Supplemental digital content

1

NONMEM CONTROL STREAMS

Population pharmacokinetic model (Run 10)

$PROBLEM XL184 PK,2-comp with first-order absorption (Base)

$INPUT C STUD PERI DOSE ID PT DAY=DROP ADAY HOUR TIME CONC DV

EVID MDV AMT ADDL II FLGPK=DROP IMPUTE=DROP

$INPUT PKDATE=DROP PKTIME=DROP FDHD=DROP FLAG=DROP FOOD AGE SEX

WT RACE HT=DROP

$INPUT SMOK BMI BSA BIL CREA=DROP CRCL ALT AST ALB

$DATA 20140427.xl184poppk.exclfed.v4.csv IGNORE=C

IGNORE(ID.EQ.292)

$SUBROUTINE ADVAN4 TRANS4

$PK

POP = 0

IF (STUD.EQ.4) POP = 1

IF (STUD.EQ.6) POP = 1

IF (STUD.EQ.7) POP = 1

COVCL = (1+ THETA(7)*(SEX-1))*(EXP(THETA(8)*(BMI-24.8)))*(EXP(THETA(9)*POP))

TVCL = THETA(1)

CL=TVCL*COVCL*DEXP(ETA(1))

TVV2 = THETA(2)

V2=TVV2*DEXP(ETA(2))

Q = THETA(3)

TV3 = THETA(4)

V3 = TV3

KA=THETA(5)*DEXP(ETA(3))

ALAG1=THETA(6)/2.4D01

S2=V2/1.D03

K10 = CL/V2

K12 = Q/V2

K21 = Q/V3

AA = K10+K12+K21

RRD = (AA+(AA*AA-4*K10*K21)**0.5)/2

RRE = (AA-(AA*AA-4*K10*K21)**0.5)/2

T12E = LOG(2)/RRE

T12D = LOG(2)/RRD

IF(AMT.GT.0) TDOS=TIME

TAD=TIME-TDOS

$ERROR

TY=F

IF(F.GT.0) THEN

IPRED=DLOG(F)

ELSE

IPRED=0

ENDIF

IRES=DV-IPRED

Y=IPRED+ EPS(1)

$THETA (0,70) ; CL

(0,349.305) ; V2

(0,20) ; Q

(0,200) ; v3

(0,62.7934) ; KA

(0,0.471162) ; ALAG1

(-0.5,-0.1,0.5) ; SEX ON CL

0.1 ; BMI on CL

0.1 ; HV on CL

$OMEGA BLOCK(2)

0.1

0.01 0.1

$OMEGA 0.1

$SIGMA 0.1

$ESTIMATION INTER MAX=9999 PRINT=5 METHOD=1 NSIG=3 SIGL=6

$COVARIANCE MATRIX=R UNCONDITIONAL

$TABLE STUD ID PT DOSE ADAY TIME TAD CONC TY IPRED IRES CWRES

NOPRINT ONEHEADER FILE=sdtab10

$TABLE STUD ID CL V2 Q V3 KA ALAG1 T12D T12E ETA(1) ETA(2) ETA(3)

NOPRINT FIRSTONLY ONEHEADER FILE=patab10

$TABLE STUD ID AGE WT BMI BSA BIL CRCL ALT AST ALB NOPRINT

FIRSTONLY ONEHEADER FILE=cotab10

$TABLE STUD ID DOSE SEX RACE SMOK NOPRINT FIRSTONLY ONEHEADER

FILE=catab10

$TABLE STUD ID PT DOSE ADAY TIME TAD EVID CONC TY IPRED IRES

CWRES CL V2 Q V3 KA ALAG1 T12D T12E ETA(1) ETA(2) ETA(3)

AGE WT BMI BSA BIL CRCL ALT AST ALB FOOD SEX RACE SMOK

NOPRINT ONEHEADER FILE=outab10.txt

Semi-mechanistic model (model 28)

$PROBLEM NONMEM PROBLEM DEFINITION

;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;;

; 1NONLINEAR MIXED EFFECTS MODEL PROGRAM (NONMEM) VERSION 7.1.0 ;

;------MODEL & DATA DESCRIPTION------;

;

;TIME: days

;CONC: ng/ml

;DV: mm (tumor volume)

;AMT: mg x 0.789

;PK model: XL184 PK,2-comp with first-order absorption (Final run10.mod)

;

;------END MODEL DESCRIPTION------

;

$INPUT C STUD ID PT TIME DV LNDV EVID MDV AMT ADDL II TV BTV LBTV

AGE SEX WT RACE HT SMOK BMI BSA TYPE TRT CLi VCi Qi VPi

KAi ALAGi

;

;$DATA Y:\184\301\POP-PK-T\FILES\PKPD-025-NS\PKPD-025-NS-D.csv IGNORE=@

$DATA 20140604.xl184pkpdQC.csv IGNORE=C WIDE

IGNORE=(TIME.GT.614)

;

$SUBROUTINE ADVAN6 TOL=3

;

$MODEL NCOMP=4 COMP=(DEP,DEFDOSE) ;CMT1 is depot site

COMP=(CENT) ;CMT2 is obs site

COMP=(PERIPH) ;CMT3 is PK peripheral compartment

COMP=(TVLM,DEFOBS) ;CMT4 is TUMOR BURDEN

;

$PK

;

;Pharmacokinetics (PK)

;READ run9.mod EBE's from PKPD database

;

CL=CLi

VC=VCi

Q=Qi

VP=VPi

KA=KAi

ALAG1=ALAGi ; RW

;

;Tumor burden dynamics (PD)

; baseline tumor volume (mm)

TV0 = THETA(1)*EXP(EXP(THETA(5))*ETA(1))

; Net disease progression rate (1/1000d)

NDPR = THETA(2)+EXP(THETA(6))*ETA(2)

; Normalized 1/day

NDPRN = NDPR/1000

; Kill slope (1/1000d)/(ng/mL)

kK = EXP(THETA(3))*EXP(EXP(THETA(7))*ETA(3))

; Normalized 1/day

kKN = kK/1000; 1/d/(ng/mL)

; Resistance rate (1/1000d)

kR = EXP(THETA(8))*EXP(EXP(THETA(9))*ETA(4))

kRN = kR/1000

A_0(4) = TV0;INITIAL (ESTIMATED) TUMOR VOLUME (MM)

;

$DES

;

;------PK Mass Balance Eqtns------

;

CP=1.D03*A(2)/VC;UG/ML to NG/ML

INH=kKN*CP

IF(TRT.EQ.0) THEN;PLACEBO DATA ONLY

;Pharmacokinetics (PK)

DADT(1)=0.D0

DADT(2)=0.D0

DADT(3)=0.D0

;Tumor Size

DADT(4)=NDPRN*A(4)

ELSE;ACTIVE TREATMENT

;Pharmacokinetics (PK)

DADT(1)=-A(1)*KA

DADT(2)=KA*A(1)-A(2)*(CL/VC)-A(2)*Q/VC+A(3)*Q/VP

DADT(3)=A(2)*Q/VC-A(3)*Q/VP

;Tumor Size

DADT(4)=NDPRN*A(4)-INH*A(4)*EXP(-kRN*T)

ENDIF

;

$ERROR

;

IPRED=F

W=EXP(THETA(4))

IRES=DV-IPRED

IWRES=IRES/(W*IPRED)

Y=IPRED*(1.D0+ERR(1)*W)

;

$THETA 8.08E+01 ; THETAINIT 1 [TV0]

8.14E-01 ; THETAINIT 2 [NDPR]

-4.58E+00 ; THETAINIT 3 log(kk)

-2.62E+00 ; THETAINIT 4 Log of sd of proportional sigma on tumor

-2.63E-01 ; THETAINIT Log of sd of log-normal eta on TV0

3.18E-01 ; THETAINIT Log of sd of additive eta on NDPR

-4.26E-01 ; THETAINIT Log of sd on log-normal eta on kK

2.89E+00 ; THETAINIT log(kR)

-1 FIX ; THETAINIT Log of sd on log-normal eta on kR

$OMEGA 1 FIX ; OMEGAINIT TV0

1 FIX ; OMEGAINIT NDPR

1 FIX ; OMEGAINIT kK

0 FIX ; OMEGAINIT kR

$SIGMA 1 FIX ; SIGMAINIT

;

$ESTIMATION INTER MAX=9990 PRINT=1 METHOD=COND POSTHOC NOABORT NSIG=3

SIGL=6 ;EVAL

$COVARIANCE

$TABLE STUD ID PT TRT AGE SEX WT BMI BSA BTV CL VC Q VP KA ALAG1

TV0 kK kR ETA1 ETA2 ETA3 ETA4 NOPRINT NOAPPEND ONEHEADER

FIRSTONLY FILE=pat28.txt

$TABLE STUD ID PT TRT TIME TYPE DV MDV AMT CP INH TIME IPRED PRED

IWRES CWRES NDPR NOPRINT NOAPPEND ONEHEADER

FILE=pred28.txt

Semi-empirical model - cabozantinib treatment arm (model 104)

$PROBLEM NONMEM PROBLEM DEFINITION

;------MODEL & DATA DESCRIPTION------;

;

;TIME: days

;CONC: ng/ml

;DV: mm (tumor volume)

;AMT: mg x 0.789

;PK model: XL184 PK,2-comp with first-order absorption (Final run10.mod)

;------END MODEL DESCRIPTION------

;

$INPUT C STUD ID PT TIME DV LNDV EVID MDV AMT ADDL II TV BTV LBTV

AGE SEX WT RACE HT SMOK BMI BSA ;INPUT

TYPE TRT CLi VCi Qi VPi KAi ALAGi ;INPUT

;

$DATA 20140604.xl184pkpdQC.csv IGNORE=@ IGNORE(TIME.GT.614)

IGNORE(TRT.EQ.0)

$SUBROUTINE ADVAN6 TOL=3

;

$MODEL NCOMP=6 COMP=(DEP,DEFDOSE) ;CMT1 is depot site

COMP=(CENT) ;CMT2 is obs site

COMP=(PERIPH) ;CMT3 is PK peripheral compartment

COMP=(TVLM,DEFOBS) ;CMT4 is TUMOR BURDEN

COMP=(AUC) ;CMT5 is AUC

COMP=(AUCPDC) ;CMT6 is PD AUC

;

$PK

CL=CLi

VC=VCi

Q=Qi

VP=VPi

KA=KAi

ALAG1=ALAGi

;

;Tumor burden dynamics (PD)

; baseline tumor volume (mm)

TV0 = THETA(1)*EXP(EXP(THETA(5))*ETA(1))

; Net disease progression rate (1/1000d)

NDPR = THETA(2)+EXP(THETA(6))*ETA(2)

; Normalized 1/day

NDPRN = NDPR/1000

; Kill slope

INTER1 = THETA(3) + EXP(THETA(8))*ETA(3)

INTER1N = INTER1/1000

SLP1 = THETA(11) + EXP(THETA(14))*ETA(6)

SLP1N = SLP1/1000000

; Stabilization Rate

INTER2 = THETA(7) + EXP(THETA(9))*ETA(4)

INTER2N = INTER2/1000

SLP2 = THETA(12) + EXP(THETA(13))*ETA(5)

SLP2N = SLP2/1000000

; Split time

TSPL=THETA(10)

A_0(4) = TV0;INITIAL (ESTIMATED) TUMOR VOLUME (MM)

;

$DES

;------PK Mass Balance Eqtns------

CONC=1.0D03*A(2)/VC

IF(T.LE.TSPL) INHN=INTER1N + SLP1N * CONC

IF(T.GT.TSPL) INHN=INTER2N + SLP2N * CONC

IF(TRT.EQ.0) THEN;PLACEBO DATA ONLY

;Pharmacokinetics (PK)

DADT(1)=0.D0

DADT(2)=0.D0

DADT(3)=0.D0

;Tumor Size

DADT(4)=NDPRN*A(4)

DADT(6)=(A(4)-TV0)/TV0

ENDIF

IF(TRT.EQ.1)THEN;ACTIVE TREATMENT

;Pharmacokinetics (PK)

DADT(1)=-A(1)*KA

DADT(2)=KA*A(1)-A(2)*(CL/VC)-A(2)*Q/VC+A(3)*Q/VP

DADT(3)=A(2)*Q/VC-A(3)*Q/VP

DADT(5)=CONC

ENDIF

;Tumor Size

IF(TRT.EQ.1) THEN

DADT(4)=(INHN)*A(4)

DADT(6)=(A(4)-TV0)/TV0

ENDIF

$ERROR

;

;calculate target concentration

IF(TRT.EQ.1) THEN

CP=1D03*A(2)/VC

AUC=A(5)

ENDIF

AUCPD=A(6)

IPRED=F

W=exp(THETA(4))

IRES=DV-IPRED

IWRES=IRES/(W*IPRED)

Y=IPRED*(1.D0+ERR(1)*W)

;

$THETA 9.35E+01 ; THETAINIT 1 [TV0]

0 FIX ; THETAINIT 2 [NDPR]

-1.69E+00 ; THETAINIT 3 [INTER1]

