The Vienna RAP Pilot Study

NCT01632605 | Completed DMC

• 1 other identifier: 003/2008/1.0
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

ADPKD

Keywords

ADPKD; Autosomal dominant polycystic kidney disease; Sirolimus; Rapamune; Polycystic liver disease; Rapamycin; Mammalian target of rapamycin; m-TOR; m-TOR inhibition

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (6)

• Leucopenia

  6 months

• Thrombopenia

  6 months

• Aphthae

  6 months

• Dysfunctional wound healing

  6 months

• Pneumonitis

  6 months

Study Arms (1)

Sirolimus

EXPERIMENTAL

Daily single oral dose of 1-3mg sirolimus with an initial loading dose of 6mg.

Drug: Sirolimus

Interventions

Sirolimus DRUG

Coated tablets, 1mg and 2mg available. Daily oral single dose with trough levels of 4-8ng/mL. Total intake for 6 months.

Also known as: RAPAMUNE

Sirolimus

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Medical University of Vienna: lead

Study Sites (1)

Division of Nephrology and Dialysis, Department of Medicine III, Medical University Vienna

Vienna, Vienna, 1090, Austria

Location

MeSH Terms

Conditions

Polycystic Kidney, Autosomal Dominant; Polycystic liver disease; Hereditary Sensory and Autonomic Neuropathies

Interventions

Sirolimus

Condition Hierarchy (Ancestors)

Polycystic Kidney Diseases; Kidney Diseases, Cystic; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Ciliopathies; Genetic Diseases, Inborn; Nervous System Malformations; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals

Study Officials

• Gere Sunder-Plassmann, MD

  Medical University Vienna

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor of Medicine

Study Record Dates

• First Submitted: May 13, 2012
• First Posted: July 3, 2012
• Study Start: November 1, 2009
• Primary Completion: April 1, 2012
• Study Completion: April 1, 2012
• Last Updated: July 3, 2012

Record last verified: 2012-06

Locations

AU (1)