Beta-Thalassemia (DBCOND0027982)

Identifiers

Synonyms
Β -Thalassemia / Β Thalassemia / Thalassemia, Beta / B-Thalassemia / Β-thalassemia / Thalassaemia beta / Beta Thalassaemia / Beta Thalassemia / B Thalassemia / Thalassemia beta / Beta thalassemia (disorder) / Beta-thalassaemia / Thalassaemia intermedia / Thalassemia intermedia (disorder) / Thalassemia intermedia / Mediterranean anemia / Mediterranean anaemia / Thalassemia major / Cooley's anaemia / Thalassaemia major / Thalassemia major (disorder) / Homozygous beta thalassemia (disorder) / Cooley's anemia

Associated Data

Indicated Drugs and Targets
DrugDescriptionTargets
Betibeglogene autotemcel
A hematopoietic stem cell-based gene therapy used in the treatment of β-thalassemia in adult and pediatric patients who require regular red blood cell transfusions.
Luspatercept
An erythroid maturation agent used to treat anemia secondary to beta thalassemia, myelodysplastic syndromes, and neoplasms.
No drug targets
Clinical Trials
IdentifierTitleDrug(s)PurposePhaseStatus
NCT06302491
A Study of Safety and Efficiency of AND017 in Patients With β-thalassemiaNo drug interventionstreatment2recruiting
NCT04411082
A Study of IMR-687 in Subjects With Beta Thalassemiatreatment2terminated
NCT06153784
Thalidomide and Hydroxyurea Combination in β-Thalassemia Patientstreatment2 / 3completed
NCT03183375
The Efficacy and Safety of HYDROXYUREA in Management of Beta Thalassemia Patients in Karachi Pakistantreatment2unknown_status
NCT05132270
Clinical Experience of Thalidomide in Thalassemic Patientstreatment2 / 3completed
NCT05736419
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)treatment2recruiting
NCT06298630
Long-term Follow-up Study of BRL-101 for TDTNo drug interventionsNot AvailableNot Availablenot_yet_recruiting
NCT05799118
Study of the Role of Genetic Modifiers in HemoglobinopathiesNo drug interventionsNot AvailableNot Availablerecruiting
NCT06041620
Safety and Efficacy Evaluation of Autologous CRISPR-Cas12b Edited Hematopoietic Stem CellsNo drug interventionstreatmentNot Availablerecruiting
NCT06314529
Long-term Follow-up Study of BHC001 for TDTNo drug interventionsNot AvailableNot Availablerecruiting
NCT03591900
The Use of CGMS to Detect Alterations of Blood Glucose in Thalassemic PatientsNo drug interventionsscreeningNot Availablecompleted
NCT01749540
Study to Evaluate the Safety and Efficacy of Luspatercept (ACE-536) in Participants With Beta-thalassemia (A536-04/MK-6143-002)treatment2completed
NCT06440603
EKLF Gene Expression in β-thalassemiaNo drug interventionsNot AvailableNot Availablenot_yet_recruiting
NCT05494333
Correlation Between Pulmonary Functions and Physical Fitness in Children With β-thalassemiaNo drug interventionsNot AvailableNot Availableunknown_status
NCT03358498
Quality of Life and Treatment Satisfaction in β-Thalassemia Patients Receiving DeferasiroxNo drug interventionsNot AvailableNot Availableunknown_status
NCT05745532
Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major PatientsNo drug interventionstreatment0recruiting
NCT05864170
the Safety and Efficacy Evaluation of HGI-001 Injection in Patients With Transfusion-Dependent β-ThalassemiaNo drug interventionstreatment0recruiting
NCT00560820
Phase I Study to Examine the Effect of Deferasirox on Renal Hemodynamics in β-thalassemia Patients With Transfusional Iron Overloadtreatment1completed
NCT05773729
Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent Beta-thalassemiaNo drug interventionstreatmentNot Availablerecruiting
NCT03802201
Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemiatreatment2completed
NCT04054921
Safety Study for Beta Thalassemia Subjects on PTG-300treatment2completed
NCT02268409
Extension Study to Evaluate the Safety and Efficacy of Luspatercept in Participants With β-Thalassemia Previously Enrolled in A536-04 (A536-06/MK-6143-004)treatment2completed
NCT06465550
A Phase 1 Study of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Treating β-thalassemia MajorNo drug interventionstreatment1recruiting
NCT03275051
