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Topical Remetinostat in Treating Patient With Cutaneous Basal Cell Cancer

Primary Purpose

Skin Basal Cell Carcinoma

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Remetinostat
Sponsored by
Kavita Sarin
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Skin Basal Cell Carcinoma

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Stanford University, School of Medicine

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Treatment (remetinostat)

Arm Description

Patients receive topical remetinostat 1% gel applied TID directly to the lesion, for 6 weeks in the absence of disease progression or unacceptable toxicity.

Outcomes

Primary Outcome Measures

Overall Response Rate
Overall response is defined as achieving either a complete response (CR) or a partial response (PR). Response is based on the Response Evaluation Criteria in Solid Tumors (RECIST), as follows. CR = tumor lesion becomes undetectable PR = ≥30% decrease in total tumor diameter Overall response (OR) = CR+PR Stable Disease (SD) = decrease in total tumor diameter is >0% and <30% Progressive Disease (PD) = increase in total tumor diameter Exact binomial 90% confidence intervals (90%) will be computed for OR. The data are reported accord to the per protocol analysis, ie, including lesions for subjects who were <70% compliant with drug treatment. For subjects who were compliant but dropped out, data from their last study visit will be used if they contribute a biopsy. The analysis population will include the participants who have provided pre-treatment and post-treatment biopsies. The outcome is reported as the percent of tumor lesions that achieve OR, with 90% CI.

Secondary Outcome Measures

Number of Participants With a Decrease in Expression of the Hedgehog Biomarker Gene GLI1
The effect of topical remetinostat gel 1% on decreasing expression of Hedgehog biomarker gene GLI1 was determined using the RNeasy Fibrous Tissue Mini Kit (Qiagen, Valencia, CA), a polymerase chain reaction (PCR) test kit. The levels observed at baseline and after 6 weeks treatment were obtained. The outcome is reported as the number of subjects for whom a decrease in expression of the Hedgehog biomarker gene GLI1 was observed, a number without dispersion.
Adverse Events Contributing to Treatment Discontinuation or Interruption
Adverse events (AEs) contributing to treatment discontinuation or interruption are reported as the number of such events, a number without dispersion.
Participants Who Discontinued Treatment or Had Treatment Interruption
The number of participants who discontinued treatment or experienced treatment interruption within the first 6 weeks of treatment are reported as the number of such participants, a number without dispersion.

Full Information

First Posted
June 6, 2017
Last Updated
May 13, 2021
Sponsor
Kavita Sarin
Collaborators
Medivir, National Institutes of Health (NIH), American Skin Association
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1. Study Identification

Unique Protocol Identification Number
NCT03180528
Brief Title
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

2. Study Status

Record Verification Date
May 2021
Overall Recruitment Status
Completed
Study Start Date
July 7, 2018 (Actual)
Primary Completion Date
July 7, 2020 (Actual)
Study Completion Date
December 31, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Kavita Sarin
Collaborators
Medivir, National Institutes of Health (NIH), American Skin Association

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Skin Basal Cell Carcinoma

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
30 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Treatment (remetinostat)
Arm Type
Experimental
Arm Description
Patients receive topical remetinostat 1% gel applied TID directly to the lesion, for 6 weeks in the absence of disease progression or unacceptable toxicity.
Intervention Type
Drug
Intervention Name(s)
Remetinostat
Other Intervention Name(s)
suberohydroxamic acid phenyl ester (SHAPE); SHAPE Gel; SHP 141; and 4 [[8 (hydroxyamino) 1,8 dioxooctyl]oxy] benzoic acid methyl ester
Intervention Description
Applied topically under bandage occlusion
Primary Outcome Measure Information:
Title
Overall Response Rate
Description
Overall response is defined as achieving either a complete response (CR) or a partial response (PR). Response is based on the Response Evaluation Criteria in Solid Tumors (RECIST), as follows. CR = tumor lesion becomes undetectable PR = ≥30% decrease in total tumor diameter Overall response (OR) = CR+PR Stable Disease (SD) = decrease in total tumor diameter is >0% and <30% Progressive Disease (PD) = increase in total tumor diameter Exact binomial 90% confidence intervals (90%) will be computed for OR. The data are reported accord to the per protocol analysis, ie, including lesions for subjects who were <70% compliant with drug treatment. For subjects who were compliant but dropped out, data from their last study visit will be used if they contribute a biopsy. The analysis population will include the participants who have provided pre-treatment and post-treatment biopsies. The outcome is reported as the percent of tumor lesions that achieve OR, with 90% CI.
Time Frame
At 6 weeks
Secondary Outcome Measure Information:
Title
Number of Participants With a Decrease in Expression of the Hedgehog Biomarker Gene GLI1
Description
The effect of topical remetinostat gel 1% on decreasing expression of Hedgehog biomarker gene GLI1 was determined using the RNeasy Fibrous Tissue Mini Kit (Qiagen, Valencia, CA), a polymerase chain reaction (PCR) test kit. The levels observed at baseline and after 6 weeks treatment were obtained. The outcome is reported as the number of subjects for whom a decrease in expression of the Hedgehog biomarker gene GLI1 was observed, a number without dispersion.
Time Frame
6 weeks
Title
Adverse Events Contributing to Treatment Discontinuation or Interruption
Description
Adverse events (AEs) contributing to treatment discontinuation or interruption are reported as the number of such events, a number without dispersion.
Time Frame
6 weeks
Title
Participants Who Discontinued Treatment or Had Treatment Interruption
Description
The number of participants who discontinued treatment or experienced treatment interruption within the first 6 weeks of treatment are reported as the number of such participants, a number without dispersion.
Time Frame
6 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Kavita Sarin
Organizational Affiliation
Stanford University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Stanford University, School of Medicine
City
Palo Alto
State/Province
California
ZIP/Postal Code
94304
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

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Topical Remetinostat in Treating Patient With Cutaneous Basal Cell Cancer

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