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

Phase 3

• Conditions: Autosomal Dominant Polycystic Kidney Disease (ADPKD)
• Interventions: Drug: Placebo; Drug: Triptolide-Containing Formulation

• Registration Number: NCT02115659
• Lead Sponsor: Shanghai Changzheng Hospital

Brief Summary

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

Randomized Controlled Trial

Study Timeline

• Status Verified: 2014-04
• Study Start: 2014-04
• Study First Submitted: 2014-04-14
• Study First Submitted QC: 2014-04-15
• Study First Posted: 2014-04-16
• Last Update Submitted: 2014-04-16
• Last Update Posted: 2014-04-17
• Primary Completion: 2016-05
• Study Completion: 2016-05

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 100

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	Placebo plus standard treatment. Anti-hypertension drug(s) for hypertension; Antibiotics for cyst infections; cause oriented treatment for flank pain.
Triptolide-Containing Formulation	Triptolide-Containing Formulation	Triptolide-Containing Formulation (1mg/kg/d) was prescribed; Dosage will be adjusted if necessary according to the adverse events monitoring.

Primary Outcome Measures

Name	Time	Method
renal volume measured by high resolution magnetic resolution imaging	2 yrs

Secondary Outcome Measures

Name	Time	Method
Estimated glomerular filtration rate (eGFR)	2 yrs
Adverse events.	2 yrs

Trial Locations

Locations (1)

Shanghai Changzheng Hospital; 🇨🇳 Shanghai, Shanghai, China