GlomerulonephritisPhase 2NCT00004990
Once-A-Month Steroid Treatment for Patients With Focal Segmental Glomerulosclerosis
A Phase 2 study testing Dexamethasone in people with Glomerulonephritis and Nephrotic Syndrome, run by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) at 1 site in United States.
Trial summary
This study will test the safety and effectiveness of a monthly dosing regimen of dexamethasone-a strong steroid medication-to treat patients with focal segmental glomerulosclerosis (FSGS). Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Glomerulonephritis, Nephrotic Syndrome
- What is being tested?
- Dexamethasone
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2000to Dec 2004
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Adults and children.
- Biopsy proven FSGS, including idiopathic FSGS and collapsing FSGS, but excluding HIV-associated FSGS and secondary FSGS associated with morbid obesity, sickle cell anemia, reflux nephropathy, chronic tubular injury, congenital renal anomalies, and reduced nephron mass.
- Glomerular filtration rate will be estimated using 4 variable MDRD GFR equation, which incorporates age, race, gender, and serum creatinine.
- If disease has been present less than or equal to 1 year, estimated GFR must be greater than or equal to 40 ml/min.
- If disease has been present greater than or equal to 1 year, estimated GFR must be greater than or equal to 60 ml/min.
- Nephrotic range proteinuria, defined as urine protein greater than or equal to 3.5 g/1.73 m(2)/d.
Show all 10Show fewer
- Patients who received steroids for FSGS and who entered complete remission but have relapsed with nephrotic range proteinuria will be eligible.
- If hypertensive, adequate blood pressure control (target BP less than or equal to 130/80 mm Hg at greater than 75% of measurement in adults).
- Patients must have been taking an angiotensin converting enzyme inhibitor or angiotensin receptor antagonist for at least 6 weeks, unless intolerant of these medications.
- Women with child-bearing potential must maintain an effective birth control regimen (oral contraceptive, intrauterine device, barrier plus spermicide).
Who cannot join
- Inability to give informed consent or cooperate with study.
- Poorly controlled diabetes (as defined by hemoglobin A1C of greater than or equal to 8.5 on entry screening, or daily insulin requirement of greater than or equal to 100 units) or hypertension (defined as systolic BP consistently greater than 160 and/or diastolic BP consistently greater than 100 on three or more drugs).
- Evidence of chronic or occult infection. Specifically, must not have evidence of active hepatitis B, hepatitis C, HIV or untreated mycobacterial infection.
- Current or prior use of cytotoxic agents or cyclosporin for FSGS. If previously treated with these agents for a non-renal indication, therapy must have concluded at least 12 months prior to study.
- Existence of any other condition which would complicate the implementation or interpretation of the study.
- History of steroid-induced psychiatric disorder, known avascular necrosis of hip or symptomatic osteoporosis (e.g., known compression fractures), brittle diabetes mellitus, or glaucoma. Patients with other psychiatric disorders will be evaluated on a case by case basis.
- Patients who have been treated with steroids equivalent to 1 mg/kg/day for greater than or equal to 8 weeks without remission will be excluded.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Bethesda, Maryland, 20892
About this study
Official title: Pulse Dexamethasone in Focal Segmental Glomerulosclerosis
This study will test the safety and effectiveness of a monthly dosing regimen of dexamethasone-a strong steroid medication-to treat patients with focal segmental glomerulosclerosis (FSGS). Patients with this disease have kidney fibrosis (scarring) and proteinuria (excessive excretion of protein in the urine) that, in about half of the patients eventually requires kidney dialysis or transplant. Currently, the most effective treatment for FSGS is high-dose steroids (prednisone) taken daily for 4 to 6 months. However, only about 30 percent of patients respond to this treatment, and it causes serious side effects in many patients. Other drugs, such as cyclosporin and cyclophosphamide, improve proteinuria in even fewer patients (about 10 percent) and also have serious side effects. This study will explore whether a monthly pulse dose of steroids will achieve disease remission with less toxicity.
