 2022 |
| NCT03513328 | Completed | Phase 1/Phase 2 | Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation | June 15, 2018 | February 19, 2023 |
| NCT03609814 | Completed | Study of Clofarabine and Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation (HCT) | January 26, 2016 | June 30, 2020 | |
| NCT03609827 | Completed | Study of Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients | September 1, 2015 | May 31, 2021 | |
| NCT03609840 | Completed | Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients | May 24, 2017 | July 31, 2023 | |
| NCT03651102 | Completed | Phase 2/Phase 3 | Efficacy and Safety of Low Dose Thalidomide in Transfusion Dependent Thalassemia | January 1, 2018 | March 1, 2022 |
| NCT03673085 | Completed | Phase 1 | Clinical Study of CN128 in Thalassemia Patients | January 5, 2018 | December 17, 2019 |
| NCT03715972 | Completed | Cerebrovascular Reserve and White Matter Disease in Patients With Chronic Anemia | July 15, 2018 | December 31, 2022 | |
| NCT03919812 | Completed | N/A | Immunomodulatory Vitamin D in Thalassemia | April 1, 2018 | August 15, 2018 |
| NCT03935633 | Completed | Phase 1 | Short-term Clinical Study of CN128 in Thalassemia Patients | December 3, 2018 | December 17, 2019 |
| NCT04582110 | Completed | The Role of OCTA in Patients Affected by Beta Thalassemia | October 1, 2019 | February 28, 2020 | |
| NCT04614779 | Completed | Phase 2 | Long-term Clinical Study of CN128 in Thalassemia Patients | September 30, 2020 | August 18, 2022 |
| NCT05255445 | Completed | Red Blood Cell - IMProving trAnsfusions for Chronically Transfused Recipients | March 16, 2022 | March 31, 2024 | |
| NCT05389891 | Completed | N/A | Hemoglobinopathy Nursing Program and Pediatric Nursing Students | February 27, 2017 | May 11, 2017 |
| NCT05529550 | Completed | Assessment of Nutritional Status and Role of Insulin-like Growth Factor-1 in Children | October 1, 2020 | December 1, 2021 | |
| NCT00661726 | Completed | Phase 2 | Evaluating the Safety and Effectiveness of Decitabine in People With Thalassemia Intermedia | January 2008 | September 2010 |
| NCT00661804 | Completed | Evaluating People With Thalassemia: The Thalassemia Longitudinal Cohort (TLC) Study | May 2007 | June 2011 | |
| NCT00669305 | Completed | Bone Marrow for Hemoglobinopathy Research | May 15, 2008 | August 29, 2019 | |
| NCT00707850 | Completed | Phase 4 | Pegasys® Plus Ribavirin in Thalassemic Patients With Hepatitis C Virus Infection | May 2007 | September 2009 |
| NCT00712738 | Completed | Phase 1 | Oral Nifedipine to Treat Iron Overload | June 20, 2008 | September 22, 2010 |
| NCT00730314 | Completed | Phase 1/Phase 2 | Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells | August 2008 | August 2015 |
| NCT00735488 | Completed | Sickle Cell Anemia Screening and Prevention in Northern Israel | May 2008 | March 2009 | |
| NCT00000588 | Completed | Phase 2 | Chelation Therapy of Iron Overload With Pyridoxal Isonicotinoyl Hydrazone | June 5, 1989 | March 31, 1995 |
| NCT00744692 | Completed | Phase 1 | Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders | October 2008 | April 2014 |
| NCT00789516 | Completed | The Change of Coagulation Markers in Children With β-thalassemia Disease After Stem Cell Transplantation | June 2006 | December 2009 | |
| NCT04208529 | Enrolling by invitation | Phase 3 | A Long-term Follow-up Study in Participants Who Received CTX001 | January 20, 2021 | September 2039 |
| NCT04310059 | Not yet recruiting | N/A | Effect of Folic Acid Supplementation in Pregnant Women Having Thalassaemia Trait | July 1, 2024 | July 31, 2026 |
| NCT06058260 | Not yet recruiting | Assessment of Cognitive Function and Quality of Life in Thalassemic Children at Sohag University Hospital | October 20, 2023 | October 20, 2024 | |
| NCT05790980 | Not yet recruiting | Assessment of Quality of Life in Thalassemic Patients at Assiut University Hospital: A Single-center Experience | April 1, 2023 | June 1, 2025 | |
| NCT05736419 | Recruiting | Phase 2 | A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT) | February 9, 2023 | February 9, 2026 |
| NCT05508932 | Recruiting | Atrial Fibrillation in Beta-Thalassemia | September 1, 2022 | July 1, 2032 | |
| NCT06313398 | Recruiting | Early Phase 1 | Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling | May 17, 2024 | June 15, 2029 |
| NCT01758042 | Recruiting | N/A | Bone Marrow and Kidney Transplant for Patients With Chronic Kidney Disease and Blood Disorders | November 2012 | July 2027 |
| NCT06210139 | Recruiting | Pharmacotherapy Prescribing Pattern and the Adherence Level to Iron Chelation Therapy in Thalassemia | March 28, 2023 | February 28, 2024 | |
| NCT02105766 | Recruiting | Phase 2 | Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure | April 21, 2014 | December 31, 2024 |
| NCT04987489 | Recruiting | Phase 2 | A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease | December 1, 2021 | December 1, 2025 |
| NCT06213402 | Recruiting | RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs) | November 30, 2021 | November 2036 | |
| NCT02386800 | Recruiting | Phase 4 | CINC424A2X01B Rollover Protocol | March 5, 2015 | September 16, 2027 |
| NCT04398628 | Recruiting | ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders | September 30, 2020 | December 2035 | |
