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ClinicalTrials.gov 8 recruiting now official registry

Fanconi Anemia clinical trials

8 of 16 indexed Fanconi Anemia trials are currently recruiting.

16 US clinical trials · 8 currently recruiting

Fanconi Anemia is the subject of 16 registered US clinical trials on ClinicalTrials.gov, 8 of them currently open to new participants. 0 are in Phase 3-4 (later-stage) and 9 in Phase 1-2 (earlier-stage). The most active sponsor is Masonic Cancer Center, University of Minnesota, running 3 of these trials.

The research picture

Fanconi Anemia has 16 registered US clinical trials, 8 of them open to new participants right now, about 50% of the total.

8
recruiting participants now
50%
of trials open to enrollment
0
in Phase 3–4 (later-stage)
3
top sponsor: Masonic Cancer Center, University of Minnesota
Phase 12Phase 27

Active & Recent Trials

16 total, page 1 of 1

RECRUITING 4,000 participants

Cancer in Inherited Bone Marrow Failure Syndromes

National Cancer Institute (NCI)

NCT00027274

RECRUITING 200 participants

Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia

National Cancer Institute (NCI)

NCT05687149

RECRUITING NA 170 participants

Role of Acetaldehyde in the Development of Oral Cancer

Masonic Cancer Center, University of Minnesota

NCT05973656

RECRUITING Phase 2 70 participants

HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy

Children's Hospital Medical Center, Cincinnati

NCT02143830

RECRUITING Phase 2 48 participants

Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders

Masonic Cancer Center, University of Minnesota

NCT03579875

RECRUITING Phase 2 27 participants

Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

NCT04232085

RECRUITING 20 participants

Experience and Management of Cancer Screening-Related Anxiety in Fanconi Anemia

National Cancer Institute (NCI)

NCT06744283

RECRUITING Phase 1 18 participants

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

Porteus, Matthew, MD

NCT04784052

ACTIVE NOT RECRUITING NA 338 participants

Widefield Fluorescence and Reflectance Imaging Systems and Oral Tissue Samples in Monitoring Participants at Risk for Developing Oral Cancer

M.D. Anderson Cancer Center

NCT00542373

ACTIVE NOT RECRUITING Phase 2 48 participants

Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia

Children's Hospital Medical Center, Cincinnati

NCT03476330

ACTIVE NOT RECRUITING Phase 2 25 participants

Eltrombopag for People With Fanconi Anemia

National Heart, Lung, and Blood Institute (NHLBI)

NCT03206086

COMPLETED Phase 1 40 participants

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

University of California, San Francisco

NCT00305708

COMPLETED NA 21 participants

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

Memorial Sloan Kettering Cancer Center

NCT00595127

COMPLETED Phase 2 14 participants

Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia

Masonic Cancer Center, University of Minnesota

NCT00258427

COMPLETED NA 3 participants

Time-restricted Feeding to Reduce Inflammation in Fanconi Anemia

Children's Hospital Medical Center, Cincinnati

NCT05598515

COMPLETED Phase 2 1 participants

Hematopoietic Stem Cell Transplant for Fanconi Anemia

Medical College of Wisconsin

NCT01071239

Reading the Fanconi Anemia Trial Landscape

16 indexed Fanconi Anemia studies · 8 recruiting (50%).

Phase mix: 0 late (III/IV) vs 9 early (I/II).

Top sponsor: Masonic Cancer Center, University of Minnesota (3 trials), 9 others listed. 16 shown below, recruiting first.

How to read this condition's trial landscape

That ratio is a useful proxy for activity level: a high share of recruiting studies often signals that research interest is current and that new enrollment opportunities are appearing, while a low share typically means the field is dominated by completed or follow-up work where most participant spots have already been filled. These counts reflect the public registry only and include studies at every stage of design, so they should be read as an index of research attention rather than as a measure of treatment availability.

Phase 1 and Phase 2 studies focus on early safety signals, dosing, and preliminary effect, while Phase 3 studies are typically the larger efficacy and safety trials submitted toward regulatory review, and Phase 4 studies follow approved interventions in real-world use. A condition weighted toward later phases often reflects a mature research pipeline with several interventions already close to or past approval, whereas a heavier early-phase tilt suggests the field is still exploring new mechanisms and candidate approaches.

All figures are derived from the public ClinicalTrials.gov dataset maintained by the National Library of Medicine and are reproduced here for reference. Inclusion of a trial, sponsor, or intervention on this page is neither an endorsement nor a recommendation, eligibility, protocol changes, and site-level status can shift frequently, so always verify current details on ClinicalTrials.gov and consult a qualified healthcare provider before acting on anything you see here.

Frequently Asked Questions

How many clinical trials are there for Fanconi Anemia?

PlainTrial tracks 16 US clinical trials for Fanconi Anemia, of which 8 are currently recruiting participants. Data sourced from ClinicalTrials.gov.

How do I find a recruiting trial for Fanconi Anemia?

Use the trial list above filtered by "Recruiting" status, or visit our trial finder at /recruiting to search by condition and state. Always discuss trial participation with your healthcare provider before enrolling.

Is this data current?

Data is sourced from ClinicalTrials.gov and reflects our most recent data pull. Trial status may have changed since then. Always verify current information at ClinicalTrials.gov before making decisions about participation.

Disclaimer: This information is provided for informational purposes only and does not constitute professional advice. Data is sourced from ClinicalTrials.gov (National Library of Medicine). Consult a qualified professional before making decisions based on this data.

Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2026 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.

Verify with NIH →

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page aggregates every registered trial for this condition, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.