Safety and Pharmacokinetics of FG-3019 in Adolescents and Adults With Focal Segmental Glomerulosclerosis (FSGS)

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the safety and tolerability of FG-3019 administered over 8 weeks to adolescent and adult subjects with steroid-resistant focal segmental glomerulosclerosis (FSGS).

Official Title
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A Phase I, Open-Label Study of the Safety and Pharmacokinetics of FG-3019 in Adolescent and Adult Subjects With Steroid-Resistant Focal Segmental Glomerulosclerosis

Conditions
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Focal Segmental Glomerulosclerosis

Intervention / Treatment
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* Drug: FG-3019


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Age 12-64 years, inclusive, and girls age 10-11 years, inclusive if Tanner stage 3 or greater Biopsy diagnosis of primary FSGS with biopsy confirmed centrally Age less than or equal to 2 years old at onset of proteinuria First morning urine protein/creatinine ratio (U p/c) >1 gm/gm Estimated glomerular filtration rate greater than or equal to 40 mL/min/1.73 m2 Exclusion Criteria: Non-FSGS renal disease other than benign cyst; or secondary FSGS History of organ transplantation History of allergic or anaphylactic reaction to human, humanized, chimeric or murine monoclonal antibodies History of malignancy, cardiovascular disease, diabetes mellitus, sickle cell disease, multiple sclerosis, systemic lupus erythematosus, or active or recurrent serious infections (including but not limited to Hepatitis B, Hepatitis C, or HIV) Participation in studies of investigational drugs within 6 weeks prior to Day 0 or receipt of an investigational drug within 12 weeks prior to Day 0 Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 2.5 times the upper limit of normal Hematocrit < 30%

Ages Eligible for Study
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Minimum Age: 12 Years
Maximum Age: 64 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: FG-3019<br>FG-3019 5 mg/kg | Drug: FG-3019<br>* FG-3019 5 mg/kg IV given over 2 hours every 2 weeks for 8 weeks<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety and tolerability of FG-3019 as measured by AEs, SAEs, physical exams, lab tests, and the Treatment Satisfaction Questionnaire for Medications |  | 32 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Standard plasma and urinary PK parameters |  | 32 weeks | 
| Change from baseline in urinary protein/creatinine, albumin/creatinine, albumin excretion rate, estimated glomerular filtration rate (eGFR), 24 hour urinary protein and fasting serum lipids |  | 8 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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steroid resistant focal segmental glomerulosclerosis, FSGS, adolescent focal segmental glomerulosclerosis, focal segmental glomerulosclerosis