Clinical Trials

Thanks to research funded by the PKD Foundation, clinical trials are now exploring several new therapies for PKD. Patients wishing to participate or learn more will find specific information about the therapies below. To participate in a clinical trial, you will need to have a formal diagnosis of PKD. To learn more, click here.


Learn about potential treatments here. Learn about PKD and heart disease here.

 		 
 
       

 

Rapamycin (Rapamune ®, Sirolimus) has been prescribed post-transplant to stop rejection of the new organ.  Everolimus is a biochemically related compound.  Both drugs have been found to inhibit cell growth in cysts by binding to a protein called mTor (mammalian target of rapamycin) in renal cells.  As a result, cyst growth is reduced.

 

 

mTOR Inhibitor Clinical Trials


 

Efficacy, Safety and Tolerability of Everolimus in Preventing End-stage Renal Disease in Patients with ADPKD

 

Intervention:

 Drug - Everolimus

Sponsor:

 Novartis
Status: Completed
Age Group: 18-65

Enrollment goal:

 300

Description:

 To evaluate the anti-proliferative drug Everolimus as a means to slow disease progression in PKD by measuring total kidney and cyst volumes and changes in renal function.
Contact: Novartis, Freiburg, Germany 41-61-324-1111
Website: NIH Study Details

 

Rapamycin as a Treatment for ADPKD: The Role of Biomarkers in Predicting a Response to Therapy

 

Intervention:

 Drug - Rapamycin

Sponsor:

 Yale Center for Clinical Investigation
Status: Completed
Age Group: 18-70

Enrollment goal:

 45

Description:

 To determine the safety & effectiveness of treatment with rapamycin for patients with ADPKD, patients will be observed for change in kidney (& cyst) size by following the patient's through volumetric magnetic resonance imaging (MRI). To evaluat e for change in biomarkers with treatment, blood and urine samples will be collected during the study.
Contact: Neera K. Dahl, MD, PhD at 203-785-4184 or neera.dahl@yale.edu
Website: NIH Study Details

 

Sirolimus (Rapamune) for ADPKD

 

Intervention:

 Drug - Rapamycin

Sponsor:

 The University of Zurich

Study Phase:

 III
Status: Active, enrollment complete
Age Group: 18-40

Enrollment goal:

 100

Description:

 To investigate whether a low dose of sirolimus retards cyst growth and slows renal functional deterioration in patients with ADPKD.
Contact: Andreas Serra, MD at 41 44 255 33 84 or andreas.serra@usz.ch
Website: NIH Study Details

 

Pilot Study of Rapamycin as Treatment for ADPKD

 

Intervention:

 Drug - Rapamycin

Sponsor:

 The Cleveland Clinic

Study Phase:

 I & II
Status: Active, not recruiting
Age Group: 18-75

Enrollment goal:

 30

Description:

 To compare the effects of an anti-proliferative, anti-angiogenesis and tumor-progression-blocking agent on disease progression in ADPKD.  Due to the intensive and frequent testing schedule, enrollment is limited to local patients only.
Contact: Susan Wirth at 216-444-0124 or wirths@ccf.org
Website: NIH Study Details
 
   
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