Epidermolysis Bullosa DystrophicaPhase 1/2NCT03786237
Rigosertib for RDEB-SCC
A Phase 1/2 study testing Rigosertib Oral Capsules / Rigosertib Intravenous in people with Epidermolysis Bullosa Dystrophica and Squamous Cell Carcinoma, run by Prof. Johann Bauer at 1 site in Austria.
Trial summary
Epidermolysis bullosa (EB) is a heritable skin disease characterized by marked fragility of epithelialized tissue with blistering in skin and mucous membranes following the slightest mechanical trauma. Read more about this study
- Who is it for?
- Ages 18 to 79 years
- Conditions the study is for
- Epidermolysis Bullosa Dystrophica, Squamous Cell Carcinoma
- What is being tested?
- Rigosertib Oral Capsules / Rigosertib Intravenous
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteAustria
- Study dates
- 12 Apr 2021to 1 Dec 2025
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 79 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 18-79 years of age;
- Diagnosis of unresectable, locally advanced or metastatic SCC confirmed prior to the Screening Visit.
- Failure to respond to RDEB SCC standard of care, such as surgical excision, radiotherapy or conventional cytotoxic chemotherapy with e.g. platin derivates (i.e.
- cisplatin or carboplatin), 5-fluorouracil, bleomycin, methotrexate, adriamycin, taxanes, gemcitabine or ifosfamide alone or in combination or failure to respond to previous alternative biologic treatments such as epidermal growth factor inhibitors (like cetuximab and panitumumab) or immune checkpoint (programmed cell death
- 1) inhibitors (such as nivolumab, pembrolizumab, cemiplimab). For recent guidelines on standard of care for RDEB SCC and non EB-SCC please see Mellerio et al., 2016; Stratigos et al., 2015 and Kim et al., 2018.
- Is not currently receiving any other cancer therapy.
- Measurable disease based on Response Criteria in Solid Tumors Version 1.1 (RECIST 1.1)
- Patient (or patient's legally authorized representative) must have signed an informed
Who cannot join
- Response to standard of care.
- Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure or unstable angina pectoris.
- Active systemic infection not adequately responding to appropriate therapy.
- Total bilirubin ≥ 1.5 mg/dL (≥5.3 mg/dL in patients if related to hemolysis or Gilbert's disease).
- Alanine transaminase (ALT)/aspartate transaminase (AST) ≥ 2.5 x upper limit of normal (ULN).
- Serum creatinine ≥2 .0 mg/dL or eGFR (estimated Glomerular Filtration Rate) <60mL/min.
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- White blood cell count ≤ 2000/μl, neutrophils ≤ 1500/μL, platelets ≤ 100 x103/μL, hemoglobin ≤ 7.9 g/dL.
- Known active HIV, hepatitis B or hepatitis C, where active is defined as follows: a. HIV or Hepatitis C - presence of viral load; b. Hepatitis B - antigen positive
- Uncorrected hyponatremia (defined as serum sodium value of <125 mmol/L).
- Male patients with partners of child-bearing potential who are unwilling to use male contraception (condom) throughout the study, up to and including the 30-day nontreatment follow-up period.
- Female subjects: pregnant or lactating women and all women physiologically capable of becoming pregnant (i.e. women of childbearing potential) UNLESS they are willing to use one or more highly effective and reliable methods of contraception with a Pearl index ≤1 including combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral or intravaginal or transdermal); progestogen-only hormonal contraception associated with inhibition of ovulation (oral or injectable or implantable); an intrauterine device (IUD); an intrauterine hormone-releasing system ( IUS); bilateral tubal occlusion; vasectomised partner (provided that partner is the sole sexual partner of the WOCBP trial participant and that the vasectomised partner has received medical assessment of the surgical success) or sexual abstinence (The reliability of sexuality abstinence needs to be evaluated in relation to the duration of the clinical trial and the preferred and usual lifestyle of the subject). Reliable contraception should be maintained throughout the study. A pregnancy test in serum will be performed at screening in all women of childbearing potential, and in urine at all visits. Any postmenopausal women (physiologic menopause defined as "12 consecutive months of amenorrhea") or women permanently sterilized (e.g. tubal occlusion, hysterectomy or bilateral salpingectomy) will not be required to undergo pregnancy test.
