Topical Remetinostat in Treating Patient With Cutaneous Basal Cell Cancer

PRIMARY OBJECTIVES:

I. Overall response rate of basal cell carcinoma (BCC) in subjects at 6 weeks.

SECONDARY OBJECTIVES:

I. Suppression of GLI1 (glioma-associated oncogene) expression in treated BCCs as compared
with baseline.

II. Safety assessment of Remetinostat after 6 weeks of topical treatment.

OUTLINE:

Tumors receive Remetinostat topically three times per day (TID) for 6 weeks in the absence of
disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed for at least 4 weeks.

Inclusion Criteria:

- Must have at least one BCC lesion > 1 cm (BCC > 5 mm) in non-cosmetically sensitive
site(s)

- Must be willing to apply the topical remetinostat 3 times daily for 6 weeks

- For women of child bearing potential, a negative urine pregnancy test

- Women of child bearing potential are expected to use an effective method of birth
control while participating in the study and for 1 month after applying the last dose

- For male subjects with female partners of childbearing potential, agreement to use
adequate contraception while participating in the study and for 1 month after applying
the last dose

- Has signed and dated the current Institutional Review Board (IRB) approved informed
consent document

Exclusion Criteria:

- Taking any medication known to interact with histone deacetylase (HDAC) inhibitors,
such as valproate or anticoagulants

- Taking any medication known to affect hedgehog (HH) signaling pathway such as
itraconazole

- Within the past 6 months, has used topical or systemic therapies that might interfere
with the evaluation of the study medication during the study; specifically, these
include the topical use to the study tumors of:

- Glucocorticoids

- Retinoids either systemically or topically (eg, etretinate, isotretinoin,
tazarotene, tretinoin, adapalene)

- Alpha hydroxy acids (eg, glycolic acid, lactic acid) to > 5% of the skin

- 5 fluorouracil or imiquimod and/or

- Itraconazole

- Has received treatment with systemic chemotherapy or agents known to be inhibitors of
HH signaling, within 60 days to starting study medication

- Currently receiving systemic medications that could affect BCC tumors (eg, oral
retinoids) or might interact with remetinostat

- Uncontrolled intercurrent illness including, but not limited to, ongoing or active
infection, recurrent seizure history or psychiatric illness/social situations that
would limit compliance with study requirements

- Moderate to severe immunosuppression due to disease or medication

- Known or previous hypersensitivity to histone deacetylase inhibitor (HDACi)

- History of congestive heart failure; cardiac arrhythmias; or other findings of
ventricular dysfunction

- History of current evidence of malabsorption or liver disease

- Pregnancy or breast feeding