Focal Segmental GlomerulosclerosisPhase 1/2NCT00007475

Permeability Factor in Focal Segmental Glomerulosclerosis

A Phase 1/2 study testing Plasma exchange and Cyclophosphamide in people with Focal Segmental Glomerulosclerosis, run by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

Who is it for?: Any ageHealthy volunteers can take part
Conditions the study is for: Focal Segmental Glomerulosclerosis
What is being tested?: Plasma exchange and Cyclophosphamide
Type of treatment: Pill or oral medicine, Surgery or procedure
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 15 participants
Where: 1 siteUnited States
Study dates: Dec 2000to Jun 2014
Phase: Phase 1/2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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
Healthy volunteers accepted
Child, Adult, Older adult

Who may be able to join

Patients with idiopathic focal segmental glomerulosclerosis on renal biopsy, including the following categories:
Untreated FSGS
Steroid-dependent FSGS
Steroid resistant FSGS
Recurrent FSGS, with functioning allograft
FSGS in ESRD, receiving hemodialysis
Adults greater than or equal to18 will be eligible for all studies.
Children greater than 20 kilograms, will be eligible for all branches of the study except for treatment of steroid resistant FSGS with pirfenidone, as pirfenidone has not previously been administered to pediatric patients in any setting. Children less than 20 kilograms will be excluded from the study for the following reason: plasma exchange in patients less than 20 kilograms requires a red blood cell transfusion, which significantly increases the risk of the procedure by exposing the patient to the risk of transfusion associated infections, and the safety of an aggressive course of plasma exchange has not been established in this population.

Who cannot join

Secondary FSGS: HIV-associated FSGS or hyperfiltration FSGS, including FSGS associated with congenital renal abnormalities, renal mass reduction, reflux nephropathy, interstitial nephritis, and sickle cell anemia are excluded.
Patients with disease associated with immunosuppression, other than chronic renal failure.
The presence of malignancy or the history of other serious, complicating illness such as myocardial infarction or cerebrovascular accident in the past six months, at the discretion of the investigators.
For plasma exchange: A Department of Transfusion Medicine consultant will evaluate all potential plasma exchange patients. Those with prolonged PT, PTT, platelet count less than 100,000 or receiving anticoagulant therapy will undergo plasma exchange only if the consultant considers this to be safe.
For prednisone: uncontrolled diabetes mellitus (requiring greater than 100 units of insulin/day with the concurrence of the Endocrinology consultant), active infection including hepatitis B or C (if that is the advice of the Hepatology consultant), infection with HIV (as these patients are at increased risk of avascular necrosis), other active infection (if that is the advice of the Infectious Disease consultant), history of avascular necrosis or bone densitometry indicating bone mass less than 2SD below normal, active ulcer disease, history of steroid-induced psychosis, morbid obesity, positive PPD or history of past positive PPD without adequate treatment are excluded.
For Cyclophosphamide:

Show all 11Show fewer

Allergy or hypersensitivity to cyclophosphamide
Leukocyte less than 3000 cells/microliter or ANC less than 1500 cells/microliter or evidence of bone marrow compromise
Prior irradiation to the heart or therapy with doxorubicin or other cardiotoxic medication (may increase the risk for cardiotoxicity)
Peritoneal dialysis, as there is no published evidence that cyclophosphamide metabolites can be safely removed.
Certain drugs will be used with caution or avoided. Barbiturates and phenytoin induce the hepatic enzymes that metabolize cyclophosphamide and therefore if these medications are required, cyclophosphamide doses may need to be increased to achieve a comparable immunosuppressive effect. Drugs that inhibit cyclophosphamide metabolism include allopurinol, imipramine, and phenothiazines, chloramphenicol and chlorpromazine; these drugs will be avoided. NSAID increase the risk of hyponatremia; these drugs will be avoided.

Trial locations and contacts

1 site in 1 country.

United States1 site

National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892

Other trials in United States

About this study

Focal segmental glomerulosclerosis (FSGS) is a renal syndrome characterized by proteinuria (usually nephrotic range), limited response to conventional therapy, and a poor renal prognosis, with progression to end stage renal failure in at least 50% of patients. As a syndrome, FSGS likely has many specific etiologies, only a few of which are well-defined. Recently, it has been suggested that some idiopathic FSGS patients have elevated circulating levels of a protein that induces glomerular permeability in vitro and in vivo. While there has been no consistent term for this factor, it will be termed here FSGS permeability factor (FPF).

