Clinical trials: Gastrointestinal System--Abnormalities
Neuroblastoma, Medulloblastoma
Molecular-Guided Therapy for Childhood Cancer
The purpose of this study is to test the feasibility (ability to be done) of experimental technologies to determine a tumor's molecular makeup.
- Conditions
- Neuroblastoma, Medulloblastoma, Glioma, Ependymoma
- Trial name
- NCT02162732
- Testing
- Guided Therapy
- Where
- 19 sites in United States, Lebanon
- Volunteers
- 186 people
- Sponsor
- Giselle Sholler
Cystic Fibrosis
A Phase 3 Study of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
This study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF…
- Trial name
- NCT03447249
- Testing
- VX-659/TEZ/IVA, IVA
- Where
- 101 sites in United States, Australia and 9 more
- Volunteers
- 385 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Familial Adenomatous Polyposis
Trial of Eflornithine Plus Sulindac in Patients With Familial Adenomatous Polyposis (FAP)
The purpose of this randomized, double-blind, Phase III trial is to determine if the combination of eflornithine plus sulindac is superior to sulindac or eflornithine as single agents in delaying time to the first occurrence of…
- Trial name
- NCT01483144
- Testing
- Eflornithine, Sulindac 150 MG
- Where
- 17 sites in United States, Belgium and 5 more
- Volunteers
- 171 people
- Sponsor
- Cancer Prevention Pharmaceuticals, Inc.
Cystic Fibrosis
A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
This study will evaluate the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF…
- Trial name
- NCT03525444
- Testing
- VX-445/TEZ/IVA, IVA
- Where
- 115 sites in United States, Australia and 11 more
- Volunteers
- 405 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Acute Intermittent Porphyria, Hereditary Coproporphyria
Panhematin for Prevention of Acute Attacks of Porphyria
The purpose of this study is to determine if Panhematin is safe and effective for prevention of acute attacks of porphyria. The study aims to provide high quality evidence for the use on hemin for prevention of acute attacks of…
- Conditions
- Acute Intermittent Porphyria, Hereditary Coproporphyria, Variegate Porphyria
- Trial name
- NCT02922413
- Testing
- Hemin for injection
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- The University of Texas Medical Branch, Galveston
Cystic Fibrosis
Ketone Supplementation in Cystic Fibrosis
This study will examine the effects of a novel ketone monester supplement on patients with cystic fibrosis experiencing an acute pulmonary exacerbation requiring hospitalization.
- Trial name
- NCT04938726
- Testing
- Ketone Monoester
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- University of Alabama at Birmingham
Cystic Fibrosis
Study to Evaluate the Safety and Efficacy of Ciprofloxacin (Inhaled) in Patients With Cystic Fibrosis
To evaluate the change in forced expiratory volume (FEV1) from baseline to Day 28-30 between Cipro Inhale-treated and placebo-treated subjects after a 4-week treatment period.
- Trial name
- NCT00645788
- Testing
- Ciprofloxacin (Cipro Inhale, BAYQ3939)
- Where
- 86 sites in United States, Australia and 7 more
- Volunteers
- 288 people
- Sponsor
- Bayer
Colorectal Cancer, Hereditary Non-polyposis Colon Cancer
Educational CD-ROM Compared With Standard Informed Consent for Patients With Colorectal Cancer or a Family History of Colorectal Cancer
RATIONALE: The use of a CD-ROM may help patients with colorectal cancer or a family history of colorectal cancer make informed decisions about undergoing microsatellite instability (MSI) testing.
- Trial name
- NCT00450424
- Testing
- counseling intervention, CD-ROM intervention
- Where
- 3 sites in United States
- Volunteers
- 239 people
- Sponsor
- Fox Chase Cancer Center
Erythropoietic Protoporphyria
Multicentre Phase III Erythropoietic Protoporphyria Study
This was a phase III, multicentre, randomised, double-blind, placebo-controlled study, to evaluate the safety and efficacy of subcutaneous bioresorbable afamelanotide implants in patients with Erythropoietic Protoporphyria (EPP).
- Trial name
- NCT04053270
- Testing
- Afamelanotide
- Volunteers
- 100 people
- Sponsor
- Clinuvel Pharmaceuticals Limited
Wilson's Disease
Study Comparing Once Daily Dose of 900mg of TETA 4HCL Against Cuprior® (450mg Trientine Base, Twice Daily).
A randomised, open-label study evaluating the pharmacokinetics, safety, and tolerability of a new once daily dose of 900mg of TETA 4HCL by comparing it against the current marketed Cuprior® formulation (450mg trientine base…
- Trial name
- NCT06128954
- Testing
- 900mg TETA 4HCl Once Daily Formulation, 900mg TETA 4HCl Cuprior®
- Where
- United Kingdom
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Orphalan
Alpha-1-antitrypsin Deficiency, Liver Cirrhosis
Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency
The primary objective is to determine if the medication Carbamazepine, can be used as a therapy for patients with severe liver disease due to Alpha-1-Antitrypsin Deficiency .
