Bone Marrow Failure Syndromes Clinical Trials

6 recruiting

Bone Marrow Failure Syndromes Trials at a Glance

9 actively recruiting trials for bone marrow failure syndromes are listed on ClinicalTrialsFinder across 6 cities in 2 countries. The largest study group is Early Phase 1 with 2 trials, with the heaviest enrollment activity in Philadelphia, Pittsburgh, and Memphis. Lead sponsors running bone marrow failure syndromes studies include Children's Hospital of Philadelphia, Monash University, and Paul Szabolcs.

Browse bone marrow failure syndromes trials by phase

Treatments under study

About Bone Marrow Failure Syndromes Clinical Trials

Looking for clinical trials for Bone Marrow Failure Syndromes? There are currently 6 studies actively recruiting participants. Clinical trials offer access to new treatments before they are widely available, and every approved therapy in use today was first tested through a clinical trial.

Below you can browse trials, sign up for alerts when new Bone Marrow Failure Syndromes trials open, and view eligibility criteria for each study. Each listing includes the study phase, locations, and enrollment details.

Frequently Asked Questions

Common questions about Bone Marrow Failure Syndromes 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 19 of 9 trials

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
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
Early Phase 1

Fludarabine Based RIC for Bone Marrow Failure Syndromes

Bone Marrow Failure Syndromes
Children's Hospital of Philadelphia25 enrolled1 locationNCT02928991
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