Thalassemia Major: Clinical Trials

50 trials for Thalassemia Major, newest first

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28 completed, 13 unknown, 4 active not recruiting, 2 recruiting. 12 Not applicable, 10 Phase 2, 8 Phase 4, 5 Phase 1, 4 Phase 3.

By phase and status

PhaseTrialsStatus
Not applicable12Completed 5, Unknown 4, Active not recruiting 1, Recruiting 1, Suspended 1
Phase 210Completed 6, Active not recruiting 1, Recruiting 1, Terminated 1, Unknown 1
Phase 48Completed 5, Unknown 3
Phase 15Completed 2, Active not recruiting 1, Terminated 1, Unknown 1
Phase 34Completed 3, Unknown 1

Follow Thalassemia Major: new trials by email Literature Review of Thalassemia Major trials Research Report on Thalassemia Major

Trials newest first · largest first

Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

TrialPhaseStatusSponsorEnrollmentStartUpdated
Effect of Incentive Respiratory Training on Pulmonary Functions and Functional Capacity in Children With B-thalassemia Major
Incentive spirometer exercises, Diaphragmatic breathing, Pursed lip breathing: - Incentive Spirometer, Pulmonary Function
Not applicableRecruitingRawan Mohammed Khairy Mostafa Elsawy502025-04-072025-08-08
Growth Evaluation, Health Promotion, and Clinical Management in Children and Adolescents With Thalassemia
MENBS clinical interventions - Thalassemia Intermedia, Thalassemia Major
Not applicableActive not recruitingInstitute of Hematology & Blood Diseases Hospital, China3852024-12-012026-09-18
Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia
Thalidomide - Fetal Hemoglobin, Thalassemia Major
Phase 2RecruitingNational Institute of Blood and Marrow Transplant (NIBMT), Pakistan542024-04-222025-05-30
NAC Effect on Iron Overload and Blood Transfusion in β-thalassemia Major
n-acetylcystine - Thalassemia Major
Phase 1UnknownAssiut University1002024-03-232023-08-23
Safety and Efficacy Evaluation of γ-globin Reactivated Autologous Hematopoietic Stem Cells
γ-globin reactivated autologous hematopoietic stem cells - Thalassemia Major
Not applicableSuspendedBioray Laboratories52023-12-252024-02-28
Longitudinal Study on the QoL of Pediatric Patients After HSCT and Its Influencing Factors
Conventional nursing - Thalassemia Major, Acute Leukemia
UnknownSun Yat-Sen Memorial Hospital of Sun Yat-Sen University2502023-10-302023-10-23
Safety and Efficacy Evaluation of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells
VGB-Ex01 - Thalassemia, Beta, Thalassemia Major
Not applicableUnknownInstitute of Hematology & Blood Diseases Hospital, China22023-08-312023-10-17
Study to Evaluate the Long- Term Safety and Efficacy of Luspatercept in Subjects Who Received at Least One Dose of Luspatercept in the Compassionate Use Phase
Luspatercept (ACE-536) - Thalassemia Major
Active not recruitingFondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia1402023-05-302025-04-06
To Evaluate Long- Term Safety and Efficacy of Luspatercept
Luspatercept (ACE-536) - Thalassemia Major
Active not recruitingFondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia3502023-04-302025-03-13
Study on the Effect of Different Risk Factors on the Growth Parameters of Thalassemic Patients in Assiut (AUCH)
serum ferritin and AST - Thalassemia Major
UnknownAssiut University502022-07-022022-03-31
The Safety and Efficiency of Luspatercept in Chinese Adults With Transfusion Dependent β-thalassemia: a Real-world Study
Luspatercept Injectable Product - Thalassemia Major, Transfusion-dependent Anemia
Phase 4UnknownSun Yat-sen University202022-07-012022-07-18
EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)
EDIT-301 - Transfusion Dependent Beta Thalassemia, Hemoglobinopathies
Phase 1 / Phase 2Active not recruitingEditas Medicine, Inc.92022-04-292025-04-02
Frequency of COVID-19 Antibodies in Patients With Hereditary Hematological Diseases
SARS-CoV-2 S1/S2 IgG - Thalassemia Major, Covid19
CompletedUniversity of Milano Bicocca632020-07-142020-11-09
Thalassemic Iron Overload Cardiomyopathy is Ameliorated by Taurine Supplementation
Taurine, Placebo - Thalassemia Major
Not applicableUnknownUniversity Health Network, Toronto602020-06-012023-10-03
Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies
CliniMACS - Sickle Cell Disease, Thalassemia Major
Not applicableCompletedTimothy Olson82020-05-142026-04-28
Hydroxy Urea, Omega 3, Nigella Sativa,Honey on Oxidative Stress and Iron Chelation in Pediatric Major Thalassemia
Omega 3, Nigella Sativa Oil, Hydroxyurea - Iron Overload, Oxidative Stress
Phase 2 / Phase 3CompletedBeni-Suef University3502019-11-012021-01-27
Pilot Study to Assess the Safety, PK and Iron Chelating Activity of DST-0509 (Deferasirox) in Thalassemia Patients Refractory to Chelation
DST-0509, Jadenu, Exjade - Thalassemia Major
Phase 2CompletedDisperSol Technologies, LLC252019-08-202022-02-01
Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study
Busulfan, Cyclophosphamide, Fludarabine - Thalassemia Major
Phase 4CompletedFirst Affiliated Hospital of Guangxi Medical University8232019-07-052024-08-09
The Effect of N-acetylcysteine on Oxidative Stress Status and Iron Overload in Thalassemia Major
N-acetyl cysteine - Thalassemia Major
Phase 1CompletedBeni-Suef University1002019-06-032020-02-07
Observing the Changes of Endocrine and Metabolism in Patients With Thalassemia Major
Thalassemia Major
UnknownNational Taiwan University Hospital1002019-04-012021-02-18

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Narrower conditions

beta-Thalassemia (50)  ·  Thalassemia (12)  ·  Anemia, Sickle Cell (6)  ·  Iron Overload (5)  ·  Genetic Diseases, Inborn (4)  ·  Anemia, Aplastic (3)  ·  Anemia, Diamond-Blackfan (3)  ·  Hematologic Diseases (3)  ·  Hemoglobinopathies (3)  ·  Osteoporosis (3)  ·  Rare Diseases (3)  ·  Bone Diseases, Metabolic (2)  ·  Fanconi Anemia (2)  ·  Hemochromatosis (2)  ·  Red-Cell Aplasia, Pure (2)

Broader conditions

Anemia (50)  ·  Anemia, Hemolytic (50)  ·  Anemia, Hemolytic, Congenital (50)  ·  Congenital, Hereditary, and Neonatal Diseases and Abnormalities (50)  ·  Hemic and Lymphatic Diseases (50)  ·  Hematologic Diseases (47)  ·  Hemoglobinopathies (47)  ·  Genetic Diseases, Inborn (46)  ·  Thalassemia (38)  ·  Metabolic Diseases (11)  ·  Nutritional and Metabolic Diseases (11)  ·  Pathological Conditions, Signs and Symptoms (9)  ·  Pathologic Processes (6)  ·  Disease Attributes (5)  ·  Iron Metabolism Disorders (5)

Interventions

Busulfan (4)  ·  Deferoxamine (4)  ·  Cyclophosphamide (3)  ·  Deferiprone (3)  ·  Tacrolimus (3)  ·  fludarabine (3)  ·  luspatercept (3)  ·  Deferasirox (2)  ·  Influenza Vaccines (2)  ·  Isoflurophate (2)  ·  Methotrexate (2)  ·  Mycophenolic Acid (2)  ·  thymoglobulin (2)  ·  6-amino-1-hydroxyhexane-1,1-diphosphonate (1)  ·  Abatacept (1)

Sponsors

Assiut University (3)  ·  Beni-Suef University (2)  ·  Ente Ospedaliero Ospedali Galliera (2)  ·  First Affiliated Hospital of Guangxi Medical University (2)  ·  Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia (2)  ·  Institute of Hematology & Blood Diseases Hospital, China (2)  ·  National Center for Research Resources (NCRR) (2)  ·  University Health Network, Toronto (2)  ·  University of Milano Bicocca (2)  ·  Ain Shams University (1)  ·  Ann & Robert H Lurie Children's Hospital of Chicago (1)  ·  ApoPharma (1)  ·  Azienda Ospedaliera V. Cervello (1)  ·  Azienda Ospedaliero-Universitaria di Modena (1)  ·  Bioray Laboratories (1)

Countries

United States (14)  ·  Italy (12)  ·  China (8)  ·  Egypt (5)  ·  Canada (3)  ·  Greece (3)  ·  Thailand (2)  ·  Turkey (Türkiye) (2)  ·  Brazil (1)  ·  Iran (1)  ·  Lebanon (1)  ·  Pakistan (1)  ·  Saudi Arabia (1)  ·  Singapore (1)  ·  Taiwan (1)

Top investigators

Yongrong Lai, MD (2)  ·  Ahmed F Mahmoud Hussein, MS.c (1)  ·  Alan Richard Cohen (1)  ·  Andrea Biondi, MD, PhD (1)  ·  Antonio Piga, M.D. (1)  ·  Antonio Piga, MD (1)  ·  Barbara B Biesecker (1)  ·  Catherine J. Wu, MD (1)  ·  Catherine Wu, MD (1)  ·  Deborah Chirnomas, MD (1)  ·  Ernesto Palummeri, MD (1)  ·  Ersi Voskaridou, Doctor (1)  ·  Gary M. Brittenham, M.D. (1)

Results publications

PMID 37975597, PMID 14550808, PMID 19236376, PMID 19724055, PMID 22571408, PMID 24081970, PMID 25222041, PMID 26653680, PMID 27862048, PMID 30381400, PMID 31907538, PMID 32311145, PMID 32314798, PMID 32330288, PMID 32394480, PMID 32395210, PMID 39891377, PMID 41730859, PMID 41931048, PMID 41984996

NIH funding

2014 (1)