A Phase 2 Open-Label, Single-Arm Trial of the Efficacy of Topical Remetinostat on Basal Cell Carcinoma in Patients
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Overall Response Rate
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
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.
干预措施: Remetinostat (Drug)
结局指标
主要结局
Overall Response Rate
时间窗: 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.
次要结局
- Number of Participants With a Decrease in Expression of the Hedgehog Biomarker Gene GLI1(6 weeks)
- Adverse Events Contributing to Treatment Discontinuation or Interruption(6 weeks)
- Participants Who Discontinued Treatment or Had Treatment Interruption(6 weeks)
研究者
Kavita Sarin
Principal Investigator
Stanford University