DDrare: Database of Drug Development for Rare Diseases

299. 嚢胞性線維症
［臨床試験数：1,592，薬物数：1,539（DrugBank：255），標的遺伝子数：81，標的パスウェイ数：162］

Searched query = "Cystic fibrosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = 0.12% saline; 0.9% Isotonic Saline (IS); 0.9% Isotonic Saline (IS), 4mL; 0.9% normal saline (control); 0.9% w/v sodium chloride solution; 033841; 1,5 (Butylimino)-1,5-dideoxy-D-glucitol; 1-cyclopropyl-6-fluoro-1,4-dihydro-4-oxo-7-[1-piperazinyl]-3-quinoline carboxylic acid; 100 mg ELX /50 mg TEZ /75 mg IVA FDC; 100 mg ELX/50 mg TEZ /75 mg IVA FDC; 100 mg ELX/50 mg TEZ/75 mg IVA fixed dose combination; 100 mg TEZ/150 mg IVA FDC; 100 mg VX-445/50 mg TEZ /75 mg IVA FDC; 100 mg/m2 dose; 100-mg ELX (VX-445) / 50-mg TEZ / 75-mg IVA FDC; 100-mg ELX (VX-455) / 50-mg TEZ / 75-mg IVA FDC; 100-mg ELX / 50-mg TEZ / 75 -mg IVA FDC; 100-mg VX-445 / 50-mg TEZ / 75-mg IVA FDC; 100-mg VX-445 /50-mg TEZ/75-mg; 100-mg VX-445/50-mg TEZ/75-mg IVA FDC; 100mg VX-445/50mg TEZ /75mg IVA FDC; 150-mg IVACAFTOR; 150mg IVACAFTOR; 2% Chlorhexidine solution wipes; 2,3-dihydroxybutanedioic acid; 2-aminoethanethiol; 200 mg/m2 dose; 25-Hydroxyvitamin D3; 2622U90 Duramycin; 291844; 4% chlorhexidine gluconate liquid skin cleanser; 4-phenylbutyrate; 4.2% NaCl/Inhalation solution; 4.2% NaCl/inhalation solution; 5-methyltetrahydrofolate and vitamin B12; 50 mg ELX/25 mg TEZ/37.5 mg IVA fixed dose combination; 5000 IU of cholecalciferol; 552-02; 6% Hypertonic Saline (HS), 4mL; 7% Hypertonic Saline (HS); 7% NaCl; 7% saline; A-F-A-F (NovoRapid-FIASP-NovoRapid-FIASP); AAC Hexal; ABBV-2222; ABBV-3067; ABELCET; ACC; ACC600Tabs; ACETYLCYSTEINE; ACETYLCYSTEINE SODIUM; AER002; AIR DNase; AIR DNase™; ALTU-135; ALTUS-135; ALX-009; AMIKACIN SULFATE; AMILASA; AMITRIPTYLINE; AMOXICILLIN; AMYLASE; ANAKINRA; AP; AP-PA02; API; ARD-3150; ARIKACE™; ARO-ENaC; ATORVASTATIN; AZD1236; AZD5634; AZD5634 for infusion; AZD5634 for inhalation; AZD7986; AZD9668; AZITHROMYCIN; AZITHROMYCIN DIHYDRATE; AZLI; AZLI 75 mg three times a day (TID); AZLI 75 mg three times daily (TID); AZLI 75 mg two times a day (BID)/ three times a day (TID); AZLI 75 mg two times a day (BID)/three times a day (TID); AZTREONAM; AZTREONAM LYSINE; Abelcet; Acebilustat; Acetate; Acetylcysteine; Active Treatment Group 7% Hypertonic Saline; Actrapid 100 IU/ml, Solution for injection in a vial; Actrapid Penfill 100UI/ml; Acute Antioxidant; Adempas; Adempas 0.125 mg; Adempas 0.5 mg; Adempas 1.0 mg; Adempas 2.0 mg; Adeno-associated virus-CFTR vector; Advagraf prolonged-release hard capsules; AeroVanc; Aeroquin; Aerosolized, human, plasma-derived Alpha-1 Antitrypsin; Aerosoltherapy; Air; Ajulemic acid; Alanine; Albuterol; Albuterol inhaler.; Alendronate; Alendronate sodium; Alginate; Alginate oligosaccharide; Allo-hMSCs; Alpha-1 HC 100 mg; Alpha-1 HC 200 mg; Alpha1-Proteinase Inhibitor (Human); Alpha1-proteinase Inhibitor; Amikacin; Amikacin Sulfate; Amikacin nebulization; Amikacin sulfate; Amikacina; Amikacina liposomal (Arikace?); Amikacine; Amikacine nebulization; Amikin, amiklin, amicacin, BB-K8, Biklin, Fabianol, Kaminax, Mikavir, Novamin, Pierami; Amikin®, amiklin, amicacin, BB-K8, Biklin®, Fabianol®, Kaminax, Mikavir, Novamin®, Pierami; Amiloride; Amiloride HCl; Amiloride Solution for Inhalation; Amitriptylin; Amitriptylin-CT; Amitriptylin-ct Tabletten; Amitriptyline; Amoxicillin; Amoxicillin and Clavulanic acid; Amphotericin; Amphotericin B; Amylase; Anakinra; Andecaliximab; Anti-pseudomonas IgY gargle; Anti-reflux pharmacotherapy; Antibiotic; Antioxidant Cocktail; AquADEK; AquADEKs-2; Arginine; Aridol; Aridol or Osmohale; Arikayce; Arikayce™; Arikayce™ 140 mg; Arikayce™ 560 mg; Arikayce™ 70 mg; Ataluren; Ataluren (PTC124®); Atorvastatin; Augmentin; Aurexis; Aurexis® (tefibazumab); Avian polyclonal IgY antibody against PA; Avibactam; Azithromycin; Azithromycin 250 mg tablets; Azithromycin 500 mg film-coated tablet; Aztreonam; Aztreonam Lysine for Inhalation; Aztreonam for Inhalation (AI); Aztreonam for Inhalation Solution; Aztreonam for Inhalation Solution (AZLI); Aztreonam lysine; Aztreonam lysine for inhalation; Aztreonam solución para inhalación; Aztreonan lysine; BAL; BAY 85-8501; BAY q 3939; BAY63-2521; BAY85-8501; BAYQ3939; BAYq3939; BH4; BH4 20mg; BH4 5mg; BI 1265162; BI 1265162 10 µg; BI 1265162 100 µg; BI 1265162 25 µg; BI 1265162 50 µg; BI 1323495; BI 443651; BIIL 283 BS (Amelubent); BIIL 284 BS, high dose; BIIL 284 BS, high dose, adult patients; BIIL 284 BS, high dose, pediatric patients; BIIL 284 BS, low dose; BIIL 284 BS, low dose, adult patients; BIIL 284 BS, low dose, pediatric patients; BIIL 284 BS, medium dose, adult patients; BIIL 284 BS, medium dose, pediatric patients; BLF; BRAMITOB*NEBUL 28F 300MG/4ML; BRAMITOB*NEBUL 56F 300MG/4ML; Bactrim; Balance; Behavioral: Aerobic exercise; Behavioral: Balance exercise; Behavioral: Environmental Decontamination; Behavioral: Exercise; Behavioral: Flexibility and postural exercise; Behavioral: Resistance exercise; Behavioral: Unsupervised exercise; Beta-Adrenergic cocktail; Bethkis; Biaxin ( clarithromycin); Biklin; Bio-25 probiotic; Bisphosphonate treatment; Blood sample; Blood test; Boost VHC; Boots High Strength Garlic Odour Controlled; Bramitob; Bramitob 300mg/4ml Nebuliser Solution; Bramitob® administered by PARI LC® PLUS nebulizer; Bramitob® administered by PARI eFlow® rapid electronic nebulizer; Brensocatib; Brensocatib 10 mg; Brensocatib 25 mg; Broccoli; Broccoli sprouts; Bronchitol; Bucelipase alfa; Budesonide; Budesonide-formoterol single inhaler; Burlulipase; CAYSTON aztreonam 75 mg powder and solvent for nebuliser solution; CB-280; CEFALEXIN; CEFTAZIDIME; CEFTAZIDIME PENTAHYDRATE; CEFUROXIME; CF patients with a G551D mutation and treated with Ivacaftor; CFTR Modulators; CFTR potentiator; CHF 6333; CHF6333 (Part 1 - SAD); CHF6333 (Part 2 - MAD); CIPROFLOXACIN; CIPROFLOXACIN HYDROCHLORIDE; CIPROFLOXACIN HYDROCHLORIDE MONOHYDRATE; CIPROXIN*6CPR RIV 500MG; CLARITHROMYCIN; CLAVULANIC ACID; CLINDAMYCIN; CLORURO SODICO 7%; COLIMICINA - 1000000 U/4 ML POLVERE E SOLVENTE PER SOLUZIONE INIETTABILE PER USO