Trial Outcomes & Findings for A Safety, Efficacy and Pharmacokinetics Study of CD11301 for the Treatment of Cutaneous T-Cell Lymphoma (CTCL) (NCT NCT03292406)

This study was conducted at 3 countries (France, Germany, USA) between 19 Dec 2017 to 17 Mar 2020. A total of 86 participants were randomized to 1 of the 3 treatment groups (placebo gel or CD11301 gel 0.03% or 0.06%) in a 1:1:1 ratio.

This study consisted of 2 cycles: Cycle 1 and Cycle 2. Each treatment cycle consisted of 8 weeks on treatment followed by 4 weeks without treatment. Cycle 1: drug product was applied on up to 5 percent (%) body surface area (BSA) and 10% BSA in cycle 2.

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

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.

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).

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.

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.

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).

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.