- Trial name
- CBZ (NCT01379469)
- Testing
- Drug-Carbamazepine (Tegretol XR)
- Where
- 3 sites in United States
- Volunteers
- 20 people
- Sponsor
- Washington University School of Medicine
Cystic Fibrosis
Effects of an Antioxidant-Enriched Multivitamin Supplement on Inflammation and Oxidative Stress in Cystic Fibrosis
The purpose of this study will be to evaluate the effects of a modified formulation of AquADEKs (AquADEKs-2) on markers of inflammation, antioxidant levels and oxidative stress.
- Trial name
- AquADEKs-2 (NCT01859390)
- Testing
- AquADEKs-2, control multivitamin
- Where
- 15 sites in United States
- Volunteers
- 73 people
- Sponsor
- University of Colorado, Denver
Chronic Hepatitis C Infection
Open-Labeled Study of PSI-7977 and RBV With and Without PEG-IFN in Treatment-Naïve Patients With HCV GT2 or GT3
This study is to assess the safety and tolerability of sofosbuvir (SOF) 400 mg with and without ribavirin (RBV) and/or with and without pegylated interferon alfa-2a (PEG) in subjects with genotype 1, 2 or 3 hepatitis C (HCV)…
- Trial name
- NCT01260350
- Testing
- SOF, RBV, PEG
- Where
- 2 sites in New Zealand
- Volunteers
- 292 people
- Sponsor
- Gilead Sciences
Cystic Fibrosis
Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA TC Combination Therapy in Participants With Cystic Fibrosis Who Are 6 Years of Age and Older
The study evaluates the long-term safety, tolerability, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in participants with cystic fibrosis (CF).
- Trial name
- NCT04183790
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 21 sites in United States, Australia and 3 more
- Volunteers
- 64 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Irritable Bowel Syndrome Constipation Predominant
Effects of ROSE-010 on GI Transit in Constipation Predominant Irritable Bowel Syndrome (C-IBS) Patients
This trial will study the effects of an investigational (not FDA approved) medication, ROSE-010, on the movement of food through the stomach, small intestine and colon in females with constipation predominant irritable bowel…
- Trial name
- NCT01056107
- Testing
- ROSE-010
- Where
- United States
- Volunteers
- 52 people
- Sponsor
- Mayo Clinic
Cystic Fibrosis
Effectiveness of Pulmozyme in Infants With Cystic Fibrosis
This is a study to find out whether Pulmozyme is effective for clearing mucus from the airways of children with cystic fibrosis less than 3 ½ years of age.
- Trial name
- NCT00179998
- Testing
- Recombinant Human DNase (Pulmozyme)
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Nationwide Children's Hospital
Exocrine Pancreatic Insufficiency
Phase 1 Study to Assess Safety and Efficacy of ANG003
Randomized, parallel, active-treatment Phase 1 study of a single dose of orally administered ANG003 with a test meal in adult subjects with cystic fibrosis-related exocrine pancreatic insufficiency.
- Trial name
- NCT06052293
- Testing
- ANG003
- Where
- 20 sites in United States
- Volunteers
- 57 people
- Sponsor
- Anagram Therapeutics, Inc.
Alpha 1-Antitrypsin Deficiency
Study of Fazirsiran (TAK-999, ARO-AAT) in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)
The purpose of this study is to evaluate the the safety and efficacy of the investigational product, fazirsiran (TAK-999, ARO-AAT), administered subcutaneously to patients with alpha-1 antitrypsin deficiency associated liver…
- Trial name
- NCT03946449
- Testing
- ARO-AAT
- Where
- 4 sites in Austria, Germany and 1 more
- Volunteers
- 16 people
- Sponsor
- Arrowhead Pharmaceuticals
Solid Tumor
Dose-Esc/Exp RMC4630 & Cobi in Relapsed/Refractory Solid Tumors & RMC4630& Osi in EGFR+ Locally Adv/Meta NSCLC
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic (PK), and pharmacodynamic (PD) profiles of RMC-4630 and cobimetinib in adult participants with relapsed/refractory solid tumors with specific…
- Trial name
- NCT03989115
- Testing
- RMC-4630, Cobimetinib, Drug: Osimertinib
- Where
- 24 sites in United States, South Korea
- Volunteers
- 113 people
- Sponsor
- Revolution Medicines, Inc.
Colorectal Cancer, Colon Cancer
CB-839 + Capecitabine in Solid Tumors and Fluoropyrimidine Resistant PIK3CA Mutant Colorectal Cancer
This study has two portions. The main goal of the Phase I portion of this research study is to see what doses of CB-839 and capecitabine can safely be given to patients without having too many side effects.
- Conditions
- Colorectal Cancer, Colon Cancer, Rectal Cancer, Solid Tumor
- Trial name
- NCT02861300
- Testing
- CB-839, Capecitabine
- Where
- 2 sites in United States
- Volunteers
- 50 people
- Sponsor
- David Bajor, MD