Clinical Trial: Novel Therapies for Resistant FSGS (FONTII): Phase II Clinical Trial

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Novel Therapies for Resistant Focal Segmental Glomerulosclerosis

Brief Summary: This project will test whether adalimumab,and/or galactose can safely reduce proteinuria (abnormal amounts of protein in the urine) and protect kidney function better than standard treatment for patients with focal segmental glomerulosclerosis (FSGS).

Detailed Summary:

SPECIFIC AIMS A significant percentage of patients with primary FSGS are resistant to corticosteroids and other immunosuppressive medications. In view of the rising incidence of this disease and the grim prognosis for patients with resistant disease, it is imperative that new therapeutic approaches be evaluated in an efficient and systematic manner. This will enable accurate assessment of the risk-benefit ratio of novel therapies and guide the design of future Phase III randomized clinical trials.

Specific Aim #1: To evaluate two novel therapies for resistant FSGS -- anti-TNF-α antibody and galactose -- against standard therapy

Specific Aim #2: To identify one or more novel agents as candidates for future study in a Phase III randomized clinical trial

OVERALL STUDY DESIGN Screening/Run-In: There is no formal run-in period in the phase II trial because patients with resistant FSGS who will be eligible for this study often have unstable kidney function and are prone to sudden decline in glomerular filtration rate (GFR). An effort will be made to achieve randomization within 2 weeks of the screening visit.

In order to achieve a comparable baseline assessment prior to initiation of one of the novel therapies, the patients must be off all immunosuppressive medications for 30 days. In addition, patients will be placed on the maximal tolerated doses of an angiotensin-converting enzyme inhibitor (ACEI), an angiotensin receptor blocker (ARB), and a lipid-lowering drug defined above based upon measurements of blood pressure, serum K+, creatinine, and cholesterol concentrations. Patients will have to be on stable doses of the ACEI/ARB treatment for a minimum of 2 weeks prior to randomization into the FONT Phase II study to insure
Sponsor: New York University School of Medicine

Current Primary Outcome: Number of Participants With a Reduction in Proteinuria at 6 Months by > 50% of the Value at Screening AND Stable GFR Defined as Greater Than 75 ml/Min/1.73m2 in Those With an Initial Value Above 90 OR Within 25% of Baseline for Remaining Patients [ Time Frame: baseline and 6 months ]

Number of participants with a reduction in proteinuria at 6 months by > 50% of the value at screening AND stable GFR defined as greater than 75 ml/min/1.73m2 in those with an initial value above 90 OR within 25% of baseline for remaining patients.


Original Primary Outcome:

  • A reduction in proteinuria at 6 months by > 50% of the value at the time of screening, and/or [ Time Frame: 6 months ]
  • An estimated GFR (GFRe) that is stable compared to value at enrollment [ Time Frame: 6 months ]


Current Secondary Outcome:

  • Patient Satisfaction Score Using the Treatment Satisfaction Questionnaire for Medication (TSQM Questionnaire) [ Time Frame: Baseline and 6 months ]
    Patient Satisfaction Score Using the Treatment Satisfaction Questionnaire for Medication (TSQM Questionnaire)
  • Number of Participants With Adverse Events [ Time Frame: Up to 7 months ]
  • Percent Change in Proteinuria [ Time Frame: Baseline and 6 months ]
  • Percent Change in or Time to Doubling of Serum Creatinine [ Time Frame: Baseline and 6 months ]


Original Secondary Outcome:

  • Adverse effect profile [ Time Frame: 6 months ]
  • Patient satisfaction score using the TSQM questionnaire (76) [ Time Frame: 6 months ]
  • Percent change in proteinuria (evaluated as a continuous variable) [ Time Frame: 6 months ]
  • Change in or time to doubling of GFRe [ Time Frame: 6 months ]


Information By: New York University School of Medicine

Dates:
Date Received: December 22, 2008
Date Started: December 2008
Date Completion:
Last Updated: July 1, 2016
Last Verified: July 2016