Trial Outcomes & Findings for Topical Resiquimod for the Treatment of Early Stage Cutaneous T Cell Lymphoma (CTCL) (NCT NCT01676831)
NCT ID: NCT01676831
Last Updated: 2019-08-13
Results Overview
After four subjects have completed at least four weeks of study drug dosing a safety review meeting will be conducted by a safety review committee. No subjects will be enrolled in the next concentration (0.03%)group until all eight have been evaluated in the 0.06% group. The safety review committee reviews all patient data including adverse events to indicate whether the patient can escalate to the highest dose.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
13 participants
Primary outcome timeframe
after 4 subjects have completed 4 weeks of study drug
Results posted on
2019-08-13
Participant Flow
Participant milestones
| Measure |
Topical Resiquimod 0.06%
Topical resiquimod 0.06% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will be 3 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.
Topical resiquimod 0.06%: topical resiquimod 0.06% dosing frequency begins at 3 times per week and evaluated every two weeks. Will be applied for a total of 8 weeks followed by 4 weeks rest and then followed by another 8 weeks of application with another 4 weeks rest.
|
Topical Resiquimod 0.03%
Topical resiquimod 0.03% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will begin 5 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.
topical resiquimod 0.03%: topical resiquimod 0.03% applied initially 5 times weekly for 8 weeks with adjustments up or down based upon tolerability followed by 4 weeks rest followed by another 8 weeks of treatment followed by another 4 week rest period.
|
|---|---|---|
|
Overall Study
STARTED
|
8
|
5
|
|
Overall Study
COMPLETED
|
8
|
4
|
|
Overall Study
NOT COMPLETED
|
0
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Topical Resiquimod for the Treatment of Early Stage Cutaneous T Cell Lymphoma (CTCL)
Baseline characteristics by cohort
| Measure |
Topical Resiquimod 0.06%
n=8 Participants
Topical resiquimod 0.06% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will be 3 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.
Topical resiquimod 0.06%: topical resiquimod 0.06% dosing frequency begins at 3 times per week and evaluated every two weeks. Will be applied for a total of 8 weeks followed by 4 weeks rest and then followed by another 8 weeks of application with another 4 weeks rest.
|
Topical Resiquimod 0.03%
n=5 Participants
Topical resiquimod 0.03% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will begin 5 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.
topical resiquimod 0.03%: topical resiquimod 0.03% applied initially 5 times weekly for 8 weeks with adjustments up or down based upon tolerability followed by 4 weeks rest followed by another 8 weeks of treatment followed by another 4 week rest period.
|
Total
n=13 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
8 Participants
n=93 Participants
|
5 Participants
n=4 Participants
|
13 Participants
n=27 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=93 Participants
|
3 Participants
n=4 Participants
|
4 Participants
n=27 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=93 Participants
|
2 Participants
n=4 Participants
|
9 Participants
n=27 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
8 Participants
n=93 Participants
|
5 Participants
n=4 Participants
|
13 Participants
n=27 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
3 Participants
n=27 Participants
|
|
Race (NIH/OMB)
White
|
5 Participants
n=93 Participants
|
4 Participants
n=4 Participants
|
9 Participants
n=27 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Region of Enrollment
United States
|
8 participants
n=93 Participants
|
5 participants
n=4 Participants
|
13 participants
n=27 Participants
|
PRIMARY outcome
Timeframe: after 4 subjects have completed 4 weeks of study drugAfter four subjects have completed at least four weeks of study drug dosing a safety review meeting will be conducted by a safety review committee. No subjects will be enrolled in the next concentration (0.03%)group until all eight have been evaluated in the 0.06% group. The safety review committee reviews all patient data including adverse events to indicate whether the patient can escalate to the highest dose.
Outcome measures
| Measure |
Topical Resiquimod 0.06%
n=8 Participants
Topical resiquimod 0.06% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will be 3 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.
Topical resiquimod 0.06%: topical resiquimod 0.06% dosing frequency begins at 3 times per week and evaluated every two weeks. Will be applied for a total of 8 weeks followed by 4 weeks rest and then followed by another 8 weeks of application with another 4 weeks rest.
|
Topical Resiquimod 0.03%
n=4 Participants
Topical resiquimod 0.03% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will begin 5 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.
topical resiquimod 0.03%: topical resiquimod 0.03% applied initially 5 times weekly for 8 weeks with adjustments up or down based upon tolerability followed by 4 weeks rest followed by another 8 weeks of treatment followed by another 4 week rest period.
|
|---|---|---|
|
The Number of Participants That Tolerated the Maximum Drug Dose
|
8 Participants
|
4 Participants
|
SECONDARY outcome
Timeframe: Up to 24 weeks or At the conclusion of patient therapyThe secondary endpoint is: • The Response Rate (Complete or Partial Response) based on Composite Assessment of Index Lesions Disease Severity (CAILDS) score at EOS for the baseline target lesions. Complete Response is defined as a score of '0' on the CAILDS scale. Partial response is defined as a reduction of at least 50% in the CAILDS. The outcome measure will indicate how many received complete response (CR), partial response (PR), and stable disease (SD).
Outcome measures
| Measure |
Topical Resiquimod 0.06%
n=8 Participants
Topical resiquimod 0.06% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will be 3 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.
