Fanconi Anemia clinical trials
The registry lists 95 studies for Fanconi Anemia across more than 10 countries. 20 are recruiting or about to start. 65 of them are mainly about Fanconi Anemia (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-12. Counting its narrower condition Fanconi Anemia, Complementation Group D1 too: 128 studies, 22 open.
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A study of drug A after surgery for early breast cancer
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· Sites: 4 added, including Seoul and Busan
· Enrollment: 300 → 450 participants
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Who runs them
Masonic Cancer Center, University of Minnesota (7) · Children's Hospital Medical Center, Cincinnati (7) · National Heart, Lung, and Blood Institute (NHLBI) (5) · University of California, San Francisco (5) · Assistance Publique - Hôpitaux de Paris (4) · National Cancer Institute (NCI) (4) · Fred Hutchinson Cancer Center (4) · Boston Children's Hospital (3)
What is being tested here
Drugs under test in Fanconi Anemia studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| Cyclophosphamide | 2 | 2 | 16 |
| Busulfan | 2 | 0 | 6 |
| Melphalan | 2 | 0 | 4 |
| Metformin | 2 | 1 | 3 |
| Fludarabine | 1 | 1 | 14 |
| Methylprednisolone | 1 | 0 | 4 |
| Granulocyte Colony-Stimulating Factor | 1 | 0 | 2 |
| Alemtuzumab | 1 | 0 | 2 |
| Naproxen | 1 | 0 | 1 |
| Ethanol | 1 | 0 | 1 |
Drug classes tested here Alkylating agents 3 open · Antimetabolites 1 open · Corticosteroids 1 open · Calcineurin inhibitors 3
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 7
- Dec 2026HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy · Children's Hospital Medical Center, Cincinnati
- Dec 2026Enhancing the Clinical Management of CMF Through a National Pediatric and Adult Network Based on Biological and Genetic Data · Istituto Giannina Gaslini
- Jan 2027Role of Acetaldehyde in the Development of Oral Cancer · Masonic Cancer Center, University of Minnesota
- Jan 2027Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders · Masonic Cancer Center, University of Minnesota
- Feb 2027French National Registry of Bone Marrow Failures · Assistance Publique - Hôpitaux de Paris
- Mar 2027Acute GVHD Suppression Using Costimulation Blockade to Expand Non-malignant Transplant · Emory University
- Apr 2027Widefield Fluorescence and Reflectance Imaging Systems and Oral Tissue Samples in Monitoring Participants at Risk for Developing Oral Cancer · M.D. Anderson Cancer Center
Who these studies take
Of the 15 Fanconi Anemia studies recruiting now, 8 (53%) set no upper age limit and 5 (33%) stop at 75 or younger. 11 (73%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 2 (13%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 11 (73%) | 11 (73%) |
| 18 | 15 (100%) | 15 (100%) |
| 30 | 14 (93%) | 14 (93%) |
| 40 | 14 (93%) | 14 (93%) |
| 50 | 13 (87%) | 13 (87%) |
| 65 | 12 (80%) | 12 (80%) |
| 75 | 10 (67%) | 10 (67%) |
| 85 | 10 (67%) | 10 (67%) |
Across all 95 studies, recruiting or not: 52% stop at 75 or younger and 49% at 65; 88% take children; 0% are for women only and 1% for men only; 7% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
What changed recently
From the registry's own updates since Oct 14, 2026.
Newly registered2
Studies
Recruiting first, then most recently updated. Showing 50 of 95. Browse all 95 → or search them.
