A Phase 1/2 Multicenter, Open Label, Two-part Study (Single Ascending Dose [Part 1], and Dose Expansion [Part 2]) to Evaluate Safety, Tolerability and Efficacy of PS-002, a Gene Therapy for the Treatment of Adult Participants With Primary IgA Nephropathy

Purespring Therapeutics Limited

Summary

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).

Eligibility

Age range: 18+ years
Sex: All
Healthy volunteers: No

Detailed criteria

Inclusion Criteria: * Diagnosis of primary IgA nephropathy (IgAN) as evidenced by renal biopsy. * A historic kidney biopsy performed within 36 months prior to screening with reported evidence of complement component 3 (C3) deposition. If the participant had a kidney biopsy performed over 36 months prior to Screening, a new kidney biopsy should be carried out during the Screening period. This biopsy must exhibit signs of ongoing complement system activity. * Proteinuria assessed during screening as UPCR greater than or equal to 0.75 g/g (greater than or equal to 750 mg/g) OR total protein excr…

Locations (10)

University of Miami Hospital
Miami, Florida
The Johns Hopkins Hospital
Baltimore, Maryland
Manchester University NHS Foundation Trust
Manchester, Greater Manchester
Salford Royal Hospital, Northern Care Alliance NHS Foundation Trust
Manchester, Greater Manchester
Leicester General Hospital
Leicester, Leicestershire
Nottingham University Hospitals NHS Trust
Nottingham, Nottinghamshire

Summary

Eligibility

Interventions

Locations (10)