Topical resiquimod 0.06%: topical resiquimod 0.06% dosing frequency begins at 3 times per week and evaluated every two weeks. Will be applied for a total of 8 weeks followed by 4 weeks rest and then followed by another 8 weeks of application with another 4 weeks rest.
|
Topical Resiquimod 0.03%
n=4 Participants
Topical resiquimod 0.03% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will begin 5 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.
topical resiquimod 0.03%: topical resiquimod 0.03% applied initially 5 times weekly for 8 weeks with adjustments up or down based upon tolerability followed by 4 weeks rest followed by another 8 weeks of treatment followed by another 4 week rest period.
|
|---|---|---|
|
Secondary End Points: Efficacy- CAILDS SCORE
Complete Response (CR)
|
4 Participants
|
0 Participants
|
|
Secondary End Points: Efficacy- CAILDS SCORE
Partial Response (PR)
|
3 Participants
|
2 Participants
|
|
Secondary End Points: Efficacy- CAILDS SCORE
Stable Disease (SD)
|
1 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: Up to 24 weeks or At the conclusion of patient therapyThe secondary endpoint is: The total surface area of involvement will also be assessed (SWAT score). A partial response will represent improvement in 50% or greater of the skin surface area with regression of lesions. A complete response will represent complete clear clearing of skin lesions and in the case of stage IIA patients, resolution of lymphadenopathy. The outcome measure will indicate how many received complete response (CR), partial response (PR), and stable disease (SD).
Outcome measures
| Measure |
Topical Resiquimod 0.06%
n=8 Participants
Topical resiquimod 0.06% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will be 3 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.
Topical resiquimod 0.06%: topical resiquimod 0.06% dosing frequency begins at 3 times per week and evaluated every two weeks. Will be applied for a total of 8 weeks followed by 4 weeks rest and then followed by another 8 weeks of application with another 4 weeks rest.
|
Topical Resiquimod 0.03%
n=4 Participants
Topical resiquimod 0.03% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will begin 5 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.
topical resiquimod 0.03%: topical resiquimod 0.03% applied initially 5 times weekly for 8 weeks with adjustments up or down based upon tolerability followed by 4 weeks rest followed by another 8 weeks of treatment followed by another 4 week rest period.
|
|---|---|---|
|
Secondary End Points: Efficacy- SWAT SCORE
Complete Response (CR)
|
2 Participants
|
0 Participants
|
|
Secondary End Points: Efficacy- SWAT SCORE
Partial Response (PR)
|
5 Participants
|
2 Participants
|
|
Secondary End Points: Efficacy- SWAT SCORE
Stable Disease (SD)
|
1 Participants
|
2 Participants
|
Adverse Events
Topical Resiquimod 0.06%
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
Topical Resiquimod 0.03%
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Topical Resiquimod 0.06%
n=8 participants at risk
Topical resiquimod 0.06% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will be 3 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.
Topical resiquimod 0.06%: topical resiquimod 0.06% dosing frequency begins at 3 times per week and evaluated every two weeks. Will be applied for a total of 8 weeks followed by 4 weeks rest and then followed by another 8 weeks of application with another 4 weeks rest.
|
Topical Resiquimod 0.03%
n=5 participants at risk
Topical resiquimod 0.03% will be applied in dosing frequencies that are periodically adjusted to tolerability. Dosing frequency will begin 5 times a week. The dosing frequency may be adjusted (1,2,3,5,or 7times per week) based on the physician assessment of tolerability. Treatment will occur for 8 weeks followed by 4 weeks rest followed by another 8 weeks of treatment with 4 weeks rest. At 24 weeks a final evaluation will be performed. Those with a partial response at week 24 will have the option to continue therapy for up to another 12 weeks.
topical resiquimod 0.03%: topical resiquimod 0.03% applied initially 5 times weekly for 8 weeks with adjustments up or down based upon tolerability followed by 4 weeks rest followed by another 8 weeks of treatment followed by another 4 week rest period.
13 patients were consented and enrolled. 1 patient dropped out of the clinical study this patient was in the 0.03% arm of the study. As such 5 patients were measured for adverse events
|
|---|---|---|
|
Immune system disorders
fever
|
25.0%
2/8 • Number of events 2 • through study completion (3years)
Patient was assessed at each study visit.
|
0.00%
0/5 • through study completion (3years)
Patient was assessed at each study visit.
|
|
Skin and subcutaneous tissue disorders
skin erosion
|
50.0%
4/8 • Number of events 4 • through study completion (3years)
Patient was assessed at each study visit.
|
0.00%
0/5 • through study completion (3years)
Patient was assessed at each study visit.
|
|
General disorders
headache
|
25.0%
2/8 • Number of events 2 • through study completion (3years)
Patient was assessed at each study visit.
|
0.00%
0/5 • through study completion (3years)
Patient was assessed at each study visit.
|
|
Skin and subcutaneous tissue disorders
skin irritation
|
12.5%
1/8 • Number of events 1 • through study completion (3years)
Patient was assessed at each study visit.
|
40.0%
2/5 • Number of events 2 • through study completion (3years)
Patient was assessed at each study visit.
|
|
General disorders
myalgia
|
12.5%
1/8 • Number of events 1 • through study completion (3years)
Patient was assessed at each study visit.
|
0.00%
0/5 • through study completion (3years)
Patient was assessed at each study visit.
|
Additional Information
Study Coordinator
Hospital of the University of Pennsylvania
Phone: 215-662-6722
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place