GDCN Clinical Trials and Research Studies

Current Clinical Trials and Research Studies

Click on the links below to learn more.

 

Please contact the GDCN study coordinators for information on current clinical trials and research studies.


Central Coordinating Office:
Glomerular Disease Collaborative Network (GDCN)
7024 Burnett-Womack Bldg
Chapel Hill, NC 27599-7155
E-Mail: gdcnunc@med.unc.edu

Read about UNC Medical Research and Volunteering in English OR Spanish.

A Multi-center, Longitudinal, Observational Study of Patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD) to Establish the Rate, Characteristics, and Determinants of Disease Progression

Principal Investigator: Patrick Nachman, MD
Sponsor : Otsuka Pharmaceutical Development & Commercialization, Inc.
Status: Enrolling

Description: This research study will investigate a correlation between the rate of kidney enlargement and the decline of kidney function in Autosomal Dominant Polycystic Kidney Disease (ADPKD) patients to see if the change in the volume of the kidneys could be used as a prognostic marker of disease progression.  This is an observational research study: no drug will be tested.

Inclusion criteria
- Men and women 12 to 70 years, inclusive
- Existing diagnosis of ADPKD
- Total kidney volume ≥ 300 cc/m height by ultrasound (within 1 year prior to baseline) or ≥ 250 cc/m height by MRI (within 1 year prior to baseline).

Exclusion Criteria
- Any medical condition that could interfere – in the opinion of the study doctor- with evaluation of the research study objectives (for example: inability to have an MRI)
- Current or expected (within the next six months) interventions for the treatment of ADPKD affecting kidney volume (for example, cysts reduction) without the prior approval of the sponsor

For more information, please contact:
Anne Froment                                                                        
(919) 966-2561 ext 247                                                         
anne_froment@med.unc.edu                                                

Sandy Grubbs
(919) 966-2561 ext 245
Sandra_grubbs@med.unc.edu

 


A Multicenter, Randomized, Prospective, Open-Label Trial of Rituximab in the Treatment of Progressive IgA Nephropathy

Sponsored by: Genentech, Inc.

Principal Investigator: Patrick Nachman, MD

The purpose of this study is to compare the ability of Rituximab to standard therapy (tight blood pressure control and fish oil) in lowering protein in the urine in patients with IgA nephropathy, and to examine the side effects occurring with Rituximab.  Approximately 50 patients (5 for UNC Kidney Center) will be randomized to Rituximab in addition to standard therapy vs. standard therapy alone in an open-label multicenter controlled trial.  Patients randomized to the Rituximab group will receive a total of 4 doses on days #1; 15; 168 and 182.  Standard therapy will consist of combination ACE inhibitors and ARBs to achieve a BP goal of 125/75 (MAP 90 mm Hg) and Omega-3 Fatty Acid Fish Oil Supplements, 3.6 gm EPA/day.  Patients will be followed for a total of 12 months.

Inclusion Criteria:

Note: All eligible patients will have undergone a renal biopsy compatible with a diagnosis of IgA nephropathy within 12 months of study entry. The diagnostic criteria for IgA nephropathy are as follows:

Exclusion Criteria:

For more information, please contact:
Anne Froment
(919) 966-2561 ext 247
anne_froment@med.unc.edu

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A Phase 2, Multicenter, Randomized, Placebo-controlled, Double-blind, Placebo-masked, Parallel-group Pilot Trial to Compare the Efficacy, Tolerability, and Safety of Tolvaptan Modified-release and Immediate-release Formulations in Subjects with Autosomal Dominant Polycystic Kidney Disease

 

Principal Investigator: Patrick Nachman, MD
Sponsor:  Otsuka Pharmaceutical Development & Commercialization, Inc.
Status: Enrolling

Otsuka Pharmaceutical Development & Commercialization, Inc. (OPDC)) is studying an investigational drug called tolvaptan (“Study Drug”).  An investigational drug is a drug that is being studied for approval by the United States Food and Drug Administration (FDA). 

Tolvaptan (Samsca®) is a drug approved for use in the United States (2009) in patients with certain types of hyponatremia. Hyponatremia is low amount of sodium or salt in the blood. Tolvaptan (Samsca®) is approved in the European Union (2009) for treatment for a specific type of hyponatremia (hyponatremia due to “syndrome of inappropriate antidiuretic hormone secretion” (SIADH)  The Study Drug has not been approved for sale in the United States to treat Polycystic Kidney Disease (PKD). PKD is a disease that causes kidney cysts (fluid-filled balloons), worsening kidney function, blood in the urine, kidney pain, high blood pressure, kidney stones, kidney infections, and cysts in the liver or other parts of the body and sometimes heart or blood vessel abnormalities. Tolvaptan is being studied as a possible treatment for PKD.  For those with PKD, the kidneys respond abnormally to the hormone vasopressin that may be involved in cyst development or growth in humans.  Tolvaptan interferes with vasopressin’s effects on the kidney, and when taken regularly, appears to block cyst growth in animal models of PKD.  It is hoped that similar effects will be seen in humans.  Tests will tell how useful tolvaptan will be in treating PKD.

There are two different formulations that exist for tolvaptan. One formulation is a tablet that releases the drug substance immediately. The second formulation is a capsule that is designed to release drug substance more slowly over time. This study will use both the immediate release tablet and the modified release capsule, to compare the efficacy, safety and tolerability of both formulations with a placebo (a pill or a capsule that does not contain any medication).

A total of approximately 180 people at approximately 20 institutions will take part in this study, including approximately 36 people from this institution.

The expected duration for trial participation, including Screening, Treatment, and Follow-up periods is 13 weeks. Treatment duration is expected to be 8 weeks.  The study involves three Magnetic Resonance Imaging (MRI) scan, without contrast; two 24hours urine collection; two 24 hours blood pressure monitoring, and the use of an electronic diary to keep track of voids and study drug ingestion.

Main inclusion criteria: Having ADPKD, with enlarged kidneys and being between 18 and 50 years old.

For more information, please contact:

Anne Froment
(919) 966-2561 ext 247
anne_froment@med.unc.edu

 

A Phase 3, Multi-center, Double-blind, Placebo-controlled, Parallel-arm Trial to Determine Long-term Safety and Efficacy of Oral Tolvaptan Tablet Regimens in Adult Subjects with Autosomal Dominant Polycystic Kidney Disease

Principal Investigator: Patrick Nachman, MD
Sponsor : Otsuka Pharmaceutical Development & Commercialization (OPDC)
Status: Ongoing – enrollment closed.

Description: Autosomal Dominant Polycystic Kidney Disease (ADPKD) is a disease that causes kidney cysts (fluid-filled balloons), worsening kidney function, blood in the urine, kidney pain, high blood pressure, kidney stones, kidney infections, and cysts in the brain or other parts of the body. Tolvaptan is being studied to see if it can help people with ADPKD. For those people with ADPKD, the kidneys respond abnormally to the hormone vasopressin, which may be involved in cyst development or growth in humans. Tolvaptan interferes with vasopressin’s effects on the kidney and, when taken continually, appears to block cyst growth in laboratory animals with ADPKD.

 

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A Randomized, Placebo-Controlled, Parallel-Group, Double-Blind Study of H.P. Acthar® Gel (Acthar) in Treatment-Resistant Subjects with Persistent Proteinuria and Nephrotic Syndrome due to Idiopathic Membranous Nephropathy (iMN)

Principal Investigator: Vimal Derebail, MD
Sponsor: Questcor Pharmaceuticals, Inc.
Status: Soon enrolling

Description: The purpose of this research study is to look at the safety and effectiveness of the study drug Acthar as compared to Placebo (inactive product) in patients who have been diagnosed with idiopathic nephrotic membranous nephropathy.  Acthar is a long-approved drug used to treat patients with proteinuria, multiple sclerosis and infantile spasms.  The drug is given by injection under the skin.

