A randomized, vehicle-controlled, double-blind, Phase I/IIa, multiple-dose, multiple-cohort, Proof of Concept study in Healthy Volunteers and Patients with Cutaneous T-cell Lymphoma to characterize the safety, efficacy, and pharmacodynamics of topical bimiralisib
- Conditions
- blood cancer affecting the skin10025321
- Registration Number
- NL-OMON48135
- Lead Sponsor
- Piqur Therapeutics AG
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 24
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 <<= 2.5x ULN
c. Total bilirubin <<= 1.5x ULN (except patients with Gilbert*s syndrome, who
may have total bilirubin <<= 3x ULN)
d. Platelet count ><= 100*000 /mm3
e. WBC count ><= 1500 /mm3
f. ANC count ><= 1500 /mm3
g. Fasting blood glucose <<= 125 mg/dL
6. Patient 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.
7. Willing to comply with all study requirements.
8. Patients of reproductive potential must agree to use double contraception
from screening until 90 days after discontinuing study treatment.
9. Females who had a menstrual cycle within 2 years of Screening must have a
negative serum pregnancy test at Screening and a negative urine pregnancy test
on their first dosing
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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Application site assessment of erythema and edema<br /><br>Change from baseline on composite Assessment of Index Lesions Disease Severity<br /><br>Score (CAILS).</p><br>
- Secondary Outcome Measures
Name Time Method <p>Healthy Volunteers: (S)AEs, Laboratory assessments, Systemic PK<br /><br><br /><br>MF patients:<br /><br>* Application site assessment of erythema and edema, (S)AEs, Lab assessments,<br /><br>Systemic PK<br /><br>* Objective Response Rate (ORR) based on the CAILS: patients who achieved<br /><br>partial (PR) or a complete response (CR) after six (6) weeks of treatment.<br /><br>* Time To Response (TTR) based on ORR up to six (6) weeks</p><br>