PMID- 17868472
OWN - NLM
STAT- MEDLINE
DCOM- 20071203
LR  - 20181113
IS  - 1471-2369 (Electronic)
IS  - 1471-2369 (Linking)
VI  - 8
DP  - 2007 Sep 15
TI  - Clinical proof-of-concept trial to assess the therapeutic effect of sirolimus in 
      patients with autosomal dominant polycystic kidney disease: SUISSE ADPKD study.
PG  - 13
AB  - 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.
FAU - Serra, Andreas L
AU  - Serra AL
AD  - Clinic for Nephrology, University Hospital, CH-8091 Zurich, Switzerland. 
      andreas.serra@usz.ch
FAU - Kistler, Andreas D
AU  - Kistler AD
FAU - Poster, Diane
AU  - Poster D
FAU - Struker, Marian
AU  - Struker M
FAU - Wuthrich, Rudolf P
AU  - Wuthrich RP
FAU - Weishaupt, Dominik
AU  - Weishaupt D
FAU - Tschirch, Frank
AU  - Tschirch F
LA  - eng
SI  - ClinicalTrials.gov/NCT00346918
PT  - Journal Article
PT  - Randomized Controlled Trial
PT  - Research Support, Non-U.S. Gov't
DEP - 20070915
PL  - England
TA  - BMC Nephrol
JT  - BMC nephrology
JID - 100967793
RN  - 0 (Immunosuppressive Agents)
RN  - W36ZG6FT64 (Sirolimus)
SB  - IM
MH  - Adolescent
MH  - Adult
MH  - Feasibility Studies
MH  - Female
MH  - Humans
MH  - Immunosuppressive Agents/administration & dosage
MH  - Male
MH  - Pilot Projects
MH  - Polycystic Kidney, Autosomal Dominant/*diagnosis/*drug therapy
MH  - Sirolimus/*administration & dosage
MH  - Treatment Outcome
PMC - PMC2048941
EDAT- 2007/09/18 09:00
MHDA- 2007/12/06 09:00
PMCR- 2007/09/15
CRDT- 2007/09/18 09:00
PHST- 2007/06/30 00:00 [received]
PHST- 2007/09/15 00:00 [accepted]
PHST- 2007/09/18 09:00 [pubmed]
PHST- 2007/12/06 09:00 [medline]
PHST- 2007/09/18 09:00 [entrez]
PHST- 2007/09/15 00:00 [pmc-release]
AID - 1471-2369-8-13 [pii]
AID - 10.1186/1471-2369-8-13 [doi]
PST - epublish
SO  - BMC Nephrol. 2007 Sep 15;8:13. doi: 10.1186/1471-2369-8-13.