Ignite Creation Date:
2024-05-05 @ 11:31 AM
Last Modification Date:
2024-10-26 @ 9:09 AM
Study NCT ID:
NCT00065611
Status:
COMPLETED
Last Update Posted:
2011-10-28
First Post:
2003-07-28
Brief Title:
Steroid Treatment for Kidney Disease
Sponsor:
National Institute of Diabetes and Digestive and Kidney Diseases NIDDK
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Pulse Dexamethasone Over 48 Weeks for Podocyte Disease
Status:
COMPLETED
Status Verified Date:
2011-09
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:
Focal segmental glomerulosclerosis FSGS and minimal change disease are kidney diseases that are associated with increased excretion of protein in the urine Approximately half of FSGS patients will lose kidney function within 8 years of diagnosis and will require dialysis The purpose of this study is to determine whether intermittent oral steroid therapy can cause sustained remission of FSGS and MCD
Approximately 70 participants including adults and children older than age 2 will be enrolled in this study They will receive 48 doses of oral dexamethasone over a period of 48 weeks One group will take two daily doses every 2 weeks the other group will take four daily doses every 4 weeks Doctors will monitor participants before during and after the steroid treatment with extensive exams and testing At the completion of the study researchers will evaluate the safety and efficacy of the drug treatment
Approximately 70 participants including adults and children older than age 2 will be enrolled in this study They will receive 48 doses of oral dexamethasone over a period of 48 weeks One group will take two daily doses every 2 weeks the other group will take four daily doses every 4 weeks Doctors will monitor participants before during and after the steroid treatment with extensive exams and testing At the completion of the study researchers will evaluate the safety and efficacy of the drug treatment
Detailed Description:
The major causes of primary nephritic syndrome in adults and children are idiopathic podocyte diseases minimal change MCD and focal segmental glomerulosclerosis FSGS Our objective is to determine whether intermittent oral dexamethasone administered over 48 weeks can induce complete remission in these patients This is an open-label multi-center pilot study designed to obtain preliminary evidence of efficacy and to establish safety This is part of a long-term effort to define the most effective mode of administering pulse dexamethasone and is expected to lead to a trial comparing daily prednisone to pulse dexamethasone
We will enroll up to 70 patients with nephritic-level proteinuria due to biopsy-proven MCD up to 30 patients or FSGS up to 40 patients We will include adults and children greater than 20 years of age Children with MCD must have received a minimum of 4 weeks and a maximum of 10 weeks of high-dose daily steroids since many children are responsive to short courses of daily steroids these requirements will define a steroid-resistant population For children with FSGS and adults with MCD or FSGS there is no minimum duration of prior steroids and there is a maximum of 8 weeks of prior high-dose daily steroids these requirements will define a population that has received a short steroid course without response If steroids have been used inclusion criteria require persistent nephrotic syndrome thus excluding steroid-sensitive nephrotic syndrome whether steroid-dependent or frequently relapsing
Patients may enroll at NIH or at collaborating centers Those patients who enroll at NIH will visit the NIH Clinical Center at least 4 times Patients enrolled at collaborating centers have the option to come to the NIH Clinical Center to complete research tests under these circumstances they will be enrolled as NIH research subjects
Patients will receive 48 doses of oral dexamethasone over a period of 48 weeks Patients will be randomized to one of two arms 2 daily doses every 2 weeks or 4 daily doses every 4 weeks The rationale is to test whether increased frequency dosing has greater efficacy with acceptable safety For adult patients we have a record of safety with pulse dexamethasone from the FSGS Dexamethasone study as well as from published studies for other diseases Therefore for adults each pulse will be 50 mgm2 during the first 12 weeks and each pulse will be 25 mgm2 during the next 36 weeks The trial for pediatric patients involves dose escalation as there is little experience with pulse dexamethasone for podocyte diseases in this age group In pediatric stage 1 each dexamethasone pulse will be 25 mgm2 over 48 weeks When 4 patients in each arm have completed 48 weeks of therapy safety and efficacy will be evaluated If the evaluation is positive we will embark on pediatric stage 2 in which dexamethasone pulses will be 50 mgm2 during the first 12 weeks and 25 mgm2 during the next 36 weeks the same as the adult regimen
The primary endpoint will be the presence of complete remission 48 weeks after beginning therapy Secondary endpoints will include complete and partial remission at 48 weeks and complete and partial remission at 104 weeks Assessment of remission will be by 24 hour urine collection in adults and children greater than 130 years and first void urine samples in children less than 130 years Patients will be evaluated for manifestations of steroid toxicity including growth rate children ophthalmologic complications adrenal suppression osteoporosis a vascular necrosis and psychological disturbances
We will enroll up to 70 patients with nephritic-level proteinuria due to biopsy-proven MCD up to 30 patients or FSGS up to 40 patients We will include adults and children greater than 20 years of age Children with MCD must have received a minimum of 4 weeks and a maximum of 10 weeks of high-dose daily steroids since many children are responsive to short courses of daily steroids these requirements will define a steroid-resistant population For children with FSGS and adults with MCD or FSGS there is no minimum duration of prior steroids and there is a maximum of 8 weeks of prior high-dose daily steroids these requirements will define a population that has received a short steroid course without response If steroids have been used inclusion criteria require persistent nephrotic syndrome thus excluding steroid-sensitive nephrotic syndrome whether steroid-dependent or frequently relapsing
Patients may enroll at NIH or at collaborating centers Those patients who enroll at NIH will visit the NIH Clinical Center at least 4 times Patients enrolled at collaborating centers have the option to come to the NIH Clinical Center to complete research tests under these circumstances they will be enrolled as NIH research subjects
Patients will receive 48 doses of oral dexamethasone over a period of 48 weeks Patients will be randomized to one of two arms 2 daily doses every 2 weeks or 4 daily doses every 4 weeks The rationale is to test whether increased frequency dosing has greater efficacy with acceptable safety For adult patients we have a record of safety with pulse dexamethasone from the FSGS Dexamethasone study as well as from published studies for other diseases Therefore for adults each pulse will be 50 mgm2 during the first 12 weeks and each pulse will be 25 mgm2 during the next 36 weeks The trial for pediatric patients involves dose escalation as there is little experience with pulse dexamethasone for podocyte diseases in this age group In pediatric stage 1 each dexamethasone pulse will be 25 mgm2 over 48 weeks When 4 patients in each arm have completed 48 weeks of therapy safety and efficacy will be evaluated If the evaluation is positive we will embark on pediatric stage 2 in which dexamethasone pulses will be 50 mgm2 during the first 12 weeks and 25 mgm2 during the next 36 weeks the same as the adult regimen
The primary endpoint will be the presence of complete remission 48 weeks after beginning therapy Secondary endpoints will include complete and partial remission at 48 weeks and complete and partial remission at 104 weeks Assessment of remission will be by 24 hour urine collection in adults and children greater than 130 years and first void urine samples in children less than 130 years Patients will be evaluated for manifestations of steroid toxicity including growth rate children ophthalmologic complications adrenal suppression osteoporosis a vascular necrosis and psychological disturbances
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
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 03-DK-0226 | None | None | None |