The purpose of the study is to evaluate safety, tolerability, and preliminary effectiveness following administration of PS-002 in adults with primary Immunoglobulin A (IgA) nephropathy. This will be a first-in-human study and will include participants at high risk of disease progression despite receiving current standard-of-care treatment. Participants will be monitored for up to one year after receiving PS-002 and invited to take part in a long-term follow-up study (total follow-up: 5 years).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
32
Adeno-associated viral vector containing the human Complement Factor I (CFI) gene
University of Miami Hospital
Miami, Florida, United States
RECRUITINGThe Johns Hopkins Hospital
Baltimore, Maryland, United States
RECRUITINGNumber of participants with: Treatment-Emergent Adverse Events (TEAEs) and serious TEAEs, TEAEs and serious TEAEs related to PS-002, TEAEs and serious TEAEs related to the PS-002 administration procedure
Time frame: Screening up to Week 48
Change in proteinuria as measured by Urine Protein:Creatinine Ratio (UPCR) derived from 24 hr urine collection
Time frame: Baseline to Week 36
Change from baseline in proteinuria as measured by urine protein creatinine ratio (UPCR) from spot urine collection. [UPCR (g/g) will be calculated from spot urine collection (first morning void)]
Time frame: Baseline to Week 48
Change from baseline in proteinuria as measured by urine albumin:creatinine ratio (UACR). [UACR (g/g) will be calculated from spot urine collection (first morning void)]
Time frame: Baseline to Week 48
Change from baseline in proteinuria as measured by UACR from 24 hour urine collection. [UACR (mg/day) will be calculated from 24 hour urine collection at Week 36 only]
Time frame: Baseline to Week 36
Change from baseline in creatinine and estimated Glomerular Filtration Rate (eGFR) values calculated using the Chronic Kidney Disease Epidemiology (CKD-EPI) creatinine formula
Time frame: Baseline to Week 48
Change from baseline in urinary soluble terminal complement complex (sC5b-9) levels
Time frame: Baseline to Week 48
Time to worsening of kidney function. Defined as: 1) sustained eGFR decline by at least 40 percent, 2) onset of end-stage kidney disease, 3) initiation of renal replacement therapy, or 4) all-cause mortality
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Manchester University NHS Foundation Trust
Manchester, Greater Manchester, United Kingdom
RECRUITINGSalford Royal Hospital, Northern Care Alliance NHS Foundation Trust
Manchester, Greater Manchester, United Kingdom
RECRUITINGLeicester General Hospital
Leicester, Leicestershire, United Kingdom
NOT_YET_RECRUITINGNottingham University Hospitals NHS Trust
Nottingham, Nottinghamshire, United Kingdom
RECRUITINGSouthmead Hospital
Bristol, United Kingdom
RECRUITINGCardiff and Vale University Health Board
Cardiff, United Kingdom
NOT_YET_RECRUITINGRoyal Infirmary of Edinburgh Clinical Research Facility
Edinburgh, United Kingdom
RECRUITINGThe Royal London Hospital
London, United Kingdom
RECRUITINGTime frame: Baseline to Week 48