| NCT05355766 | Recruiting | Phase 2 | Long-term Clinical Study of CN128 in Thalassemia With Sever Liver Iron Overloaded Patients | June 2, 2022 | December 31, 2024 |
| NCT05356195 | Recruiting | Phase 3 | Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) | May 3, 2022 | May 2026 |
| NCT06250595 | Recruiting | European Rare Blood Disorders Platform (ENROL) | July 1, 2022 | July 2037 | |
| NCT05477563 | Recruiting | Phase 3 | Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease | August 2, 2022 | February 2025 |
| NCT03367546 | Terminated | Phase 2 | Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases | July 2, 2018 | December 19, 2022 |
| NCT00578292 | Terminated | N/A | Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO | February 2004 | May 2016 |
| NCT01319851 | Terminated | N/A | Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation | September 2010 | September 2013 |
| NCT00034528 | Terminated | Phase 2 | Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or Thalassemia | September 2001 | November 2003 |
| NCT02512679 | Terminated | Phase 2 | Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells | February 2007 | February 2014 |
| NCT02065869 | Terminated | Phase 1/Phase 2 | Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant | April 2014 | September 7, 2021 |
| NCT02504619 | Terminated | Phase 1/Phase 2 | Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies | April 4, 2016 | December 2017 |
| NCT03825341 | Terminated | Phase 2 | Hydroxyurea Therapy: Optimizing Access in Pediatric Populations Everywhere | June 10, 2019 | January 20, 2022 |
| NCT01534676 | Terminated | N/A | Effects of Transfusion of Older Stored Red Cells | February 2012 | March 2013 |
| NCT00586209 | Terminated | Phase 2 | L-Glutamine Therapy for Sickle Cell Anemia | February 6, 2004 | November 2009 |
| NCT03733249 | Terminated | Phase 1/Phase 2 | Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study | January 2017 | April 14, 2023 |
| NCT00040469 | Terminated | Phase 2 | Bone Marrow Transplant From Related Donor for Patients With High Risk Hemoglobinopathies | August 2000 | November 21, 2003 |
| NCT00040417 | Terminated | Phase 2 | Bone Marrow Transplant From Donor Using Less Toxic Conditioning for Patient With High Risk Hemoglobinopathies | August 2000 | November 21, 2003 |
| NCT00481221 | Unknown status | Detection of β Thalassemia Carriers by Red Cell Parameters Obtained From the H2 Automatic Counter | March 2007 | December 31, 2020 | |
| NCT02772016 | Unknown status | Early Phase 1 | Therapeutic Effect of Colla Corii Asini on Improving Anemia and Hemoglobin Composition in Pregnant Women With Thalassemia | March 2015 | |
| NCT02995707 | Unknown status | Phase 2 | The Effective and Safety of Thalidomide in NTDT | September 2016 | March 2018 |
| NCT00738413 | Unknown status | Phase 1/Phase 2 | Iron Balance Study of Deferasirox, Deferoxamine and the Combination of Both | August 2008 | December 2009 |
| NCT04706585 | Unknown status | Thalassemia Knowledge in Young People of Indonesia | June 1, 2021 | October 10, 2021 | |
| NCT03184844 | Unknown status | Phase 2 | The Effective and Safety of Thalidomide in TI | May 2, 2017 | May 2, 2019 |
| NCT00327639 | Unknown status | Factors Promoting Increased Rate and Success of Pregnancy in the Thalassemia Population in Toronto | July 2005 | ||
| NCT03374111 | Unknown status | Phase 4 | Colla Corii Asini Treating Anemia in Pregnant Women With Thalassemia(Presenting the Syndrome of Blood Deficiency) | March 1, 2018 | December 30, 2019 |
| NCT03392298 | Unknown status | Phase 4 | Study on the Mechanism of Colla Corri Asini in the Treatment of Thalassemia Patients With Pregnancy Anemia | December 1, 2017 | February 28, 2019 |
| NCT03402191 | Unknown status | Phase 4 | L-arginine Versus Sildenafil in Children With Beta Thalassemia Associated With Pulmonary Hypertension | July 2016 | December 2023 |
| NCT05326503 | Unknown status | N/A | Inhibitory Effect of a Polyphenol Supplement on Dietary Iron Absorption in Adults With Thalassemia | May 1, 2022 | December 31, 2022 |
| NCT03728322 | Unknown status | Early Phase 1 | iHSCs With the Gene Correction of HBB Intervent Subjests With β-thalassemia Mutations | January 2019 | January 2021 |
| NCT01935661 | Unknown status | A Case Control Study to Evaluate the Cognitive and Brain Function of β-thalassemia Patients. | September 2013 | December 31, 2020 | |
| NCT01419704 | Withdrawn | Phase 1/Phase 2 | Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies | May 2011 | May 2016 |
- Disase is a (Disease Ontology)
- DOID:11252
- Cross Reference ID (Disease Ontology)
- GARD:7756
- Cross Reference ID (Disease Ontology)
- ICD10CM:D56
- Cross Reference ID (Disease Ontology)
- ICD9CM:282.4
- Cross Reference ID (Disease Ontology)
- MESH:D013789
- Cross Reference ID (Disease Ontology)
- NCI:C35069
- Cross Reference ID (Disease Ontology)
- SNOMEDCT_US_2023_03_01:191192008
- Cross Reference ID (Disease Ontology)
- UMLS_CUI:C0039730
- Exact Synonym (Disease Ontology)
- Sickle-cell thalassemia with crisis
- Exact Synonym (Disease Ontology)
- Sickle-cell thalassemia without crisis
- Exact Synonym (Disease Ontology)
- thalassemia Hb-S disease with crisis
- Exact Synonym (Disease Ontology)
- thalassemia Hb-S disease without crisis
- MeSH unique ID (MeSH (Medical Subject Headings))
- D013789