Condition (MeSH)
Anemia, Diamond-Blackfan
ClinicalTrials.gov lists 56 trials for Anemia, Diamond-Blackfan or a narrower condition beneath it.
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Trials per phase
56 of the 56 trials are tagged with Anemia, Diamond-Blackfan itself; the rest are tagged with a narrower condition beneath it.
When did Anemia, Diamond-Blackfan trials start? (26)
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.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Phase 2/3
- Phase 3
- Phase 4
- Not applicable
- Observational
Top of scale: 5
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) | 2 |
| 2022 | 2 |
| 2021 | 1 |
| 2020 | 2 |
| 2019 | 1 |
| 2018 | 2 |
| 2017 | 4 |
| 2016 | 1 |
| 2015 | 1 |
| 2014 | 5 |
Showing 10 of 26 earlier years.
No forecast is made for this condition: it averages 0.8 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 3 trials still running at the 2026-10-09 snapshot, fewer than the 5 needed for a median. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Anemia, Diamond-Blackfan trials in? (8)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 5 |
| Phase 1/2 | 12 |
| Phase 2 | 18 |
| Phase 2/3 | 2 |
| Phase 3 | 2 |
| Phase 4 | 1 |
| Not applicable | 7 |
| Observational | 9 |
How many Anemia, Diamond-Blackfan trials are recruiting? (6)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 10 |
| Active, not recruiting | 1 |
| Completed | 31 |
| Terminated | 11 |
| Withdrawn | 1 |
Showing 5 of 6 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Anemia, Diamond-Blackfan trials? (4)
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 |
| Novartis Pharmaceuticals | 5 |
| Bellicum Pharmaceuticals | 3 |
| Apriligen, Inc. | 1 |
Which drugs are being tested for Anemia, Diamond-Blackfan? (3)
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 |
|---|---|
| Busulfan | 6 |
| Cytoxan | 4 |
| Melphalan | 4 |
Interventions as named in the registry (12)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| SQ Fludarabine | 10 |
| Thymoglobulin [Anti-Thymocyte Globulin (Rabbit)] | 7 |
| Peripheral blood stem cell transplantation | 6 |
| Deferasirox FCT | 6 |
| Allogeneic bone marrow transplantation | 4 |
Showing 5 of 12 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 Anemia, Diamond-Blackfan 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 |
|---|---|---|
| Pyrexia | 28 | 5 |
| Sickle cell anaemia with crisis | 27 | 1 |
| Infection | 15 | 4 |
| Acute graft versus host disease | 12 | 2 |
| Sepsis | 11 | 6 |
Showing 5 of 10 adverse events.
Which publications cover Anemia, Diamond-Blackfan? (57)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Probable digenic inheritance of Diamond-Blackfan anemia. | American journal of medical genetics. Part A | 2024 | Yes |
| Diagnosis, treatment, and surveillance of Diamond-Blackfan anaemia syndrome: international consensus statement. | The Lancet. Haematology | 2024 | Yes |
| Successful gene therapy of Diamond-Blackfan anemia in a mouse model and human CD34<sup>+</sup> cord blood hematopoietic stem cells using a clinically applicable lentiviral vector. | Haematologica | 2022 | Yes |
| Downregulation of SATB1 by miRNAs reduces megakaryocyte/erythroid progenitor expansion in preclinical models of Diamond-Blackfan anemia. | Experimental hematology | 2022 | Yes |
| Genotype-phenotype association and variant characterization in Diamond-Blackfan anemia caused by pathogenic variants in <i>RPL35A</i>. | Haematologica | 2021 | Yes |
Showing 5 of 57 publications.
Prevalence (18)
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 |
|---|---|---|---|---|---|
| Diamond-Blackfan anemia | Prevalence at birth | 1-9 / 1 000 000 | United Kingdom | GB | Unspecified |
| Diamond-Blackfan anemia | Prevalence at birth | 1-9 / 1 000 000 | United Kingdom | GB | Exact |
| Diamond-Blackfan anemia | Prevalence at birth | 1-9 / 1 000 000 | United Kingdom | GB | Exact |
| Diamond-Blackfan anemia | Prevalence at birth | 1-9 / 1 000 000 | United States | US | Exact |
| Diamond-Blackfan anemia | Prevalence at birth | 1-9 / 1 000 000 | United States | US | Exact |
Showing 5 of 18 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Diamond-Blackfan anemia | 124 | Exact | Orphadata Science knowledge files, Product 1 |
| Diamond-Blackfan anemia | 124 | Exact | Orphanet Rare Disease Ontology (ORDO) |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.