A Safety, Efficacy and Pharmacokinetics Study of CD11301 for the Treatment of Cutaneous T-Cell Lymphoma (CTCL)

Phase 2

Completed

Conditions: Cutaneous T Cell Lymphoma

Interventions: Drug: CD11301 0.03%
Drug: Placebo
Drug: CD11301 0.06%

Registration Number: NCT03292406

Lead Sponsor: Galderma R&D

Brief Summary: To assess the efficacy, safety and pharmacokinetics in participants treated with CD11301 gel vs. placebo for early stage CTCL (IA, IB, or IIA).

Detailed Description: To assess the efficacy and safety of two concentrations (0.03% and 0.06%) of CD11301 gel in the treatment of early stage CTCL (stage IA, IB, or IIA) versus placebo.

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 86

Inclusion Criteria

Clinical Diagnosis of CTCL stage IA, IB, or IIA with biopsy within last 3 months
Have BSA involvement corresponding to stages IA, IB or IIA CTCL with at least 3 distinct lesions

Exclusion Criteria

CTCL that is stage IIB or great or stage IIA with stage N2 with >5% circulating Sezary cells or CD8+ or large cell transformation or Progressive CTCL
History of autoimmune disease
Laboratory test values at screening outside of the normal range and judged clinically significant by the investigator
Current participation in another clinical trial of a drug or device or past participation within 4 weeks before Baseline or participant is in exclusion period from a previous clinical trial

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
CD11301 Gel 0.03%	CD11301 0.03%	Participants applied 0.03% CD11301 gel (up to 500 mg per dose) topically once daily, 3 to 5 times per week, for cycle 1 and 2 i.e. 24 weeks.
Placebo	Placebo	Participants applied placebo gel during cycle one followed by 0.03% CD11301 gel topically during cycle two once daily, 3 to 5 times per week, for 24 weeks.
Placebo	CD11301 0.03%	Participants applied placebo gel during cycle one followed by 0.03% CD11301 gel topically during cycle two once daily, 3 to 5 times per week, for 24 weeks.
CD11301 Gel 0.06%	CD11301 0.06%	Participants applied 0.06% CD11301 gel (up to 500 mg per dose) topically once daily, 3 to 5 times per week, for cycle 1 and 2 i.e. 24 weeks.

Primary Outcome Measures

Name	Time	Method
Number of Participants Reported Overall Response (Complete and Partial) of Target Treated Lesions Based on Modified Composite Assessment of Index Lesion Severity (mCAILS) Score at Week 12	Week 12	Overall response is defined as the number of participants that achieved a complete response (CR) or partial response (PR) as assessed by mCAILS. The mCAILS assessment total was derived from components collected on the case report form (CRF). Target treated lesions (1-5 lesions) were rated in erythema (0-8, where 0=no evidence and 8= very severe), scaling (0-8, where 0=no evidence and 8= very severe), plaque elevation (0-3, where 0=no evidence and 3= marked elevation), and size (scale=0-18, where 0= no measurable area and 18= size of lesion \>300 centimeter \[cm\]\^2). These 4 ratings were summed to create subtotals, 1 per lesion. The final mCAILS assessment score was the sum of these subtotals. Total summation Score: 0-50 where higher score indicated higher severity. Complete response is defined as a 100% decrease from baseline i.e. score of '0' on the mCAILS scale. Partial response is defined as at least a 50%, but less than 100%, decrease from baseline.

Secondary Outcome Measures

Name	Time	Method
Number of Participants Reported Overall Response (OR) of Target Treated Lesions Based on Modified Severity-Weighted Assessment Tool (mSWAT) Score at Week 12	Week 12	OR is defined as the number of participants that achieved a complete response or partial response as assessed by mSWAT. mSWAT composite score involved the direct assessment of the BSA of each type of lesion (palm plus fingers of the participant= approximately 1% BSA) in each of 12 areas (Head, Neck, Anterior trunk, Arms, Forearms, Hands, Posterior trunk, Buttocks, Thighs, Legs, Feet, Groin) of the body, multiplying the sum of the BSA of each lesion type by a weighting factor (patch = 1, plaque = 2, and tumor = 3 or 4) and generating a sum of the subtotals of each lesion subtype. mSWAT score (0=no lesions; 400= lesions covering all areas). Complete response is defined as a 100% decrease from baseline. Partial response is defined as at least a 50%, but less than 100%, decrease from baseline, and with a tumor subscore of zero (no tumor).
Duration of Overall Response (Complete Response or Partial Response) Based on mCAILS Score	Up to Week 36	The duration of overall response (complete or partial) of the target treated lesions based on the mCAILS score was calculated in days as: (date of first non-response after responding) - (date of response) + 1. The mCAILS assessment total was derived from components collected on the case report form (CRF). Target treated lesions (1-5 lesions) were rated in erythema (0-8, where 0=no evidence and 8= very severe), scaling (0-8, where 0=no evidence and 8= very severe), plaque elevation (0-3, where 0=no evidence and 3= marked elevation), and size (scale=0-18, where 0= no measurable area and 18= size of lesion \>300 centimeter \[cm\]\^2). These 4 ratings were summed to create subtotals, 1 per lesion. The final mCAILS assessment score was the sum of these subtotals. Total summation Score: 0-50 where higher score indicated higher severity.
Time to Participant's First Overall Response (Complete or Partial) of the Target Treated Lesions Based on the mCAILS Score	Up to Week 36	Time to overall response (CR or PR) is the number of days from the start of drug application to the first documentation of objective response assessed by mCAILS Score. The 25th, 50th, and 75th percentiles were presented along with 95% confidence intervals using the log-log transformation. The mCAILS assessment total was derived from components collected on the case report form (CRF). Target treated lesions (1-5 lesions) were rated in erythema (0-8, where 0=no evidence and 8= very severe), scaling (0-8, where 0=no evidence and 8= very severe), plaque elevation (0-3, where 0=no evidence and 3= marked elevation), and size (scale=0-18, where 0= no measurable area and 18= size of lesion \>300 centimeter \[cm\]\^2). These 4 ratings were summed to create subtotals, 1 per lesion. The final mCAILS assessment score was the sum of these subtotals. Total summation Score: 0-50 where higher score indicated higher severity.
Time to Progressive Disease Using mSWAT	Up to Week 36	Progressive disease is defined as ≥ 25% increase in skin disease from baseline, or loss of response: in those with CR or PR, increase of skin score of greater than the sum of nadir plus 50% baseline score, Nadir is defined as the lowest skin score (best response).
Change From Baseline in Skindex-29 Survey Results at Week 12, 24 and 36	Week 12, 24 and Follow up (Week 36)	Participants answered 30 questions as part of the Skindex-29 survey. A composite score and 3 sub scores were calculated from the results. Item 18 of the survey was not used in any scoring. First, answers to each item were given a numeric value: Never = 0; Rarely = 25; Sometimes = 50; Often = 75; All the time = 100. The items used to calculate each subscore were: Emotions: 3, 6, 9, 12, 13, 15, 21, 23, 26, and 28 (10 items), Symptoms: 1, 7, 10, 16, 19, 24, and 27 (7 items), Functioning: 2, 4, 5, 8, 11, 14, 17, 20, 22, 25, 29, and 30 (12 items). The composite score is the average of the 3 sub scores ranging from 0 (no effect)-100 (maximum effect), higher score corresponds to lower quality of life.