Trial Outcomes & Findings for Clarification of Abatacept Effects in SLE With Integrated Biologic and Clinical Approaches (NCT NCT02270957)

NCT ID: NCT02270957

Last Updated: 2022-01-26

Results Overview

The British Isles Lupus Assessment Group Index is a scoring system for progress of disease activity over the prior month with a scoring system that rates each organ system as "A" or severe, "B" or moderate, "C" or mild vs no activity in the past month. To meet the BICLA endpoint requires all baseline severe features (BILAG A) improving to moderate (BILAG B), mild or resolved, and all baseline BILAG B features improving to mild or resolved without increase in any other feature on either the BILAG or a different measure called the SLEDAI (SLE Disease Activity Index). Furthermore there must be no increase in Physician's Global Assessment or any rescue medications after the month 2 visit. Only those meeting all of these criteria meet the primary endpoint.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

66 participants

Primary outcome timeframe

6 months

Results posted on

2022-01-26

Participant Flow

Participant milestones

Participant milestones
Measure
Abatacept
Patients receive Abatacept 125 mg subcutaneously weekly for six months. An optional continuation up until 12 months is allowed. Background immune suppressants are withdrawn at the beginning of the study and the option of depomedrol up to 320 mg total (in divided doses) is allowed at any time up through the visit 2 months after study medication is started. After this additional rescue is allowed with any standard of care treatment and/or open label abatacept (since patients are blinded) but this additional rescue will define non-response in the primary endpoint at six months. Abatacept
Placebo
Patients receive Placebo but at the time they meet non-response criteria they may elect open label abatacept or any standard of care to treat their SLE
Overall Study
STARTED
31
35
Overall Study
COMPLETED
27
31
Overall Study
NOT COMPLETED
4
4

Reasons for withdrawal

Reasons for withdrawal
Measure
Abatacept
Patients receive Abatacept 125 mg subcutaneously weekly for six months. An optional continuation up until 12 months is allowed. Background immune suppressants are withdrawn at the beginning of the study and the option of depomedrol up to 320 mg total (in divided doses) is allowed at any time up through the visit 2 months after study medication is started. After this additional rescue is allowed with any standard of care treatment and/or open label abatacept (since patients are blinded) but this additional rescue will define non-response in the primary endpoint at six months. Abatacept
Placebo
Patients receive Placebo but at the time they meet non-response criteria they may elect open label abatacept or any standard of care to treat their SLE
Overall Study
Lack of Efficacy
2
4
Overall Study
Adverse Event
2
0

Baseline Characteristics

Clarification of Abatacept Effects in SLE With Integrated Biologic and Clinical Approaches

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Abatacept
n=31 Participants
Patients receive Abatacept 125 mg subcutaneously weekly for six months. An optional continuation up until 12 months is allowed. Background immune suppressants are withdrawn at the beginning of the study and the option of depomedrol up to 320 mg total (in divided doses) is allowed at any time up through the visit 2 months after study medication is started. After this additional rescue is allowed with any standard of care treatment and/or open label abatacept (since patients are blinded) but this additional rescue will define non-response in the primary endpoint at six months. Abatacept
Placebo
n=35 Participants
Patients receive Placebo. At any time that they meet non-response criteria (primary endpoint) they may elect to be treated with any standard of care medication or open label abatacept Placebo
Total
n=66 Participants
Total of all reporting groups
Age, Continuous
43.8 years
STANDARD_DEVIATION 10.9 • n=5 Participants
46.9 years
STANDARD_DEVIATION 11.9 • n=7 Participants
45.4 years
STANDARD_DEVIATION 11.5 • n=5 Participants
Sex: Female, Male
Female
29 Participants
n=5 Participants
34 Participants
n=7 Participants
63 Participants
n=5 Participants
Sex: Female, Male
Male
2 Participants
n=5 Participants
1 Participants
n=7 Participants
3 Participants
n=5 Participants
Race/Ethnicity, Customized
Caucasian
17 Participants
n=5 Participants
26 Participants
n=7 Participants
43 Participants
n=5 Participants
Race/Ethnicity, Customized
African
8 Participants
n=5 Participants
6 Participants
n=7 Participants
14 Participants
n=5 Participants
Race/Ethnicity, Customized
Asian
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race/Ethnicity, Customized
Native American/Alaskan Native
6 Participants
n=5 Participants
3 Participants
n=7 Participants
9 Participants
n=5 Participants
Region of Enrollment
United States
31 participants
n=5 Participants
35 participants
n=7 Participants
66 participants
n=5 Participants
anti-dsDNA positive
5 participants
n=5 Participants
12 participants
n=7 Participants
17 participants
n=5 Participants
low complement
5 participants
n=5 Participants
9 participants
n=7 Participants
14 participants
n=5 Participants
Number with at least one BILAG "B" score
31 Participants
n=5 Participants
35 Participants
n=7 Participants
66 Participants
n=5 Participants

