Clinical trials: Functional Deformities
Abnormality, Respiratory System, Autonomic Nervous System
Effects of Deployment Exposures on Cardiopulmonary and Autonomic Function
Following deployment to Operations Enduring and Iraqi Freedom and Operation New Dawn (OEF/OIF/OND), Veterans report breathing problems, difficulty exercising and other symptoms.
- Trial name
- AirHzds (NCT01754922)
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- VA Office of Research and Development
Cystic Fibrosis
A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis and F/MF Genotypes
This study will evaluate the efficacy and safety of elexacaftor (ELX) / tezacaftor (TEZ) / ivacaftor (IVA) triple combination (TC) in subjects 6 through 11 years of age with cystic fibrosis (CF) who are heterozygous for F508del…
- Trial name
- NCT04353817
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 34 sites in Australia, Canada and 8 more
- Volunteers
- 121 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Growth Hormone Deficiency, Panhypopituitarism
Growth Hormone and Endothelial Function in Children
Objective: This study is designed to determine whether growth hormone treatment in children 8 to 18 years of age alters function of the lining of the arteries.
- Conditions
- Growth Hormone Deficiency, Panhypopituitarism, Short Stature
- Trial name
- NCT00373386
- Testing
- growth hormone
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Ohio State University
Cystic Fibrosis
A Phase 3 Study to Evaluate the Efficacy and Safety of Ivacaftor and VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Mutation
The purpose of this study is to evaluate the efficacy and safety of VX-661 in combination with ivacaftor (IVA, VX-770) and IVA monotherapy in participants with Cystic Fibrosis (CF) who are heterozygous for F508del-CFTR allele and…
- Trial name
- NCT02392234
- Testing
- VX-661/Ivacaftor, Ivacaftor
- Where
- 93 sites in United States, Australia and 9 more
- Volunteers
- 248 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Huntington Disease
PRidopidine's Outcome On Function in Huntington Disease, PROOF- HD
This study will evaluate the efficacy and safety of pridopidine 45mg twice daily (BID) in patients with early stage manifest Huntington Disease (HD).
- Trial name
- NCT04556656
- Testing
- Pridopidine
- Where
- 58 sites in United States, Austria and 9 more
- Volunteers
- 499 people
- Sponsor
- Prilenia
Sickle Cell Disease (SCD)
Study Exploring the Effect of Crizanlizumab on Kidney Function in Patients With Chronic Kidney Disease Caused by Sickle Cell Disease
The goal of the study was to evaluate descriptively the effect of crizanlizumab + standard of care and standard of care alone on renal function in sickle cell disease patients ≥ 16 years with chronic kidney disease due to sickle…
- Trial name
- STEADFAST (NCT04053764)
- Testing
- Crizanlizuamb, Standard of Care
- Where
- 24 sites in United States, Brazil and 9 more
- Volunteers
- 58 people
- Sponsor
- Novartis Pharmaceuticals
Cystic Fibrosis
Cystic Fibrosis and Endothelial Function: At Rest and During Exercise
Perhaps one of the most disturbing aspects of Cystic Fibrosis (CF) is the associated premature death. Oxidative stress has been observed in patients with CF and exercise intolerance has been shown to predict mortality in patients…
- Trial name
- NCT01772758
- Testing
- BH4 5mg, BH4 20mg, Antioxidant Cocktail
- Where
- United States
- Volunteers
- 64 people, healthy volunteers welcome
- Sponsor
- Augusta University
Cystic Fibrosis
A Phase 3 Study of VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)
This study will evaluate the efficacy, safety and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for…
- Trial name
- NCT04058353
- Testing
- ELX/TEZ/IVA, IVA, TEZ/IVA
- Where
- 93 sites in United States, Australia and 11 more
- Volunteers
- 271 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Sickle Cell Disease
Gerofit Exercise Intervention for Older Adults With Sickle Cell Disease (SICKLE-FIT Study)
The purpose of this study to assess the feasibility, acceptability, and safety of a personalized exercise training program adapted from Gerofit to improve physical health and quality of life for adults with SCD
- Trial name
- SICKLE-FIT (NCT05714098)
- Testing
- Gerofit Exercise Program
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- Duke University
Sickle Cell Disease
Assessment of Algorithm-Based Hydroxyurea Dosing on Fetal Hemoglobin Response, Acute Complications, and Organ Function in People With Sickle Cell Disease
Background: Sickle cell disease (SCD) is a blood disease. The drug hydroxyurea (HU) is approved to prevent pain crises in people with SCD. Researchers want to see how higher doses of HU affect the blood.
- Trial name
- NCT02225132
- Testing
- Hydroxyurea
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Migraine With Aura, Patent Foramen Ovale
Comorbidities Associated With Migraine and Patent Foramen Ovale (CAMP)
The purpose of the study is to compare the rate of comorbidities associated with migraine aura (MA) between persons who have a large circulatory right-to-left shunt (RLS) and those who do not have RLS.
- Trial name
- CAMP (NCT01257880)
- Where
- 2 sites in United States
- Volunteers
- 31 people
- Sponsor
- Swedish Medical Center
Atopic Eczema/Dermatitis (Non-Specific)
Skin bioMARkers for Atopic Eczema Therapy Evaluation
The study aims to investigate two new non-invasive technologies for assessing skin properties to identify and validate a range of safety biomarkers that may be considered useful as primary outcome measures for evaluating the…
- Trial name
- SMART (NCT04194814)
- Testing
- crisaborole (2%) ointment, betamethasone valerate 0.1% cream
- Where
- United Kingdom
- Volunteers
- 37 people
- Sponsor
- Sheffield Teaching Hospitals NHS Foundation Trust
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
Hypophosphatasia, Bone Disease
Non-interventional Substudy of ALX-HPP-502 to Assess Natural History of Patients With Juvenile-onset HPP Who Served as Historical Controls in ENB-006-09
The purpose of this study is to characterize the natural history of HPP in patients with Juvenile-onset HPP who served as historical controls in ENB-006-09.
- Trial name
- NCT02235493
- Testing
- Retrospective Case Only
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
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
Constipation-predominant Irritable Bowel Syndrome
Cheno Effect on Transit in Health and IBS-C
The study hypothesis is that the naturally occurring bile acid, chenodeoxycholic acid, induces acceleration of colonic transit in health and in patients with constipation-predominant Irritable Bowel Syndrome (IBS-C).
- Trial name
- Chenotransit (NCT00912301)
- Testing
- Sodium chenodeoxycholate (NaCDC)
- Where
- United States
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Mayo Clinic
Muscular Dystrophy, Duchenne, Ambulatory Care
Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)
The aim of this Phase III study was to assess the efficacy of idebenone on pulmonary function, motor function, muscle strength and quality of life in patients with DMD.
- Trial name
- DELOS (NCT01027884)
- Testing
- Idebenone
- Where
- 23 sites in United States, Austria and 8 more
- Volunteers
- 65 people
- Sponsor
- Santhera Pharmaceuticals
Lennox-Gastaut Syndrome, Dravet Syndrome
The Effects of Cannabidiol (CBD) on Electrical and Autonomic Cardiac Function in Children With Severe Epilepsy
The investigators propose to study the effects of cannabidiol (CBD) on cardiac electrical function and the autonomic nervous system in children with Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS), when the CBD is…
- Trial name
- CBD1 (NCT02815540)
- Testing
- 12-Lead ECG, Cannabidiol
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Gillette Children's Specialty Healthcare
Cystic Fibrosis Related Diabetes, Pancreatic Insufficiency
Pilot and Feasibility Study for the Treatment of Pre-diabetes in Patients With Cystic Fibrosis
The purpose of this study is to provide the necessary data and experience to design a larger, full scale clinical trial to determine if a certain medicine (repaglinide), which increases the amount of insulin secreted by the…
- Trial name
- NCT00763412
- Testing
- repaglinide
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Arbelaez, Ana Maria
Fabry Disease
Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease
The purpose of this study is to compare the safety and effectiveness of various doses of Replagal in patients with cardiomyopathy due to Fabry disease.
- Trial name
- NCT00864851
- Testing
- Replagal
- Where
- 12 sites in United States, Australia and 6 more
- Volunteers
- 44 people
- Sponsor
- Shire