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
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 as assessed at the Screening visit by UPCR at least 1g/g (at least 1000 mg/g) OR total protein excretion at least 1 g/24 h (a…
Interventions
- GeneticPS-002
Adeno-associated viral vector containing the human Complement Factor I (CFI) gene
Locations (11)
- University of Miami HospitalMiami, Florida
- The Johns Hopkins HospitalBaltimore, Maryland
- University of Michigan HospitalAnn Arbor, Michigan
- Manchester University NHS Foundation TrustManchester, Greater Manchester
- Salford Royal Hospital, Northern Care Alliance NHS Foundation TrustManchester, Greater Manchester
- Leicester General HospitalLeicester, Leicestershire