PRIMARY outcome

Timeframe: 6 months

Population: Full Analysis Set: All Randomized Patients

The British Isles Lupus Assessment Group Index is a scoring system for progress of disease activity over the prior month with a scoring system that rates each organ system as "A" or severe, "B" or moderate, "C" or mild vs no activity in the past month. To meet the BICLA endpoint requires all baseline severe features (BILAG A) improving to moderate (BILAG B), mild or resolved, and all baseline BILAG B features improving to mild or resolved without increase in any other feature on either the BILAG or a different measure called the SLEDAI (SLE Disease Activity Index). Furthermore there must be no increase in Physician's Global Assessment or any rescue medications after the month 2 visit. Only those meeting all of these criteria meet the primary endpoint.

Outcome measures

Outcome measures
Measure
Abatacept
n=31 Participants
Patients receive Abatacept 125 mg subcutaneously weekly for six months. An optional continuation up until 12 months is allowed. Background immune suppressants are withdrawn at the beginning of the study and the option of depomedrol up to 320 mg total (in divided doses) is allowed at any time up through the visit 2 months after study medication is started. After this additional rescue is allowed with any standard of care treatment and/or open label abatacept (since patients are blinded) but this additional rescue will define non-response in the primary endpoint at six months. Abatacept
Placebo
n=35 Participants
Patients receive Abatacept 125 mg subcutaneously weekly for six months. An optional continuation up until 12 months is allowed. Background immune suppressants are withdrawn at the beginning of the study and the option of depomedrol up to 320 mg total (in divided doses) is allowed at any time up through the visit 2 months after study medication is started. After this additional rescue is allowed with any standard of care treatment and/or open label abatacept (since patients are blinded) but this additional rescue will define non-response in the primary endpoint at six months. Placebo
British Isles Lupus Assessment Group Index-based Combined Lupus Assessment (BICLA)
8 Participants
8 Participants

POST_HOC outcome

Timeframe: 6 months

Population: Full Analysis Set

Comparing the endpoint date at six months to Baseline, there must be a 4 point decreased in SLEDAI score (SLEDAI is defined as the SLE Disease Activity Index). The SLEDAI is a discontinuous scale in which each type of sign or symptom of active SLE is assigned a fixed number of points. Although the scale includes possible signs or symptoms adding up to more than 100 points it is rare for any (even very severe) patient to ever have a total score \> 20. To meet the SLE Responder Index endpoint, There must also be no worsening of BILAG (British Isles Lupus Assessment Group Index (described in the primary endpoint section) and no worsening of PGA (a visual analogue scale reflecting physicians global assessment) by more than 10% of the scale. To meet this endpoint there must also be no new or increased medication initiated after Baseline other than the steroid rescues up to Month 2.

Outcome measures

Outcome measures
Measure
Abatacept
n=31 Participants
Patients receive Abatacept 125 mg subcutaneously weekly for six months. An optional continuation up until 12 months is allowed. Background immune suppressants are withdrawn at the beginning of the study and the option of depomedrol up to 320 mg total (in divided doses) is allowed at any time up through the visit 2 months after study medication is started. After this additional rescue is allowed with any standard of care treatment and/or open label abatacept (since patients are blinded) but this additional rescue will define non-response in the primary endpoint at six months. Abatacept
Placebo
n=35 Participants
Patients receive Abatacept 125 mg subcutaneously weekly for six months. An optional continuation up until 12 months is allowed. Background immune suppressants are withdrawn at the beginning of the study and the option of depomedrol up to 320 mg total (in divided doses) is allowed at any time up through the visit 2 months after study medication is started. After this additional rescue is allowed with any standard of care treatment and/or open label abatacept (since patients are blinded) but this additional rescue will define non-response in the primary endpoint at six months. Placebo
SLE Responder Index-4 (SRI-4)
9 Participants
8 Participants

POST_HOC outcome

Timeframe: Comparison of Baseline to 6 months

Population: Full Analysis Set of all randomized patients

The SLEDAI is a discontinuous scoring system that weights disease activity not by severity of individual symptoms but by the weighting of organs. This makes it less robust for comparing one group of patients to another, but it is quite useful to gave numbers of patients with improvement, since to lower the score a rigorous improvement must be documented

Outcome measures

Outcome measures
Measure
Abatacept
n=31 Participants
Patients receive Abatacept 125 mg subcutaneously weekly for six months. An optional continuation up until 12 months is allowed. Background immune suppressants are withdrawn at the beginning of the study and the option of depomedrol up to 320 mg total (in divided doses) is allowed at any time up through the visit 2 months after study medication is started. After this additional rescue is allowed with any standard of care treatment and/or open label abatacept (since patients are blinded) but this additional rescue will define non-response in the primary endpoint at six months. Abatacept
Placebo
n=35 Participants
Patients receive Abatacept 125 mg subcutaneously weekly for six months. An optional continuation up until 12 months is allowed. Background immune suppressants are withdrawn at the beginning of the study and the option of depomedrol up to 320 mg total (in divided doses) is allowed at any time up through the visit 2 months after study medication is started. After this additional rescue is allowed with any standard of care treatment and/or open label abatacept (since patients are blinded) but this additional rescue will define non-response in the primary endpoint at six months. Placebo
4 Point Improvement in the SLE Disease Activity Index (SLEDAI)
9 Participants
8 Participants

Adverse Events

Abatacept

Serious events: 6 serious events
Other events: 29 other events
Deaths: 0 deaths

Placebo

Serious events: 4 serious events
Other events: 35 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Abatacept
n=31 participants at risk
Patients receive Abatacept 125 mg subcutaneously weekly for six months.
Placebo
n=35 participants at risk
Patients receive Placebo subcutaneously weekly for six months.
Gastrointestinal disorders
gastroenteritis
3.2%
1/31 • Number of events 1 • 12 months
0.00%
0/35 • 12 months
Infections and infestations
viral syndrome
3.2%
1/31 • Number of events 1 • 12 months
2.9%
1/35 • Number of events 1 • 12 months
Blood and lymphatic system disorders
sickle cell crisis
3.2%
1/31 • Number of events 1 • 12 months
0.00%
0/35 • 12 months
Musculoskeletal and connective tissue disorders
fracture
3.2%
1/31 • Number of events 1 • 12 months
0.00%
0/35 • 12 months
Psychiatric disorders
suicidal ideation
3.2%
1/31 • Number of events 1 • 12 months
0.00%
0/35 • 12 months
Gastrointestinal disorders
cholecystitis
3.2%
1/31 • Number of events 1 • 12 months
0.00%
0/35 • 12 months
Cardiac disorders
cardiac valve dysfunction
0.00%
0/31 • 12 months
2.9%
1/35 • Number of events 1 • 12 months
Social circumstances
opiate overdose
0.00%
0/31 • 12 months
2.9%
1/35 • Number of events 1 • 12 months
Respiratory, thoracic and mediastinal disorders
respiratory compromise
0.00%
0/31 • 12 months
2.9%
1/35 • Number of events 1 • 12 months
Renal and urinary disorders
Acute renal failure
0.00%
0/31 • 12 months
2.9%
1/35 • Number of events 1 • 12 months
Renal and urinary disorders
blocked ureter
0.00%
0/31 • 12 months
2.9%
1/35 • Number of events 1 • 12 months

Other adverse events

Other adverse events
Measure
Abatacept
n=31 participants at risk
Patients receive Abatacept 125 mg subcutaneously weekly for six months.
Placebo
n=35 participants at risk
Patients receive Placebo subcutaneously weekly for six months.
Respiratory, thoracic and mediastinal disorders
bronchitis
16.1%
5/31 • Number of events 5 • 12 months
11.4%
4/35 • Number of events 5 • 12 months
Gastrointestinal disorders
gastroenteritis
54.8%
17/31 • Number of events 18 • 12 months
31.4%
11/35 • Number of events 11 • 12 months
Infections and infestations
oral candida
0.00%
0/31 • 12 months
22.9%
8/35 • Number of events 8 • 12 months
Injury, poisoning and procedural complications
injection site reaction
12.9%
4/31 • Number of events 4 • 12 months
8.6%
3/35 • Number of events 4 • 12 months
Skin and subcutaneous tissue disorders
rash
6.5%
2/31 • Number of events 2 • 12 months
11.4%
4/35 • Number of events 6 • 12 months
Ear and labyrinth disorders
otitis media
3.2%
1/31 • Number of events 1 • 12 months
11.4%
4/35 • Number of events 5 • 12 months
Infections and infestations
urinary tract infection
16.1%
5/31 • Number of events 7 • 12 months
48.6%
17/35 • Number of events 24 • 12 months
Infections and infestations
upper respiratory infection
48.4%
15/31 • Number of events 20 • 12 months
45.7%
16/35 • Number of events 27 • 12 months
Infections and infestations
influenza
3.2%
1/31 • Number of events 1 • 12 months
8.6%
3/35 • Number of events 4 • 12 months
Infections and infestations
sinusitis
12.9%
4/31 • Number of events 4 • 12 months
22.9%
8/35 • Number of events 14 • 12 months
Infections and infestations
vaginal yeast infestation
3.2%
1/31 • Number of events 1 • 12 months
14.3%
5/35 • Number of events 7 • 12 months

Additional Information

Joan T. Merrill, M.D.

Oklahoma Medical Research Foundation

Phone: 405 822 2336

Results disclosure agreements

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