Trial Outcomes & Findings for Elotuzumab in Immunoglobulin G4-Related Disease (IgG4-RD) (NCT NCT04918147)
NCT ID: NCT04918147
Last Updated: 2026-02-05
Results Overview
The severity of AEs were classified using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE), version 5.0
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
8 participants
Primary outcome timeframe
Up to Week 48 post treatment initiation
Results posted on
2026-02-05
Participant Flow
Three study sites were activated in the United States, beginning in October 2021. A total of nine participants were screened from October 2021 to October 2023 at two sites. Six participants were enrolled in Cohort 1a. Two participants were enrolled in Cohort 1b; enrollment in Cohort 1b was terminated early No participants were enrolled in Part 2.
Participant milestones
| Measure |
Cohort 1a: Elotuzumab, 1-Month Regimen + Prednisone Taper
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Days 0, 7, 14, 21. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
Cohort 1b: Elotuzumab, 12-Month Regimen + Prednisone Taper
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Day 0 and Weeks 8, 16, 24, 32, and 40. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
|---|---|---|
|
Overall Study
STARTED
|
6
|
2
|
|
Overall Study
COMPLETED
|
6
|
0
|
|
Overall Study
NOT COMPLETED
|
0
|
2
|
Reasons for withdrawal
| Measure |
Cohort 1a: Elotuzumab, 1-Month Regimen + Prednisone Taper
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Days 0, 7, 14, 21. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
Cohort 1b: Elotuzumab, 12-Month Regimen + Prednisone Taper
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Day 0 and Weeks 8, 16, 24, 32, and 40. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
|---|---|---|
|
Overall Study
Disease flare as defined in protocol
|
0
|
2
|
Baseline Characteristics
Elotuzumab in Immunoglobulin G4-Related Disease (IgG4-RD)
Baseline characteristics by cohort
| Measure |
Cohort 1a: Elotuzumab, 1-Month Regimen + Prednisone Taper
n=6 Participants
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Days 0, 7, 14, 21. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
Cohort 1b: Elotuzumab, 12-Month Regimen + Prednisone Taper
n=2 Participants
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Day 0 and Weeks 8, 16, 24, 32, and 40. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
Total
n=8 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=25 Participants
|
0 Participants
n=26 Participants
|
0 Participants
n=51 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
2 Participants
n=25 Participants
|
2 Participants
n=26 Participants
|
4 Participants
n=51 Participants
|
|
Age, Categorical
>=65 years
|
4 Participants
n=25 Participants
|
0 Participants
n=26 Participants
|
4 Participants
n=51 Participants
|
|
Age, Continuous
|
63.8 years
STANDARD_DEVIATION 6.27 • n=25 Participants
|
56.0 years
STANDARD_DEVIATION 0.00 • n=26 Participants
|
61.9 years
STANDARD_DEVIATION 6.42 • n=51 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=25 Participants
|
2 Participants
n=26 Participants
|
3 Participants
n=51 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=25 Participants
|
0 Participants
n=26 Participants
|
5 Participants
n=51 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=25 Participants
|
0 Participants
n=26 Participants
|
0 Participants
n=51 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
6 Participants
n=25 Participants
|
2 Participants
n=26 Participants
|
8 Participants
n=51 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=25 Participants
|
0 Participants
n=26 Participants
|
0 Participants
n=51 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=25 Participants
|
0 Participants
n=26 Participants
|
0 Participants
n=51 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=25 Participants
|
0 Participants
n=26 Participants
|
0 Participants
n=51 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=25 Participants
|
0 Participants
n=26 Participants
|
0 Participants
n=51 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=25 Participants
|
2 Participants
n=26 Participants
|
3 Participants
n=51 Participants
|
|
Race (NIH/OMB)
White
|
5 Participants
n=25 Participants
|
0 Participants
n=26 Participants
|
5 Participants
n=51 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=25 Participants
|
0 Participants
n=26 Participants
|
0 Participants
n=51 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=25 Participants
|
0 Participants
n=26 Participants
|
0 Participants
n=51 Participants
|
|
Region of Enrollment
United States
|
6 Participants
n=25 Participants
|
2 Participants
n=26 Participants
|
8 Participants
n=51 Participants
|
|
IgG4-Related Disease Responder Index (IgG4-RD RI) Total Activity Score
|
9.3 Scores on a scale
STANDARD_DEVIATION 3.01 • n=25 Participants
|
7.0 Scores on a scale
STANDARD_DEVIATION 4.24 • n=26 Participants
|
8.8 Scores on a scale
STANDARD_DEVIATION 3.20 • n=51 Participants
|
PRIMARY outcome
Timeframe: Up to Week 48 post treatment initiationPopulation: The safety population includes all participants who received any amount of investigational product.
