Search clinical trials for: Hereditary Hemolytic Anemias

Total 4550 results

Closed Joint-Stock Company NeoCor
Research Institute for Complex Problems of Cardiovascular Diseases, Russia; CT Medical Limited Liability Company

Recruiting

Post-market Clinical Monitoring of the Safety and Efficacy of the Aortic Valve Bioprothesis (TiAra)

Aortic Valve Disease | Artificial Heart Valves

Russian Federation
Assiut University

Recruiting

Association of Auditory Dysfunction With Retinal Ischemia in Behcet Disease

Behcet's Disease With Multisystem Involvement

Egypt
Duke University
American Society of Hematology

Recruiting

COVID-19 Vaccination Hesitancy in Adults With Sickle Cell Disease

Sickle Cell Disease | COVID-19 Vaccine | Vaccine Hesitancy

United States
Mario Negri Institute for Pharmacological Research

Recruiting

Developing a Pipeline to Employ RNA-Seq as a Complementary Diagnostic Tool in Rare Diseases (ANTHEM)

Healthy | Autosomal Dominant Polycystic Kidney | Atypical Hemolytic Uremic Syndrome | Membranoproliferative Glomerulonephritis

Italy
Memorial Sloan Kettering Cancer Center

Recruiting

A Study of Selinexor in People With Wilms Tumors and Other Solid Tumors

Solid Tumor | Rhabdoid Tumor | Wilms Tumor | Nephroblastoma | Malignant Peripheral Nerve Sheath Tumors | MPNST | XPO1 Gene Mutation

United States
Mario Negri Institute for Pharmacological Research

Recruiting

New Analytic Tools for aHUS and C3G Diagnosis (COMPRare)

Healthy | Hemolytic-Uremic Syndrome | Membranoproliferative Glomerulonephritis

Italy
Institute of Hematology & Blood Diseases Hospital

Recruiting

Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy

Gene Therapy | Transfusion-dependent Beta-Thalassemia

China
Hoffmann-La Roche

Recruiting

A Study To Evaluate The Efficacy, Safety, Pharmacokinetics, And Pharmacodynamics Of Satralizumab In Participants With Thyroid Eye Disease

Thyroid Eye Disease | TED

Japan, Australia, Singapore, United States, Argentina, Germany, Italy, Hong Kong
University Hospital, Tours

Recruiting

French National Cohort MATRIX "Renal and Systemic Thrombotic Microangiopathy" (MATRIX)

Kidney Diseases | Hemolytic Uremic Syndrome | Acute Kidney Injury | Thrombotic Microangiopathy | Nephrology

France
Istanbul University - Cerrahpasa (IUC)

Recruiting

The Effect of Video Game on Children With Familial Mediterranean Fever

FMF

Turkey
Laurent Servais
SYSNAV

Recruiting

Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next Study

Duchenne Muscular Dystrophy | Myotonic Dystrophy 1 | Congenital Muscular Dystrophy | Centronuclear Myopathy | Charcot-Marie-Tooth | Fascioscapulohumeral Muscular Dystrophy

Belgium
University Hospital, Toulouse

Not yet recruiting

Evaluate the Evolution of Body and Scalp Skin Discomfort in Patients With Hereditary Ichthyosis After Wraps (EnvelopIchtyose)

Ichthyosis Inherited

France
Virginia Commonwealth University

Not yet recruiting

Southeastern ATTR Amyloidosis Consortium: SEATTRAC Family Registry

Amyloidosis, Hereditary

United States
Omeros Corporation

Recruiting

Safety and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria Patients With a Sub-optimal Response to Ravulizumab

Paroxysmal Nocturnal Hemoglobinuria

Germany, Greece, Switzerland, United Kingdom
PERROD Guillaume

Not yet recruiting

CAD-EYE System for the Detection of Neoplastic Lesions in Patients With Lynch Syndrome (CADLYNCH)

Lynch Syndrome
University Hospital, Antwerp
Kom Op Tegen Kanker; Stichting tegen Kanker

Recruiting

First-in-human Interleukin-15-transpresenting Wilms' Tumor Protein 1-targeting Autologous Dendritic Cell Vaccination in Cancer Patients (IL15-TransDC)

Esophageal Cancer | Ovarian Cancer | Pancreas Cancer | Liver Cancer

Belgium
Ocugen

Recruiting

Study to Assess the Safety and Efficacy of OCU410ST for Stargardt Disease (GARDian)

Stargardt Disease

United States
Institut National de la Santé Et de la Recherche...

