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. Author manuscript; available in PMC: 2014 Jul 1.
Published in final edited form as: Pediatr Res. 2013 Oct 31;75(0):148–157. doi: 10.1038/pr.2013.191

Table 1.

The Future: Therapies for Childhood Pkd

Class
(compound)
ADPKD ARPKD CLINICAL TRIALS or pre-clinical
reference
(www.clinicaltrials.gov)
VPV2R Antagonists (Tolvaptin) ++/− +/−− Phase II–III
NCT01022424 (III) in progress
NCT01280721 (III) in progress
NCT01214421 (III) recruiting
NCT01451827 (II) recruiting
Src Inhibitors (Bosutinib) +++ +++ Phase II
NCT01233869 (II) recruiting
mTOR Inhibitors +/− − +/− − Phase I–II
NCT00414440-in progress
NCT00286156
NCT01009957 (II,III)-recruiting
Multi-Kinase Inhibitors (KD-019) +++ ++/? Phase I–II
NCT01559363 (I–II) recruiting
Somatostatins Long Acting analogues (Octreonide & Laneonide) ++/? ++/? Phase III
NCT01377246 (III) recruiting
NCT01354405 (III) in progress
(Lisinopril and Telmisartan) HALT PKD ++/? ++/? NCT00283686 (III) in progress
(Diterpene) (Triptolide Woldifii) +/?? +/?? NCT00801268 (II) (China only)
EGFR-Axis Inhibitors +++ +++ (40)
(42)
Angiogenesis Inhibitors/MMP-inhibitors ++/? ++/? (74)
SMAC Mimetics/ HDAC-i/TNF-α ++/? ++/? (75)
(76)
20-HETE ++/? ++/? (77)
(78)
ROS-i ++/? ++/? (79)
Manipulation of Genetic Modifiers ++/? ++/? (80)