Clinical trial NCT05759754: Effects of Traditional Chinese Medicine (GSJYF) in Children With Inherited Proteinuric Kidney Disease
Official title: Treatment Effects of Traditional Chinese Medicine (Gu Shen Juan Yu Formula) in Children With Inherited Proteinuric Kidney Disease：A Multicenter, Open-Label, Two-Arm, Crossover Randomized Clinical Trial
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Proteinuria
Kidney Diseases
Hereditary Nephropathy
Keywords: Traditional Chinese Medicine
anti-proteinuric
Lead sponsor: Children's Hospital of Fudan University
Sponsor class: OTHER
Collaborators: name: Anhui Provincial Children's Hospital
name: Henan Provincial People's Hospital
name: Wuhan Union Hospital, China
name: Children's Hospital of Nanjing Medical University
name: The Children's Hospital of Zhejiang University School of Medicine
name: First Affiliated Hospital, Sun Yat-Sen University
name: Shandong Provincial Hospital
name: Xuzhou Children Hospital
First posted: 2023-03-08
Enrollment: 72
Sex: ALL
Minimum age (years): 3
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to determine whether traditional Chinese medicine, Gu Shen Juan Yu Formula, as complementary treatment is safe and effective in the treatment of Inherited Proteinuric Kidney Disease.

Detailed description: Current therapy for inherited proteinuric kidney disease remains limited. Renin-angiotensin-aldosterone-system (RAAS) inhibitors such as Angiotensin converting enzyme inhibitors (ACEI) or angiotensin II receptor blockers (ARBs) help to control proteinuria and slow the progression of kidney function loss. However, a substantial proportion of patients progress to end-stage renal disease, which is partly associated with high residual proteinuria. Novel therapies to decrease proteinuria and limit CKD progression are needed. Traditional Chinese medicine (TCM) has been extensively used in China for thousands of years and favored in treatment of chronic disease for multi-factorial, multi-target action.

The investigators' hypothesis is that this traditional Chinese medicine, targeted to podocyte injury and repair named Gu Shen Juan Yu Formula, can significantly reduce proteinuria and delay renal function loss in patients with proteinuric kidney disease. To test this hypothesis, the investigators plan to initiate a multicenter, open-label, two-arm, crossover, randomized clinical trial in 72 children with proteinuria kidney disease. The study population will consist of children with proteinuria who are on receiving routine therapy such as ACE inhibitor or angiotensin II receptor blocker treatment. In this study, participants were randomly assigned (1:1) to receive first either TCM+Routine Therapy or Routine Therapy for 12 weeks. A 2 weeks washout period was used before crossover.

The primary objective was to measure the change of urine protein excretion and estimated glomerular filtration rate (eGFR) during the treatment period.

Eligibility criteria: Inclusion Criteria:

* Aged 3-18 years；
* Proteinuria associated with hereditary nephropathy, which was confirmed by mutations in podocyte genes or syndromal genes (including collagens);
* Urine protein more than 500 mg/24 hours and/or UPCR (in first-morning void) more than 0.5 mg/mg at time of baseline despite ACEI or ARB treatment for at least 3 months;
* Estimated GFR ≥ 45 ml/min/1.73m2 (estimated with Schwartz formula);
* Without any immunosuppressive medications such as corticosteroids, calcineurin inhibitors, etc；
* On a stable dose of ACEI or ARB for at least 4 weeks；
* Willingness to give written consent and comply with the study protocol.

Exclusion Criteria:

* Diagnosed with Nephronophthisis, Polycystic kidney disease etc;
* With abnormal liver function, ALT or AST \>3.0 x upper limit of normal (ULN) at enrollment;
* Receiving immunosuppressive therapy (including corticosteroids, and other immune suppressive medicine) within three months prior to enrollment;
* Receiving other traditional Chinese medicine and/or its analogue which can reduce proteinuria within the past 2 weeks. Patients who are taking other TCM treatment could be enrolled after 2 weeks of wash out period;
* Has undergone major organ transplantation (e.g. heart, kidney, liver);
* Any medication, surgical or medical condition which might significantly alter the absorption, distribution, or metabolism including, but not limited to any of the following: active inflammatory bowel disease, received major gastrointestinal tract surgery;
* History of noncompliance to medical regimens or inability to comply with the study and follow-up procedures.

Primary outcomes:
measure: Percentage change in urinary protein-creatinine ratio (UPCR) from baseline to week 26
timeFrame: baseline, week 12, week 14, week 26
measure: Changes in estimated glomerular filtration rate (eGFR) from baseline to week 26
timeFrame: baseline, week 12, week 14, week 26
measure: Percentage change in urinary albumin-creatinine ratio (UACR) from baseline to week 26
timeFrame: baseline, week 12, week 14, week 26
measure: Percentage change in 24-hour protein from baseline to week 26
timeFrame: baseline, week 12, week 14, week 26
Secondary outcomes:
measure: Changes in serum albumin from baseline to week 26
timeFrame: baseline, week 12, week 14, week 26
measure: Changes of Traditional Chinese Medicine syndrome scores after treatment
timeFrame: Baseline, week 26
measure: Changes in liver function parameters from baseline to week 26: Alanine Aminotransferase (ALT)
timeFrame: baseline, week 12, week 14, week 26
measure: Changes in liver function parameters from baseline to week 26: Aspartate Aminotransferase (AST)
timeFrame: baseline, week 12, week 14, week 26
Other outcomes:
measure: Number of subjects reporting adverse events （AEs）during the treatment period
timeFrame: baseline to week 26
Locations:
city: Hefei
country: China
facility: Anhui Provincial Children's Hospital
state: Anhui
city: Guangzhou
country: China
facility: First Affiliated Hospital, Sun Yat-Sen University
state: Guangzhou
city: Zhengzhou
country: China
facility: Children's Hospital Affiliated to Zhengzhou University/Henan Children's Hospital
state: Henan
city: Wuhan
country: China
facility: Wuhan Children's Hospital,Tongji Medical College, Huazhong University of Science and Technology.
state: Hubei
city: Nanjing
country: China
facility: Children's Hospital of Nanjing Medical University
state: Jiangsu
city: Shandong
country: China
facility: Shandong Provincal Hospital
state: Shandong
city: Hangzhou
country: China
facility: The Children's Hospital of Zhejiang University School of Medicine
state: Zhejiang
city: Shanghai
country: China
facility: Children's Hospital of Fudan University
city: Xuzhou
country: China
facility: Xuzhou Children's Hospital
Study officials:
affiliation: Children's Hospital of Fudan University
name: Hong Xu, PhD
role: STUDY_CHAIR
References: