Trial Outcomes & Findings for Proof-of-Concept Study With BT063 in Subjects With Systemic Lupus Erythematosus (NCT NCT02554019)
NCT ID: NCT02554019
Last Updated: 2020-01-27
Results Overview
Number of Participants with Adverse Events (Including SAEs and AEs leading to discontinuation) from Baseline through End of Trial Visit (Week 14)
COMPLETED
PHASE2
36 participants
Baseline through End of Trial Visit (Week 14)
2020-01-27
Participant Flow
In total 36 subjects were enrolled in study parts I and II (18 in part I and 18 in part II). 12 subjects in part I received 50 mg BT063, 12 subjects in part II received 100 mg BT063. 6 subjects in part I and 6 subjects in part II received Placebo and were pooled for analysis. Only final results and no partial or interim results are presented.
Participant milestones
| Measure |
BT063 50 mg
50 mg BT063 administered by intravenous (IV) infusion 8 times
BT063: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
BT063 100 mg
100 mg BT063 administered by intravenous (IV) infusion 8 times
BT063: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
Placebo
Placebo administered by IV infusion 8 times
Placebo: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
|---|---|---|---|
|
Overall Study
STARTED
|
12
|
12
|
12
|
|
Overall Study
COMPLETED
|
10
|
12
|
10
|
|
Overall Study
NOT COMPLETED
|
2
|
0
|
2
|
Reasons for withdrawal
| Measure |
BT063 50 mg
50 mg BT063 administered by intravenous (IV) infusion 8 times
BT063: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
BT063 100 mg
100 mg BT063 administered by intravenous (IV) infusion 8 times
BT063: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
Placebo
Placebo administered by IV infusion 8 times
Placebo: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
|---|---|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
0
|
2
|
|
Overall Study
Adverse Event
|
1
|
0
|
0
|
Baseline Characteristics
Proof-of-Concept Study With BT063 in Subjects With Systemic Lupus Erythematosus
Baseline characteristics by cohort
| Measure |
BT063 50 mg
n=12 Participants
50 mg BT063 administered by intravenous (IV) infusion 8 times
BT063: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
BT063 100 mg
n=12 Participants
100 mg BT063 administered by intravenous (IV) infusion 8 times
BT063: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
Placebo
n=12 Participants
Placebo administered by IV infusion 8 times
Placebo: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
Total
n=36 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
37.4 years
STANDARD_DEVIATION 12.77 • n=93 Participants
|
50.7 years
STANDARD_DEVIATION 7.18 • n=4 Participants
|
48.6 years
STANDARD_DEVIATION 13.5 • n=27 Participants
|
45.6 years
STANDARD_DEVIATION 12.63 • n=483 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=93 Participants
|
11 Participants
n=4 Participants
|
12 Participants
n=27 Participants
|
33 Participants
n=483 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
3 Participants
n=483 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Race (NIH/OMB)
White
|
12 Participants
n=93 Participants
|
12 Participants
n=4 Participants
|
12 Participants
n=27 Participants
|
36 Participants
n=483 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
PRIMARY outcome
Timeframe: Baseline through End of Trial Visit (Week 14)Number of Participants with Adverse Events (Including SAEs and AEs leading to discontinuation) from Baseline through End of Trial Visit (Week 14)
Outcome measures
| Measure |
BT063 50 mg
n=12 Participants
50 mg BT063 administered by intravenous (IV) infusion 8 times
BT063: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
BT063 100 mg
n=12 Participants
100 mg BT063 administered by intravenous (IV) infusion 8 times
BT063: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
Placebo
n=12 Participants
Placebo administered by IV infusion 8 times
Placebo: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
|---|---|---|---|
|
Number of Participants With Adverse Events
Subjects with TEAEs
|
5 Participants
|
8 Participants
|
8 Participants
|
|
Number of Participants With Adverse Events
Subjects with TEAEs leading to early termination
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Adverse Events
Subjects with treatment-emergent SAEs
|
1 Participants
|
1 Participants
|
0 Participants
|
|
Number of Participants With Adverse Events
Subjects with drug-related TEAEs
|
1 Participants
|
2 Participants
|
0 Participants
|
|
Number of Participants With Adverse Events
Subjects with severe TEAEs
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Adverse Events
Subjects with TEAEs leading to death
|
0 Participants
|
0 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: Baseline through End of Trial Visit (Week 14)Number of Participants with changes in vital signs, ECGs, Safety laboratory parameters (full blood count including white differential count, clinical chemistry, thyroid hormones, urinalysis, and faecal occult blood test), Development of anti-drug antibodies against BT063 (anti-BT063), Immunological status of potential viral and bacterial infections (HBV, HCV, HIV, tetanus, diphtheria tuberculosis), EBV / CMV Serology, Premature withdrawals.
