Trial Outcomes & Findings for Abatacept for the Treatment of Myositis-associated Interstitial Lung Disease (NCT NCT03215927)
NCT ID: NCT03215927
Last Updated: 2025-01-07
Results Overview
The primary outcome criteria for efficacy will be the absolute change of % predicted FVC from the baseline visit to week 24 between the 2 treatment arms (SOC/placebo vs. SOC/Abatacept).
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
20 participants
Primary outcome timeframe
24 Weeks
Results posted on
2025-01-07
Participant Flow
Subjects were enrolled from four clinical sites between June 2017 through May 2021.
Participant milestones
| Measure |
Placebo
Subcutaneous placebo injection weekly for 24 weeks. 24 week optional follow up phase all subjects receive abatacept 125 mg weekly.
Placebo: Placebo
|
Abatacept
Subcutaneous injection of abatacept 125 mg weekly for 24 weeks. 24 week optional follow up phase all subjects receive abatacept 125 mg weekly.
Abatacept: Abatacept 125mg subcutaneous weekly
|
|---|---|---|
|
Randomized, Placebo-controlled
STARTED
|
11
|
9
|
|
Randomized, Placebo-controlled
COMPLETED
|
11
|
8
|
|
Randomized, Placebo-controlled
NOT COMPLETED
|
0
|
1
|
|
Open Label Extension
STARTED
|
11
|
8
|
|
Open Label Extension
COMPLETED
|
9
|
7
|
|
Open Label Extension
NOT COMPLETED
|
2
|
1
|
Reasons for withdrawal
| Measure |
Placebo
Subcutaneous placebo injection weekly for 24 weeks. 24 week optional follow up phase all subjects receive abatacept 125 mg weekly.
Placebo: Placebo
|
Abatacept
Subcutaneous injection of abatacept 125 mg weekly for 24 weeks. 24 week optional follow up phase all subjects receive abatacept 125 mg weekly.
Abatacept: Abatacept 125mg subcutaneous weekly
|
|---|---|---|
|
Randomized, Placebo-controlled
Lack of Efficacy
|
0
|
1
|
|
Open Label Extension
Adverse Event
|
1
|
0
|
|
Open Label Extension
Lack of Efficacy
|
1
|
0
|
|
Open Label Extension
Death
|
0
|
1
|
Baseline Characteristics
Abatacept for the Treatment of Myositis-associated Interstitial Lung Disease
Baseline characteristics by cohort
| Measure |
Placebo
n=11 Participants
Subcutaneous placebo injection weekly for 24 weeks.
Placebo: Placebo
|
Abatacept
n=9 Participants
Subcutaneous injection of abatacept 125 mg weekly for 24 weeks. 24 week optional follow up phase all subjects receive abatacept 125 mg weekly.
Abatacept: Abatacept 125mg subcutaneous weekly
|
Total
n=20 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
55.95 years
STANDARD_DEVIATION 6.87 • n=5 Participants
|
51.56 years
STANDARD_DEVIATION 10.69 • n=7 Participants
|
53.98 years
STANDARD_DEVIATION 8.83 • n=5 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
11 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
20 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
10 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Forced Vital Capacity (FVC) %
|
61.0 %
n=5 Participants
|
66.0 %
n=7 Participants
|
63.5 %
n=5 Participants
|
PRIMARY outcome
Timeframe: 24 WeeksThe primary outcome criteria for efficacy will be the absolute change of % predicted FVC from the baseline visit to week 24 between the 2 treatment arms (SOC/placebo vs. SOC/Abatacept).
Outcome measures
| Measure |
Placebo
n=11 Participants
Subcutaneous placebo injection weekly for 24 weeks. 24 week optional follow up phase all subjects receive abatacept 125 mg weekly.
Placebo: Placebo
|
Abatacept
n=9 Participants
Subcutaneous injection of abatacept 125 mg weekly for 24 weeks. 24 week optional follow up phase all subjects receive abatacept 125 mg weekly.
