Clinical Trials on Thalassemia Alpha

Total 612 results

University of California, San Francisco

Recruiting

International Registry of Patients With Alpha Thalassemia (ATM Registry)

Alpha-Thalassemia | Alpha Thalassemia Major | Alpha Thalassemia Minor

United States
University of California, San Francisco
California Institute for Regenerative Medicine (CIRM)

Enrolling by invitation

In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM)

Hemoglobinopathies | Alpha Thalassemia Major | Thalassemia Major | Fetal Hydrops | Hemoglobinopathy; With Thalassemia | Fetal Anemia | Alpha; Thalassemia | Thalassemia Alpha | A-Thalassemia

United States
Agios Pharmaceuticals, Inc.

Active, not recruiting

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) (ENERGIZE-T)

Transfusion-dependent Alpha-Thalassemia | Transfusion-dependent Beta-Thalassemia

Spain, Taiwan, Thailand, United States, France, Canada, Malaysia, Germany, Netherlands, Bulgaria, United Kingdom, Turkey, Italy, Greece, United Arab Emirates, Brazil, Denmark, Lebanon, Saudi Arabia
Agios Pharmaceuticals, Inc.

Active, not recruiting

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT) (ENERGIZE)

Non-Transfusion-dependent Alpha-Thalassemia | Non-Transfusion-dependent Beta-Thalassemia

Spain, Taiwan, Thailand, United Kingdom, Malaysia, United States, Netherlands, Bulgaria, Turkey, Italy, Canada, Brazil, France, United Arab Emirates, Denmark, Greece, Lebanon, Saudi Arabia
HaEmek Medical Center, Israel

Completed

Demographic, Clinical and Laboratory Characteristics of Children With Alpha Thalassemia in Northern Israel

Hemolytic Anemia | Thalassemia Alpha

Israel
The 923rd Hospital of Joint Logistics Support Force...
Guangzhou Reforgene Medicine Co., Ltd.

Recruiting

Safety and Efficacy of RM-004 Cells for Hemoglobin H-Constant Spring Disease

Hemoglobinopathies | Hereditary Diseases | Alpha Thalassemia Hemoglobin H Constant Spring

China
Cyprus Institute of Neurology and Genetics

Recruiting

Study of the Role of Genetic Modifiers in Hemoglobinopathies (INHERENT)

Sickle Cell Disease | Hemoglobinopathies | Thalassemia Alpha | Thalassemia, Beta

United States, Denmark, Spain, Belgium, Portugal, Congo, The Democratic Republic of the, Greece, Argentina, Brunei Darussalam, Cyprus, Israel, Italy, Nigeria, Malaysia, Angola, Pakistan
National Institute of Allergy and Infectious Diseases...

Recruiting

Screening for Alpha Thalassemia in Healthy Volunteers

Alpha Thalassemia

United States
Deborah Rund

Completed

Genetics of Alpha Thalassemia in Israeli Ethnic Groups

Alpha Thalassemia

Israel
Talaris Therapeutics Inc.
Duke University

Withdrawn

Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies

Anemia, Sickle Cell | Thalassemia | Beta-Thalassemia | Bone Marrow Failure Syndromes | Diamond-Blackfan Anemia | Alpha-Thalassemia | Complex and Transfusion-dependent Hemoglobinopathies

United States
Shenzhen Hemogen

Recruiting

the Safety and Efficacy Evaluation of HGI-002 Injection in Patients With Transfusion-Dependent α-Thalassemia

α-thalassemia

China
First Affiliated Hospital of Guangxi Medical University
Genmedicn Biopharma Ltd.

Recruiting

A Study Evaluating the Safety and Efficacy of the GMCN-508A Drug Product in Transfusion-dependent α-Thalassemia Participants

Transfusion-dependent α-Thalassemia

China
IRCCS San Raffaele
Telethon Institute for Gene Therapy (OSR-TIGET)

Active, not recruiting

Long-term Follow-up of Subjects Treated With OTL-300 for Transfusion Dependent Beta-thalassemia Study (TIGET-BTHAL)

Beta Thalassaemia

Italy
EdiGene (GuangZhou) Inc.

Active, not recruiting

A Safety and Efficacy Study Evaluating ET-01 in Subjects With Transfusion Dependent β-Thalassaemia (ET-01)

Transfusion Dependent Beta-Thalassaemia

China
EdiGene (GuangZhou) Inc.
PLA 923 Hospital

Active, not recruiting

A Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia.