- Uncontrolled hypertension. (i.e.. systolic blood pressure greater than or equal to 140mmHg and diastolic blood pressure greater than or equal to 90mmHg despite intake of ≥ 3 antihypertensive medications with complementary mechanisms of action (a diuretic should be 1 component); (Whelton et al., 2018).
- Patient is currently participating and receiving study therapy or systemic therapy or has participated in a study of an investigational agent and received study therapy or used an investigational device within 4 weeks of the first dose of treatment.
- Psychiatric illness or social situation that would limit the patient's ability to tolerate and/or comply with study requirements.
- Patients (or parents in case of paediatric subject) unlikely to comply with the study protocol or unable to understand the nature and scope of the study or the possible benefits or unwanted effects of the study procedures and treatments.
- History or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the study, interfere with the patient's participation for the full duration of the study, or is not in the best interest of the patient to participate, in the opinion of the treating Investigator.
- Known hypersensitivity reaction to any of the components of study treatment.
- Presence of clinically significant ECG abnormalities based on the inverstigator´s criteria.
Trial locations and contacts
1 site in 1 country.
Austria1 site
EB House Austria/Dept. of Dermatology University Hospital
Salzburg, State of Salzburg, 5020
About this study
Official title: A Phase II, Open Study to Assess Efficacy and Safety of Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated Locally Advanced/Metastatic Squamous Cell Carcinoma
Epidermolysis bullosa (EB) is a heritable skin disease characterized by marked fragility of epithelialized tissue with blistering in skin and mucous membranes following the slightest mechanical trauma. Eighty percent of all patients suffering from recessive dystrophic EB (RDEB), a subtype originating from mutations in the COL7A1 gene, develop squamous cell carcinoma (SCC). In RDEB patients SCC presents early (most patients are in their 20s or 30s) and shows a highly aggressive metastatic course which often leads to premature death at this young age.
In light of scarce data on the efficacy and safety of systemic treatment regimens for advanced SCC, the investigators propose to perform a small, "first in EB " trial of an experimental drug called rigosertib for the treatment of EB cancer. The trial will be conducted in two study centres, in London and Salzburg, and will last approximately 2.5 years with each patient recruited being in the study for 1 year. The drug is a polo-like kinase inhibitor interfering with different molecular pathways that are essential for cancer cell growth. Rigosertib was developed by Onconova Therapeutics and is currently tested in several clinical trials for a number of other cancers including myelodysplastic syndrome (a cancer of the blood). The investigators have identified that rigosertib most selectively kills EB cancer cells in vitro while leaving normal EB skin cells unaffected. This project will evaluate whether rigosertib is capable of inducing an anti-cancer response in EB patients and whether the drug is well-tolerated. Mechanisms of molecular targeting of squamous cancer cells by rigosertib will further be investigated in EB patients, also aiming at the identification of biomarkers that may allow the predictive identification of best responders.
- Being tested
- Rigosertib Oral Capsules / Rigosertib Intravenous
- Medical subject headings
- Epidermolysis Bullosa Dystrophica, Carcinoma, Squamous Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Rigosertib Oral Capsules / Rigosertib IntravenousDrug
Patients will take Rigosertib either as oral capsules or will get intravenous infusions depending on the needs of the patients.
Oral Capsules:
Patients will take oral Rigosertib continuously for a total of three weeks, every four week cycle (three weeks on, one week off drug) for up to 13 cycles. Patients will take 560 mg of oral Rigosertib (ie, 2 capsules of 280 mg) in the morning and in the afternoon, total of 1120mg/day.
