Search clinical trials for: Rare Anemia Disorders

Total 238968 results

Novartis Pharmaceuticals

Not yet recruiting

A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE) (SPARKLE)

Sickle Cell Disease
Memorial Sloan Kettering Cancer Center

Recruiting

A Study of Emapalumab for Pediatric Aplastic Anemia

Aplastic Anemia | Cytopenia | Hypocellular Marrow

United States
China-Japan Friendship Hospital

Recruiting

The Effects of Low-Dose Versus High-Dose Intravenous IRON Therapy With Ferric DerisomaltOSE in Patients With Chronic Heart Failure and Iron Deficiency (IRONDOSE)

Heart Failure | Iron Deficiency

China
Peking Union Medical College Hospital

Not yet recruiting

A Prospective Study on the Treatment of Recurrent/Refractory/Intolerable NSAA With Lusutrombopag

Aplastic Anemia

China
Peking Union Medical College Hospital

Not yet recruiting

Luspatercept Plus CsA vs CsA for the Treatment of Newly Diagnosed Non-Transfusion-Dependent NSAA

Aplastic Anemia

China
Regeneron Pharmaceuticals

Not yet recruiting

A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body (FERVENT-1)

Non-transfusion Dependent Beta-thalassemia (NTDT)
Masonic Cancer Center, University of Minnesota

Not yet recruiting

MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.

Acquired Pure Red Cell Aplasia | Paroxysmal Nocturnal Hemoglobinuria | Severe Aplastic Anemia | Acquired Amegakaryocytic Thrombocytopenia
Novartis Pharmaceuticals

Not yet recruiting

Home Reported Outcomes in PNH

Paroxysmal Nocturnal Hemoglobinuria
Sidney Kimmel Comprehensive Cancer Center at Johns...
Janssen Research & Development, LLC

Not yet recruiting

Darzalex Faspro (Daratumumab and Hyaluronidase-fihj) Before Standard Desensitization and Allogeneic Peripheral Blood Stem Cell Transplantation in Adult Patients at High-risk for Primary Graft Failure Secondary to Donor Specific Antibodies

Lymphoma | Multiple Myeloma | Hodgkin Lymphoma | Myelofibrosis | Non Hodgkin Lymphoma | Chronic Myeloid Leukemia | Hematologic Malignancy | Aplastic Anemia | Myelodysplastic Syndromes (MDS) | Acute Myeloid Leukemia (AML) | Bone Marrow Transplant Rejection | Acute Lymphoblastic Leukemia (ALL), Adult
Vanderbilt University Medical Center
Aminu Kano Teaching Hospital; Murtala Muhammed Specialist Hospital

Not yet recruiting

Prevention of Malnutrition in Children With Sickle Cell Disease (PMC-SCD)

Malnutrition | Sickle Cell Disease
Inmunova S.A.
Chemo Research; KLIXAR; Linical; Crovelis

Not yet recruiting

Efficacy of INM004 in Children With STEC-HUS

Hemolytic-Uremic Syndrome

Argentina
Hematology department of the 920th hospital

Recruiting

Clinical Study on Modified Allogeneic Hematopoietic Stem Cell Transplantation Regimen for Severe Aplastic Anemia

Severe Aplastic Anemia

China
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Comparative Health Status and Quality of Life of Patients With Sickle Cell Disease (SCD) Who Underwent Matched-sibling Hematopoietic Stem Cell Transplantation Versus Non Transplanted SCD Case-control Patients (TRANSPLANTORN2)

Sickle Cell Disease
European Institute of Oncology

Recruiting

Total Body Irradiation Treatments for Patients Treated at European Institute of Oncology

Lymphoma | Leukemia | Aplastic Anemia | Leukemia, Acute | Leukemia, Chronic

Italy
Alexion Pharmaceuticals, Inc.

Not yet recruiting

Study of Ultomiris® (Ravulizumab) Safety in Pregnancy

Pregnancy | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Atypical Hemolytic Uremic Syndrome (aHUS) | Ultomiris-exposed Pregnant/ Postpartum | Generalized Myasthenia Gravis (gMG) | Neuromyelitis Optica Spectrum Disorder (NMOSD)
National Cancer Institute (NCI)

Not yet recruiting

Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms

Myelodysplastic Syndromes

United States
Omeros Corporation

Recruiting

Long-Term Safety, Tolerability and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria

Paroxysmal Nocturnal Hemoglobinuria

Switzerland, Germany, Ukraine, United Kingdom
Longbio Pharma

Recruiting

A Study of Single and Multiple Doses of LP-005 in Healthy Adult Participants

Paroxysmal Nocturnal Hemoglobinuria (PNH)

China
University Health Network, Toronto
Sunnybrook Health Sciences Centre; Queen's University; Heart and Stroke Foundation...

Not yet recruiting

Post-operative Intravenous Iron to Treat Iron-deficiency Anemia in Patients Undergoing Cardiac Surgery (POAM)

Cardiac Surgery | Chronic Iron Deficiency Anaemia

Canada
City of Hope Medical Center
National Cancer Institute (NCI)

Not yet recruiting

225Ac-DOTA-Anti-CD38 Daratumumab Monoclonal Antibody With Fludarabine, Melphalan and Total Marrow and Lymphoid Irradiation as Conditioning Treatment for Donor Stem Cell Transplant in Patients With High-Risk Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia and Myelodysplastic Syndrome

Acute Myeloid Leukemia | Acute Lymphoblastic Leukemia | Myelodysplastic Syndrome

United States
Novartis Pharmaceuticals

Not yet recruiting

Revolade Tablets Specified Drug-use Survey

Aplastic Anemia
Kuang Tien General Hospital

Recruiting

Sucrosomial Iron on Insomnia in Non-dialysis Chronic Kidney Disease Patients With Iron Deficiency Anemia

Sleep Disorder | CKD

Taiwan
Anemia Working Group Romania
Carol Davila University of Medicine and Pharmacy; Sf Ioan Emergency Clinical... and other collaborators

Recruiting

Effect of Low Protein Diet on Top of Dapagliflozin on Chronic Kidney Disease in Patients With Type 2 Diabetes Mellitus (PRODAPA-CKD)

Chronic Kidney Diseases | Type 2 Diabetes Mellitus

Romania
Peking University People's Hospital

Not yet recruiting

Sirolimus+Abatacept+Mycophenolate Mofetil for Prophylaxis of aGVHD in Patients Receiving Haplo-HSCT Who Are Intolerant to Calcineurin Inhibitors

Myelodysplastic Syndromes | Acute Leukemia | Severe Aplastic Anemia
Raffaele De Caterina
University of Pisa, Italy

Recruiting

Effect of Oral sucRosomIal Iron on exerciSE Capacity and Quality of Life in Patients With Heart Failure (RISE-HF)

Iron-deficiency | Chronic Heart Failure | Left Ventricular Systolic Dysfunction

Italy
Institute of Hematology & Blood Diseases Hospital...
Fosun Pharmaceutical Distribution Jiangsu co., Limited

Not yet recruiting

Efficacy and Safety of Avatrombopag vs. Avatrombopag Combined With rhTPO in the Treatment of SAA

Anemia, Aplastic
Institute of Hematology & Blood Diseases Hospital...

Recruiting

Rituximab for Serious Aplastic Anemia With Platelet Transfusion Refractoriness

Platelet Transfusion Refractoriness | Severe Aplastic Anemia

China
Hospital Universitari Vall d'Hebron Research Institute
ERN-EuroBloodNet (European Reference Network on Rare Hematological Diseases

Recruiting

European Rare Blood Disorders Platform (ENROL) (ENROL)

Leukemia | Anemia | Iron Metabolism Disorders | Anemia, Sickle Cell | Thalassemia | Bleeding Disorder | Myeloma | Lymphoid Neoplasm | Blood Cancer | Bone Marrow Failure | Red Cell Membrane and Enzyme Abnormalities | Myeloma, Malignant

Spain
Wuhan Union Hospital, China

Not yet recruiting

UCMSC-Exo for Chemotherapy-induced Myelosuppression in Acute Myeloid Leukemia

Infections | Acute Myeloid Leukemia | Anemia | Bleeding | Thrombocytopenia | Neutropenia
Chengdu Suncadia Medicine Co., Ltd.

Recruiting

Long-term Safety and Tolerability of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria

Paroxysmal Nocturnal Hemoglobinuria

China
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Early-goal Directed Automated Red Blood Cell Exchange for Acute Chest Syndrome in Sickle Cell Disease (ARCAD)

Acute Chest Syndrome

France
Hospital Universitari Vall d'Hebron Research Institute
Erasme University Hospital; Cyprus Institute of Neurology and Genetics; EuroBloodNet...

