Trial Outcomes & Findings for Treatment of Resistant Nephrotic Syndrome With ACTH Gel (ACTHAR) (NCT NCT01129284)
NCT ID: NCT01129284
Last Updated: 2014-04-24
Results Overview
Complete remission is defined as stable or improved renal function, serum creatinine \< or = 125% baseline, with proteinuria falling \<500 mg/day at month 6. Partial remission is defined as stable or improved renal function, serum creatinine \< or = 125% baseline, with 50% reduction in proteinuria and final proteinuria 500-3500 mg/day at month 6.
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
15 participants
Primary outcome timeframe
6 months
Results posted on
2014-04-24
Participant Flow
15 subjects with resistant glomerular diseases were enrolled and treated with ACTH gel (80 units subcutaneously twice weekly) for 6 months.
Participant milestones
| Measure |
Membranous Nephropathy
Subjects diagnosed with membranous nephropathy were treated with ACTH gel (80 units subcutaneously twice a week) for 6 months.
|
FSGS/MCD
Subjects diagnosed with focal segmental glomerulosclerosis (FSGS) or minimal change disease (MCD) were treated with ACTH gel (80 units subcutaneously twice a week) for 6 months.
|
Immunoglobulin A (IgA) Nephropathy
Subjects diagnosed with Immunoglobulin A (IgA) nephropathy (Berger's disease) were treated with ACTH gel (80 units subcutaneously twice a week) for 6 months.
|
|---|---|---|---|
|
Overall Study
STARTED
|
5
|
5
|
5
|
|
Overall Study
COMPLETED
|
3
|
5
|
4
|
|
Overall Study
NOT COMPLETED
|
2
|
0
|
1
|
Reasons for withdrawal
| Measure |
Membranous Nephropathy
Subjects diagnosed with membranous nephropathy were treated with ACTH gel (80 units subcutaneously twice a week) for 6 months.
|
FSGS/MCD
Subjects diagnosed with focal segmental glomerulosclerosis (FSGS) or minimal change disease (MCD) were treated with ACTH gel (80 units subcutaneously twice a week) for 6 months.
|
Immunoglobulin A (IgA) Nephropathy
Subjects diagnosed with Immunoglobulin A (IgA) nephropathy (Berger's disease) were treated with ACTH gel (80 units subcutaneously twice a week) for 6 months.
|
|---|---|---|---|
|
Overall Study
Adverse Event
|
2
|
0
|
1
|
Baseline Characteristics
Treatment of Resistant Nephrotic Syndrome With ACTH Gel (ACTHAR)
Baseline characteristics by cohort
| Measure |
Membranous Nephropathy
n=5 Participants
Subjects diagnosed with membranous nephropathy
|
FSGS/MCD
n=5 Participants
Subjects diagnosed with focal segmental glomerulosclerosis (FSGS) or minimal change disease (MCD)
|
IgA Nephropathy
n=5 Participants
Subjects diagnosed with IgA nephropathy (Berger's disease)
|
Total
n=15 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
2 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
11 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
3 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
4 Participants
n=4 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
10 Participants
n=4 Participants
|
|
Race/Ethnicity, Customized
White
|
3 participants
n=5 Participants
|
4 participants
n=7 Participants
|
1 participants
n=5 Participants
|
8 participants
n=4 Participants
|
|
Race/Ethnicity, Customized
African American
|
1 participants
n=5 Participants
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
1 participants
n=4 Participants
|
|
Race/Ethnicity, Customized
Asian
|
1 participants
n=5 Participants
|
0 participants
n=7 Participants
|
3 participants
n=5 Participants
|
4 participants
n=4 Participants
|
|
Race/Ethnicity, Customized
Hispanic
|
0 participants
n=5 Participants
|
1 participants
n=7 Participants
|
1 participants
n=5 Participants
|
2 participants
n=4 Participants
|
|
Previous Immunosuppressive therapy
steroids
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
2 participants
n=5 Participants
|
2 participants
n=4 Participants
|
|
Previous Immunosuppressive therapy
steroids, cyclosporine
|
0 participants
n=5 Participants
|
1 participants
n=7 Participants
|
0 participants
n=5 Participants
|
1 participants
n=4 Participants
|
|
Previous Immunosuppressive therapy
steroids, cyclosporine, MMF
|
0 participants
n=5 Participants
|
1 participants
n=7 Participants
|
0 participants
n=5 Participants
|
1 participants
n=4 Participants
|
|
Previous Immunosuppressive therapy
steroids, cyclosporine, tacrolimus
|
1 participants
n=5 Participants
|
1 participants
n=7 Participants
|
0 participants
n=5 Participants
|
2 participants
n=4 Participants
|
|
Previous Immunosuppressive therapy
steroids, MMF
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
1 participants
n=5 Participants
|
1 participants
n=4 Participants
|
|
Previous Immunosuppressive therapy
steroids, MMF, cyclophosphamide
|
1 participants
n=5 Participants
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
1 participants
n=4 Participants
|
|
Previous Immunosuppressive therapy
steroids, tacrolimus
|
0 participants
n=5 Participants
|
1 participants
n=7 Participants
|
0 participants
n=5 Participants
|
1 participants
n=4 Participants
|
|
Previous Immunosuppressive therapy
steroids, tacrolimus, cyclosporine, MMF
|
0 participants
n=5 Participants
|
1 participants
n=7 Participants
|
0 participants
n=5 Participants
|
1 participants
n=4 Participants
|
|
Previous Immunosuppressive therapy
cyclosporine, MMF
|
1 participants
n=5 Participants
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
1 participants
n=4 Participants
|
|
Previous Immunosuppressive therapy
MMF, cyclophosphamide
|
1 participants
n=5 Participants
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
1 participants
n=4 Participants
|
|
Previous Immunosuppressive therapy
MMF, tacrolimus, cyclosporine, rituximab
|
1 participants
n=5 Participants
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
1 participants
n=4 Participants
|
|
Previous Immunosuppressive therapy
none
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
2 participants
n=5 Participants
|
2 participants
n=4 Participants
|
|
Renal Function
Impaired function: sCr range 1.6-2.9 mg/dl
|
5 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
8 Participants
n=4 Participants
|
|
Renal Function
Preserved function: sCr range 0.6-1.2 mg/dl
|
0 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
|
Renal Function
Preserved function: eGFR>60 ml/min/1.73m2
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: 6 monthsComplete remission is defined as stable or improved renal function, serum creatinine \< or = 125% baseline, with proteinuria falling \<500 mg/day at month 6. Partial remission is defined as stable or improved renal function, serum creatinine \< or = 125% baseline, with 50% reduction in proteinuria and final proteinuria 500-3500 mg/day at month 6.
Outcome measures
| Measure |
Membranous Nephropathy
n=5 Participants
Subjects diagnosed with membranous nephropathy, treated with ACTH gel (80 units subcutaneously twice a week) for 6 months
|
FSGS/MCD
n=5 Participants
Subjects diagnosed with focal segmental glomerulosclerosis (FSGS) or minimal change disease (MCD), treated with ACTH gel (80 units subcutaneously twice a week) for 6 months
|
IgA Nephropathy
n=5 Participants
Subjects diagnosed with IgA nephropathy (Berger's disease), treated with ACTH gel (80 units subcutaneously twice a week) for 6 months
|
|---|---|---|---|
|
Significant Reduction in Proteinuria to Remission Levels During the Treatment Period (Includes Complete and Partial Remission, as Defined in the Outcome Measure Description Below)
|
2 participants
|
2 participants
|
3 participants
|
SECONDARY outcome
Timeframe: Up to 1 year after treatmentComplete remission defined as stable or improved renal function, serum creatinine \< or = 125% baseline, with proteinuria falling \<500 mg/day at last follow-up visit (6 to 12 months post treatment). Partial remission defined as stable or improved renal function, serum creatinine \< or = 125% baseline, with 50% reduction in proteinuria and final proteinuria 500-3500 mg/day at last follow-up visit (6 to 12 months post treatment).
Outcome measures
| Measure |
Membranous Nephropathy
n=3 Participants
Subjects diagnosed with membranous nephropathy, treated with ACTH gel (80 units subcutaneously twice a week) for 6 months
|
FSGS/MCD
n=2 Participants
Subjects diagnosed with focal segmental glomerulosclerosis (FSGS) or minimal change disease (MCD), treated with ACTH gel (80 units subcutaneously twice a week) for 6 months
|
IgA Nephropathy
n=3 Participants
Subjects diagnosed with IgA nephropathy (Berger's disease), treated with ACTH gel (80 units subcutaneously twice a week) for 6 months
|
|---|---|---|---|
|
Sustained Remissions up to 1 Year After Discontinuing Therapy
|
3 participants
|
2 participants
|
2 participants
|
Adverse Events
Membranous Nephropathy
Serious events: 2 serious events
Other events: 1 other events
Deaths: 0 deaths
FSGS/MCD
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
IgA Nephropathy
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Membranous Nephropathy
n=5 participants at risk
Subjects diagnosed with membranous nephropathy, treated with ACTH gel (80 units subcutaneously twice a week) for 6 months
|
FSGS/MCD
n=5 participants at risk
Subjects diagnosed with focal segmental glomerulosclerosis (FSGS) or minimal change disease (MCD), treated with ACTH gel (80 units subcutaneously twice a week) for 6 months
|
IgA Nephropathy
n=5 participants at risk
Subjects diagnosed with IgA nephropathy (Berger's disease), treated with ACTH gel (80 units subcutaneously twice a week) for 6 months
|
|---|---|---|---|
|
Metabolism and nutrition disorders
exacerbation of diabetes
|
40.0%
2/5 • Number of events 2
|
0.00%
0/5
|
0.00%
0/5
|
Other adverse events
| Measure |
Membranous Nephropathy
n=5 participants at risk
Subjects diagnosed with membranous nephropathy, treated with ACTH gel (80 units subcutaneously twice a week) for 6 months
|
FSGS/MCD
n=5 participants at risk
Subjects diagnosed with focal segmental glomerulosclerosis (FSGS) or minimal change disease (MCD), treated with ACTH gel (80 units subcutaneously twice a week) for 6 months
|
IgA Nephropathy
n=5 participants at risk
Subjects diagnosed with IgA nephropathy (Berger's disease), treated with ACTH gel (80 units subcutaneously twice a week) for 6 months
|
|---|---|---|---|
|
Skin and subcutaneous tissue disorders
Increased skin pigmentation
|
20.0%
1/5 • Number of events 1
|
20.0%
1/5 • Number of events 1
|
0.00%
0/5
|
|
General disorders
Cushingoid response
|
0.00%
0/5
|
0.00%
0/5
|
20.0%
1/5 • Number of events 1
|
Additional Information
Dr. Andrew S. Bomback, MD, MPH
Columbia University Medical Center
Phone: 212-305-0320
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place