blood cancer affecting the skin
Conditions
Interventions
Topical bimiralisib/vehicle non-aqueous gel to be administered to target
lesion(s) of up to 400cm2 (in healthy volunteers) or up to 200cm2 (in MF
patients) in the following dose levels:
* Vehicle
*
Sponsors
Piqur Therapeutics AG
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: Healthy volunteers may be included in the study if they meet all the below inclusion criteria: 1. Males * 18 years of age 2. Fitzpatrick skin type I, II, III or IV 3. No clinical significant skin disease in the treatment area(s) and no hypertrophic scarring 4. Willing and able to washout and withhold any topical treatment in the treatment area (2 weeks) 5. Subjects must understand the investigational nature of this study and sign an independent ethics committee-approved written informed consent form prior to any study related procedure. 6. Willing to comply with all study requirements. 7. Subjects of reproductive potential must agree to use double contraception from screening until 90 days after discontinuing study treatment and withhold from any sperm donation.Patients may be included in the study if they meet all the below inclusion criteria: 1. Males or females * 18 years of age 2. Confirmed diagnosis of CTCL (MF): MF stage 1A or 1B (maximum T2N0M0B1) as per Modified ISCL/EORTC revisions (Olsen-2011): a. Confirmed histopathological diagnosis from skin biopsy representative of current disease by pathologist with expertise in cutaneous lymphoma. The date of biopsy should be within the last 5 years. If diagnosis is not confirmed by light microscopic examination, ISCL diagnostic criteria must be used. 3. At least 1, 2 and up to 3 target lesions with a (combined) total size of at least 150 cm2 4. Willing and able to washout any previous topical treatment (at least 2 weeks) and any systemic treatment (at least 4 weeks) prior to first application of topical bimiralisib. 5. Otherwise healthy, i.e. absence of clinically significant or unstable disease, with acceptable organ function with lab values within normal range or as specified below: a. eGFR (mCockcroft-Gault) > 30 mL/min. b. AST and ALT
Exclusion criteria
Exclusion criteria: Healthy volunteers will be excluded from the study if they meet any of the below exclusion criteria: 1. Known hypersensitivity to any of the excipients of bimiralisib gel. 2. Evidence of any clinically significant active or unstable chronic disease or condition that could interfere with, or for which the treatment of might interfere with, the conduct of the study, or that would pose an unacceptable risk to the subject in the opinion of the investigator (following a detailed medical history, physical examination, vital signs (systolic and diastolic blood pressure, pulse rate, body temperature) and 12-lead electrocardiogram (ECG) at screening or pre-dose). Minor deviations from the normal range may be accepted, if judged by the Investigator to have no clinical relevance. 3. Clinically significant abnormalities, as judged by the investigator, in laboratory test results (including hepatic and renal panels, complete blood count, chemistry panel and urinalysis) at screening or pre-dose. In the case of uncertain or questionable results, tests performed during screening may be repeated before randomization to confirm eligibility or judged to be clinically irrelevant for healthy subjects; 4. Positive Hepatitis B surface antigen (HBsAg), Hepatitis C antibody (HCV Ab), or human immunodeficiency virus antibody (HIV Ab) at screening, or other known infection requiring antibiotic therapy within the last three months prior to the study. 5. Participation in an investigational drug or device study within 3 months prior to screening or more than 4 times in the past year; 6. Donation of blood or blood loss of >500 mL within 3 months prior to screening or donation of plasma within 14 days 7. Any condition that in the opinion of the investigator would complicate or compromise the study or the well-being of the subjectPatients will be excluded from the study if they meet any of the below exclusion criteria: 1. Known hypersensitivity to any of the excipients of bimiralisib gel. 2. Patients who are on (or will require) any systemic treatment to treat their disease (MF) during the study. 3. Concurrent severe and/or uncontrolled medical conditions that would, in the investigator*s judgment, contraindicate patient participation in the clinical study or require concomitant skin-directed or systemic therapy (e.g., active or uncontrolled severe infection, chronic active hepatitis, immuno-compromised, acute or chronic pancreatitis, uncontrolled high blood pressure, interstitial lung disease, etc.). 4. Has other active malignancies that require systemic treatment. 5. Has a known history of HIV infection or hepatitis (testing not mandatory). 6. Pregnant or nursing (lactating) women. 7. Has a known history of alcohol or drug abuse within the past 1 year. 8. Psychiatric illness, disability or social situation that would compromise the subject*s safety or ability to provide consent, or limit compliance with study requirements.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Application site assessment of erythema and edema Change from baseline on composite Assessment of Index Lesions Disease Severity Score (CAILS). | — |
Secondary
| Measure | Time frame |
|---|---|
| Healthy Volunteers: (S)AEs, Laboratory assessments, Systemic PK MF patients: * Application site assessment of erythema and edema, (S)AEs, Lab assessments, Systemic PK * Objective Response Rate (ORR) based on the CAILS: patients who achieved partial (PR) or a complete response (CR) after six (6) weeks of treatment. * Time To Response (TTR) based on ORR up to six (6) weeks | — |
Countries
Netherlands
Outcome results
None listed