Cystic Fibrosis
Watchlist
Retrieved
2022-04-26
Source
Trials
—
Genes
CFTR,
TGFB1,
SCNN1B,
DCTN4,
SCNN1A,
SCNN1G,
CLCA4,
TNFRSF1A,
FCGR2A,
STX1A,
ENG,
PRSS1,
CLCN2,
MUC2,
MUC1,
MBL2,
SERPINA1,
SLC9A3,
HFE,
MUC4,
SMOX,
MPO,
NOS2,
COPD,
CXCL8,
MET,
IL10,
IL6,
IL1B,
FEV
CFTR,
TGFB1,
SCNN1B,
DCTN4,
SCNN1A,
SCNN1G,
CLCA4,
TNFRSF1A,
FCGR2A,
STX1A,
ENG,
PRSS1,
CLCN2,
MUC2,
MUC1,
MBL2,
SERPINA1,
SLC9A3,
HFE,
MUC4,
SMOX,
MPO,
NOS2,
COPD,
CXCL8,
MET,
IL10,
IL6,
IL1B,
FEV,
ANO1,
HLA-DRB9,
SLC26A9,
ELANE,
DNASE1,
ABCB1,
MUC5AC,
ABCB6,
SLC9A6,
MUC5B,
TNF,
TLE5,
MMP9,
TLR4,
ADRB2,
IL1A,
PLF,
SPINK1,
GSTM1,
IL17A,
BPIFA1,
ABCC1,
MFAP1,
ALB,
APRT,
TLR2,
S100A8,
NOS1,
CLCA1,
GLB1,
IGF1,
ACTB,
AAVS1,
SMPD1,
DEFB1,
BPI,
ABCG2,
SLC6A14,
LUM,
CELA3B,
PTGS2,
PARP9,
GCG,
SPX,
SUMF2,
HPGDS,
GSTP1,
GSTT1,
SFTPD,
MIR155,
CEL,
SLC9A3R1,
PPARG,
PDE5A,
SLC27A5,
IFNG,
SCT,
SULT1E1,
HSPA4,
TLR5,
S100A9,
XBP1,
MBL3P,
TBC1D9,
NLRP3,
HMGB1,
VEGFA,
BEST1,
CXCR1,
GH1,
A2M,
WNT2,
SFTPA1,
BRCA1,
COL1A2,
BCL2,
CAT,
CAMP,
CRP,
ARSB,
PGD,
EHF,
WNK1,
ATP12A,
SLC26A4,
GCLC,
NFE2L2,
PIK3CA,
PIK3CB,
IFRD1,
PIK3CG,
EBP,
PRTN3,
ABCC11,
GSTK1,
MYD88,
TRPV4,
CASP3,
IFNB1,
IL1RN,
CD14,
ICAM1,
TREM1,
CXCR2,
IL13,
ISYNA1,
KNG1,
CPOX,
KRT8,
TBX22,
LCN2,
LIPE,
MDM4,
HIF1A,
MSH3,
COX2,
HDAC6,
PIK3CD,
DECR1,
ADIPOQ,
SLCO6A1,
BECN1,
CELA1,
TRBV20OR9-2,
DMD,
TGM2,
ESR1,
ABCC8,
VIP,
EZR,
ZFP36,
TRBC1,
ABCA4,
MIF,
TRB,
CTNNB1,
MMP2,
MMP7,
TCF7L2,
CD40LG,
TLR9,
SMAD3,
TRBV7-9,
PIAS1,
TRBV16,
TSPO,
TAP1,
STK39,
ADAM17,
XBP1P1,
SLC39A1,
CD59,
LPO,
MIR17,
IL9,
VCP,
MIR146A,
FZD4,
TXN,
IL18,
IL23A,
TWIST1,
KCNH2,
TRPC6,
KCNN4,
LOC111674463,
MTCO2P12,
CASP1,
KRT18,
KRT19,
HAVCR1,
ERICD,
LEP,
CAST,
VIM2P,
AGTR2,
AFP,
AGER,
MASP2,
TCFL5,
AKT1,
TNFSF13B,
PON1,
SFTPB,
PREP,
AHSA1,
MAPK1,
AREG,
PRSS8,
DEFB4B,
CELA3A,
CCL21,
ABCC5,
PARP3,
FGF19,
RHO,
RNF5,
SAA1,
ANXA5,
CCL5,
SFTPA2,
PLCB3,
ARRB2,
PKD1,
ASAH1,
SCNN1D,
VN1R17P,
NOS3,
GPR166P,
LPAR3,
SIRT1,
P2RY2,
PAEP,
NT5C2,
CTRC,
STAT3,
SLC11A1,
CAPN10,
STAT1,
PGF,
PGM3,
SP1,
AIF1,
PI3,
SLPI,
NR1I2,
SQSTM1,
IGFBP3,
MRGPRX1,
MMP1,
HMOX1,
EDNRA,
HGF,
GOPC,
DNMT3B,
CHRM3,
HLA-DQA1,
HLA-DQB1,
EPO,
HLA-DRB1,
DNAH5,
HLA-G,
LGALS14,
CLCN1,
DEFB4A,
DNAAF3,
GPR151,
EGFR,
FSD1,
CHI3L1,
ERN1,
CXCL2,
CHIT1,
F2RL1,
GSTM3,
F5,
LGR6,
HBB,
GUCY2C,
SLC26A6,
MRGPRX3,
MRGPRX4,
GPT,
HBA1,
LRRC26,
RGMA,
FLNC,
GABPA,
HSH2D,
CCR5,
FSD1L,
CYP3A4,
FUT3,
FUT4,
CDKN2A,
CFI,
OXER1,
ACE,
MMAA,
HSPD1,
CTSS,
GPRC6A,
CTSB,
CTSG,
FOXI1,
GC,
CDKN1A,
DDIT3,
CYCSP51,
IL17F,
SLC46A1,
CDK2AP2,
ZGLP1,
GRAP2,
ENPP7,
ENDO1,
RACK1,
ORAI1,
ZMPSTE24,
NCR1,
KLF2,
SCGB3A2,
STARD13,
SYTL3,
HDAC9,
TOM1,
SEC1P,
DNER,
BCAP31,
BPIFB1,
FRMD7,
PLCZ1,
SLC23A2,
DELE1,
CLEC6A,
EIF2AK3,
CLCA3P,
MUC16,
BAG3,
TBPL1,
FAM13A,
G3BP1,
MIR598,
MIR636,
SLC4A7,
VAMP3,
DNAH14,
CD163,
H4C12,
COMMD1,
H4C13,
P2RX5-TAX1BP3,
H4C5,
H4C2,
H4C8,
TIPRL,
RN7SL263P,
PLB1,
LINC02605,
H4C3,
H4C11,
H4C6,
LINC01672,
H4C4,
H4C1,
H4C9,
FGF23,
NR4A3,
AIMP2,
MOGS,
PAOX,
MUC20,
YWHAZ,
H3P8,
YY1,
H19,
H4C14,
NR0B2,
RECK,
LPAR2,
ATG12,
CCNE2,
P2RX6,
USP10,
TRPA1,
HSPB3,
MGAM,
H4-16,
GPNMB,
SUMF1,
PROKR2,
SOCS2,
RBM45,
OCLN,
SNAP23,
ABCC3,
DNAH11,
AHSA2P,
IL31RA,
DEFB104A,
PTGR2,
KHSRP,
WIPF2,
C1orf52,
RAPGEF3,
MED18,
FST,
MIR93,
RETN,
SDF4,
MEMO1,
SPHK2,
F11R,
EJM2,
ACF,
IL22,
SLC12A9,
STOML2,
ACKR3,
SLC39A2,
MIR199A1,
MIR199A2,
AKR1B10,
CD274,
NDUFS7,
PYCARD,
MIR215,
SETD2,
MIR25,
MIR302A,
LAMTOR2,
NLN,
RPTOR,
GBA2,
GBA3,
POLE4,
CPA4,
ASAH2,
TRPV6,
TRPM4,
TRPM7,
UGT1A1,
SYTL2,
MIR125A,
MARCHF1,
SLC52A1,
MIR126,
MIR140,
MIR145,
ELP2,
CHDH,
PLXNA3,
HGH1,
ATF7IP,
FXYD5,
ASIC5,
SF3B6,
GDE1,
CISD1,
MIRLET7E,
TRPV2,
SPG21,
USE1,
SEPTIN3,
TRPV5,
MIR31,
POLD4,
DCTN5,
SGSM3,
CHAC1,
CHEK2,
ADIPOR2,
HSPBAP1,
EHMT1,
METTL8,
DEFB104B,
LPCAT1,
RPP14,
WLS,
WDR26,
COASY,
SPACA9,
STIP1,
SUB1,
CCL27,
MIRLET7G,
AMN,
PTGES3,
CD226,
CXCR6,
H4C15,
KHDRBS1,
CRISPLD2,
ERN2,
KAT5,
MIR494,
TMC6,
WNK4,
UBE2Z,
SLC39A6,
HAMP,
MIR99B,
NLRC4,
SELENOK,
DNAI1,
SACS,
PTPN22,
POLDIP2,
LRRC4,
RNF19A,
CLEC7A,
PART1,
SH3BP4,
LMTK2,
NUP62,
PADI4,
TSPAN12,
BRD4,
NEPNP,
GORASP1,
RRP8,
JMJD6,
WG,
MARCKSL1,
P2RX2,
SIRT2,
ITFG1,
PTH,
VWF,
GNAO1,
GHRHR,
GHSR,
GIP,
GJB2,
GLI1,
GLP1R,
GLS,
GNAS,
GBA,
GOT2,
GP2,
UTS2R,
GPR42,
NR3C1,
GRM1,
GRM5,
GBAP1,
FUT2,
F2,
FGFR2,
PTK2B,
BPTF,
FCGR3A,
FCGR3B,
FCN1,
FGF7,
FGF10,
FGFR4,
FUT1,
FOXJ1,
FOXO1,
FLNA,
FLT1,
FNTA,
FPR2,
MTOR,
CXCL1,
GSR,
GSTM2,
HSP90AA1,
PRMT1,
HSD11B2,
HSF1,
HSPA5,
HSPA9,
HSPB1,
HSPB2,
HSPG2,
GTF2H1,
HTC2,
HTR2A,
IFI16,
IFNA1,
IFNA13,
IFNGR1,
IGFBP2,
HP,
HOXD13,
HOXA2,
HMBS,
GUSB,
GYPA,
GYPB,
GYPE,
H2AX,
HBA2,
HCFC1,
HTT,
HEXA,
NRG1,
HLA-A,
HLA-B,
HLA-DQA2,
HLA-DQB2,
HLA-H,
F10,
EPHB2,
VTN,
BID,
RHOA,
STS,
ATP2A2,
ATP5F1E,
AVP,
AZU1,
BAG1,
