Focal Segmental Glomerulosclerosis Clinical Trial (FSGS-CT)

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00135811
• Date of registration: 24/08/2005
• Prospective Registration: No
• Primary sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
• Public title: Focal Segmental Glomerulosclerosis Clinical Trial (FSGS-CT)
• Scientific title: Focal Segmental Glomerulosclerosis Clinical Trial
• Date of first enrolment: November 2004
• Target sample size: 207
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00135811
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 3

Countries of recruitment

United States

Contacts

• Name: Marva Moxey-Mims, MD, FAAP
• Affiliation: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

• Name: Aaron Friedman, MD
• Affiliation: University of Minnesota - Clinical and Translational Science Institute

Key inclusion & exclusion criteria

Inclusion Criteria:

- Age 2-40 years at onset of signs or symptoms of FSGS

- Age = 40 years at time of randomization (randomization date before 41st birthday)

- Estimated GFR = 40 ml/min/1.73 m2 at most recent measure prior to randomization

- For participants < age 18 years: Schwartz formula

- For participants = age 18 years: Cockroft-Gault formula

- Up/c > 1.0 g protein/g creatinine on first am void at time of randomization

- Biopsy confirmed as primary FSGS (including all subtypes) by study pathologist. A
minimum of 1 glomerulus demonstrating segmental sclerosis on light microscopy will be
required to confirm the diagnosis.

- Steroid resistance: The participant must have demonstrated steroid resistance
(defined as a failure to achieve a sustained Up/c = 1.0) based on at least one
treatment course with high dose steroids prior to randomization which satisfies both
of the following conditions:

- minimal treatment duration of 4 weeks

- minimum cumulative dose of 56 mg/kg or 1680 mg of prednisone or its equivalent.
In addition, the participant must not have had a complete remission of
proteinuria (Up/c < 0.2 or dipstick urine protein 0/trace) subsequent to the
latest qualifying 4-week course demonstrating steroid resistance.

- Willingness to follow the clinical trial protocol, including medications, and
baseline and follow-up visits and procedures.

- Participants may be taking ACEI, ARB, Vitamin E, or lipid lowering therapy.

Exclusion Criteria:

- Secondary FSGS

- Prior therapy with sirolimus, CSA, tacrolimus, MMF, or azathioprin (Imuran)

- Treated with cytoxan, chlorambucil, levamisole, methotrexate, or nitrogen mustard in
the last 30 days

- Lactation, pregnancy, or refusal of birth control in women of child bearing potential

- Participation in another therapeutic trial concurrently or 30 days prior to
randomization

- Active/serious infection (including, but not limited to Hepatitis B, C, or HIV)

- Malignancy

- Blood pressure > 140/95 or > 95th percentile for age/height.

- Participant is receiving 4 or more antihypertensive agents for the primary purpose of
controlling blood pressure.

- Participants with previously diagnosed diabetes mellitus type I or II: the diagnosis
of DM I or II will be based on local criteria for participants with an established
diagnosis. If hyperglycemia is detected during the screening period, the WHO
criteria for the diagnosis of DM I and II will be used.

- Clinical evidence of cirrhosis or chronic active liver disease

- Abnormal laboratory values at the time of study entry:

- Absolute neutrophil count (ANC) < 2000/mm3, or

- Hematocrit (HCT) < 28%

- History of significant gastrointestinal disorder, e.g, severe chronic diarrhea (> 5
watery stools per day) or active peptic ulcer disease.

- Organ transplantation

- Obesity (based on estimated dry weight at onset of disease prior to steroid therapy)
defined as

- BMI > 97th percentile for age if aged 2-20 years

- BMI > 40 kg/m2 for age =21 years

- Allergy to study medications

- Inability to consent/assent

Note: Participants with conditions meeting exclusion criteria at a particular evaluation
for eligibility may be re-evaluated at a later time to determine if the conditions have
changed so that all entry criteria are met. In particular, if blood pressure > 140/95 or
> 95th percentile for age/height while the participant is on less than three
antihypertensive agents, the participant may be re-evaluated for eligibility after adding
other antihypertensive agents so long as the total number of agents does not exceed three.



Age minimum: 2 Years
Age maximum: 40 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Glomerulosclerosis, Focal

Intervention(s)

Drug: Cyclosporin

Drug: MMF and Dexamethasone

Primary Outcome(s)

The primary outcome is a 6-level ordinal variable defined based on the achievement of remission from proteinuria during the first 52 weeks after randomization. [Time Frame: first 52 weeks after randomization.]

Secondary Outcome(s)

The main secondary outcome is a 5-level ordinal variable defined based on the persistence of remissions after immunosuppressive agents are withdrawn. [Time Frame: based on the participant's level of proteinuria during the period from week 52 through week 78 following withdrawal of CSA or MMF/Pulse steroids]

Secondary ID(s)

63490 (completed)

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

The Cleveland Clinic