2023-504597-37-00招募中3 期
A pivotal Phase 3, multicenter, randomized, double-blind, placebo-controlled study of the efficacy and safety of DMX-200 in patients with focal segmental glomerulosclerosis (FSGS) who are receiving an angiotensin II receptor blocker (ARB)
Dimerix Bioscience Pty Limited47 个研究点 分布在 7 个国家目标入组 95 人开始时间: 2023年6月29日最近更新:
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 95
- 试验地点
- 47
- 主要终点
- Percent change in urine PCR (based on 24-hour urine collection) following treatment with DMX-200 compared with placebo
研究概览
简要总结
To evaluate the efficacy of DMX-200 in terms of urine PCR and eGFR slope in adult patients with FSGS who are receiving an ARB. OLE: To assess the long-term safety and tolerability of open-label treatment with DMX-200 in patients with FSGS who are receiving an ARB.
研究设计
- 分配方式
- Not Applicable
- 主要目的
- Open Label Extension
- 盲法
- None
入排标准
- 年龄范围
- 0 years 至 65+ years（0-17 Years, 65+ Years, 18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •Primary FSGS, genetic FSGS, or FSGS of undetermined cause (FSGS-UC) with timing of initial diagnosis within 7 years prior to screening. NOTE: i) Primary FSGS and FSGS-UC, the initial diagnosis must be biopsy-proven within 7 years prior to Screening. The kidney biopsy could have been obtained at any time within the previous 7 years and should be based on light microscopy with supportive findings on either electron microscopy or immunofluorescence. ii) For Genetic FSGS, no biopsy is required where there is a documented genetic mutation of a podocyte protein associated with FSGS. The genetic testing should have been obtained within 7 years prior to screening. iii) All patients should demonstrate a clinical history and disease course consistent with FSGS.
- •OLE: The patient received blinded IP throughout the duration of the double-blind period up to the Week 104 (EOT – DB) visit (ie, did not prematurely and permanently discontinue the IP).
- •Must be either receiving an ARB at the maximal tolerated dose and ≥ 50% of the maximum recommended dose per the product label prior to Screening, or willing to transition to this treatment (including transition from an ACE inhibitor) prior to stabilization
- •If taking corticosteroids, the dosage must be ≤ 10mg / day prednisone (or equivalent) and stable for ≥4 weeks prior to and during both Screening and Stabilization, and there must be no plan to change their corticosteroid treatment regimen during study. Use of inhaled corticosteroids for respiratory diseases is allowed.
- •If taking aldosterone inhibitors, mineralocorticoid receptor antagonists, direct renin inhibitors, sodium-glucose co-transporter-2 inhibitors, or endothelin receptor antagonists (including dual antagonists), the dose and regimen must be stable for ≥12 weeks prior to Screening and maintained during Stabilization and patients must have no plan to change their treatment regimen during the study
- •Urine protein/creatinine ratio (PCR) >1.5 g/g (>169.5 mg/mmol) or 24-hour total protein >1.5 g/day based on 24-hour urine collection during Screening and Qualification
- •Estimated glomerular filtration rate (eGFR) at screening: For adults (≥ 18 years): eGFR ≥25 and ≤120 mL/min/1.73 m2 using the CKD Epidemiology Collaboration (CKD-EPI) Creatinine Equation (2009) For adolescent patients (<18): eGFR ≥25mL/min/1.73 m2using the Modified Schwartz formula
- •Seated blood pressure ≤160/100 mm Hg (mean of 3 values) (patients ≥18 years of age) or between the 5th and 95th percentile for age, sex, and height (patients <18 years of age) at Screening
- •Body weight ≥35 kg (all patients) AND a body mass index (BMI) ≤40 kg/m2 (patients ≥18 years of age) or between the 5th and 98th percentile for age and sex (patients <18 years of age) at Screening. For patients with moderate or severe edema, BMI will be calculated based on remission or premorbid weight measured within 3 months prior to Screening, if available. If not available, BMI will be calculated based on the estimated dry weight, based on the Investigator’s clinical judgment
- •OLE: Patients who have completed participation in the double-blind period, including the Week 104 visit (including non-responders, those with disease worsening, and those receiving additional FSGS-directed therapies)
排除标准
- •Has FSGS secondary to another condition
- •OLE: The patient has met the criteria for premature and permanent IP discontinuation as defined in Section 7.1 or study discontinuation as defined in Section 7.2 at Week 104, or prior to the first open-label dose of DMX 200 at Week
- •Patients with nephrotic syndrome (>3.5 g/day proteinuria and serum albumin <30 g/L) who have not previously been treated with standard of care FSGS- directed therapies (including steroids).