Long-term Follow-up of Subjects Treated With OTL-300 for Transfusion Dependent Beta-thalassemia Study (TIGET-BTHAL)No drug interventionsotherNot Availableactive_not_recruiting
NCT05357482
Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemiatreatment1 / 2recruiting
NCT00001958
Hydroxyurea to Treat Beta-Thalassemia (Cooley's Anemia)treatment2completed
NCT04929574
Evaluation of Heart Status in Patients of Beta Thalassemia Using EchocardiogramNo drug interventionsNot AvailableNot Availablerecruiting
NCT00790127
Phase 1/2 Study of HQK-1001 in Patients With Beta Thalassemiatreatment1 / 2completed
NCT06073860
A Post-Marketing Surveillance Study to Assess Safety of Luspatercept in Korean Patients With Myelodysplastic Syndrome or β-thalassemiaNot AvailableNot Availablerecruiting
NCT01049854
CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplanttreatment2completed
NCT00408447
Stem Cell Transplant in Sickle Cell Disease and Thalassemiatreatment2active_not_recruiting
NCT04917978
Association of Hb F Level With Clinical Severity of Beta ThalassemiaNo drug interventionsNot AvailableNot Availablerecruiting
NCT04918056
Genetic Variants Affecting the Clinical Severity of Beta ThalassemiaNo drug interventionsNot AvailableNot Availableunknown_status
NCT00159042
Genetic Factors Affecting the Severity of Beta ThalassemiaNo drug interventionsNot AvailableNot Availablecompleted
NCT00005934
5-Azacytidine and Phenylbutyrate to Treat Severe Thalassemiatreatment2completed
NCT01931644
At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic ConditionsNo drug interventionsNot AvailableNot Availablecompleted
NCT02597595
Effects of Spirulina on Cardiac Functions in Children With Beta Thalassemia MajorNo drug interventionspreventionNot Availablecompleted
NCT06647979
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe Β-HemoglobinopathiesNo drug interventionstreatment1not_yet_recruiting
NCT06308159
An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-ThalassemiaNo drug interventionstreatment1 / 2recruiting
NCT03655678
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemiatreatment2 / 3active_not_recruiting
NCT06364774
ALS20-101 Lentiviral Gene Therapy for Beta ThalassemiaNo drug interventionstreatment1 / 2recruiting
NCT05567458
A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia.treatment2active_not_recruiting
NCT06271512
A Study of Participants with Β-Thalassemia Treated with Betibeglogene AutotemcelNo drug interventionsNot AvailableNot Availablerecruiting
NCT03462784
Complications in Children With B- Thalassemia MajorNo drug interventionsNot AvailableNot Availableunknown_status
NCT02274233
Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-Thalassemiatreatment1terminated
NCT02453477
Gene Therapy for Transfusion Dependent Beta-thalassemiaNo drug interventionstreatment1 / 2unknown_status
NCT03894605
Dermatological Abnormalities in Beta-thalassemia MajorNo drug interventionsNot AvailableNot Availableunknown_status
NCT03961828
Hyalornic Acid Level in β-Thalassemic Children Treated for Hepatitis C Virustreatment4completed
NCT00171171
A Study of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosistreatment3completed
NCT03877809
Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patientstreatment2completed
NCT03822585
Detection Of β-thalassemia Carriers In AssiutNo drug interventionspreventionNot Availableunknown_status
NCT01927913
Treatment of Iron Overload Requiring Chelation Therapytreatment2withdrawn
NCT01186419
Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron Overloadtreatment2completed
NCT03271541
A Study of Bitopertin (RO4917838) in Adults With Non-Transfusion-Dependent (NTD) Beta-Thalassemiatreatment2completed
NCT05506358
Evaluation of Low-cost Techniques for Detecting Sickle Cell Disease and β-thalassemia in Nepal and CanadaNo drug interventionsdiagnosticNot Availablecompleted
NCT05635266
Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research InitiativesNo drug interventionsNot AvailableNot Availablerecruiting
NCT02474420
Amlodipine as Adjuvant Treatment to Iron Chelation for Prevention of Cardiac Iron Overload in Thalassemia PatientstreatmentNot Availableunknown_status
NCT04064060
