Clinical Trials on Shwachman-Diamond Syndrome-Like

Total 414 results

Boston Children's Hospital
Children's Hospital Medical Center, Cincinnati

Recruiting

Shwachman Diamond Syndrome Registry and Study (SDSR)

Shwachman-Diamond Syndrome | Shwachman-Diamond Syndrome-Like

United States
Emory University
Children's Healthcare of Atlanta

Terminated

Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation

Sickle Cell Disease | Fanconi Anemia | Thalassemia | Severe Congenital Neutropenia | Wiskott-Aldrich Syndrome | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Glanzmann Thrombasthenia | Leukocyte Adhesion Deficiency | Dyskeratosis-congenita | Chediak-Higashi Syndrome | Chronic-granulomatous Disease | Schwachman-Diamond...

United States
National Cancer Institute (NCI)

Recruiting

Cancer in Inherited Bone Marrow Failure Syndromes

Fanconi Anemia | Dyskeratosis Congenita | Diamond Blackfan Anemia | Shwachman Diamond Syndrome | Inherited Bone Marrow Failure Syndrome, Aplastic Anemia

United States
Masonic Cancer Center, University of Minnesota

Completed

Stem Cell Transplant for Bone Marrow Failure Syndromes

Diamond-Blackfan Anemia | Shwachman-Diamond Syndrome | Kostmann's Neutropenia

United States
University Hospital Freiburg

Completed

Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to Treatment

Myelodysplastic Syndromes | Fanconi Anemia | Dyskeratosis Congenita | Pearson Marrow-pancreas Syndrome | Shwachman-diamond Syndrome

Spain, Germany, Switzerland, Austria, Netherlands, Italy, Czech Republic, Belgium, Denmark, Ireland
Masonic Cancer Center, University of Minnesota
National Marrow Donor Program

Completed

Stem Cell Transplant for Hemoglobinopathy

Sickle Cell Disease | Thalassemia | Severe Congenital Neutropenia | Diamond-Blackfan Anemia | Shwachman-Diamond Syndrome

United States
Diane George

Active, not recruiting

CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation

Sickle Cell Disease | Hemoglobinopathies | Severe Congenital Neutropenia | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Macrophage Activation Syndrome | Bone Marrow Failure Syndrome | Amegakaryocytic Thrombocytopenia | Schwachman Diamond Syndrome | Primary Immunodeficiency Syndromes | Acquired Immunodeficiency... and other conditions

United States
Washington University School of Medicine
Shwachman Diamond Syndrome Foundation; Barnes-Jewish Hospital

Recruiting

Diabetes/ Endocrine Surveillance in SDS

Shwachman-Diamond Syndrome

United States
National Center for Research Resources (NCRR)
James Whitcomb Riley Hospital for Children

Completed

Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes

Thrombocytopenia | Dyskeratosis Congenita | Fanconi's Anemia | Shwachman Syndrome
University of California, San Francisco
National Cancer Institute (NCI)

Completed

Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders

Myelodysplastic Syndromes | Leukemia | Congenital Amegakaryocytic Thrombocytopenia | Severe Congenital Neutropenia | Diamond-blackfan Anemia

United States
Novartis

Completed

Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload

Myelodysplastic Syndromes | Hemoglobinopathies | Diamond-Blackfan Anemia | Transfusional Iron Overload | Other Inherited or Acquired Anaemia | MPD Syndrome | Other Rare Anaemias

Australia
St. Jude Children's Research Hospital

Completed

Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias

Anemia, Aplastic | Diamond-Blackfan Anemia | Amegakaryocytic Thrombocytopenia | Kostmann Syndrome

United States
Northwell Health

Completed

Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia (LeucineDBA)

Pure Red Cell Aplasia | Diamond Blackfan Anemia | Blackfan Diamond Syndrome | DBA | Congenital Hypoplastic Anemia

United States
Emory University

Completed

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

Sickle Cell Disease | Fanconi Anemia | Hurler Syndrome | Chronic Granulomatous Disease | Severe Congenital Neutropenia | Hemophagocytic Lymphohistiocytosis | Wiskott-Aldrich Syndrome | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Glanzmann Thrombasthenia | Shwachman-Diamond Syndrome | Thalassemia Major | Leukocyte... and other conditions

United States
National Heart, Lung, and Blood Institute (NHLBI)

Recruiting

Study of the Selective GlyT1 Inhibitor Bitopertin for Steroid-Refractory Diamond-Blackfan Anemia

Steroid-refractory Diamond-Blackfan Anemia (DBA)

United States
Jason Robert Gotlib
Celgene Corporation

Terminated

Pilot Lenalidomide in Adult Diamond-Blackfan Anemia Patients w/ RBC Transfusion-Dependent Anemia