- •OLE: Any safety concerns identified during the double-blind period which, in the Investigator’s opinion, may interfere with the patient’s continued participation during the OLE period.
- •History of type 1 diabetes mellitus, or uncontrolled type 2 diabetes mellitus (defined as glycated hemoglobin >8%)
- •History of lymphoma, leukemia, or any active malignancy within the past 2 years (except for basal cell or squamous cell carcinomas of the skin or cervical carcinoma in situ that have been resected and with no evidence of metastatic disease)
- •Active clinically significant hepatobiliary disease
- •Documented history of heart failure (New York Heart Association Class III/IV) or a major adverse cardiac event within 12 weeks prior to Screening
- •Serum potassium levels >5.5 mmol/L at Screening
- •Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) >2 × upper limit of normal at Screening
- •Received any of the following with the specified timeframe prior to Screening - Treatment with non-steroid immunosuppressant agents including biological drugs (eg. rituximab), calcineurin inhibitors, cyclophosphamide, azathioprine, or mycophenolate mofetil within 12 weeks prior to Screening
结局指标
主要结局
Percent change in urine PCR (based on 24-hour urine collection) following treatment with DMX-200 compared with placebo
Percent change in urine PCR (based on 24-hour urine collection) following treatment with DMX-200 compared with placebo
Slope of eGFR following treatment with DMX-200 compared with placebo
Slope of eGFR following treatment with DMX-200 compared with placebo
OLE: Incidence and severity of treatment-related AEs and any AESIs and SAEs following long-term treatment with DMX-200
OLE: Incidence and severity of treatment-related AEs and any AESIs and SAEs following long-term treatment with DMX-200
次要结局
- Incidence and severity of AEs following treatment with DMX-200 compared with placebo
- Incidence of clinically significant changes in the safety profile of patients treated with DMX-200 compared with placebo, as measured by changes from baseline in clinical laboratory evaluations (hematology, coagulation, clinical chemistry, and urinalysis), ECGs, vital signs, and physical examinations
- Proportion of patients achieving proteinuria response following treatment with DMX-200 compared with placebo at any time during the double-blind period, defined as: - Complete response: 24-hour urine PCR reduction to <0.3 g/g [<33.9 mg/mmol] - Modified partial remission (FPRE): 24-hour urine PCR reduction ≥40% from Baseline and <1.5 g/g [<169.5 mg/mmol] - No response (failure to meet any response criteria)
- Proportion of patients on treatment with DMX-200 compared with placebo that meet a composite endpoint of worsening in kidney function, as defined by the onset of kidney failure (initiation of chronic dialysis, kidney transplantation, or a sustained eGFR of <15 mL/min/1.73 m2), a 40% decline in eGFR from Baseline, or death from kidney or cardiovascular causes
- OLE: Slope of eGFR from Week 108 (Baseline)
- OLE: Percent change in urine PCR (based on first morning void urine samples) from Week 108 (Baseline) at each visit
- OLE: Proportion of patients on treatment with DMX 200 that meet a composite endpoint of worsening in kidney function, as defined by the onset of kidney failure (initiation of chronic dialysis, kidney transplantation, or a sustained eGFR of <15 mL/min/1.73 m2), a 40% decline in eGFR from Baseline, or death from kidney or cardiovascular causes
- OLE: Change in eGFR from Week 108 (Baseline) at each visit
研究者
Clinical Trials Information
Scientific
Dimerix Bioscience Pty Limited
研究点 (47)
Loading locations...
相似试验
招募中
1 期
A Study of GFH375 Combined With Cetuximab or Chemotherapy in Participants With Solid Tumors Harboring KRAS G12D MutationNCT07259590Genfleet Therapeutics (Shanghai) Inc.126
尚未招募
2 期
Efficacy, Safety, Tolerability, and Biomarker Effects of GT-02287 in Early Parkinson's DiseaseParkinson DiseaseNCT07280299Gain Therapeutics, Inc.111
招募中
3 期
Trial of T-DXd Versus SoC Chemotherapy as Adjuvant Treatment in Endometrial Cancer2024-519444-33-00Daiichi Sankyo Inc.45
招募中
3 期
EASE SBS 5 (Efficacy And Safety Evaluation of Glepaglutide in Treatment of SBS)Short Bowel Syndrome2024-512486-14-00Zealand Pharma A/S3
已完成
2 期
A Biomarker-Directed Phase 2 Trial of SY-1425, a Selective Retinoic Acid Receptor Alpha Agonist, in Adult Patients with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)2024-516281-11-00Syros Pharmaceuticals Inc.1