INTRAMUSCOLARE1 FLACONCINO POLVERE + 1 FIALA SOLVENTE 4 ML; COLIMYCINE inhalation; COLIMYCINE injectable; COLISTIMETATO SODICO; COLISTIMETHATE; COLISTIMETHATE SODIUM; COLISTIMETHATE SODIUM (A COMPONENT); COLISTIN; COLISTIN MESILATE SODIUM; COLISTINEB 2MUI; CPX; CR002; CR002 Liquid API; CRC3357, ZK-355322, Leukoton; CREON; CREON 10000 Capsules; CREON IR; CREON N 25000; CSL787; CTX-4430; CURCUMIN; CYC202; 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]; Calcium; Calcium carbonate; Calfactant; Carbonate; Cavosonstat; Cayston; Cayston 75 mg Pulver und Lösungsmittel zur Herstellung einer Lösung für einen Vernebler; Cayston Aztreonam 75 mg powder and solvent for nebuliser solution; Cefalexin; Cefepime; Ceftaroline; Ceftazidim; Ceftazidim Fresenius Kabi; Ceftazidim Fresenius Kabi 2000 mg poeder voor oplossing voor injectie of infusie; Ceftazidime; Ceftazidime and tobramycin; Ceftazidime/avibactam; Ceftolozane; Ceftolozane/Tazobactam; Cefuroxim; Cefuroxime; Cephalexin; Cftr Modulators; Chlorhexidine; Chlorhexidine Gluconate; Chlorhexidine gluconate oral rinse; Chloride; Cholecalciferol; Cholecalciferol (Vitamin D3); Cholecalciferol (vitaminD3); Choline; Choline Chloride; Choline supplementation; Chronic Antioxidant; Cipro (Ciprofloxacin, BAYQ3939); Cipro Inhale; Ciprofloxacin; Ciprofloxacin (BAYQ3939) dry powder for inhalation; Ciprofloxacin (Cipro Inhale, BAYQ3939); Ciprofloxacin (Cipro, BAYQ3939); Ciprofloxacin (ciproxin); Ciprofloxacin DPI; Ciprofloxacin DPI (BAYQ3939); Ciprofloxacin Hydrochloride; Ciprofloxacin Inhale; Ciprofloxacin dispersion for inhalation; Ciprofloxacin for Inhalation; Ciprofloxacin hydrochloride; Ciproxin; Ciproxin Suspension; Clarithromycin; Claritromycin; Claritromycin 500mg film coated tablets; Clavulanate; Clavulanic acid; Clindamycin; Cobamamide; Colimicina; Colimycine; Colistimethat-Natrium; Colistimethate; Colistimethate 75 mg inhaled two times daily; Colistimethate Sodium; Colistimethate sodium; Colistimethate sodium (BAN, USAN), Colistimethatum natricum, Colistin sodium methanesulfonate; Colistimethate sodium (promixin); Colistin; Colistin CF; Colistin Forest - Trockenstechampullen mit Lösungsmittel; Colistineb; Colobreathe; Colomycin; Compound 1a; Continuous infusion Cefepime; Continuous infusion Ceftazidime; Continuous infusion Meropenem; Continuous infusion Piperacillin tazobactam; Continuous infusion Ticarcillin-clavulanate; Control; Control Group 0.9% Isotonic Saline; Control multivitamin; Creon 10.000, hårde enterokapsler; Creon 10000; Creon 25.000, hårde enterokapsler; Creon 25000; Creon 40.000, hårde enterokapsler; Creon IR; Creon N; Creon®; Creon® (DR/GR); Creon® 25,000; Crestor; Curcuma Longa; Curcuma Longa Extract; Curcuma Longa Extract cpl formaat 00 23,2 x 8,5 mm 723 mg; Curcumin; Cystagon; Cystagon (cysteamine bitartrate) immediate-release capsules; Cysteamine; Cysteamine bitartrate; Cysteamine bitartrate (Cystagon); Cystic Fibrosis Ready to Use Supplemental Food; D-a-tocopheryl PEG 1000 succinate; D-glucitol; D6-25-hydroxyvitamin D3; DCI; DCI 1020; DEPELESTAT; DEUTIVACAFTOR; DHA; DICLOXACILLIN; DORIBAX; DORIPENEM HYDRATE; DORNASE ALFA; DOXYCYCLINE; DP Mannitol; DTPA; DURAMYCIN; DX-890; Decolonization; Denosumab; Denufosol; Denufosol Tetrasodium (INS37217) Inhalation Solution; Denufosol tetrasodium (INS37217); Denufosol tetrasodium (INS37217) Inhalation Solution; Denufosol tetrasodium Inhalation Solution; Depelestat; Desoxyribonuclease; Detemir; Detoxifying agents for antineoplastic treatment; Deuterated Ivacaftor; Deutivacaftor; Device: 129Xe Small and Large Human Lung Coil; Device: 3He Human Lung Coil; Device: Continuous Glucose Monitoring; Device: Continuous glucose monitor (CGM); Device: Frequently Sampled Oral Glucose Tolerance Test and CGM; Device: HFNT during exercise; Device: High flow nasal oxygen cannula; Device: Home Oral Glucose Tolerance Test kit; Device: Insulin Pump; Device: Lung Clearance Index; Device: Macroduct sweat stimulator; Device: Magnetic Resonance Imaging; Device: Manuka honey sinus rinse; Device: Nocturnal oxygen , nocturnal bi-level positive pressure ventilation; Device: Non invasive ventilation; Device: Noninvasive ventilation; Device: Oxygen; Device: PARI eFlow nebulizer system; Device: PFP and SF6 Human Lung Coil; Device: Perform three multiple breath nitrogen washout tests (N2-MBW-test); Device: Positive expiratory pressure; Device: RELiZORB (immobilized lipase) cartridge; Device: SmartPill Motility System & PillCam Patency Capsule; Device: Sperti Del Sol Lamp; Device: Standard oxygen therapy/RA; Device: Standard sinus rinse; Device: Tobi Podhaler; Device: activated smart device; Device: de-activated smart device; Dexamethasone; Dexamethasone sodium phosphate 250 mg/10 ml solution; Diagnostic Test: Candy Glucose Tolerance Test; Diagnostic Test: Continuous glucose monitoring; Diagnostic Test: Dexa scan; Diagnostic Test: Infant PFT; Diagnostic Test: Injected abdominal CT; Diagnostic Test: Iontophoresis sessions in forearm of subjects with pilocarpine; Diagnostic Test: Lung Clearance Index; Diagnostic Test: Measurement of Sweat Chloride and Sweat Bromide; Diagnostic Test: Nasal brushing; Diagnostic Test: Oral glucose tolerance test; Diagnostic Test: Passive tidal breathing; Dicloxacillin; Diet; Digitoxin; Docosahexaenoic acid; Docosahexaenoic acid (DHA); Docosahexaenoic acid administration: 50 mg/kg/day during 12 months; Docosahexanoic Acid Supplement; Doribax; Doripenem; Dornase alfa; Dornase alfa (Pulmozyme); Dornase alfa [Pulmozyme®]; Dornase alpha; Doxycyclin; Doxycycline; Dry powder tobramycin; Duramycin; Duramycin, 2622U90; ECGC; EGCG; EGCG + Tocotrienol; ELEXACAFTOR; ELX; ELX (VX-445)/TEZ (VX-661)/IVA (VX-770); ELX-02; ELX/TEZ/IVA; EMEA/H/C/000125; EPI-hNE4 DX-890; EU Orphan Designation Number EU/3/02/120; EUR-1008; EUR-1008 (APT-1008); EUR-1008 25,000 Units; EZ-2053; EZ-2053 5mg/kg; Ebselen; Elexacaftor; Elexacaftor-tezacaftor-ivacaftor exposure; Engineerd Protein Inhibitor of human Neutrophil Elastase; Engineered Protein Inhibitor of human Neutrophil Elastase; Ensure High Protein; Ensure