Recruiting

FARD (RaDiCo Cohort) (RaDiCo-FARD) (FARD)

Neurofibromatosis Type 1 | Ichthyosis | Ectodermal Dysplasia | Albinism | Pemphigus | Mucous Membrane Pemphigoid | Inherited Epidermolysis Bullosa | Palmoplantar Keratoderma | Incontinentia Pigmenti

France
Hospices Civils de Lyon

Recruiting

Stress-Echography in Hereditary Haemorrhagic Telangiectasia Patient With Hepatic Involvement (ROSE)

Hereditary Hemorrhagic Telangiectasia | Rendu Osler Disease

France
Forma Therapeutics, Inc.
Novo Nordisk A/S

Recruiting

A Phase 2 Open-label Study to Evaluate the Activity of Etavopivat on Transcranial Doppler Velocities in Pediatric Patients With Sickle Cell Disease Who Are at Increased Risk for Primary Stroke

Sickle Cell Disease

Nigeria, India, Oman
Institut National de la Santé Et de la Recherche...

Recruiting

National Cohort on Congenital Defects of the Eye (RaDiCoACOEIL)

Microphthalmia | Aniridia | Anophthalmia | Anterior Segment Dysgenesis 6, Peters Anomaly Subtype | Anterior Segment Dysgenesis 3, Rieger Subtype

France
Afnan Bkri

Not yet recruiting

Physiotherapy for Children With Sickle Cell Disease: A Randomized Controlled Trial (RCT)

Blood Disease
Vertex Pharmaceuticals Incorporated

Not yet recruiting

Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/βC Genotype

Sickle Cell Disease
Beijing Tiantan Hospital

Recruiting

Registry of X-linked Adrenoleukodystrophy

X-linked Adrenoleukodystrophy

China
RwHealth
Northern Care Alliance NHS Foundation Trust; Alnylam Pharmaceuticals

Not yet recruiting

RWE-based Treatment Patterns and Outcomes in CIDP

Hereditary Amyloidosis | Guillain-Barre Syndrome | Chronic Inflammatory Demyelinating Polyneuropathy

United Kingdom
University Hospital, Bordeaux

Recruiting

OBS'CEREVANCE: French Cohort of Pediatric Autoimmune Cytopenia (OBS'CEREVANCE)

Immune Thrombocytopenia | Autoimmune Hemolytic Anemia | Evans Syndrome

France
CSL Behring

Recruiting

First-In-Human Study To Evaluate The Safety, Tolerability, Pharmacokinetics, And Pharmacodynamics Of Escalating Single And Multiple Doses Of CSL040 In Healthy Subjects

Complement Deficiencies

Australia
Novartis Pharmaceuticals

Recruiting

Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)

Atypical Hemolytic Uremic Syndrome

Japan, Turkey
EuroBloodNet Association

Not yet recruiting

Safety and Efficacy of Mitapivat Sulfate in Adult Patients With Erythrocyte Membranopathies (SATISFY)

Hereditary Red Blood Cell Disorder (Disorder)

Denmark, Netherlands
Odense University Hospital

Recruiting

Hereditary Hemorrhagic Telangiectasia and Neurovascular Manifestations, in the Danish HHT Database (DAN-HEMTEL-VM)

Telangiectasia, Hereditary Hemorrhagic | Intracranial Arteriovenous Malformations

Denmark
Fondazione IRCCS Ca' Granda, Ospedale Maggiore...

Recruiting

Prospective Evaluation of Diagnosis and Treatment of Patients With Autoimmune Cytopenias Including Autoimmune Hemolytic Anemia, Immune Thrombocytopenia, and Chronic Idiopathic/Autoimmune Neutropenia (AIHA ITP CIN)

Myelodysplastic Syndromes | Immune Thrombocytopenia | Autoimmune Hemolytic Anemia | Cold Agglutinin Disease | Autoimmune Neutropenia | Chronic Idiopathic Neutropenia

Italy
Institut National de la Santé Et de la Recherche...

Recruiting

Eurbio-Alport (RaDiCo Cohort) (RaDiCo Eurbio-Alport) (Eurbio-Alport)

Alport Syndrome

France
Fondazione Policlinico Universitario Agostino Gemelli...

Recruiting

Exploring Biomarkers in Hereditary Transthyretin Amyloidosis (ELBA)

Hereditary Transthyretin Amyloidosis

Italy
Janssen Pharmaceutical K.K.

