The Vienna RAP Pilot Study

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT01632605
• Date of registration: 13/05/2012
• Prospective Registration: No
• Primary sponsor: Medical University of Vienna
• Public title: The Vienna RAP Pilot Study RAP
• Scientific title: Rapamycin in Advanced Polycystic Kidney Disease Pilot Study
• Date of first enrolment: November 2009
• Target sample size: 8
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT01632605
• Study type: Interventional
• Study design: Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label
• Phase: N/A

Countries of recruitment

Austria

Contacts

• Name: Gere Sunder-Plassmann, MD
• Affiliation: Medical University Vienna

Key inclusion & exclusion criteria

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.



Age minimum: 18 Years
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

ADPKD

Intervention(s)

Drug: Sirolimus

Primary Outcome(s)

Slope in estimated glomerular filtration rate (eGFR; 4 variables MDRD equation) and proteinuria within six months of exposure to sirolimus. [Time Frame: Six months]

Secondary Outcome(s)

Aphthae [Time Frame: 6 months]

Acne [Time Frame: 6 months]

Dysfunctional wound healing [Time Frame: 6 months]

Pneumonitis [Time Frame: 6 months]

Leucopenia [Time Frame: 6 months]

Thrombopenia [Time Frame: 6 months]

Secondary ID(s)

003/2008/1.0

Source(s) of Monetary Support

Please refer to primary and secondary sponsors