IVIg Efficacy Study to Treat Cutaneous Lupus Erythematosus

Proof-of-Concept Study of IVIg Efficacy in Patients With Cutaneous Lupus Erythematosus

Status

Completed

Phases

Early Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01841619

Enrollment

Registered

2013-04-26

Start date

2013-03-31

Completion date

2014-11-30

Last updated

2015-08-10

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Cutaneous Lupus Erythematosus

Keywords

skin, lupus, intravenous immunoglobulin, ivig

Brief 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.

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: 1. the beneficial effects of IVIg for patients with CLE should be prompt, with marked improvement within a few weeks; 2. clinical improvement should last several weeks after the last infusion; and 3. 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.

Interventions

DRUGIVIg

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 design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

* 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

Exclusion criteria

* 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.

Design outcomes

Primary

Measure	Time frame	Description
Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Activity Score (CLASI - TAS)	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	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.

Secondary

Measure	Time frame	Description
Mean Percent Change in Physician's Subjective Assessment of Improvement (PSAI)	Initial, 1st Visit - 9th Visit	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.
Mean Percent Change in Physician's Subjective Assessment of Severity (PSAS)	Initial, 1st Visit - 9th Visit	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.
Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Damage Score (CLASI - TDS)	Initial, 1st Visit - 9th Visit	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.

Countries

United States

Participant flow

Participants by arm

Arm	Count
IVIg as a Monotherapy IVIg will be used as a first line treatment. Topical treatment will be stopped at the beginning of IVIg therapy. IVIg: 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.	16
Total	16

Arm

Count

IVIg as a Monotherapy

IVIg will be used as a first line treatment. Topical treatment will be stopped at the beginning of IVIg therapy. IVIg: 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.

Total

Baseline characteristics

Characteristic	IVIg as a Monotherapy
Age, Categorical <=18 years	0 Participants
Age, Categorical >=65 years	1 Participants
Age, Categorical Between 18 and 65 years	15 Participants
Sex: Female, Male Female	15 Participants
Sex: Female, Male Male	1 Participants

Adverse events

Event type	EG000 affected / at risk
deaths Total, all-cause mortality	— / —
other Total, other adverse events	9 / 16
serious Total, serious adverse events	0 / 16

Outcome results

Primary

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.