Trial Outcomes & Findings for Idiopathic Focal Segmental Glomerulosclerosis (FSGS) and Treatment With ACTH (NCT NCT01155141)

NCT ID: NCT01155141

Last Updated: 2014-09-01

Results Overview

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

15 participants

Primary outcome timeframe

Baseline - Month 6

Results posted on

2014-09-01

Participant Flow

Participant milestones

Participant milestones
Measure
H.P. Acthar Gel
Patients treated with 40 units subcutaneously (SC) weekly for 2 weeks, 80 units SC weekly for 2 weeks, then 80 units SC twice weekly to complete 16 weeks of therapy.
Overall Study
STARTED
16
Overall Study
COMPLETED
14
Overall Study
NOT COMPLETED
2

Reasons for withdrawal

Reasons for withdrawal
Measure
H.P. Acthar Gel
Patients treated with 40 units subcutaneously (SC) weekly for 2 weeks, 80 units SC weekly for 2 weeks, then 80 units SC twice weekly to complete 16 weeks of therapy.
Overall Study
Withdrawal by Subject
1
Overall Study
Screen Failure
1

Baseline Characteristics

Idiopathic Focal Segmental Glomerulosclerosis (FSGS) and Treatment With ACTH

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
H.P. Acthar Gel
n=15 Participants
Patients treated with 40 units subcutaneously (SC) weekly for 2 weeks, 80 units SC weekly for 2 weeks, then 80 units SC twice weekly to complete 16 weeks of therapy.
Age, Continuous
40 years
STANDARD_DEVIATION 15.1 • n=5 Participants
Sex: Female, Male
Female
6 Participants
n=5 Participants
Sex: Female, Male
Male
9 Participants
n=5 Participants
24 hour proteinuria
3.6 g/day
STANDARD_DEVIATION 5.7 • n=5 Participants
Serum Creatinine
1.4 mg/dL
STANDARD_DEVIATION 0.9 • n=5 Participants
Protein/creatinine ratio
3.1 unitless
STANDARD_DEVIATION 2.3 • n=5 Participants
Glucose
95 mg/dL
STANDARD_DEVIATION 8.4 • n=5 Participants
Weight
87.3 kg
STANDARD_DEVIATION 24.3 • n=5 Participants
Systolic blood pressure
128 mm Hg
STANDARD_DEVIATION 15.9 • n=5 Participants
Diastolic blood pressure
82 mm Hg
STANDARD_DEVIATION 9.8 • n=5 Participants
Total cholesterol
236 mg/dL
STANDARD_DEVIATION 99.5 • n=5 Participants
eGFR
56 mL/min/1.73m^2
STANDARD_DEVIATION 34.8 • n=5 Participants

PRIMARY outcome

Timeframe: Baseline - Month 6

Outcome measures

Outcome measures
Measure
H.P. Acthar Gel
n=15 Participants
Patients treated with 40 units subcutaneously (SC) weekly for 2 weeks, 80 units SC weekly for 2 weeks, then 80 units SC twice weekly to complete 16 weeks of therapy.
24 Hour Proteinuria
Baseline (N=15)
3.6 g/day
Standard Deviation 5.7
24 Hour Proteinuria
Week 5 (N=15)
3.8 g/day
Standard Deviation 8.6
24 Hour Proteinuria
Week 16 (N=14)
2.2 g/day
Standard Deviation 4.7
24 Hour Proteinuria
Month 6 (N=12)
3.0 g/day
Standard Deviation 3.2

PRIMARY outcome

Timeframe: Baseline - Month 6

Outcome measures

Outcome measures
Measure
H.P. Acthar Gel
n=15 Participants
Patients treated with 40 units subcutaneously (SC) weekly for 2 weeks, 80 units SC weekly for 2 weeks, then 80 units SC twice weekly to complete 16 weeks of therapy.
Serum Creatinine
Baseline (N=15)
1.4 mg/dL
Standard Deviation 0.9
Serum Creatinine
Week 5 (N=15)
1.8 mg/dL
Standard Deviation 0.8
Serum Creatinine
Week 9 (N=14)
1.8 mg/dL
Standard Deviation 1
Serum Creatinine
Week 16 (N=14)
1.9 mg/dL
Standard Deviation 0.9
Serum Creatinine
Month 6 (N=12)
2.0 mg/dL
Standard Deviation 1.1

PRIMARY outcome

Timeframe: Baseline - Month 6

Outcome measures

Outcome measures
Measure
H.P. Acthar Gel
n=15 Participants
Patients treated with 40 units subcutaneously (SC) weekly for 2 weeks, 80 units SC weekly for 2 weeks, then 80 units SC twice weekly to complete 16 weeks of therapy.
Protein/Creatinine Ratio
Month 6 (N=11)
1.6 unitless
Standard Deviation 1.3
Protein/Creatinine Ratio
Baseline (N=15)
3.1 unitless
Standard Deviation 2.3
Protein/Creatinine Ratio
Week 5 (N=15)
3.1 unitless
Standard Deviation 5.3
Protein/Creatinine Ratio
Week 16 (N=14)
2.4 unitless
Standard Deviation 1.8

SECONDARY outcome

Timeframe: Baseline - Month 6

Outcome measures

Outcome measures
Measure
H.P. Acthar Gel
n=15 Participants
Patients treated with 40 units subcutaneously (SC) weekly for 2 weeks, 80 units SC weekly for 2 weeks, then 80 units SC twice weekly to complete 16 weeks of therapy.
eGFR
Baseline (N=15)
56.0 mL/min/1.73m^2
Standard Deviation 34.8
eGFR
Week 5 (N=15)
44.0 mL/min/1.73m^2
Standard Deviation 37.1
eGFR
Week 9 (N=14)
37.5 mL/min/1.73m^2
Standard Deviation 32.0
eGFR
Week 16 (N=14)
43.0 mL/min/1.73m^2
Standard Deviation 28.0
eGFR
Month 6 (n=12)
38.0 mL/min/1.73m^2
Standard Deviation 33.5

SECONDARY outcome

Timeframe: Baseline - Month 6

Outcome measures

Outcome measures
Measure
H.P. Acthar Gel
n=15 Participants
Patients treated with 40 units subcutaneously (SC) weekly for 2 weeks, 80 units SC weekly for 2 weeks, then 80 units SC twice weekly to complete 16 weeks of therapy.
Weight
Baseline (N=15)
87.5 kg
Standard Deviation 24.3
Weight
Week 5 (N=15)
89.8 kg
Standard Deviation 24.7
Weight
Week 9 (N=13)
94.0 kg
Standard Deviation 25.2
Weight
Week 16 (N=13)
93.8 kg
Standard Deviation 26.1
Weight
Month 6 (N=11)
91.3 kg
Standard Deviation 28.2

SECONDARY outcome

Timeframe: Baseline - Month 6

Outcome measures

Outcome measures
Measure
H.P. Acthar Gel
n=15 Participants
Patients treated with 40 units subcutaneously (SC) weekly for 2 weeks, 80 units SC weekly for 2 weeks, then 80 units SC twice weekly to complete 16 weeks of therapy.
Systolic Blood Pressure
Baseline (N=15)
128.0 mm Hg
Standard Deviation 15.9
Systolic Blood Pressure
Week 5 (N=15)
136.0 mm Hg
Standard Deviation 15.2
Systolic Blood Pressure
Week 9 (N=13)
136.0 mm Hg
Standard Deviation 18.3
Systolic Blood Pressure
Week 16 (N=14)
134.0 mm Hg
Standard Deviation 18.6
Systolic Blood Pressure
Month 6 (N=11)
128.0 mm Hg
Standard Deviation 15.6

SECONDARY outcome

Timeframe: Baseline - Month 6

Outcome measures

Outcome measures
Measure
H.P. Acthar Gel
n=15 Participants
Patients treated with 40 units subcutaneously (SC) weekly for 2 weeks, 80 units SC weekly for 2 weeks, then 80 units SC twice weekly to complete 16 weeks of therapy.
Diastolic Blood Pressure
Baseline (N=15)
82.0 mm Hg
Standard Deviation 9.8
Diastolic Blood Pressure
Week 5 (N=15)
85.0 mm Hg
Standard Deviation 11.1
Diastolic Blood Pressure
Week 9 (N=13)
80.0 mm Hg
Standard Deviation 10.1
Diastolic Blood Pressure
Week 16 (N=14)
78.0 mm Hg
Standard Deviation 12.1
Diastolic Blood Pressure
Month 6 (N=11)
77.0 mm Hg
Standard Deviation 8.4

SECONDARY outcome

Timeframe: Baseline - Month 6

Outcome measures

Outcome measures
Measure
H.P. Acthar Gel
n=15 Participants
Patients treated with 40 units subcutaneously (SC) weekly for 2 weeks, 80 units SC weekly for 2 weeks, then 80 units SC twice weekly to complete 16 weeks of therapy.
Glucose
Baseline (N=15)
95 mg/dL
Standard Deviation 8.4
Glucose
Week 5 (N=15)
92.0 mg/dL
Standard Deviation 14.9
Glucose
Week 9 (N=14)
93.0 mg/dL
Standard Deviation 24.0
Glucose
Week 16 (N=14)
97.0 mg/dL
Standard Deviation 17.2
Glucose
Month 6 (N=13)
88.0 mg/dL
Standard Deviation 14.3

SECONDARY outcome

Timeframe: Baseline - Month 6

Outcome measures

Outcome measures
Measure
H.P. Acthar Gel
n=14 Participants
Patients treated with 40 units subcutaneously (SC) weekly for 2 weeks, 80 units SC weekly for 2 weeks, then 80 units SC twice weekly to complete 16 weeks of therapy.
Total Cholesterol
Baseline (N=15)
236.0 mg/dL
Standard Deviation 99.5
Total Cholesterol
Week 5 (N=14)
215.0 mg/dL
Standard Deviation 86.2
Total Cholesterol
Week 16 (N=13)
229.0 mg/dL
Standard Deviation 90.4
Total Cholesterol
Month 6 (N=13)
217.0 mg/dL
Standard Deviation 133.3

Adverse Events

H.P. Acthar Gel

Serious events: 2 serious events
Other events: 13 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
H.P. Acthar Gel
n=15 participants at risk
Patients were treated with 40 units subcutaneously (SC) weekly for 2 weeks, then dose increased to 80 units SC weekly for 2 weeks followed by 80 units SC twice weekly to complete 16 weeks of therapy.
Metabolism and nutrition disorders
Hyperglycemia
6.7%
1/15
Renal and urinary disorders
Acute Kidney Injury
6.7%
1/15

Other adverse events

Other adverse events
Measure
H.P. Acthar Gel
n=15 participants at risk
Patients were treated with 40 units subcutaneously (SC) weekly for 2 weeks, then dose increased to 80 units SC weekly for 2 weeks followed by 80 units SC twice weekly to complete 16 weeks of therapy.
Vascular disorders
Elevated Blood Pressure
13.3%
2/15
Psychiatric disorders
Altered Mood
6.7%
1/15
Metabolism and nutrition disorders
Increased Energy
26.7%
4/15
Ear and labyrinth disorders
Vertigo
6.7%
1/15
Skin and subcutaneous tissue disorders
Slow Wound Healing
6.7%
1/15
Injury, poisoning and procedural complications
Bruising at Injection Site
6.7%
1/15
Gastrointestinal disorders
Nausea
6.7%
1/15
Metabolism and nutrition disorders
Increased Appetite
6.7%
1/15
Infections and infestations
Cold
6.7%
1/15
General disorders
Fatigue
13.3%
2/15
Investigations
Weight Gain
13.3%
2/15
Musculoskeletal and connective tissue disorders
Muscle Cramps
6.7%
1/15
Nervous system disorders
Headache
6.7%
1/15
Renal and urinary disorders
Polyuria
6.7%
1/15
Skin and subcutaneous tissue disorders
Rash
6.7%
1/15
General disorders
Facial Swelling
6.7%
1/15
General disorders
Leg Swelling
13.3%
2/15
Skin and subcutaneous tissue disorders
Acne
20.0%
3/15
Endocrine disorders
Cushingoid
6.7%
1/15
Renal and urinary disorders
Nephrotic Syndrome
6.7%
1/15
Nervous system disorders
Felt Faint after Injection
6.7%
1/15
Metabolism and nutrition disorders
Hypokalemia
6.7%
1/15
Musculoskeletal and connective tissue disorders
Myalgia
6.7%
1/15
Eye disorders
Blurred Vision
6.7%
1/15
Nervous system disorders
Right Arm Numbness
6.7%
1/15

Additional Information

Dr. Richard Lafayette

Stanford University

Phone: 650-498-6063

Results disclosure agreements

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