Clinical trial NCT03180528: Topical Remetinostat in Treating Patient With Cutaneous Basal Cell Cancer
Official title: A Phase 2 Open-Label, Single-Arm Trial of the Efficacy of Topical Remetinostat on Basal Cell Carcinoma in Patients
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Skin Basal Cell Carcinoma
Lead sponsor: Stanford University
Sponsor class: OTHER
Collaborators: name: Medivir
name: National Institutes of Health (NIH)
name: American Skin Association
First posted: 2017-06-08
Enrollment: 30
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This phase 2 trial studies how well remetinostat works in treating patients with skin basal cell cancer. Remetinostat may slow the growth of basal cell cancer cells.

Detailed description: 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.

Eligibility criteria: 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

Primary outcomes:
measure: Overall Response Rate
timeFrame: At 6 weeks
Secondary outcomes:
measure: Number of Participants With a Decrease in Expression of the Hedgehog Biomarker Gene GLI1
timeFrame: 6 weeks
measure: Adverse Events Contributing to Treatment Discontinuation or Interruption
timeFrame: 6 weeks
measure: Participants Who Discontinued Treatment or Had Treatment Interruption
timeFrame: 6 weeks
Other outcomes:
Locations:
city: Palo Alto
country: United States
facility: Stanford University, School of Medicine
state: California
Study officials:
affiliation: Stanford University
name: Kavita Sarin
role: PRINCIPAL_INVESTIGATOR
References: