StudyFinder
Search Results Within Category "Dermatology"
5 Study Matches
Mirdametinib in Histiocytic Disorders
Monica Trapp - Monica.Trapp@cchmc.org
ALL
2 years and over
PHASE2
NCT06153173
Inclusion Criteria:
• Subjects must be ≥ 2 years of age AND have a diagnosis of a histiocytic disorder that requires systemic therapy * If patient has had a diagnostic biopsy, biopsy must be reviewed and confirmed by CCHMC pathologist as feasible * If patient has had a biopsy but has not had molecular testing done, must have tissue available for mutational analysis * If patient has isolated pituitary/CNS disease or situations where biopsy is not feasible, positive ddPCR blood test for mutation associated with histiocytic neoplasm with clinical features of histiocytosis is sufficient
• Must have measurable disease on PET scan or brain MRI
• Subjects must demonstrate adequate organ function as defined: * Renal: maximum serum creatinine 2x the upper limit of normal (ULN) OR a creatinine clearance or radioisotope GFR ≥ 70ml/min/1.73 m2 * Liver: ALT ≤ 3x ULN AND normal INR (≤ 1.5) * Hematologic: Hematology: Albumin ≥ 2.8 g/dL; Absolute neutrophil count ≥ 1.5 x 109/L; Platelets ≥ 100 x 109/L; Hemoglobin ≥ 9.0 g/dL * Patients with organ function abnormalities outside of these thresholds deemed to be the result of histiocytic disease will be considered eligible
Exclusion Criteria:
• Prior therapy with stipulations as described: * Myelosuppressive Chemotherapy: Must not have received any cytotoxic chemotherapy which impacts the growth and development of cells in the bone marrow within 14 days of enrollment onto this study (i.e. cytarabine, cladribine, clofarabine, mercaptopurine, methotrexate, vinblastine) * MEK Inhibitors: Must not have received a MEK inhibitor within 30 days (or 5 half-lives, whichever is longer) of enrollment, NOR have had disease progression on MEK inhibitor * Steroids: Due to the increased risk of an ocular event, the use of systemic oral, inhaled, or ocular glucocorticoid therapy is prohibited within 14 days prior to first dose of mirdametinib. Throughout the treatment period, short term glucocorticoid treatment (30 days or less) is permitted. Any patients requiring long-term steroid use (more than 30 consecutive days) are not eligible. The exception to this rule is subjects with endocrine deficiencies who require physiologic steroids * Radiation: Must not have received radiation within 14 days of study enrollment or have received radiation to the orbit at any time
• Risk factors for retinal vein occlusion (RVO) are listed. Exclusion should be considered by clinical discretion if they have any of the following risk factors for RVO at screening: * Intraocular pressure (IOP) \> 21 mmHg; if IOP is unable to be obtained (eg age, cooperation, tolerability), ophthalmologist's exam findings and overall assessment will be utilized. If in the ophthalmologist's assessment there are no signs of raised IOP, the subject will be considered eligible for this parameter * Glaucoma or any significant abnormality (≥ grade 2) on ophthalmologic exam that is uncontrolled with intervention * Serum cholesterol \> 300 mg/dL * Serum triglycerides \> 300 mg/dL * Hyperglycemia (either fasting blood glucose \> 125 mg/dL OR random blood glucose \> 200 mg/dL) * Uncontrolled hypertension (participants ≤ 12 years of age with a blood pressure ≥ 95th percentile for age + 12 mmHg; participants ≥ 13 years of age with a blood pressure ≥ 140/90 mm Hg) unresolved on repeat measurement
• LVEF \< 55% at screening OR history of clinically significant cardiac disease, unless deemed to be the direct result of disease
• Subjects who are pregnant or breastfeeding, or are at risk of pregnancy or fathering a baby and are unable to use acceptable methods of birth control during the length of the study
DRUG: Mirdametinib
Langerhans Cell Histiocytosis (LCH), Juvenile Xanthogranuloma (JXG), Rosai-Dorfman Disease (RDD), Histiocytic Disorders
Targeted Investigation of Microbiome 2 Treat Atopic Dermatitis (TIME-2)
Elsie Parmar - elsie.parmar@cchmc.org
ALL
6 years and over
PHASE1
NCT06504160
Inclusion Criteria:
Each individual must meet all of the following criteria at Screening to be eligible for enrollment as a study participant:
• Participant and/or parent/legal guardian must be able to understand and provide informed consent and assent (if applicable).