The severity of AEs were classified using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE), version 5.0
Outcome measures
| Measure |
Cohort 1a: Elotuzumab, 1-Month Regimen + Prednisone Taper
n=6 Participants
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Days 0, 7, 14, 21. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
Cohort 1b: Elotuzumab, 12-Month Regimen + Prednisone Taper
n=2 Participants
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Day 0 and Weeks 8, 16, 24, 32, and 40. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
|---|---|---|
|
The Percent of Participants in Each Cohort Who Experience at Least One Grade 3 or Higher Adverse Event (AE).
|
5 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Up to Week 48 post treatment initiationPopulation: The safety population includes all participants who received any amount of investigational product.
The severity of AEs was classified using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE), version 5.0.
Outcome measures
| Measure |
Cohort 1a: Elotuzumab, 1-Month Regimen + Prednisone Taper
n=6 Participants
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Days 0, 7, 14, 21. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
Cohort 1b: Elotuzumab, 12-Month Regimen + Prednisone Taper
n=2 Participants
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Day 0 and Weeks 8, 16, 24, 32, and 40. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
|---|---|---|
|
The Percent of Participants Who Experience at Least One Grade 2 or Higher Adverse Event (AE).
|
6 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: Up to Week 48 post treatment initiationPopulation: The safety population includes all participants who received any amount of investigational product.
The number of AEs classified as infections that were Grade 3 or higher. The severity of AEs was classified using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE), version 5.0.
Outcome measures
| Measure |
Cohort 1a: Elotuzumab, 1-Month Regimen + Prednisone Taper
n=6 Participants
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Days 0, 7, 14, 21. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
Cohort 1b: Elotuzumab, 12-Month Regimen + Prednisone Taper
n=2 Participants
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Day 0 and Weeks 8, 16, 24, 32, and 40. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
|---|---|---|
|
The Percent of Participants With a Grade 3 or Higher Infection
|
3 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Up to Week 48 post treatment initiationPopulation: The safety population includes all participants who received any amount of investigational product.
The number of participants with malignancies reported as AEs. The severity of AEs was classified using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE), version 5.0.
Outcome measures
| Measure |
Cohort 1a: Elotuzumab, 1-Month Regimen + Prednisone Taper
n=6 Participants
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Days 0, 7, 14, 21. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
Cohort 1b: Elotuzumab, 12-Month Regimen + Prednisone Taper
n=2 Participants
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Day 0 and Weeks 8, 16, 24, 32, and 40. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
|---|---|---|
|
The Percent of Participants Who Experience a Malignancy.
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Up to Week 48 post treatment initiationPopulation: The safety population includes all participants who received any amount of investigational product.
Hepatotoxicity is defined as an increase in the aspartate aminotransferase (AST) or alanine aminotransferase (ALT) to elevations three times the upper limit of normal.
Outcome measures
| Measure |
Cohort 1a: Elotuzumab, 1-Month Regimen + Prednisone Taper
n=6 Participants
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Days 0, 7, 14, 21. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
Cohort 1b: Elotuzumab, 12-Month Regimen + Prednisone Taper
n=2 Participants
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Day 0 and Weeks 8, 16, 24, 32, and 40. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
|---|---|---|
|
The Percent of Participants Who Experience a Hepatotoxicity
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Up to Week 48 post treatment initiationPopulation: The safety population includes all participants who received any amount of investigational product.
An adverse event (AE) is considered "serious" if, in the view of either the investigator or sponsor, it results in any of the following outcomes (21 CFR 312.32(a)): 1\. Death. 2. Life-threatening event: An AE is considered "life-threatening" if, in the view of either the investigator or Sponsor, its occurrence places the participant at immediate risk of death. It does not include an AE that, had it occurred in a more severe form, might have caused death. 3. Inpatient hospitalization or prolongation of existing hospitalization. 4. Persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. 5. Congenital anomaly or birth defect. 6. Important medical events that may not result in death, be life threatening, or require hospitalization may be considered serious when, based upon appropriate medical judgment, they may jeopardize the participant and may require medical or surgical intervention to prevent one of the outcomes listed above.
Outcome measures
| Measure |
Cohort 1a: Elotuzumab, 1-Month Regimen + Prednisone Taper
n=6 Participants
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Days 0, 7, 14, 21. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
Cohort 1b: Elotuzumab, 12-Month Regimen + Prednisone Taper
n=2 Participants
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Day 0 and Weeks 8, 16, 24, 32, and 40. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
|---|---|---|
|
The Percent of Participants Who Experience a Serious Adverse Event
|
4 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Up to 24 hours post treatment infusionPopulation: The safety population includes all participants who received any amount of investigational product.
