Clinical Trials on Congenital Non-Spherocytic Hemolytic Anemia in Germany

Total 104 results

Boston Children's Hospital
Agios Pharmaceuticals, Inc.

Completed

Pyruvate Kinase Deficiency Natural History Study (PKD NHS)

Pyruvate Kinase Deficiency | Congenital Non-Spherocytic Hemolytic Anemia

United States, Germany, Canada, Czechia, Italy, Netherlands
Bioverativ, a Sanofi company

Active, not recruiting

Safety and Tolerability Study in Adults With Cold Agglutinin Disease Previously Treated With SAR445088 or Never Treated With SAR445088

Autoimmune Haemolytic Anaemia

Netherlands, Germany, Italy, Norway, United Kingdom
Bioverativ, a Sanofi company

Completed

A Safety and Tolerability Study of BIVV020 in Adults With Cold Agglutinin Disease

Autoimmune Haemolytic Anaemia

United States, Germany, Italy, Netherlands, Norway, United Kingdom
Sanofi

Terminated

Safety, Pharmacokinetics, and Efficacy of Subcutaneous Isatuximab in Adults With Warm Autoimmune Hemolytic Anemia (wAIHA)

Warm Autoimmune Hemolytic Anemia (wAIHA)

United Kingdom, Belgium, Netherlands, France, United States, Germany, Hungary, Italy
Sanofi

Active, not recruiting

Efficacy, Safety and Pharmacokinetics of Rilzabrutinib in Patients With Warm Autoimmune Hemolytic Anemia (wAIHA)

Warm Autoimmune Hemolytic Anemia (wAIHA)

United States, Austria, China, Denmark, Germany, Hungary, Italy, Spain, United Kingdom
Rigel Pharmaceuticals

Enrolling by invitation

A Phase 3 Open Label Extension Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia

Warm Antibody Autoimmune Hemolytic Anemia

Spain, United States, Australia, Norway, Belarus, Germany, Netherlands, Belgium, Austria, Bulgaria, Czechia, France, Georgia, Italy, Russian Federation, Serbia, Ukraine, United Kingdom
Incyte Corporation

Terminated

Study of the Efficacy and Safety of Parsaclisib in Participants With Primary Warm Autoimmune Hemolytic Anemia (PATHWAY)

Warm Autoimmune Hemolytic Anemia (wAIHA)

Spain, United States, Austria, Belgium, Canada, France, Germany, Israel, Italy, Japan, Netherlands, Poland, United Kingdom
Rigel Pharmaceuticals

Completed

A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of wAIHA

Warm Antibody Autoimmune Hemolytic Anemia

United States, Spain, Canada, Australia, Norway, France, Belarus, Germany, Netherlands, Belgium, United Kingdom, Austria, Czechia, Georgia, Italy, Russian Federation, Serbia, Ukraine, Romania, Denmark, Hungary, Bulgaria
Novartis Pharmaceuticals

Recruiting

A Study of Efficacy and Safety of Ianalumab in Previously Treated Patients With Warm Autoimmune Hemolytic Anemia (VAYHIA)

Warm Autoimmune Hemolytic Anemia (wAIHA)

China, Japan, Spain, Singapore, France, Germany, Taiwan, United States, Italy, India, Malaysia, Argentina, Hungary, Israel, Australia, Thailand, United Kingdom, Romania
Janssen Research & Development, LLC

Recruiting

Efficacy and Safety of M281 in Adults With Warm Autoimmune Hemolytic Anemia (ENERGY)

Warm Autoimmune Hemolytic Anemia

United States, Israel, Korea, Republic of, Spain, Czechia, France, Brazil, China, United Kingdom, Malaysia, Hungary, Japan, Poland, Germany, Ukraine, Italy, Greece, Netherlands, Egypt
University Hospital Heidelberg
Charite University, Berlin, Germany; Pfizer; German Cancer Research Center; Johann... and other collaborators

Recruiting

Sickle-cell Disease Registry of the GPOH (SichReg)

Sickle Cell Disease

Germany
AstraZeneca
Parexel

Completed

A Study to Compare if the Uptake of Ticagrelor in the Body Differs When Different Tablets Are Administered

Sickle Cell Disease

Germany
Alexion Pharmaceuticals

Completed

Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-Resistant aHUS (aHUS)

Atypical Hemolytic Uremic Syndrome

France, United States, Austria, Germany, United Kingdom
Agios Pharmaceuticals, Inc.

Active, not recruiting

A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period (ACTIVATE-KidsT)

Pediatric Pyruvate Kinase Deficiency | Pediatric Hemolytic Anemia

United States, Spain, Denmark, Turkey, Netherlands, Czechia, United Kingdom, Germany, Canada, Switzerland
Alexion Pharmaceuticals

Completed

Open Label Controlled Trial of Eculizumab in Adolescent Patients With Plasma Therapy-Resistant aHUS (aHUS)

Atypical Hemolytic Uremic Syndrome

France, United States, Austria, Germany, United Kingdom
Alexion

Completed

Open Label Controlled Trial of Eculizumab in Adolescent Patients With Plasma Therapy-Sensitive aHUS (aHUS)

Atypical Hemolytic Uremic Syndrome

Canada, United Kingdom, France, Germany, Netherlands, Sweden, Italy
Alexion Pharmaceuticals

Completed

Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-sensitive Atypical Hemolytic Uremic Syndrome (aHUS) (aHUS)

Atypical Hemolytic Uremic Syndrome

Canada, United Kingdom, France, Germany, Sweden, Netherlands, Italy
Agios Pharmaceuticals, Inc.