Outcome measures
| Measure |
BT063 50 mg
n=12 Participants
50 mg BT063 administered by intravenous (IV) infusion 8 times
BT063: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
BT063 100 mg
n=12 Participants
100 mg BT063 administered by intravenous (IV) infusion 8 times
BT063: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
Placebo
n=12 Participants
Placebo administered by IV infusion 8 times
Placebo: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
|---|---|---|---|
|
Number of Participants With Changes of Safety Parameters
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: At week14 and week 28Population: Intension-To-Treat Set included 36 subjects (12 of each Group). At week 14, 1 subject in Placebo Group had no end of Treatment result; at week 28, 1 subject of 50 mg and 2 subjects of Placebo had no results. The efficacy outcome was based on observed cases.
Number of Participants with 50% improvement of swollen/tender joints. A total of 66/68 joints was assessed for the swollen/tender joint count. A joint that is normal (no tenderness or swelling), without signs of inflammation will be graded as 0. A joint with tenderness will be graded as 1 for tender joint count and a joint with swelling will be graded as 1 for swollen joint count. Joints suspected or known to have ischemic osteonecrosis are not to be taken into consideration. Higher scores indicate more disease activity.
Outcome measures
| Measure |
BT063 50 mg
n=12 Participants
50 mg BT063 administered by intravenous (IV) infusion 8 times
BT063: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
BT063 100 mg
n=12 Participants
100 mg BT063 administered by intravenous (IV) infusion 8 times
BT063: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
Placebo
n=12 Participants
Placebo administered by IV infusion 8 times
Placebo: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
|---|---|---|---|
|
Number of Participants With Improvements of Joints
50% improvement in swollen joints at week 14
|
8 Participants
|
5 Participants
|
8 Participants
|
|
Number of Participants With Improvements of Joints
50% improvement in swollen joints at week 28
|
7 Participants
|
4 Participants
|
7 Participants
|
|
Number of Participants With Improvements of Joints
50% improvement in tender joints at week 14
|
5 Participants
|
7 Participants
|
6 Participants
|
|
Number of Participants With Improvements of Joints
50% improvement in tender joints at week 28
|
7 Participants
|
6 Participants
|
6 Participants
|
SECONDARY outcome
Timeframe: At week14 and week 28Population: Intension-To-Treat Set included 36 subjects (12 of each Group). At week 14, 1 subject in Placebo Group had no end of Treatment result; at week 28, 1 subject of 50 mg and 2 subjects of Placebo had no results. The efficacy outcome was based on observed cases.
Number of Participants with 50% improvement in Cutaneous Lupus Erythematosus Disease Area and Sensitivity Index (CLASI) Activity score. The CLASI is an assessment over 13 body regions (scalp, ears, nose - including malar area, rest of the face, V-area neck - frontal, post. neck \& shoulders, chest, abdomen, back and buttocks, arms, hands, legs, feet) and consists of 2 scores: total activity score and total damage score. Only the activity score was used in this study. The minimum score possible on this scale is 0 and the maximum score is 70. The higher scores mean a worse outcome.
