A Randomized, Double-Blind, Placebo-Controlled, Dose-Escalating Phase 1b Study to Assess the Safety, Pharmacodynamics and Pharmacokinetics of SHP 141, A Histone Deacetylase Inhibitor, Administered Topically Up to 28 Days to Patients With Stage IA, IB or IIA Cutaneous T-Cell Lymphoma
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 18
- 试验地点
- 6
- 主要终点
- Percentage of Patients With Complete or Partial Response as Measured by Change in Lesion Severity Using CAILS (Composite Assessment of Index Lesion Severity)
研究概览
简要总结
The purpose of this study is to investigate the safety and tolerability of topical SHP141 applied directly to skin lesions in patients with Stage IA, IB, or IIA Cutaneous T-cell Lymphoma. This study will also investigate the effect of SHP141 on skin lesions in patients with Stage IA, IB, or IIA CTCL.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- •Histopathologically confirmed CTCL; a documented verifiable biopsy report is required.
- •Documented clinical Stage IA, IB, or IIA CTCL.
- •Skin lesion involvement of at least 3% of BSA accessible for topical application of study drug and biopsy.
- •ECOG performance status of 0-2.
排除标准
- •CTCL with histologic evidence of folliculotropic variant or large cell transformed CTCL.
- •Severe pruritus requiring systemic or topical treatment.
- •Palpable lymph node ≥1.5 cm in diameter (unless the lymph node has been biopsied and has been designated as Stage IA-IIA disease).
- •Coexistent second malignancy or history of prior solid organ malignancy within previous 5 years (excluding basal or squamous cell carcinoma of the skin, in situ carcinoma of the cervix (CIN 3), papillary or follicular thyroid cancer that has been treated curatively, or prostate cancer that has been treated curatively).
- •Any prior history of a hematologic malignancy (other than CTCL).
- •History of or current major renal, hepatic, gastrointestinal, pulmonary, cardiovascular, genito-urinary or hematological disease, CNS disorders, infectious disease or coagulation disorders as determined by the Investigator.
- •Evidence of active Hepatitis B or C or HIV.
- •Circulating atypical cells >5%
研究组 & 干预措施
placebo for SHAPE (SHP-141)
placebo for SHAPE (SHHP-141) topical gelled solution
干预措施: placebo for SHAPE (SHP-141) (Drug)
SHAPE (SHP-141) 0.1%BID
SHAPE (SHP-141) topical gelled solution at 0.1% concentration twice weekly
干预措施: SHAPE (SHP-141) 0.1% BID (Drug)
SHAPE (SHP-141) 0.5% BID
SHAPE (SHP-141) topical gelled solution at 0.5% concentration twice weekly
干预措施: SHAPE (SHP-141) 0.5% BID (Drug)
SHAPE (SHP-141) 1.0% BID
SHAPE (SHP-141) topical gelled solution at 1.0% concentration twice weekly
干预措施: SHAPE (SHP-141) 1.0% BID (Drug)
结局指标
主要结局
Percentage of Patients With Complete or Partial Response as Measured by Change in Lesion Severity Using CAILS (Composite Assessment of Index Lesion Severity)
时间窗: Weekly through day 28 (days 1, 7, 14, 21, 28) and again day 42
Response assessed by change in lesion severity using Composite Assessment of Index Lesion Severity (CAILS) Assessment Tool which measures clinical signs of CTCL by erythema; scaling; plaque elevation; hypo- or hyperpigmentation, each on a scale of 0-8; and lesion size (cm2), on a scale of 0 (no lesion; 0 cm2) to 18 (300 cm2). Up to five index lesions are each scored, and a subtotal CAILS score is provided for each index lesion. A total score is calculated by summing these subtotals. Response criteria measure the change in CAILS score from baseline to follow-up as follows: Complete Response (CR): 100% decrease in CAILS score; Partial Response (PR): 50% - 99% decrease in CAILS score; Stable Disease (SD): \< 25% increase to \< 50% decrease in CAILS score; Progressive Disease (PD) ≥ 25% increase in CAILS score.
次要结局
未报告次要终点