Trial Outcomes & Findings for IVIg Efficacy Study to Treat Cutaneous Lupus Erythematosus (NCT NCT01841619)

NCT ID: NCT01841619

Last Updated: 2015-08-10

Results Overview

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.

• Recruitment status: COMPLETED
• Study phase: EARLY_PHASE1
• Target enrollment: 16 participants
• Primary outcome timeframe: Initial, 1st Visit - 9th Visit
• Results posted on: 2015-08-10

Participant Flow

Participant milestones

Measure	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.
Overall Study STARTED	16
Overall Study COMPLETED	16
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

IVIg Efficacy Study to Treat Cutaneous Lupus Erythematosus

Baseline characteristics by cohort

Measure	IVIg as a Monotherapy n=16 Participants 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.
Age, Categorical <=18 years	0 Participants n=93 Participants
Age, Categorical Between 18 and 65 years	15 Participants n=93 Participants
Age, Categorical >=65 years	1 Participants n=93 Participants
Sex: Female, Male Female	15 Participants n=93 Participants
Sex: Female, Male Male	1 Participants n=93 Participants

PRIMARY outcome

Timeframe: 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.

Outcome measures

Measure	IVIg as a Monotherapy n=16 Participants 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.
Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Activity Score (CLASI - TAS) Initial	100 Percent of Baseline Standard Deviation 0
Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Activity Score (CLASI - TAS) 1st Visit	94 Percent of Baseline Standard Deviation 30
Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Activity Score (CLASI - TAS) 2nd Visit	84 Percent of Baseline Standard Deviation 29
Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Activity Score (CLASI - TAS) 3rd Visit	94 Percent of Baseline Standard Deviation 52
Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Activity Score (CLASI - TAS) 4th Visit	86 Percent of Baseline Standard Deviation 19
Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Activity Score (CLASI - TAS) 5th Visit	85 Percent of Baseline Standard Deviation 14
Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Activity Score (CLASI - TAS) 6th Visit	72 Percent of Baseline Standard Deviation 37
Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Activity Score (CLASI - TAS) 7th Visit	68 Percent of Baseline Standard Deviation 33
Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Activity Score (CLASI - TAS) 8th Visit	81 Percent of Baseline Standard Deviation 17
Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Activity Score (CLASI - TAS) 9th Visit	71 Percent of Baseline Standard Deviation 13

PRIMARY outcome

Timeframe: 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.

Outcome measures

Measure	IVIg as a Monotherapy n=16 Participants 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.
Skindex 29 Initial	100 Percent of Baseline Standard Deviation 0
Skindex 29 1st Visit	97 Percent of Baseline Standard Deviation 3
Skindex 29 2nd Visit	94 Percent of Baseline Standard Deviation 9
Skindex 29 3rd Visit	92 Percent of Baseline Standard Deviation 12
Skindex 29 4th Visit	90 Percent of Baseline Standard Deviation 15
Skindex 29 5th Visit	81 Percent of Baseline Standard Deviation 21
Skindex 29 6th Visit	79 Percent of Baseline Standard Deviation 23
Skindex 29 7th Visit	83 Percent of Baseline Standard Deviation 30
Skindex 29 8th Visit	99 Percent of Baseline Standard Deviation 11
Skindex 29 9th Visit	82 Percent of Baseline Standard Deviation 21

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	IVIg as a Monotherapy n=16 Participants 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.
Mean Percent Change in Physician's Subjective Assessment of Improvement (PSAI) Intial	100 Percent of Baseline Standard Deviation 0
Mean Percent Change in Physician's Subjective Assessment of Improvement (PSAI) 1st Visit	100 Percent of Baseline Standard Deviation 0
Mean Percent Change in Physician's Subjective Assessment of Improvement (PSAI) 2nd Visit	100 Percent of Baseline Standard Deviation 0
Mean Percent Change in Physician's Subjective Assessment of Improvement (PSAI) 3rd Visit	136 Percent of Baseline Standard Deviation 79
Mean Percent Change in Physician's Subjective Assessment of Improvement (PSAI) 4th Visit	104 Percent of Baseline Standard Deviation 48
Mean Percent Change in Physician's Subjective Assessment of Improvement (PSAI) 5th Visit	105 Percent of Baseline Standard Deviation 52
Mean Percent Change in Physician's Subjective Assessment of Improvement (PSAI) 6th Visit	117 Percent of Baseline Standard Deviation 37
Mean Percent Change in Physician's Subjective Assessment of Improvement (PSAI) 7th Visit	133 Percent of Baseline Standard Deviation 47
Mean Percent Change in Physician's Subjective Assessment of Improvement (PSAI) 8th Visit	150 Percent of Baseline Standard Deviation 77
Mean Percent Change in Physician's Subjective Assessment of Improvement (PSAI) 9th Visit	100 Percent of Baseline Standard Deviation 29

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	IVIg as a Monotherapy n=16 Participants 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.
Mean Percent Change in Physician's Subjective Assessment of Severity (PSAS) Initial	100 Percent of Baseline Standard Deviation 0
Mean Percent Change in Physician's Subjective Assessment of Severity (PSAS) 1st Visit	100 Percent of Baseline Standard Deviation 0
Mean Percent Change in Physician's Subjective Assessment of Severity (PSAS) 2nd Visit	98 Percent of Baseline Standard Deviation 8
Mean Percent Change in Physician's Subjective Assessment of Severity (PSAS) 3rd Visit	100 Percent of Baseline Standard Deviation 10
Mean Percent Change in Physician's Subjective Assessment of Severity (PSAS) 4th Visit	97 Percent of Baseline Standard Deviation 18
Mean Percent Change in Physician's Subjective Assessment of Severity (PSAS) 5th Visit	92 Percent of Baseline Standard Deviation 19
Mean Percent Change in Physician's Subjective Assessment of Severity (PSAS) 6th Visit	92 Percent of Baseline Standard Deviation 18
Mean Percent Change in Physician's Subjective Assessment of Severity (PSAS) 7th Visit	92 Percent of Baseline Standard Deviation 20
Mean Percent Change in Physician's Subjective Assessment of Severity (PSAS) 8th Visit	90 Percent of Baseline Standard Deviation 18
Mean Percent Change in Physician's Subjective Assessment of Severity (PSAS) 9th Visit	90 Percent of Baseline Standard Deviation 17

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	IVIg as a Monotherapy n=16 Participants 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.
Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Damage Score (CLASI - TDS) Initial	100 Percent of Baseline Standard Deviation 0
Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Damage Score (CLASI - TDS) 1st Visit	100 Percent of Baseline Standard Deviation 0
Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Damage Score (CLASI - TDS) 2nd Visit	100 Percent of Baseline Standard Deviation 0
Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Damage Score (CLASI - TDS) 3rd Visit	100 Percent of Baseline Standard Deviation 0
Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Damage Score (CLASI - TDS) 4th Visit	100 Percent of Baseline Standard Deviation 0
Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Damage Score (CLASI - TDS) 5th Visit	100 Percent of Baseline Standard Deviation 0
Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Damage Score (CLASI - TDS) 6th Visit	100 Percent of Baseline Standard Deviation 0
Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Damage Score (CLASI - TDS) 7th Visit	100 Percent of Baseline Standard Deviation 0
Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Damage Score (CLASI - TDS) 8th Visit	100 Percent of Baseline Standard Deviation 0
Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Damage Score (CLASI - TDS) 9th Visit	100 Percent of Baseline Standard Deviation 0

Adverse Events

IVIg as a Monotherapy

Serious events: 0 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	IVIg as a Monotherapy n=16 participants at risk 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.
Blood and lymphatic system disorders Anemia	12.5% 2/16
Blood and lymphatic system disorders Neutropenia	6.2% 1/16
Nervous system disorders Headache	18.8% 3/16 • Number of events 5
Immune system disorders Oral Lupus Flare	6.2% 1/16
Musculoskeletal and connective tissue disorders Back Spasms	6.2% 1/16
Infections and infestations Diabetic Foot Ulcer	6.2% 1/16
Skin and subcutaneous tissue disorders Herpes - Associated Erythema Multiforme	6.2% 1/16
Immune system disorders Lupus Flare	12.5% 2/16
Gastrointestinal disorders GI Upset	6.2% 1/16
General disorders Fever	12.5% 2/16
Gastrointestinal disorders Nausea/Vomting	12.5% 2/16

Additional Information

Sergei A. Grando

UC Irvine Dermatology Research Center

Phone: 949 824 7103

Email: sgrando@uci.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place