Trial Outcomes & Findings for Safety, Tolerability, and Efficacy of JBT-101 in Subjects With Dermatomyositis (NCT NCT02466243)

Last Updated: 2023-01-19

Results Overview

The CDASI is a validated outcome measure that systematically quantifies cutaneous DM disease activity, In the CDASI, DM skin disease activity is scored from 0 to 100 based on the physician's evaluation of erythema, scale, and erosion or ulceration at 15 anatomic locations as well as alopecia, Gottron's sign or papules on the hands, and periungual changes. A 5-point or greater decrease in the CDASI activity score indicates clinically relevant improvement based on statistical analysis using a receiver operating characteristic curve to maximize sensitivity and specificity

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

22 participants

Primary outcome timeframe

Part A: 84-day treatment period (Change from the Baseline CDSAI score at Day 84)

Results posted on

2023-01-19

Participant Flow

Participant milestones

Participant milestones
Measure	JBT-101 Part A: JBT-101 20 mg capsule once a day on Days 1-28, then 20 mg capsule twice a day on Days 29-84. Part B: JBT-101 20 mg twice daily on Days 1 - 365 of the OLE. JBT-101: Part A: 20 mg once daily on Days 1-28, then 20 mg twice daily on Days 29-84. Part B: JBT-101 20 mg twice daily on Days 1 - 365 of the OLE.	Placebo Part A: Placebo capsule once a day on Days 1-28, then placebo capsule twice a day on Days 29-84. Part B: Placebo twice daily on Days 1 - 365 of the OLE. Placebo: Part A: Once daily on Days 1-28, then twice daily on Days 29-84. Part B: Placebo twice daily on Days 1 - 365 of the OLE.
Part A Treatment Period (Double Blind) STARTED	11	11
Part A Treatment Period (Double Blind) COMPLETED	11	11
Part A Treatment Period (Double Blind) NOT COMPLETED	0	0
Part B Open-label Treatment Period STARTED	11	10
Part B Open-label Treatment Period COMPLETED	0	0
Part B Open-label Treatment Period NOT COMPLETED	11	10

Reasons for withdrawal

Reasons for withdrawal
Measure	JBT-101 Part A: JBT-101 20 mg capsule once a day on Days 1-28, then 20 mg capsule twice a day on Days 29-84. Part B: JBT-101 20 mg twice daily on Days 1 - 365 of the OLE. JBT-101: Part A: 20 mg once daily on Days 1-28, then 20 mg twice daily on Days 29-84. Part B: JBT-101 20 mg twice daily on Days 1 - 365 of the OLE.	Placebo Part A: Placebo capsule once a day on Days 1-28, then placebo capsule twice a day on Days 29-84. Part B: Placebo twice daily on Days 1 - 365 of the OLE. Placebo: Part A: Once daily on Days 1-28, then twice daily on Days 29-84. Part B: Placebo twice daily on Days 1 - 365 of the OLE.
Part B Open-label Treatment Period Adverse Event	1	0
Part B Open-label Treatment Period Withdrawal by Subject	0	2
Part B Open-label Treatment Period Sponsor Decision	8	6
Part B Open-label Treatment Period Missing	2	2

Baseline Characteristics

Safety, Tolerability, and Efficacy of JBT-101 in Subjects With Dermatomyositis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	JBT-101 n=11 Participants Part A: JBT-101 20 mg capsule once a day on Days 1-28, then 20 mg capsule twice a day on Days 29-84. Part B: JBT-101 20 mg twice daily on Days 1 - 365 of the OLE. JBT-101: Part A: 20 mg once daily on Days 1-28, then 20 mg twice daily on Days 29-84. Part B: JBT-101 20 mg twice daily on Days 1 - 365 of the OLE.	Placebo n=11 Participants Part A: Placebo capsule once a day on Days 1-28, then placebo capsule twice a day on Days 29-84. Part B: Placebo twice daily on Days 1 - 365 of the OLE. Placebo: Part A: Once daily on Days 1-28, then twice daily on Days 29-84. Part B: Placebo twice daily on Days 1 - 365 of the OLE.	Total n=22 Participants Total of all reporting groups
Age, Continuous	53.1 years STANDARD_DEVIATION 9.31 • n=5 Participants	52.5 years STANDARD_DEVIATION 10.44 • n=7 Participants	52.8 years STANDARD_DEVIATION 9.66 • n=5 Participants
Sex: Female, Male Female	10 Participants n=5 Participants	11 Participants n=7 Participants	21 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	3 Participants n=5 Participants	0 Participants n=7 Participants	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	8 Participants n=5 Participants	11 Participants n=7 Participants	19 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 White	11 Participants n=5 Participants	10 Participants n=7 Participants	21 Participants n=5 Participants
Race Black or African American	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: Part A: 84-day treatment period (Change from the Baseline CDSAI score at Day 84)