Transfusion Dependent Beta-Thalassaemia

China
Institute of Hematology & Blood Diseases Hospital...
EdiGene Inc.; The Affiliated Hospital Of Guizhou Medical University; Zunyi Medical...

Active, not recruiting

Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia

Transfusion Dependent Beta-Thalassaemia

China
Tanta University

Unknown

Study of The Therapeutic Benefits of Al-hijamah in Children With Beta Thalassemia Major (Al-hijamah)

AL-Hijama in Thalassmia Major

Egypt
National Institute of Allergy and Infectious Diseases...

Recruiting

Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants

Malaria | Sickle Cell Disease | Alpha and Beta Thalassemia | Human Physiology

United States
Chinese University of Hong Kong

Recruiting

In-utero Hematopoietic Stem Cell Transplantation for the Treatment of Fetuses With Bart's Hydrops Fetalis Syndrome

Haemoglobin Barts Hydrops

Hong Kong
Shanghai First Maternity and Infant Hospital

Unknown

Precision Medical Research of Non-immune Fetal Hydrops (NIFH)-From Prenatal Diagnosis to Intrauterine Treatment

Fetal Hydrops
Baxalta now part of Shire
Arriva Pharmaceuticals, Inc.

Completed

Study of the Effect of Aerosolized, Recombinant Alpha 1-Antitrypsin on Epithelial Lining Fluid Analytes in Subjects With Alpha 1-Antitrypsin Deficiency

Alpha1-antitrypsin Deficiency

United States
Grifols Therapeutics LLC

Recruiting

A Study to Evaluate Safety, Tolerability and Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% in Participants With Alpha1-Antitrypsin Deficiency

Alpha1-Antitrypsin Deficiency

United States
Vertex Pharmaceuticals Incorporated

Completed

Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype

Alpha1-Antitrypsin Deficiency

United States, Germany, Canada, Ireland, Sweden, United Kingdom
Grifols Therapeutics LLC
Grifols Japan K.K.

Completed

Safety and Pharmacokinetics of Alpha-1 MP (Alpha1-proteinase Inhibitor (Human), Modified Process) in Participants With Alpha1-Antitrypsin Deficiency

Alpha1-Antitrypsin Deficiency

Japan
Baxalta now part of Shire

Completed

GLASSIA Safety, Immunogenicity, and Bronchoalveolar Lavage Study

Alpha1-antitrypsin Deficiency

United States, Canada
Baxalta now part of Shire

Terminated

Aralast alpha1-proteinase Inhibitor Surveillance Study

Alpha1-antitrypsin Deficiency

United States
Obstetrix Medical Group

Terminated

Amniotic Fluid Tandem Mass Spectrometry for Pregnancies Complicated by NIH and Severe Symmetrical IUGR (TandemMS)

Fetal Growth Retardation | Hydrops Fetalis

United States
Grifols Therapeutics LLC
Grifols Japan K.K.

Completed

Long-term Safety of Alpha1-Proteinase Inhibitor (Human) in Japanese Subjects With Alpha1 Antitrypsin Deficiency (GTI1401-OLE)

Alpha1-Antitrypsin Deficiency

Japan
Baxalta now part of Shire
Arriva Pharmaceuticals, Inc.

Completed

Safety Study of an Aerosolized, Recombinant Alpha 1-Antitrypsin in Subjects With Alpha 1-Antitrypsin Deficiency

Alpha1-antitrypsin Deficiency

United States
Z Factor Limited
Hammersmith Medicines Research; Centessa Pharmaceuticals plc

Terminated

A Single Ascending and Repeated Dose Study of Oral ZF874 in Healthy Volunteers and PiXZ Subjects

Alpha1 Anti-Trypsin Deficiency

United Kingdom
Baxalta now part of Shire

Completed

GLASSIA Infusion Rate Study

Healthy Volunteers | Alpha1-antitrypsin Deficiency

United States
RWTH Aachen University

Unknown

Liver Disease in Patients With alpha1-antitrypsin Deficiency

alpha1-antitrypsin Deficiency

Germany
Takeda

Recruiting

Study to Learn About the Safety of Fazirsiran and if it Can Help People With Alpha-1 Antitrypsin Liver Disease With Mild Liver Scarring (Fibrosis)

Alpha1-Antitrypsin Deficiency

United States
Takeda
Takeda Development Center Americas, Inc.

Recruiting

Study to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Protein

Alpha1-Antitrypsin Deficiency

United States, Belgium, Switzerland, Canada, Australia, Germany, United Kingdom, Austria, France, Spain, Portugal, Italy, Poland
Takeda

Enrolling by invitation

An Extension Study to Learn About the Long-Term Safety of Fazirsiran and if Fazirsiran Can Help People With Alpha-1 Antitrypsin Liver Disease

Alpha1-Antitrypsin Deficiency

United Kingdom, Germany, United States, Austria, Portugal
University of California, San Francisco
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Enrolling by invitation

Hydrops: Diagnosing & Redefining Outcomes With Precision Study (HyDROPS)

Hydrops Fetalis | Birth Defect | Fetal Anomaly

United States
University of California, San Francisco
Johns Hopkins University; Eunice Kennedy Shriver National Institute of Child... and other collaborators

Enrolling by invitation

Uncovering the Etiologies of Non-immune Hydrops Fetalis

Non-Immune Hydrops Fetalis

United States
CSL Behring

Completed

Extension Study of Zemaira® i.v. Administration in Subjects With Emphysema Due to alpha1-proteinase Inhibitor Deficiency.

Emphysema | Alpha 1-proteinase Inhibitor Deficiency

Australia, Canada, Czech Republic, Denmark, Estonia, Finland, Germany, Ireland, Poland, Romania, Sweden
Thomas Jefferson University

Recruiting

Whole Exome Sequencing and Whole Genome Sequencing for Nonimmune Fetal/Neonatal Hydrops

Genetic Disorders | Nonimmune Fetal Hydrops | Nonimmune Hydrops in Neonate

United States
Johns Hopkins University
National Institute on Drug Abuse (NIDA)

Recruiting

Behavioral Pharmacology of THC and Alpha-pinene

THC | Alpha-pinene

United States
Baxalta now part of Shire
Baxalta Innovations GmbH, now part of Shire

Terminated

Stage 1 Study of ARALAST NP and GLASSIA in A1PI Deficiency

Chronic Obstructive Pulmonary Disease | Alpha1-antitrypsin Deficiency

United States, Canada, Australia
Shire

Withdrawn

ARALAST NP Alpha-1 Lung Density Chronic Obstructive Pulmonary Disease-Emphysema (COPD-E) Study

Chronic Obstructive Pulmonary Disease | Alpha1-antitrypsin Deficiency
Editas Medicine, Inc.

Recruiting

EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)

Hemoglobinopathies | Thalassemia Major | Thalassemia Intermedia | Transfusion Dependent Beta Thalassemia

United States, Canada
Accademia Italiana Medicina Osteopatica

Completed

Effect of Cranial Manipulation on ANS Activity

Manipulation, Osteopathic | Alpha Amylase

Italy
Shiraz University of Medical Sciences

Completed

Combination Therapy of Hydroxyurea With L-Carnitine and Magnesium Chloride in Thalassemia Intermedia

β-Thalassemia Intermedia

Iran, Islamic Republic of
BGI-research
Shenzhen Children's Hospital

Not yet recruiting

Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients

β-Thalassemia Major

China
Ionis Pharmaceuticals, Inc.

Terminated

Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)

Beta Thalassemia Intermedia

Australia, Thailand, Greece, Lebanon, Turkey
M.D. Anderson Cancer Center

Withdrawn

Pre-transplant Immunosuppression and Donor Stem Cell Transplant for the Treatment of Severe Hemoglobinopathies

Sickle Cell Disease | Sickle Beta Thalassemia | Beta Thalassemia Major | Sickle Cell-SS Disease | Sickle Beta 0 Thalassemia | Sickle Beta Plus Thalassemia

United States
Celgene

Terminated

Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia.

Beta Thalassemia Intermedia | Beta Thalassemia Major

France, United Kingdom, Italy, Greece
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Mannosidosis Disease (BioMannosidosis) (BioMannosido)

Alpha-Mannosidase B Deficiency | Lysosomal Alpha B Mannosidosis | Alpha-Mannosidase Deficiency

India, Sri Lanka, Germany

Clinical Trials on Thalassemia Alpha

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CROs in Taiwan

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