plus; Ergocalciferol; Ergocalciferol (vitamin D2); Esomeprazole; Essential amino acid intake + Leucine vs total AA supplement; Estradiol; Estradiol 2 mg; Ethambutol; Ethinyl estradiol; F-A-F-A (FIASP-NovoRapid-FIASP-NovoRapid); FDL169; FDL176; FDL176 & FDL169 coadministration; FLUCLOXACILLIN; FLUCLOXACILLIN SODIUM; FORLAX 4 g, poudre pour solution buvable en sachet; FTI, AZLI; FUSIDIC ACID; Fenretinide; Ferinject; Ferric carboxymaltose; Ferrous sulfate 325mg; Finely ground flaxseed powder; Fish oil; Flaxseed; FluMist; Flucloxacillin; Flucloxacillin 250mg/5ml Sugar-Free Powder for Oral Solution; Flucloxicillin; Flumist®; Fluticasone; Formoterol; Fortum; Fucidin; Fucidin Tablets; Fusidic acid; Fusidin; G1117337; G914167; G957389; GENISTEIN; GIP; GLP-1; GLPG1837; GLPG1837 dose 1; GLPG1837 dose 2; GLPG1837 dose 3; GLPG2222; GLPG2222 100 mg; GLPG2222 150 mg q.d.; GLPG2222 200 mg; GLPG2222 300 mg q.d.; GLPG2222 400 mg; GLPG2222 50 mg; GLPG2451 dose regimen A; GLPG2451 dose regimen B; GLPG2737; GLPG2737 single dose; GLPG3067; GLPG3067 single dose; GLUTATHIONE; GLUTATHIONE SODIUM; GS-5737; GS-5745; GS-9411; GSH; GSH (Glutathione); Galicaftor; Gallium nitrate; Garlic; Genetic: Blood or Saliva Sample Collection; Genistein; Genistein (Unconjugated Isoflavones 100); Genisteine Plus 150 mg cpl formaat 1 18,7 x 6,4 mm 438 mg; Gentamicin; Gernebcin; Gernebcin 80 mg; Ghrelin; Giving DHA-rich supplement; Glargine; Glargine insulin; Glucose; Glutamine; Glutathione; Glycine; Glycine Buffer; Gold; Growth Hormone; Growth hormone; HEPARIN SODIUM; HMSCs; HP 129Xe; HP 3He; HYPERTONIC SALINE; Helium; Helium-3; Heparin; Heparin 25mg inhalation powder, hard capsule; Heracillin; High dose of budesonide; High-Dose Vitamin D3; Honey; Human recombinant growth hormone; Hyaluronan; Hydrocortisone; Hydroxychloroquine; Hyperglycemic Clamp with concurrent GIP infusion; Hyperglycemic clamp; Hyperglycemic clamp with concurrent GLP-1 infusion; Hyperpolarized Helium-3; Hyperpolarized Helium-3 MRI of the chest; Hyperpolarized Xe129; Hyperpolarized Xenon; Hyperpolarized Xenon 129; Hyperpolarized Xenon gas; Hyperpolarized xenon-129; Hypertonic Saline; Hypertonic Saline 7%; Hypertonic bicarbonate; Hypertonic saline; Hypertonic saline (7 %) and isotonic saline (0.9%); Hypertonic saline (7%); Hypertonic saline 6% -; Hypertonic saline 7%; Hypertonic saline solution; ICS+LABA; IDPM; IDPM: Inhaled Dry Powder Mannitol; IGE025; INCRELEX*SC 1FL 4ML 10MG/ML; INDUSIL*OS GTT FL 30MG+FL 15ML; INOmax; INOmax 400ppm mol/mol inhalation gas; INS1007; INS1007 10 mg oral tablet; INS1007 25 mg oral tablet; IONIS-ENaCRx; ISOTONIC SALINE; ITRACONAZOLE; IV amikacin; IV ceftazidime; IV meropenem; IV piperacillin-tazobactam; IV ticarcillin-clavulanate; IV tobramycin; IV vancomycin hydrochloride; IVA; IVACAFTOR; Ibuprofen; Icenticaftor; IgY; Impact; Impact-Nutridrink; Increlex; Indium; Indium-DTPA; Influenza vaccination; Inhaled 7% hypertonic saline (HS); Inhaled AR-501; Inhaled Dry Powder Mannitol; Inhaled Mannitol; Inhaled Reduced Glutathione; Inhaled SNSP113; Inhaled Sodium Pyruvate; Inhaled Vancomycin; Inhaled dry powder mannitol; Inhaled hypertonic saline (7%); Inhaled mannitol; Inhaled tobramycin; Initiation of CFTR Modulator; Inomax; Insulin; Insulin Asparte; Insulin detemir; Insulin detemir [rDNA origin] injection; Insulin glargine; Insuline humaine recombinante; Interferon Gamma; Interferon gamma-1b; Intermittent, short infusion Cefepime; Intermittent, short infusion Ceftazidime; Intermittent, short infusion Meropenem; Intermittent, short infusion Piperacillin tazobactam; Intermittent, short infusion Ticarcillin-clavulanate; Inulin; Isotonic Saline; Isotonic saline; Isotonic saline 0.9%; Itraconazole; Itraconazole/voriconazole; Ivacaftor; Ivacaftor (proposed INN); Ivacaftor 125mg tablet; Ivacaftor 150 mg; Ivacaftor 150 mg or 250 mg; Ivacaftor 25 mg/75 mg; Ivacaftor 75 mg; Ivacaftor 75 mg/150 mg; Ivacaftor Exposed; Ivacaftor or elexacaftor/tezacaftor/ivacaftor; Ivacaftor+lumacaftor; Ivacaftor/Ataluren; Ivacaftor/Kalydeco; J01GB01; JBT-101; JBT-101 (lenabasum); KB001; KB001-A; KREON 25000; KREON N 25000; KREON de Liberación Inmediata; Kalydeco; Kalydeco 150 mg film-c; Kalydeco 150 mg film-coated tablets; Kalydeco 150mg; Kineret; Klacid; Kreon 25 000; Kreon 25,000 Units; Kreon 25000; Kreon® 25,000; L-Glutamine; L-Lysine; L-Lysine N acetylcysteinate; L-Lysine N-acetylcysteinate; L-Lysine- N-acetylcysteinate; L-Lysine-N-acetyl-L-cysteinate; L-Lysine-N-acetylcysteinate; L-alanine; L-arginine; LABA/LAMA; LANTUS*SC 5CAR3ML100UI/ML; LAU-7b; LENABASUM; LEVOFLOXACIN HEMIHYDRATE; LINEZOLID; LIPASA; LIPASE; LIPASE LIP2; LU 70274; LUM; LUM/IVA; LUM/IVA fixed-dose combination; LUMACAFTOR; Lactic acid; Lactic acid producing organisms; Lactizole nebulization; Lactobacillus reuteri; Lactobacillus rhamnosus; Lactobacillus rhamnosus GG; Lansoprazole; Lantus 100 units/ml solution for injection in a cartridge; Lantus® 100 Units/ml solution for injection in a vial. Lantus® 100 Units/ml solution for injection in a cartridge. Lantus® 100 Units/ml solution for injection in a pre-filled pen; Lenabasum; Lenabasum 20 mg; Lenabasum 5 mg; Leucine; Levemir; Levemir 100 U/ml solution for injection in a cartridge, Levemir 100 U/ml solution for injection in a pre-filled pen; Levemir FlexPen; Levemir Penfill; Levemir insulin; Levofloxacin; Levofloxacin Kabi; Levofloxacin hemihydrate; Linez; Linezolid; Linoleic acid; Linoleic acid supplementation; Lipase CLEC; Lipase Lip2 from Yarrowia lipolytica; Lipitor; Liposomal Amikacin; Liposomal Amikacin (Arikace TM); Liposomal Amikacin (Arikace™); Liposomal amikacin for inhalation; Liposomal amikacin for inhalation (Arikayce™) using the PARI Investigational eFlow® Nebulizer.; Liposomal amphotericin B (Ambisome®); Liprotamasa; Liprotamase; Liprotamase Powder for Oral Solution; Loestrin (norethindrone acetate and ethinyl estradiol); Lonodelestat; Losartan; Lubiprostone; Lucinactant; Lucinactant first; Lumacaftor; Lumacaftor / ivacaftor; Lumacaftor Plus Ivacaftor Combination; Lumacaftor plus Ivacaftor; Lumacaftor, Ivacaftor Drug Combination; Lumacaftor-Ivacaftor