Search clinical trials for: Congenital Nonspherocytic Hemolytic Anemia

Total 12381 results

Rocket Pharmaceuticals Inc.

Not yet recruiting

Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency

Pyruvate Kinase Deficiency

Spain, United States
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
Massachusetts General Hospital
Agios Pharmaceuticals, Inc.

Recruiting

Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms

Acute Myeloid Leukemia | Myelodysplastic Syndromes | Myeloproliferative Neoplasm | Myelodysplastic/Myeloproliferative Neoplasm | Clonal Cytopenia of Undetermined Significance | Pyruvate Kinase Deficiency | Pyruvate Kinase Deficiency Anemia | Hereditary Hemolytic Anemia | Clonal Myeloid Neoplasm | Other... and other conditions

United States
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
Agios Pharmaceuticals, Inc.

Active, not recruiting

A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency

Pyruvate Kinase Deficiency

United States, Canada, Netherlands, United Kingdom, France, Italy
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
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
Rocket Pharmaceuticals Inc.

Active, not recruiting

Gene Therapy for Pyruvate Kinase Deficiency (PKD)

Pyruvate Kinase Deficiency

Spain, United States
Agios Pharmaceuticals, Inc.

Completed

Pyruvate Kinase Deficiency Global Longitudinal Registry Substudy of Protocol AG348-C-008

Pyruvate Kinase Deficiency

United States, Italy, Netherlands
Agios Pharmaceuticals, Inc.

Terminated

Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO) (PKD)

Anemia | Anemia, Hemolytic | Pyruvate Kinase Deficiency | Pyruvate Kinase Deficiency Anemia

United States, United Kingdom, Italy, Spain, Netherlands
CENTOGENE GmbH Rostock

Completed

Pyruvate Kinase Deficiency Epidemiological Study (PIECE) (PIECE)

Pyruvate Kinase Deficiency

United States
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
Agios Pharmaceuticals, Inc.

Completed

A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)

Anemia, Hemolytic | Pyruvate Kinase Deficiency

United States, United Kingdom, Netherlands, Canada, Denmark, France, Italy, Ireland, Thailand
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
Rofaida Hassan Ahmed

Not yet recruiting

EKLF Gene Expression in β-thalassemia

β-thalassemia
St. Jude Children's Research Hospital
BioLineRx, Ltd.

Not yet recruiting

SCD Stem Cell Mobilization and Apheresis Using Motiixafortide

Sickle Cell Disease

United States
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
Novo Nordisk A/S

Not yet recruiting

A Research Study of the Effect of Food on Etavopivat in Healthy Participants

Healthy Volunteers Sickle Cell Disease, Thalassemia

United States
Assiut University

Not yet recruiting

Detection of Krupple Like Factor -1(KLF1/ EKLF) DNA Mutations in Beta Thalassemia Patients

EKLF Mutations in Beta Thalassemia Patients
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)
Instituto Nacional de Investigacao em Saude, Angola
FUNDAÇAO CALOUSTE GULBENKIAN; FUNDACIÓN BANCARIA CAIXA D'ESTALVIS I PENSIONS... and other collaborators

Recruiting

LEARNER- Low dosE AspiRiN prEterm tRial (Angola) (LEARNER)

Pregnancy Complications | Pregnancy Related | Sickle Cell Disease | Pre-Eclampsia

Angola
Essen Biotech

Not yet recruiting

Investigation Into the Use of BAH243 Lentiviral Vector for Gene Therapy in Treating Sickle Cell Disease (BAH243)

Sickle Cell Disease | Sickle-Cell Disease With Crisis

China
Academisch Medisch Centrum - Universiteit van Amsterdam...

Recruiting

The Role of Red Cell Characteristics, Angiogenesis, Viscosity and Oxygenation in the Pathophysiology of Sickle Cell Related Retinopathy (RAVOS)

Sickle Cell Disease | Sickle Cell SC Disease | Sickle Cell-SS Disease | Sickle Cell Retinopathy

Netherlands
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
University of Mississippi Medical Center
University of Alabama at Birmingham

Recruiting

Low Systemic/High Local Exercise Load in Peds SCD

Sickle Cell Anemia in Children

United States
RTI International

Recruiting

Recruitment and Engagement in Care to Impact Practice Enhancement (RECIPE) for Sickle Cell Disease (RECIPE)

Sickle Cell Disease

United States
Assiut University

Not yet recruiting

Thyroid Function , HbA1c in Relation to Ferritin Level in Adullt Patient e B Thalassemia

B Thalassemia
University of Pittsburgh
National Heart, Lung, and Blood Institute (NHLBI)

Not yet recruiting

Peer Support for Adolescents and Emerging Adults With Sickle Cell Pain (PRESENCE)

Pain | Sickle Cell Disease
St. Jude Children's Research Hospital
National Heart, Lung, and Blood Institute (NHLBI)

Recruiting

School Readiness Intervention for Preschool Children With Sickle Cell Disease

Sickle Cell Disease

United States
Children's Hospital of Philadelphia

Not yet recruiting

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

Beta-Thalassemia

United States
Editas Medicine, Inc.