The purposes of the present study are five fold:

To identify a population of FSGS patients with elevated FPF levels
To examine RNA expression profiles of peripheral blood mononuclear cells (PBMC) in FSGS patients with elevated FPF levels
To define the kinetics of FPF disappearance and reappearance in FSGS patients receiving immunomodulatory therapy and in the case of patients with recurrent FSGS following renal transplant, those receiving plasma exchange
To identify immunosuppressive agents which are successful in inducing sustained reduction in FPF levels
To determine in patients with FSGS who are awaiting renal transplant, whether sustained reduction in FPF levels is associated with reduced risk of recurrent FSGS.

Patient participation is divided into an evaluation phase, in which FPF levels, RNA expression profiles, and patient eligibility for participation in treatment protocols are determined, and a treatment phase in which specific immunomodulatory therapy is introduced in an open label fashion. We propose to define carefully the relationship between elevated FPF and remission of proteinuria in patients with FSGS in native kidneys, following treatment with standard therapies (daily prednisone, cyclophosphamide) and experimental therapies (pulse dexamethasone, pirfenidone). In patients with recurrent FSGS in renal allografts, we will determine the kinetics of FPF following plasma exchange and following plasma exchange plus cyclophosphamide. In patients with elevated FPF levels who are awaiting renal transplantation, we will determine the kinetics of FPF following plasma exchange and following plasma exchange plus cyclophosphamide, and examine the rate of recurrent FSGS in these patients.

Read the detailed description

The purposes of the present study are five fold:

To identify a population of FSGS patients with elevated FPF levels
To examine RNA expression profiles of peripheral blood mononuclear cells (PBMC) in FSGS patients with elevated FPF levels
To define the kinetics of FPF disappearance and reappearance in FSGS patients receiving immunomodulatory therapy and in the case of patients with recurrent FSGS following renal transplant, those receiving plasma exchange
To identify immunosuppressive agents which are successful in inducing sustained reduction in FPF levels
To determine in patients with FSGS who are awaiting renal transplant, whether sustained reduction in FPF levels is associated with reduced risk of recurrent FSGS.

patients.

Conditions: Focal Segmental Glomerulosclerosis
Being tested: Plasma exchange, Cyclophosphamide
Keywords: Plasma Exchange, Cyclophosphamide, Proteinuria, Hemodialysis, Expression Profiling
Medical subject headings: Glomerulosclerosis, Focal Segmental, Proteinuria

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Plasma exchangeProcedure or surgery
Also called Plasmapheresis
A course of plasma exchange of 5 treatments over 10 days, then administration of cyclophosphamide.
CyclophosphamideDrug
Also called Cytophosphane
For GFR > 50 ml/min/1.73 m2 received oral cyclophosphamide at a dose of 2 mg/kg/ day for 3 months. For GFR < 50 ml/min/1.73 m2 but > 10 ml/min/1.73 m2 will receive oral cyclophosphamide at a 25% reduced dose or 1.5 mg/kg/d for 3 months.

Study groups in NCT00007475
Study group	Role	What they receive
Plasma Exchange + Cyclophosphamide	Experimental	Procedure/Surgery: Plasma exchange A course of plasma exchange of 5 treatments over 10 days, then administration of cyclophosphamide. Drug: Cyclophosphamide For GFR > 50 ml/min/1.73 m2 received oral cyclophosphamide at a dose of 2 mg/kg/ day for 3 months. For GFR < 50 ml/min/1.73 m2 but > 10 ml/min/1.73 m2 will receive oral cyclophosphamide at a 25% reduced dose or 1.5 mg/kg/d for 3 months. Receives: Procedure: Plasma exchange; Drug: Cyclophosphamide

What is being measured

The primary outcome is the main question the study is designed to answer.

Reduction in Proteinuria in Recurrent FSGS Following Renal Transplant With Plasma Exchange and Cyclophosphamide.
Outcomes for FSGS occurring in native kidneys:
1. Complete remission: proteinuria <0.3 g/d ; B. Partial remission: proteinuria between 0.3 and 2 g/d ; C. Incomplete response: proteinuria between 2 and 3.5 g/d ; D. Relapse: return to proteinuria ≥3.5 g/d ; Note that counts within each category A-D may be summarized relative to remaining categories, as a proportion (relative to complement of the whole group count) with calculations implicitly based on zero/one valued binary variables, whose means are proportions, so to report 95% confidence intervals calculated using an exact binomial distribution.
Measured: every 3 months up to a year followed with native kidneys

