PS-002 treatment for adults with IgA nephropathy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This first-in-human Phase 1/2 interventional trial will give PS-002 to adults with biopsy-proven primary IgA nephropathy who have evidence of complement C3 deposition, persistent proteinuria (≥1 g/g or ≥1 g/24 h), and an eGFR ≥45 mL/min/1.73 m2 despite best supportive care. Participants will be monitored closely for safety, tolerability, and preliminary signs of biological activity for up to one year after dosing, with an option for long-term follow-up extending to five years. The study will be conducted at multiple U.S. academic centers and will include regular clinic visits, blood and urine testing, and kidney function monitoring. A typical Phase 1/2 dose-escalation and expansion design will be used to define an acceptable safety profile and initial signals of efficacy.

Why it matters

Who can join: the full rules

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 excretion greater than or equal to 1 g/24 h (greater than or equal to 1000 mg/24h) sampled from 24 h urine collection.
* eGFR calculated using the CKD-EPI formula greater than or equal to 30 mL/min/1.73m\^2.
* Sitting office systolic blood pressure equal to or less than 140 mmHg, diastolic blood pressure equal to or less than 90 mmHg.
* All participants must have been on best supportive care for IgAN, as per region-specific requirements defined in the protocol.

Exclusion Criteria:

* A participant has nephrotic syndrome, defined for this purpose as 24 h urine protein greater than 3.5g with concurrent hypoalbuminemia (serum albumin less than 3.0 g/dL \[less than 30 g/L\]).
* Any secondary IgAN defined as associated with gastrointestinal and liver disorders (liver cirrhosis, celiac disease, Crohn's disease, ulcerative colitis), autoimmune disorders (dermatitis herpetiformis, psoriasis, seronegative arthritis, systemic lupus erythematosus, rheumatoid arthritis), malignancy (IgA myeloma, lymphoma, lung cancer, renal cell cancer, cutaneous T-cell lymphoma), respiratory disorders (bronchiolitis obliterans, idiopathic pulmonary fibrosis) etc.
* Having a major concurrent non-IgAN-related disease that, in the opinion of the investigator, prevents the assessment of IgAN.
* History of malignancy; or bone marrow or organ transplant.
* History of, or currently active primary or secondary immunodeficiency, including known history of human immunodeficiency virus infection, and other severe immunodeficiency blood disorders.
* Presence of other significant medical conditions that would create an unacceptable procedure or anesthesia risk.
* Aspartate aminotransferase or alanine aminotransferase greater than 1.5 times the upper limit of normal.
* History of serious infection requiring parenteral antibiotics within the past 8 weeks prior to study drug administration.
* Participants previously treated with immunosuppressive/immunomodulatory agents such as, but not limited to, cyclophosphamide, infliximab, complement inhibitor, canakinumab, mycophenolate mofetil, mycophenolate sodium, cyclosporine, tacrolimus, everolimus, or systemic corticosteroids (exposure greater than 7.5mg/day prednisone/prednisolone equivalent) within 90 days (or 180 days for rituximab) prior to Screening. Participants previously or currently receiving oral budesonide (Kinpeygo/Tarpeyo) require wash out for 90 days prior to the study drug administration.
* Exposed to a live or attenuated vaccine within the 6 weeks prior to study drug administration.
* Participants with a known sensitivity or intolerance to corticosteroid therapy.
* Known hypersensitivity to study drug ingredients.
* Prior treatment with PS-002 or any other gene therapy, or participation in any other investigational trial during this study.
* Positive serology for hepatitis B or C, i.e., positive hepatitis B surface antigen or hepatitis C ribonucleic acid (RNA) viral load positive.
* Participants treated with potentially hepatotoxic medications unless they have been monitored in accordance with the drug label and have received a stable dose since \>90 days prior to dosing without clinically significant liver enzyme fluctuations.

Note: Other protocol-defined inclusion/exclusion criteria may apply.

Where it's running

• University of Miami Hospital
  Miami, Florida, United States Recruiting
• The Johns Hopkins Hospital
  Baltimore, Maryland, United States Recruiting
• Manchester University NHS Foundation Trust
  Manchester, Greater Manchester, United Kingdom Recruiting
• Salford Royal Hospital, Northern Care Alliance NHS Foundation Trust
  Manchester, Greater Manchester, United Kingdom Recruiting
• Nottingham University Hospitals NHS Trust
  Nottingham, Nottinghamshire, United Kingdom Recruiting
• Southmead Hospital
  Bristol, United Kingdom Recruiting

Study contacts

• Study coordinator: Clinical Operations
• Email: contact@purespringtx.com
• Phone: +44 (0)20 3855 6324

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.