Ignite Creation Date:
2024-05-05 @ 10:38 PM
Last Modification Date:
2024-10-26 @ 10:22 AM
Study NCT ID:
NCT01155141
Status:
COMPLETED
Last Update Posted:
2014-09-01
First Post:
2010-06-29
Brief Title:
Idiopathic Focal Segmental Glomerulosclerosis FSGS and Treatment With ACTH
Sponsor:
Stanford University
Organization:
Stanford University
Study Overview
Official Title:
Idiopathic Focal Segmental Glomerulosclerosis FSGS and Treatment With ACTH
Status:
COMPLETED
Status Verified Date:
2014-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
FSGS is an immunologic disorder wherein circulating immune proteins cause damage to the kidneys and progressive injury and scarring Corticosteroid therapy is occasionally but not nearly universally successful in reducing proteinuria and when patients respond they have a favorable prognosis The investigators believe that ACTH therapy HP Acthar Gel can provide a more rapid well tolerated reduction in glomerular injury
Detailed Description:
EXPERIMENTAL TREATMENT Patients with biopsy proven FSGS will be treated with ACTH in an open-label pilot study of tolerability and efficacy The investigators will recruit 18-20 patients to complete this study over 2 years
ACTH therapy Patients will receive HP Acthar gel IM or SQ initially at 40 IU SQ every week for 16 weeks of therapy All patients will be treated to goal BP of 14090mmHg with all patients treated with ACEi or ARB therapy as tolerated H2 receptor blockade or proton pump inhibitor therapy will also be offered all patients Dose will be titrated up to 160 IU SQ every week if at 1 month the patient has had no substantial reduction in proteinuria no deterioration of blood pressure and no development of hyperglycemia as well as no serious adverse events felt to be related to the medication
CLINICAL OUTCOME Patients will be followed for the primary outcome of remission of proteinuria This will be defined as partial remission when the proteinuria is reduced below 50 of the initial pre treatment value and as complete remission when the proteinuria is reduced to 05 gg or 500 mgday on a 24 hour urine sample Secondary outcomes will include effects on eGFR effects on glucose levels effects on blood pressure total doses of antihypertensive medications and absolute changes in blood pressure and on immune status Outcomes will be determined by looking at 3 month and 6 month values of urine protein and eGFR following initiation of treatment
IMMUNOLOGIC TESTING In order to further assess the role of immunologic perturations on FSGS and the effect of ACTH on the immune system all patients will bank blood and urine before the start of the study for cytokine analysis RNA DNA protein and protoarray testing
MONITORING AND SAFETY All patients will undergo full informed consent per the Stanford Institutional Review Board Contact numbers will be provided and study staff will be available at all times in case of any medical emergencies All patients will continue with routine health monitoring with a minimal of monthly assessments A comprehensive interview will be undertaken to assess for side effects complete physical exams will be accomplished including vital signs CBC clinical chemistries including electrolytes creatinine glucose and liver function tests urine for protein and creatinine and fasting lipid profiles every 3 months Also at screening and baseline
PATIENT WITHDRAWALTERMINATION OF STUDY Patients will be closely monitored as detailed above Patients may voluntarily leave the study at any time although every effort will be made to follow their clinical course and monitor for safety issues and possible benefits of therapy Patient will be monitored for adverse events Patients with severe adverse events will be evaluated for the relatedness of their event to the study medication If the event is considered severe and possibly or probably related to the study medication the medication will be discontinued and the patient will continue to be monitored In the case the adverse event is possibly related the medication may be restarted if the investigator and subject agree For patients with probably related severe adverse events study treatment will be discontinued however the investigators will still follow the patient to see if the course of their underlying disease was modified by treatment As this is an open label pilot study no data safety monitoring board is felt to be necessary If drug related SAEs develop in more than 20 of patients the study will be submitted back to the IRB to determine if it should continue
ACTH therapy Patients will receive HP Acthar gel IM or SQ initially at 40 IU SQ every week for 16 weeks of therapy All patients will be treated to goal BP of 14090mmHg with all patients treated with ACEi or ARB therapy as tolerated H2 receptor blockade or proton pump inhibitor therapy will also be offered all patients Dose will be titrated up to 160 IU SQ every week if at 1 month the patient has had no substantial reduction in proteinuria no deterioration of blood pressure and no development of hyperglycemia as well as no serious adverse events felt to be related to the medication
CLINICAL OUTCOME Patients will be followed for the primary outcome of remission of proteinuria This will be defined as partial remission when the proteinuria is reduced below 50 of the initial pre treatment value and as complete remission when the proteinuria is reduced to 05 gg or 500 mgday on a 24 hour urine sample Secondary outcomes will include effects on eGFR effects on glucose levels effects on blood pressure total doses of antihypertensive medications and absolute changes in blood pressure and on immune status Outcomes will be determined by looking at 3 month and 6 month values of urine protein and eGFR following initiation of treatment
IMMUNOLOGIC TESTING In order to further assess the role of immunologic perturations on FSGS and the effect of ACTH on the immune system all patients will bank blood and urine before the start of the study for cytokine analysis RNA DNA protein and protoarray testing
MONITORING AND SAFETY All patients will undergo full informed consent per the Stanford Institutional Review Board Contact numbers will be provided and study staff will be available at all times in case of any medical emergencies All patients will continue with routine health monitoring with a minimal of monthly assessments A comprehensive interview will be undertaken to assess for side effects complete physical exams will be accomplished including vital signs CBC clinical chemistries including electrolytes creatinine glucose and liver function tests urine for protein and creatinine and fasting lipid profiles every 3 months Also at screening and baseline
PATIENT WITHDRAWALTERMINATION OF STUDY Patients will be closely monitored as detailed above Patients may voluntarily leave the study at any time although every effort will be made to follow their clinical course and monitor for safety issues and possible benefits of therapy Patient will be monitored for adverse events Patients with severe adverse events will be evaluated for the relatedness of their event to the study medication If the event is considered severe and possibly or probably related to the study medication the medication will be discontinued and the patient will continue to be monitored In the case the adverse event is possibly related the medication may be restarted if the investigator and subject agree For patients with probably related severe adverse events study treatment will be discontinued however the investigators will still follow the patient to see if the course of their underlying disease was modified by treatment As this is an open label pilot study no data safety monitoring board is felt to be necessary If drug related SAEs develop in more than 20 of patients the study will be submitted back to the IRB to determine if it should continue
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None