Recruiting

A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regularly Transfused, Followed by a 5-Year Extension Period (ACTIVATE-Kids)

Pediatric Pyruvate Kinase Deficiency | Pediatric Hemolytic Anemia

United States, Spain, Germany, Netherlands, Canada, France, Israel, Switzerland, Italy
UCSF Benioff Children's Hospital Oakland
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Medical... and other collaborators

Completed

Multi-Center Study of Iron Overload: Pilot Study (MCSIO)

Sickle Cell Disease | Thalassemia | Diamond-Blackfan Anemia

United Kingdom, United States, Germany
Alexion

Completed

An Open-label, Multi-center Clinical Trial of Eculizumab in Adult Patients With Atypical Hemolytic-uremic Syndrome

Atypical Hemolytic-Uremic Syndrome

United Kingdom, United States, France, Italy, Germany, Spain, Belgium
Alexion Pharmaceuticals

Completed

An Open-Label, Multi-Center Clinical Trial of Eculizumab in Pediatric Patients With Atypical Hemolytic-Uremic Syndrome (aHUS)

Atypical Hemolytic-Uremic Syndrome

United Kingdom, Belgium, United States, Germany, Italy, France, Netherlands, Australia, Canada
German CLL Study Group

Completed

Combination Chemotherapy and Rituximab in Treating Patients With Chronic Lymphocytic Leukemia (CLL) That Did Not Respond to Fludarabine, CLL With Autoimmune Haemolytic Anemia (AIHA) or Richter's Transformation (RT)

Chronic Lymphocytic Leukemia

Germany
Alexion Pharmaceuticals

Terminated

Evaluation of Potential Predictors of Disease Progression in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Including Genetics, Biomarkers, and Treatment (EVIDENCE)

Atypical Hemolytic Uremic Syndrome

United States, Australia, Germany, United Kingdom
Novartis Pharmaceuticals

Completed

Study of Efficacy, Safety and Tolerability of ACZ885 (Canakinumab) in Pediatric and Young Adult Patients With Sickle Cell Anemia

Sickle Cell Anemia

Turkey, United States, Germany, Canada, United Kingdom, Israel, South Africa
Alexion Pharmaceuticals

Terminated

aHUS Observational Long Term Follow-Up (LTFU)

Atypical Hemolytic Uremic Syndrome

France, Canada, United States, Belgium, Italy, United Kingdom, Netherlands, Australia, Austria, Germany, Sweden, Switzerland
Vertex Pharmaceuticals Incorporated
CRISPR Therapeutics

Recruiting

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

Hematologic Diseases | Genetic Diseases, Inborn | Hemoglobinopathies | Thalassemia | Beta-Thalassemia

United States, Germany, Italy, United Kingdom, Canada
Alexion
Syneos Health

Recruiting

Atypical Hemolytic-Uremic Syndrome (aHUS) Registry

Atypical Hemolytic-Uremic Syndrome

France, Germany, Israel, Italy, United Kingdom, Canada, United States, Australia, Belgium, Denmark, Korea, Republic of, Poland, Russian Federation, Spain, Taiwan
Vertex Pharmaceuticals Incorporated
CRISPR Therapeutics

Recruiting

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

Sickle Cell Disease | Hemoglobinopathies | Hematological Diseases | Hydroxyurea Failure | Hydroxyurea Intolerance

United States, Italy, United Kingdom, Germany
bluebird bio

Completed

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype

Beta-Thalassemia

France, United Kingdom, United States, Thailand, Italy, Germany
Vertex Pharmaceuticals Incorporated
CRISPR Therapeutics

Recruiting

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

Hematologic Diseases | Genetic Diseases, Inborn | Sickle Cell Disease | Hemoglobinopathies | Sickle Cell Anemia | Thalassemia | Beta-Thalassemia

United States, Italy, Germany, Saudi Arabia
bluebird bio

Completed

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia

Beta-Thalassemia

United States, Germany, United Kingdom, France, Greece, Italy
UCSF Benioff Children's Hospital Oakland
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Medical... and other collaborators

Completed

Multi-Center Study of Iron Overload: Survey Study (MCSIO) (MCSIO)

Sickle Cell Disease | Thalassemia | Diamond-Blackfan Anemia

United States, United Kingdom, Germany
Agios Pharmaceuticals, Inc.

