Trial Outcomes & Findings for Safety and Efficacy of Abatacept in IgG4-Related Disease (NCT NCT03669861)

NCT ID: NCT03669861

Last Updated: 2021-08-24

Results Overview

Effect of weekly subcutaneous (SC) administration of abatacept on complete remission

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 10 participants
• Primary outcome timeframe: 24 weeks
• Results posted on: 2021-08-24

Participant Flow

Concurrent glucocorticoid treatment was permitted but was required to be discontinued by week 4.

Participant milestones

Measure	Abatacept To assess the effect of weekly subcutaneous (SC) administration of abatacept on complete remission of IgG4-RD Abatacept: Subjects will receive weekly subcutaneous doses of abatacept (125mg) for 24 doses (24 weeks)
Overall Study STARTED	10
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	7

Reasons for withdrawal

Measure	Abatacept To assess the effect of weekly subcutaneous (SC) administration of abatacept on complete remission of IgG4-RD Abatacept: Subjects will receive weekly subcutaneous doses of abatacept (125mg) for 24 doses (24 weeks)
Overall Study Disease Flare	5
Overall Study Lack of Efficacy	2

Baseline Characteristics

Safety and Efficacy of Abatacept in IgG4-Related Disease

Baseline characteristics by cohort

Measure	Abatacept n=10 Participants To assess the effect of weekly subcutaneous (SC) administration of abatacept on complete remission of IgG4-RD. Abatacept: Subjects will receive weekly subcutaneous doses of abatacept (125mg) for 24 doses (24 weeks). The protocol called for a final study visit at 36 weeks, three months after the patients discontinued their active treatment, primarily for safety
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=5 Participants
Age, Categorical >=65 years	6 Participants n=5 Participants
Age, Continuous	64.8 years n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	7 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	9 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	9 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	10 participants n=5 Participants
Baseline Serum IgG4	597 units on a scale n=5 Participants
Baseline Serum IgE	480.5 IU n=5 Participants
Disease activity: IgG4-RD Responder Index score	7.2 points range 0-75 n=5 Participants
BAS-Duration of IgG4-RD (months)	54.4 months n=5 Participants
Number of active organs affected	3.4 count of number of organs involved n=5 Participants

PRIMARY outcome

Timeframe: 24 weeks

Population: The primary outcome, complete remission at week 24

Effect of weekly subcutaneous (SC) administration of abatacept on complete remission

Outcome measures

Measure	Abatacept n=10 Participants To assess the effect of weekly subcutaneous (SC) administration of abatacept on complete remission of IgG4-RD Abatacept: Subjects will receive weekly subcutaneous doses of abatacept (125mg) for 24 doses (24 weeks)
Treatment Response	3 Participants

SECONDARY outcome

Timeframe: 12 weeks

Population: All 10 patients were treated initially with abatacept

Assess the effect of abatacept on disease response at week 12

Outcome measures

Measure	Abatacept n=10 Participants To assess the effect of weekly subcutaneous (SC) administration of abatacept on complete remission of IgG4-RD Abatacept: Subjects will receive weekly subcutaneous doses of abatacept (125mg) for 24 doses (24 weeks)
Disease Response	6 Participants

SECONDARY outcome

Timeframe: disease response at 24 weeks

Population: disease response at week 24

Percentage of patients achieving disease response at week 24

Outcome measures

Measure	Abatacept n=10 Participants To assess the effect of weekly subcutaneous (SC) administration of abatacept on complete remission of IgG4-RD Abatacept: Subjects will receive weekly subcutaneous doses of abatacept (125mg) for 24 doses (24 weeks)
Disease Response at Week 24	5 Participants

SECONDARY outcome

Timeframe: 24 weeks

Population: Subjects with reported disease flare on treatment. Disease flare was defined as recurrence of disease activity or demonstration of a disease exacerbation such that additional therapy beyond the trial protocol was indicated

number of disease flares per subject

Outcome measures

Measure	Abatacept n=10 Participants To assess the effect of weekly subcutaneous (SC) administration of abatacept on complete remission of IgG4-RD Abatacept: Subjects will receive weekly subcutaneous doses of abatacept (125mg) for 24 doses (24 weeks)
Disease Remission: Flares Over Time Per Subject	0.2 units on a scale Interval 0.0 to 2.0

SECONDARY outcome

Timeframe: 24 Weeks

Population: serum IgG4 concentration can be used as a surrogate marker for disease activity

Serum IgG4 measured at baseline and week 24

Outcome measures

Measure	Abatacept n=6 Participants To assess the effect of weekly subcutaneous (SC) administration of abatacept on complete remission of IgG4-RD Abatacept: Subjects will receive weekly subcutaneous doses of abatacept (125mg) for 24 doses (24 weeks)
Decline in Serum IgG4 Concentration of Responders	3 Participants

SECONDARY outcome

Timeframe: 24 weeks

Population: Serum IgE concentrations declined in all 6 patients (100%) who demonstrated a disease response with abatacept

Serum IgE concentration was measured at baseline and Week 24

Outcome measures

Measure	Abatacept n=6 Participants To assess the effect of weekly subcutaneous (SC) administration of abatacept on complete remission of IgG4-RD Abatacept: Subjects will receive weekly subcutaneous doses of abatacept (125mg) for 24 doses (24 weeks)
Decline in Serum IgE Concentration of Responders	6 Participants

Adverse Events

Abatacept

Serious events: 1 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Measure

Abatacept n=10 participants at risk To assess the effect of weekly subcutaneous (SC) administration of abatacept on complete remission of IgG4-RD Abatacept: Subjects will receive weekly subcutaneous doses of abatacept (125mg) for 24 doses (24 weeks)

Cardiac disorders Myocardial Infarction

10.0% 1/10 • Number of events 1 • Adverse Event monitoring (from time of study drug administration) to end of study (visit 10 (week 36) or safety follow up visit, if needed). Ten subjects were enrolled in the study between December 2018 and August 2019. Adverse Event (AE) is defined as any new untoward medical occurrence or worsening of a preexisting medical condition in a clinical investigation participant administered study drug a nd that does not necessarily have a causal relationship with this treatment. A Serious Adverse Event (SAE) is any untoward medical occurrence that at any dose: results in death; is life-threatening; requires inpatient hospitalization or causes prolongation of existing hospitalization; results in persistent or significant disability/incapacity/congenital anomaly. All Serious Adverse Events (SAEs) that occur following the subject's written consent to participate in the study through 30 days of discontinuation of dosing must be reported.

Other adverse events

Measure	Abatacept n=10 participants at risk To assess the effect of weekly subcutaneous (SC) administration of abatacept on complete remission of IgG4-RD Abatacept: Subjects will receive weekly subcutaneous doses of abatacept (125mg) for 24 doses (24 weeks)
Blood and lymphatic system disorders thrombocytopenia	10.0% 1/10 • Number of events 1 • Adverse Event monitoring (from time of study drug administration) to end of study (visit 10 (week 36) or safety follow up visit, if needed). Ten subjects were enrolled in the study between December 2018 and August 2019. Adverse Event (AE) is defined as any new untoward medical occurrence or worsening of a preexisting medical condition in a clinical investigation participant administered study drug a nd that does not necessarily have a causal relationship with this treatment. A Serious Adverse Event (SAE) is any untoward medical occurrence that at any dose: results in death; is life-threatening; requires inpatient hospitalization or causes prolongation of existing hospitalization; results in persistent or significant disability/incapacity/congenital anomaly. All Serious Adverse Events (SAEs) that occur following the subject's written consent to participate in the study through 30 days of discontinuation of dosing must be reported.
Eye disorders episcleritis	10.0% 1/10 • Number of events 1 • Adverse Event monitoring (from time of study drug administration) to end of study (visit 10 (week 36) or safety follow up visit, if needed). Ten subjects were enrolled in the study between December 2018 and August 2019. Adverse Event (AE) is defined as any new untoward medical occurrence or worsening of a preexisting medical condition in a clinical investigation participant administered study drug a nd that does not necessarily have a causal relationship with this treatment. A Serious Adverse Event (SAE) is any untoward medical occurrence that at any dose: results in death; is life-threatening; requires inpatient hospitalization or causes prolongation of existing hospitalization; results in persistent or significant disability/incapacity/congenital anomaly. All Serious Adverse Events (SAEs) that occur following the subject's written consent to participate in the study through 30 days of discontinuation of dosing must be reported.
Gastrointestinal disorders abdominal pain	10.0% 1/10 • Number of events 1 • Adverse Event monitoring (from time of study drug administration) to end of study (visit 10 (week 36) or safety follow up visit, if needed). Ten subjects were enrolled in the study between December 2018 and August 2019. Adverse Event (AE) is defined as any new untoward medical occurrence or worsening of a preexisting medical condition in a clinical investigation participant administered study drug a nd that does not necessarily have a causal relationship with this treatment. A Serious Adverse Event (SAE) is any untoward medical occurrence that at any dose: results in death; is life-threatening; requires inpatient hospitalization or causes prolongation of existing hospitalization; results in persistent or significant disability/incapacity/congenital anomaly. All Serious Adverse Events (SAEs) that occur following the subject's written consent to participate in the study through 30 days of discontinuation of dosing must be reported.

Additional Information

DRAI Director of Clinical Trials

Massachusetts General Hospital

Phone: 6176920668

Email: JHSTONE@mgh.harvard.edu

Results disclosure agreements

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