Triptolide-Containing Formulation as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Study Overview
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Brief Summary
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Triptolide was shown in experimental studies to inhibit the cyst formation and growth in ADPKD models, while triptolide-containing formulation was revealed to potentially slow the disease progression in several proteinuric ADPKD patients in our clinical practice. It remains to be shown the effect of triptolide-containing formulation on total kidney volume (TKV) enlargement and renal function protection in ADPKD patients.

Detailed Description
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Randomized Controlled Trial

Official Title
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Randomized Controlled Trial of Triptolide-Containing Formulation for Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Conditions
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Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Intervention / Treatment
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* Drug: Triptolide-Containing Formulation
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: ADPKD patient older then 40 years of age without gender limitation Estimated glomerular filtration rate (eGFR) higher than 60 ml/min 1.73m2 documented kidney volume progression with yearly increasing rate more than 6% informed consent Exclusion Criteria: Female who is planning to become pregnant, who is pregnant and/or lactating, who is unwilling to use effective means of contraception impaired liver function as increased liver enzymes (2-fold above normal values) uncontrolled hypercholesterolemia (fasting cholesterol > 8 mmol/l) or hypertriglyceridaemia (> 5 mmol/l) under lipid lowering therapy granulocytopenia (white blood cell < 3,000/mm3) or thrombocytopenia (platelets < 100,000/mm3) hepatitis B or C, HIV infection malignancy mental illness that interfere with the patient ability to comply with the protocol drug or alcohol abuse known hypersensitivity to similar drugs as Triptolide-Containing Formulation

Ages Eligible for Study
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Minimum Age: 40 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Placebo Comparator: Placebo<br>Placebo plus standard treatment. Anti-hypertension drug(s) for hypertension; Antibiotics for cyst infections; cause oriented treatment for flank pain. | Drug: Placebo<br>* Placebo plus standard treatment of complications. Anti-hypertension drug(s) for hypertension; Antibiotics for cyst infections; cause oriented treatment for flank pain, etc.<br>* Other names: Placebo plus standard treatment.;|
| Experimental: Triptolide-Containing Formulation<br>Triptolide-Containing Formulation (1mg/kg/d) was prescribed; Dosage will be adjusted if necessary according to the adverse events monitoring. | Drug: Triptolide-Containing Formulation<br>* Triptolide-Containing Formulation (1mg/kg/d) plus standard treatment of complications. Anti-hypertension drug(s) for hypertension; Antibiotics for cyst infections; cause oriented treatment for flank pain.<br>* Other names: Triptolide-Containing Formulation plus standard treatment.;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| renal volume measured by high resolution magnetic resolution imaging |  | 2 yrs | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Estimated glomerular filtration rate (eGFR) |  | 2 yrs | 
| Adverse events. |  | 2 yrs | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ADPKD, triptolide