- Clinical Genetics Branch Eligibility Screening SurveyNot yet recruitingNCT070052971 country
- Cancer in Inherited Bone Marrow Failure SyndromesRecruitingNCT000272741 country
- Defining the Natural History of Squamous Cell Carcinoma in Fanconi AnemiaRecruitingNCT056871491 country
- Safety and Tolerability of Metformin and Naproxen for Cancer Prevention in Fanconi AnemiaRecruitingNCT07901959Phase 11 country
- Enhancing the Clinical Management of CMF Through a National Pediatric and Adult Network Based on Biological and Genetic DataRecruitingNCT078777271 country
- Role of Acetaldehyde in the Development of Oral CancerRecruitingNCT05973656Not applicable1 country
- MRI as Noninvasive Innovative Approach in Detection and Monitoring of Malignant Oral Lesions in Fanconi Anemia PatientsRecruitingNCT076490311 country
- Familial Investigations of Childhood Cancer PredispositionRecruitingNCT030502681 country
- Prenatal Transplantation for Fetuses With Fanconi AnemiaNot yet recruitingNCT07408583Phase 1/21 country
- Investigation of the Genetics of Hematologic DiseasesRecruitingNCT027206791 country
- Afatinib in Patients With Fanconi Anemia (FA) and Advanced Head and Neck Squamous Cell Carcinoma (HNSCC)RecruitingNCT06648096Phase 1/22 countries
- Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102Enrolling by invitationNCT075279753 countries
- Expanded Access to CD34+ Selection Utilizing Miltenyi CliniMACS Prodigy® for Patients Receiving Peripheral Blood Stem Cell Transplantations and Stem Cell BoostsAvailableNCT06731036
- Alpha/Beta TCD HCT in Patients With Inherited BMF DisordersRecruitingNCT03579875Phase 21 country
- Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing BriquilimabRecruitingNCT04784052Phase 1/21 country
- Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow FailuresRecruitingNCT04232085Phase 21 country
- HSCT for Patients With Fanconi Anemia Using Risk-Adjusted ChemotherapyRecruitingNCT02143830Phase 21 country
- Safety and Efficacy of Metformin for Treatment of Cytopenia in Children and Adolescents With Fanconi AnemiaRecruitingNCT06519786Phase 31 country
- French National Registry of Bone Marrow FailuresRecruitingNCT047817901 country
- Use Massive Parallel Sequencing and Exome Capture Technology to Sequence the Exome of Fanconi Anemia Children and Their PatentsAvailableNCT019953051 country
- FANCA Gene Transfer for Fanconi Anemia Using a High-safety, High-efficiency, Self-inactivating Lentiviral VectorTerminatedNCT03351868Not applicable1 country
- Experience and Management of Cancer Screening-Related Anxiety in Fanconi AnemiaActive, not recruitingNCT067442831 country
- Eltrombopag for Fanconi AnemiaActive, not recruitingNCT03206086Phase 21 country
- Widefield Fluorescence and Reflectance Imaging Systems and Oral Tissue Samples in Monitoring Participants at Risk for Developing Oral CancerActive, not recruitingNCT00542373Not applicable1 country
- Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi AnemiaCompletedNCT03476330Phase 21 country
- Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene TherapyActive, not recruitingNCT044377711 country
- Acute GVHD Suppression Using Costimulation Blockade to Expand Non-malignant TransplantActive, not recruitingNCT03924401Phase 21 country
- A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 PatientsWithdrawnNCT062274291 country
- Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and PlerixaforCompletedNCT02678533Phase 1/21 country
- Corrective Exercise and Rehabilitation in Fanconi Anemia: A Case StudyCompletedNCT06958380Not applicable1 country
- Baby Detect : Genomic Newborn ScreeningCompletedNCT056874741 country
- A Dose Escalation Study of FP-045 in Patients With Fanconi AnemiaWithdrawnNCT04522375Phase 1/2
- Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant DiseasesCompletedNCT01019876Phase 2/31 country
- Time-restricted Feeding to Reduce Inflammation in Fanconi AnemiaCompletedNCT05598515Not applicable1 country
- Prognostic Significance of Mutation Type and Chromosome Fragility in Fanconi AnemiaCompletedNCT064905101 country
- Gene Therapy for Fanconi AnemiaTerminatedNCT01331018Phase 11 country
- Whole Blood Biospecimen Collection for Subjects With Fanconi AnemiaTerminatedNCT059108531 country
- Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant DiseasesTerminatedNCT02231710Phase 11 country
- Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype ACompletedNCT03157804Phase 1/21 country
- Quercetin in Children With Fanconi Anemia; a Pilot StudyCompletedNCT01720147Phase 11 country
- Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell TransplantTerminatedNCT02065869Phase 1/22 countries
- Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical StudyTerminatedNCT03733249Phase 1/21 country
- Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant PatientsCompletedNCT036098401 country
- Eltrombopag for Treatment of Fanconi AnemiaCompletedNCT06045052Phase 21 country
- A Phase 1/2 Trial SRA737 in Combination With Gemcitabine and Cisplatin or Gemcitabine Alone in Advanced Cancer SubjectsCompletedNCT02797977Phase 1/22 countries
- A Phase 1/2 Trial of SRA737 in Subjects With Advanced CancerCompletedNCT02797964Phase 1/21 country
- Microcephaly, Fanconi Anemia and Praxial DisordersWithdrawnNCT04656171Not applicable1 country
- Pilot Study of Metformin for Patients With Fanconi AnemiaCompletedNCT03398824Phase 21 country
- A Study of LY2606368 (Prexasertib) in Patients With Solid Tumors With Replicative Stress or Homologous Repair DeficiencyCompletedNCT02873975Phase 21 country
- Research Study in Healthy Volunteers of Patients With Fanconi Anemia, Myeloproliferative Disorders, or MyelomaCompletedNCT009000551 country