Pilot Studies of Novel Therapies to Treat Resistant Focal Segmental Glomerulosclerosis (FSGS)

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00193648
• Date of registration: 10/09/2005
• Prospective Registration: No
• Primary sponsor: North Shore Long Island Jewish Health System
• Public title: Pilot Studies of Novel Therapies to Treat Resistant Focal Segmental Glomerulosclerosis (FSGS)
• Scientific title: Novel Therapies for Resistant FSGS
• Date of first enrolment: July 2005
• Target sample size: 21
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00193648
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Howard Trachtman, MD
• Affiliation: Schneider Children's Hospital of North Shore-LIJ Health System

• Name: Tom Greene, PhD
• Affiliation: The Cleveland Clinic

• Name: Debbie Gipson, MD
• Affiliation: University of North Carolina

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Aged 2-42 years at onset of proteinuria

2. Aged = 42 years at time of randomization (randomization date before 43rd birthday)

3. Estimated glomerular filtration rate (GFR) = 40 ml/min/1.73 m2 at most recent
measurement prior to randomization

1. For patients < age 18 years: Schwartz formula

2. For patients = age 18 years: Cockroft-Gault formula

4. Up/c > 1.0 g/g creatinine on first morning void at time of randomization

5. Biopsy confirmed as primary FSGS (including all subtypes) by study pathologist.

6. Steroid resistance: During the last treatment course with high dose steroids prior
to randomization, the patient must have demonstrated steroid resistance defined below
and not have had a complete remission of proteinuria (Up/c < 0.2 or dipstick urine
protein negative/trace) subsequently. The course of steroid treatment that defines
resistance must be the same or equivalent to at least 4 weeks of every day dosing
with a minimum cumulative dose of 56 mg/kg or 1680 mg of prednisone or its
equivalent.

7. May be taking angiotensin-converting enzyme inhibitor (ACEI), angiotensin receptor
blocking agent (ARB), vitamin E, or lipid lowering therapy

8. Willingness to comply with clinical trial protocol, medications, and follow-up
visits, etc.

9. Screen failure in FSGS-CT based on prior treatment with excluded medication

10. Treatment failure in FSGS-CT based on failure to achieve remission after 26 weeks or
52 weeks of test therapy, i.e., cyclosporine or mycophenolate mofetil (MMF) + oral
dexamethasone pulses

Exclusion Criteria

1. Secondary FSGS

2. Treated with cyclophosphamide, chlorambucil, levamisole, methotrexate, nitrogen
mustard, or other immunosuppressive medications in the 30 days prior to
randomization

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

4. Participation in another therapeutic trial concurrently or for 30 days prior to
randomization

5. Active/serious infection (including, but not limited to hepatitis B or C, HIV)

6. Malignancy

7. Systemic lupus erythematosus (SLE) or multiple sclerosis

8. Hepatic disease defined as serum AST/ALT > 2.5X the upper limit of normal

9. Patients with blood pressure > 140/95 or > 95th percentile for age/height while
receiving maximal doses of 3 or more antihypertensive agents.

10. Diabetes mellitus (DM) type I or II.

11. Hematocrit < 30%

12. Organ transplantation

13. Obesity (based on estimated dry weight at disease onset prior to steroid therapy)
defined as:

1. Body mass index (BMI) > 97th percentile for age if aged 2-20 years

2. BMI > 40 kg/m2 if aged = 21 years

14. Allergy to study medications

15. Inability to consent/assent



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

Health Condition(s) or Problem(s) studied

Focal Glomerulosclerosis

Intervention(s)

Drug: Adalimumab (Humira)

Drug: Rosiglitazone (Avandia)

Primary Outcome(s)

Safety and tolerance of medications [Time Frame: 16 week treatment period]

Secondary Outcome(s)

Reduction in proteinuria [Time Frame: 16 week treatment period]

Secondary ID(s)

DK70341

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

The Cleveland Clinic

University of North Carolina