Skin Basal Cell CarcinomaPhase 2NCT03180528
Topical Remetinostat in Treating Patient With Cutaneous Basal Cell Cancer
A Phase 2 study testing Remetinostat in people with Skin Basal Cell Carcinoma, run by Kavita Sarin at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Skin Basal Cell Carcinoma
- What is being tested?
- Remetinostat
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- 7 Jul 2018to 31 Dec 2020
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 16Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Stanford University, School of Medicine
Palo Alto, California, 94304
About this study
Official title: A Phase 2 Open-Label, Single-Arm Trial of the Efficacy of Topical Remetinostat on Basal Cell Carcinoma in Patients
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.
Read the detailed description
PRIMARY OBJECTIVES:
- Overall response rate of basal cell carcinoma (BCC) in subjects at 6 weeks.
SECONDARY OBJECTIVES:
- 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.
- Conditions
- Skin Basal Cell Carcinoma
- Being tested
- Remetinostat
- Medical subject headings
- Carcinoma, Basal Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RemetinostatDrug
Also called suberohydroxamic acid phenyl ester (SHAPE); SHAPE Gel; SHP 141; and 4 [[8 (hydroxyamino) 1,8 dioxooctyl]oxy] benzoic acid methyl ester
Applied topically under bandage occlusion
| Study group | Role | What they receive |
|---|---|---|
| Treatment (remetinostat) | Experimental | Patients receive topical remetinostat 1% gel applied TID directly to the lesion, for 6 weeks in the absence of disease progression or unacceptable toxicity. Receives: Drug: Remetinostat |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: At 6 weeks
Secondary outcomes (3)
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.
Measured: 6 weeks
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.
Measured: 6 weeks
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.
Measured: 6 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jun 2017
- Study start7 Jul 2018
- Primary completion7 Jul 2020
- Study completion31 Dec 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Skin Basal Cell CarcinomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kavita Sarin
- Sponsor type
- University, hospital or other
- Collaborators
- Medivir, National Institutes of Health (NIH), American Skin Association
- Responsible party
- Sponsor-investigator, Kavita Sarin, Stanford University
- Investigators
- Kavita Sarin, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03180528
- Sponsor's protocol ID
- IRB-40947
- Other IDs
- NCI-2017-00981, SKIN0037
- Status last verified
- May 2021
- First submitted
- 6 Jun 2017
- Last updated
- 8 Jun 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Open-Label, Single-Arm Trial of the Efficacy of Topical Remetinostat on Basal Cell Carcinoma in Patients
- ClinicalTrials.gov ID
- NCT03180528
- Acronym / sponsor ID
- IRB-40947
- Conditions
- Skin Basal Cell Carcinoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Kavita SarinUniversity, hospital or other
- Collaborators
- Medivir, National Institutes of Health (NIH), American Skin Association
- Study start
- 7 Jul 2018 (actual)
- Primary completion
- 7 Jul 2020 (actual)
- Study completion
- 31 Dec 2020 (actual)
- First posted
- 8 Jun 2017 (actual)
- Results
- Posted 5 Jan 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- Overall response rate of basal cell carcinoma (BCC) in subjects at 6 weeks.
SECONDARY OBJECTIVES:
- 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (remetinostat) | Experimental | Drug: RemetinostatPatients receive topical remetinostat 1% gel applied TID directly to the lesion, for 6 weeks in the absence of disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (16 listed)
- 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:
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RemetinostatAlso: suberohydroxamic acid phenyl ester (SHAPE); SHAPE Gel; SHP 141; and 4 [[8 (hydroxyamino) 1,8 dioxooctyl]oxy] benzoic acid methyl esterApplied topically under bandage occlusion | Drug | Treatment (remetinostat) |
Primary outcome
Overall Response Rate
Time frame: At 6 weeks
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.
3 secondary outcomes
Number of Participants With a Decrease in Expression of the Hedgehog Biomarker Gene GLI1
Time frame: 6 weeks
Adverse Events Contributing to Treatment Discontinuation or Interruption
Time frame: 6 weeks
Participants Who Discontinued Treatment or Had Treatment Interruption
Time frame: 6 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jun 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kavita Sarin principal investigator
Stanford University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Remetinostat in people with Skin Basal Cell Carcinoma, run by Kavita Sarin at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 5 Jan 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Response Rate (time frame: At 6 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jun 2021.