Recruiting

RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs) (RADeep)

Iron Metabolism Disorders | Sickle Cell Disease | Thalassemia | Hemolytic; Anemia, Hereditary, Due to Enzyme Disorder | Anemia Due to Membrane Defect | CDA | Sideroblastic Anemia | Constitutional Aplastic Anemia | Hereditary Anemia

Spain
Shaheed Zulfiqar Ali Bhutto Medical University

Not yet recruiting

Comparison of Twice Weekly Versus Daily Iron Therapy in Treating Anemia in Children With Cerebral Palsy

Cerebral Palsy | Iron Deficiency Anemia
AstraZeneca

Recruiting

A Real-world, Multi-center, Prospective, Observational Study for PNH in China (ReWoPNH)

Paroxysmal Nocturnal Hemoglobinuria

China
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders (DANNIgene)

Hemophilia A | Hemophilia B | Cystic Fibrosis | Sickle Cell Disease | Muscular Dystrophy, Duchenne | Fragile X Syndrome | Huntington Disease | Myotonic Dystrophy | Autosomal Recessive Polycystic Kidney Disease | Neurofibromatosis-Noonan Syndrome | Muscular Dystrophy, Becker | Invasive PreNatal Diagnosis in a... and other conditions

France
Assistance Publique - Hôpitaux de Paris

Recruiting

Peripheral Serotonin and Albinism (SEPIAs)

Oculocutaneous Albinism

France
Wuhan Createrna Science and Technology Co., Ltd

Not yet recruiting

Study of Safety and Efficacy of MY008211A in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Paroxysmal Nocturnal Hemoglobinuria (PNH)
Azienda Ospedaliero-Universitaria di Modena

Recruiting

"Iron Overload and Endocrinological Diseases"

Iron Overload | Hemochromatosis | Thalassemia Major | Thalassemia Intermedia | Endocrinopathy

Italy
BioCryst Pharmaceuticals

Recruiting

Dose Escalation of BCX10013 in Subjects With Paroxysmal Nocturnal Hemoglobinuria

Paroxysmal Nocturnal Hemoglobinuria

South Africa, Malaysia
AstraZeneca

Recruiting

A Prospective, Non-interventional, Observational Study of Presentation, Treatment Patterns and Outcomes in Atypical Hemolytic Uremic Syndrome Patients

Atypical Hemolytic Uremic Syndrome(aHUS)

China
National Human Genome Research Institute (NHGRI)

Recruiting

A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders

Metabolic Disease | Purine-Pyrimidine Metabolism | AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2 | UNG, OMIM *191525, Hyper-IgM Syndrome 5 | NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency | UMPS, OMIM *613891, Orotic Aciduria | DHODH, OMIM *126064,... and other conditions

United States
Markus Mapara
ITB-Med LLC

Recruiting

Siplizumab for Sickle Cell Disease Transplant (CD2 SCD)

Anemia, Sickle Cell

United States
King's College London

Recruiting

The Living With a Long-Term Condition Study (LTC)

Liver Diseases | Heart Diseases | Heart Failure | Stroke | Cerebrovascular Disorders | Epilepsy | Pain | Multiple Sclerosis | Hypertension | Obesity | Diabetes | Cancer | Arthritis | Osteoarthritis | Rheumatoid Arthritis | Parkinson Disease | Migraine | Hepatitis | Atrial Fibrillation | Asthma | Endometriosis | Dementia | Fibromyalgia | Celiac... and other conditions

United Kingdom
Peking University People's Hospital

Recruiting

The Optimization of Conditioning Regimen for HLA Matched HSCT in SAA

Severe Aplastic Anemia

China
UK Kidney Association

Recruiting

National Registry of Rare Kidney Diseases (RaDaR)

Vasculitis | AL Amyloidosis | Tuberous Sclerosis | Fabry Disease | Cystinuria | Focal Segmental Glomerulosclerosis | IgA Nephropathy | Bartter Syndrome | Pure Red Cell Aplasia | Membranous Nephropathy | Atypical Hemolytic Uremic Syndrome | Autosomal Dominant Polycystic Kidney Disease | Cystinosis | Nephronophthisis | BK Nephropathy and other conditions

United Kingdom
Sohag University

Not yet recruiting

Assessment of Cognitive Function and Quality of Life in Thalassemic Children at Sohag University Hospital

Thalassemia

Egypt
Wuhan Createrna Science and Technology Co., Ltd

Recruiting

A Study of MY008211A in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Paroxysmal Nocturnal Hemoglobinuria

China
Bastiaan Driehuys
National Institutes of Health (NIH)

Recruiting

129Xe MRI Cardiopulmonary

Polycythemia | Anemia | Interstitial Lung Disease | Dyspnea | Chronic Thromboembolic Pulmonary Hypertension | Acute Pulmonary Embolism

United States
Anhui Provincial Hospital

Recruiting

Conditioning Regimen Containing Low Dose ATG for The Treatment of Acquired SAA Receiving sUCBT

Severe Aplastic Anemia

China
Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University

Not yet recruiting

Longitudinal Study on the QoL of Pediatric Patients After HSCT and Its Influencing Factors

Acute Leukemia | Thalassemia Major

China

Search clinical trials for: Rare Anemia Disorders

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