Discoid Lupus ErythematosusPhase 1/2NCT00708916
Safety Study of Clinical and Immune Effects of Phosphodiesterase 4 (PDE-4) Inhibitor in Cutaneous Lupus Patients
A Phase 1/2 study testing CC-10004 in people with Discoid Lupus Erythematosus, run by NYU Langone Health at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Discoid Lupus Erythematosus
- What is being tested?
- CC-10004
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2008to Aug 2010
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of cutaneous discoid lupus by clinical and histopathological exam
Who cannot join
- Systemic lupus involving the internal organs
- Systemic vasculitis
- History of other clinically significant disease process
- History of HIV, hepatitis B or C
- Concurrent use of immune modulating therapy
- Evidence of incompletely treated tuberculosis
- Pregnant or lactating female
Trial locations and contacts
1 site in 1 country.
United States1 site
New York University Tisch Hospital
New York, New York, 10016
About this study
Official title: Clinical and Immune-modulating Effects of CC-10004 in Discoid Lupus Erythematosus
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.
Read the detailed description
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.
- Conditions
- Discoid Lupus Erythematosus
- Being tested
- CC-10004
- Keywords
- Cutaneous lupus, Intervention, Discoid lupus, Phosphodiesterase 5 inhibitor
- Medical subject headings
- Lupus Erythematosus, Discoid
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CC-10004Drug
Also called Apremilast
20 mg twice daily by mouth for 12 weeks, followed by a 4 week washout period and final assessment
| Study group | Role | What they receive |
|---|---|---|
| Apremilast | Experimental | CC-10004 20 mg twice daily by mouth for 12 weeks, followed by a 4 week washout period and final assessment Receives: Drug: CC-10004 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 16 Weeks
Secondary outcomes (5)
Dermatology Quality of Life Index (DQLI)
- To evaluate the clinical safety of CC-10004 in cutaneous lupus patients
- To determine the effect of CC-10004 on immune parameters in the skin
- To determine the effect of CC-10004 on immune parameters in the blood
- in vivo
- in vitro
- To determine the effect of CC-10004 on QOL and psychological outcome measures
Measured: 16 Weeks
Common Terminology Criteria for Adverse Events v3.0 (CTCAE)
- To evaluate the clinical safety of CC-10004 in cutaneous lupus patients
- To determine the effect of CC-10004 on immune parameters in the skin
- To determine the effect of CC-10004 on immune parameters in the blood
- in vivo
- in vitro
- To determine the effect of CC-10004 on QOL and psychological outcome measures
Measured: Weeks 1, 2, 4, 6, 8, 10, 12, 16
Dermal and Circulating Blood Plasmacytoid Dendritic Cell Levels
Measured: Weeks 0, 4 (dermal and circulating); week 12 (circulating only)
Dermal and Circulating Blood T Regulatory Cell Levels
- To evaluate the clinical safety of CC-10004 in cutaneous lupus patients
- To determine the effect of CC-10004 on immune parameters in the skin
- To determine the effect of CC-10004 on immune parameters in the blood
- in vivo
- in vitro
- To determine the effect of CC-10004 on QOL and psychological outcome measures
Measured: Weeks 0, 4 (dermal and blood); Week 12 (blood only)
Plasma Cytokine Levels
- To evaluate the clinical safety of CC-10004 in cutaneous lupus patients
- To determine the effect of CC-10004 on immune parameters in the skin
- To determine the effect of CC-10004 on immune parameters in the blood
- in vivo
- in vitro
- To determine the effect of CC-10004 on QOL and psychological outcome measures
Measured: Weeks 0, 4, 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Jul 2008 (estimated)
- Study startJun 2008
- Primary completionAug 2010
- Study completionAug 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Discoid Lupus ErythematosusStudies of the same condition, open and under way.
- More trials run by NYU Langone HealthThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- NYU Langone Health
- Sponsor type
- University, hospital or other
- Collaborators
- Celgene Corporation
- Responsible party
- Sponsor
- Investigators
- Andrew G Franks, Jr., MD, Principal investigator, NYU Langone Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00708916
- Sponsor's protocol ID
- 07-580
- Status last verified
- Feb 2021
- First submitted
- 1 Jul 2008
- Last updated
- 3 Mar 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical and Immune-modulating Effects of CC-10004 in Discoid Lupus Erythematosus
- ClinicalTrials.gov ID
- NCT00708916
- Acronym / sponsor ID
- 07-580
- Conditions
- Discoid Lupus Erythematosus
- Keywords
- Cutaneous lupus, Intervention, Discoid lupus, Phosphodiesterase 5 inhibitor
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- NYU Langone HealthUniversity, hospital or other
- Collaborators
- Celgene Corporation
- Study start
- Jun 2008
- Primary completion
- Aug 2010 (actual)
- Study completion
- Aug 2010 (actual)
- First posted
- 3 Jul 2008 (estimated)
- Results
- Posted 3 Mar 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Apremilast | Experimental | Drug: CC-10004CC-10004 20 mg twice daily by mouth for 12 weeks, followed by a 4 week washout period and final assessment |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- Diagnosis of cutaneous discoid lupus by clinical and histopathological exam
Key exclusion criteria (7 listed)
- Systemic lupus involving the internal organs
- Systemic vasculitis
- History of other clinically significant disease process
- History of HIV, hepatitis B or C
- Concurrent use of immune modulating therapy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CC-10004Also: Apremilast20 mg twice daily by mouth for 12 weeks, followed by a 4 week washout period and final assessment | Drug | Apremilast |
Primary outcome
Cutaneous LE Diseases Area and Severity Index (CLASI) Score Based on Extent of Symptoms
Time frame: 16 Weeks
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.
5 secondary outcomes
Dermatology Quality of Life Index (DQLI)
Time frame: 16 Weeks
Common Terminology Criteria for Adverse Events v3.0 (CTCAE)
Time frame: Weeks 1, 2, 4, 6, 8, 10, 12, 16
Dermal and Circulating Blood Plasmacytoid Dendritic Cell Levels
Time frame: Weeks 0, 4 (dermal and circulating); week 12 (circulating only)
Dermal and Circulating Blood T Regulatory Cell Levels
Time frame: Weeks 0, 4 (dermal and blood); Week 12 (blood only)
Plasma Cytokine Levels
Time frame: Weeks 0, 4, 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Mar 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
Related trials for Discoid Lupus Erythematosus
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A Study of Enpatoran in Participants With Cutaneous Manifestations of Lupus With or Without Systemic Disease (ELOWEN-2)
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Study of Afimetoran in Adults With Cutaneous Lupus Erythematosus
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A 2-Part Study to Learn Whether Litifilimab (BIIB059) Injections Can Improve Symptoms of Adult Participants Who Have Active Cutaneous Lupus Erythematosus
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Active, not recruitingPhase 2
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Andrew G Franks, Jr., MD principal investigator
NYU Langone Health
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing CC-10004 in people with Discoid Lupus Erythematosus, run by NYU Langone Health at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 3 Mar 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cutaneous LE Diseases Area and Severity Index (CLASI) Score Based on Extent of Symptoms (time frame: 16 Weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Mar 2021.