Cutaneous Lupus ErythematosusEarly Phase 1NCT01841619
IVIg Efficacy Study to Treat Cutaneous Lupus Erythematosus
A Early Phase 1 study testing IVIg in people with Cutaneous Lupus Erythematosus, run by University of California, Irvine at 2 sites in United States.
Trial summary
The purpose of this research study is to show that non-steroidal treatment with intravenous immunoglobulin (IVIg) can replace current systemic immunosuppressive therapy in cutaneous lupus erythematosis (CLE) patients. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cutaneous Lupus Erythematosus
- What is being tested?
- IVIg
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 2 sitesUnited States
- Study dates
- Mar 2013to Nov 2014
- Phase
- Early Phase 1
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Evidence of a personally signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the trial.
- Subjects who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
- Be at least 18 years of age at time of informed consent.
- Have had a diagnosis of CLE
- Currently has active CLE (any subtype) established by standard clinical and histo- and immunopathologic criteria
- Falls into one of the two following cohorts:
- Cohort 1 - Has received a standard systemic therapy without a therapeutic response for a minimum of one month
- Cohort 2 - Has not received any systemic treatment
Who cannot join
- Subject is not over 18 years of age.
- Subject cannot understand or follow directions.
- Subject is a female of child-bearing potential and unwilling to use a form of highly effective birth control.
- Subject is pregnant, planning to get pregnant, or breast feeding.
- Subject has a known history of immunoglobulin A (IgA) deficiency.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
The Institute for Clinical and Translational Science (ICTS)
Irvine, California, 92697
Unversity of California, Irvine Healthcare, Dermatology, Gottschalk Medical Plaza
Irvine, California, 92697
About this study
Official title: Proof-of-Concept Study of IVIg Efficacy in Patients With Cutaneous Lupus Erythematosus
The purpose of this research study is to show that non-steroidal treatment with intravenous immunoglobulin (IVIg) can replace current systemic immunosuppressive therapy in cutaneous lupus erythematosis (CLE) patients.
Read the detailed description
The ultimate goal of this pilot project is to generate proof-of-concept data showing that treatment with intravenous immunoglobulin (IVIg) can replace current systemic immunosuppressive therapy in cutaneous lupus erythematosis (CLE) patients. This project has relevant clinical implications due to the severe side effects of and lack of response to current therapies.
From the review of literature, it can be postulated that:
- the beneficial effects of IVIg for patients with CLE should be prompt, with marked improvement within a few weeks;
- clinical improvement should last several weeks after the last infusion; and
- remission may be prolonged by maintenance IVIg therapy.
Although this is only a non-controlled study, the investigator expects that IVIg will improve CLE, including those resistant to standard treatments. It is anticipated that treatment with IVIg will facilitate healing of extensive cutaneous lesions and achieve rapid remission. Maintenance therapy with repeated monthly pulses of IVIg is expected to keep the disease in remission during the treatment-free follow up observational period. The results will provide the basis a multicenter randomized controlled study to identify which CLE subsets will benefit the most and which protocol will provide the optimal clinical outcome.
- Conditions
- Cutaneous Lupus Erythematosus
- Being tested
- IVIg
- Keywords
- skin, lupus, intravenous immunoglobulin, ivig
- Medical subject headings
- Lupus Erythematosus, Cutaneous
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IVIgDrug
Also called Intravenous Immunoglobin
All enrolled subjects will receive IVIg treatment following the protocol that proved to be efficacious in the treatment of patients with autoimmune blistering diseases as well as some patients with CLE. The drug will be administered at 500 mg/kg/day on consecutive days up to a total of 2 g/kg/month for 3 months in the Institute for Clinical and Translational Science (ICTS) at University of California, Irvine. After 3 months of treatment, IVIg will be discontinued and the subjects will be monitored for additional 6 months for a possible relapse. In the case of relapse, which is expected to occur in <25% subjects, the subjects will be re-treated by the standard protocol.
| Study group | Role | What they receive |
|---|---|---|
| IVIg as a monotherapy | Active comparator | IVIg will be used as a first line treatment. Topical treatment will be stopped at the beginning of IVIg therapy. Receives: Drug: IVIg |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Activity Score (CLASI - TAS)
Disease activity will be measured using the CLASI activity score that describes the activity of the disease. This score ranges from 0-70, with higher scores indicating more severe skin disease. This clinical assessment tool enables standardized assessments of response to therapy.
Results expressed relative to the mean initial value of all patients, taken as 100%. Each proceeding visit will be relative to the initial visit. A decrease in percentage represents improvement while an increase in percentage indicates worsening of CLE. Visits occur on a month-to-month basis.
