Idiopathic Focal Segmental Glomerulosclerosis
Conditions
Keywords
FSGS, primary FSGS, idiopathic FSGS
Brief summary
Focal segmental glomerulosclerosis (FSGS) is a condition that harms the kidney filters that remove waste from the blood. Proteins are supposed to stay in the blood. Damaged filters let protein get into the kidney. FSGS is a serious condition that can lead to kidney failure. The only treatment for kidney failure is dialysis or kidney transplant. Proteinuria means too much protein came through the kidneys into the urine. If the doctor cannot figure out what is causing the problem, it is primary (idiopathic) FSGS. This kind of FSGS is very hard to treat. This study will test Acthar in patients with this condition who have not responded to other treatments. It primarily investigates how well the therapy is tolerated by the patients and how well they respond to this treatment.
Interventions
DRUGActhar Gel
Acthar Gel 80 U/mL solution for subcutaneous injection
Sponsors
Mallinckrodt ARD LLC
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Potential participants must meet the following summary criteria for inclusion in the study: * Is male or non-pregnant, non-lactating female * Has primary FSGS, is nephrotic and did not achieve at least partial proteinuria response with prior therapy * Has been treated with an angiotensin converting enzyme inhibitor or receptor blocker (or have documented intolerance), for at least 4 weeks before screening * Has blood pressure no higher than 150/90 mmHg * Meets all other inclusion criteria detailed in the protocol
Exclusion criteria
Potential participants will not be eligible for the the study if they meet the following summary criteria: * Has hepatitis B or C, tuberculosis, or other contraindication listed on the United States (US) Prescribing Information for Acthar * Has Type 1 or Type 2 diabetes mellitus or any clinically significant infection * Has received specific treatments at exclusionary time points per protocol * Has been involved in a therapeutic drug/device trial (other than for FSGS) within 4 weeks before screening * Meets any other
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Adverse Events | within 56 weeks | Clinically significant changes in laboratory or physical examination findings are counted as adverse events. Descriptive statistics are collected for participants with: 1. death for any reason (all cause mortality) 2. treatment emergent serious adverse events (TESAEs) 3. any non-serious treatment emergent adverse events (TEAEs) |
Countries
Argentina, Australia, Chile, Mexico, Peru, Turkey (Türkiye), United States
Participant flow
Recruitment details
Multiple locations enrolled participants in Argentina (3), Australia (5), Chile (4), Mexico (5), Peru (8), Turkey (13), and the United States (25).
Participants by arm
| Arm | Count |
|---|---|
| Acthar Gel All participants receive open-label treatment with Acthar Gel per protocol regimen | 63 |
| Total | 63 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 8 |
| Overall Study | Increased proteinuria | 2 |
| Overall Study | Mutual decision patient and physician | 1 |
| Overall Study | Participant decided not to continue with open label extension | 1 |
| Overall Study | Physician Decision | 7 |
| Overall Study | Withdrawal by Subject | 8 |
Baseline characteristics
| Characteristic | Acthar Gel |
|---|---|
| Age, Continuous | 36.1 years STANDARD_DEVIATION 13.54 |
| Age, Customized 18-25 years of age | 16 Participants |
| Age, Customized 26-45 years of age | 29 Participants |
| Age, Customized 46-65 years of age | 17 Participants |
| Age, Customized greater than 65 years of age | 1 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 2 Participants |
| Race (NIH/OMB) Asian | 1 Participants |
| Race (NIH/OMB) Black or African American | 4 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 8 Participants |
| Race (NIH/OMB) White | 48 Participants |
| Region of Enrollment Argentina | 3 participants |
| Region of Enrollment Australia | 5 participants |
| Region of Enrollment Chile | 4 participants |
| Region of Enrollment Mexico | 5 participants |
| Region of Enrollment Peru | 8 participants |
| Region of Enrollment Turkey | 13 participants |
| Region of Enrollment United States | 25 participants |
| Sex: Female, Male Female | 29 Participants |
| Sex: Female, Male Male | 34 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 63 |
| other Total, other adverse events | 50 / 63 |
| serious Total, serious adverse events | 11 / 63 |
Outcome results
Primary
Number of Participants With Adverse Events
Clinically significant changes in laboratory or physical examination findings are counted as adverse events. Descriptive statistics are collected for participants with: 1. death for any reason (all cause mortality) 2. treatment emergent serious adverse events (TESAEs) 3. any non-serious treatment emergent adverse events (TEAEs)
Time frame: within 56 weeks
Population: Safety population
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Acthar Gel | Number of Participants With Adverse Events | Deaths | 0 Participants |
| Acthar Gel | Number of Participants With Adverse Events | Serious TEAEs | 11 Participants |
| Acthar Gel | Number of Participants With Adverse Events | Non-serious TEAEs | 60 Participants |