A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trialstreatment3recruiting
NCT00029380
Cord Blood Transplantation for Sickle Cell Anemia and Thalassemiatreatment2completed
NCT04208529
A Long-term Follow-up Study in Participants Who Received CTX001other3enrolling_by_invitation
NCT06065189
Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia MajorNo drug interventionstreatment0recruiting
NCT02626689
To Document the Burden of Illness on the Quality of Life and the Impact on Healthcare Utilization in (Beta) β-thalassemia Subjects Who Are Transfusion Dependent (TD) and Non-transfusion Dependent (NTD) Receiving Standard of CareNo drug interventionsNot AvailableNot Availablecompleted
NCT04947540
Iron Status in BTM With Blood TransfusionNo drug interventionsNot AvailableNot Availableunknown_status
NCT01419704
Phase I/II Pilot Study of Mixed Chimerism to Treat HemoglobinopathiesNo drug interventionstreatment1 / 2withdrawn
NCT04364269
Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Preliminary Efficacy of VIT-2763 in β-thalassaemiatreatment2completed
NCT06539169
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare DiseasesNo drug interventionsNot AvailableNot Availablerecruiting
NCT05577312
Safety and Efficacy Evaluation of BRL-101 in Subjects With Transfusion-Dependent β-ThalassemiaNo drug interventionstreatment1enrolling_by_invitation
NCT00000595
Evaluation of Subcutaneous Desferrioxamine as Treatment for Transfusional Hemochromatosistreatment2completed
NCT00061750
Safety & Efficacy of ICL670 vs. Deferoxamine in Beta-thalassemia Patients With Iron Overload Due to Blood Transfusionstreatment3completed
NCT03655223
Early Check: Expanded Screening in NewbornsNo drug interventionsNot AvailableNot Availableenrolling_by_invitation
NCT03728543
the Efficacy and Safety of Sugammadex in Children 0-2 Years Oldtreatment2 / 3unknown_status
NCT06328764
CS-101 in Patients With β-thalassemiaNo drug interventionstreatment0enrolling_by_invitation
NCT02906202
A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 GenotypeNo drug interventionstreatment3completed
NCT02604433
An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemiatreatment3completed
NCT03381833
A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemiatreatment2terminated
NCT00103753
Combined Chelation Treatment With Deferiprone and Deferoxamine in Thalassemia Majortreatment4unknown_status
NCT03222453
Thalassemia Treatment Based on the Stem Cell TechnologyNo drug interventionstreatmentNot Availableunknown_status
NCT05370677
The Prevelence of IVS 1-6 (T-C) [HBB:c.92 +6 T-C] Gene Mutation in Suspected Cases of β Thalassemia in Assiut University HospitalsNo drug interventionsNot AvailableNot Availablenot_yet_recruiting
NCT03889977
Resistance Exercise on Postprandial Hyperglycemia in Patients With B-thalassemia Exhibiting Resistance to InsulinNo drug interventionstreatmentNot Availablecompleted
NCT06512298
Plasticizer Exposure and Its Consequences on HealthNo drug interventionsNot AvailableNot Availablenot_yet_recruiting
NCT01996683
Efficacy and Safety of Efficacy and Safety of Continued Iron Chelation Therapy In Poly-transfused Thalassemia Patients With Low Serum Ferritin (< 500 ng/ml)treatmentNot Availableunknown_status
NCT00733811
Efficacy Study of the Use of Sequential DFP-DFO Versus DFPtreatment4completed
NCT01016093
Zoledronic Acid for the Prevention of Bone Loss Post-bone Marrow Transplantation for Thalassemia Major Patientstreatment2 / 3unknown_status
NCT00000588
Chelation Therapy of Iron Overload With Pyridoxal Isonicotinoyl HydrazoneNo drug interventionstreatment2completed
NCT05133388
The Prevelence of HBB c.93-21 G-A in β Thalassemia PatientsNo drug interventionsNot AvailableNot Availablerecruiting
NCT03207009
A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-ThalassemiaNo drug interventionstreatment3completed
NCT01206075
Evaluating the Safety and Effectiveness of Mozobil Mobilization in Adults With Beta-Thalassemia MajortreatmentNot Availablecompleted
NCT00115349
Combination Therapy Compared With Single-Drug Therapy in Patients With Cardiac Diseasestreatment2terminated
NCT04219449