• Male or female participant 6 years of age or older.
• Meet ADRN Standard Diagnostic Criteria for active AD. Each individual must meet all of the following criteria at Baseline to be eligible for enrollment as a study participant:
• Have at least 7 cm2 of lesional skin within the upper extremities, lower extremities, and/or trunk. Lesions on the face, neck, hands, feet, and intertriginous areas do not count toward the required area, as samples may not be taken from these areas. The required area may be one contiguous area or may be comprised of multiple areas with a compliant total area.
• Have at least 3% body surface area of AD involvement as indicated by derived total area of involvement score during SCORAD assessment.
• Have an IGA score of two or greater.
• Each potential participant who can become pregnant must meet either of the following criteria prior to randomization to be eligible for enrollment as a study participant.
• Willing to remain abstinent from intercourse that may result in a pregnancy.
• Willing to use an FDA-approved method of contraception for the duration of study participation. Acceptable methods include the following: * Permanent sterilization of partner * Long-acting reversible contraceptives (e.g., intrauterine devices or systems, implantable rods, contraceptive injections) when used as directed for at least 7 days prior to Baseline. * Short-acting hormonal contraceptives (e.g., oral contraceptive pills, patch, vaginal ring) when used as directed for at least 30 days prior to Baseline * Barrier methods (e.g., condoms; diaphragm, sponge, or cervical cap with spermicide)
• Have obtained negative pregnancy test results during both the Screening and Baseline Visits.
Exclusion Criteria:
Individuals who meet any of the following criteria at Screening or Baseline are not eligible for enrollment as study participants:
• Inability or unwillingness to give written informed consent or comply with study protocol.
• Has self-reported as pregnant or lactating during the Screening or Baseline Visit, or is pregnant as indicated by a positive pregnancy test result obtained at the Screening or Baseline Visit.
• Sensitivity to or difficulty tolerating Dove® fragrance-free bar soap, Cetaphil® lotion, alcohol-based cleaners, clobetasol and fluocinonide ointments, triamcinolone ointment, hydrocortisone ointment, glycerol, hydroxyethylcellulose or soy products.
• Known recalcitrance to topical steroids, including class 1 steroids, within 6 months of the Screening Visit.
• History of serious life-threatening reaction to tape or adhesives.
• Known allergy to all antibiotics to which S. hominis A9 is sensitive. These include ampicillin-sulbactam, cefazolin, cefoxitin, clindamycin, daptomycin, doxycycline, levofloxacin, linezolid, minocycline, moxifloxacin, mupirocin, nitrofurantoin, oxacillin, rifampin, trimethoprim-sulfamethoxazole, and vancomycin.
• Has a major defect in the epidermal barrier such as open wounds or genodermatoses (e.g., Netherton's syndrome).
• Is immunocompromised (e.g., Human Immunodeficiency Virus (HIV)/Acquired Immunodeficiency Syndrome (AIDS), Wiskott-Aldrich Syndrome) or has an immune system disorder (e.g., autoimmune disease).
• Has current malignant disease (except non-melanoma skin cancer in an area not affected by treatment).
• Has a history of psychiatric disease or history of alcohol or drug abuse that, in the opinion of the study investigator, would interfere with the ability to comply with the study protocol.
• Ongoing participation in another investigational trial or use of investigational drugs within 8 weeks, or five half-lives (if known), whichever is longer, of the Screening Visit.
• Treatment with non-steroid systemic immunosuppressant within 6 months of the Screening Visit.
• Treatment with any biologic, including dupilumab, within 16 weeks of the Screening Visit.
• Treatment with oral or injectable therapy for AD (excluding oral steroids) within five half-lives (if known) or 16 weeks before the Screening Visit, whichever is longer.
• Treatment with oral retinoids (e.g., isotretinoin) within 60 days of the Screening Visit.
• Treatment with allergen immunotherapy within 30 days of Screening Visit.
• Has close contacts (e.g., spouse, children, or members in the same household) who have severe barrier defects or are immunocompromised.
• May, in the opinion of the investigator, have difficulty tolerating the medication washout requirements for topical AD treatments, prescription moisturizers, antibiotics, oral steroid therapies, and phototherapy ahead of Baseline.
• Past or current medical conditions or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the investigator, may pose additional risks from participation in the study, may interfere with the participant's ability to comply with study requirements or that may impact the quality or interpretation of the data obtained from the study. Individuals who meet any of the following criteria at Baseline are not eligible for enrollment as study participants:
• Have more than 30% body surface area of AD involvement, as indicated by the derived total area of involvement score during SCORAD assessment.