Leukemia | Anemia | Myelodysplastic Syndromes (MDS) | Acute Myeloid Leukemia (AML)

United States
Masonic Cancer Center, University of Minnesota

Recruiting

Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

Sickle Cell Disease | Severe Congenital Neutropenia | Paroxysmal Nocturnal Hemoglobinuria | Glanzmann Thrombasthenia | Shwachman-Diamond Syndrome | Diamond Blackfan Anemia | Transfusion Dependent Alpha- or Beta- Thalassemia | Non-Malignant Hematologic Disorders

United States
Stanford University
University of Alabama at Birmingham; University of Minnesota

Completed

Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs

Sickle Cell Disease | Thalassemia | Diamond-Blackfan Anemia

United States
Ryan St. Pierre-Hetz

Active, not recruiting

Innovative Central Line Securement Device in the Pediatric Population

Quality of Life | Pediatric Disorder | Central Line Complication | Short Gut Syndrome | Central Line Infection | Central Line-Associated Infection | Central Line Sepsis

United States
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
Fred Hutchinson Cancer Center
National Cancer Institute (NCI); National Heart, Lung, and Blood Institute... and other collaborators

Recruiting

Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)

Congenital Amegakaryocytic Thrombocytopenia | Paroxysmal Nocturnal Hemoglobinuria | Shwachman-Diamond Syndrome | Bone Marrow Failure Syndrome | Congenital Pure Red Cell Aplasia | Hereditary Sideroblastic Anemia | Hematologic Neoplasm With Germline GATA2 Mutation | Hematologic Neoplasm With Germline... and other conditions

United States
Northwell Health

Terminated

Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia (ACE-011-DBA)

Diamond Blackfan Anemia

United States
National Heart, Lung, and Blood Institute (NHLBI)

Completed

Mobilization of Stem Cells With G-CSF for Collection From Patients With Diamond-Blackfan Anemia

Diamond Blackfan Anemia

United States
Paul Szabolcs

Recruiting

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

Sickle Cell Anemia | Diamond-blackfan Anemia | Beta-thalassemia Major

United States
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
Federal Scientific Clinical Centre of Pediatric...

Unknown

L-leucine in Diamond Blackfan Anemia Patients

Diamond Blackfan Anemia

Russian Federation
Feinstein Institute for Medical Research
Diamond Blackfan Anemia Foundation, Inc.

Recruiting

Diamond Blackfan Anemia Registry (DBAR) (DBAR)

Anemia | Blood Disease

United States
National Heart, Lung, and Blood Institute (NHLBI)

Active, not recruiting

Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag

Anemia, Diamond-Blackfan

United States
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
Adrianna Vlachos, MD

Terminated

The Use of Trifluoperazine in Transfusion Dependent DBA (DBA)

Pure Red Cell Aplasia | Diamond Blackfan Anemia

United States
National Heart, Lung, and Blood Institute (NHLBI)

Completed

Rituximab to Treat Moderate Aplastic Anemia, Pure Red Cell Aplasia, or Diamond Blackfan Anemia

Anemia, Aplastic | Red-Cell Aplasia, Pure | Anemia, Diamond-Blackfan

United States
National Heart, Lung, and Blood Institute (NHLBI)

Terminated

A Study to Determine Whether Therapy With Daclizumab Will Benefit Patients With Bone Marrow Failure

Aplastic Anemia | Pure Red Cell Aplasia | Diamond Blackfan Anemia

United States
St. Jude Children's Research Hospital
Baylor College of Medicine; Children's Hospital of Philadelphia; Dana-Farber... and other collaborators

Recruiting

Investigation of the Genetics of Hematologic Diseases

Myelodysplastic Syndromes | Sickle Cell Disease | Fanconi Anemia | Severe Congenital Neutropenia | Bone Marrow Failure Syndromes | Blood Coagulation Disorder | Erythrocyte Disorder | Leukocyte Disorder | Hemostasis | Dyskeratosis Congenita | Diamond-Blackfan Anemia | Congenital Thrombocytopenia | Myeloproliferative...

United States
Masonic Cancer Center, University of Minnesota
National Cancer Institute (NCI)

Terminated

Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer

Lymphoma | Myelodysplastic Syndromes | Leukemia | Chronic Myeloproliferative Disorders | Fanconi Anemia | Multiple Myeloma and Plasma Cell Neoplasm | Myelodysplastic/Myeloproliferative Diseases | Graft Versus Host Disease | Diamond-blackfan Anemia

United States
National Heart, Lung, and Blood Institute (NHLBI)

Completed

Medical Treatment for Diamond Blackfan Anemia

Hematologic Disease | Fanconi's Anemia

United States
University of California, San Francisco
National Cancer Institute (NCI)

