980 1030
Table 2 – Ongoing trials including exclusively or in part patients with RKCs for advanced or metastatic disease
Trial
Design and line
Included subtypes
Estimated
enrollment (
n
)
Treatment
Primary
end points
Secondary
end points
PAPMET
NCT02761057
Randomized
Phase 2 One prior
systemic therapy
allowed except
study drugs
pRCC types 1 and 2
180
Sunitinib versus
cabozantinib versus
crizotinib versus
savolitinib
PFS
AE rate
OS
ORR
MET mutation rate
SAVOIR
NCT03091192
Randomized
Phase 3
any line allowed
except study drugs
pRCC any type, MET-
driven only after
screening
180
Sunitinib versus
savolitinib
PFS
PRO
AE rate
OS
ORR
CALYPSO
NCT02819596
Randomized
Phases 1 and 2
pRCC cohort: VEGF
treatment-naı¨ve or
treatment
refractory
pRCC and clear-cell RCC
195
Savolitinib versus
durvalumab versus
savolitinib + durvalumab
versus tremelimumab +
durvalumab
Phase 1
DLT
ORR
Phase 1
PK
Phase 2
PFS
OS
DOR
Biomarkers
SUNNIFORECAST
NCT03075423
Randomized
Phase 2
Treatment naı¨ve
Non-ccRCC with at least
50% non-cc component
306
Nivolumab + Ipilimumab
versus sunitinib
OS rate
at 12 mo
OS rate 6/18 mo
OS
PFS
ORR
AE rate
QoL
NCT02724878 Single arm
Phase 2
Prior VEGFR-TKI or
cytokines allowed
pRCC any type
chRCC
Translocation
CDC
RMC
uRCC
Any histology with 20%
sarcomatoid
40
Atezolizumab +
bevacizumab
ORR
DOR
AE rate
IORR
PFS
OS
QoL
NCT02915783 Single arm
Phase 2
Treatment naı¨ve
pRCC any type chRCC
CDC
RMC
uRCC
31
Lenvatinib + everolimus
ORR
PFS
OS
NCT01130519 Single arm
Phase 2
No more than two
prior systemic
therapies
excluding
bevacizumab
Cohort A: HLRCC
Cohort B: sporadic pRCC
85
Bevacizumab + erlotinib ORR
PFS
OS
DOR
Biomarkers
Somatic FH mutations
Effect on leimyomas
Keynote-427
NCT02853344
Single arm
Phase 2
Treatment naı¨ve
Cohort A: ccRCC
Cohort B: non-ccRCC with
or without sarcomatoid
255
Pembrolizumab
ORR
DOR
PFS
OS
AE rate
Checkmate 920
NCT02982954
Nonrandomized
Phase 3b/4
Treatment naı¨ve
Cohorts:
ccRCC KPS
>
70%
Non-ccRCC KPS
>
70%
Any RCC KPS 50–60%
Any RCC with nonactive
brain mets
200
Nivolumab + ipilimumab High-grade
IMAE
Time to IMAE
PFS
ORR
TTR
DOR
NCT01767636 Single arm
Phase 2
One prior systemic
therapy allowed
pRCC any type
chRCC
CDC
RMC
Translocation
Sarcomatoid
39
Pazopanib
OS rate at
12 mo
AE rate
PFS
ORR
NCT01672775 Single arm
Phase 1
Treatment naı¨ve
for non-ccRCC
ccRCC and non-ccRCC
ENPP3-positive at
prescreening
Expansion cohort with
ENPP3 + pRCC
34
AGS-16C3F at various
dose levels
AE rate
PK
ORR
Antibodies against
AGS-16C3F
FH = fumarate hydratase; HLRCC = hereditary leiomyomatosis and renal cell cancer; KPS = Karnofsky performance status; pRCC = papillary renal cell carcinoma;
chRCC = chromophobe renal cell carcinoma; CDC = collecting duct carcinoma; RMC = renal medullary carcinoma; ccRCC = clear-cell renal cell carcinoma;
uRCC = unclassified renal cell carcinoma; PFS = progression free survival; OS = overall survival; ORR = objective response rate; IORR = immune-related objective
response rate; AE = adverse event; IMAE = immune-mediated adverse events; DLT = dose-limiting toxicity; PK = pharmacokinetics; DOR = duration of response;
TTR = time to response; QoL = quality of life; ENPP3 = ectonucleotide pyrophosphatase/phosphodiesterase family member 3; PRO = patient-related outcomes;
RKC = rare kidney cancer; RCC = renal cell carcinoma; VEGF = vascular endothelial growth factor.
E U R O P E A N U R O L O G Y 7 2 ( 2 0 1 7 ) 9 7 4 – 9 8 3
980