Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Celgene Corporation | INDUSTRY |
Not provided
Not provided
Not provided
Not provided
The purpose of this study is to determine the clinical and immunological effects of the phosphodiesterase type 4 inhibitor, CC-10004, on skin inflammation associated with cutaneous lupus erythematosus.
Discoid cutaneous lupus is the most common cutaneous manifestation of lupus erythematosus, a chronic, immune mediated disease of unknown etiology. The immune processes underlying cutaneous lupus remain largely unexplored, but recent evidence suggests a role for dendritic cells (DCs), type 1 interferons (IFN) and Th1-type immune processes. Treatment of cutaneous lupus remains limited primarily to anti-malarials, with thalidomide an effective secondary agent. However, side effects associated with these treatments are potentially problematic with chronic use. Phosphodiesterases (PDE) are critical enzymes that degrade cAMP. In particular, PDE type 4 (PDE4) activity is found in inflammatory and immune cells, including DCs. The immune modulator CC-10004 is a PDE4 inhibitor with demonstrated low toxicity in phase I and II clinical studies with potential efficacy in cutaneous lupus. CC-10004 is a well-tolerated, selective PDE4 inhibitor with demonstrated inhibitory effects on Th1-type cytokines and other inflammatory mediators and is under development for the treatment of inflammatory and immune mediated conditions. Prior studies include pilot trials in psoriasis and exercise-induced asthma, with results suggesting clinical efficacy in the former study. This open label, pilot study of 16 weeks duration will explore the clinical and immune-modulating effects of CC-10004 in 10 cutaneous discoid lupus patients. Patients meeting study criteria will receive the drug for 12 weeks, followed by a 4-week washout period. Study visit time points will include weeks 0, 1, 2, 4, 6, 8, 10, 12 and 16, during which we will measure outcomes for clinical, immunological and safety parameters. To investigate early immunological changes occurring in response to treatment, we will also perform skin punch biopsies of lesional sites at week 0 and week 4 for immunohistochemical and molecular analysis.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Apremilast | Experimental | CC-10004 20 mg twice daily by mouth for 12 weeks, followed by a 4 week washout period and final assessment |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CC-10004 | Drug | 20 mg twice daily by mouth for 12 weeks, followed by a 4 week washout period and final assessment |
|
| Measure | Description | Time Frame |
|---|---|---|
| Cutaneous LE Diseases Area and Severity Index (CLASI) Score Based on Extent of Symptoms | 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. | 16 Weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Dermatology Quality of Life Index (DQLI) |
| 16 Weeks |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Andrew G Franks, Jr., MD | NYU Langone Health | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| New York University Tisch Hospital | New York | New York | 10016 | United States |
Not provided
Not provided
| ID | Type | URL | Comment |
|---|---|---|---|
| PMID: 23134988 | Clinical Study Report | View IPD |
Not provided
Not provided
Not provided
Not provided
Not provided
Screen Fail subjects were excluded from participation in trial.
Drs. Franks/Oliver and other physician (attendings) would refer subjects to the unit if they were interested.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Apremilast | CC-10004 20 mg twice daily by mouth for 12 weeks, followed by a 4 week washout period and final assessment |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Must be male or female and aged ≥ 18 years at time of consent Must have a clinical exam consistent with discoid lupus erythematosus as follows: the presence of chronic cutaneous lesions without evidence of systemic disease of lupus
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Apremilast | 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 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Cutaneous LE Diseases Area and Severity Index (CLASI) Score Based on Extent of Symptoms | 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. | 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 | Posted | 16 Weeks |
|
2 years
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Apremilast | CC-10004 20 mg twice daily by mouth for 12 weeks, followed by a 4 week washout period and final assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Lichenoid Dermatitis | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nausea | Gastrointestinal disorders | Non-systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Andrew Franks, MD, Principal Investigator | New York University School of Medicine | 212-263-5244 | andrew.franks@nyumc.org |
Not provided
| ID | Term |
|---|---|
| D008179 | Lupus Erythematosus, Discoid |
| ID | Term |
|---|---|
| D008178 | Lupus Erythematosus, Cutaneous |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012871 | Skin Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C505730 | apremilast |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Common Terminology Criteria for Adverse Events v3.0 (CTCAE) |
| Weeks 1, 2, 4, 6, 8, 10, 12, 16 |
| Dermal and Circulating Blood Plasmacytoid Dendritic Cell Levels | Weeks 0, 4 (dermal and circulating); week 12 (circulating only) |
| Dermal and Circulating Blood T Regulatory Cell Levels |
| Weeks 0, 4 (dermal and blood); Week 12 (blood only) |
| Plasma Cytokine Levels |
| Weeks 0, 4, 12 |
Apremilast for discoid lupus erythematosus: results of a phase 2, open-label, single-arm, pilot study. De Souza A, Strober BE, Merola JF, Oliver S, Franks AG Jr. J Drugs Dermatol. 2012 Oct;11(10):1224-6. PMID: 23134988 |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| OG000 | Apremilast | CC-10004 20 mg twice daily by mouth for 12 weeks, followed by a 4 week washout period and final assessment |
|
| Secondary | Dermatology Quality of Life Index (DQLI) |
| Secondary outcome measures were not measured in this study. | Posted | 16 Weeks |
|
|
| Secondary | Common Terminology Criteria for Adverse Events v3.0 (CTCAE) |
| PI no longer with institution. No secondary outcome measure data available for reporting. | Posted | Weeks 1, 2, 4, 6, 8, 10, 12, 16 |
|
|
| Secondary | Dermal and Circulating Blood Plasmacytoid Dendritic Cell Levels | PI no longer with institution. No secondary outcome measure data available for reporting. | Posted | Weeks 0, 4 (dermal and circulating); week 12 (circulating only) |
|
|
| Secondary | Dermal and Circulating Blood T Regulatory Cell Levels |
| PI no longer with institution. No secondary outcome measure data available for reporting. | Posted | Weeks 0, 4 (dermal and blood); Week 12 (blood only) |
|
|
| Secondary | Plasma Cytokine Levels |
| PI no longer with institution. No secondary outcome measure data available for reporting. | Posted | Weeks 0, 4, 12 |
|
|
| 2 |
| 8 |
| 4 |
| 8 |
| Neuraopathy Sensory | Nervous system disorders | Non-systematic Assessment |
|
| Headache | Nervous system disorders | Non-systematic Assessment |
|
| Diarrhea | Gastrointestinal disorders | Non-systematic Assessment |
|
Not provided
Not provided