Participants:  Patients with idiopathic membranous nephropathy with a low chance of remission and who have previously been treated with standard treatment without success (treatment-resistant). .

Length of the study: Up to 14 months

Inclusion criteria:

a. If you stopped taking this therapy because of a change in your health or bad side effect
before you could tell if you were getting better or not, you may still eligible
b. If you got better or almost got better taking this medication(s) and then had a relapse of
your disease, you will not be eligible

Exclusion Criteria:

For more information, please contact:

Anne Froment
(919) 966-2561 ext 247
anne_froment@med.unc.edu                                    

 

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Cognitive impairment in patients with End Stage Kidney Disease

Principal Investigator: Patrick Nachman, MD
Sponsor : TRACs Institute
Status: Enrolling

Description: To test if hemolialysis has a long-term impact on cognitive function, we are recruiting volunteers for a prospective longitudinal analysis of the changes in cognitive function in a large cohort of patients on chronic hemodialysis over 12 months.  The primary outcome will be the rate of change in cognitive function based on a battery of validated cognitive tests.  Repeated measures of hemodynamic and metabolic variables will be recorded. Risk factors for progressive cognitive impairment will be identified by multivariate analysis.

Inclusion criteria:
ESKD on chronic hemodialysis for at least 3 months
Age 50- 80 years .old.

Exclusion criteria:
Established history of severe dementia.
History of cerebro-vascular accident with persistent neurologic deficits (patients with a history of transient ischemic attack will not be excluded)
Patients who are severely visually impaired.
Patients with known HIV/AIDS (as this may impact the risk for and progression of cognitive impairment/dementia).   No testing for HIV will be performed as part of the study.

For more information, please contact:
Anne Froment
(919) 966-2561 ext 247
anne_froment@med.unc.edu

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Cyclophosphamide-Related Urothelial Tumor Evaluation

 

Principal Investigator: Patrick Nachman, MD
Sponsor : none
Status: Enrolling

Description: The purpose of this research study is to create a registry of patients who have received treatment for vasculitis or glomerulonephritis with the chemotherapy drugs cyclophosphamide or chlorambucil.  These drugs are known to increase the risk of bladder cancer.  For this reason, screening for bladder cancer is recommended although specific guidelines as to the frequency of such screening are not well established.  Although screening for bladder cancer is offered to patients previously treated with cyclophosphamide or chlorambucil, the optimal screening method and frequency are not established.   

The specific aims of the registry are to:
1- identify patients who have been treated with cyclophosphamide or chlorambucil and are at risk for bladder cancer.
2- offer these patients a systematic screening regimen with scheduled cystoscopies.
3- collect demographic, clinical, and epidemiologic data on factors susceptible to influence a patient’s risk for bladder cancer.
4- assess the utility of a cytology test on voided urine in detecting a bladder cancer.

For more information, please contact:
Anne Froment
(919) 966-2561 ext 247
anne_froment@med.unc.edu

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Multi-center, Open-label, Extension Study to Evaluate the Long-term Efficacy and Safety of Oral Tolvaptan Tablet Regimens in Subjects with Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Principal Investigator: Patrick Nachman, MD
Sponsor : Otsuka Pharmaceutical Development & Commercialization, Inc. (OPDC)
Status: Ongoing – enrollment open only to participants in the previous double blind research study of tolvaptan.

Description: Autosomal Dominant Polycystic Kidney Disease (ADPKD) is a disease that causes kidney cysts (fluid-filled balloons), worsening kidney function, blood in the urine, kidney pain, high blood pressure, kidney stones, kidney infections, and cysts in the brain or other parts of the body. Tolvaptan is being studied to see if it can help people with ADPKD. For those people with ADPKD, the kidneys respond abnormally to the hormone vasopressin, which may be involved in cyst development or growth in humans. Tolvaptan interferes with vasopressin’s effects on the kidney and, when taken continually, appears to block cyst growth in laboratory animals with PKD

This open label extension research trial studies the potential long-term benefits and safety of tolvaptan.

