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About the CODY Study

CODY is a multi-center Phase 2b clinical study evaluating the safety and effectiveness of a topical gel (experimental topical rapamycin, or PTX-367) for Gorlin syndrome. The CODY study will determine if PTX-367 is safe and effective in preventing the number of new basal cell carcinomas (BCCs) on the face. Your participation in the study is expected to last for six months.

About Gorlin Syndrome

Gorlin syndrome, also known as nevoid basal cell carcinoma syndrome, is a rare genetic disease caused by a mutation in one of several genes (PTCH1, PTCH2, SUFU). These genes are responsible for suppressing the formation of tumors.

People with Gorlin syndrome can develop hundreds of basal cell carcinomas (BCCs) in their lifetimes. Many afflicted individuals undergo repeated surgical removal of these cancerous lesions.

There are no FDA-approved medical therapies for Gorlin syndrome.

About PTX-367 (QTORIN rapamycin)

PTX-367 is a topical gel formulation of a medicine called rapamycin being developed for the potential treatment of Gorlin syndrome. Rapamycin blocks the activation of a naturally occurring protein molecule (mTOR) that has been shown to help tumors form in several studies1. A recent study also showed that mTOR could be a factor in the formation of BCCs in those with Gorlin syndrome2.

1. Hua, H., Kong, Q., Zhang, H. et al. Targeting mTOR for cancer therapy. J Hematol Oncol 12, 71 (2019) doi:10.1186/s13045-019-0754-1

2. Back, J. et al. SOX9 Transcriptionally Regulates mTOR-Induced Proliferation of Basal Cell Carcinomas. J Invest Dermatol. 2018 August; 138(8): 1716–1725.

Interested in Enrolling?

Palvella plans to begin enrolling for the CODY Study in late spring 2021. We anticipate that your participation will last approximately six months. To qualify for CODY, participants must meet certain criteria, including confirmed diagnosis of Gorlin syndrome with the PTCH1 mutation.

If travel is required, financial and logistical assistance will be provided. As we plan for the study, we are mindful of the impact of COVID-19 on the health of our participants and clinicians.

Deciding whether to participate in a clinical trial is a voluntary and personal decision. We encourage you to talk with your family about whether enrolling in any clinical trial is the right decision for you.

Anticipated Requirements Include:
  • Confirmed Gorlin Syndrome with PTCH1 mutation
  • Adults, age 18 or older
  • History of at least 10 BCCs on the face within past 24 months
  • Cannot have participated in another Gorlin syndrome clinical study in past 3 months

Find a Trial Location

Multiple locations across USA (TBD)

United States

Multiple locations across USA (TBD)

Travel Assistance Available