Intravenous Infusions:
For IV treatment Rigosertib 1800 mg/24 hr is diluted in 0.9% sodium chloride for injection just prior to dosing and is administered as a 72-hr CIV infusion on days 1, 2, and 3 of a 2-week cycle for the first eight 2-week cycles, then on days 1, 2, and 3 of a 4-week cycle thereafter.
| Study group | Role | What they receive |
|---|---|---|
| Treatment Oral Capsules / Intravenous | Experimental | Not described Receives: Drug: Rigosertib Oral Capsules / Rigosertib Intravenous |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy: Objective Response Rate (ORR)
The primary (efficacy) objective of this trial is to determine the Objective Response Rate (ORR) of therapy with Rigosertib in RDEB patients with locally advanced/metastatic squamous cell carcinoma of the skin using Response Criteria in Solid Tumors Version 1.1 (RECIST1.1) per site assessment up to 52 weeks by CT/MR Scan.
Measured: 1 year
Number of Treatment-related adverse events
Safety will be evaluated for all treated patients, who receive at least one dose of Rigosertib, using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), Version 4.0 (http://ctep.cancer.gov). Safety assessments will be based on number of treatment related adverse events per grade.
Measured: 1 year
Secondary outcomes (2)
Evaluation of quality of life using the "Quality of life in Epidermolysis bullosa questionnaire (QOLEB)"
Assess impact on quality of life using an Epidermolysis bullosa (EB) specific questionnaire developed and published by Murrell et al.2009. Total score is reported (range 0-75; 0 means EB has no affect on ones life, the higher the score the more affect and annoyance/handicap)
Measured: 1 year
Rate of presence or absence of cancer specific biomarkers
Fixed tissue will be assessed using immuno-histochemistry with antibodies raised against phosphorylated AKT (p473 Akt), phosphorylated C-RAF (p-S338 RAF), phosphorylated ERK and cleaved caspase.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Dec 2018
- Study start12 Apr 2021
- Primary completion1 Dec 2025
- Study completion1 Dec 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Epidermolysis Bullosa DystrophicaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Prof. Johann Bauer
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Prof. Johann Bauer, Salzburger Landeskliniken
- Investigators
- Johann W Bauer, Prof., MD, Principal investigator, Department of Dermatology, Paracelsus Medical University, Salzburger Landeskliniken
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03786237
- Sponsor's protocol ID
- Rigosertib 5.0
- Status last verified
- Feb 2026
- First submitted
- 14 Nov 2018
- Last updated
- 24 Feb 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 24 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Open Study to Assess Efficacy and Safety of Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated Locally Advanced/Metastatic Squamous Cell Carcinoma
- ClinicalTrials.gov ID
- NCT03786237
- Acronym / sponsor ID
- Rigosertib 5.0
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Prof. Johann BauerUniversity, hospital or other
- Study start
- 12 Apr 2021 (actual)
- Primary completion
- 1 Dec 2025 (actual)
- Study completion
- 1 Dec 2025 (actual)
- First posted
- 24 Dec 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Epidermolysis bullosa (EB) is a heritable skin disease characterized by marked fragility of epithelialized tissue with blistering in skin and mucous membranes following the slightest mechanical trauma. Eighty percent of all patients suffering from recessive dystrophic EB (RDEB), a subtype originating from mutations in the COL7A1 gene, develop squamous cell carcinoma (SCC). In RDEB patients SCC presents early (most patients are in their 20s or 30s) and shows a highly aggressive metastatic course which often leads to premature death at this young age.
In light of scarce data on the efficacy and safety of systemic treatment regimens for advanced SCC, the investigators propose to perform a small, "first in EB " trial of an experimental drug called rigosertib for the treatment of EB cancer. The trial will be conducted in two study centres, in London and Salzburg, and will last approximately 2.5 years with each patient recruited being in the study for 1 year. The drug is a polo-like kinase inhibitor interfering with different molecular pathways that are essential for cancer cell growth. Rigosertib was developed by Onconova Therapeutics and is currently tested in several clinical trials for a number of other cancers including myelodysplastic syndrome (a cancer of the blood). The investigators have identified that rigosertib most selectively kills EB cancer cells in vitro while leaving normal EB skin cells unaffected. This project will evaluate whether rigosertib is capable of inducing an anti-cancer response in EB patients and whether the drug is well-tolerated. Mechanisms of molecular targeting of squamous cancer cells by rigosertib will further be investigated in EB patients, also aiming at the identification of biomarkers that may allow the predictive identification of best responders.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment Oral Capsules / Intravenous | Experimental | Drug: Rigosertib Oral Capsules / Rigosertib Intravenous |
Who may qualify
- Ages
- 18 to 79 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 18-79 years of age;
- Diagnosis of unresectable, locally advanced or metastatic SCC confirmed prior to the Screening Visit.
