Clinical trials: Physical Function
- Condition Physical Function
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Fabry Disease
A Study of Renal Function in Treatment-naïve, Young Male Patients With Fabry Disease
No investigational drug will be administered in this study for the treatment of Fabry disease. This will be a multicenter, multinational, non-treatment, cross-sectional study of young male patients with Fabry disease who have not…
- Trial name
- NCT01839526
- Testing
- Iohexol
- Where
- 22 sites in United States, Argentina and 12 more
- Volunteers
- 39 people
- Sponsor
- Genzyme, a Sanofi Company
Kidney Transplant
Study Comparing Sirolimus With Cyclosporine in a Calcineurin Inhibitor (CNI)-Free Regimen in Kidney Transplant Recipients
The purpose of this study is to determine if one drug is superior to another with regard to safety and the preservation of renal function after a kidney transplant.
- Trial name
- NCT00137345
- Testing
- sirolimus
- Where
- 82 sites in United States, Argentina and 19 more
- Volunteers
- 500 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Diabetes Mellitus, Type 1
A Study of Baricitinib (LY3009104) to Preserve Beta Cell Function in Children and Adults Newly Diagnosed With Type 1 Diabetes (BARICADE-PRESERVE)
The purpose of this study is to find out if baricitinib can preserve beta-cell function in participants newly diagnosed with type 1 diabetes. Participation in the study will last about 60 weeks.
- Trial name
- NCT07222332
- Testing
- Baricitinib
- Where
- 131 sites in United States, Australia and 18 more
- Volunteers
- 300 people
- Sponsor
- Eli Lilly and Company
Duchenne Muscular Dystrophy
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of KER-065 administered to adult and pediatric ambulatory and nonambulatory male participants with…
- Trial name
- NCT07704099
- Testing
- KER-065
- Where
- 2 sites in New Zealand
- Volunteers
- 36 people
- Sponsor
- Keros Therapeutics, Inc.
Spinal Muscular Atrophy
ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)
The primary purpose is to address critical evidence in the treatment landscape for Spinal Muscular Atrophy (SMA), specifically focusing on the intrathecal formulation of onasemnogene abeparvovec-brve (ITVISMA®). U.S.
- Trial name
- NCT07448610
- Testing
- Onasemnogene Abeparvovec-brve
- Where
- 6 sites in United States
- Volunteers
- 36 people
- Sponsor
- Novartis Pharmaceuticals
Impaired Hepatic Function, Healthy
Study of ALXN2050 in Participants With Hepatic Impairment
This study will investigate the impact of impaired hepatic function (IHF) on the plasma pharmacokinetics of ALXN2050 in order to provide dosing recommendations for future indications in individuals with varying degrees of IHF.
- Trial name
- NCT05259085
- Testing
- ALXN2050
- Where
- 2 sites in United States
- Volunteers
- 26 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hypothyroidism
Thyroid Function of Pediatric Subjects Following Isovue® Administration
This is a Phase IV prospective, multicenter, observational study to estimate the proportion of subjects 0 to 3 years of age who develop abnormal thyroid function after exposure to intravascular administration of ISOVUE for the…
- Trial name
- NCT03779906
- Testing
- Isovue
- Where
- 4 sites in United States
- Volunteers
- 17 people
- Sponsor
- Bracco Diagnostics, Inc
Psychotic Disorders
Improving Cognitive Function of Aripiprazole in Treating Adolescents and Young Adults With Psychotic Disorder
This is a 24-week, observational, prospective study to evaluate the effectiveness of improving cognitive function of aripiprazole in treating adolescents and young adults with psychotic disorder in Taiwan.
- Trial name
- NCT00541502
- Sponsor
- Genovate Biotechnology Co., Ltd.,
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
Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD
PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)
The purpose of this Phase 1/2a trial is to evaluate the safety, tolerability, and preliminary efficacy of PBGENE-DMD in patients with DMD harboring mutations amenable to excision of exons 45-55.
- Trial name
- NCT07429240
- Testing
- PBGENE-DMD (IV)
- Where
- 2 sites in United States
- Volunteers
- 18 people
- Sponsor
- Precision BioSciences, Inc.
Cystic Fibrosis in Children
The Effectiveness of the Jamboxx Respiratory Therapy Device: Study 2
The Effectiveness of the Jamboxx Respiratory Therapy Device in Treatment of Patients with Decreased Respiratory Function is a proposal for investigation of the application of gaming to improving respiratory health.
- Trial name
- NCT03522480
- Testing
- Jamboxx Respiratory Therapy Device, Autogenic Draining Training
- Where
- United States
- Sponsor
- My Music Machines Inc.
