Trial Outcomes & Findings for Intratumoral Injections of LL37 for Melanoma (NCT NCT02225366)

Last Updated: 2021-12-09

Results Overview

Dose Limiting Toxicity defined as: a. Any grade 3 or 4 non-hematologic toxicity regardless of duration, except: Grade 3 skin reactions at injection sites - Grade 3 fever b. Grade 4 thrombocytopenia c. Grade 4 neutropenia lasting \>2 weeks or associated with infection.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

4 participants

Primary outcome timeframe

once a week, up to 8 weeks duration

Results posted on

2021-12-09

Participant Flow

Participant milestones

Participant milestones
Measure	Cohort 1 This cohort received starting dose of 250 ug/tumor injection once a week	Cohort 2 This cohort received 500 ug/tumor injection once a week
Overall Study STARTED	2	1
Overall Study COMPLETED	1	1
Overall Study NOT COMPLETED	1	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Cohort 1 This cohort received starting dose of 250 ug/tumor injection once a week	Cohort 2 This cohort received 500 ug/tumor injection once a week
Overall Study Lack of Efficacy	1	0

Baseline Characteristics

Intratumoral Injections of LL37 for Melanoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cohort 1 n=2 Participants This cohort received starting dose of 250 ug/tumor injection once a week	Cohort 2 n=1 Participants This cohort received 500 ug/tumor injection once a week	Total n=3 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	2 participants n=5 Participants	1 participants n=7 Participants	3 participants n=5 Participants

PRIMARY outcome

Timeframe: once a week, up to 8 weeks duration

Outcome measures

Outcome measures
Measure	Cohort 1 n=2 Participants This cohort received starting dose of 250 ug/tumor injection once a week	Cohort 2 n=1 Participants This cohort received 500 ug/tumor injection once a week
Number of Participants With Optimal Biological Dose (OBD) of LL37 Based Upon Toxicity	2 Participants	1 Participants

SECONDARY outcome

Timeframe: Radiologic evaluations in the form of CT scans of affected disease sites will be performed every 8 weeks (+/- 14 days) while on study, up to 1 year

Response defined as experiencing either an immune-related complete or partial response (irCR or irPR), and the association between response and disease characteristics and T-cell responses will be assessed using logistic regression.

Outcome measures

Outcome measures
Measure	Cohort 1 n=2 Participants This cohort received starting dose of 250 ug/tumor injection once a week	Cohort 2 n=1 Participants This cohort received 500 ug/tumor injection once a week
Number of Participants With Antitumor Immune Response of Intra-Tumoral Injection of LL37	2 Participants	1 Participants

Adverse Events

Cohort 1

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Cohort 2

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Cohort 1 n=2 participants at risk This cohort received starting dose of 250 ug/tumor injection once a week	Cohort 2 n=1 participants at risk This cohort received 500 ug/tumor injection once a week
Skin and subcutaneous tissue disorders Squamous Cell Carcinoma	50.0% 1/2 • adverse events were collected every week at the time of the injections for up to 8 weeks, All-Cause Mortality was assessed up to 14 months	0.00% 0/1 • adverse events were collected every week at the time of the injections for up to 8 weeks, All-Cause Mortality was assessed up to 14 months
Skin and subcutaneous tissue disorders Actinic Keratosis	50.0% 1/2 • adverse events were collected every week at the time of the injections for up to 8 weeks, All-Cause Mortality was assessed up to 14 months	0.00% 0/1 • adverse events were collected every week at the time of the injections for up to 8 weeks, All-Cause Mortality was assessed up to 14 months
Blood and lymphatic system disorders Anemia	50.0% 1/2 • adverse events were collected every week at the time of the injections for up to 8 weeks, All-Cause Mortality was assessed up to 14 months	100.0% 1/1 • adverse events were collected every week at the time of the injections for up to 8 weeks, All-Cause Mortality was assessed up to 14 months
Skin and subcutaneous tissue disorders Skin hypopigmentation	50.0% 1/2 • adverse events were collected every week at the time of the injections for up to 8 weeks, All-Cause Mortality was assessed up to 14 months	0.00% 0/1 • adverse events were collected every week at the time of the injections for up to 8 weeks, All-Cause Mortality was assessed up to 14 months
Endocrine disorders Hypothyroidism	50.0% 1/2 • adverse events were collected every week at the time of the injections for up to 8 weeks, All-Cause Mortality was assessed up to 14 months	0.00% 0/1 • adverse events were collected every week at the time of the injections for up to 8 weeks, All-Cause Mortality was assessed up to 14 months
Blood and lymphatic system disorders Lymphocyte Count Decrease	0.00% 0/2 • adverse events were collected every week at the time of the injections for up to 8 weeks, All-Cause Mortality was assessed up to 14 months	100.0% 1/1 • adverse events were collected every week at the time of the injections for up to 8 weeks, All-Cause Mortality was assessed up to 14 months
Blood and lymphatic system disorders White blood cell decrease	0.00% 0/2 • adverse events were collected every week at the time of the injections for up to 8 weeks, All-Cause Mortality was assessed up to 14 months	100.0% 1/1 • adverse events were collected every week at the time of the injections for up to 8 weeks, All-Cause Mortality was assessed up to 14 months
Blood and lymphatic system disorders Blood bilirubin increase	0.00% 0/2 • adverse events were collected every week at the time of the injections for up to 8 weeks, All-Cause Mortality was assessed up to 14 months	100.0% 1/1 • adverse events were collected every week at the time of the injections for up to 8 weeks, All-Cause Mortality was assessed up to 14 months

Additional Information

Dr. Rodabe Amaria,MD, Associate Professor, Melanoma Medical Oncology

UT MD Anderson Cancer Center

Phone: (713) 792-2921

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place