A Phase 3, Multicenter, Open-Label Extension Study of Patidegib Topical Gel, 2% in Subjects with Gorlin Syndrome (Basal Cell Nevus Syndrome)

Phase 3

Completed

• Conditions: Gorlin Syndrome; Basal Cell Nevus Syndrome; 10040900

• Registration Number: NL-OMON49176
• Lead Sponsor: PellePharm, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 15 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 10

Inclusion Criteria

1. The subject must be at least 18 years old.
2. The subject must provide written informed consent prior to any study
procedures.
3. The subject must have completed PellePharm Study 926-201 or 926-301 with
adequate compliance
4. Study 926-301 subjects must have completed the End of Treatment Visit in
Study 301, prior to the Screening Visit in this study. They must also complete
all Study 301 related procedures prior to the Baseline Visit of this study.
5. The subject must meet diagnostic criteria for Gorlin (basal cell nevus)
syndrome, including major criterion #a plus 1 additional major criterion or
plus 2 additional minor criteria listed in Appendix 17.2
6. The subject must be willing to abstain from application of a non-study
topical medication (prescription or over the counter) to facial skin for the
duration of the trial except as prescribed by the Investigator. Moisturizers
and emollients are allowed. Subjects will be encouraged to use their preferred
sunscreen with a sunscreen protection factor (SPF) of at least 30 daily on all
exposed skin sites.
7. Female subjects must have a negative pregnancy test (serum pregnancy test at
Screening Visit, urine pregnancy test at Baseline Visit). For Study 301
subjects, a negative serum pregnancy test result from Study 301 is acceptable
if the test was done within 7 days of the Screening Visit of this study.
8. If the subject is a woman of child bearing potential (WOCBP), she must be
willing to use birth control methods which may be considered highly effective
(Appendix 17.1). Hormonal contraception must be supplemented with a barrier
method (preferably condom). Birth control must start prior to Baseline,
continue through the duration of the study, and for 30 days after last
application of IP
9. If the subject is a male with a female sex partner who is a WOCBP, the
subject must be willing to use condoms, even after a vasectomy, starting prior
to Baseline, through the duration of the study, and for at least 3 months after
the last application of IP.
10. The subject is willing for all facial BCCs to be evaluated and follow
treatment recommendations made only by the Investigator
11. The subject is willing to forego treatment of facial BCCs with anything
other than the study IP except when the Investigator believes that delay of
treatment of a BCC potentially might compromise the health of the subject. In
such instances, the only other allowed form of treatment is surgical.

Exclusion Criteria

1. The subject has used topical treatment to the face or systemic therapies
that might interfere with the evaluation of the study IP. Among these are use
of the following:
a. 5-fluorouracil, imiquimod, or Ingenol mebutate (except as topical treatment
to anatomical areas other than the face) systemically or topically to the skin
within the 2 months prior to the Screening and Baseline Visit.
b. Systemic chemotherapy within 1 year prior to the Screening and Baseline
Visit.
c. Known inhibitors of the Hedgehog signaling pathway (e.g., vismodegib,
sonidegib, itraconazole) topically (except as topical treatment to anatomical
areas other than the face) or systemically within 2 months prior to the
Screening and Baseline Visit.
d. Photodynamic therapy (PDT) except to localized non-facial, individual BCCs
within 2 months prior to the Screening and Baseline Visit.
2. The subject has a known hypersensitivity to any of the ingredients in the
study IP formulation.
3. The subject is unable or unwilling to make a good faith effort to return to
the study site for all study visits and tests.
4. The subject has current, recent (within five half lives of the experimental
drug or if half life not known, within the past 6 months prior to the Screening
Visit), or planned participation in an experimental drug study (excluding Study
926-301) while enrolled in this study.
5. The subject is a WOCBP who is unwilling or unable to comply with pregnancy
prevention measures.
6. The subject is pregnant or breastfeeding.
7. The subject has any condition or situation which, in the Investigator*s
opinion, may put the subject at significant risk, could confound the study
results, or could interfere significantly with the subject*s participation in
the study. This may include a history of other skin conditions (e.g., severe
facial eczema) or diseases, metabolic dysfunction, physical examination (PE)
findings, or clinical laboratory findings giving reasonable suspicion of a
disease or condition that contraindicates use of an investigational drug or
that might affect interpretation of the results of the study or render the
subject at high risk from treatment complications.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>* The number of lesions on the face that are suspicious for BCC<br /><br>* The number of lesions on the face that are suspicious for BCC and that are<br /><br>surgically removed<br /><br>* The number of lesions on the face that were suspicious for BCC, removed and<br /><br>confirmed on histology to be BCC<br /><br>* Quality of Life Assessments</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>* Safety Assessments<br /><br>* Dermal Safety and Tolerability Events<br /><br>* Physical examinations<br /><br>* Pregnancy tests</p><br>