Safety, Pharmacodynamics (PD), Pharmacokinetics (PK) Study of SHP141 in 1A, 1B, or 2A Cutaneous T-Cell Lymphoma (CTCL)

Phase 1

Completed

Interventions: Drug: placebo for SHAPE (SHP-141)
Drug: SHAPE (SHP-141) 0.1% BID
Drug: SHAPE (SHP-141) 0.5% BID
Drug: SHAPE (SHP-141) 1.0% BID

Brief Summary: 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.

Recruitment & Eligibility

Inclusion Criteria

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.

Exclusion Criteria

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%

Arm && Interventions

Group	Intervention	Description
placebo for SHAPE (SHP-141)	placebo for SHAPE (SHP-141)	placebo for SHAPE (SHHP-141) topical gelled solution
SHAPE (SHP-141) 0.1%BID	SHAPE (SHP-141) 0.1% BID	SHAPE (SHP-141) topical gelled solution at 0.1% concentration twice weekly
SHAPE (SHP-141) 0.5% BID	SHAPE (SHP-141) 0.5% BID	SHAPE (SHP-141) topical gelled solution at 0.5% concentration twice weekly
SHAPE (SHP-141) 1.0% BID	SHAPE (SHP-141) 1.0% BID	SHAPE (SHP-141) topical gelled solution at 1.0% concentration twice weekly

Primary Outcome Measures

Name	Time	Method
Percentage of Patients With Complete or Partial Response as Measured by Change in Lesion Severity Using CAILS (Composite Assessment of Index Lesion Severity)	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.

Secondary Outcome Measures

Name	Time	Method

Locations (6): Stanford University
🇺🇸
Stanford, California, United States
MD Anderson Cancer Center
🇺🇸
Houston, Texas, United States
University Hospitals Case Medical Center
🇺🇸
Cleveland, Ohio, United States
Cleveland Clinic
🇺🇸
Cleveland, Ohio, United States
Northwestern University Dept of Dermatology
🇺🇸
Chicago, Illinois, United States
Ohio State University
🇺🇸
Gahanna, Ohio, United States