Bone Marrow Failure Syndrome Clinical Trials

5 recruiting

Frequently Asked Questions

Common questions about Bone Marrow Failure Syndrome clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 117 of 17 trials

Recruiting
Phase 1

Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency

Inherited Bone Marrow Failure SyndromeFamilial Platelet Disorder With Predisposition to Myeloid Malignancies
National Cancer Institute (NCI)75 enrolled1 locationNCT06090669
Recruiting

Natural History of Acquired and Inherited Bone Marrow Failure Syndromes

Severe Aplastic AnemiaTelomere Biology DisordersInherited Bone Marrow Failure Syndromes
National Heart, Lung, and Blood Institute (NHLBI)1,000 enrolled1 locationNCT05012111
Recruiting
Phase 2

A Pilot Study to Evaluate the Feasibility of Post-Hematopoietic Stem Cell Transplant Prophylaxis With Decitabine Combined With Filgrastim for Children and Young Adults With AML, MDS and Related Myeloid Malignancies

Acute Myeloid LeukemiaMDSMyelodysplastic Syndromes+3 more
Franziska Wachter37 enrolled2 locationsNCT05796570
Recruiting

Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia

Fanconi AnemiaInherited Bone Marrow Failure Syndrome
National Cancer Institute (NCI)200 enrolled1 locationNCT05687149
Recruiting
Not Applicable

Pre-myeloid Cancer and Bone Marrow Failure Clinic Study

Hematopoietic and Lymphatic System NeoplasmHematologic NeoplasmsMyeloid Malignancy+10 more
Mayo Clinic2,000 enrolled3 locationsNCT02958462
Recruiting
Early Phase 1

Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC

LeukemiaInborn Errors of MetabolismBone Marrow Failure Syndromes+2 more
Children's Hospital of Philadelphia100 enrolled1 locationNCT02356653
Recruiting

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

Primary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)+2 more
Paul Szabolcs50 enrolled1 locationNCT04528355
Recruiting
Phase 1Phase 2

Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders

HemoglobinopathiesBone Marrow Failure SyndromesMetabolic Disorders+3 more
Washington University School of Medicine29 enrolled4 locationsNCT03128996
Recruiting
Phase 2

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

Juvenile Rheumatoid Arthritis (JRA)Primary Immunodeficiency (PID)Congenital Bone Marrow Failure Syndromes+4 more
Paul Szabolcs100 enrolled1 locationNCT01962415
Recruiting

Investigation of the Genetics of Hematologic Diseases

Diamond-Blackfan AnemiaDyskeratosis CongenitaFanconi Anemia+10 more
St. Jude Children's Research Hospital1,716 enrolled1 locationNCT02720679
Recruiting
Phase 2

Haploidentical HCT for Severe Aplastic Anemia

Aplastic AnemiaBone Marrow Failure Syndrome
St. Jude Children's Research Hospital21 enrolled1 locationNCT04558736
Recruiting
Phase 2

TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children

Severe Aplastic AnemiaHemoglobinopathy (Disorder)Bone Marrow Failure Syndrome
Johns Hopkins All Children's Hospital17 enrolled1 locationNCT04356469
Recruiting
Early Phase 1

Fludarabine Based RIC for Bone Marrow Failure Syndromes

Bone Marrow Failure Syndromes
Children's Hospital of Philadelphia25 enrolled1 locationNCT02928991
Recruiting
Early Phase 1

CD7 CAR-T Cell Sequential Allo-HSCT for Non-malignant Blood and Immune System Diseases

Bone Marrow Failure Syndrome
Zhejiang University20 enrolled1 locationNCT06787560
Recruiting

Aplastic Anaemia and Other Bone Marrow Failure Syndromes Registry (AAR)

Hereditary Predisposition to Haematological Malignancy (HPHM)Aplastic AnaemiaVEXAS Syndrome+1 more
Monash University1,000 enrolled37 locationsACTRN12623000461695
Recruiting

Australian Marrow Failure Biobank

Bone Marrow Failure Syndromes
Monash University200 enrolled5 locationsACTRN12623000424606
Recruiting

French National Registry of Bone Marrow Failures

Bone Marrow Failure Syndrome
Assistance Publique - Hôpitaux de Paris5,000 enrolled1 locationNCT04781790