Condition (MeSH)
Epidermolysis Bullosa Dystrophica
ClinicalTrials.gov lists 77 trials for Epidermolysis Bullosa Dystrophica or a narrower condition beneath it. Krystal Biotech, Inc. has the most among industry and government lead sponsors, with 7.
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Trials per phase
77 of the 77 trials are tagged with Epidermolysis Bullosa Dystrophica itself; the rest are tagged with a narrower condition beneath it.
When did Epidermolysis Bullosa Dystrophica trials start? (18)
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
- Not applicable
- Observational
Top of scale: 9
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) | 7 |
| 2025 | 5 |
| 2024 | 7 |
| 2023 | 5 |
| 2022 | 1 |
| 2021 | 7 |
| 2020 | 9 |
| 2019 | 4 |
| 2018 | 5 |
| 2017 | 5 |
Showing 10 of 18 earlier years.
Forecast of the full-year count, planned starts included, not rows of this table: about 6 trials starting in 2026 and about 5 in 2027, the average of the 24 months through September, counted as registered and carried forward. The rows of this table count trials registered so far with a start date in the year, planned starts included; the forecast looks ahead to the count for the whole year, so it is not the same measure. In backtests on conditions of this size, forecasts made part-way through the year were typically off by about 28 percent and ran about 20 percent low against the count later registered; forecasts made before the year began were off by about 39 percent and ran about 37 percent low against the count later registered. These figures rest on a few years of history (target years from 2018 to 2025, varying by figure), place each condition as it is mapped today rather than as it was then, and score against a count for the newest year that was still being revised. Only condition series were scored. The 2026 forecast is below the 7 already registered with a 2026 start date: at the cutoff, 2 trials were already registered to start in October to December, more than the method's monthly average allows for, and the method does not use those planned starts.
Prediction, not a registry value: median of 24 months from start to primary completion across 10 trials still running at the 2026-10-09 snapshot; registered median of 17 months across 40 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Epidermolysis Bullosa Dystrophica trials in? (7)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 6 |
| Phase 1/2 | 22 |
| Phase 2 | 10 |
| Phase 3 | 10 |
| Phase 4 | 2 |
| Not applicable | 6 |
| Observational | 21 |
How many Epidermolysis Bullosa Dystrophica trials are recruiting? (7)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 3 |
| Recruiting | 13 |
| Active, not recruiting | 8 |
| Completed | 28 |
| Terminated | 8 |
Showing 5 of 7 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Epidermolysis Bullosa Dystrophica trials? (8)
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 |
|---|---|
| Krystal Biotech, Inc. | 7 |
| Abeona Therapeutics, Inc | 5 |
| Anterogen Co., Ltd. | 3 |
| Castle Creek Biosciences, LLC. | 3 |
| Phoenix Tissue Repair, Inc. | 3 |
Showing 5 of 8 sponsors.
Which drugs are being tested for Epidermolysis Bullosa Dystrophica? (2)
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 |
|---|---|
| FCX-007 (dabocemagene autoficel; see below for FCX-007 description) | 2 |
| Busulfan | 2 |
Interventions as named in the registry (13)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Gentamicins | 3 |
| PTR-01 | 3 |
| Topical Beremagene Geperpavec | 2 |
| SQ Fludarabine | 2 |
| HP802-247 | 1 |
Showing 5 of 13 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 Epidermolysis Bullosa Dystrophica 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 |
|---|---|---|
| Bacterial infection | 6 | 1 |
| Hyperbilirubinemia | 4 | 1 |
| Fungal infection | 2 | 1 |
| Neoplasms benign, malignant and unspecified, squamous cell carcinoma | 2 | 1 |
| Renal insufficiency | 2 | 1 |
Showing 5 of 10 adverse events.
Which publications cover Epidermolysis Bullosa Dystrophica? (39)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Long-Term Safety and Tolerability of Beremagene Geperpavec-svdt (B-VEC) in an Open-Label Extension Study of Patients with Dystrophic Epidermolysis Bullosa. | American journal of clinical dermatology | 2025 | Yes |
| Prademagene zamikeracel for recessive dystrophic epidermolysis bullosa wounds (VIITAL): a two-centre, randomised, open-label, intrapatient-controlled phase 3 trial. | Lancet (London, England) | 2025 | Yes |
| Pregabalin for Neuropathic Pain and Itch in Recessive Dystrophic Epidermolysis Bullosa: A Randomized Crossover Trial. | JAMA dermatology | 2024 | Yes |
| ABCB5<sup>+</sup> mesenchymal stromal cells facilitate complete and durable wound closure in recessive dystrophic epidermolysis bullosa. | Cytotherapy | 2023 | Yes |
| In vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase 1 and 2 trial. | Nature medicine | 2022 | Yes |
Showing 5 of 39 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 |
|---|---|---|---|---|---|
| Dystrophic epidermolysis bullosa | Point prevalence | 1-9 / 1 000 000 | United States | US | Unspecified |
| Dystrophic epidermolysis bullosa | Point prevalence | 1-9 / 1 000 000 | United States | US | Exact |
| Dystrophic epidermolysis bullosa | Point prevalence | 1-9 / 1 000 000 | United States | US | Exact |
| Dystrophic epidermolysis bullosa | Annual incidence | 1-9 / 100 000 | Netherlands | Not stated | Exact |
| Dystrophic epidermolysis bullosa | Point prevalence | 1-9 / 1 000 000 | Romania | Not stated | Exact |
Showing 5 of 21 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Dystrophic epidermolysis bullosa | 303 | Exact | Orphadata Science knowledge files, Product 1 |
| Dystrophic epidermolysis bullosa | 303 | 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.