Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Randomized Controlled Trial
. 2007 Sep 15:8:13.
doi: 10.1186/1471-2369-8-13.

Clinical proof-of-concept trial to assess the therapeutic effect of sirolimus in patients with autosomal dominant polycystic kidney disease: SUISSE ADPKD study

Andreas L Serra  1 , Andreas D Kistler, Diane Poster, Marian Struker, Rudolf P Wüthrich, Dominik Weishaupt, Frank Tschirch
Affiliations
Randomized Controlled Trial

Clinical proof-of-concept trial to assess the therapeutic effect of sirolimus in patients with autosomal dominant polycystic kidney disease: SUISSE ADPKD study

Andreas L Serra et al. BMC Nephrol. .

Abstract

Background: Currently there is no effective treatment available to retard cyst growth and to prevent the progression to end-stage renal failure in patients with autosomal dominant polycystic kidney disease (ADPKD). Evidence has recently been obtained from animal experiments that activation of the mammalian target of rapamycin (mTOR) signaling pathway plays a crucial role in cyst growth and renal volume expansion, and that the inhibition of mTOR with rapamycin (sirolimus) markedly slows cyst development and renal functional deterioration. Based on these promising results in animals we have designed and initiated the first randomized controlled trial (RCT) to examine the effectiveness, safety and tolerability of sirolimus to retard disease progression in ADPKD.

Method/design: This single center, randomised controlled, open label trial assesses the therapeutic effect, safety and tolerability of the mTOR inhibitor sirolimus (Rapamune) in patients with autosomal dominant polycystic kidney disease and preserved renal function. The primary outcome will be the inhibition of kidney volume growth measured by magnetic resonance imaging (MRI) volumetry. Secondary outcome parameters will be preservation of renal function, safety and tolerability of sirolimus.

Discussion: The results from this proof-of-concept RCT will for the first time show whether treatment with sirolimus effectively retards cyst growth in patients with ADPKD.

Trial registration: ClinicalTrials.gov NCT00346918.

PubMed Disclaimer

Figures

Figure 1
Figure 1
Flow chart of SUISSE ADPKD study.
See this image and copyright information in PMC

References

    1. Dalgaard OZ. Bilateral polycystic disease of the kidneys; a follow-up of two hundred and eighty-four patients and their families. Acta Med Scand Suppl. 1957;328:1–255. - PubMed
    1. Hateboer N, v Dijk MA, Bogdanova N, Coto E, Saggar-Malik AK, San Millan JL, Torra R, Breuning M, Ravine D. Comparison of phenotypes of polycystic kidney disease types 1 and 2. European PKD1-PKD2 Study Group. Lancet. 1999;353:103–107. doi: 10.1016/S0140-6736(98)03495-3. - DOI - PubMed
    1. Wilson PD. Polycystic kidney disease. N Engl J Med. 2004;350:151–164. doi: 10.1056/NEJMra022161. - DOI - PubMed
    1. Harris PC, Rossetti S. Molecular genetics of autosomal recessive polycystic kidney disease. Mol Genet Metab. 2004;81:75–85. doi: 10.1016/j.ymgme.2003.10.010. - DOI - PubMed
    1. Torra R, Badenas C, Darnell A, Nicolau C, Volpini V, Revert L, Estivill X. Linkage, clinical features, and prognosis of autosomal dominant polycystic kidney disease types 1 and 2. J Am Soc Nephrol. 1996;7:2142–2151. - PubMed

Publication types

Associated data