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NEPTUNE (The Nephrotic Syndrome Study Network)

Principal Investigator: Patrick Nachman, MD
Sponsors : National Institutes of Health, NIDDK, Nephcure Foundation, University of Michigan
Status: ongoing – enrollment open
Description: The five-year NEPhroTic Syndrome StUdy NEtwork  (NEPTUNE) is studying three kidney diseases: Focal Segmental Glomerulosclerosis (FSGS), Minimal Change Disease (MCD) and Membranous Nephropathy (MN)
NEPTUNE enrolls volunteers –adults and children with protein in their urine- at the time of their diagnosis.
Volunteers for that study give kidney tissue, nail clippings, blood and urine samples, and give information about their medications, their health, their quality of life at regular interval (4 times the first year, 2 times a year after that).
All those samples and information will be used for research on FSGS, MCD and MN, looking for risk factors, markers of the disease, better treatments, etc.
UNC is one of 15 participating sites in the United States and Canada which are conducting the research over the five years of the project.

Inclusion criteria:
New diagnosis of FSGS, MCD, MN
Consent form signed before biopsy, in order for the study to receive an extra core for research.

Exclusion criteria:
Prior solid organ transplant or bone marrow transplant
A clinical diagnosis of FSGS/MCD/MN  without diagnostic renal biopsy
Clinical or histological evidence of other renal diseases (Alport, Nail Patella, Diabetic Nephropathy, IgA or Lupus nephritis, genito-urinary malformations with vesico-uretheral reflux or renal dysplasia in pediatric patients).
Renal function (measured with 4-variable MDRD) estimated GFR<30m/1.73m2/24hr

For more information, please contact:
Anne Froment or Sandy Grubbs
(919) 966-2561 ext 247 or 245
anne_froment@med.unc.edu 
sandra_grubbs@med.unc.edu

 

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Open Label Research Study of Eculizumab - Targeting Complement Activation in ANCA-Vasculitis


Sponsors: National Institutes of Health (NIH) and Alexion Pharmaceuticals, Inc.

Principal investigator: Patrick Nachman, MD

Status: Enrolling

Overview and rationale.  This is a proof-of-concept pilot research study of eculizumab as adjunctive therapy in patients with active ANCA disease. Eculizumab is a monoclonal antibody to C5, which is currently FDA approved for the treatment of paroxysmal nocturnal hemoglobinuria.  Eculizumab will be used over a period of 12 weeks in addition to the standard induction treatment. Patients may be enrolled no later than 10 days after the start of induction therapy. The research study requires 15 visits over a period of 52 weeks.

Total number of patients: 10 (6 patients randomized to Eculizumab arm, 4 patients randomized to control arm)

Inclusion criteria: 

Exclusion criteria:


For more information, please contact:
Anne Froment
(919) 966-2561 ext 247
anne_froment@med.unc.edu

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Plasma exchange and glucocorticoid dosing in the treatment of ANCA-associated vasculitis: a multicentre randomized controlled trial (PEXIVAS)

Principal Investigator: Patrick Nachman, MD

Sponsors: Food and Drug Administration

Status: Enrolling

Description:  Multi-centre, international, open label, factorial design, randomized control research study in severe Anti-Neutrophils Cytoplasmic Antibodies (ANCA)-associated vasculitis (AAV) to determine the efficacy of plasma exchange (PLEX) in addition to immunosuppressive therapy and glucocorticoids (GC) in reducing death and end-stage renal disease (ESRD) and  to determine the non-inferiority of a reduced dose glucocorticoids (GC) regimen in reducing death and ESRD

This research study will randomize patients with AAV in a two-by-two factorial design. Randomization to each intervention will be in a one-to-one ratio stratified by the other intervention. Patients will be randomized to receive either adjunctive PLEX or no PLEX and randomized to receive either a standard glucocorticoid (GC) dose or a low GC dose. All patients will receive standard immunosuppressive induction therapy with cyclophosphamide or rituximab. The primary outcome of the research study will be the composite endpoint of all-cause mortality or end-stage renal disease.