Thrombophilia In Beta ThalassemiaNo drug interventionsNot AvailableNot Availablenot_yet_recruiting
NCT01039636
Safety and Pharmacokinetic Study of Escalating Multiple Doses of an Iron Chelator in Patients With Iron Overloadtreatment1completed
NCT00006136
Phase II Study of Arginine Butyrate With or Without Epoetin Alfa in Patients With Thalassemia Intermediatreatment2completed
NCT00564941
Evaluating the Efficacy of Deferasirox in Transfusion Dependent Chronic Anaemias (Myelodysplastic Syndrome, Beta-thalassaemia Patients) With Chronic Iron Overloadtreatment4completed
NCT04143724
Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemiatreatment2recruiting
NCT06479616
A Long-term Follow-up Study in Participants Who Received CS-101No drug interventionsotherNot Availablerecruiting
NCT04067258
Choroidal Thickness in Beta-thalassemia PatientsNo drug interventionsNot AvailableNot Availablecompleted
NCT04938635
Efficacy and Safety Study of Multiple Doses of VIT-2763 in Adults With Transfusion-dependent Beta-thalassemiatreatment2withdrawn
NCT00061763
Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemiastreatment2completed
NCT05477563
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Diseasetreatment3recruiting
NCT05469230
Assessment of Cognitive Function, Fatigue and Health Related Quality of Life in Children With Beta ThalassemiaNo drug interventionsNot AvailableNot Availablecompleted
NCT02950857
A Study of EPEG in Beta Thalassemia PatientsNo drug interventionstreatment1completed
NCT06568926
Adherence of Beta Thalssemia Patients to Oral Chelation TherapyNo drug interventionshealth_services_researchNot Availablerecruiting
NCT04099966
AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletiontreatment2recruiting
NCT06024876
A Clinical Study Evaluating the Safety and Efficacy of CS-101 in Treating Subjects With β-thalassemiaNo drug interventionstreatment0active_not_recruiting
NCT00447694
Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatmenttreatment2completed
NCT00336362
Evaluating the Safety of G-CSF Mobilization in Individuals With Beta Thalassemia MajortreatmentNot Availablecompleted
NCT00069862
Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-Thalassemiatreatment1 / 2completed
NCT04973280
Study to Evaluate Additional Risk Minimisation Measures (aRMMs) for REBLOZYL Among Healthcare Professionals (HCPs)No drug interventionsNot AvailableNot Availablecompleted
NCT03351829
Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral VectorNo drug interventionstreatmentNot Availableunknown_status
NCT03801889
SP-420 in Subjects With Transfusion-dependent Beta-Thalassemia or Other Rare Anemiastreatment2withdrawn
NCT06239389
Comparison Of Efficacy And Safety Of Thalidomide Vs Hydroxyurea In Thalassemia Patients: A Single-Centre Pilot Study.treatment2completed
NCT06490601
Long Term Beta Thalassemia Treatment: Findings From The Extension Periodtreatment2active_not_recruiting
NCT01092169
Prevalence Of Microalbuminuria Among Children Suffering From Sickle Cell Nephropathy and Sickle Cell/Beta-ThalassemiaNo drug interventionsNot AvailableNot Availableunknown_status
NCT06509581
Vitamin E Supplementation for Children With Transfusion Dependent Beta Thalassemia on Different Iron Chelation RegimenNo drug interventionstreatmentNot Availablecompleted
NCT05356195
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)treatment3recruiting
NCT04247750
Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients (THALA-RAP)treatment2unknown_status
NCT00000623
Thalassemia (Cooley's Anemia) Clinical Research Network (TCRN)Not AvailableNot Availablecompleted
NCT01363908
Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron Overloadtreatment2terminated
NCT05105659
Assessment of Cognitive Functions, Psychological Symptoms, and Quality of Life Among B Thalassemia Patients and Their Clinical Correlates.No drug interventionsNot AvailableNot Availableunknown_status
NCT06377865
Thyroid Function , HbA1c in Relation to Ferritin Level in Adullt Patient e B ThalassemiaNo drug interventionsNot AvailableNot Availablenot_yet_recruiting
NCT01863173
Effect of Metoprolol on Thalassemia Cardiomyopathytreatment2 / 3completed