Not yet recruiting

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

Sickle Cell Disease | Hemoglobinopathies | Transfusion-dependent Beta-Thalassemia
Children's National Research Institute
The Hospital for Sick Children; Ann & Robert H Lurie Children's Hospital of... and other collaborators

Recruiting

Sickle Cell Disease Transplant Using a Nonmyeloablative Approach for Patients With Anti-donor Red Cell AntibodY (SUN-RAY)

Sickle Cell Disease

United States
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
New York University

Not yet recruiting

mAnaging siCkle CELl disEase Through incReased AdopTion of hydroxyurEa in Nigeria (ACCELERATE)

Sickle Cell Disease
Oman Medical Speciality Board
Armed Forces Hospital, Oman

Not yet recruiting

Analgesic Effect of Parecoxib Versus Morphine in SCD Patients Presenting to the Emergency Department (PASC)

Sickle-Cell Disease With Crisis
Bioray Laboratories
Xiangya Hospital of Central South University; The 923rd Hospital of Joint Logistics...

Recruiting

Long-term Follow-up Study of BHC001 for TDT

Thalassemia, Beta

China
National Heart, Lung, and Blood Institute (NHLBI)
Children's National Research Institute

Recruiting

Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling

Sickle Cell Disease | Thalassemia | Hemoglobinopathy

United States
Lantu Biopharma

Not yet recruiting

An Open-label Study of a Gene Therapy Product in Beta-Thalassemia

Beta-Thalassemia
Bioray Laboratories
First Affiliated Hospital of Guangxi Medical University

Not yet recruiting

Clinical Study of BRL-101 in Severe SCD

Sickle Cell Disease

China
Children's Hospital Medical Center, Cincinnati
Makerere University; Ministry of Health, Uganda; Mulago Hospital, Uganda

Recruiting

Uganda Sickle Surveillance Study (US-3)

Sickle Cell Disease

Uganda
Kind Pharmaceuticals LLC

Not yet recruiting

A Study of Safety and Efficiency of AND017 in Patients With Transfusion Dependent and Non-transfusion Dependent β-thalassemia

β -Thalassemia
Bangabandhu Sheikh Mujib Medical University, Dhaka...

Recruiting

Efficacy of Combination of Hdroxyurea and Thalidomide Over Either Hydroxyurea or Thalidomide Alone in the Treatment of Transfusion Dependent Thalassemia in Children: A Quasi-Randomised Clinical Trial

Transfusion-dependent Thalassemia

Bangladesh
Bioray Laboratories
Xiangya Hospital of Central South University; Sun Yat-Sen Memorial Hospital... and other collaborators

Not yet recruiting

Long-term Follow-up Study of BRL-101 for TDT

Thalassemia, Beta

China
CorrectSequence Therapeutics Co., Ltd

Recruiting

A Safety and Efficacy Study Evaluating CS-101 in Subjects With β-Thalassemia Major

Beta-Thalassemia Major

China
Children's Hospital Medical Center, Cincinnati
Children's Hospital of Philadelphia; Emory University; Nemours Children's Health...

Not yet recruiting

A Socio-ecological Approach for Improving Self-management in Adolescents With SCD (SC-Thrive)

Anemia, Sickle Cell
Agios Pharmaceuticals, Inc.

Not yet recruiting

A Study of Mitapivat in Participants With Sickle Cell Disease and Nephropathy

Sickle Cell Disease | Nephropathy
Bioray Laboratories
Institute of Hematology & Hospital of Blood Diseases, Chinese Academy of...

Not yet recruiting

Clinical Study on the Safety and Efficacy of BRL-101 in the Treatment of Sickle Cell Disease

Sickle Cell Disease
Bioray Laboratories
Nanfang Hospital, Southern Medical University

Not yet recruiting

Clinical Study of BRL-101 in the Treatment of Sickle Cell Disease

Sickle Cell Disease
Kanglin Biotechnology (Hangzhou) Co., Ltd.

Not yet recruiting

A Phase I/II Clinical Study of the KL003 Cell Injection in β-Thalassemia Major Participants

Transfusion-dependent Beta-Thalassemia

China
bluebird bio
Center for International Blood and Marrow Transplant Research

Recruiting

A Study of Participants With β-Thalassemia Treated With Betibeglogene Autotemcel

Beta-Thalassemia

United States

Search clinical trials for: Congenital Nonspherocytic Hemolytic Anemia

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Trinidad & Tobago

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations