Disease · MONDO_0017610
epidermolysis bullosa simplex
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. epidermolysis bullosa simplex. Diseases, record MONDO_0017610. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0017610
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Disease
Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.
Drugs9
The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.
| Drug | ChEMBL ID | Type | Open Targets' stage | Stage reported by | Reports | Of them trials | Trials shown here | Source |
|---|---|---|---|---|---|---|---|---|
| ALLANTOIN | CHEMBL593429 | Small molecule | Phase 3 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| ERYTHROMYCIN | CHEMBL532 | Small molecule | Phase 3 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| NELITOLIMOD | CHEMBL6068457 | Oligonucleotide | Phase 3 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| DIACEREIN | CHEMBL41286 | Small molecule | Phase 2/3 | ClinicalTrials.gov | 5 | 5 | 5 | Drugs and targets |
| ONABOTULINUMTOXINA | CHEMBL1201574 | Phase 2/3 | ClinicalTrials.gov | 2 | 2 | 0 | Drugs and targets | |
| SIROLIMUS | CHEMBL413 | Small molecule | Phase 2 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
| APREMILAST | CHEMBL514800 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| BOTULINUM TOXIN TYPE A | CHEMBL4297862 | Protein | Phase 2 | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets |
| LETIBOTULINUMTOXINA | CHEMBL3707322 | Phase 2 | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets |
Targets6
The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.
| Target | Protein | Its drugs that act on it | Mechanisms | Highest stage of those drugs | Gene in Genes | Source |
|---|---|---|---|---|---|---|
| SNAP25 | Synaptosomal-associated protein 25 | 3 | Synaptosomal nerve-associated protein 25 (SNAP-25) hydrolytic enzyme; Synaptosomal nerve-associated protein 25 (SNAP-25) inhibitor | Phase 2/3 | https://conductscience.org/library/genes/6616 | Drugs and targets |
| FKBP1A | Peptidyl-prolyl cis-trans isomerase FKBP1A | 1 | FK506-binding protein 1A inhibitor | Phase 2 | https://conductscience.org/library/genes/2280 | Drugs and targets |
| PDE4A | Phosphodiesterase 4 | 1 | Phosphodiesterase 4 inhibitor | Phase 2 | https://conductscience.org/library/genes/5141 | Drugs and targets |
| PDE4B | Phosphodiesterase 4 | 1 | Phosphodiesterase 4 inhibitor | Phase 2 | https://conductscience.org/library/genes/5142 | Drugs and targets |
| PDE4C | Phosphodiesterase 4 | 1 | Phosphodiesterase 4 inhibitor | Phase 2 | https://conductscience.org/library/genes/5143 | Drugs and targets |
| PDE4D | Phosphodiesterase 4 | 1 | Phosphodiesterase 4 inhibitor | Phase 2 | https://conductscience.org/library/genes/5144 | Drugs and targets |
Trials10
Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs. 2 more are not shown, since the checks do not confirm them.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT03154333 | Safety and Efficacy of Diacerein 1% Ointment for Subjects With Epidermolysis Bullosa Simplex (EBS) | Phase 2 | 54 | 2019-11-05 | Epidermolysis Bullosa Simplex | DIACEREIN | Its record names the disease | ClinicalTrials.gov |
| NCT03389308 | Long Term Open-label Study Evaluating Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex | Phase 2 | 51 | 2020-09-16 | Epidermolysis Bullosa, Epidermolysis Bullosa Simplex | DIACEREIN | Its record names the disease | ClinicalTrials.gov |
| NCT03472287 | To Evaluate the Pharmacokinetic of Diacerein and Rhein After Maximum Use in Patients With Epidermolysis Bullosa (EB) | Phase 1 | 11 | 2020-02-10 | Epidermolysis Bullosa (EB), Epidermolysis Bullosa Simplex, Dystrophic Epidermolysis Bullosa, Junctional Epidermolysis Bullosa | DIACEREIN | Its record names the disease | ClinicalTrials.gov |
| NCT02960997 | Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study | Phase 2 | 8 | 2020-01-30 | Epidermolysis Bullosa Simplex, Epidermolysis Bullosa Simplex Kobner, Weber-Cockayne Syndrome | SIROLIMUS | Its record names the disease | ClinicalTrials.gov |
| NCT06073132 | An International, Multicenter, Randomized, Double-Blind, Parallel Group, Vehicle-Controlled, Phase 2/3 Study With Open-Label Extension Evaluating the Efficacy and Safety of Diacerein 1% Ointment for the Treatment of Generalized Epidermolysis Bullosa Simplex (EBS) | Phase 2/3 | 80 | Generalized Epidermolysis Bullosa Simplex | DIACEREIN | Its record names the disease | ClinicalTrials.gov | |
| NCT07482787 | Efficacy and Safety Study to Evaluate SD-101 in Epidermolysis Bullosa | Phase 3 | 80 | Epidermolysis Bullosa (EB) | ALLANTOIN, NELITOLIMOD | Its record names the disease | ClinicalTrials.gov | |
| NCT06509984 | A 20-Week Study Assessing the Efficacy of Apremilast in Patients with EB Simplex Generalized | Phase 2 | 20 | Epidermolysis Bullosa Simplex, Genodermatosis | APREMILAST | Its record names the disease | ClinicalTrials.gov | |
| NCT03468322 | A Double-blind, Intra-individual Comparison, POC Trial of AC-203 in EB Patients | Phase 2 | 9 | Inherited Epidermolysis Bullosa | DIACEREIN | Its record names the disease | ClinicalTrials.gov | |
| NCT01340235 | Treatment of Dowling Maera Type of Epidermolysis Bullosa Simplex by Oral Erythromycin | Phase 3 | 8 | Epidermolysis Bullosa | ERYTHROMYCIN | Its record names the disease | ClinicalTrials.gov | |
| NCT03016715 | Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study | Phase 2 | 8 | Epidermolysis Bullosa Simplex, Epidermolysis Bullosa Simplex Kobner, Weber-Cockayne Syndrome | SIROLIMUS | Its record names the disease | ClinicalTrials.gov |
Comparisons in its trials8
Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.
| Comparison | Group 1, with the outcome | Group 2, with the outcome | Participants | Trial | Source |
|---|---|---|---|---|---|
| Diacerein 1% Ointment vs Vehicle Ointment: The Proportion of Subjects Who Achieved Success on the Investigator's Global Assessment (IGA) | 12 of 28 | 7 of 26 | 54 | NCT03154333 | Trial comparisons |
| Diacerein 1% Ointment vs Vehicle Ointment: deaths from any cause | 0 of 28 | 0 of 26 | 54 | NCT03154333 | Trial comparisons |
| Diacerein 1% Ointment vs Vehicle Ointment: Proportion of Subjects Who Achieved ≥ 60% Reduction in Body Surface Area (BSA) of EBS Lesions Within Assessment Area | 16 of 28 | 14 of 26 | 54 | NCT03154333 | Trial comparisons |
| Diacerein 1% Ointment vs Vehicle Ointment: serious adverse events | 0 of 28 | 2 of 26 | 54 | NCT03154333 | Trial comparisons |
| Sirolimus vs Placebo: deaths from any cause | 0 of 8 | 0 of 8 | 16 | NCT02960997 | Trial comparisons |
| Sirolimus vs Placebo: serious adverse events | 0 of 8 | 0 of 8 | 16 | NCT02960997 | Trial comparisons |
| Cohort 1 (Adolescents, Adults) vs Cohort 2 (Children): deaths from any cause | 0 of 8 | 0 of 3 | 11 | NCT03472287 | Trial comparisons |
| Cohort 1 (Adolescents, Adults) vs Cohort 2 (Children): serious adverse events | 0 of 8 | 0 of 3 | 11 | NCT03472287 | Trial comparisons |
Papers from its trials1
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
| PMID | Listed as | Trials listing it |
|---|---|---|
| 41224305 | DERIVED | 1 |
Source PubMed
Data dates
The date of each source this release read.
Sources 6
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ChEMBL, through Open TargetsCC BY-SA 3.0
- ClinicalTrials.govNo license stated
- NLM's MeSHNLM's terms
- Open TargetsCC0 1.0
- PubMedNLM's terms
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- ClinicalTrials.govnone stated
- NLM's MeSHNLM's
- Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- PubMedNLM's terms
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this disease from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/diseases/v1/MONDO_0017610
GET https://conductscience.org/api/diseases/v1/MONDO_0017610/drugs