Trial Outcomes & Findings for Ustekinumab for the Treatment of Giant Cell Arteritis (NCT NCT02955147)

NCT ID: NCT02955147

Last Updated: 2020-06-12

Results Overview

The primary study endpoint, prednisone-free remission, was defined as: 1) absence of relapse from the time that remission was achieved through week 52; 2) normalization of ESR (\<40 mm/hour) and CRP (\<10 mg/L); and, 3) adherence to the protocol prednisone taper.

• Recruitment status: TERMINATED
• Study phase: PHASE1/PHASE2
• Target enrollment: 13 participants
• Primary outcome timeframe: 52 weeks
• Results posted on: 2020-06-12

Participant Flow

Between December 2016 and August 2018, we screened 16 GCA patients for this trial. Three patients failed the screening process and 13 patients were enrolled.

Three patients failed the screening due to dementia (N = 1), severe prednisone-induced depression (N = 1), and positive hepatitis B core antibody (N = 1)

Participant milestones

Measure	Ustekinumab Plus Prednisone 1. Ustekinumab 90 mg was administered subcutaneously at baseline, week 4, week 12, week 20, week 28, week 36 and week 44. Ustekinumab: Ustekinumab is a humanized monoclonal antibody that targets the p40 subunit of IL-12 and IL-23 and inhibits cytokine - cytokine receptor coupling and signaling 2. All patients received a 6 month prednisone course tapered according to predefined schedules starting at either 60 mg, 40 mg or 20 mg. The initial dose of prednisone was chosen by the investigators according to disease severity and comorbid medical conditions. The initial prednisone dose was 60 mg in 3 patients, 40 mg in 9 patients, and 20 mg in 1 patient. Prednisone is an anti-inflammatory medication
Overall Study STARTED	13
Overall Study COMPLETED	13
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Ustekinumab for the Treatment of Giant Cell Arteritis

Baseline characteristics by cohort

Measure	Ustekinumab Plus Prednisone n=13 Participants 1. Ustekinumab: 90 mg of ustekinumab will be administered subcutaneously at baseline, week 4, week 12, week 20, week 28, week 36 and week 44. 2. Prednisone: All patients will receive a prednisone course tapered according to predefined schedules starting at either 60 mg, 40 mg or 20 mg. The initial dose of prednisone will be chosen by the investigators according to disease severity and comorbid medical conditions. The duration of the prednisone taper will be 6 months in all cases. Ustekinumab: Ustekinumab is a humanized monoclonal antibody that targets the p40 subunit of IL-12 and IL-23 and inhibits cytokine - cytokine receptor coupling and signaling Prednisone: Prednisone is an anti-inflammatory medication
Age, Categorical <=18 years	0 Participants n=93 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=93 Participants
Age, Categorical >=65 years	11 Participants n=93 Participants
Age, Continuous	71 years STANDARD_DEVIATION 7 • n=93 Participants
Sex: Female, Male Female	11 Participants n=93 Participants
Sex: Female, Male Male	2 Participants n=93 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=93 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	13 Participants n=93 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants
Race (NIH/OMB) Black or African American	0 Participants n=93 Participants
Race (NIH/OMB) White	13 Participants n=93 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants
Region of Enrollment United States	13 participants n=93 Participants

PRIMARY outcome

Timeframe: 52 weeks

The primary study endpoint, prednisone-free remission, was defined as: 1) absence of relapse from the time that remission was achieved through week 52; 2) normalization of ESR (<40 mm/hour) and CRP (<10 mg/L); and, 3) adherence to the protocol prednisone taper.

Outcome measures

Measure	Ustekinumab Plus Prednisone n=13 Participants 1. Ustekinumab 90 mg was administered subcutaneously at baseline, week 4, week 12, week 20, week 28, week 36 and week 44. 2. All patients received a 6 month prednisone course tapered according to predefined schedules starting at either 60 mg (n = 3), 40 mg (n = 9) or 20 mg (n = 1).
Percentage of Patients in Glucocorticoid-free Remission	3 Participants

SECONDARY outcome

Timeframe: 52 weeks

Disease relapse was defined as the recurrence of signs or symptoms of GCA (e.g., cranial or PMR) that required treatment intensification, regardless of the ESR and CRP levels.