Recruiting

A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa

X-Linked Retinitis Pigmentosa

Japan
Peking Union Medical College Hospital

Recruiting

Sirolimus in the Treatment of Refractory/Relapsed wAIHA

Warm Autoimmune Hemolytic Anemia

China
Chen Miao

Recruiting

Zanubrutinib in the Treatment of Relapsed/Refractory wAIHA

Warm Autoimmune Hemolytic Anemia

China
Fog Pharmaceuticals, Inc.

Recruiting

FOG-001 in Locally Advanced or Metastatic Solid Tumors

Cancer | Gastric Cancer | Colorectal Cancer | Solid Tumor | Metastatic Cancer | Adenomatous Polyposis Coli | Non-small Cell Lung Cancer | Non-small Cell Lung Cancer Metastatic | Non-small Cell Carcinoma | Locally Advanced Solid Tumor | Non-small Cell Lung Cancer Stage IIIB | Gastroesophageal-junction Cancer | Non-Small... and other conditions

United States
Shanghai Fosun Pharmaceutical Industrial Development...

Recruiting

FCN-159 in Adult Patients With Symptomatic, Inoperable Neurofibromatosis Type 1-Related Plexiform Neurofibromas

Neurofibromatosis 1 | NF1 | Plexiform Neurofibroma

China
University of Arkansas

Recruiting

Evaluating Mitochondrial Dysfunction in Patients With Neurofibromatosis Type 1

Neurofibromatosis 1

United States
Assistance Publique - Hôpitaux de Paris
Braintale

Not yet recruiting

Validation of a Prognostic Biomarker Using Brain Diffusion MRI in X-linked Adrenoleukodystrophy (CALDIFF)

X-linked Adrenoleukodystrophy
University of Southern California

Not yet recruiting

Non-invasive Ultrasound Retinal Stimulation for Vision Restoration

Age-Related Macular Degeneration | Retinitis Pigmentosa | Ultrasound Therapy; Complications
Janssen Research & Development, LLC

Recruiting

A Study of Nipocalimab in Pregnancies at Risk for Severe Hemolytic Disease of the Fetus and Newborn (HDFN) (AZALEA)

Hemolytic Disease of the Fetus and Newborn

Israel, Japan, Australia, United States, United Kingdom, Brazil
West Kazakhstan Medical University

Recruiting

Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in Kazakhstan

Ornithine Transcarbamylase Deficiency | Biotinidase Deficiency | Citrullinemia | Glutaric Acidemia Type II | Argininosuccinic Aciduria | Maple Syrup Urine Disease | Primary Carnitine Deficiency | Homocystinuria | Carnitine Palmitoyltransferase II Deficiency | Arginase Deficiency | Very Long-chain Acyl-CoA... and other conditions

Kazakhstan
Piazza della Vittoria 14 Studio Medico - Ginecologia...

Not yet recruiting

Perception About Benefits and Risks Related to Combined Hormonal Contraceptives Use in Patients With Lynch Syndrome

Lynch Syndrome | Contraception

Italy
HuidaGene Therapeutics Co., Ltd.
Cholgene Therapeutics, Inc.

Recruiting

Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR) (STAR)

Leber Congenital Amaurosis | Inherited Retinal Diseases Caused by RPE65 Mutations

United States, China
The University of The West Indies

Recruiting

Enablers and Barriers to Hydroxyurea Use for Sickle Cell Disease Jamaica

Sickle Cell Disease

Jamaica
University of Minnesota

Recruiting

Treatment of LSCD With DM

Limbal Stem-cell Deficiency | Congenital Aniridia

United States
PT. Prodia Stem Cell Indonesia

Recruiting

Role of UC-MSC and CM to Inhibit Vision Loss in Retinitis Pigmentosa Phase I/II

Retinitis Pigmentosa

Indonesia
Santé Arménie French-Armenian Research Center

Recruiting

Armenian NAtionwide REGistry of Systemic Autoimmune and Autoinflammatory Diseases (NAREG)

Rheumatoid Arthritis | Celiac Disease | Giant Cell Arteritis | Sjogren's Syndrome | Takayasu Arteritis | Primary Antiphospholipid Syndrome | Behcet Disease | Antineutrophil Cytoplasmic Antibody (ANCA) Positive Vasculitis | Hereditary and Acquired Angioedema

Armenia
National Heart, Lung, and Blood Institute (NHLBI)

Not yet recruiting

Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease

Sickle Cell Disease | Sickle Cell Disorders | Hb-SS Disease | Hemoglobin S | Disease Sickle Cell Anemia | Hemoglobin Beta Thalassemia Disease

United States

Search clinical trials for: Hereditary Hemolytic Anemias

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