The number of participants with infusion reactions reported as AEs. Infusion reactions are defined as any adverse reaction within 24 hours of infusion which are Grade 2 or higher events and at least possibly related to study drug. The severity of AEs was classified using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE), version 5.0.
Outcome measures
| Measure |
Cohort 1a: Elotuzumab, 1-Month Regimen + Prednisone Taper
n=6 Participants
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Days 0, 7, 14, 21. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
Cohort 1b: Elotuzumab, 12-Month Regimen + Prednisone Taper
n=2 Participants
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Day 0 and Weeks 8, 16, 24, 32, and 40. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
|---|---|---|
|
The Percent of Participants Who Experience Infusion Reactions
|
1 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline and Week 24Population: The safety population includes all participants who received any amount of investigational product.
The IgG4-RD RI detects change in disease activity and identifies improvements/worsening in the same or different organ systems. It encompasses more than 25 organs/sites and records the following for each organ/site: (i) activity trend (through a 0-3 \[normal/resolved - worsening\] organ/site score); (ii) presence of symptoms due to active disease; (iii) need for urgent care; (iv) presence of damage; and (v) presence of symptoms due to damage. The final activity score at each visit is obtained by summing all organ/site scores (i) and by doubling items needing urgent care (iii). Higher scores represent greater (i.e. worse) disease activity. % improvement in the IgG4-RD RI = 100 x ( (Baseline IgG4-RD RI Activity Score - Week 24 IgG4-RD RI Activity Score) / Baseline IgG4-RD RI Activity Score). Participants who use glucocorticoids/immunosuppressants beyond that permitted by protocol, or who experience a disease flare before 24 weeks will be defined as achieving no disease response (0%).
Outcome measures
| Measure |
Cohort 1a: Elotuzumab, 1-Month Regimen + Prednisone Taper
n=6 Participants
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Days 0, 7, 14, 21. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
Cohort 1b: Elotuzumab, 12-Month Regimen + Prednisone Taper
n=2 Participants
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Day 0 and Weeks 8, 16, 24, 32, and 40. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
|---|---|---|
|
The Percent of Participants That Reach the Following Threshold Levels of IgG4-Related Disease Responder Index (IgG4-RD RI) Improvement at 24 Weeks: 50% and 75%.
50% Improvement
|
2 Participants
|
0 Participants
|
|
The Percent of Participants That Reach the Following Threshold Levels of IgG4-Related Disease Responder Index (IgG4-RD RI) Improvement at 24 Weeks: 50% and 75%.
75% Improvement
|
2 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Up to Week 48 post treatment initiationPopulation: The safety population includes all participants who received any amount of investigational product.
IgG4-Related Disease flare was defined as recurrence of disease activity such that additional immunosuppressive therapy beyond the trial protocol is indicated. Such additional therapy may include glucocorticoids or alternative immunosuppressive agents. At the time of a potential disease flare, investigators documented the features of the disease flare in each involved organ, including laboratory evaluations, radiology studies, and other procedures (e.g., biopsy). Disease flares were reported as adverse events.
Outcome measures
| Measure |
Cohort 1a: Elotuzumab, 1-Month Regimen + Prednisone Taper
n=6 Participants
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Days 0, 7, 14, 21. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
Cohort 1b: Elotuzumab, 12-Month Regimen + Prednisone Taper
n=2 Participants
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Day 0 and Weeks 8, 16, 24, 32, and 40. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
|---|---|---|
|
Number of Participants With Disease Flares
|
4 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: Baseline, Weeks 12, 24 and 48Population: The safety population includes all participants who received any amount of investigational product.
The Physician Global Assessment of the participant's current disease activity was recorded on a 100 mm linear horizontal visual analog scale, where the left hand extreme of the line is considered "Very Good" (0 mm, symptom free and no IgG4-RD symptoms) and the right hand extreme is considered "Very Bad" (100 mm, maximum IgG4-RD activity). Only active disease (as opposed to damage) is considered in the scoring. Change was calculated as the value at Week 12, 24 or 48 minus the value at Baseline.
