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Rigosertib for RDEB-SCC
EU CTIS ID: 2024-518846-25-00
What this study is testing
To evaluate the safety and tolerability of Rigosertib administered either orally daily for three weeks on, one week off or as 72h CIV infusions on day 1-3 of a two week-cycle for 8 cycles and then on day 1-3 of a 4 week cycle thereafter. To estimate the anti-tumor activity of Rigosertib in RDEB patients with advanced SCC that has failed prior standard of care by determining the overall response rate (ORR) which is defined as the proportion of patients who achieve either a CR or a PR.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Diagnosis of RDEB confirmed by genetic testing or by a skin biopsy with immunofluorescence mapping (IFM).
- 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).
You likely can't join if
- Response to standard of care;
- 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 non-treatment follow-up period;
- Female subjects: pregnant or lactating women and all women Rigosertib for RDEB-SCC Version 5.0; 15.09.2023 Confidential 9 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 (either oral or intravaginal or transdermal); progestogen-only hormonal contraception associated with inhibition of ovulation (either 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 sexual 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 repeated 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.
See the full eligibility criteria
Who can join
- Diagnosis of RDEB confirmed by genetic testing or by a skin biopsy with immunofluorescence mapping (IFM).
- 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 consent document indicating that the patient understands the purpose of and procedures required for the study and is willing to participate in the study
What rules you out
- Response to standard of care;
- 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 non-treatment follow-up period;
- Female subjects: pregnant or lactating women and all women Rigosertib for RDEB-SCC Version 5.0; 15.09.2023 Confidential 9 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 (either oral or intravaginal or transdermal); progestogen-only hormonal contraception associated with inhibition of ovulation (either 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 sexual 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 repeated 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 investigator´s criteria.
- 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) <60 mL/min;
- 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);
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.