• Active bacterial, viral, or fungal skin infections, except for onychomycosis and tinea pedis.
• Any noticeable breaks or cracks in the skin on the target areas of investigational product application, including severely excoriated skin or skin with open or weeping wounds suggestive of an active infection. a. At the investigator's discretion, participants with minor breaks, cracks, or excoriations on body areas not eligible for sampling during the trial may be enrolled, provided there is no evidence of infection and investigational product is not applied to these areas until healed.
• Use of topical AD treatments - including steroids and calcineurin inhibitors - on the upper extremities (exclusive of the hands), lower extremities (exclusive of the feet), or trunk within seven days of the Baseline Visit.
• Treatment with prescription moisturizers classified as medical device (e.g., Atopiclair®, MimyX®, Epiceram®, etc.) on the upper extremities (exclusive of the hands), lower extremities (exclusive of the feet), or trunk within seven days of the Baseline Visit.
• Use of any systemic microbialª within fourteen days of the Baseline Visit, or use of any topical antimicrobial on the upper extremities (exclusive of the hands), lower extremities (exclusive of the feet), or trunk within fourteen days of the Baseline Visit. a. Antimicrobials include antibiotics, antifungals, antiparasitics, and antivirals. Certain systemic antivirals that do not exhibit systemic anti-inflammatory effects may be permitted with prior approval from a protocol co-chair).
• Within the upper extremities (exclusive of the hands), lower extremities (exclusive of the feet), and trunk, use of topical products - prescription or over-the-counter, all formulations - not specified per protocol, within seven days of the Baseline Visit.
• Use of systemic corticosteroid therapies for any indication within 28 days of the Baseline Visit.
• Use of systemic corticosteroid therapies for treatment of an asthma exacerbation within 3 months of the Baseline Visit.
• Require a dose greater than 880 mcg/day of fluticasone propionate or equivalent inhaled corticosteroid to maintain asthma control, at the time of the Baseline Visit.
• Any phototherapy for skin disease (such as narrow band ultraviolet B \[NBUVB\], ultraviolet B \[UVB\], ultraviolet A1 \[UVA1\], psoralen + UVA \[PUVA\]) or regular use (more than 2 visits per week) of a tanning bed within 28 days of the Baseline Visit.
DRUG: ShA9 Topical Gel, DRUG: Hydrocortisone Ointment, DRUG: Clobetasol Ointment, DRUG: Fluocinonide Ointment, DRUG: Placebo (Vehicle) Topical Gel, DRUG: Triamcinolone Ointment
Atopic Dermatitis
atopic dermatitis, eczema, ADRN, TIME-2, microbiome, Staphylococcus hominis
NRSTS2021, A Risk Adapted Study Evaluating Maintenance Pazopanib, Limited Margin, Dose-Escalated Radiation Therapy and Selinexor in Non-Rhabdomyosarcoma Soft Tissue Sarcoma (NRSTS)
cancer@cchmc.org
ALL
Up to 30 years old
PHASE1
NCT06239272
Inclusion Criteria:
Inclusion Criteria - All Patients
* Patients must be 1-30 years at the time of the biopsy that established the diagnosis of NRSTS.
* Surgical Resection: Patients who had an upfront resection prior to enrollment will be eligible if they are able to begin therapy within 28 days of resection assuming other eligibility criteria are met. Delayed resection is preferred for all patients with intermediate and high-risk disease.
* Lansky performance status score ≥ 60 for patients ≤ 16 years of age. Karnofsky performance status score ≥ 60 for patients \>16 years of age. Note patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
Diagnosis
• Patients with CIC-DUX 4 rearranged sarcomas will be enrolled on the high-risk stratum only, regardless of presence of metastasis, size, or resection status.
Patient has low-risk disease if the patient has a:
* Low-grade tumor of any size where R0 or R1 surgical margins are anticipated or achieved.
* High-grade tumors that are \< 5 cm where R0 or R1 resection margins are anticipated or achieved.
Patient must have adequate organ function in the organs that will be within the radiotherapy field.