Measured: Initial, 1st Visit - 9th Visit
Skindex 29
The subjects also evaluated their skin-specific quality of life with the Skindex-29 - the questionnaire consisting of 29 items used to calculate three subscales: symptoms (pain, itch, burning, sensitivity), emotions (depression, anxiety, embarrassment, anger) and functioning (sleep, relationships with others). All assessments were repeated at all study visits. Results expressed relative to the mean initial value of all patients, taken as 100%. Each proceeding visit will be relative to the initial visit. A decrease in percentage represents improvement while an increase in percentage indicates worsening of CLE. Visits occur on a month-to-month basis.
Measured: Initial, 1st Visit - 9th Visit
Secondary outcomes (3)
Mean Percent Change in Physician's Subjective Assessment of Improvement (PSAI)
At clinic visits, the investigator will categorize the change in disease activity in each patient as improved, unchanged, or worse since the last visit. Estimated change in disease activity will be based on the investigator's subjective assessment of the patient's skin disease.
Results expressed relative to the mean initial value of all patients, taken as 100%. Each proceeding visit will be relative to the initial visit. A decrease in percentage represents improvement while an increase in percentage indicates worsening of CLE. Visits occur on a month-to-month basis.
Measured: Initial, 1st Visit - 9th Visit
Mean Percent Change in Physician's Subjective Assessment of Severity (PSAS)
At clinic visits, the investigator will categorize the change in disease activity in each patient as improved, unchanged, or worse since the last visit. Estimated change in disease activity will be based on the investigator's subjective assessment of the patient's skin disease.
Results expressed relative to the mean initial value of all patients, taken as 100%. Each proceeding visit will be relative to the initial visit. A decrease in percentage represents improvement while an increase in percentage indicates worsening of CLE. Visits occur on a month-to-month basis.
Measured: Initial, 1st Visit - 9th Visit
Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Damage Score (CLASI - TDS)
Disease activity will be measured using the CLASI activity score that describes the damage of the disease. This score ranges from 0-70, with higher scores indicating more severe skin disease. This clinical assessment tool enables standardized assessments of response to therapy.
Results expressed relative to the mean initial value of all patients, taken as 100%. Each proceeding visit will be relative to the initial visit. A decrease in percentage represents improvement while an increase in percentage indicates worsening of CLE. Visits occur on a month-to-month basis.
All patients were measured identically in all visits.
Measured: Initial, 1st Visit - 9th Visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Apr 2013 (estimated)
- Study startMar 2013
- Primary completionNov 2014
- Study completionNov 2014
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 Cutaneous Lupus ErythematosusStudies of the same condition, open and under way.
- More trials run by University of California, IrvineThe 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, Irvine
- Sponsor type
- University, hospital or other
- Collaborators
- Grifols Therapeutics LLC
- Responsible party
- Principal investigator, Sergei Grando, University of California, Irvine
- Investigators
- Sergei Grando, MD PhD D.Sc., Principal investigator, University of California, Irvine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01841619
- Sponsor's protocol ID
- 2013-9351
- Status last verified
- Mar 2015
- First submitted
- 21 Apr 2013
- Last updated
- 10 Aug 2015
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 10 Aug 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Proof-of-Concept Study of IVIg Efficacy in Patients With Cutaneous Lupus Erythematosus
- ClinicalTrials.gov ID
- NCT01841619
- Acronym / sponsor ID
- 2013-9351
- Conditions
- Cutaneous Lupus Erythematosus
- Keywords
- skin, lupus, intravenous immunoglobulin, ivig
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- University of California, IrvineUniversity, hospital or other
- Collaborators
- Grifols Therapeutics LLC
- Study start
- Mar 2013
- Primary completion
- Nov 2014 (actual)
- Study completion
- Nov 2014 (actual)
- First posted
- 26 Apr 2013 (estimated)
- Results
- Posted 10 Aug 2015
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 research study is to show that non-steroidal treatment with intravenous immunoglobulin (IVIg) can replace current systemic immunosuppressive therapy in cutaneous lupus erythematosis (CLE) patients.
Detailed description
The ultimate goal of this pilot project is to generate proof-of-concept data showing that treatment with intravenous immunoglobulin (IVIg) can replace current systemic immunosuppressive therapy in cutaneous lupus erythematosis (CLE) patients. This project has relevant clinical implications due to the severe side effects of and lack of response to current therapies.
From the review of literature, it can be postulated that:
- the beneficial effects of IVIg for patients with CLE should be prompt, with marked improvement within a few weeks;
- clinical improvement should last several weeks after the last infusion; and
- remission may be prolonged by maintenance IVIg therapy.
Although this is only a non-controlled study, the investigator expects that IVIg will improve CLE, including those resistant to standard treatments. It is anticipated that treatment with IVIg will facilitate healing of extensive cutaneous lesions and achieve rapid remission. Maintenance therapy with repeated monthly pulses of IVIg is expected to keep the disease in remission during the treatment-free follow up observational period. The results will provide the basis a multicenter randomized controlled study to identify which CLE subsets will benefit the most and which protocol will provide the optimal clinical outcome.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| IVIg as a monotherapy | Active comparator | Drug: IVIgIVIg will be used as a first line treatment. Topical treatment will be stopped at the beginning of IVIg therapy. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Evidence of a personally signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the trial.