Abatacept: Abatacept 125mg subcutaneous weekly
|
|---|---|---|
|
% Predicted Forced Vital Capacity (FVC) Absolute Change
|
-1.70 % predicted FVC
Interval -5.9 to 2.5
|
-2.05 % predicted FVC
Interval -6.97 to 2.87
|
SECONDARY outcome
Timeframe: 48 weeksThe first occurrence of any of the following: death or lung transplantation or decline in % predicted FVC ≥ 10% or decline in % predicted FVC ≥ 5% with a decline in % predicted DLCO ≥ 15%
Outcome measures
| Measure |
Placebo
n=11 Participants
Subcutaneous placebo injection weekly for 24 weeks. 24 week optional follow up phase all subjects receive abatacept 125 mg weekly.
Placebo: Placebo
|
Abatacept
n=9 Participants
Subcutaneous injection of abatacept 125 mg weekly for 24 weeks. 24 week optional follow up phase all subjects receive abatacept 125 mg weekly.
Abatacept: Abatacept 125mg subcutaneous weekly
|
|---|---|---|
|
Time to Progression Free Survival
|
27.71 Weeks
Interval 12.71 to 46.86
|
16.86 Weeks
Interval 15.36 to 34.93
|
SECONDARY outcome
Timeframe: 24 weeksMeasured by University of California San Diego Shortness of Breath Questionnaire (UCSD SOBQ) (range 0-120, higher score is worsening dyspnea).
Outcome measures
| Measure |
Placebo
n=11 Participants
Subcutaneous placebo injection weekly for 24 weeks. 24 week optional follow up phase all subjects receive abatacept 125 mg weekly.
Placebo: Placebo
|
Abatacept
n=9 Participants
Subcutaneous injection of abatacept 125 mg weekly for 24 weeks. 24 week optional follow up phase all subjects receive abatacept 125 mg weekly.
Abatacept: Abatacept 125mg subcutaneous weekly
|
|---|---|---|
|
Comparison of Change in Patient Reported Dyspnea Scores (University of California San Diego Shortness of Breath Questionnaire)
|
-4.59 score on a scale
Interval -15.54 to 6.36
|
3.20 score on a scale
Interval -8.86 to 15.26
|
SECONDARY outcome
Timeframe: 48 weeksComparison of percent predicted FVC results from pulmonary function tests from baseline to week 48. Improvement is defined as a % predicted FVC change ≥10%
Outcome measures
| Measure |
Placebo
n=11 Participants
Subcutaneous placebo injection weekly for 24 weeks. 24 week optional follow up phase all subjects receive abatacept 125 mg weekly.
Placebo: Placebo
|
Abatacept
n=9 Participants
Subcutaneous injection of abatacept 125 mg weekly for 24 weeks. 24 week optional follow up phase all subjects receive abatacept 125 mg weekly.
Abatacept: Abatacept 125mg subcutaneous weekly
|
|---|---|---|
|
Time to Improvement in % Predicted FVC ≥10%
|
17.86 weeks
Interval 9.86 to 25.86
|
11.86 weeks
Interval 11.86 to 11.86
|
Adverse Events
Placebo
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
Abatacept
Serious events: 3 serious events
Other events: 6 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Placebo
n=11 participants at risk
Subcutaneous placebo injection weekly for 24 weeks.
Placebo: Placebo
|
Abatacept
n=20 participants at risk
Subcutaneous injection of abatacept 125 mg weekly for 24 weeks. 24 week optional follow up phase all subjects receive abatacept 125 mg weekly.
Abatacept: Abatacept 125mg subcutaneous weekly
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Failure
|
0.00%
0/11 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
5.0%
1/20 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
0.00%
0/11 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
5.0%
1/20 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
0.00%
0/11 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
5.0%
1/20 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
Other adverse events
| Measure |
Placebo
n=11 participants at risk
Subcutaneous placebo injection weekly for 24 weeks.
Placebo: Placebo
|
Abatacept
n=20 participants at risk
Subcutaneous injection of abatacept 125 mg weekly for 24 weeks. 24 week optional follow up phase all subjects receive abatacept 125 mg weekly.