The participation of the subject to the research study will last between 2 and 7 years. The exact duration for the subject will depend on how long the research study has been running before he/she is recruited. We anticipate recruiting for 5 years.

Subjects will need to visit the clinic 7 times within the first year and then every 6 months until the end of the research study (minimum 9-maximum19 visits in total). Each visit will last approximately half an hour.

Patients will be followed more frequently when they begin the research study when the interventions are most intense and treatment is designed to induce remission of disease (Induction of Remission Period) and follow-up will be less intense after this period (Maintenance of Remission Period).

Inclusion Criteria

Patients must meet all of the following criteria:

1. New or previous clinical diagnosis of Wegener’s granulomatosis, or microscopic polyangiitis consistent with the Chapel-Hill consensus definitions AND
2. Positive test for proteinase 3-ANCA or myeloperoxidase-ANCA  AND
3. Severe vasculitis defined by at least one of the following manifestations:
a. Renal involvement characterized by both of the following:
i. Evidence of glomerulonephritis by either of the following:
1. Renal biopsy demonstrating focal necrotizing glomerulonephritis or
2. Active urine sediment characterized by glomerular hematuria/cellular casts and proteinuria
AND
ii. An estimated glomerular filtration (eGFR) rate of <50 ml/min/1.73 m2. Patients known to have a stable eGFR <50 ml/min/1.73 m2 for greater than three months prior to enrollment are NOT eligible.

b. Pulmonary hemorrhage due to active vasculitis defined by the following:
i. A compatible chest x-ray or CT scan (diffuse pulmonary infiltrates)
AND
ii. The absence of an alternative explanation for all pulmonary infiltrates (i.e. volume overload or pulmonary infection)
AND
iii. At least one of the following:
1. Evidence of alveolar hemorrhage on bronchoscopic examination or increasingly bloody returns with bronchoalveolar lavage
2. Observed hemoptysis
3. Unexplained anemia (<10 g/dL) or documented drop in hemoglobin (>1 g/dL) from less than 10g/dl
4. an increased diffusing capacity of carbon dioxide

Exclusion Criteria
Patients must have none of the following:

  1. A diagnosis of vasculitis other than Wegener’s granulomatosis or microscopic polyangiitis
  2. A positive serum test for anti-glomerular basement membrane or a renal biopsy demonstrating linear glomerular immunoglobulin deposition
  3. Receipt of dialysis for greater than 21 days prior to randomization
  4. Age <15 years. In centers that do not routinely treat patients <18 years or if no local investigator routinely treats patients <18 years, enrollment may be restricted to patients 18 years or older
  5. Pregnancy
  6. Inability or unwillingness to comply with birth control/abstinence
  7. Inability to provide informed consent
  8. Treatment with >1 IV dose of cyclophosphamide and/or >14 days of oral cyclophosphamide and/or >14 days of prednisone/prednisolone (>30 mg/day) and/or treatment with >1 dose of rituximab within the last 28 days
  9. A comorbidity that, in the opinion of the investigator, precludes the use of cyclophosphamide, glucocorticoids, or plasma exchange or absolutely mandates the use of plasma exchange

For more information, please contact:
Anne Froment or Sandy Grubbs
(919) 966-2561 ext 247 or 245
anne_froment@med.unc.edu 
sandra_grubbs@med.unc.edu

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Rituximab in the Treatment of Idiopathic Membranous Nephropathy

Principal Investigator: Patrick H. Nachman, MD
Sponsor: Mayo Clinic and Genentech
Status : Not recruiting – Ongoing - follow-up of treatment only

Description: This study aims to determine if the medication Rituximab is effective in treating membranous nephropathy.  Rituximab, also called Rituxanâ, is a monoclonal antibody produced by Genentech, Inc. and Biogen IDEC Pharmaceuticals, using recombinant DNA technology.  Rituximab is not approved to treat idiopathic membranous nephropathy.

For more information, please contact:
Anne Froment
(919) 966-2561 ext 247
anne_froment@med.unc.edu

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