BCCs in Gorlin Syndrome Patients
Conditions
Keywords
Gorlin Syndrome, Basal Cell Carcinoma, Nevoid basal cell carcinoma syndrome, Basal cell nevus syndrome BCNS, Gorlin Syndrome Alliance, Prevention, Topical, Sirolimus
Brief summary
A Phase 2b study looks at the safety and efficacy of a treatment that is being investigated for people with certain diseases. This study will be conducted at multiple centers in the United States where participants with Gorlin Syndrome, also known as basal cell nevus syndrome, will be randomly placed into two groups; one group will receive the active topical gel, the other a topical vehicle gel, also know as placebo. Participants will apply this topical product to their face once a day for 6 months. The study will be looking at the number of new BCCs that develop on the faces of all the participants during this time.
Interventions
DRUGPTX-022
QTORIN 3.9 % Sirolimus Topical Gel
Vehicle comparator topical gel
Sponsors
Palvella Therapeutics, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
PREVENTION
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Adults, 18 years or older * Meet diagnostic criteria for Gorlin Syndrome * Willing to avoid application of a non-study topical medication to the face (prescription or over the counter) during the study. * Willing to forego treatment of BCCs with anything other than the study medication except when the Investigator believes that delay of treatment of a BCC potentially might compromise the health of the subject. During the trial, the only allowed form of BCC treatment is surgical. * Participant will be encouraged to use their preferred sunscreen with a sun protector factor (SPF) of at least 30 daily Key
Exclusion criteria
* Recently participated in a clinical trial evaluating an investigational product for the treatment of BCCs or Gorlin Syndrome within 3 months prior to to starting study medication. Participant can start the study screening period prior to completing the 3 month washout. * Recently used topical or systemic (oral) treatment that might interfere with the evaluation of the study medication.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of new biopsy confirmed BCCs that develop on the face compared between the active and vehicle treatment arms | Month 6 |
| Incidence of dermatological, treatment emergent adverse events after treatment with active | Month 6 |
Secondary
| Measure | Time frame |
|---|---|
| Percentage of participants developing 2 or more new biopsy confirmed BCCs on the face. | Month 6 |
| Percentage of participants developing 1 or more new biopsy confirmed BCCs on the face. | Month 6 |
Countries
United Kingdom, United States
Outcome results
None listed