Abatacept: Abatacept 125mg subcutaneous weekly
|
|---|---|---|
|
Blood and lymphatic system disorders
Excessive bruising
|
18.2%
2/11 • Number of events 2 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
5.0%
1/20 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Cardiac disorders
Sinus Tachycardia
|
18.2%
2/11 • Number of events 2 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
0.00%
0/20 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Infections and infestations
Eye Infection
|
0.00%
0/11 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
11.1%
1/9 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Gastrointestinal disorders
Abdominal Pain
|
0.00%
0/11 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
5.0%
1/20 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Gastrointestinal disorders
Nausea
|
18.2%
2/11 • Number of events 2 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
0.00%
0/20 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
General disorders
Fatigue
|
9.1%
1/11 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
0.00%
0/20 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
General disorders
Fever
|
9.1%
1/11 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
0.00%
0/20 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Infections and infestations
Herpes Zoster
|
9.1%
1/11 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
0.00%
0/20 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Infections and infestations
Upper Respiratory Infection
|
18.2%
2/11 • Number of events 2 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
15.0%
3/20 • Number of events 3 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Infections and infestations
Lip infection
|
0.00%
0/11 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
5.0%
1/20 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Investigations
Deceased lymphocyte count
|
0.00%
0/11 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
10.0%
2/20 • Number of events 2 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Investigations
Aspartate aminotransferase
|
9.1%
1/11 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
0.00%
0/20 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Investigations
alanine aminotransferase increased
|
9.1%
1/11 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
0.00%
0/20 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
9.1%
1/11 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
0.00%
0/20 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
0.00%
0/11 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
5.0%
1/20 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Nervous system disorders
Headache
|
9.1%
1/11 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
5.0%
1/20 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Psychiatric disorders
Insomnia
|
0.00%
0/11 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
5.0%
1/20 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Psychiatric disorders
Anxiety
|
0.00%
0/11 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
5.0%
1/20 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Psychiatric disorders
Depression
|
0.00%
0/11 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
5.0%
1/20 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
18.2%
2/11 • Number of events 2 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
15.0%
3/20 • Number of events 3 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Respiratory, thoracic and mediastinal disorders
Decreased DLCO
|
9.1%
1/11 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
0.00%
0/20 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Respiratory, thoracic and mediastinal disorders
Voice alteration
|
9.1%
1/11 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
0.00%
0/20 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
9.1%
1/11 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
5.0%
1/20 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Metabolism and nutrition disorders
Anorexia
|
9.1%
1/11 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
5.0%
1/20 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Cardiac disorders
Hypertension
|
0.00%
0/11 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
5.0%
1/20 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Infections and infestations
Oral thrush
|
0.00%
0/11 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
5.0%
1/20 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Cardiac disorders
Chest pain
|
0.00%
0/11 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
10.0%
2/20 • Number of events 2 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Gastrointestinal disorders
colitis
|
0.00%
0/11 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
5.0%
1/20 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Gastrointestinal disorders
diarrhea
|
0.00%
0/11 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
15.0%
3/20 • Number of events 3 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Gastrointestinal disorders
oral mucositis
|
0.00%
0/11 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
5.0%
1/20 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Gastrointestinal disorders
vomiting
|
0.00%
0/11 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
5.0%
1/20 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Infections and infestations
Streptococcal infection
|
0.00%
0/11 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
5.0%
1/20 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Musculoskeletal and connective tissue disorders
pain in extremities
|
0.00%
0/11 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
5.0%
1/20 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Musculoskeletal and connective tissue disorders
shoulder pain
|
0.00%
0/11 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
5.0%
1/20 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Renal and urinary disorders
renal calculi
|
0.00%
0/11 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
10.0%
2/20 • Number of events 2 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Respiratory, thoracic and mediastinal disorders
voice alteration
|
0.00%
0/11 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
5.0%
1/20 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Reproductive system and breast disorders
other
|
0.00%
0/11 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
5.0%
1/20 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Skin and subcutaneous tissue disorders
dry skin
|
0.00%
0/11 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
5.0%
1/20 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
|
Skin and subcutaneous tissue disorders
rash on extremities
|
0.00%
0/11 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
5.0%
1/20 • Number of events 1 • Adverse events were collected from Baseline (week 0) through end of study Visit 5 (week 48)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60