- Subjects who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
- Be at least 18 years of age at time of informed consent.
- Have had a diagnosis of CLE
- Currently has active CLE (any subtype) established by standard clinical and histo- and immunopathologic criteria
Key exclusion criteria (5 listed)
- Subject is not over 18 years of age.
- Subject cannot understand or follow directions.
- Subject is a female of child-bearing potential and unwilling to use a form of highly effective birth control.
- Subject is pregnant, planning to get pregnant, or breast feeding.
- Subject has a known history of immunoglobulin A (IgA) deficiency.
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 |
|---|---|---|
| IVIgAlso: Intravenous ImmunoglobinAll enrolled subjects will receive IVIg treatment following the protocol that proved to be efficacious in the treatment of patients with autoimmune blistering diseases as well as some patients with CLE. The drug will be administered at 500 mg/kg/day on consecutive days up to a total of 2 g/kg/month for 3 months in the Institute for Clinical and Translational Science (ICTS) at University of California, Irvine. After 3 months of treatment, IVIg will be discontinued and the subjects will be monitored for additional 6 months for a possible relapse. In the case of relapse, which is expected to occur in <25% subjects, the subjects will be re-treated by the standard protocol. | Drug | IVIg as a monotherapy |
Primary outcome
Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Activity Score (CLASI - TAS)
Time frame: Initial, 1st Visit - 9th Visit
Disease activity will be measured using the CLASI activity score that describes the activity of the disease. This score ranges from 0-70, with higher scores indicating more severe skin disease. This clinical assessment tool enables standardized assessments of response to therapy. Results expressed relative to the mean initial value of all patients, taken as 100%. Each proceeding visit will be relative to the initial visit. A decrease in percentage represents improvement while an increase in percentage indicates worsening of CLE. Visits occur on a month-to-month basis.
Skindex 29
Time frame: Initial, 1st Visit - 9th Visit
The subjects also evaluated their skin-specific quality of life with the Skindex-29 - the questionnaire consisting of 29 items used to calculate three subscales: symptoms (pain, itch, burning, sensitivity), emotions (depression, anxiety, embarrassment, anger) and functioning (sleep, relationships with others). All assessments were repeated at all study visits. Results expressed relative to the mean initial value of all patients, taken as 100%. Each proceeding visit will be relative to the initial visit. A decrease in percentage represents improvement while an increase in percentage indicates worsening of CLE. Visits occur on a month-to-month basis.
3 secondary outcomes
Mean Percent Change in Physician's Subjective Assessment of Improvement (PSAI)
Time frame: Initial, 1st Visit - 9th Visit
Mean Percent Change in Physician's Subjective Assessment of Severity (PSAS)
Time frame: Initial, 1st Visit - 9th Visit
Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Damage Score (CLASI - TDS)
Time frame: Initial, 1st Visit - 9th Visit
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Aug 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Cutaneous Lupus Erythematosus
Not yet recruitingPhase 2
This Study Will Evaluate the Effect and Safety of 626 in Patients With CLE
Cutaneous Lupus Erythematosus (CLE)
RecruitingPhase 3
A Study of Enpatoran in Participants With Cutaneous Manifestations of Lupus With or Without Systemic Disease
Systemic Lupus Erythematosus (SLE), Cutaneous Lupus Erythematosus (CLE)
RecruitingPhase 3
A Study of Enpatoran in Participants With Cutaneous Manifestations of Lupus With or Without Systemic Disease (ELOWEN-2)
Systematic Lupus Erythematosus, Cutaneous Lupus Erythematosus
Active, not recruitingPhase 2
A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Phase 2a Study With an Open-Label Extension Evaluating the Efficacy and Safety of VENT-03 in Adult Participants With Active Cutaneous Lupus Erythematosus With or Without Systemic Lupus Erythematosus
Cutaneous Lupus Erythematosus (CLE), Systemic Lupus Erythematosus
RecruitingPhase 2/3
Study of Afimetoran in Adults With Cutaneous Lupus Erythematosus
Cutaneous Lupus Erythematosus (CLE), Systematic Lupus Erythematosus
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.
Sergei Grando, MD PhD D.Sc. principal investigator
University of California, Irvine
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing IVIg in people with Cutaneous Lupus Erythematosus, run by University of California, Irvine at 2 sites 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 8 inclusion and 5 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 10 Aug 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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 phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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 10 Aug 2015). 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 Lupus Erythematosus Disease Area and Severity Index - Total Activity Score (CLASI - TAS) (time frame: Initial, 1st Visit - 9th Visit). The study lists 2 primary outcomes in all. 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 10 Aug 2015.