treatment; Lumacaftor-ivacaftor; Lumacaftor/Ivacaftor; Lumacaftor/ivacaftor; Lumacaftor/ivacaftor 100mg/125mg tablets; Lumacaftor/ivacaftor 200mg/125mg; Lumacaftor/ivacaftor 200mg/125mg tablets; Lumacaftor/ivacaftor 200mg/83mg tablet; Lym-X-Sorb powder; Lysine; Lysomucil 10 %; MAG-DHA; MANNITOL; MECASERMIN; MEROPENEM; MEROPENEM TRIHYDRATE; MINOCYCLINE HYDROCHLORIDE; MMF; MOLI 1901; MONITOR; MP-376; MP-376 (Levofloxacin Solution for Inhalation); MP-376 (Levofloxacin solution for Inhalation); MRI with PFP; MRT5005; MS1819; MS1819-SD; MSI-1995; MTT; Macrogol; Macrogol 4000; Mannite; Mannitol; Mannitol + pulmozyme; Mannitol inhalation powder; Matched isoglycemic clamp; Mecasermin; Medical Grade Oxygen; Melphalan; Menadiol Diphosphate; Menadiol diphosphate; Menadiol sodium diphosphate; Meropenem; Meropenem Fresenius Kabi; Meropenem Fresenius Kabi 1 g poeder voor oplossing voor injectie of infusie; Meropenem Infusion; Mesenchymal Stem Cells; Metformin; Metformin Hydrochloride; Metformine; Metformine HCL 500 PCH; Miglustat; Minocycline; Modified Oral Glucose Tolerance Test (50g 4-hours); Modified lipid formulation; Molgramostim; Molgramostim nebulizer solution; Moli 1901; Moli 1901 (2622U90, duramycin); Moli1901; Moli1901 (2622U90, duramycin); Moxifloxacin; Moxifloxacin hydrochloride; Mucoid exopolysaccharide P. aeruginosa immune globulin IV; Mucolysin; Mucomyst; Multienzymes (lipase, protease etc.); Mupirocin; Mupirocin Intranasal Creme; N-(5-hydroxy-2,4- bis(trimethylsilyl)phenyl)-4-oxo- 1,4-dihydroquinoline-3-carboxamide; N-(5-hydroxy-2,4-bis(trimethylsilyl)phenyl)-4-oxo-1,4-dihydroquinoline-3-carboxamide; N-(trans-3-(5-((R)-1-hydroxyethyl)-1,3,4-oxadiazol-2-yl)cyclobutyl)-3-phenylisoxazole-5-carboxamide; N-acetylcystein; N-acetylcysteine; N-acetylcysteine (NAC); N6022; N91115; NAC; NAL; NEB/01-07; NEBCINE; NO; NOXAFIL - 40 MG/ML SOSPENSIONE ORALE USO ORALE FLACONE (VETRO) 105 ML 1 FLACONE; NR; Nacystelyn; Nacystelyn®; Nasal Aztreonam; Nebcin; Nebcin (tobramycin); Nebcinal; Nebcinal Tobi; Nebulization of Amikacin during NIV (RR: 15 cycles/minute); Nebulization of Amikacin during NIV (RR: 25 cycles/minute); Nebulization tobramycin; Nexium; Nexium 20; Nitrate; Nitric Oxide; Nitric Oxide 160 ppm; Nitric Oxide for Inhalation; Nitric oxide; Nitrite; Nitrogen; None; None assigned; Norethindrone; Norgestimate; Normal Saline; Normal saline; Normal saline IV, salt tablets; Normal saline solution; Normal saline:; Norvir; Norvir 100 mg film-coated tablets; Novamin; Novo Rapid; Novo Rapid Insulin (Novonordisk); NovoNorm 0.5 mg; NovoNorm 1 mg; NovoNorm 2 mg; NovoRapid 100 U/ml, solution for injection in a vial. NovoRapid Penfill 100 U/ml, solution for injection in a cartridge. NovoRapid FlexPen 100 U/ml, solution for injection in a pre-filled pen.; NovoRapid Aspartate; Novolog insulin; Novorapid / humalog short acting insulin; Noxafil; Noxafil - oral suspension 40 mg/ml; Noxafil 100 mg; Noxafil®; Nutridrink-Impact; Nutropin AQ; Nutropin AQ [somatropin (DNA origin) injection]; OGT 918; OLIGOG CF-5/20; OMEGA-3-ACID TRIGLYCERIDES; OSCN-; Oleic Acid; Oleic acid supplementation; OligoG; OligoG (60 mg/ml); OligoG CF-5/20; OligoG CF-5/20 - 17.5 mg; OligoG CF-5/20, 17,5 mg, Hartkapsel mit Pulver zur Inhalation; OligoG CF/20 - 27.5 mg; OligoG CF/20 - 37.5 mg; OligoG CF/20, 27,5 mg, Hartkapsel mit Pulver zur Inhalation; OligoG CF/20, 37,5 mg, Hartkapsel mit Pulver zur Inhalation; OligoG DPI; OligoG Dry powder for inhalation; OligoG-CF5/20, Alginate oligosaccharide (G-block) fragment; Oligomer of Sodium Alginate; Omadacycline; Omadacycline Injection [Nuzyra]; Omadacycline Oral Tablet [Nuzyra]; Omalizumab; Omega 3; Omega 3 Premium; Omega-3; Omega-3 triglycerides; Omeprazole; Omeprazole 40mg Capsule; Once-daily insulin detemir; Oral Aztreonam; Oral Glucose Tolerance test (75g 2-hour); Oral Glutathione; Oral ciprofloxacin; Oral ciprofloxacin plus inhaled colistin; Oral reduced l-glutathione; Oral roflumilast; Orkambi; Orkambi 100 mg/125 mg film-coated tablets; Orkambi 200 mg/125 mg film-coated tablets; Orkambi: contains lumacaftor and ivacaftor; Orkambi®; Ortho-cyclen, Ethinyl estradiol/norgestimate, 0.035 mg/0.250 mg; Oseltamivir; Osmohale; Other non-sponsor pancreatic enzyme replacement therapy; Other respiratory system products; Other: A-STEP; Other: A-STEP (New Protocol); Other: Absorptive clearance scan; Other: Air - oxygen; Other: Autogenic drainage; Other: Balance; Other: Blood glucose results; Other: Comparator: CPET cycle ergometer (Gold Standard); Other: Continuation of dornase alfa (dnase); Other: Continuation of hypertonic saline (HS); Other: Diagnostics for glucose tolerance with 3 different methods.; Other: Discontinuation of dornase alfa (dnase); Other: Discontinuation of hypertonic saline (HS); Other: Exercise capacity; Other: Fruit juice; Other: Glucose -potentiated arginine (GPA) stimulation tests; Other: Glucose profile for 2 weeks; Other: Hepatic elastography; Other: High Glycemic Index Meal; Other: Hyperpolarized Xenon MRI; Other: Hypoglycemic Clamp; Other: Impact Peptide 1.5; Other: Inhalable Hypertonic saline 7%; Other: Inhalable Hypertonic saline 7% + Hyaluronan 0.1%; Other: Liquid Meal Test (Carbohydrate-rich); Other: Liquid Meal Test (Fat-rich); Other: Liquid Meal Test (Mixed); Other: Low Glycemic Index Meal; Other: Mixed Meal Tolerance Test; Other: Mixed meal test evaluation test arm; Other: Muscle oxygenation; Other: No doxycycline; Other: No pancreatic enzyme replacement therapy; Other: Non-hormonal period; Other: OGTT; Other: On-demand treatment; Other: Ophthalmologic examinations; Other: Oral Glucose Tolerance Test; Other: Oral Glucose Tolerance Test (OGTT) assessment arm; Other: Oxygen - Air; Other: Pentadecanoic acid (PA) and Triheptadecanoin (THA); Other: Reaction Time and Postural Control; Other: Sham Comparator; Other: Sputum Sample; Other: Sputum sample; Other: Stool Sample; Other: Technetium [Tc-99m] sulfur colloid and Indium [In-111] DTPA; Other: Test Soda; Other: Tobacco questionnaire; Other: Tobramycin time of administration; Other: blood sample; Other: glucose solution at a dose of 1.75 g/kg (up to a maximum of 75 g) for the OGTT and glucose solution at a dose of 0.5 g/kg (up to a maximum of 35 g) was injected in 2.5-3 min for the IGTT; Other: measure of endothelial function; Other: quercetin; Other: routine therapy; Oxygen; P-1037; PAAG15; PANCREASE MT; PANCREASLIPASE; PANCREATIN; PANCREATIN (PAncreas Powder); PANCREATIN (Pancreas Powder); PANCREATINA; PANCREATINA (Páncreas Polvo; PANCREATINA (Páncreas Polvo); PANCREAZE; PANCREAZE®; PANCRECARB® (pancrelipase); PANCRELIPASE AMYLASE; PANZYTRAT 25000; PBI4050; PC945; PEG; PERTZYE; PFP; PFT; PGM169/GL67A; PIPERACILLIN; PIPERACILLIN SODIUM; PL 0010/0211; PO azithromycin; PO ciprofloxacin; POL6014; POL7080; POSACONAZOLO; PPI treatment; PREFOLIC*30CPR GASTROR 15MG; PROMIXIN*NEBUL 30MONOD 1MUI; PROTEASA; PROTEASE; PTC-0161480; PTC-124; PTC-C124; PTC124; PTI-428; PTI-801; PTI-808; PUR118; Pancreas Powder; Pancrease; Pancrease MT 10.5, or MT 21; Pancreatin; Pancrelipase; Pancrelipase Delayed Release; Pancrelipase Delayed Release Capsule; Pancrelipase Microtablets; Pancrelipase amylase; Pancrelipase microtablets; Pancrelipase with bicarbonate; Pancrelipase/Pancreas Powder; Pankreatin; Panzytrat® 25,000; Panzytrat® ok; Perfluorinated gas/oxygen mixture; Phenylbutyrate; Phosphate; Physiologic serum nebulized nasally; Physiological solution; Pilocarpine; Pilocarpine Nitrate 5%; Pioglitazone; Piperacillin; Piperacillin sodium , tazobactam sodium; Piperacillin with Tazobactam (tazocin); Piperacillin-tazobactam combination product; Piperacillin/Tazobactam; Piperacillin/Tazobactam Kabi - Fresenius Kabi Austria; Piperacilline/Tazobactam Fresenius Kabi 2 g/0,25 g poeder voor oplossing voor infusie; Piperacilline/Tazobactam Fresenius Kabi 4 g/0,5 g poeder voor oplossing voor infusie; Plerixafor; Plerixafor mobilization of autologous CD117 stem cells; Polyethylene Glycol 3350; Polyethylene glycol; Porcine (pig) PERT; Porcine PERT; Posaconazole; Posaconazolo; Prednisolone; Prednisone; Procedure: Airway clearance technique; Procedure: Blood draw; Procedure: Blood draw.; Procedure: Blood sampling for determination of serum Zn; Procedure: Continuous Glucose Monitoring System (CGMS); Procedure: Dual Energy X-ray Absorptiometry (DEXA); Procedure: Exhaled Nitric Oxide; Procedure: FDG-PET; Procedure: Growth Hormone Stimulation Test; Procedure: HGPIV diagnosis test; Procedure: HOMAR-IR diagnosis test; Procedure: History and physical exam.; Procedure: IV glucose tolerance test; Procedure: Indirect Calorimetry; Procedure: Oral Glucose Tolerance Test; Procedure: Oral Glucose tolerance test; Procedure: Physiotherapy, exercise, percutaneous gastrostomy feeds; Procedure: Pulmonary Function Testing; Procedure: Pulmonary function testing.; Procedure: Sputum Collection; Procedure: Urinary collect; Procedure: Whole body Protein Turnover; Procedure: Whole body Protein Turnover Study; Procedure: Whole body protein turnover; Procedure: bronchoscopy with BAL; Procedure: sodium bicarbonate; Procedure: spirometry; Prograft capsules; Prolastin; Prolastin (drug); Promixin; Promixin 1 million International Units (IU) Powder for Nebuliser Solution; Promixin, 1 million International Units (IU) Powder for Nebuliser Solution; Promixin,1 millón de Unidades Internacionales,polvo para solución para inhalación por nebulizador; Promixin/Tadim; Protease; PsAer IgY; Pulmaquin®; Pulmozyme; Pulmozyme (Dornase alpha); Pulmozyme (dornase alfa); Pulmozyme single use ampule; Pulmozyme®; QAU145; QBW251; QBW276; QBW276 1500mcg; QBW276 300mcg; QBW276 Succinate; QR-010; Quercetin; R03AC02; R03CC02; R05C B01; RIFAMPICIN; RIOCIGUAT; RPL554; RPS 2505; Radiation: MRI Pancreas and Liver; Radiolabelled OligoG CF-5/20 6% Solution; Radiolabelled OligoG CF-5/20 6% Solution for Nebulisation; Radiolabelled OligoG CF-5/20 DPI; Radiolabelled OligoG CF-5/20 Dry Powder for Inhalation (DPI); Rapeglinide; Ravicti; Ravicti high dose; Ravicti low dose; Recombiant human Desoxyribonuclease ( rhDNase); Recombinant Human DNase (Pulmozyme); Recombinant human IGF-1; Recombinant humanized monoclonal antibody omalizumab to IgE; Recombinant truncated SPINT2 protease inhibitor; Red Blood Cell Zn; Reduced glutathione; Reduced glutathione sodium salt; Repaglinide; Repaglinide and Insulin; Reparixin; Resunab, ajulemic acid, anabasum; Resveratrol; RhBSSL; RhDNAse; Rifampicin; Rifampin; Rimactan; Riociguat; Riociguat (Adempas, BAY63-2521); Roflumilast; Roflumilast Oral Tablet; Roscovitine; Rosuvastatin; Rosuvastatin calcium; Roxithromycin; Répaglinide; S-1226; SALBUTAMOL; SALINE; SB-656933 Tablets; SB-656933-AAA; SB656933; SE10599; SELICICLIB; SF6; SIN ASIGNAR; SLIT Amikacin; SLIT(TM) Amikacin; SMX; SNSP113; SODIUM ALGINATE; SODIUM BICARBONATE BP; SODIUM CHLORIDE; SODIUM CHLORIDE 7%; SODIUM CHLORIDE PH. EUR.; SODIUM CHLORIDE SOLUTION 0.9%; SPI-1005 Ebselen 200mg Capsule x1; SPI-1005 Ebselen 200mg Capsule x2; SPI-1005 Ebselen 200mg Capsule x3; SPORANOX; SPX-101; STERILE PYROGEN-FREE ISOTONIC NACL SOLUTE (0.9% W / V); SUB185183; SULFAMETHOXAZOLE; Salbutamol; Saline; Saline 0.9% inhalation solution; Saline Solution; Salmeterol; Saxagliptin; Seliciclib; Serum Zn; Short-acting Insulin (Actrapid); Sildenafil; Sildenafil (Acute-1 hour); Sildenafil (Subchronic-4 weeks); Sildenafil 40mg oral capsule; Simvastatin; Simvastatin treatment for 28 days; Sitagliptin; Snack beverage; Sodium (R)-8-methyl-2-(3-methylbenzofuran-2-yl)-5-(1-(tetrahydro-2H-pyran-4-yl)ethoxy)quinoline-4-ca; Sodium 4-Phenylbutyrate; Sodium 4-Phenylbutyrate (4PBA); Sodium Bicarbonate; Sodium Chloride; Sodium Pyruvate in 0.9% Sodium Chloride Solution; Sodium alginate; Sodium bicarbonate; Sodium chloride; Sodium chloride (7%); Sodium chloride 0,9%; Sodium chloride 6%; Sodium chloride 7%; Sodium nitrite; Soluble fiber supplementation; Somalgen; Somatropin; Spiriva; Spiriva Respimat; Spiriva Respimat 2.5 mcg; Spiriva Respimat 2.5 microgram; Spiriva Respimat 2.5 microgram, solution for inhalation; Sporanox; Sporanox 10 mg/ml Oral Solution; Sputum; Ss-Adrenergic cocktail; Standard Care; Standard Formula; Standard formula (Enfamil); Standard