Clinical proof-of-concept trial to assess the therapeutic effect of sirolimus in patients with autosomal dominant polycystic kidney disease: SUISSE ADPKD study
<p>Abstract</p> <p>Background</p> <p>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 obt...
| Main Authors: | , , , , , , |
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| Format: | Article |
| Language: | English |
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BMC
2007-09-01
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| Series: | BMC Nephrology |
| Online Access: | http://www.biomedcentral.com/1471-2369/8/13 |
| _version_ | 1818791303699234816 |
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| author | Wüthrich Rudolf P Struker Marian Poster Diane Kistler Andreas D Serra Andreas L Weishaupt Dominik Tschirch Frank |
| author_facet | Wüthrich Rudolf P Struker Marian Poster Diane Kistler Andreas D Serra Andreas L Weishaupt Dominik Tschirch Frank |
| author_sort | Wüthrich Rudolf P |
| collection | DOAJ |
| description | <p>Abstract</p> <p>Background</p> <p>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.</p> <p>Method/design</p> <p>This single center, randomised controlled, open label trial assesses the therapeutic effect, safety and tolerability of the mTOR inhibitor sirolimus (Rapamune<sup>®</sup>) 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.</p> <p>Discussion</p> <p>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.</p> <p>Trial registration</p> <p>NCT00346918</p> |
| first_indexed | 2024-12-18T15:09:13Z |
| format | Article |
| id | doaj.art-9d7b38e4174b444596083f179c405514 |
| institution | Directory Open Access Journal |
| issn | 1471-2369 |
| language | English |
| last_indexed | 2024-12-18T15:09:13Z |
| publishDate | 2007-09-01 |
| publisher | BMC |
| record_format | Article |
| series | BMC Nephrology |
| spelling | doaj.art-9d7b38e4174b444596083f179c4055142022-12-21T21:03:43ZengBMCBMC Nephrology1471-23692007-09-01811310.1186/1471-2369-8-13Clinical proof-of-concept trial to assess the therapeutic effect of sirolimus in patients with autosomal dominant polycystic kidney disease: SUISSE ADPKD studyWüthrich Rudolf PStruker MarianPoster DianeKistler Andreas DSerra Andreas LWeishaupt DominikTschirch Frank<p>Abstract</p> <p>Background</p> <p>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.</p> <p>Method/design</p> <p>This single center, randomised controlled, open label trial assesses the therapeutic effect, safety and tolerability of the mTOR inhibitor sirolimus (Rapamune<sup>®</sup>) 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.</p> <p>Discussion</p> <p>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.</p> <p>Trial registration</p> <p>NCT00346918</p>http://www.biomedcentral.com/1471-2369/8/13 |
| spellingShingle | Wüthrich Rudolf P Struker Marian Poster Diane Kistler Andreas D Serra Andreas L Weishaupt Dominik Tschirch Frank Clinical proof-of-concept trial to assess the therapeutic effect of sirolimus in patients with autosomal dominant polycystic kidney disease: SUISSE ADPKD study BMC Nephrology |
| title | Clinical proof-of-concept trial to assess the therapeutic effect of sirolimus in patients with autosomal dominant polycystic kidney disease: SUISSE ADPKD study |
| title_full | Clinical proof-of-concept trial to assess the therapeutic effect of sirolimus in patients with autosomal dominant polycystic kidney disease: SUISSE ADPKD study |
| title_fullStr | Clinical proof-of-concept trial to assess the therapeutic effect of sirolimus in patients with autosomal dominant polycystic kidney disease: SUISSE ADPKD study |
| title_full_unstemmed | Clinical proof-of-concept trial to assess the therapeutic effect of sirolimus in patients with autosomal dominant polycystic kidney disease: SUISSE ADPKD study |
| title_short | Clinical proof-of-concept trial to assess the therapeutic effect of sirolimus in patients with autosomal dominant polycystic kidney disease: SUISSE ADPKD study |
| title_sort | clinical proof of concept trial to assess the therapeutic effect of sirolimus in patients with autosomal dominant polycystic kidney disease suisse adpkd study |
| url | http://www.biomedcentral.com/1471-2369/8/13 |
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