Outcome measures

Measure	Ustekinumab Plus Prednisone n=13 Participants 1. Ustekinumab 90 mg was administered subcutaneously at baseline, week 4, week 12, week 20, week 28, week 36 and week 44. 2. All patients received a 6 month prednisone course tapered according to predefined schedules starting at either 60 mg (n = 3), 40 mg (n = 9) or 20 mg (n = 1).
Number of Participants With Disease Flare	7 Participants

SECONDARY outcome

Timeframe: 52 weeks

Outcome measures

Measure	Ustekinumab Plus Prednisone n=13 Participants 1. Ustekinumab 90 mg was administered subcutaneously at baseline, week 4, week 12, week 20, week 28, week 36 and week 44. 2. All patients received a 6 month prednisone course tapered according to predefined schedules starting at either 60 mg (n = 3), 40 mg (n = 9) or 20 mg (n = 1).
Cumulative Prednisone Dose	2289 mg of prednisone Standard Deviation 498

OTHER_PRE_SPECIFIED outcome

Timeframe: 52 weeks

Outcome measures

Measure	Ustekinumab Plus Prednisone n=13 Participants 1. Ustekinumab 90 mg was administered subcutaneously at baseline, week 4, week 12, week 20, week 28, week 36 and week 44. 2. All patients received a 6 month prednisone course tapered according to predefined schedules starting at either 60 mg (n = 3), 40 mg (n = 9) or 20 mg (n = 1).
Number of Participants With at Least One Adverse Event	12 Participants

Adverse Events

Ustekinumab Plus Prednisone

Serious events: 1 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Measure

Ustekinumab Plus Prednisone n=13 participants at risk 1. Ustekinumab 90 mg was administered subcutaneously at baseline, week 4, week 12, week 20, week 28, week 36 and week 44. Ustekinumab: Ustekinumab is a humanized monoclonal antibody that targets the p40 subunit of IL-12 and IL-23 and inhibits cytokine - cytokine receptor coupling and signaling 2. All patients received a 6 month prednisone course tapered according to predefined schedules starting at either 60 mg, 40 mg or 20 mg. The initial dose of prednisone was chosen by the investigators according to disease severity and comorbid medical conditions. The initial prednisone dose was 60 mg in 3 patients, 40 mg in 9 patients, and 20 mg in 1 patient. Prednisone is an anti-inflammatory medication

Gastrointestinal disorders Diverticulitis

7.7% 1/13 • Number of events 1 • 60 weeks

Other adverse events

Measure	Ustekinumab Plus Prednisone n=13 participants at risk 1. Ustekinumab 90 mg was administered subcutaneously at baseline, week 4, week 12, week 20, week 28, week 36 and week 44. Ustekinumab: Ustekinumab is a humanized monoclonal antibody that targets the p40 subunit of IL-12 and IL-23 and inhibits cytokine - cytokine receptor coupling and signaling 2. All patients received a 6 month prednisone course tapered according to predefined schedules starting at either 60 mg, 40 mg or 20 mg. The initial dose of prednisone was chosen by the investigators according to disease severity and comorbid medical conditions. The initial prednisone dose was 60 mg in 3 patients, 40 mg in 9 patients, and 20 mg in 1 patient. Prednisone is an anti-inflammatory medication
Respiratory, thoracic and mediastinal disorders Pneumonia	7.7% 1/13 • Number of events 1 • 60 weeks
Respiratory, thoracic and mediastinal disorders Bronchitis	7.7% 1/13 • Number of events 1 • 60 weeks
Gastrointestinal disorders Gastroenteritis	7.7% 1/13 • Number of events 1 • 60 weeks
Renal and urinary disorders urinary tract infection	7.7% 1/13 • Number of events 1 • 60 weeks

Additional Information

Dr. Sebastian Unizony

Mass General Hospital

Phone: (617) 726-7938

Email: sunizony@mgh.harvard.edu

Results disclosure agreements

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