of care IV antibiotic(s); Standardized turmeric root extract; Study medication, azithromycin; Sulfamethoxazole; Sulfate; Sulfato de amikacina; Sulfur Colloid; Sunflower Oil; Sunflower oil; Supplementation with vitamin D2/D3; Symbiotic Formula with DHA and antioxidants; Symdeko; Symkevi; Symkevi 100mg/150mg Film-coated tablets; Synrinse; T 100 PARI; T-326; T100; T100 PARI; TACROLIMUS; TAD 600; TAFLUPROST; TAZOBACTAM; TAZOBACTAM SODIUM; TBD; TBM100; TBM100C; TEZ; TEZ/IVA; TEZACAFTOR; THA; TIOTROPIUM; TIOTROPIUM BROMIDE MONOHYDRATE; TIP; TIP (Tobramycin inhalation powder); TIS (Tobramycin Inhalation Solution); TIS or TIP; TMP; TNSE; TOBI; TOBI (tobramycin); TOBI 300 mg / 5 mL nebuliser solution; TOBI 300 mg/5 ml Lösung für einen; TOBI 300 mg/5 ml Lösung für einen Vernebler; TOBI 300 mg/5 ml Nebuliser Solution; TOBI 300 mg/5 ml solución para inhalación por nebulizador; TOBI 300 mg/5 ml, solution pour inhalation par nébuliseur; TOBI 300mg/ 5ml solución para inhalación con nebulizador; TOBI 300mg/5mL Nebuliser Solution; TOBI 300mg/5mL nebuliser solution; TOBI 300mg/5ml Nebuliser solution; TOBI PODHALER; TOBI PODHALER - 28 MG POLVERE PER INALAZIONE CAPSULA RIGIDA USO INALATORIO BLISTER(ALU/ALU) 56 CAPSULE + 1 INALATORE; TOBI PODHALER*448CPS 28MG+10IN; TOBI Podhaler; TOBI Podhaler 28 mg Hartkapseln mit Pulver zur Inhalation; TOBI Podhaler 28 mg inhalációs por, kemény kapszula; TOBI*NEBUL 56F 1D 300MG/5ML; TOBI®; TOBI™ Podhaler™ 112 mg inhaled twice daily; TOBRAMICINA; TOBRAMYCIN; TOBRAMYCIN SULFATE; TR02; TRIMETHOPRIM; Tacrolimus; Tacrolimus and MMF; Tacrolimus capsules; Tacrolimus prolonged-release hard capsules; Tad; Tadim; Tafluprost; Talniflumate; Tauroursodeoxycholic acid; Tazobactam; Technetium Sulfur Colloid; Tedizolid; Tedizolid IV; Tedizolid PO; Tefibazumab; Teicoplanin; Teicoplanin Sandoz 200 mg powder and solvent for solution for injection or infusion or oral solution.; Telavancin; Telavancin Injection; Tezacaftor; Tezacaftor (TEZ); Tezacaftor/Ivacaftor; Tezacaftor/Ivacaftor + Ivacaftor; Tezacaftor/Ivacaftor 100mg/150mg; Tezacaftor/Ivacaftor/100mg/150mg; Tezacaftor/ivacaftor; Tezacaftor/ivacaftor 100mg/150mg; Tham; The gut mycobiota and microbiota profile; The lung mycobiota and microbiota profile; Theophylline; Ticarcillin; Tigerase®; Tiotropium; Tiotropium Bromide (monohydrate); Tiotropium Respimat; Tiotropium Respimat 1.25 microgram solution for inhalation; Tiotropium Respimat 1.25 microgram, solution for inhalation; Tiotropium Respimat 1.25 micrograms solution for inhalation; Tiotropium Respimat® inhaler; Tiotropium bromide; Tiotropium bromide 5 mcg; Tiotropium bromide high; Tiotropium bromide low; Tiotropium bromide medium; Tiotropium bromide monohydrate; Tiotropium bromide-low dose-2.5mcg; Tobacco; Tobi; Tobi 300 mg/5 ml Nebuliser Solution; Tobi 300mg/5ml Nebuliser solution.; Tobi 300mg/5ml Solución para inhalación por nebulizador.; Tobi Inhalant Product; Tobi Nebcinal; Tobramcyin; Tobramicina; Tobramicina polvere inalatoria; Tobramycin; Tobramycin (Gernebcin®); Tobramycin / Bramitob; Tobramycin / TOBI; Tobramycin 100 PARI; Tobramycin Inhalation Powder; Tobramycin Inhalation Powder (TIP); Tobramycin Inhalation Powder TIP delivered by the T-326 Inhaler; Tobramycin Inhalation Solution (TIS); Tobramycin Powder; Tobramycin Solution for Inhalation; Tobramycin Steri-Neb 300 mg/5 ml, inhalation solution; Tobramycin dry powder; Tobramycin for Inhalation; Tobramycin inhalation powder; Tobramycin inhalation solution; Tobramycin inhalation solution using a PARI LC® Plus nebulizer.; Tobramycin injection 40mg/1ml; Tobramycin nebulized nasally; Tobramycin solution for inhalation; Tobramycin solution for inhalation (TOBI); Tobramycin solution for inhalation 300 mg; Tobramycine; Tobramycinum; Tobrineb/Bramitob; Tocotrienol; Treatment; Treatment with polyethylene glycol (Macrogol 4000); Trifakta; Trikafta; Trimethoprim; Trimethoprim Sulfamethoxazole (TMP/SMX); Trimethoprim and Sulfamethoxazole; Trimethoprim-sulfamethoxazole; Trimethoprim/Sulfamethoxazole; Trimethoprim/Sulfamethoxazole (TMP/SMX); Trimetoprim; Triple combination therapy; Turmeric; ULTRESA; Ultrase; Ultrase® MT12; Ultrase® MT20; Unconjugated Isoflavones 100 (PTI G-4660, 87% Genistein); Uridine; Urinary levels of PIIINP as a marker of changes in renal function; Ursodiol; V-Fend; VEDROP; VFEND 40 mg/ml powder for oral suspension; VITAMIN E - TGPS; VORICONAZOLE; VR496; VRT-813077; VRT-826809; VRT-893661; VRT-893661 VRT-0893661; VX-121; VX-152; VX-371; VX-371 in hypertonic saline; VX-371 in saline; VX-440; VX-445; VX-445 / TEZ / IVA; VX-445 / TEZ / IVA FDC; VX-445 /TEZ/IVA; VX-445/TEZ/IVA; VX-561; VX-561 (CTP-656); VX-659; VX-659/ TEZ/IVA; VX-659/TEZ/IVA; VX-659/Tezacaftor/Ivacaftor; VX-661; VX-661 / VX-770; VX-661 Plus Ivacaftor Combination; VX-661 plus ivacaftor combination; VX-661, VRT-893661; VX-661/Ivacaftor; VX-661/VX-770; VX-661/VX-770 (TEZ/IVA); VX-661/ivacaftor (VX-770) 100mg/150 mg; VX-661/ivacaftor 100 mg/ 150 mg; VX-661/ivacaftor 100mg/150 mg; VX-661/ivacaftor 100mg/150mg; VX-661/ivacaftor 50 mg/ 75 mg; VX-770; VX-770, VRT-8130077; VX-770, VRT-813077; VX-809; VX-809 & VX-770; VX-809 / VX-770; VX-809, VRT-826809; VX-809/VX-770; Vaccin grippal pandémique H1N1; Vaccination with vaccine against hepatitis A and B; Valganciclovir; Valganciclovir [Valcyte]; Vancomycin; Vancomycin hydrochloride inhalation powder; Vancomycin inhalation powder; Vantobra (tobramycin); Vardenafil; Vedrop; Vfend; Vitamin B12; Vitamin D; Vitamin D Oil; Vitamin D Powder; Vitamin D2; Vitamin D3; Vitamin E; Vitamin K; Vitamin K supplementation, dose #1; Vitamin K supplementation, dose #2; Vitamin: Choline Bitartrate (2-hydroxyethyl) trimethylammonium salt 1:1; Voriconazole; WATER FOR INJECTIONS, EP; Water; Water for injections; XOLAIR; Xenon; Xenon-129; Xolair; Xolair 150 mg Pulver und Injektionslösung zur Herstellung einer Injektionslösung; Xylitol; ZENPEP; ZITHOMAX 40 mg/ml ENFANTS; ZITHROMAX; ZITHROMAX 250 mg; Zavesca; Zenpep; Zinc; Zinc acetate; Zinc acetate (20mg/d); Zithromax; Zitromax; Zn; Zoledronic acid; [N/A]; [NA]; [SPX-101]; [na]

