NCT07182227招募中1 期
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 Limited10 个研究点 分布在 2 个国家目标入组 32 人开始时间: 2026年3月30日最近更新:
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 32
- 试验地点
- 10
- 主要终点
- 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
研究概览
简要总结
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).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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^
- •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.
排除标准
- •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.
研究组 & 干预措施
PS-002
Experimental
Part 1: Dose escalation in three groups: Group 1: Low dose, Group 2: Intermediate Dose, Group 3: High Dose.
Part 2: Dose expansion in a fourth group with a selected dose.
干预措施: PS-002 (Genetic)
结局指标
主要结局
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
时间窗: Screening up to Week 48
次要结局
- 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)](Baseline to Week 48)
- 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)](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](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(Baseline to Week 48)
- Change from baseline in urinary soluble terminal complement complex (sC5b-9) levels(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(Baseline to Week 48)
- Change in proteinuria as measured by Urine Protein:Creatinine Ratio (UPCR) derived from 24 hr urine collection(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)](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)](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](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(Baseline to Week 48)
- Change from baseline in urinary soluble terminal complement complex (sC5b-9) levels(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(Baseline to Week 48)
研究者
研究点 (10)
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