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

Naval Military Medical University (Second Military Medical University)

Status and phase: Unknown; Phase 3

Conditions: Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Treatments: Drug: Placebo; Drug: Triptolide-Containing Formulation

Study type: Interventional

Funder types: Other

Identifiers

NCT02115659

CZKIPLA-ADPKD-002

Details and patient eligibility

About

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.

Full description

Randomized Controlled Trial

Enrollment: 100 estimated patients

Sex: All

Ages: 40 to 75 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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100 participants in 2 patient groups, including a placebo group

Placebo

Placebo Comparator group

Description:

Placebo plus standard treatment. Anti-hypertension drug(s) for hypertension; Antibiotics for cyst infections; cause oriented treatment for flank pain.

Treatment:

Drug: Placebo

Triptolide-Containing Formulation

Experimental group

Description:

Triptolide-Containing Formulation (1mg/kg/d) was prescribed; Dosage will be adjusted if necessary according to the adverse events monitoring.

Treatment:

Drug: Triptolide-Containing Formulation