- Failure to respond to RDEB SCC standard of care, such as surgical excision, radiotherapy or conventional cytotoxic chemotherapy with e.g. platin derivates (i.e.
- cisplatin or carboplatin), 5-fluorouracil, bleomycin, methotrexate, adriamycin, taxanes, gemcitabine or ifosfamide alone or in combination or failure to respond to previous alternative biologic treatments such as epidermal growth factor inhibitors (like cetuximab and panitumumab) or immune checkpoint (programmed cell death
- 1) inhibitors (such as nivolumab, pembrolizumab, cemiplimab). For recent guidelines on standard of care for RDEB SCC and non EB-SCC please see Mellerio et al., 2016; Stratigos et al., 2015 and Kim et al., 2018.
Key exclusion criteria (18 listed)
- Response to standard of care.
- Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure or unstable angina pectoris.
- Active systemic infection not adequately responding to appropriate therapy.
- Total bilirubin ≥ 1.5 mg/dL (≥5.3 mg/dL in patients if related to hemolysis or Gilbert's disease).
- Alanine transaminase (ALT)/aspartate transaminase (AST) ≥ 2.5 x upper limit of normal (ULN).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Rigosertib Oral Capsules / Rigosertib IntravenousPatients will take Rigosertib either as oral capsules or will get intravenous infusions depending on the needs of the patients. Oral Capsules: Patients will take oral Rigosertib continuously for a total of three weeks, every four week cycle (three weeks on, one week off drug) for up to 13 cycles. Patients will take 560 mg of oral Rigosertib (ie, 2 capsules of 280 mg) in the morning and in the afternoon, total of 1120mg/day. Intravenous Infusions: For IV treatment Rigosertib 1800 mg/24 hr is diluted in 0.9% sodium chloride for injection just prior to dosing and is administered as a 72-hr CIV infusion on days 1, 2, and 3 of a 2-week cycle for the first eight 2-week cycles, then on days 1, 2, and 3 of a 4-week cycle thereafter. | Drug | Treatment Oral Capsules / Intravenous |
Primary outcome
Efficacy: Objective Response Rate (ORR)
Time frame: 1 year
The primary (efficacy) objective of this trial is to determine the Objective Response Rate (ORR) of therapy with Rigosertib in RDEB patients with locally advanced/metastatic squamous cell carcinoma of the skin using Response Criteria in Solid Tumors Version 1.1 (RECIST1.1) per site assessment up to 52 weeks by CT/MR Scan.
Number of Treatment-related adverse events
Time frame: 1 year
Safety will be evaluated for all treated patients, who receive at least one dose of Rigosertib, using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), Version 4.0 (http://ctep.cancer.gov). Safety assessments will be based on number of treatment related adverse events per grade.
2 secondary outcomes
Evaluation of quality of life using the "Quality of life in Epidermolysis bullosa questionnaire (QOLEB)"
Time frame: 1 year
Rate of presence or absence of cancer specific biomarkers
Time frame: 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Feb 2026.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Johann W Bauer, Prof., MD principal investigator
Department of Dermatology, Paracelsus Medical University, Salzburger Landeskliniken
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Rigosertib Oral Capsules / Rigosertib Intravenous in people with Epidermolysis Bullosa Dystrophica and Squamous Cell Carcinoma, run by Prof. Johann Bauer at 1 site in Austria.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Austria. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Efficacy: Objective Response Rate (ORR) (time frame: 1 year). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Feb 2026.