Trial Outcomes & Findings for Topical Remetinostat in Treating Patient With Cutaneous Basal Cell Cancer (NCT NCT03180528)

Last Updated: 2021-06-08

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

30 participants

Primary outcome timeframe

At 6 weeks

Results posted on

2021-06-08

Participant Flow

Participant milestones

Participant milestones
Measure	Treatment (Remetinostat) Patients receive topical remetinostat 1% gel applied TID directly to the lesion, for 6 weeks in the absence of disease progression or unacceptable toxicity. Remetinostat: Applied topically under bandage occlusion
Overall Study STARTED	30
Overall Study COMPLETED	27
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Treatment (Remetinostat) Patients receive topical remetinostat 1% gel applied TID directly to the lesion, for 6 weeks in the absence of disease progression or unacceptable toxicity. Remetinostat: Applied topically under bandage occlusion
Overall Study Adverse Event	1
Overall Study Withdrawal by Subject	1
Overall Study Eligible and enrolled, but withdrawn due to abnormal baseline laboratory values.	1

Baseline Characteristics

Topical Remetinostat in Treating Patient With Cutaneous Basal Cell Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (Remetinostat) n=30 Participants Patients receive topical remetinostat 1% gel applied TID directly to the lesion, for 6 weeks in the absence of disease progression or unacceptable toxicity. Remetinostat: Applied topically under bandage occlusion
Age, Categorical <=18 years	0 Participants n=99 Participants
Age, Categorical Between 18 and 65 years	21 Participants n=99 Participants
Age, Categorical >=65 years	9 Participants n=99 Participants
Age, Continuous	59.26 years STANDARD_DEVIATION 10.6 • n=99 Participants
Sex: Female, Male Female	11 Participants n=99 Participants
Sex: Female, Male Male	19 Participants n=99 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=99 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	27 Participants n=99 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	2 Participants n=99 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants
Race (NIH/OMB) Asian	0 Participants n=99 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=99 Participants
Race (NIH/OMB) Black or African American	0 Participants n=99 Participants
Race (NIH/OMB) White	27 Participants n=99 Participants
Race (NIH/OMB) More than one race	0 Participants n=99 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=99 Participants
Region of Enrollment United States	30 participants n=99 Participants

PRIMARY outcome

Timeframe: At 6 weeks

Population: Analysis is on a per-lesion basis, and not a per-participant basis. Per protocol, analysis does not include lesions from participants \<70% compliant with treatment regimen.

Outcome measures

Outcome measures
Measure	Treatment (Remetinostat) n=33 Tumor lesions Patients receive topical remetinostat 1% gel applied TID directly to the lesion, for 6 weeks in the absence of disease progression or unacceptable toxicity. Remetinostat: Applied topically under bandage occlusion
Overall Response Rate	69.7 percentage of tumor lesions Interval 54.0 to 82.5

SECONDARY outcome

Timeframe: 6 weeks

Population: Results are reported for those participants for whom baseline and 6-week analysis data were available. The COVID-19 epidemic adversely affected the ability of the laboratory to process and return results.

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.

Outcome measures

Outcome measures
Measure	Treatment (Remetinostat) n=6 Participants Patients receive topical remetinostat 1% gel applied TID directly to the lesion, for 6 weeks in the absence of disease progression or unacceptable toxicity. Remetinostat: Applied topically under bandage occlusion
Number of Participants With a Decrease in Expression of the Hedgehog Biomarker Gene GLI1	5 Participants

SECONDARY outcome

Timeframe: 6 weeks

Population: The outcome is reported as a number of adverse events (AEs), not as a number of participants. For some participants, multiple AEs contributing to treatment decisions.

Adverse events (AEs) contributing to treatment discontinuation or interruption are reported as the number of such events, a number without dispersion.

Outcome measures

Outcome measures
Measure	Treatment (Remetinostat) n=29 Participants Patients receive topical remetinostat 1% gel applied TID directly to the lesion, for 6 weeks in the absence of disease progression or unacceptable toxicity. Remetinostat: Applied topically under bandage occlusion
Adverse Events Contributing to Treatment Discontinuation or Interruption AEs contributing to treatment interruption	9 adverse events
Adverse Events Contributing to Treatment Discontinuation or Interruption AEs contributing to treatment discontinuation	3 adverse events

SECONDARY outcome

Timeframe: 6 weeks

Population: For some participants, multiple AEs contributing to the treatment decision.

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.

Outcome measures

Outcome measures
Measure	Treatment (Remetinostat) n=29 Participants Patients receive topical remetinostat 1% gel applied TID directly to the lesion, for 6 weeks in the absence of disease progression or unacceptable toxicity. Remetinostat: Applied topically under bandage occlusion
Participants Who Discontinued Treatment or Had Treatment Interruption Participants who experienced treatment interruption	5 Participants
Participants Who Discontinued Treatment or Had Treatment Interruption Participants who discontinued treatment	3 Participants

Adverse Events

Treatment (Remetinostat)

Serious events: 0 serious events

Other events: 29 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Treatment (Remetinostat) n=29 participants at risk Patients receive topical remetinostat 1% gel applied TID directly to the lesion, for 6 weeks in the absence of disease progression or unacceptable toxicity. Remetinostat: Applied topically under bandage occlusion
Skin and subcutaneous tissue disorders Eczema	93.1% 27/29 • Number of events 27 • 12 weeks Data are reported for subjects that received the study treatment only. Subjects who were enrolled, but were withdrawn without receiving study treatment due to abnormal baseline laboratory values are not included.
Skin and subcutaneous tissue disorders Pain of Skin	20.7% 6/29 • Number of events 6 • 12 weeks Data are reported for subjects that received the study treatment only. Subjects who were enrolled, but were withdrawn without receiving study treatment due to abnormal baseline laboratory values are not included.
Skin and subcutaneous tissue disorders Pruritis	3.4% 1/29 • Number of events 1 • 12 weeks Data are reported for subjects that received the study treatment only. Subjects who were enrolled, but were withdrawn without receiving study treatment due to abnormal baseline laboratory values are not included.
Skin and subcutaneous tissue disorders Skin Ulceration	3.4% 1/29 • Number of events 1 • 12 weeks Data are reported for subjects that received the study treatment only. Subjects who were enrolled, but were withdrawn without receiving study treatment due to abnormal baseline laboratory values are not included.
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders - Other, tumor hemorrhage	3.4% 1/29 • Number of events 1 • 12 weeks Data are reported for subjects that received the study treatment only. Subjects who were enrolled, but were withdrawn without receiving study treatment due to abnormal baseline laboratory values are not included.

Additional Information

Kavita Yang Sarin

Stanford University

Phone: (650) 723-6316

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place