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Fanconi Anemia

Part of Anemia

1 published article · Updated continuously

Clinical Trial Landscape

Clinical Trials for Fanconi Anemia

10 trials tracked for Fanconi Anemia: 1 in phase 3 or 4 and 1 with published results. The most-cited published study has 111 citations.

10Trials tracked
1Phase 3 & 4
0Recruiting
1With published results
Phase distribution
Phase 3 1 Phase 2 7 Other / NA 2
  1. Phase 3 TBI Dose De-escalation for Fanconi Anemia Completed · 111 cited
  2. Phase 2 NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders Completed
  3. Phase 2 Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi Anemia Completed
  4. Phase 2 Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia Completed
  5. Phase 2 Pilot Study of Metformin for Patients With Fanconi Anemia Completed
  6. Phase 2 Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia Completed
Show 4 more trials
  1. Phase 2 Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and AMD3100 Completed
  2. Phase 2 Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant, Mycophenolate Mofetil, and Cyclosporine in Treating Patients With Fanconi Anemia Completed
  3. N/A Hematopoietic Stem Cell Transplantation for Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and Fludarabine Completed
  4. N/A Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders Completed

Showing the 10 most-cited and recently-updated of 10 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found Updated — new results For clinicians

Fanconi Anemia: what the trials found

Established clinical evidence includes the use of Cyclophosphamide, which was evaluated in multiple trials for its effects on transplant outcomes [2, 8, 9]. Specifically, data from one study showed that 71 patients experienced Grade ≥3 regimen-related toxicity, while another cohort reported a mean IgG level of 550.6 as a measure of immune reconstitution at 100 days post-transplant 2.

Anti-thymocyte globulin is established as an intervention across several trials [4, 5, 7].

Recent results — preliminary, needs further review

  • Quercetin (dietary supplement) was evaluated in a recent phase 2 trial 1.

For the clinician treating this condition

  • Cyclophosphamide has been utilized in clinical settings with documented outcomes regarding toxicity and immune reconstitution [2, 8, 9].
  • Anti-thymocyte globulin is an established intervention in the current literature [4, 5, 7].

AI synthesis of 2 cited trials, updated Jun 29, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.

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