Secondary outcomes (4)

Comparison of RNA Expression Profiles in PBMC From Patients With FPF, Without FPF and Control Subjects
No RNA expression profiles have been obtained as FSGS Permeability Factor (FPF) levels NOT available -- its assay has not yet been developed to an extent that it could be applied to all enrollees of this current trial.
Note that provisional values (targeting current candidate molecule: cardiotrophin-like cytokine 1) were assayed for 3 of the first 4 enrollees using assay by Dr. Virginia Savin, whose lab is actively investigating a molecular identification of FPF using an isolation approach based on sequential precipitation results in a 100-fold purification, but the fraction remains a complex mixture on SDS-PAGE (Sharma 1999). Other investigators of FPF include:
1. Terry Phillips at NIH developed an assay that looked promising but after his retirement it has not been possible for other researchers to get this working.
2. Avi Rosenberg, NCI has developed a promising ELISA-style assay, as well as some work in a mass spectrometry assay, and this is being further refined.
Measured: End of study
Define the Kinetics of FPF in FSGS Patients Receiving Immunomodulatory Therapy or Plasma Exchange.
Provisional assay of a molecular identification of FPF (current candidate: cardiotrophin-like cytokine 1) using an isolation approach based on sequential precipitation results in a 100-fold purification.
Note: Focal segmental glomerulosclerosis Permeability Factor (FPF) assay had not yet been developed to an extent that it could be applied to all enrollees in the current trial; provisional values were assayed for 3 of the first 4 enrollees using a version implemented by Dr. Virginia Savin, whose lab is actively investigating the above isolation approach, but the fraction remains a complex mixture on SDS-PAGE (Sharma 1999). Other investigators of FPF, mentioned here for interested readers of this trial report, include:
1. Terry Phillips at NIH developed an assay that looked promising prior to his retirement.
2. Avi Rosenberg, NCI has developed promising ELISA-style assay, and mass spectrometry assay, being refined.
Measured: End of study
Correlate the Effect of Immunosuppressive Agents Which Reduce Proteinuria in Recurrent FSGS With the Effect on FPF Levels
Provisional assay of a molecular identification of FPF (current candidate: cardiotrophin-like cytokine 1) using an isolation approach based on sequential precipitation results in a 100-fold purification.
Note: Focal segmental glomerulosclerosis Permeability Factor (FPF) assay had not yet been developed to an extent that it could be applied to all enrollees in the current trial; provisional values were assayed for 3 of the first 4 enrollees using a version implemented by Dr. Virginia Savin, whose lab is actively investigating the above isolation approach, but the fraction remains a complex mixture on SDS-PAGE (Sharma 1999). Other investigators of FPF, mentioned here for interested readers of this trial report, include:
1. Terry Phillips at NIH developed an assay that looked promising prior to his retirement.
2. Avi Rosenberg, NCI has developed promising ELISA-style assay, and mass spectrometry assay, being refined.
Measured: End of study
Determine Whether Renal Transplantation in Patients Whose Elevated FPF Levels Have Been Reduced for a Sustained Period is Associated With a Reduced Prevalence of Recurrent FSGS.
Provisional assay of a molecular identification of FPF (current candidate: cardiotrophin-like cytokine 1) using an isolation approach based on sequential precipitation results in a 100-fold purification.
Note: Focal segmental glomerulosclerosis Permeability Factor (FPF) assay had not yet been developed to an extent that it could be applied to all enrollees in the current trial; provisional values were assayed for 3 of the first 4 enrollees using a version implemented by Dr. Virginia Savin, whose lab is actively investigating the above isolation approach, but the fraction remains a complex mixture on SDS-PAGE (Sharma 1999). Other investigators of FPF, mentioned here for interested readers of this trial report, include:
1. Terry Phillips at NIH developed an assay that looked promising prior to his retirement.
2. Avi Rosenberg, NCI has developed promising ELISA-style assay, and mass spectrometry assay, being refined.
Measured: End of study

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Dec 2000Jun 2014

First posted25 Dec 2000 (estimated)
Study startDec 2000
Primary completionJun 2014
Study completionJun 2014