Population: modified intent-to-treat population LS mean (SE) at Day 84

Outcome measures

Outcome measures
Measure	Lenabasum n=11 Participants lenabasum 20 mg BID	Placebo n=11 Participants Placebo BID
Change in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) From Baseline in Part A.	-8.0 CDASI Activity Score Standard Error 8.16	-5.5 CDASI Activity Score Standard Error 5.56

PRIMARY outcome

Timeframe: Part A: to Day 84

Population: Safety Population

Number of participants with treatment emergent adverse events were assessed as a measure of safety and tolerability

Outcome measures

Outcome measures
Measure	Lenabasum n=11 Participants lenabasum 20 mg BID	Placebo n=11 Participants Placebo BID
Number of Participants With Treatment Emergent Adverse Events as a Measure of Safety and Tolerability Any TEAE	11 Participants	8 Participants
Number of Participants With Treatment Emergent Adverse Events as a Measure of Safety and Tolerability Any serious TEAE	0 Participants	0 Participants
Number of Participants With Treatment Emergent Adverse Events as a Measure of Safety and Tolerability Any TEAE leading to discontinuation	0 Participants	0 Participants
Number of Participants With Treatment Emergent Adverse Events as a Measure of Safety and Tolerability Any severe TEAE	0 Participants	0 Participants
Number of Participants With Treatment Emergent Adverse Events as a Measure of Safety and Tolerability No TEAE	0 Participants	3 Participants

SECONDARY outcome

Timeframe: Part A: 84-day treatment period

Population: modified intent to treat population

LS mean (SE) change from baseline to Week 6 (Day 84) for lenabasum vs. placebo using a mixed model repeated measures analysis The CDASI is a validated outcome measure that systematically quantifies cutaneous DM disease activity and damage, In the CDASI, the Damage Score is scored from 0 to 32 based on the physician's evaluation of poikiloderma and calcinosis. 0 representing no damage and 32 representing the greatest level of damage.

Outcome measures

Outcome measures
Measure	Lenabasum n=11 Participants lenabasum 20 mg BID	Placebo n=11 Participants Placebo BID
Change in Patient-reported Outcomes From Baseline at 84 Days for Part A	-5.5 CDASI Damage Score Standard Error 5.56	-8.0 CDASI Damage Score Standard Error 8.16

Adverse Events

JBT-101

Serious events: 0 serious events

Other events: 11 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	JBT-101 n=11 participants at risk Part A: JBT-101 20 mg capsule once a day on Days 1-28, then 20 mg capsule twice a day on Days 29-84. Part B: JBT-101 20 mg twice daily on Days 1 - 365 of the OLE. JBT-101: Part A: 20 mg once daily on Days 1-28, then 20 mg twice daily on Days 29-84. Part B: JBT-101 20 mg twice daily on Days 1 - 365 of the OLE.	Placebo n=11 participants at risk Part A: Placebo capsule once a day on Days 1-28, then placebo capsule twice a day on Days 29-84. Part B: Placebo twice daily on Days 1 - 365 of the OLE. Placebo: Part A: Once daily on Days 1-28, then twice daily on Days 29-84. Part B: Placebo twice daily on Days 1 - 365 of the OLE.
Gastrointestinal disorders Diarrhoea	27.3% 3/11 • Number of events 3 • 84 days	9.1% 1/11 • Number of events 2 • 84 days
Gastrointestinal disorders Dry mouth	36.4% 4/11 • Number of events 4 • 84 days	9.1% 1/11 • Number of events 1 • 84 days
Gastrointestinal disorders Dyspepsia	18.2% 2/11 • Number of events 2 • 84 days	0.00% 0/11 • 84 days
Gastrointestinal disorders Nausea	9.1% 1/11 • Number of events 1 • 84 days	18.2% 2/11 • Number of events 2 • 84 days
General disorders Fatigue	9.1% 1/11 • Number of events 1 • 84 days	27.3% 3/11 • Number of events 3 • 84 days
Infections and infestations Upper respiratory tract infection	0.00% 0/11 • 84 days	18.2% 2/11 • Number of events 2 • 84 days
Injury, poisoning and procedural complications Fall	18.2% 2/11 • Number of events 3 • 84 days	0.00% 0/11 • 84 days
Metabolism and nutrition disorders Increased appetite	18.2% 2/11 • Number of events 2 • 84 days	9.1% 1/11 • Number of events 1 • 84 days
Nervous system disorders Dizziness	45.5% 5/11 • Number of events 8 • 84 days	18.2% 2/11 • Number of events 2 • 84 days
Nervous system disorders Headache	18.2% 2/11 • Number of events 2 • 84 days	0.00% 0/11 • 84 days

Additional Information

Head of Clinical Operations

Corbus Pharmaceuticals, Inc.

Phone: 6179630100

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place