Outcome measures
| Measure |
BT063 50 mg
n=12 Participants
50 mg BT063 administered by intravenous (IV) infusion 8 times
BT063: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
BT063 100 mg
n=12 Participants
100 mg BT063 administered by intravenous (IV) infusion 8 times
BT063: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
Placebo
n=12 Participants
Placebo administered by IV infusion 8 times
Placebo: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
|---|---|---|---|
|
Number of Participants With Improvement of Skin
50% improvement in CLASI Activity score at week 14
|
6 Participants
|
5 Participants
|
3 Participants
|
|
Number of Participants With Improvement of Skin
50% improvement in CLASI Activity score at week 28
|
7 Participants
|
4 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: Baseline to week 14 and at week 28Population: Intension-To-Treat Set included 36 subjects (12 of each Group). At week 14, 1 subject in Placebo Group had no end of Treatment result; at week 28, 1 subject of 50 mg and 2 subjects of Placebo had no results. The efficacy outcome was based on observed cases.
Percent changes in Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) scores from baseline SLEDAI-2K score. The SLEDAI-2K is a global index that measures SLE disease activity. It includes 24 items for the 9 organs/systems. Scores range from 0 to 105; a score of 6 is considered clinically important. The index measures disease activity within the last 10 days. Higher scores mean worse outcome. Negative percent change means reduced disease activity.
Outcome measures
| Measure |
BT063 50 mg
n=12 Participants
50 mg BT063 administered by intravenous (IV) infusion 8 times
BT063: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
BT063 100 mg
n=12 Participants
100 mg BT063 administered by intravenous (IV) infusion 8 times
BT063: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
Placebo
n=12 Participants
Placebo administered by IV infusion 8 times
Placebo: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
|---|---|---|---|
|
Percent Changes in Systemic Lupus Erythematosus Disease Activity Index 2000
Percent changes in SLEDAI-2K scores at week 14
|
-29.3 percentage of change
Standard Deviation 31.43
|
-18.0 percentage of change
Standard Deviation 37.304
|
-18.2 percentage of change
Standard Deviation 25.75
|
|
Percent Changes in Systemic Lupus Erythematosus Disease Activity Index 2000
Percent changes in SLEDAI-2K scores at week 28
|
-28.9 percentage of change
Standard Deviation 36.05
|
-24.4 percentage of change
Standard Deviation 35.19
|
-13.5 percentage of change
Standard Deviation 32.36
|
Adverse Events
BT063 50 mg
BT063 100 mg
Placebo
Serious adverse events
| Measure |
BT063 50 mg
n=12 participants at risk
50 mg BT063 administered by intravenous (IV) infusion 8 times
BT063: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
BT063 100 mg
n=12 participants at risk
100 mg BT063 administered by intravenous (IV) infusion 8 times
BT063: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
Placebo
n=12 participants at risk
Placebo administered by IV infusion 8 times
Placebo: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
|---|---|---|---|
|
Musculoskeletal and connective tissue disorders
Synovitis
|
8.3%
1/12 • Number of events 1 • Incidence of AEs (including SAEs and AEs leading to discontinuation) from baseline through End of Trial Visit (Week 14)
|
0.00%
0/12 • Incidence of AEs (including SAEs and AEs leading to discontinuation) from baseline through End of Trial Visit (Week 14)
|
0.00%
0/12 • Incidence of AEs (including SAEs and AEs leading to discontinuation) from baseline through End of Trial Visit (Week 14)
|
|
Infections and infestations
Bronchopneumonia
|
0.00%
0/12 • Incidence of AEs (including SAEs and AEs leading to discontinuation) from baseline through End of Trial Visit (Week 14)
|
8.3%
1/12 • Number of events 1 • Incidence of AEs (including SAEs and AEs leading to discontinuation) from baseline through End of Trial Visit (Week 14)
|
0.00%
0/12 • Incidence of AEs (including SAEs and AEs leading to discontinuation) from baseline through End of Trial Visit (Week 14)
|
|
Injury, poisoning and procedural complications
Overdose
|
0.00%