-2.41E+00 ; THETAINIT 4 Log of sd of proportional sigma on tumor

-2.96E-01 ; THETAINIT 5 Log of sd of log-normal eta on TV0

-200 FIX ; THETAINIT 6 Log of sd of additive eta on NDPR

5.92E-01 ; THETAINIT 7 [INTER2]

5.75E-02 ; THETAINIT 8 Log of sd of additive eta on INTER1

-5.78E-01 ; THETAINIT 9 Log of sd of additive eta on INTER2

110 FIX ; THETAINIT 10 split time

0 FIX ; THETAINIT 11 [SLP1]

9.06E-01 ; THETAINIT 12 [SLP2]

-5.53E-01 ; THETAINIT 13 [SLP2] Log of sd of log-normal eta on SLP2

0 FIX ; THETAINIT 14 [SLP1] Log of sd of log-normal eta on SLP1

$OMEGA 1 FIX ; OMEGAINIT TV0

1 FIX ; OMEGAINIT NDPR

1 FIX ; OMEGAINIT INTER1

1 FIX ; OMEGAINIT INTER2

1 FIX ; OMEGAINIT S2

0 FIX ; OMEGAINIT S1

$SIGMA 1 FIX ; SIGMAINIT

;

$ESTIMATION INTER MAX=9990 PRINT=1 METHOD=COND POSTHOC NOABORT NSIG=3

SIGL=6 ;EVAL

;COVA

$COVARIANCE

$TABLE STUD ID TRT TIME AMT CP LNDV MDV IPRED IWRES,CWRES NDPR

SLP1 SLP2 TSPL AUC AUCPD TV BTV NOPRINT ONEHEADER

FILE=104-NSA.TXT ;FILEA

$TABLE STUD ID PT TIME NDPR NDPRN INTER1 INTER1N INTER2 INTER2N

SLP1 SLP1N SLP2 SLP2N TSPL NOPRINT ONEHEADER

FILE=104-NSC.TXT ;FILEC

$TABLE ID PT CL VC Q VP KA ALAG1 ETA1 ETA2 ETA3 ETA4 ETA5 ETA6

NOPRINT ONEHEADER FILE=104-NSE.TXT ;FILEE

$TABLE STUD ID PT AGE SEX WT BMI BSA TYPE TRT TV0 BTV NOPRINT

ONEHEADER FILE=104-NSF.TXT ;FILEF

Semi-empirical model - placebo arm (model 101)

$PROBLEM NONMEM PROBLEM DEFINITION

;------MODEL & DATA DESCRIPTION------;

;

;TIME: days

;CONC: ng/ml

;DV: mm (tumor volume)

;AMT: mg x 0.789

;PK model: XL184 PK,2-comp with first-order absorption (Final run10.mod)

;------END MODEL DESCRIPTION------

;

$INPUT C STUD ID PT TIME DV LNDV EVID MDV AMT ADDL II TV BTV LBTV

AGE SEX WT RACE HT SMOK BMI BSA ;INPUT

TYPE TRT CLi VCi Qi VPi KAi ALAGi ;INPUT

;

$DATA 20140604.xl184pkpdQC.csv IGNORE=@ IGNORE(TIME.GT.614)

IGNORE(TRT.EQ.1)

$SUBROUTINE ADVAN6 TOL=3

;

$MODEL NCOMP=6 COMP=(DEP,DEFDOSE) ;CMT1 is depot site

COMP=(CENT) ;CMT2 is obs site

COMP=(PERIPH) ;CMT3 is PK peripheral compartment

COMP=(TVLM,DEFOBS) ;CMT4 is TUMOR BURDEN

COMP=(AUC) ;CMT5 is AUC

COMP=(AUCPDC) ;CMT6 is PD AUC

;

$PK

CL=CLi

VC=VCi

Q=Qi

VP=VPi

KA=KAi

ALAG1=ALAGi

;

;Tumor burden dynamics (PD)

; baseline tumor volume (mm)

TV0 = THETA(1)*EXP(EXP(THETA(5))*ETA(1))

; Net disease progression rate (1/1000d)

NDPR = THETA(2)+EXP(THETA(6))*ETA(2)

; Normalized 1/day

NDPRN = NDPR/1000

; Kill slope

INTER1 = THETA(3) + EXP(THETA(8))*ETA(3)

INTER1N = INTER1/1000

SLP1 = THETA(11) + EXP(THETA(14))*ETA(6)

SLP1N = SLP1/1000000

; Stabilization Rate

INTER2 = THETA(7) + EXP(THETA(9))*ETA(4)

INTER2N = INTER2/1000

SLP2 = THETA(12) + EXP(THETA(13))*ETA(5)

SLP2N = SLP2/1000000

; Split time

TSPL=THETA(10)

A_0(4) = TV0;INITIAL (ESTIMATED) TUMOR VOLUME (MM)

;

$DES

;------PK Mass Balance Eqtns------

CONC=1.0D03*A(2)/VC

IF(T.LE.TSPL) INHN=INTER1N + SLP1N * CONC

IF(T.GT.TSPL) INHN=INTER2N + SLP2N * CONC

IF(TRT.EQ.0) THEN;PLACEBO DATA ONLY

;Pharmacokinetics (PK)

DADT(1)=0.D0

DADT(2)=0.D0

DADT(3)=0.D0

;Tumor Size

DADT(4)=NDPRN*A(4)

DADT(6)=(A(4)-TV0)/TV0

ENDIF

IF(TRT.EQ.1)THEN;ACTIVE TREATMENT

;Pharmacokinetics (PK)

DADT(1)=-A(1)*KA

DADT(2)=KA*A(1)-A(2)*(CL/VC)-A(2)*Q/VC+A(3)*Q/VP

DADT(3)=A(2)*Q/VC-A(3)*Q/VP

DADT(5)=CONC

ENDIF

;Tumor Size

IF(TRT.EQ.1) THEN

DADT(4)=(INHN)*A(4)

DADT(6)=(A(4)-TV0)/TV0

ENDIF

$ERROR

;

;calculate target concentration

IF(TRT.EQ.1) THEN

CP=1D03*A(2)/VC

AUC=A(5)

ELSE

AUC=0

ENDIF

AUCPD=A(6)

IPRED=F

W=exp(THETA(4))

IRES=DV-IPRED

IWRES=IRES/(W*IPRED)

Y=IPRED*(1.D0+ERR(1)*W)

;

$THETA 9.35E+01 ; THETAINIT 1 [TV0]

0.01 ; THETAINIT 2 [NDPR]

0 FIX ; THETAINIT 3 [INTER1]

-2.41E+00 ; THETAINIT 4 Log of sd of proportional sigma on tumor

-2.96E-01 ; THETAINIT 5 Log of sd of log-normal eta on TV0

0.5 ; THETAINIT 6 Log of sd of additive eta on NDPR

0 FIX ; THETAINIT 7 [INTER2]

0 FIX ; THETAINIT 8 Log of sd of additive eta on INTER1

0 FIX ; THETAINIT 9 Log of sd of additive eta on INTER2

110 FIX ; THETAINIT 10 split time

0 FIX ; THETAINIT 11 [SLP1]

0 FIX ; THETAINIT 12 [SLP2]

0 FIX ; THETAINIT 13 [SLP2] Log of sd of log-normal eta on SLP2

0 FIX ; THETAINIT 14 [SLP1] Log of sd of log-normal eta on SLP1

$OMEGA 1 FIX ; OMEGAINIT TV0

1 FIX ; OMEGAINIT NDPR

1 FIX ; OMEGAINIT INTER1

1 FIX ; OMEGAINIT INTER2

1 FIX ; OMEGAINIT S2

1 FIX ; OMEGAINIT S1

$SIGMA 1 FIX ; SIGMAINIT

;

$ESTIMATION INTER MAX=9990 PRINT=1 METHOD=COND POSTHOC NOABORT NSIG=3

SIGL=6 ;EVAL

;COVA

$COVARIANCE

$TABLE STUD ID TRT TIME AMT CP LNDV MDV IPRED IWRES,CWRES NDPR

SLP1 SLP2 TSPL AUC AUCPD TV BTV NOPRINT ONEHEADER

FILE=101-NSA.TXT ;FILEA

$TABLE STUD ID PT TIME NDPR NDPRN INTER1 INTER1N INTER2 INTER2N

SLP1 SLP1N SLP2 SLP2N TSPL NOPRINT ONEHEADER

FILE=101-NSC.TXT ;FILEC

$TABLE ID PT CL VC Q VP KA ALAG1 ETA1 ETA2 ETA3 ETA4 ETA5 ETA6

NOPRINT ONEHEADER FILE=101-NSE.TXT ;FILEE

$TABLE STUD ID PT AGE SEX WT BMI BSA TYPE TRT TV0 BTV NOPRINT

ONEHEADER FILE=101-NSF.TXT ;FILEF

1