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (3)Papers the sponsor has linked to this study.
Other trials for Focal Segmental GlomerulosclerosisStudies 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 1/2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 15 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00007475
Sponsor's protocol ID: 010053
Other IDs: 01-DK-0053
Status last verified: Feb 2016
First submitted: 22 Dec 2000
Last updated: 10 Feb 2016
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Korbet SM. Primary focal segmental glomerulosclerosis. J Am Soc Nephrol. 1998 Jul;9(7):1333-40. doi: 10.1681/ASN.V971333. No abstract available.
PubMed 9644647
Cameron JS. Recurrent renal disease after renal transplantation. Curr Opin Nephrol Hypertens. 1994 Nov;3(6):602-7. doi: 10.1097/00041552-199411000-00007.
PubMed 7881983
Valeri A, Barisoni L, Appel GB, Seigle R, D'Agati V. Idiopathic collapsing focal segmental glomerulosclerosis: a clinicopathologic study. Kidney Int. 1996 Nov;50(5):1734-46. doi: 10.1038/ki.1996.493.
PubMed 8914044

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 2
An Open-Label Phase 2 Study of N-Acetyl-D-Mannosamine (ManNAc) in Subjects With Podocyte Diseases
Focal Segmental Glomerulosclerosis, Minimal Change Disease
Not yet recruitingObservational study
MENA/Levant Epidemiological, Registry of Immunological & Clinical Traits (MERIT)
Antiphospholipid (aPL)-Positive, Rheumatic Arthritis
RecruitingPhase 2
A Study to Evaluate the Efficacy and Safety of Frexalimab, Brivekimig, or Rilzabrutinib in Participants Aged 16 to 75 Years With Primary Focal Segmental Glomerulosclerosis or Minimal Change Disease
Focal Segmental Glomerulosclerosis, Glomerulonephritis Minimal Lesion
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 2
A Study to Evaluate the Effectiveness and Safety of Sibeprenlimab in Participants With Primary Membranous Nephropathy, Immune-mediated Focal Segmental Glomerulosclerosis, and Minimal Change Disease
Primary Membranous Nephropathy, Minimal Change Disease

All Focal Segmental Glomerulosclerosis trials

Study at a glance

From the registry record, last updated by the sponsor on 10 Feb 2016. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Permeability Factor in Focal Segmental Glomerulosclerosis
ClinicalTrials.gov ID: NCT00007475
Acronym / sponsor ID: 010053
Conditions: Focal Segmental Glomerulosclerosis
Keywords: Plasma Exchange, Cyclophosphamide, Proteinuria, Hemodialysis, Expression Profiling
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Completed
Enrolment: 15 participants (actual)
Lead sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)US National Institutes of Health
Study start: Dec 2000
Primary completion: Jun 2014 (actual)
Study completion: Jun 2014 (actual)
First posted: 25 Dec 2000 (estimated)
Results: Posted 10 Feb 2016

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

The purposes of the present study are five fold:

To identify a population of FSGS patients with elevated FPF levels
To examine RNA expression profiles of peripheral blood mononuclear cells (PBMC) in FSGS patients with elevated FPF levels
To define the kinetics of FPF disappearance and reappearance in FSGS patients receiving immunomodulatory therapy and in the case of patients with recurrent FSGS following renal transplant, those receiving plasma exchange
To identify immunosuppressive agents which are successful in inducing sustained reduction in FPF levels
To determine in patients with FSGS who are awaiting renal transplant, whether sustained reduction in FPF levels is associated with reduced risk of recurrent FSGS.

Detailed description

The purposes of the present study are five fold:

To identify a population of FSGS patients with elevated FPF levels
To examine RNA expression profiles of peripheral blood mononuclear cells (PBMC) in FSGS patients with elevated FPF levels
To define the kinetics of FPF disappearance and reappearance in FSGS patients receiving immunomodulatory therapy and in the case of patients with recurrent FSGS following renal transplant, those receiving plasma exchange
To identify immunosuppressive agents which are successful in inducing sustained reduction in FPF levels
To determine in patients with FSGS who are awaiting renal transplant, whether sustained reduction in FPF levels is associated with reduced risk of recurrent FSGS.

patients.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Plasma Exchange + Cyclophosphamide	Experimental	Procedure: Plasma exchange, Drug: CyclophosphamideProcedure/Surgery: Plasma exchange A course of plasma exchange of 5 treatments over 10 days, then administration of cyclophosphamide. Drug: Cyclophosphamide For GFR > 50 ml/min/1.73 m2 received oral cyclophosphamide at a dose of 2 mg/kg/ day for 3 months. For GFR < 50 ml/min/1.73 m2 but > 10 ml/min/1.73 m2 will receive oral cyclophosphamide at a 25% reduced dose or 1.5 mg/kg/d for 3 months.