Adults and children with FSGS who: 1) have not received steroid treatment, or 2) could not tolerate daily steroid treatment, or 3) relapsed after conventional steroid treatment may be eligible for this study. Those enrolled will take dexamethasone by mouth for 4 days every 4 weeks for a total of 8 months. Patients will undergo various tests before treatment starts (baseline), during the course of treatment, and in follow-up visits to evaluate the effects of treatment as follows:
Read more
- Review of kidney biopsy, medical evaluation, measurement of total daily urine protein excretion and kidney function, psychiatric testing for depression or other mood disorder
- Measurements of blood pressure, blood chemistries and urine protein excretion - monthly during treatment
- Questionnaire about the effects of treatment, if any, on mood and feelings - monthly during treatment
- Photographs of the face and body (in underwear or shorts and tank top) to evaluate body fat distribution- baseline and 8 months
- Eye examinations for cataracts and glaucoma - baseline and 8 months
- Bone density scan (DEXA scan) of the lower spine and hip - baseline, 4 and 12 months
- Magnetic resonance imaging (MRI) of the hips
- Psychological evaluation and quality of life evaluation - baseline, 1, 2 and 8 months
- Blood tests for adrenal gland function - baseline, 4 and 8 months
- Blood and urine tests - 10, 12, 15, and 18 months
Patients who achieve remission (whose urine protein levels decrease to normal) before completing the 8 months of dexamethasone will take one more dose and then stop therapy, but continue with follow-up. Patients who achieve remission but relapse may be offered a second course of treatment.
Read the detailed description
The objective of this study is to evaluate the effectiveness and toxicity of an alternative steroid dosing regimen for patients with focal segmental glomerulosclerosis (FSGS), using a pilot study design that will enroll 20 patients. Although the literature reports variable steroid responsiveness, remission rates of up to 30-40 percent have recently been reported in nephrotic adult patients treated with daily prednisone at 1 mg/kg/day for at least 4 months, followed by a taper over 3-4 months. Such a prolonged and aggressive steroid course is fraught with significant morbidity, but this approach has been advocated by some authors because of the poor prognosis for renal survival in nephrotic patients with FSGS who do not achieve remission with steroid treatment. We plan to test an eight month course of high dose steroid therapy administered in monthly pulses instead of daily doses, for comparable efficacy in achieving remission, and for the occurrence of adverse steroid side effects. We plan to enroll patients with nephrotic syndrome due to biopsy-proven FSGS, who have either not been treated, or have responded to conventional steroid dosing regimens and relapsed. We plan to treat them with monthly oral pulses of dexamethasone (40-60 mg/d x 4 days), for 8 months. The primary endpoint will be induction of complete remission, defined as urine protein less than 300 mg/d. Patients will also be evaluated for manifestations of steroid toxicity. Patients will be seen in follow up at intervals up to 24 months following study entry. If this study suggests that remission of nephrotic syndrome can be attained with this regimen, and with an acceptable toxicity profile, we will plan a randomized controlled trial of this regimen compared with daily or alternate day oral steroids.
- Conditions
- Glomerulonephritis, Nephrotic Syndrome
- Being tested
- Dexamethasone
- Keywords
- Nephrotic Syndrome, Steroids, Osteoporosis, Adrenal Suppression, Proteinuria, Focal Segmental Glomerulosclerosis
- Medical subject headings
- Glomerulonephritis, Nephrotic Syndrome, Osteoporosis, Proteinuria, Glomerulosclerosis, Focal Segmental
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DexamethasoneDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Mar 2000 (estimated)
- Study startMar 2000
- Study completionDec 2004
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for GlomerulonephritisStudies of the same condition, open and under way.
- More trials run by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Enrolment
- 20 participants
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00004990
- Sponsor's protocol ID
- 000100
- Other IDs
- 00-DK-0100
- Status last verified
- Dec 2004
- First submitted
- 18 Mar 2000
- Last updated
- 4 Mar 2008
- Results posted
- No
Publications
Adams DM, Kinney TR, O'Branski-Rupp E, Ware RE. High-dose oral dexamethasone therapy for chronic childhood idiopathic thrombocytopenic purpura. J Pediatr. 1996 Feb;128(2):281-3. doi: 10.1016/s0022-3476(96)70410-9.