BRS3,
AQP9,
C3,
VPS51,
CA12,
CALCR,
CALR,
CALU,
CAPN1,
ARG1,
FAS,
EPHB1,
AHR,
ABO,
ACHE,
ACO2,
ADA,
ADCYAP1,
ADRA1A,
ADRA2B,
ALOX15,
APOE,
AMFR,
AMPD1,
AMPD2,
ANGPT2,
ANK1,
ANXA1,
XIAP,
CASP8,
CAV1,
CAV2,
EDNRB,
DAB2,
DCN,
DCT,
DNAH6,
SLC26A3,
ATN1,
EDN3,
EFNA5,
SERPINA6,
EGF,
ELF5,
ELK3,
ELN,
ADGRE1,
SLC29A1,
EPHA3,
CYP2C19,
CYP1A2,
CTSL,
CCN2,
CD1D,
ENTPD1,
CDC25C,
CEACAM5,
CEBPB,
CETN1,
CEACAM3,
CEACAM8,
CLCN7,
ATF2,
CREBBP,
CRK,
MAPK14,
CSK,
CSTB,
IGHM,
IKBKB,
IL2,
S100A12,
RBP1,
REG1A,
RELA,
RGS2,
RNASE3,
RPS27A,
S100A1,
S100B,
RANGAP1,
SAA2,
SAG,
CCL3,
CCL17,
CXCL6,
SELP,
SFTPC,
RARRES2,
MOK,
IL4,
PRL,
PRKAA2,
PRKCA,
PRKCD,
PRKCE,
MAPK3,
MAPK8,
MAPK9,
PRNP,
RAD51,
PROS1,
MASP1,
TAS2R38,
PTEN,
SERPINA3,
PTX3,
RAB27A,
SGK1,
SI,
SIPA1,
TYR,
TIMP1,
TLR1,
TNFRSF1B,
TP53,
TRH,
TRPC3,
TYMS,
TYRP1,
SLC1A5,
UBC,
UCHL1,
UQCRC1,
UROD,
VCAM1,
VDAC1,
VDR,
THBS1,
TF,
TCF4,
TCEA1,
SLC2A1,
SLC6A2,
SLC6A8,
SLC12A2,
SLC15A2,
SLC22A3,
SMS,
SRI,
SSTR4,
ST14,
STAT6,
STATH,
SYT1,
TAP2,
TAT,
PPP2R1A,
PPARA,
PON2,
MSRA,
MARCKS,
MDK,
MEFV,
MIP,
MMP8,
MPI,
MSH2,
COX1,
NHS,
ND4,
MUC6,
MUC7,
MYC,
MYLK,
CEACAM6,
NCF4,
LMNA,
LIF,
LCT,
LCN1,
IL7,
IDO1,
CXCL10,
INS,
INPP5D,
IRF1,
IRF6,
ITGA2B,
ITK,
KCNK3,
KCNQ1,
KRAS,
KRT13,
LAMB3,
LBP,
NFKB1,
NM,
POMC,
PKM,
PCK1,
PCNA,
PDE7A,
PDK1,
PECAM1,
ATP8B1,
SERPINE2,
PLA2G1B,
NME1,
PLA2G2A,
PLA2G4A,
PLAU,
PLCD1,
PLG,
SERPINF2,
PLOD1,
PCBD1,
PAM,
SERPINE1,
PAH,
NPC1,
NPPA,
NPR3,
NTRK1,
OAS1,
TNFRSF11B,
OPRD1,
P2RX1,
P2RX3,
P2RX4,
P2RX5,
P2RX7,
P2RY1,
P2RY11,
FURIN,
H3P5
Drugs
(R)-1-(2,2-difluorobenzo [d][1,3] dioxol-5-yl)-N-(1-(2,3-dihydroxypropyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl) cyclopropanecarboxamide,
(R)-2-(5-cyano-2-(6-(methoxycarbonyl)-7-methyl-3-oxo-8-(3-(trifluoromethyl)phenyl)-2,3,5,8-tetrahydro-[1,2,4]triazolo[4,3-a]pyrimidine-5-yl)phenyl)-N,N,N-trimethylethanaminium methanesulfonate dehydrate,
11-(4-Dimethylamino-3-hydroxy-6-methyl-tetrahydro-pyran-2-yloxy)-2-ethyl-3,4,10-trihydroxy-3,5,6,8,10,12,14-heptamethyl-1-oxa-6-aza-cyclopentadecane-13,15-dione
(R)-1-(2,2-difluorobenzo [d][1,3] dioxol-5-yl)-N-(1-(2,3-dihydroxypropyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl) cyclopropanecarboxamide,
(R)-2-(5-cyano-2-(6-(methoxycarbonyl)-7-methyl-3-oxo-8-(3-(trifluoromethyl)phenyl)-2,3,5,8-tetrahydro-[1,2,4]triazolo[4,3-a]pyrimidine-5-yl)phenyl)-N,N,N-trimethylethanaminium methanesulfonate dehydrate,
