Clinical trials: Gastrointestinal System--Abnormalities
Wilson Disease, Wilson's Disease
A Study of the Safety and Efficacy of Prime Editing (PM577) in Participants With Wilson Disease (WD)
The purpose of this study is to evaluate the safety, tolerability, biological activity, and initial efficacy of PM577a, an investigational Prime Editing therapy, in adults and adolescents with Wilson disease (WD).
- Conditions
- Wilson Disease, Wilson's Disease, Wilsons Disease
- Trial name
- PM577a (NCT07748403)
- Testing
- PM577a
- Where
- 4 sites in United States, New Zealand
- Volunteers
- 42 people
- Sponsor
- Prime Medicine, Inc.
Polycystic Liver Disease, Hepatomegaly
Open-Label Extension of LOCKCYST Trial
Positive data originating from two polycystic liver patients treated with somatostatin analogues, showed a volume reduction of 38.3% and 14.9%.
- Conditions
- Polycystic Liver Disease, Hepatomegaly, Liver Diseases, Polycystic Kidney
- Trial name
- LOCKCYST-ext (NCT00771888)
- Testing
- lanreotide
- Where
- 2 sites in Belgium, Netherlands
- Volunteers
- 54 people
- Sponsor
- Radboud University Medical Center
Cystic Fibrosis
Personalized Theratyping Trial
The purpose of this study is to explore the use of off-label CFTR modulators that may affect CFTR function in patients with CFTR mutations that are not currently approved for these drugs.
- Trial name
- NCT03587961
- Testing
- Symdeko
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- George Solomon
Cancer
MS-275 in Treating Patients With Advanced Solid Tumors or Lymphoma
RATIONALE: MS-275 may stop the growth of cancer cells by blocking the enzymes necessary for their growth. PURPOSE: This phase I trial is studying the side effects and best dose of MS-275 in treating patients with advanced solid…
- Trial name
- NCT00020579
- Testing
- entinostat
- Where
- 3 sites in United States
- Volunteers
- 75 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
Cystic Fibrosis, Respiratory Infection
Sputum Validation for the Molecular Diagnosis of Respiratory Viral Infections in Cystic Fibrosis
The aim of this pilot study is to demonstrate the feasibility of viral biomolecular diagnosis in sputum compared to nasopharyngeal swab in cystic fibrosis acute respiratory infection.
- Trial name
- EXPIRE (NCT03684759)
- Where
- 3 sites in France
- Volunteers
- 30 people
- Sponsor
- University Hospital, Brest
Obscure Gastrointestinal Bleeding, Angiodysplasia
Long-term Effects of Thalidomide for Recurrent Gastrointestinal Bleeding Due to Vascular Malformation
Background: Repeated episodes of bleeding from gastrointestinal vascular malformations refractory to endoscopic or surgical therapy often pose a major therapeutic challenge.
- Conditions
- Obscure Gastrointestinal Bleeding, Angiodysplasia, Gastric Antral Vascular Ectasia, Thalidomide
- Trial name
- NCT00964496
- Testing
- Thalidomide, Iron
- Volunteers
- 55 people
- Sponsor
- Shanghai Jiao Tong University School of Medicine
Cystic Fibrosis and Non CF Bronchiectasis
The Role of Vitamin D3 in Pediatric Bronchiectasis Severity
Vitamin D3 therapy was effective in decreasing the frequency of pulmonary exacerbations and preserving lung functions , thereby improving the disease severity even more in non CF than CF bronchiectasis patients
- Trial name
- NCT04411901
- Testing
- Cholecalciferol (vitaminD3)
- Where
- Egypt
- Volunteers
- 40 people
- Sponsor
- Heba Omara
Cystic Fibrosis
Electro Flo 5000 and Vest Therapy
It is the goal of the proposed study to compare the efficacy, as assessed primarily by sputum weight, of these two different devices (the Electroflo 500 and VEST therapy) for airway clearance (AC) in CF patients with mild to…
- Trial name
- EF/Vest (NCT02277626)
- Testing
- Electro Flo 5000, Incourage Vest System
- Volunteers
- 15 people
- Sponsor
- Stanford University
Anorectal Malformations, Transition
Transition of Care for Patients With Hirschsprung Disease and Anorectal Malformations
Transition from paediatric to adult health care is crucial for preventing deterioration of chronic diseases. At present, transitional care (TC) is not established for patients with the Hirschsprung disease (H) and Anorectal…
- Conditions
- Anorectal Malformations, Transition, Quality of Life, Hirschsprung Disease
- Trial name
- NOHARM (NCT04106947)
- Testing
- Questionnaires
- Where
- Norway
- Volunteers
- 1,000 people
- Sponsor
- Oslo University Hospital
Cystic Fibrosis
Host and Bacterial Mechanisms During Cystic Fibrosis Pulmonary Exacerbations
Cystic fibrosis pulmonary exacerbations (CF PEx) vary greatly in their severity, their pathogens, and their treatment responses.
- Trial name
- NCT04354038
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- National Jewish Health
Cystic Fibrosis
Clinical and Genetic Profile of Pediatric Patients With Cystic Fibrosis in Sohag.
