Condition (MeSH)
Anemia, Dyserythropoietic, Congenital
ClinicalTrials.gov lists 10 trials for Anemia, Dyserythropoietic, Congenital or a narrower condition beneath it. UCB Pharma has the most among industry and government lead sponsors, with 2.
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Trials per phase
10 of the 10 trials are tagged with Anemia, Dyserythropoietic, Congenital itself; the rest are tagged with a narrower condition beneath it.
When did Anemia, Dyserythropoietic, Congenital trials start? (9)
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 3
- Observational
Top of scale: 2
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) | 1 |
| 2023 | 1 |
| 2020 | 1 |
| 2017 | 1 |
| 2016 | 1 |
| 2010 | 2 |
| 2008 | 1 |
| 2004 | 1 |
| 2003 | 1 |
No forecast is made for this condition: it averages 0.4 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 1 trial 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, Dyserythropoietic, Congenital trials in? (5)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 1 |
| Phase 1/2 | 2 |
| Phase 2 | 1 |
| Phase 3 | 2 |
| Observational | 4 |
How many Anemia, Dyserythropoietic, Congenital trials are recruiting? (4)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 3 |
| Completed | 4 |
| Withdrawn | 1 |
| Unknown status | 2 |
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Anemia, Dyserythropoietic, Congenital 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 |
|---|---|
| UCB Pharma | 2 |
| Telavant, Inc. | 1 |
| Wolfson Medical Center | 1 |
Which drugs are being tested for Anemia, Dyserythropoietic, Congenital? (1)
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 |
|---|---|
| Certolizumab | 2 |
Interventions as named in the registry (4)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| MOC31-PE | 1 |
| acid Zoledronic | 1 |
| Hematopoietic Stem Cell Transplantation | 1 |
| Investigational Product | 1 |
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 publications cover Anemia, Dyserythropoietic, Congenital? (6)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Mutations in the <i>RACGAP1</i> gene cause autosomal recessive congenital dyserythropoietic anemia type III. | Haematologica | 2023 | Yes |
| Novel Majeed Syndrome-Causing LPIN2 Mutations Link Bone Inflammation to Inflammatory M2 Macrophages and Accelerated Osteoclastogenesis. | Arthritis & rheumatology (Hoboken, N.J.) | 2021 | Yes |
| Congenital dyserythropoietic anemia type I: First report from the Congenital Dyserythropoietic Anemia Registry of North America (CDAR). | Blood cells, molecules & diseases | 2021 | Yes |
| VPS4A Mutations in Humans Cause Syndromic Congenital Dyserythropoietic Anemia due to Cytokinesis and Trafficking Defects. | American journal of human genetics | 2020 | Yes |
| Designing a single-arm phase 2 clinical trial of mitapivat for adult patients with erythrocyte membranopathies (SATISFY): a framework for interventional trials in rare anaemias - pilot study protocol. | BMJ open | 2024 | No |
Showing 5 of 6 publications.
Prevalence (12)
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 |
|---|---|---|---|---|---|
| Congenital dyserythropoietic anemia | Prevalence at birth | 1-9 / 1 000 000 | Europe | Not stated | Exact |
| Congenital dyserythropoietic anemia | Point prevalence | 1-9 / 1 000 000 | Europe | Not stated | Unspecified |
| Congenital dyserythropoietic anemia | Point prevalence | 1-9 / 1 000 000 | Europe | Not stated | Exact |
| Congenital dyserythropoietic anemia | Point prevalence | 1-9 / 1 000 000 | Worldwide | Not stated | Exact |
| Congenital dyserythropoietic anemia | Prevalence at birth | 1-9 / 1 000 000 | Europe | Not stated | Unspecified |
Showing 5 of 12 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Congenital dyserythropoietic anemia | 85 | Exact | Orphadata Science knowledge files, Product 1 |
| Congenital dyserythropoietic anemia | 85 | Exact | Orphanet Rare Disease Ontology (ORDO) |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: serious adverse events in this condition's trials and sub-conditions in mesh.