PubMed 8636831Alexanian R, Dimopoulos MA, Delasalle K, Barlogie B. Primary dexamethasone treatment of multiple myeloma. Blood. 1992 Aug 15;80(4):887-90.
PubMed 1498331Andersen JC. Response of resistant idiopathic thrombocytopenic purpura to pulsed high-dose dexamethasone therapy. N Engl J Med. 1994 Jun 2;330(22):1560-4. doi: 10.1056/NEJM199406023302203.
PubMed 8177245
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2008. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pulse Dexamethasone in Focal Segmental Glomerulosclerosis
- ClinicalTrials.gov ID
- NCT00004990
- Acronym / sponsor ID
- 000100
- Conditions
- Glomerulonephritis, Nephrotic Syndrome
- Keywords
- Nephrotic Syndrome, Steroids, Osteoporosis, Adrenal Suppression, Proteinuria, Focal Segmental Glomerulosclerosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants
- Lead sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)US National Institutes of Health
- Study start
- Mar 2000
- Study completion
- Dec 2004
- First posted
- 20 Mar 2000 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will test the safety and effectiveness of a monthly dosing regimen of dexamethasone-a strong steroid medication-to treat patients with focal segmental glomerulosclerosis (FSGS). Patients with this disease have kidney fibrosis (scarring) and proteinuria (excessive excretion of protein in the urine) that, in about half of the patients eventually requires kidney dialysis or transplant. Currently, the most effective treatment for FSGS is high-dose steroids (prednisone) taken daily for 4 to 6 months. However, only about 30 percent of patients respond to this treatment, and it causes serious side effects in many patients. Other drugs, such as cyclosporin and cyclophosphamide, improve proteinuria in even fewer patients (about 10 percent) and also have serious side effects. This study will explore whether a monthly pulse dose of steroids will achieve disease remission with less toxicity.
Adults and children with FSGS who: 1) have not received steroid treatment, or 2) could not tolerate daily steroid treatment, or 3) relapsed after conventional steroid treatment may be eligible for this study. Those enrolled will take dexamethasone by mouth for 4 days every 4 weeks for a total of 8 months. Patients will undergo various tests before treatment starts (baseline), during the course of treatment, and in follow-up visits to evaluate the effects of treatment as follows:
- Review of kidney biopsy, medical evaluation, measurement of total daily urine protein excretion and kidney function, psychiatric testing for depression or other mood disorder
- Measurements of blood pressure, blood chemistries and urine protein excretion - monthly during treatment
- Questionnaire about the effects of treatment, if any, on mood and feelings - monthly during treatment
- Photographs of the face and body (in underwear or shorts and tank top) to evaluate body fat distribution- baseline and 8 months
- Eye examinations for cataracts and glaucoma - baseline and 8 months
- Bone density scan (DEXA scan) of the lower spine and hip - baseline, 4 and 12 months
- Magnetic resonance imaging (MRI) of the hips
- Psychological evaluation and quality of life evaluation - baseline, 1, 2 and 8 months
- Blood tests for adrenal gland function - baseline, 4 and 8 months
- Blood and urine tests - 10, 12, 15, and 18 months
Patients who achieve remission (whose urine protein levels decrease to normal) before completing the 8 months of dexamethasone will take one more dose and then stop therapy, but continue with follow-up. Patients who achieve remission but relapse may be offered a second course of treatment.
Detailed description
The objective of this study is to evaluate the effectiveness and toxicity of an alternative steroid dosing regimen for patients with focal segmental glomerulosclerosis (FSGS), using a pilot study design that will enroll 20 patients. Although the literature reports variable steroid responsiveness, remission rates of up to 30-40 percent have recently been reported in nephrotic adult patients treated with daily prednisone at 1 mg/kg/day for at least 4 months, followed by a taper over 3-4 months. Such a prolonged and aggressive steroid course is fraught with significant morbidity, but this approach has been advocated by some authors because of the poor prognosis for renal survival in nephrotic patients with FSGS who do not achieve remission with steroid treatment. We plan to test an eight month course of high dose steroid therapy administered in monthly pulses instead of daily doses, for comparable efficacy in achieving remission, and for the occurrence of adverse steroid side effects. We plan to enroll patients with nephrotic syndrome due to biopsy-proven FSGS, who have either not been treated, or have responded to conventional steroid dosing regimens and relapsed. We plan to treat them with monthly oral pulses of dexamethasone (40-60 mg/d x 4 days), for 8 months. The primary endpoint will be induction of complete remission, defined as urine protein less than 300 mg/d. Patients will also be evaluated for manifestations of steroid toxicity. Patients will be seen in follow up at intervals up to 24 months following study entry. If this study suggests that remission of nephrotic syndrome can be attained with this regimen, and with an acceptable toxicity profile, we will plan a randomized controlled trial of this regimen compared with daily or alternate day oral steroids.