Outcome measures
| Measure |
Cohort 1a: Elotuzumab, 1-Month Regimen + Prednisone Taper
n=6 Participants
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Days 0, 7, 14, 21. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
Cohort 1b: Elotuzumab, 12-Month Regimen + Prednisone Taper
n=2 Participants
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Day 0 and Weeks 8, 16, 24, 32, and 40. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
|---|---|---|
|
Change From Baseline in Physician Global Assessment
Week 12
|
—
|
3.0 millimeter
Standard Deviation 24.04
|
|
Change From Baseline in Physician Global Assessment
Week 24
|
-18.5 millimeter
Standard Deviation 15.60
|
—
|
|
Change From Baseline in Physician Global Assessment
Week 48
|
-30.0 millimeter
Standard Deviation 17.32
|
—
|
SECONDARY outcome
Timeframe: Baseline, Weeks 12, 24 and 48Population: The safety population includes all participants who received any amount of investigational product.
The Patient Global Assessment of the participant's current disease activity was recorded on a 100 mm linear horizontal visual analog scale, where the left hand extreme of the line is considered "Very Good" (0 mm, symptom free and no IgG4-RD symptoms) and the right hand extreme is considered "Very Bad" (100 mm, maximum IgG4-RD activity). Change was calculated as the value at Week 12, 24 or 48 minus the value at Baseline.
Outcome measures
| Measure |
Cohort 1a: Elotuzumab, 1-Month Regimen + Prednisone Taper
n=6 Participants
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Days 0, 7, 14, 21. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
Cohort 1b: Elotuzumab, 12-Month Regimen + Prednisone Taper
n=2 Participants
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Day 0 and Weeks 8, 16, 24, 32, and 40. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
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|---|---|---|
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Change From Baseline in Patient Global Assessment
Week 12
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—
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-15.0 millimeter
Standard Deviation 32.53
|
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Change From Baseline in Patient Global Assessment
Week 24
|
18.8 millimeter
Standard Deviation 40.53
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—
|
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Change From Baseline in Patient Global Assessment
Week 48
|
17.0 millimeter
Standard Deviation 34.04
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—
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SECONDARY outcome
Timeframe: Screening and Week 24Population: The safety population includes all participants who received any amount of investigational product.
An assessment of damage caused by IgG4-RD in each affected organ is part of the Responder Index. The IgG4-RD RI encompasses more than 25 organs/sites and records the following set of information for each organ/site: (i) activity trend (through a 0-3 \[normal/resolved - worsening\] organ/site score); (ii) presence of symptoms due to active disease; (iii) need for urgent care; (iv) presence of damage; and (v) presence of symptoms due to damage. Activity and damage are considered separately from the standpoint of scoring, because only disease activity can be expected to respond to treatment. Damage is defined as organ dysfunction that has occurred as a result of IgG4-RD and is considered permanent. The total damage score at each visit is defined as the total number of damaged organs and can range from 0 (no damage) to more than 26 (severe damage).
Outcome measures
| Measure |
Cohort 1a: Elotuzumab, 1-Month Regimen + Prednisone Taper
n=6 Participants
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Days 0, 7, 14, 21. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
Cohort 1b: Elotuzumab, 12-Month Regimen + Prednisone Taper
n=2 Participants
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Day 0 and Weeks 8, 16, 24, 32, and 40. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
|---|---|---|
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Disease-Related Damage, as Measured by the Damage Section of the IgG4-Related Disease (IgG4-RD) Responder Index.
Screening
|
1.3 Scores on a scale
Standard Deviation 1.21
|
0 Scores on a scale
Standard Deviation 0
|
|
Disease-Related Damage, as Measured by the Damage Section of the IgG4-Related Disease (IgG4-RD) Responder Index.
Week 24
|
1.5 Scores on a scale
Standard Deviation 1.52
|
—
|
Adverse Events
Cohort 1a: Elotuzumab, 1-Month Regimen + Prednisone Taper
Serious events: 4 serious events
Other events: 6 other events
Deaths: 0 deaths
Cohort 1b: Elotuzumab, 12-Month Regimen + Prednisone Taper
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Cohort 1a: Elotuzumab, 1-Month Regimen + Prednisone Taper
n=6 participants at risk
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Days 0, 7, 14, 21. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
Cohort 1b: Elotuzumab, 12-Month Regimen + Prednisone Taper
n=2 participants at risk
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Day 0 and Weeks 8, 16, 24, 32, and 40. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
16.7%
1/6 • Number of events 1 • Up to Week 48 post treatment initiation.
|
0.00%
0/2 • Up to Week 48 post treatment initiation.
|
|
Infections and infestations
Appendicitis
|
16.7%
1/6 • Number of events 1 • Up to Week 48 post treatment initiation.
|
0.00%
0/2 • Up to Week 48 post treatment initiation.
|
|
Infections and infestations
COVID-19
|
33.3%
2/6 • Number of events 2 • Up to Week 48 post treatment initiation.
|
0.00%
0/2 • Up to Week 48 post treatment initiation.
|
|
Injury, poisoning and procedural complications
Hip fracture
|
16.7%
1/6 • Number of events 1 • Up to Week 48 post treatment initiation.
|
0.00%
0/2 • Up to Week 48 post treatment initiation.
|
Other adverse events
| Measure |
Cohort 1a: Elotuzumab, 1-Month Regimen + Prednisone Taper
n=6 participants at risk
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Days 0, 7, 14, 21. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
Cohort 1b: Elotuzumab, 12-Month Regimen + Prednisone Taper
n=2 participants at risk
Elotuzumab was administered open label as an intravenous infusion, 10 mg/kg (based on participant's actual weight), on Day 0 and Weeks 8, 16, 24, 32, and 40. Pre-medication with methylprednisolone, diphenhydramine, acetominophen, and an H2 blocker (famotidine or equivalent) occurred prior to each elotuzumab infusion. Participants also received a standardized prednisone taper, starting at either 40 mg orally daily or 30 mg orally based on doses received during the screening period. This was tapered to discontinuation over a 10-week period.
|
|---|---|---|
|
Injury, poisoning and procedural complications
Infusion related reaction
|
16.7%
1/6 • Number of events 1 • Up to Week 48 post treatment initiation.
|
0.00%
0/2 • Up to Week 48 post treatment initiation.
|
|
Investigations
Alanine aminotransferase increased
|
16.7%
1/6 • Number of events 1 • Up to Week 48 post treatment initiation.
|
0.00%
0/2 • Up to Week 48 post treatment initiation.
|
|
Investigations
Blood albumin decreased
|
16.7%
1/6 • Number of events 1 • Up to Week 48 post treatment initiation.
|
0.00%
0/2 • Up to Week 48 post treatment initiation.
|
|
Investigations
Blood potassium increased
|
16.7%
1/6 • Number of events 1 • Up to Week 48 post treatment initiation.
|
0.00%
0/2 • Up to Week 48 post treatment initiation.
|
|
Investigations
Glomerular filtration rate decreased
|
16.7%
1/6 • Number of events 1 • Up to Week 48 post treatment initiation.
|
0.00%
0/2 • Up to Week 48 post treatment initiation.
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
16.7%
1/6 • Number of events 1 • Up to Week 48 post treatment initiation.
|
0.00%
0/2 • Up to Week 48 post treatment initiation.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Bone cyst
|
16.7%
1/6 • Number of events 1 • Up to Week 48 post treatment initiation.
|
0.00%
0/2 • Up to Week 48 post treatment initiation.
|
|
Skin and subcutaneous tissue disorders
Acne
|
16.7%
1/6 • Number of events 1 • Up to Week 48 post treatment initiation.
|
0.00%
0/2 • Up to Week 48 post treatment initiation.
|
|
Cardiac disorders
Tachycardia
|
16.7%
1/6 • Number of events 1 • Up to Week 48 post treatment initiation.
|
0.00%
0/2 • Up to Week 48 post treatment initiation.
|
|
Endocrine disorders
Cushing's syndrome
|
16.7%
1/6 • Number of events 1 • Up to Week 48 post treatment initiation.
|
0.00%
0/2 • Up to Week 48 post treatment initiation.
|
|
Gastrointestinal disorders
Abdominal pain
|
16.7%
1/6 • Number of events 1 • Up to Week 48 post treatment initiation.
|
0.00%
0/2 • Up to Week 48 post treatment initiation.
|
|
Immune system disorders
Immunoglobulin G4 related disease
|
66.7%
4/6 • Number of events 4 • Up to Week 48 post treatment initiation.
|
100.0%
2/2 • Number of events 2 • Up to Week 48 post treatment initiation.
|
|
Immune system disorders
Secondary immunodeficiency
|
16.7%
1/6 • Number of events 1 • Up to Week 48 post treatment initiation.
|
0.00%
0/2 • Up to Week 48 post treatment initiation.
|
|
Infections and infestations
Bronchitis
|
16.7%
1/6 • Number of events 1 • Up to Week 48 post treatment initiation.
|
0.00%
0/2 • Up to Week 48 post treatment initiation.
|
|
Infections and infestations
COVID-19
|
33.3%
2/6 • Number of events 2 • Up to Week 48 post treatment initiation.
|
0.00%
0/2 • Up to Week 48 post treatment initiation.
|
Additional Information
Director, Clinical Research Operations Program
DAIT/NIAID
Phone: 301-594-7669
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place