Clinical Trials on Bone Marrow Failure Disorders

Total 9665 results

Masonic Cancer Center, University of Minnesota

Terminated

Stem Cell Transplantation for Fanconi Anemia

Fanconi Anemia

United States
OHSU Knight Cancer Institute
National Cancer Institute (NCI)

Terminated

Gene Function in Bone Marrow Cells From Patients With Fanconi Anemia and From Healthy Participants

Fanconi Anemia

United States
Memorial Sloan Kettering Cancer Center
National Cancer Institute (NCI); Miltenyi Biotec, Inc.

Completed

Hematopoietic Stem Cell Transplantation for Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and Fludarabine

Fanconi Anemia

United States
Fairview University Medical Center

Completed

Study of Allogeneic Bone Marrow Transplantation Following Cyclophosphamide and Radiotherapy in Patients With Fanconi's Anemia

Fanconi's Anemia

United States
Dana-Farber Cancer Institute

Unknown

Phase I Study of Amifostine in Patients With Bone Marrow Failure Related to Fanconi's Anemia

Fanconi's Anemia

United States
Boston Children's Hospital
Massachusetts General Hospital; Mayo Clinic; Karolinska University Hospital; Baylor... and other collaborators

Active, not recruiting

Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita

Aplastic Anemia | Dyskeratosis Congenita | Hoyeraal Hreidarsson Syndrome | Revesz Syndrome

United States, Norway, Sweden
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
Assistance Publique - Hôpitaux de Paris
EuroFancolen

Completed

Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and Plerixafor (FancoMob)

Fanconi Anemia

France
Masonic Cancer Center, University of Minnesota

Completed

TBI Dose De-escalation for Fanconi Anemia

Fanconi Anemia

United States
Children's Hospital Medical Center, Cincinnati
National Heart, Lung, and Blood Institute (NHLBI)

Completed

Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and AMD3100

Fanconi Anemia

United States
Shenzhen Geno-Immune Medical Institute

Unknown

FANCA Gene Transfer for Fanconi Anemia Using a High-safety, High-efficiency, Self-inactivating Lentiviral Vector

Fanconi Anemia

China
Medical College of Wisconsin
Memorial Sloan Kettering Cancer Center

Completed

Hematopoietic Stem Cell Transplant for Fanconi Anemia (FA)

Fanconi Anemia

United States
Neena Kapoor, M.D.

Withdrawn

Unrelated HSCT in Patients With Fanconi Anemia

Fanconi Anemia

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

Completed

Gene Therapy for the Treatment of Fanconi's Anemia Type C

Pancytopenia | Fanconi's Anemia

United States
Boston Children's Hospital
National Heart, Lung, and Blood Institute (NHLBI); Children's Hospital Medical...

Completed

Bone Marrow Cell Gene Transfer in Individuals With Fanconi Anemia

Fanconi Anemia

United States
Fred Hutchinson Cancer Center
National Cancer Institute (NCI)

Completed

Cyclophosphamide in Treating Patients Who Are Undergoing a Donor Bone Marrow Transplant for Fanconi's Anemia

Fanconi Anemia

United States, Brazil
University of Minnesota

Unknown

Phase I Pilot Study of CD34 Enriched, Fanconi's Anemia Complementation Group C Gene Transduced Autologous Peripheral Blood Stem Cell Transplantation in Patients With Fanconi's Anemia

Fanconi's Anemia
Regeneron Pharmaceuticals

Recruiting

REGN7257 in Adult Patients With Severe Aplastic Anemia That Is Refractory to or Relapsed on Immunosuppressive Therapy

Severe Aplastic Anemia (SAA)

United States, France, Korea, Republic of, United Kingdom
BioLineRx, Ltd.

Completed

Phase IIA Open Label Study to Evaluate Efficacy and Safety of BL-8040 Followed by (hATG), Cyclosporine and Methyprednisolone in Adult Subjects With Aplastic Anemia or Hypoplastic Myelodysplastic Syndrome

Aplastic Anemia | Hypoplastic Myelodysplastic Syndrome

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

Recruiting

Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome

Myelodysplastic Syndrome (MDS) | Severe Aplastic Anemia | Hypo-Plastic MDS

United States
National Heart, Lung, and Blood Institute (NHLBI)
National Cancer Institute (NCI); National Institute of Allergy and Infectious... and other collaborators

Recruiting

Safety Study of Cord Blood Units for Stem Cell Transplants

Lymphoma | Leukemia | Aplastic Anemia | Myelodysplastic Syndrome (MDS)

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
Children's Hospital of Philadelphia
National Cancer Institute (NCI); University of Pennsylvania; City of Hope Medical... and other collaborators

Recruiting

Exercise Training and NR Supplementation Trial to Improve Fitness in AYA HCT Survivors (IAMFIT)

Myelodysplastic Syndromes | Aplastic Anemia | Lymphoblastic Leukemia | Cancer Survivors

United States
Rocket Pharmaceuticals Inc.