Study design
- Primary purpose
- Treatment
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- Adults and children.
- Biopsy proven FSGS, including idiopathic FSGS and collapsing FSGS, but excluding HIV-associated FSGS and secondary FSGS associated with morbid obesity, sickle cell anemia, reflux nephropathy, chronic tubular injury, congenital renal anomalies, and reduced nephron mass.
- Glomerular filtration rate will be estimated using 4 variable MDRD GFR equation, which incorporates age, race, gender, and serum creatinine.
- If disease has been present less than or equal to 1 year, estimated GFR must be greater than or equal to 40 ml/min.
- If disease has been present greater than or equal to 1 year, estimated GFR must be greater than or equal to 60 ml/min.
Key exclusion criteria (7 listed)
- Inability to give informed consent or cooperate with study.
- Poorly controlled diabetes (as defined by hemoglobin A1C of greater than or equal to 8.5 on entry screening, or daily insulin requirement of greater than or equal to 100 units) or hypertension (defined as systolic BP consistently greater than 160 and/or diastolic BP consistently greater than 100 on three or more drugs).
- Evidence of chronic or occult infection. Specifically, must not have evidence of active hepatitis B, hepatitis C, HIV or untreated mycobacterial infection.
- Current or prior use of cytotoxic agents or cyclosporin for FSGS. If previously treated with these agents for a non-renal indication, therapy must have concluded at least 12 months prior to study.
- Existence of any other condition which would complicate the implementation or interpretation of the study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Dexamethasone | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Mar 2008.
- Background: PubMed 8636831Adams DM, Kinney TR, O'Branski-Rupp E, Ware RE. High-dose oral dexamethasone therapy for chronic childhood idiopathic thrombocytopenic purpura. J Pediatr. 1996 Feb;128(2):281-3. doi: 10.1016/s0022-3476(96)70410-9.
- Background: PubMed 1498331Alexanian R, Dimopoulos MA, Delasalle K, Barlogie B. Primary dexamethasone treatment of multiple myeloma. Blood. 1992 Aug 15;80(4):887-90.
- Background: PubMed 8177245Andersen JC. Response of resistant idiopathic thrombocytopenic purpura to pulsed high-dose dexamethasone therapy. N Engl J Med. 1994 Jun 2;330(22):1560-4. doi: 10.1056/NEJM199406023302203.
Related trials for Glomerulonephritis
RecruitingPhase 3
Study to Assess the Efficacy, Pharmacokinetics, Safety and Tolerability of Atrasentan in Pediatric Patients With Primary IgAN
Berger Disease, Bergers Disease
RecruitingPhase 4
Dapagliflozin in Active Lupus Nephritis
Lupus Nephritis (LN)
RecruitingPhase 3
A Study to Find Out if BI 764198 Helps Adults and Adolescents With a Kidney Condition Called Focal Segmental Glomerulosclerosis (FSGS)
Focal Segmental Glomerulosclerosis
Not yet recruitingPhase 3
A Study to Assess the Efficacy and Safety of Pegcetacoplan for the Prevention of Recurrent C3G or Primary IC-MPGN in Kidney Transplant Recipients
C3 Glomerulopathy (C3G), Primary Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN), Recurrent Glomerular Disease After Kidney Transplantation
Not yet recruiting
MYTP01 for Immune-Mediated Diseases
IgA Nephropathy, Kidney Transplant Recipients
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Dexamethasone in people with Glomerulonephritis and Nephrotic Syndrome, run by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Mar 2008. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00004990, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Mar 2008.