Condition (MeSH)
Fanconi Anemia
ClinicalTrials.gov lists 93 trials for Fanconi Anemia or a narrower condition beneath it. National Heart, Lung, and Blood Institute (NHLBI) has the most among industry and government lead sponsors, with 5.
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Trials per phase (log scale)
93 of the 93 trials are tagged with Fanconi Anemia itself; the rest are tagged with a narrower condition beneath it.
When did Fanconi Anemia trials start? (32)
Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year. 1 trial is registered to start in 2027 or later, and not listed here.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Phase 2/3
- Phase 3
- Not applicable
- Observational
Top of scale: 8
Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.
| Year | Trials started |
|---|---|
| 2026 (partial year) | 3 |
| 2025 | 2 |
| 2024 | 3 |
| 2023 | 4 |
| 2022 | 5 |
| 2021 | 1 |
| 2020 | 3 |
| 2019 | 1 |
| 2018 | 5 |
| 2017 | 5 |
Showing 10 of 32 earlier years.
No forecast is made for this condition: it averages 3.4 trial starts a year over the five years to September, under the 5 a year the method needs.
Prediction, not a registry value: median of 36 months from start to primary completion across 5 trials still running at the 2026-10-09 snapshot; registered median of 61 months across 55 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Fanconi Anemia trials in? (8)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 13 |
| Phase 1/2 | 17 |
| Phase 2 | 19 |
| Phase 2/3 | 2 |
| Phase 3 | 1 |
| Not applicable | 15 |
| Observational | 24 |
| Expanded access | 2 |
How many Fanconi Anemia trials are recruiting? (9)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 2 |
| Recruiting | 12 |
| Enrolling by invitation | 1 |
| Active, not recruiting | 5 |
| Available | 2 |
Showing 5 of 9 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Fanconi Anemia trials? (3)
Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.
| Sponsor | Trials |
|---|---|
| National Heart, Lung, and Blood Institute (NHLBI) | 5 |
| National Cancer Institute (NCI) | 4 |
| Bellicum Pharmaceuticals | 3 |
Which drugs are being tested for Fanconi Anemia? (6)
Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.
| Drug | Trials |
|---|---|
| Cytoxan | 19 |
| Busulfan | 9 |
| METHYLPREDNISOLONE | 8 |
| Melphalan | 6 |
| G-CSF | 4 |
Showing 5 of 6 drugs.
Interventions as named in the registry (9)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| SQ Fludarabine | 22 |
| Thymoglobulin [Anti-Thymocyte Globulin (Rabbit)] | 11 |
| Filgrastime (G-CSF) | 9 |
| Allogeneic bone marrow transplantation | 7 |
| Cyclosporine 0.05% | 7 |
Showing 5 of 9 interventions.
These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.
Which serious adverse events do Fanconi Anemia trials report? (10)
Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.
| Adverse event | Subjects affected | Trials |
|---|---|---|
| Neutrophil count decreased | 24 | 1 |
| Pyrexia | 23 | 4 |
| White blood cell decreased | 20 | 1 |
| Acute graft versus host disease | 12 | 2 |
| Hospitalisation | 12 | 1 |
Showing 5 of 10 adverse events.
Which publications cover Fanconi Anemia? (74)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Haematopoietic gene therapy of non-conditioned patients with Fanconi anaemia-A: results from open-label phase 1/2 (FANCOLEN-1) and long-term clinical trials. | Lancet (London, England) | 2025 | Yes |
| Prognostic significance of mutation type and chromosome fragility in Fanconi anemia. | American journal of hematology | 2025 | Yes |
| Phase 1 study of quercetin, a natural antioxidant for children and young adults with Fanconi anemia. | Blood advances | 2025 | Yes |
| Eltrombopag for Bone Marrow Failure in Fanconi Anemia: Results From the Phase II Clinical Trial FANCREV. | European journal of haematology | 2025 | Yes |
| Irradiation- and busulfan-free stem cell transplantation in Fanconi anemia using an anti-CD117 antibody: a phase 1b trial. | Nature medicine | 2025 | Yes |
Showing 5 of 74 publications.
Prevalence (21)
Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.
| Orphanet disease | Type | Class | Geography | Jurisdiction | Mapping relation |
|---|---|---|---|---|---|
| Fanconi anemia | Prevalence at birth | 1-9 / 100 000 | Israel | Not stated | Exact |
| Fanconi anemia | Point prevalence | 1-9 / 100 000 | Specific population | Not stated | Exact |
| Fanconi anemia | Point prevalence | 1-9 / 1 000 000 | Europe | Not stated | Unspecified |
| Fanconi anemia | Point prevalence | 1-9 / 100 000 | Specific population | Not stated | Unspecified |
| Fanconi anemia | Point prevalence | 1-9 / 100 000 | Specific population | Not stated | Unspecified |
Showing 5 of 21 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Fanconi anemia | 84 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Fanconi anemia | 84 | Exact | Orphadata Science knowledge files, Product 1 |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.