Fragile X Syndrome
Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X Syndrome
There will be four study periods: Screening (up to 14 days in length), the Treatment Period (8 weeks), the Withdrawal Period (22 days), and Follow-up Period (up to 31 days).
- Trial name
- Harbor-C (NCT01325220)
- Testing
- arbaclofen
- Where
- 28 sites in United States
- Volunteers
- 172 people
- Sponsor
- Seaside Therapeutics, Inc.
Asthma
Efficacy and Safety Study of Mepolizumab Adjunctive Therapy in Participants With Severe Eosinophilic Asthma on Markers of Asthma Control
This is a multi-centre, placebo-controlled, double-blind, parallel-group study to evaluate the efficacy and safety of mepolizumab adjunctive therapy in participants with severe eosinophilic asthma on markers of asthma control.
- Trial name
- NCT02281318
- Testing
- Mepolizumab, SOC
- Where
- 144 sites in United States, Argentina and 17 more
- Volunteers
- 556 people
- Sponsor
- GlaxoSmithKline
Single Ventricle Heart Disease After Fontan Surgery
Pharmacokinetic/Pharmacodynamic Study of Udenafil in Adolescents
To determine the pharmacokinetic profile and safety of udenafil in adolescents with Fontan physiology and to assess the short-term pharmacodynamic effect of udenafil on pharmacodynamic measures of exercise capacity, ventricular…
- Trial name
- NCT02201342
- Testing
- Udenafil
- Where
- 6 sites in United States, Canada
- Volunteers
- 36 people
- Sponsor
- Mezzion Pharma Co. Ltd
Pro-opiomelanocortin (POMC) Deficiency Obesity
Setmelanotide for the Treatment of Early-Onset Pro-Opiomelanocortin (POMC) Deficiency Obesity
To demonstrate statistically significant and clinically meaningful effects of setmelanotide on percent body weight change in participants with pro-opiomelanocortin (POMC) deficiency or proprotein convertase subtilisin/kexin type…
- Trial name
- NCT02896192
- Testing
- Setmelanotide
- Where
- 7 sites in United States, Belgium and 5 more
- Volunteers
- 15 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Cystic Fibrosis
Natural History Study of Exocrine Pancreatic Function in Infants With Cystic Fibrosis (CF)
The purpose of the study is to evaluate the natural history of exocrine pancreatic function by assessing Fecal elastase-1 (FE-1) in infants with CF during their first year of life.
- Trial name
- NCT06506773
- Where
- 50 sites in United States, Australia and 9 more
- Volunteers
- 79 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
POMC Deficiency Obesity, PCSK1 Deficiency Obesity
An Observational,Prospective Natural History Study of Early-Onset Extreme Obesity Due to Bi-Allelic Loss-of-Function Mutations in the POMC, PCSK1 or LEPR Genes
This is an observational study. There are no protocol-defined visits, although patients are expected to have routine office visits approximately every 6 months.
- Conditions
- POMC Deficiency Obesity, PCSK1 Deficiency Obesity, LEPR Deficiency Obesity
- Trial name
- NHS (NCT03621007)
- Where
- 3 sites in Turkey (Türkiye)
- Volunteers
- 8 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Kidney Transplant Failure and Rejection
Assessment of QSant™ for Underlying Allograft Rejection
The AQUA registry is a multi-center observational study to assess the clinical management of kidney transplant recipients (KTRs) with use of the QSant test.
- Trial name
- AQUA (NCT05282966)
- Testing
- QSant-TM Urine Test
- Volunteers
- 4,000 people
- Sponsor
- NephroSant
Healthy
Form, Fit, and Function of INVSENSOR00061
The objective of this study is to evaluate the form, fit, and function of INVSENSOR00061 when used on newborn and infant subjects 0-18 months.
- Trial name
- NCT05926648
- Testing
- INVSENSOR00061
- Where
- United States
- Volunteers
- 53 people, healthy volunteers welcome
- Sponsor
- Masimo Corporation
CHAPLE Disease
A Study to Test the Safety of Pozelimab in Pediatric Participants 1 to 5 Years of Age With a Rare Disease Called CHAPLE (Complement Hyperactivation, Angiopathic Thrombosis, Protein-losing Enteropathy) Disease
This study is researching a drug called pozelimab (called "study drug"). The main aim of this study is to monitor the safety and tolerability of the study drug.
- Trial name
- NCT07142343
- Testing
- Pozelimab
- Where
- Turkey (Türkiye)
- Volunteers
- 5 people
- Sponsor
- Regeneron Pharmaceuticals