ADPKD Clinical Trial
— RAPOfficial title:
Rapamycin in Advanced Polycystic Kidney Disease Pilot Study
The purpose of this study is to evaluate the safety of a daily single oral dose of sirolimus in patients with advanced autosomal dominant polycystic kidney disease.
Status | Completed |
Enrollment | 8 |
Est. completion date | April 2012 |
Est. primary completion date | April 2012 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | 18 Years and older |
Eligibility |
Inclusion Criteria: - ADPKD - Eighteen years of age, or older. - Baseline eGFR of 20-40mL/min per 1.73m2. - Negative serum pregnancy test prior to administration of sirolimus and agreement to use contraception throughout the pilot safety study and three months after. Any participant who is getting pregnant during the pilot safety study period will have to discontinue. - Written informed consent. Exclusion Criteria: - Pregnancy or lactation or plans to become pregnant in the near future or disagreement to use contraception. - History of life threatening complications of ADPKD. - Evidence of active systemic- or localized major infection. - Evidence of infiltrate, cavities or consolidation on chest X-ray. - Use of any investigational drug or -treatment up to 4 weeks prior to the enrolment and during the pilot safety study. - Known hypersensitivity to sirolimus and its derivatives. - Treatment with substances known to interfere with the cytochrome p-450 (CYP) 3A4/3A5 systems. - Screening/baseline total white blood cell count below or equal to 3000/mm3. - Screening/baseline platelet count below or equal to 100.000/mm3. - Screening/baseline fasting triglycerides above or equal to 400 mg/dL. - Screening/baseline fasting total cholesterol above or equal to 300 mg/dL. - Concomitant glomerular diseases. - Psychiatric disorders or any condition that might prevent the full comprehension of the purposes and risks of the pilot safety study. - History of malignancies with the exception of adequately treated basal- and squamous-cell carcinomas of the skin. - HIV infection. |
Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label
Country | Name | City | State |
---|---|---|---|
Austria | Division of Nephrology and Dialysis, Department of Medicine III, Medical University Vienna | Vienna |
Lead Sponsor | Collaborator |
---|---|
Medical University of Vienna |
Austria,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Slope in estimated glomerular filtration rate (eGFR; 4 variables MDRD equation) and proteinuria within six months of exposure to sirolimus. | A single daily oral dose of sirolimus with trough levels of 4 to 8ng/dL in patients with advanced polycystic kidney disease and an eGFR of 20-40mL/min per 1.73m2 does not lead to a greater decline in kidney function as represented by the eGFR than -8.8mL/min per 1.73m2 within 6 months (one-sided) as well as it does not lead to an incline in proteinuria, as represented by the logarithm of the protein-creatinine ratio, greater than 0.39 within 6 months (one-sided). | Six months | Yes |
Secondary | Leucopenia | Drop in WBC below 4 G/L | 6 months | Yes |
Secondary | Thrombopenia | Drop in platelets below 150 G/L | 6 months | Yes |
Secondary | Aphthae | New onset of aphthaeous stomatitis under therapy with sirolimus | 6 months | Yes |
Secondary | Dysfunctional wound healing | Dysfunctional and/or prolonged wound healing attributed to sirolimus therapy | 6 months | Yes |
Secondary | Pneumonitis | Persisting cough and infiltrates on chest x-ray | 6 months | Yes |
Secondary | Acne | Acne attributed to sirolimus therapy | 6 months | Yes |
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