Condition (MeSH)
Dyskeratosis Congenita
ClinicalTrials.gov lists 21 trials for Dyskeratosis Congenita or a narrower condition beneath it. National Cancer Institute (NCI) has the most among industry and government lead sponsors, with 2.
Search trials, drugs, companies and investigators, and follow each one to the others. Individual plan, $249/seat/mo. Card required.
Trials per phase
21 of the 21 trials are tagged with Dyskeratosis Congenita itself; the rest are tagged with a narrower condition beneath it.
When did Dyskeratosis Congenita trials start? (15)
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
- Not applicable
- 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 |
| 2025 | 1 |
| 2021 | 2 |
| 2020 | 1 |
| 2018 | 1 |
| 2017 | 1 |
| 2016 | 1 |
| 2015 | 2 |
| 2014 | 2 |
| 2012 | 1 |
Showing 10 of 15 earlier years.
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: 4 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 Dyskeratosis Congenita trials in? (7)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 4 |
| Phase 1/2 | 1 |
| Phase 2 | 7 |
| Phase 2/3 | 1 |
| Not applicable | 2 |
| Observational | 5 |
| Expanded access | 1 |
How many Dyskeratosis Congenita trials are recruiting? (5)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 8 |
| Active, not recruiting | 1 |
| Available | 1 |
| Completed | 7 |
| Terminated | 4 |
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Dyskeratosis Congenita 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 Cancer Institute (NCI) | 2 |
| Bellicum Pharmaceuticals | 1 |
| National Center for Research Resources (NCRR) | 1 |
Which drugs are being tested for Dyskeratosis Congenita? (4)
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 | 4 |
| Melphalan | 3 |
| Danazol | 2 |
| METHYLPREDNISOLONE | 2 |
Interventions as named in the registry (11)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| SQ Fludarabine | 7 |
| Alemtuzumab s.c. | 6 |
| Total Body Irradiation | 4 |
| Thymoglobulin [Anti-Thymocyte Globulin (Rabbit)] | 3 |
| IR Tacrolimus | 3 |
Showing 5 of 11 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 Dyskeratosis Congenita 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 |
|---|---|---|
| Hypoxia: Decreased O2 sat requiring support | 4 | 1 |
| Anaphylaxis reaction (rATG) | 2 | 1 |
| Chronic graft versus host disease | 2 | 1 |
| Fever (not neutropenic) | 2 | 1 |
| Fever (not neutropenic): attributed to rATG | 2 | 1 |
Showing 5 of 10 adverse events.
Which publications cover Dyskeratosis Congenita? (21)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Germline RTEL1 Variants in Telomere Biology Disorders. | American journal of medical genetics. Part A | 2025 | Yes |
| Clinical Use of ZSCAN4 for Telomere Elongation in Hematopoietic Stem Cells. | NEJM evidence | 2025 | Yes |
| A rare homozygous variant in TERT gene causing variable bone marrow failure, fragility fractures, rib anomalies and extremely short telomere lengths with high serum IgE. | British journal of haematology | 2024 | Yes |
| The distribution and accumulation of the shortest telomeres in telomere biology disorders. | British journal of haematology | 2023 | Yes |
| Disease progression and clinical outcomes in telomere biology disorders. | Blood | 2022 | Yes |
Showing 5 of 21 publications.
Prevalence (3)
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 |
|---|---|---|---|---|---|
| Dyskeratosis congenita | Point prevalence | 1-9 / 1 000 000 | Europe | Not stated | Exact |
| Dyskeratosis congenita | Point prevalence | 1-9 / 1 000 000 | Europe | Not stated | Exact |
| Dyskeratosis congenita | Point prevalence | 1-9 / 1 000 000 | Europe | Not stated | Unspecified |
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Dyskeratosis congenita | 1775 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Dyskeratosis congenita | 1775 | 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.