Completed

Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First Remission

Leukemia | Thrombocytopenia | Fanconi Anemia | Congenital Amegakaryocytic Thrombocytopenia | Severe Congenital Neutropenia | Diamond-blackfan Anemia

United States
Children's Hospital Los Angeles
Lucile Packard Children's Hospital

Terminated

Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells

Metabolic Diseases | Stem Cell Transplantation | Chronic Granulomatous Disease | Bone Marrow Transplantation | Thalassemia | Wiskott-Aldrich Syndrome | Genetic Diseases | Peripheral Blood Stem Cell Transplantation | Pediatrics | Diamond-Blackfan Anemia | Allogeneic Transplantation | Combined Immune Deficiency | X-linked Lymphoproliferative Disease
Abramson Cancer Center of the University of Pennsylvania
Children's Hospital of Philadelphia

Recruiting

Li-Fraumeni Syndrome/TP53 Biobank

Li-Fraumeni Syndrome | Li-Fraumeni-Like Syndrome

United States
Istituto Ortopedico Rizzoli

Recruiting

Registry of Li Fraumeni and Li Fraumeni Like Syndromes (ReLF)

Li-Fraumeni Syndrome | Li-Fraumeni-Like Syndrome

Italy
Novartis Pharmaceuticals

Completed

Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload

Myelofibrosis | Sickle Cell Disease | Thalassemia | Diamond Blackfan Anemia

United States, Netherlands, Taiwan, Thailand, Italy, Germany, Spain, Canada, Belgium, Greece, Turkey, United Kingdom
University Hospital, Montpellier

Not yet recruiting

Validation of a Quality of Life Assessment Tool in Adult Generalized Dystonodyskinetic Syndromes Treated by Continuous Electrical Neuromodulation (DBS-QoLs)

Dystono-dyskinetic Syndromes
Bellicum Pharmaceuticals

Terminated

Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study

Lymphoma, Non-Hodgkin | Myelodysplastic Syndromes | Acute Lymphoblastic Leukemia | Primary Immunodeficiency | Anemia, Sickle Cell | Hemoglobinopathies | Fanconi Anemia | Anemia, Aplastic | Thalassemia | Cytopenia | Diamond Blackfan Anemia | Leukemia, Acute Myeloid (AML), Child

Italy
Novartis Pharmaceuticals

Completed

Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias

Myelodysplastic Syndromes | Fanconi Syndrome | Anemia, Aplastic | Beta-thalassemia | Anemia, Diamond-Blackfan

United States
Janssen-Cilag S.p.A.

Completed

Survey on QUality of Life In myeloDisplasia (SQUID)

Myelodysplastic Syndromes
Gruppo Italiano Malattie EMatologiche dell'Adulto

Unknown

Quality of Life and Symptoms in Patients With Newly Diagnosed Myelodysplastic Syndromes (PROMYS)

Myelodysplastic Syndromes | Adult

Belgium, United States, Austria, United Kingdom, Italy, Taiwan, Brazil, China, Croatia, Czechia, France, Germany
Bellicum Pharmaceuticals

Terminated

Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant

Lymphoma, Non-Hodgkin | Acute Lymphoblastic Leukemia | Primary Immunodeficiency | Anemia, Sickle Cell | Hemoglobinopathies | Fanconi Anemia | Anemia, Aplastic | Myelodysplastic Syndrome | Thalassemia | Cytopenia | Diamond Blackfan Anemia | Osteopetrosis | Leukemia, Acute Myeloid (AML), Child

United Kingdom, Italy
Lille Catholic University

Terminated

Impact of 2 Transfusion Strategies on Quality of Life of Multitransfused Patients With Low-risk Myelodysplastic Syndrome (SMD-transfu)

Myelodysplastic Syndromes | Transfusion-dependent Anemia

France
Charite University, Berlin, Germany
Takeda

Unknown

Quality of Life in Patients With Short Bowel Syndrome Treated Without and With Teduglutide - a Prospective Nested Matched Pair Analysis (MatchedQoL)

Short Bowel Syndrome | Intestinal Failure

Germany
Center for International Blood and Marrow Transplant...
Medical College of Wisconsin

Completed

Study of Quality of Life in Older vs. Younger Adult Patients Undergoing Allogeneic Hematopoietic Cell Transplantation for Myelodysplastic Syndromes

Myelodysplastic Syndromes | Hematopoietic Cell Transplant

United States
Sunnybrook Health Sciences Centre
Princess Margaret Hospital, Canada; Juravinski Cancer Center

Unknown

Red Blood Cell Transfusion Thresholds and QOL in MDS (EnhanceRBC)

Quality of Life | Myelodysplastic Syndromes (MDS) | Red Blood Cell (RBC) Transfusions

Canada

Clinical Trials on Shwachman-Diamond Syndrome-Like

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