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Results for “Sickle Beta Plus Thalassemia”

54 trials

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Showing 20 of 54 results

Not applicableLooking for participantsNCT06539169
What this trial is testing

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

Who this might be right for
Alpha-ThalassemiaBeta-ThalassemiaAmyloidosis+14 more
xCures 1,000
Not applicableEnrolling By InvitationNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
Testing effectiveness (Phase 2)Study completedNCT01186419
What this trial is testing

Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron Overload

Who this might be right for
Transfusional Iron OverloadBeta-thalassemia
Shire 51
Testing effectiveness (Phase 2)Active Not RecruitingNCT05357482
What this trial is testing

Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia

Who this might be right for
Sickle Cell AnemiaBeta Thalassemia
National Heart, Lung, and Blood Institute (NHLBI) 40
Testing effectiveness (Phase 2)Looking for participantsNCT03653338
What this trial is testing

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

Who this might be right for
Sickle Cell AnemiaBeta-thalassemia MajorDiamond-blackfan Anemia
Paul Szabolcs 5
Not applicableActive Not RecruitingNCT05145062
What this trial is testing

Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.

Who this might be right for
Blood and Lymphatic Diseases
Sangamo Therapeutics 8
Testing effectiveness (Phase 2)Looking for participantsNCT04008368
What this trial is testing

Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels

Who this might be right for
Myeloid Chimerism
National Heart, Lung, and Blood Institute (NHLBI) 32
Testing effectiveness (Phase 2)Ended earlyNCT01363908
What this trial is testing

Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron Overload

Who this might be right for
Transfusional Iron OverloadBeta-Thalassemia
Shire 30
Early research (Phase 1)Study completedNCT00005783
What this trial is testing

A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients With Sickling Disorders

Who this might be right for
Hemoglobin SC DiseaseSickle Cell Anemia
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) 50
Large-scale testing (Phase 3)Enrolling By InvitationNCT04208529
What this trial is testing

A Long-term Follow-up Study in Participants Who Received CTX001

Who this might be right for
Beta-ThalassemiaThalassemiaSickle Cell Disease+4 more
Vertex Pharmaceuticals Incorporated 160
Large-scale testing (Phase 3)UnknownNCT01511848
What this trial is testing

Study Of Efficacy,Safety of Combined Deferasirox and Deferiprone Versus Combined Deferiprone and Desferal In Conditions of Iron Overload

Who this might be right for
Beta-thalassemia MajorSickle Cell DiseaseIron Hemosiderosis
Ain Shams University 60
Not applicableActive Not RecruitingNCT05675436
What this trial is testing

Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease

Who this might be right for
Sickle Cell AnemiaSickle Cell ThalassemiaSickle Cell Pain+5 more
National Heart, Lung, and Blood Institute (NHLBI) 6
Not applicableEnrolling By InvitationNCT06363760
What this trial is testing

A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301

Who this might be right for
Sickle Cell DiseaseTransfusion-dependent Beta-ThalassemiaHemoglobinopathies
Editas Medicine, Inc. 54
Not applicableActive Not RecruitingNCT04691323
What this trial is testing

The Longitudinal Relationship of HU Adherence to HRQOL, Barriers to Adherence and Habit in SCD.

Who this might be right for
Sickle Cell DiseaseSickle B+ ThalassemiaSickle Beta Zero Thalassemia+1 more
Ann & Robert H Lurie Children's Hospital of Chicago 68
Not applicableStudy completedNCT00005893
What this trial is testing

Study of Allogeneic Bone Marrow Transplantation Using Matched, Related Donors in Patients With Nonmalignant Hematologic Disorders

Who this might be right for
NeutropeniaSickle Cell AnemiaThalassemia Major+1 more
Fairview University Medical Center
Early research (Phase 1)Study completedNCT03492931
What this trial is testing

PK Study of Ticagrelor in Children Aged Less Than 24 Months, With Sickle Cell Disease (HESTIA4)

Who this might be right for
Sickle Cell Disease
AstraZeneca 21
Testing effectiveness (Phase 2)UnknownNCT00061568
What this trial is testing

Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias

Who this might be right for
Congenital Hemolytic AnemiaSickle Cell Disease
National Heart, Lung, and Blood Institute (NHLBI) 130
Testing effectiveness (Phase 2)Enrolling By InvitationNCT04432623
What this trial is testing

The BENeFiTS Trial in Beta Thalassemia Intermedia

Who this might be right for
Beta Thalassemia IntermediaSickle Cell Disease
Phoenicia BioScience 36
Large-scale testing (Phase 3)Looking for participantsNCT05477563
What this trial is testing

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

Who this might be right for
Beta-ThalassemiaThalassemiaHematologic Diseases+4 more
Vertex Pharmaceuticals Incorporated 26
Not applicableStudy completedNCT06831799
What this trial is testing

ERN-EuroBloodNet Registry on Patients With Rare Red Blood Cell Defects and COVID-19

Who this might be right for
HemoglobinopathiesSickle Cell DiseaseSickle Cell Anemia+21 more
Hospital Universitari Vall d'Hebron Research Institute 684
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