11-(4-Dimethylamino-3-hydroxy-6-methyl-tetrahydro-pyran-2-yloxy)-2-ethyl-3,4,10-trihydroxy-3,5,6,8,10,12,14-heptamethyl-1-oxa-6-aza-cyclopentadecane-13,15-dione,
2-(7-ethoxy-4-(3-fluorophenyl)-1-oxophthalazin-2(1H)-yl)-N-methyl-N-(2-methylbenzo[d]oxazol-6-yl) acetamide,
3-(6-(1-(2,2-difluorobenzo [d] [1,3] dioxol-5-yl)cyclopropanecarboxamido)-3- methylpyridin-2-yl)benzoic acid,
4,6,4'-trymethylangelicin,
4-[[(1S,4S)-5-[[4-[4-(oxazol-2-yl)phenoxy]phenyl]methyl]-2,5-diazabicyclo[2.2.1]hept-2-yl]methyl]benzoic acid,
6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-alpha-L-talofuranosyl)-paromamine sulfate,
8-cyclopentyl-1,3-dipropylxanthine,
Alginate oligosaccharide (G-block) fragment,
Alpha-1 antitrypsin (inhalation use),
Alpha-1 proteinase inhibitor (inhalation use),
Amikacin sulfate,
Amikacin sulfate (liposomal)
(
ARIKAYCE
),
Amiloride hydrochloride dihydrate,
Ataluren
(
TRANSLARNA
),
Avian polyclonal IgY antibody against Pseudomonas aeruginosa,
Aztreonam lysinate (inhalation use)
(
CAYSTON
),
Bacterial lipase,
Carbamic acid, [[4-[[3-[[4-[1-(4-hydroxyphenyl)-1-methylethyl] phenoxy]methyl]phenyl]methoxy]-phenyl]iminomethyl]-,ethyl ester,
Ciprofloxacin (inhalation use),
Ciprofloxacin (liposomal),
Colistimethate sodium
(
COLIMYCINE,
COLOBREATHE
),
Cyclo[L-alanyl-L-seryl-L-isoleucyl-L-prolyl-L-prolyl-L-glutaminyl-L-lysyl-L-tyrosyl-D-prolyl-L-prolyl-(2S)-2-aminodecanoyl-L-alpha-glutamyl-L-threonyl] acetate salt,
Cysteamine,
Cystic fibrosis transmembrane conductance regulator (CFTR) amplifier,
Denufosol tetrasodium,
Dexamethasone sodium phosphate encapsulated in human erythrocytes,
Dornase alfa
(
PULMOZYME
),
Duramycin,
Elexacaftor/Tezacaftor/Ivacaftor
(
Kaftrio,
TRIKAFTA
),
Eluforsen,
Engineered protein inhibitor of human neutrophil elastase,
Fixed-dose combination of fosfomycin disodium and tobramycin,
Gallium citrate,
Glutathione,
Heparin sodium,
Hypothiocyanite / lactoferrin,
Ivacaftor
(
KALYDECO
),
L-Lysine-N-acetyl-L-cysteinate,
Lenabasum,
Liprotamase,
Lumacaftor / Ivacaftor
(
ORKAMBI
),
Mannitolum
(
BRONCHITOL
),
Multilamellar microvesicle comprising phosphatidylcholine, sphingomyelin, phosphatidylethanolamine, phosphatidylserine, phosphatidylinositol and cholesterol,
Nafamostat mesilate,