Completed

A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)

Anemia, Hemolytic | Pyruvate Kinase Deficiency

United States, Spain, Switzerland, Turkey, Germany, France, United Kingdom, Japan, Netherlands, Brazil, Canada, Czechia, Denmark, Italy, Korea, Republic of, Thailand
Vertex Pharmaceuticals Incorporated
CRISPR Therapeutics

Active, not recruiting

A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia

Hematologic Diseases | Genetic Diseases, Inborn | Hemoglobinopathies | Thalassemia | Beta-Thalassemia

United States, United Kingdom, Canada, Germany, Italy
Vertex Pharmaceuticals Incorporated
CRISPR Therapeutics

Active, not recruiting

A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease

Sickle Cell Disease | Hemoglobinopathies | Hematological Diseases

United States, Belgium, Canada, France, Germany, Italy, United Kingdom
Agios Pharmaceuticals, Inc.

Active, not recruiting

Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007

Pyruvate Kinase Deficiency

United States, Spain, Japan, Canada, United Kingdom, Korea, Republic of, Ireland, Thailand, Italy, Turkey, Denmark, Brazil, France, Germany, Netherlands, Switzerland
ADDMEDICA SASA

Completed

European Sickle Cell Disease Cohort - Hydroxyurea (ESCORT-HU)

Sickle Cell Disease

France, Germany, French Guiana, Greece, Guadeloupe, Italy, Martinique
Dr. med. Mahir Karakas

Terminated

Effect of Platelet Inhibition and / or Lipid Lowering in Non-ACS-patients With Positive Troponin (GRAY-ZONE)

Acute Chest Syndrome

Germany
Agios Pharmaceuticals, Inc.

Recruiting

Pyruvate Kinase Deficiency Global Longitudinal Registry (PEAK Registry)

Pyruvate Kinase Deficiency

United States, Germany, Spain, Italy, United Kingdom, Czechia, France, Canada, Korea, Republic of, Netherlands, Turkey, Portugal, Denmark, Ireland, Switzerland, Thailand
Novartis Pharmaceuticals

Recruiting

Rollover Study for Patients With Sickle Cell Disease Who Have Completed a Prior Novartis-Sponsored Crizanlizumab Study

Sickle Cell Disease

United States, Belgium, Spain, Lebanon, Brazil, Italy, Oman, Turkey, Germany, Colombia, France
Alexion Pharmaceuticals, Inc.

Completed

Study of Ravulizumab in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS)

Atypical Hemolytic Uremic Syndrome (aHUS)

Italy, United States, Korea, Republic of, Spain, Germany, Belgium, United Kingdom
University of Regensburg

Recruiting

A Trial to Assess Haploidentical T-depleted Stem Cell Transplantation in Patients With SCD

Sickle Cell Disease | Sickle Cell Anemia | Sickle Cell Disorders | HbS Disease | Hemoglobin S Disease | Sickling Disorder Due to Hemoglobin S

Germany, Austria
Alexion Pharmaceuticals

Completed

Safety and Efficacy Study of Eculizumab in Shiga-Toxin Producing Escherichia Coli Hemolytic-Uremic Syndrome (STEC-HUS) (STEC-HUS)

Shiga-like Toxin-producing Escherichia Coli

Germany
Vertex Pharmaceuticals Incorporated
CRISPR Therapeutics

Enrolling by invitation

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

Hematologic Diseases | Genetic Diseases, Inborn | Sickle Cell Disease | Hemoglobinopathies | Sickle Cell Anemia | Thalassemia | Beta-Thalassemia

United States, Canada, Italy, United Kingdom, Germany
ADDMEDICA SASA
International Clinical Trials Association

Recruiting

ESCORT-HU Extension: European Sickle Cell Disease Cohort - Hydroxyurea - Extension Study

Sickle Cell Disease

France, French Guiana, Germany, Greece, Guadeloupe, Italy, Martinique, Réunion
Celgene
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway...

Recruiting

Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia

Beta-Thalassemia

United States, Turkey, Italy, Thailand, Germany, Greece, China, India, Lebanon
Alexion

Completed

A Retrospective, Observational, Non-interventional Trial to Assess Eculizumab Treatment Effect in Patients With Atypical Hemolytic Uremic Syndrome (aHUS)

Atypical Hemolytic Uremic Syndrome (aHUS)

United States, France, Germany, Spain, Switzerland, Netherlands, Canada, Austria
bluebird bio

Active, not recruiting

Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy

Transfusion-dependent Beta-Thalassemia

United States, France, United Kingdom, Australia, Thailand, Italy, Germany, Greece
Forma Therapeutics, Inc.
Novo Nordisk A/S

Recruiting

A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS)

Sickle Cell Disease

United States, Italy, Spain, United Kingdom, France, Lebanon, Germany, Oman, Canada, Greece
Alexion Pharmaceuticals, Inc.

Completed

Study of ALXN1210 in Complement Inhibitor Treatment-Naïve Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)

Atypical Hemolytic Uremic Syndrome (aHUS)

United States, France, Spain, Taiwan, United Kingdom, Australia, Belgium, Germany, Italy, Japan, Korea, Republic of, Russian Federation, Austria, Canada

Clinical Trials on Congenital Non-Spherocytic Hemolytic Anemia in Germany

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