0/12 • Incidence of AEs (including SAEs and AEs leading to discontinuation) from baseline through End of Trial Visit (Week 14)
|
8.3%
1/12 • Number of events 1 • Incidence of AEs (including SAEs and AEs leading to discontinuation) from baseline through End of Trial Visit (Week 14)
|
0.00%
0/12 • Incidence of AEs (including SAEs and AEs leading to discontinuation) from baseline through End of Trial Visit (Week 14)
|
Other adverse events
| Measure |
BT063 50 mg
n=12 participants at risk
50 mg BT063 administered by intravenous (IV) infusion 8 times
BT063: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
BT063 100 mg
n=12 participants at risk
100 mg BT063 administered by intravenous (IV) infusion 8 times
BT063: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
Placebo
n=12 participants at risk
Placebo administered by IV infusion 8 times
Placebo: Repeated IV infusions over 12 weeks (at weeks 0, 1, 2, 4, 6, 8, 10, 12)
|
|---|---|---|---|
|
General disorders
ASTHENIA
|
0.00%
0/12 • Incidence of AEs (including SAEs and AEs leading to discontinuation) from baseline through End of Trial Visit (Week 14)
|
16.7%
2/12 • Number of events 2 • Incidence of AEs (including SAEs and AEs leading to discontinuation) from baseline through End of Trial Visit (Week 14)
|
0.00%
0/12 • Incidence of AEs (including SAEs and AEs leading to discontinuation) from baseline through End of Trial Visit (Week 14)
|
|
General disorders
FATIGUE
|
0.00%
0/12 • Incidence of AEs (including SAEs and AEs leading to discontinuation) from baseline through End of Trial Visit (Week 14)
|
8.3%
1/12 • Number of events 1 • Incidence of AEs (including SAEs and AEs leading to discontinuation) from baseline through End of Trial Visit (Week 14)
|
8.3%
1/12 • Number of events 1 • Incidence of AEs (including SAEs and AEs leading to discontinuation) from baseline through End of Trial Visit (Week 14)
|
|
Infections and infestations
BRONCHITIS
|
8.3%
1/12 • Number of events 1 • Incidence of AEs (including SAEs and AEs leading to discontinuation) from baseline through End of Trial Visit (Week 14)
|
0.00%
0/12 • Incidence of AEs (including SAEs and AEs leading to discontinuation) from baseline through End of Trial Visit (Week 14)
|
8.3%
1/12 • Number of events 1 • Incidence of AEs (including SAEs and AEs leading to discontinuation) from baseline through End of Trial Visit (Week 14)
|
|
Infections and infestations
NASOPHARYNGITIS
|
0.00%
0/12 • Incidence of AEs (including SAEs and AEs leading to discontinuation) from baseline through End of Trial Visit (Week 14)
|
8.3%
1/12 • Number of events 1 • Incidence of AEs (including SAEs and AEs leading to discontinuation) from baseline through End of Trial Visit (Week 14)
|
8.3%
1/12 • Number of events 1 • Incidence of AEs (including SAEs and AEs leading to discontinuation) from baseline through End of Trial Visit (Week 14)
|
|
Infections and infestations
PHARYNGITIS
|
8.3%
1/12 • Number of events 1 • Incidence of AEs (including SAEs and AEs leading to discontinuation) from baseline through End of Trial Visit (Week 14)
|
8.3%
1/12 • Number of events 1 • Incidence of AEs (including SAEs and AEs leading to discontinuation) from baseline through End of Trial Visit (Week 14)
|
8.3%
1/12 • Number of events 1 • Incidence of AEs (including SAEs and AEs leading to discontinuation) from baseline through End of Trial Visit (Week 14)
|
|
Vascular disorders
HYPERTENSION
|
0.00%
0/12 • Incidence of AEs (including SAEs and AEs leading to discontinuation) from baseline through End of Trial Visit (Week 14)
|
8.3%
1/12 • Number of events 1 • Incidence of AEs (including SAEs and AEs leading to discontinuation) from baseline through End of Trial Visit (Week 14)
|
16.7%
2/12 • Number of events 3 • Incidence of AEs (including SAEs and AEs leading to discontinuation) from baseline through End of Trial Visit (Week 14)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The Investigator shall not publish, present, or use any Study data or results arising out of the performance of the Study for their own instruction, research, or publication purposes without the prior express written consent of Sponsor, which consent may be withheld at Sponsor's discretion.
- Publication restrictions are in place
Restriction type: OTHER