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
NOT_YET_RECRUITINGThe Johns Hopkins Hospital
Baltimore, Maryland, United States
Number 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.
University of Michigan Hospital
Ann Arbor, Michigan, United States
NOT_YET_RECRUITINGManchester University NHS Foundation Trust
Manchester, Greater Manchester, United Kingdom
NOT_YET_RECRUITINGSalford Royal Hospital, Northern Care Alliance NHS Foundation Trust
Manchester, Greater Manchester, United Kingdom
NOT_YET_RECRUITINGLeicester General Hospital
Leicester, Leicestershire, United Kingdom
NOT_YET_RECRUITINGNottingham University Hospitals NHS Trust
Nottingham, Nottinghamshire, United Kingdom
NOT_YET_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
NOT_YET_RECRUITING...and 1 more locations
Time frame: Baseline to Week 48