Unknown

A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A

Fanconi Anemia Complementation Group A

United States
Children's Hospital Medical Center, Cincinnati
Amgen

Completed

Pilot Study of Etanercept (Enbrel) in Children With Fanconi Anemia

Fanconi Anemia

United States
Rocket Pharmaceuticals Inc.

Enrolling by invitation

Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy

Fanconi Anemia | Fanconi Anemia Complementation Group A

Spain
Baylor College of Medicine
The Methodist Hospital Research Institute; Center for Cell and Gene Therapy...

Terminated

Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Fanconi Anemia (Mafia)

Fanconi Anemia | Severe Aplastic Anemia

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

Completed

Alemtuzumab to Treat Severe Aplastic Anemia

Severe Aplastic Anemia, Refractory | Severe Aplastic Anemia, Relapse

United States
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
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
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
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
UNC Lineberger Comprehensive Cancer Center

Recruiting

Impact of Physical and Occupational Therapy Therapy in New BMT Patients

Bone Marrow Transplantation | Bone Marrow Disease

United States
M.D. Anderson Cancer Center

Terminated

Long Term Minor Sibling Donor Well-Being

Blood Stem Cell Transplant Failure | Disorder Related to Bone Marrow Transplantation | Transmission, Blood, Recipient/Donor | Disturbance of Emotions Specific to Childhood and Adolescence | Sibling

United States
Joanne Kurtzberg, MD
Miltenyi Biomedicine GmbH

Completed

Unrelated Cord Blood Transplant Plus a Haplo-Identical (Half-Matched), T-Cell Depleted Stem Transplant From a Related Donor for Subjects With High Risk Malignancies

Hematologic Malignancy | Aplastic Anemia | Myelodysplastic Syndrome (MDS)

United States
National Center for Research Resources (NCRR)
University of California, San Diego

Completed

Evaluation of Fanconi Syndrome and Cystinosis

Fanconi Syndrome | Cystinosis
Peking Union Medical College Hospital
Novartis

Unknown

Study of MRI Monitoring in Patients With Aplastic Anemia and Low or Int-1 Risk of MDS Complicated With Iron Overload

Iron Overload | Aplastic Anemia | Dysmyelopoietic Syndromes
Eunice Kennedy Shriver National Institute of Child...

Terminated

Treatment of Menorrhagia in Women With Thrombocytopenia Using Platelets or Platelets and Hormones

Amenorrhea | Aplastic Anemia | Menorrhagia

United States
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
St. Justine's Hospital

Withdrawn

Denosumab for Prevention of Bone Complications After Bone Marrow Transplantation in Children

Disorder Related to Bone Marrow Transplantation

Canada
New York Medical College

Completed

CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant

Lymphoma | Leukemia | Sickle Cell Disease | Histiocytosis | Immunodeficiencies | Inborn Errors of Metabolism | Bone Marrow Failure | Beta Thalassemia

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
M.D. Anderson Cancer Center
National Cancer Institute (NCI)

Recruiting

Methylprednisolone, Horse Anti-Thymocyte Globulin, Cyclosporine, Filgrastim, and/or Pegfilgrastim or Pegfilgrastim Biosimilar in Treating Patients With Aplastic Anemia or Low or Intermediate-Risk Myelodysplastic Syndrome

Previously Treated Myelodysplastic Syndrome | Myelodysplastic Syndrome | Aplastic Anemia | de Novo Myelodysplastic Syndrome

United States
Tufts Medical Center

Terminated

A Phase II Study of Umbilical Cord Blood Transplantation

Lymphoma | Leukemia | Multiple Myeloma | Aplastic Anemia

United States
University Hospital, Basel, Switzerland
Novartis; ProPatient foundation of the University Hospital Basel

Completed

Electronic Patient-reported Outcome Monitoring in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (ePRO-AA-PNH)

Aplastic Anemia | Paroxysmal Nocturnal Hemoglobinuria

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

Completed

Drug Etiology of Aplastic Anemia and Related Dyscrasias

Purpura, Thrombocytopenic | Agranulocytosis | Anemia, Aplastic | Blood Disease
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Observational Follow-up Study of Haplo-identical Transplants in Fanconi Disease (HAPLO-FANCONI)

Fanconi Syndrome
Porteus, Matthew, MD

Recruiting

Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab

Fanconi Anemia

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

Recruiting

Eltrombopag for People With Fanconi Anemia

Fanconi Anemia

United States
Fred Hutchinson Cancer Center
National Heart, Lung, and Blood Institute (NHLBI); Rocket Pharma Limited

Terminated

Gene Therapy for Fanconi Anemia

Fanconi Anemia

United States

Clinical Trials on Bone Marrow Failure Disorders

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Equatorial Guinea

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations