Bone Marrow Failure Clinical Trials

3 recruiting

Frequently Asked Questions

Common questions about Bone Marrow Failure 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 111 of 11 trials

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 LeukemiaAMLMDS+4 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
Phase 2

Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders

AMLMDSHLH+6 more
Memorial Sloan Kettering Cancer Center31 enrolled1 locationNCT04644016
Recruiting

Cancer in Inherited Bone Marrow Failure Syndromes

Diamond-Blackfan AnemiaDyskeratosis CongenitaFanconi Anemia+2 more
National Cancer Institute (NCI)4,000 enrolled2 locationsNCT00027274
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 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
Phase 2

Myeloablative Allo HSCT With Related or Unrelated Donor for Heme Disorders

Acute Lymphoblastic LeukemiaAcute Myeloid LeukemiaChronic Lymphocytic Leukemia+25 more
Masonic Cancer Center, University of Minnesota300 enrolled1 locationNCT03314974
Recruiting

Shwachman-Diamond Syndrome Global Patient Survey and Partnering Platform

Myelodysplastic SyndromesShwachman-Diamond SyndromeSDS+24 more
Shwachman-Diamond Syndrome Alliance Inc8,000 enrolled1 locationNCT06999954
Recruiting
Phase 1Phase 2

Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT

LeukemiaInborn Errors of MetabolismHLH+11 more
Children's Hospital of Philadelphia100 enrolled1 locationNCT06839456
Recruiting

European Rare Blood Disorders Platform (ENROL)

LeukemiaMyelomaAnemia+9 more
Hospital Universitari Vall d'Hebron Research Institute37,090 enrolled1 locationNCT06250595