Adequate renal function defined as:
* Creatinine clearance or radioisotope GFR \> 70 mL/min/1.73 m2, or
* A normal serum creatinine based on age/gender as follows
Age Maximum Serum Creatinine (mg/dL) Male Female 2 to \< 6 years 0.8 0.8 6 to \< 10 years 1 1 10 to \< 13 years 1.2 1.2 13 to \< 16 years 1.5 1.4 \> 16 years 1.5 1.4
Adequate liver function defined as:
* Total bilirubin \< 1.5 x upper limit of normal (ULN) for age
* SGOT (AST) or SGPT (ALT) \< 2.5 x ULN for age
Adequate cardiac function defined as:
* Ejection fraction of \> 55% by echocardiogram or cardiac MRI
* QTc \< 480 msec
Adequate pulmonary function defined as:
* No evidence of dyspnea at rest, no exercise intolerance, and a resting pulse oximetry reading \> 94% on room air if there is clinical indication for determination.
Inclusion Criteria - Intermediate and High Risk Participants
Patient has intermediate-risk if the patient has a:
* Low-grade non-metastatic initially unresectable disease at study enrollment where delayed resection is planned.
* High-grade \< 5 cm non-metastatic initially unresectable disease at study enrollment where delayed resection is planned. Of note, patients enrolled on the low-risk arm who were unable to achieve gross total resection where delayed re-resection is planned are eligible for this arm.
* High-grade tumor \> 5 cm that is potentially resectable.
Patient high-risk if the patient has:
* Metastatic disease at presentation
* Unresectable disease at study enrollment where delayed resection is not anticipated. Of note, patients enrolled on the low-risk arm who were unable to achieve gross total resection where delayed re-resection is not-planned are eligible for this arm.
* CIC-DUX4 rearranged sarcoma
Organ Function
Adequate bone marrow function defined as:
* Absolute neutrophil count \> 1000/µL
* Platelet count \> 100,000/µL
* Hemoglobin \> 8 g/dL for patients \< 16 years of age
* Hemoglobin \> 9 g/dL for patients \> 16 years of age
Note: No transfusions are permitted 7 days prior to laboratory studies to determine eligibility.
Adequate renal function defined as:
* Creatinine clearance or radioisotope GFR \> 70 mL/min/1.73 m2, or
* A normal serum creatinine based on age/gender as follows
Age Maximum Serum Creatinine (mg/dL) Male Female 2 to \< 6 years 0.8 0.8 6 to \< 10 years 1 1 10 to \< 13 years 1.2 1.2 13 to \< 16 years 1.5 1.4 \> 16 years 1.5 1.4
Adequate liver function defined as:
* Total bilirubin \< 1.5 x upper limit of normal (ULN) for age
* SGOT (AST) or SGPT (ALT) \< 2.5 x ULN for age
Adequate cardiac function defined as:
* Ejection fraction of \> 55% by echocardiogram or cardiac MRI
* QTc \< 480 msec
Adequate pulmonary function defined as:
* No evidence of dyspnea at rest, no exercise intolerance, and a resting pulse oximetry reading \> 94% on room air if there is clinical indication for determination.
Anticoagulation
Patients on low molecular weight heparin, warfarin (with a stable INR), or direct oral anticoagulants (DOAC) who have been on a stable dose of are eligible. Patients being treated for a pulmonary embolism or deep venous thrombosis (DVT) must have been treated for a minimum of 6 weeks prior to starting therapy treatment.
Life Expectancy
Patient must have a life expectancy of at least 3 months with appropriate therapy.
Exclusion Criteria:
* Patients with known primary CNS sarcoma or CNS metastases are not eligible. Note: Brain imaging is not an eligibility requirement. Tumors with intracranial extension will be allowed.
* Patients with the following histologic diagnosis are not eligible: intermediate locally aggressive tumors as defined by WHO, malignant rhabdoid tumor, alveolar soft part sarcoma, infantile fibrosarcoma, unresectable/metastatic dermatofibrosarcoma protuberans, inflammatory myofibroblastic tumor, desmoid fibromatosis, rhabdomyosarcoma, desmoplastic small round cell tumor, BCOR-CCNB3 fusion positive sarcoma.
* Bleeding diathesis: Patients with evidence of active bleeding or bleeding diathesis will be excluded (Note: Patients aged \> 17 years with excess of 2.5 mL of hemoptysis are not eligible).
* Uncontrolled hypertension: Patients with uncontrolled hypertension (CTCAE v5 Grade ≥ 2) are ineligible. Hypertension must be well controlled on stable doses of medication for at least two weeks.
Prior Therapy
* Patients must have had no prior systemic therapy for the treatment of the NRSTS
* Patients must have had no prior anthracycline or ifosfamide chemotherapy
* Patients must have had no prior use of pazopanib or similar multi-targeted TKI.
Patients must have had no prior radiotherapy to tumor-involved sites.
Note: Patients previously treated for a non-NRSTS cancer are eligible provided they meet the prior therapy requirements. Patients who have had chemotherapy or radiotherapy within 4 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier are excluded.
* CYP3A4 Substrates WITH Narrow Therapeutic Indices: Patients chronically receiving medications known to be metabolized by CYP3A4 and with narrow therapeutic indices within 7 days prior to study enrollment, including but not limited to pimozide, aripiprazole, triazolam, ergotamine and halofantrine are not eligible. Note: the use of fentanyl is permitted.
* CYP3A4 Inhibitors: Patients chronically receiving drugs that are known potent CYP3A4 inhibitors within 7 days prior to study enrollment, including but not limited to itraconazole, clarithromycin, erythromycin, many NNRTIs, diltiazem, verapamil, and grapefruit juice are not eligible.
* CYP3A4 Inducers: Patients chronically receiving drugs that are known potent CYP3A4 inducers within 14 days prior to study enrollment, including but not limited to carbamazepine, phenobarbital, phenytoin, rifampin, and St. John's wort are not eligible (with the exception of glucocorticoids).
* Certain medications that are associated with a risk for QTc prolongation and/or Torsade's de Pointes, although not prohibited, should be avoided or replaced with medications that do not carry these risks, if possible.
* Subjects with any condition that may impair the ability to absorb oral medications/investigational product including:
* prior surgical procedures affecting absorption including, but not limited to major resection of stomach or small bowel
* active peptic ulcer disease
* malabsorption syndrome
• 4.12 Thyroid Replacement Therapy: Patients who require thyroid replacement therapy are not eligible if they have not been receiving a stable replacement dose for at least 4 weeks prior to study enrollment.
• 4.13 Subjects with any condition that may increase the risk of gastrointestinal bleeding or gastrointestinal perforation, including: * active peptic ulcer disease * known intraluminal metastatic lesions * inflammatory bowel disease (e.g., ulcerative colitis, Crohn's disease) or other gastrointestinal conditions which increase the risk of perforation * history of abdominal fistula, gastrointestinal perforation or intra- abdominal abscess within 28 days prior to beginning study treatment.
• 4.14 Pulmonary embolism or DVT. Patients must not have experienced: * An untreated pulmonary embolism or DVT in last 6 months or * treated pulmonary embolism or DVT which has been treated with therapeutic anticoagulation for less than 6 weeks * arterial thrombosis in last 12 months History of serious or non-healing wound, ulcer, or bone fracture. Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements. HIV-positive subjects on combination antiretroviral therapy are ineligible because of the potential for pharmacokinetic interactions with pazopanib. In addition, these subjects are at increased risk of lethal infections when treated with marrow-suppressive therapy. Patients who are receiving any other investigational agent(s). Pregnancy and Breast Feeding * Pregnancy and Breast Feeding Female patients who are pregnant are ineligible due to risks of fetal and teratogenic adverse events as seen in animal/human studies. * Lactating females are not eligible unless they have agreed not to breastfeed their infants during treatment and for a period of 1 month following completion of treatment. * Female patients of childbearing potential are not eligible unless a negative pregnancy test result has been obtained. * Unwillingness to use an effective contraceptive method for the duration of their study participation and for at least 1 month after treatment is completed if sexually active with reproductive potential.
PROCEDURE: Surgical resection, RADIATION: Proton beam radiation therapy, DRUG: Pazopanib, DRUG: Ifosfamide, DRUG: Doxorubicin, DRUG: Selinexor
Adipocytic Neoplasm, Liposarcoma, Atypical Fibroxanthoma, Angiomatoid Fibrous Histiocytoma, Fibrosarcoma NOS, Myxofibrosarcoma, Angiosarcoma, Osteosarcoma, Extraskeletal, Dedifferentiated Liposarcoma, Myxoid Liposarcoma, Pleomorphic Liposarcoma, Myxoid Pleomorphic Liposarcoma, Low Grade Fibromyxoid Sarcoma, Sclerosing Epithelioid Fibrosarcoma, Malignant Tenosynovial Giant Cell Tumor of Soft Tissue, Epithelioid Hemangioendothelioma, Glomus Tumor, Inflammatory Leiomyosarcoma, Leiomyosarcoma, Ossifying Fibromyxoid Tumor, Malignant, Myoepithelioma, Synovial Sarcoma, Epithelioid Sarcoma, Perineurioma, Malignant, Clear Cell Sarcoma, Extraskeletal Myxoid Chondrosarcoma, Granular Cell Tumor, Malignant, Melanotic Malignant Nerve Sheath Tumor, Malignant Peripheral Nerve Sheath Tumor, Perivascular Epithelioid Tumor, Malignant, Intimal Sarcoma, Myoepithelial Carcinoma, Undifferentiated Sarcoma, Pleomorphic Sarcoma, Undifferentiated, Round Cell Sarcoma, Undifferentiated, NTRK-Rearranged Spindle Cell Neoplasm, Phosphaturic Mesenchymal Tumor, Malignant, Round Cell Sarcoma, Well Differentiated Liposarcoma, Giant Cell Tumor of Soft Parts NOS, Low Grade Myofibroblastic Sarcoma, Dermatofibrosarcoma Protuberans, Fibrosarcomatous
Proton therapy
A Study Comparing KB803 and Matched Placebo in Patients With Dystrophic Epidermolysis Bullosa (IOLITE)
Bret Augsburger - Bret.Augsburger@cchmc.org
ALL
6 month(s) and over
PHASE3
NCT07016750
Inclusion Criteria:
• The subject and/or their parent/legal guardian must provide informed consent/assent and must be able to and willing to follow study procedures and instructions.
• Age 6 months or older at time of informed consent/assent.
• Confirmed diagnosis of DEB with a mutation in the COL7A1 gene.
• Meets minimum corneal abrasion symptom frequency in the NHS study.
Exclusion Criteria:
• Initiation of any new treatment regimen or change in treatment for ocular disease during the run-in period except for preservative free topical antibiotics or artificial tears/lubricants associated with standard of care treatment of corneal abrasions.
• Treatment with an investigational agent or off-label ophthalmic use of an approved product during the run-in period or planned use during the study (Exceptions may be approved by the medical monitor on a case-by-case basis).
• Any condition that, in the opinion of the Investigator, would impact the completion of all study-related assessments, interfere with the administration of study drug, and/or poses an additional risk to the subject.
• Women who are pregnant or nursing.
• Subject who is unwilling to comply with contraception requirements per protocol.
• Subject is known to be noncompliant or is unlikely to comply with the requirements of the study protocol in the opinion of the Investigator.
BIOLOGICAL: KB803, DRUG: Placebo
Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa
Dystrophic Epidermolysis Bullosa, DEB, Corneal Abrasions
A Phase 2 Study of TCP-25 Gel in Patients With Epidermolysis Bullosa, STEP-study (STEP)
- bret.augsburger@cchmc.org
ALL
4 years and over
PHASE2
NCT06594393
Inclusion Criteria:
* Male or female patients with documented diagnosis of DEB or JEB, confirmed by genetic testing and/or by a skin biopsy with immunofluorescence mapping.
* Patients ≥4 years old. Note: Initially, patients ≥12 years will be enrolled. Enrollment will be open to 4 to 11 year old pediatric patients (both inclusive), after a DMC reviews and provides a positive opinion regarding the safety and tolerability of the IMP in at least 3 patients 12 to 18 years old who have completed at least 4 weeks of IMP use.
Exclusion Criteria:
* The patient has any subtype of EB other than DEB or JEB.
* The patient is currently being treated or planned to be treated with systemic antibiotics.
Note: Use of preventive and/or anti-inflammatory antibiotic treatment, including doxycycline, on an established treatment regimen (stable dose for ≥6 weeks before the Baseline Visit) is permitted. Use of topical antibiotics on the index wounds within 7 days before the Baseline Visit is prohibited.
• Use of systemic corticosteroids \>0.2 mg/kg prednisone dose equivalent per day within 30 days or use of topical corticosteroids on index wounds within 7 days before the Screening Visit.
Note: Corticosteroids for inhalation, ophthalmic, or intranasal use are permitted.
• The patient has a history of or current malignancy over the index wound, eg, basal cell carcinoma or squamous cell carcinoma. DRUG: TCP-25 gel, DRUG: Vehicle (placebo)
Epidermolysis Bullosa (EB), Dystrophic Epidermolysis Bullosa, Junctional Epidermolysis Bullosa
epidermolysis bullosa, topical treatment