Who may qualify

Sex: All
Healthy volunteers: Accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (8 listed)

Key exclusion criteria (11 listed)

Secondary FSGS: HIV-associated FSGS or hyperfiltration FSGS, including FSGS associated with congenital renal abnormalities, renal mass reduction, reflux nephropathy, interstitial nephritis, and sickle cell anemia are excluded.
Patients with disease associated with immunosuppression, other than chronic renal failure.
The presence of malignancy or the history of other serious, complicating illness such as myocardial infarction or cerebrovascular accident in the past six months, at the discretion of the investigators.
For plasma exchange: A Department of Transfusion Medicine consultant will evaluate all potential plasma exchange patients. Those with prolonged PT, PTT, platelet count less than 100,000 or receiving anticoagulant therapy will undergo plasma exchange only if the consultant considers this to be safe.
For prednisone: uncontrolled diabetes mellitus (requiring greater than 100 units of insulin/day with the concurrence of the Endocrinology consultant), active infection including hepatitis B or C (if that is the advice of the Hepatology consultant), infection with HIV (as these patients are at increased risk of avascular necrosis), other active infection (if that is the advice of the Infectious Disease consultant), history of avascular necrosis or bone densitometry indicating bone mass less than 2SD below normal, active ulcer disease, history of steroid-induced psychosis, morbid obesity, positive PPD or history of past positive PPD without adequate treatment are excluded.

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
Plasma exchangeAlso: PlasmapheresisA course of plasma exchange of 5 treatments over 10 days, then administration of cyclophosphamide.	Procedure or surgery	Plasma Exchange + Cyclophosphamide
CyclophosphamideAlso: CytophosphaneFor GFR > 50 ml/min/1.73 m2 received oral cyclophosphamide at a dose of 2 mg/kg/ day for 3 months. For GFR < 50 ml/min/1.73 m2 but > 10 ml/min/1.73 m2 will receive oral cyclophosphamide at a 25% reduced dose or 1.5 mg/kg/d for 3 months.	Drug	Plasma Exchange + Cyclophosphamide

Primary outcome

Reduction in Proteinuria in Recurrent FSGS Following Renal Transplant With Plasma Exchange and Cyclophosphamide.
Time frame: every 3 months up to a year followed with native kidneys
Outcomes for FSGS occurring in native kidneys: A. Complete remission: proteinuria <0.3 g/d ; B. Partial remission: proteinuria between 0.3 and 2 g/d ; C. Incomplete response: proteinuria between 2 and 3.5 g/d ; D. Relapse: return to proteinuria ≥3.5 g/d ; Note that counts within each category A-D may be summarized relative to remaining categories, as a proportion (relative to complement of the whole group count) with calculations implicitly based on zero/one valued binary variables, whose means are proportions, so to report 95% confidence intervals calculated using an exact binomial distribution.

4 secondary outcomes

Comparison of RNA Expression Profiles in PBMC From Patients With FPF, Without FPF and Control Subjects
Time frame: End of study
Define the Kinetics of FPF in FSGS Patients Receiving Immunomodulatory Therapy or Plasma Exchange.
Time frame: End of study
Correlate the Effect of Immunosuppressive Agents Which Reduce Proteinuria in Recurrent FSGS With the Effect on FPF Levels
Time frame: End of study
Determine Whether Renal Transplantation in Patients Whose Elevated FPF Levels Have Been Reduced for a Sustained Period is Associated With a Reduced Prevalence of Recurrent FSGS.
Time frame: End of study

Results and safety

Results posted to the registry on 10 Feb 2016, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
FPF NOT Assayed, Plasma Exchange + Cyclophosphamide Completed	12	8
FPF Assayed Pre-Tx as Low, PE + Cyclophosphamide Completed	1	1
FPF Assayed Pre-Tx as High, PE + Cyclophosphamide Completed	2	2
Historical Controls	0	0

Primary outcome: Reduction in Proteinuria in Recurrent FSGS Following Renal Transplant With Plasma Exchange and Cyclophosphamide.

