Trial Outcomes & Findings for Safety Study of Clinical and Immune Effects of Phosphodiesterase 4 (PDE-4) Inhibitor in Cutaneous Lupus Patients (NCT NCT00708916)

Results Overview

To evaluate the clinical response of cutaneous lupus patients to CC-10004. From J Invest Dermatol. 2005 Nov; 125(5): 889-894.doi: 10.1111/j.0022-202X.2005.23889.x: The CLASI consists of two scores. One summarizes the activity of the disease on a scale from 0 to 30, higher score translates to more severe disease. The second is a measure of the damage done by the disease on a scale form 0 to 30, higher score translates to more severe disease. Activity is scored on the basis of erythema, scale/hyperkeratosis, mucous membrane involvement, acute hair loss and non-scarring alopecia. Damage is scored in terms of dyspigmentation and scarring, including scarring alopecia. Each score is reported separately. Outcome measures are reported for each time point for each subject due to the low numbers of enrollment.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

8 participants

Primary outcome timeframe

16 Weeks

Results posted on

2021-03-03

Participant Flow

Drs. Franks/Oliver and other physician (attendings) would refer subjects to the unit if they were interested.

Screen Fail subjects were excluded from participation in trial.

Participant milestones

Participant milestones
Measure	Apremilast CC-10004 20 mg twice daily by mouth for 12 weeks, followed by a 4 week washout period and final assessment
Overall Study STARTED	12
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Reasons for withdrawal
Measure	Apremilast CC-10004 20 mg twice daily by mouth for 12 weeks, followed by a 4 week washout period and final assessment
Overall Study Subject Noncompliance	2
Overall Study Adverse Event	2

Baseline Characteristics

Safety Study of Clinical and Immune Effects of Phosphodiesterase 4 (PDE-4) Inhibitor in Cutaneous Lupus Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Apremilast n=8 Participants CC-10004 20 mg twice daily by mouth for 12 weeks, followed by a 4 week washout period and final assessment CC-10004: 20 mg twice daily by mouth for 12 weeks, followed by a 4 week washout period and final assessment
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	8 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants
Region of Enrollment United States	8 participants n=5 Participants

PRIMARY outcome

Timeframe: 16 Weeks

Population: The PI and study team has left the institution. Efforts were made to contact the PI/study team members, but were unsuccessful. No study data for primary outcome measure are available

To evaluate the clinical response of cutaneous lupus patients to CC-10004. From J Invest Dermatol. 2005 Nov; 125(5): 889-894.doi: 10.1111/j.0022-202X.2005.23889.x: The CLASI consists of two scores. One summarizes the activity of the disease on a scale from 0 to 30, higher score translates to more severe disease. The second is a measure of the damage done by the disease on a scale form 0 to 30, higher score translates to more severe disease. Activity is scored on the basis of erythema, scale/hyperkeratosis, mucous membrane involvement, acute hair loss and non-scarring alopecia. Damage is scored in terms of dyspigmentation and scarring, including scarring alopecia. Each score is reported separately. Outcome measures are reported for each time point for each subject due to the low numbers of enrollment.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 16 Weeks

Population: Secondary outcome measures were not measured in this study.

1. To evaluate the clinical safety of CC-10004 in cutaneous lupus patients 2. To determine the effect of CC-10004 on immune parameters in the skin 3. To determine the effect of CC-10004 on immune parameters in the blood 1. in vivo 2. in vitro 4. To determine the effect of CC-10004 on QOL and psychological outcome measures

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Weeks 1, 2, 4, 6, 8, 10, 12, 16

Population: PI no longer with institution. No secondary outcome measure data available for reporting.

1. To evaluate the clinical safety of CC-10004 in cutaneous lupus patients 2. To determine the effect of CC-10004 on immune parameters in the skin 3. To determine the effect of CC-10004 on immune parameters in the blood 1. in vivo 2. in vitro 4. To determine the effect of CC-10004 on QOL and psychological outcome measures

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Weeks 0, 4 (dermal and circulating); week 12 (circulating only)

Population: PI no longer with institution. No secondary outcome measure data available for reporting.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Weeks 0, 4 (dermal and blood); Week 12 (blood only)

Population: PI no longer with institution. No secondary outcome measure data available for reporting.

1. To evaluate the clinical safety of CC-10004 in cutaneous lupus patients 2. To determine the effect of CC-10004 on immune parameters in the skin 3. To determine the effect of CC-10004 on immune parameters in the blood 1. in vivo 2. in vitro 4. To determine the effect of CC-10004 on QOL and psychological outcome measures

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Weeks 0, 4, 12

Population: PI no longer with institution. No secondary outcome measure data available for reporting.

1. To evaluate the clinical safety of CC-10004 in cutaneous lupus patients 2. To determine the effect of CC-10004 on immune parameters in the skin 3. To determine the effect of CC-10004 on immune parameters in the blood 1. in vivo 2. in vitro 4. To determine the effect of CC-10004 on QOL and psychological outcome measures

Outcome measures

Outcome data not reported

Adverse Events

Apremilast

Serious events: 2 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Apremilast n=8 participants at risk CC-10004 20 mg twice daily by mouth for 12 weeks, followed by a 4 week washout period and final assessment
Skin and subcutaneous tissue disorders Lichenoid Dermatitis	12.5% 1/8 • Number of events 1 • 2 years
Nervous system disorders Neuraopathy Sensory	12.5% 1/8 • Number of events 1 • 2 years

Other adverse events

Other adverse events
Measure	Apremilast n=8 participants at risk CC-10004 20 mg twice daily by mouth for 12 weeks, followed by a 4 week washout period and final assessment
Gastrointestinal disorders Nausea	50.0% 4/8 • Number of events 4 • 2 years
Nervous system disorders Headache	25.0% 2/8 • Number of events 2 • 2 years
Gastrointestinal disorders Diarrhea	12.5% 1/8 • Number of events 1 • 2 years

Additional Information

Andrew Franks, MD, Principal Investigator

New York University School of Medicine

Phone: 212-263-5244

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place