Nitric oxide
(
GENOSYL,
KINOX,
INOMAX
),
Octovalent Pseudomonas aeruginosa O-polysaccharie-toxin A conjugate vaccine,
Pegylated recombinant anti-Pseudomonas aeruginosa PcrV Fab' antibody,
Phosphoinositide 3-kinase gamma peptide,
Plasmid DNA encoding the human cystic fibrosis transmembrane conductance regulator gene complexed with a non-viral, cationic lipid based gene transfer agent,
Recombinant dog gastric lipase,
Recombinant human CXCL8 mutant,
Recombinant human acid ceramidase,
Recombinant human bile salt-stimulated lipase,
Recombinant microbial lipase,
Sinapultide, dipalmitoylphosphatidylcholine, palmitoyloleoylphosphatidylglycerol and palmitic acid,
Tamoxifen citrate,
Teicoplanin,
Temocillin sodium,
Tezacaftor,
Tezacaftor / Ivacaftor
(
SYMDEKO,
SYMKEVI
),
Tobramycin (inhalation powder)
(
TOBI PODHALER
),
Tobramycin (inhalation solution)
(
TOBI,
TOBRAMYCIN PARI
),
Tobramycin (inhalation use),
Tobramycin (liposomal),
codon-optimised human cystic fibrosis transmembrane conductance regulator messenger ribonucleic acid complexed with lipid-based nanoparticles,
ivacaftor, potassium(benzenesulfonyl)({[6-(3-{2-[1-(trifluoromethyl) cyclopropyl]ethoxy}-1H-pyrazol-1-yl)-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridin-3-yl]carbonyl})azanide, tezacaftor,
levofloxacin hemihydrate
(
QUINSAIR
),
poly-N (acetyl, arginyl) glucosamine (PAAG),
sodium nitrite and EDTA
Registered!
Cystic fibrosis (CF) is a genetic disorder that causes mucus to build up and damage organs in the body, particularly the lungs and pancreas. Signs and symptoms may include salty-tasting skin; persistent coughing; frequent lung infections; wheezing or shortness of breath; poor growth; weight loss; greasy, bulky stools; difficulty with bowel movements; and in males, infertility. Over time, mucus buildup and infections can lead to permanent lung damage, including the formation of scar tissue (fibrosis) and cysts in the lungs. CF is caused by mutations in the CFTR gene and inheritance is autosomal recessive. Treatment aims to relieve symptoms and usually includes respiratory therapies, inhaled medicines, pancreatic enzyme supplement, and nutritional supplements. Newer medications such as CFTR modulators have been approved for use in the United States. Ongoing research is focused on finding a cure for the disease.