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT03894657 (ClinicalTrials.gov)	December 18, 2019	12/2/2019	Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®.	Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®. Pathway to Personalized Therapy in Cystic Fibrosis	Cystic Fibrosis;Homozygous F508del Mutation	Diagnostic Test: Nasal brushing;Other: Sputum sample;Other: blood sample;Drug: Orkambi	Assistance Publique - Hôpitaux de Paris	NULL	Recruiting	5 Years	N/A	All	104	N/A	France
2	NCT03150719 (ClinicalTrials.gov)	May 24, 2017	3/5/2017	A Study to Evaluate Safety, Efficacy, and Tolerability of TEZ/IVA in Orkambi® (Lumacaftor/Ivacaftor) -Experienced Subjects With Cystic Fibrosis (CF)	Phase 3b, Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Assess the Safety, Efficacy, and Tolerability of Tezacaftor/Ivacaftor (TEZ/IVA) in an Orkambi-experienced Population Who Are Homozygous for the F508del CFTR Mutation	Cystic Fibrosis	Drug: Tezacaftor /Ivacaftor;Drug: Ivacaftor;Drug: Placebo	Vertex Pharmaceuticals Incorporated	NULL	Completed	12 Years	N/A	All	98	Phase 3	United States;France;Germany
3	EUCTR2018-002624-16-FR (EUCTR)		21/11/2018	NA	Validation of respiratory epithelial functional assessment to predict clinical efficacy of Orkambi®. Pathway to personalized therapy in Cystic Fibrosis PREDICT-CF - PREDICT-CF	Homozygous F508del patient aged 12 years or older MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]		ASSISTANC-PUBLIQUE-HOPITAUX DE PARIS (AP-HP)	NULL	NA			Female: yes Male: yes	104	Phase 4	France