Cystic fibrosis (CF) is an autosomal recessive genetic disorder caused by mutations in the gene encoding CF transmembrane conductance regulator (CFTR), which is located at 7q31.2 and encodes 1480 amino acids.
- Trial name
- NCT05599958
- Testing
- sweat chloride test, genetic testing
- Where
- Egypt
- Volunteers
- 152 people
- Sponsor
- Sohag University
Cystic Fibrosis
Energy Balance and Weight Gain With Ivacaftor Treatment
Ivacaftor is a novel, FDA approved new therapy that addresses Cystic fibrosis transmembrane conductance regulator (CFTR) dysfunctions in subjects with Cystic fibrosis (CF) and "gating mutations".
- Trial name
- NCT02141464
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Children's Hospital of Philadelphia
Cystic Fibrosis
A Decentralized Study to Evaluate Physical Activity and Cough Frequency Using Wearable Technology in Cystic Fibrosis
This study will evaluate the performance of wearable technology in cystic fibrosis (CF) participants taking commercial Elexacaftor (ELX)/Tezacaftor (TEZ)/Ivacaftor (IVA) utilizing a fully decentralized trial design.
- Trial name
- NCT04923464
- Where
- 17 sites in United States, United Kingdom
- Volunteers
- 51 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Transthyretin (TTR) Amyloid Cardiomyopathy, Cardiac Amyloidosis
Liver and Coagulation Disorders in Cardiac Transthyretin Amyloidosis
Transthyretin cardiac amyloidosis (ATTR-CA) is a progressive infiltrative cardiomyopathy caused by the deposition of misfolded transthyretin protein within the myocardium.
- Conditions
- Transthyretin (TTR) Amyloid Cardiomyopathy, Cardiac Amyloidosis, Cardiomyopathies, Wild-Type Transthyretin Cardiac Amyloidosis
- Trial name
- LICA2025 (NCT07766135)
- Where
- Italy
- Volunteers
- 70 people
- Sponsor
- University of Messina
Cystic Fibrosis, Diabetes
The Effect of Combination Lumacaftor and Ivacaftor on Markers of Hyperglycemia
The purpose of this research study is to find out if the combined therapy lumacaftor-ivacaftor affects glycemia in patient with cystic fibrosis.
- Trial name
- NCT02858843
- Testing
- lumacaftor-ivacaftor
- Volunteers
- 1 people
- Sponsor
- Massachusetts General Hospital
Alpha-1 Antitrypsin Deficiency, Emphysema
Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin Deficiency
The aim of this study is to test whether aspirin improves endothelial function in alpha-1 antitrypsin deficiency-associated lung disease, measured by pulmonary microvascular blood flow on magnetic resonance imaging (MRI) and with…
- Conditions
- Alpha-1 Antitrypsin Deficiency, Emphysema, Chronic Obstructive Pulmonary Disease
- Trial name
- NCT03008915
- Testing
- Aspirin, Withdrawal from alpha1 antitrypsin replacement therapy
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Columbia University
Cystic Fibrosis (CF), Gene Therapy
A Prospective, Single-center, Open-label Study of Inhaled BMD003 (CFTR mRNA) in Chinese Adults With Cystic Fibrosis
The study uses a prospective, single-center, open-label clinical trial design to evaluate the safety and efficacy of inhaled BMD003 (CFTR mRNA) in adult Chinese patients with cystic fibrosis.
- Trial name
- NCT06963008
- Testing
- inhaled BMD003(CFTR mRNA)
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Peking Union Medical College Hospital
Cystic Fibrosis
Study to Evaluate the Effect of KB001-A on Time-to-Need for Antibiotic Treatment
The purpose of this study is to confirm and extend the Phase 1-2 KB001 findings of an airway anti-inflammatory effect in CF individuals with chronic Pseudomonas aeruginosa (Pa) airway infection.
- Trial name
- KB001-A (NCT01695343)
- Testing
- KB001-A
- Where
- 67 sites in United States, Australia and 2 more
- Volunteers
- 182 people
- Sponsor
- Humanigen, Inc.
Erythropoietic Protoporphyria
Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP)
This is a randomized placebo-controlled study to be conducted in two parallel study arms for a six month period (three doses). Approximately 10 eligible patients per center will be enrolled and will receive afamelanotide (16 mg…
- Trial name
- NCT01097044
- Testing
- Afamelanotide
- Where
- 6 sites in United States
- Volunteers
- 77 people
- Sponsor
- Clinuvel Pharmaceuticals Limited
Biliary Atresia
Biliary Atresia, Hepatic Buffer Response and Sevoflurane
To evaluate the effects of sevoflurane on hepatic blood flow (HBF) and hepatic arterial buffer response (HABR) in infants with obstructive jaundice by Doppler ultrasound.Twenty-five infants with biliary atresia (1-3…
- Trial name
- NCT02471209
- Testing
- Sevoflurane
- Volunteers
- 25 people
- Sponsor
- Children's Hospital of Fudan University