Time frame: every 3 months up to a year followed with native kidneys. Mean (95% Confidence Interval). Unit: proportion of participants with outcome

Measure	FPF NOT Assayed, Plasma Exchange + Cyclophosphamide Completed n = 12	FPF Assayed Pre-Tx as Low, PE + Cyclophosphamide Completed n = 1	FPF Assayed Pre-Tx as High, PE + Cyclophosphamide Completed n = 2
A. Complete remission: proteinuria <0.3 g/d	0 (0 to 0.00049)	0 (0 to 0.975)	0.5 (0.01258 to 0.9874)
B. Partial remission: proteinuria in [0.3,2] g/d	0.25 (0.05486 to 0.5719)	0 (0 to 0.975)	0 (0 to 0.8419)
C. Incomplete response: proteinuria in (2,3.5) g/d	0 (0 to 0)	0 (0 to 0)	0 (0 to 0)
D. Relapse: return to proteinuria ≥3.5 g/d	0.1667 (0.02086 to 0.4841)	1 (0.025 to 1)	0 (0 to 0.8419)

FPF NOT Assayed, Plasma Exchange + Cyclophosphamide Completed vs FPF Assayed Pre-Tx as Low, PE + Cyclophosphamide Completed vs FPF Assayed Pre-Tx as High, PE + Cyclophosphamide Completed: Proportion with reduced proteinuria: 0.6363 (95% CI 0.3079 to 0.8907), p <0.0001, Exact binomial test

Population analysed: all participants, regardless of amount of follow-up

4 secondary outcomes are reported in the full results.

Adverse events

Time frame: Entire course of study follow-up, from study enrollment to closure. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Plasma Exchange + Cyclophosphamide, Complete Treatment	3 of 12 (25%)	0 of 12 (0%)	Not reported
FPF Assayed Pre-Tx as Low, PE + Cyclophosphamide Completed	0 of 1 (0%)	0 of 1 (0%)	Not reported
FPF Assayed Pre-Tx as High, PE + Cyclophosphamide Completed	0 of 2 (0%)	0 of 2 (0%)	Not reported

Limitations and caveats (from the sponsor): Note: definitive FSGS Permeability Factor (FPF) assays still in development. Data/samples transferred to the NIH-CC Glomerulosclerosis Consolidation protocol: focus on cardiotrophin like cytokine 1 \& antibodies to a panel of podocyte proteins as FPF.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 10 Feb 2016.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 9644647Korbet SM. Primary focal segmental glomerulosclerosis. J Am Soc Nephrol. 1998 Jul;9(7):1333-40. doi: 10.1681/ASN.V971333. No abstract available.
Background: PubMed 7881983Cameron JS. Recurrent renal disease after renal transplantation. Curr Opin Nephrol Hypertens. 1994 Nov;3(6):602-7. doi: 10.1097/00041552-199411000-00007.
Background: PubMed 8914044Valeri A, Barisoni L, Appel GB, Seigle R, D'Agati V. Idiopathic collapsing focal segmental glomerulosclerosis: a clinicopathologic study. Kidney Int. 1996 Nov;50(5):1734-46. doi: 10.1038/ki.1996.493.

Related trials for Focal Segmental Glomerulosclerosis

RecruitingPhase 2
An Open-Label Phase 2 Study of N-Acetyl-D-Mannosamine (ManNAc) in Subjects With Podocyte Diseases
Focal Segmental Glomerulosclerosis, Minimal Change Disease
Not yet recruitingObservational study
MENA/Levant Epidemiological, Registry of Immunological & Clinical Traits (MERIT)
Antiphospholipid (aPL)-Positive, Rheumatic Arthritis
RecruitingPhase 2
A Study to Evaluate the Efficacy and Safety of Frexalimab, Brivekimig, or Rilzabrutinib in Participants Aged 16 to 75 Years With Primary Focal Segmental Glomerulosclerosis or Minimal Change Disease
Focal Segmental Glomerulosclerosis, Glomerulonephritis Minimal Lesion
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 2
A Study to Evaluate the Effectiveness and Safety of Sibeprenlimab in Participants With Primary Membranous Nephropathy, Immune-mediated Focal Segmental Glomerulosclerosis, and Minimal Change Disease
Primary Membranous Nephropathy, Minimal Change Disease

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.

Jeffrey B Kopp, M.D. principal investigator

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

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 8 inclusion and 11 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 10 Feb 2016. 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 combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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?

Yes. Results are posted in the registry (first posted 10 Feb 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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.

What does the primary outcome measure?

Reduction in Proteinuria in Recurrent FSGS Following Renal Transplant With Plasma Exchange and Cyclophosphamide. (time frame: every 3 months up to a year followed with native kidneys). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00007475, 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 10 Feb 2016.