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

NCT03894657
(ClinicalTrials.gov)

December 18, 2019

12/2/2019

Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®.

Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®. Pathway to Personalized Therapy in Cystic Fibrosis

Cystic Fibrosis;Homozygous F508del Mutation

Diagnostic Test: Nasal brushing;Other: Sputum sample;Other: blood sample;Drug: Orkambi

Assistance Publique - Hôpitaux de Paris

NULL

Recruiting

5 Years

N/A

All

104

N/A

France

NCT03150719
(ClinicalTrials.gov)

May 24, 2017

3/5/2017

A Study to Evaluate Safety, Efficacy, and Tolerability of TEZ/IVA in Orkambi® (Lumacaftor/Ivacaftor) -Experienced Subjects With Cystic Fibrosis (CF)

Phase 3b, Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Assess the Safety, Efficacy, and Tolerability of Tezacaftor/Ivacaftor (TEZ/IVA) in an Orkambi-experienced Population Who Are Homozygous for the F508del CFTR Mutation

Cystic Fibrosis

Drug: Tezacaftor /Ivacaftor;Drug: Ivacaftor;Drug: Placebo

Vertex Pharmaceuticals Incorporated

NULL

Completed

12 Years

N/A

All

Phase 3

United States;France;Germany

EUCTR2018-002624-16-FR
(EUCTR)

21/11/2018

Validation of respiratory epithelial functional assessment to predict clinical efficacy of Orkambi®. Pathway to personalized therapy in Cystic Fibrosis PREDICT-CF - PREDICT-CF

Homozygous F508del patient aged 12 years or older
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

ASSISTANC-PUBLIQUE-HOPITAUX DE PARIS (AP-HP)

NULL

Female: yes
Male: yes

104

Phase 4

France