Clinical trials: Functional Status
- Condition Functional Status
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Gamma-sarcoglycanopathy
Natural History Study in Patients With LGMDR5/2c
Prospective, longitudinal, interventional, multicenter natural history study to better understand the LGMDR5/2c disease physiopathology. The duration of participation for each patient will be up to 24 months.
- Trial name
- NCT06210672
- Where
- 3 sites in France, Tunisia
- Sponsor
- Atamyo Therapeutics
Centronuclear Myopathy
Natural History Study of Patients With Centronuclear Myopathies
This is a prospective, longitudinal study of the natural disease course intended to recruit approximately 60 patients with centronuclear myopathies (CNM) in Europe and the United States.
- Trial name
- NatHis-CNM (NCT04977648)
- Sponsor
- Dynacure
Diarrhea, Upper Respiratory Infections
Children Immune Functions
The purpose of the planned study is to investigate the effect of heat-treated lactobacilli on diarrhea outcome in healthy children 1-4 years old in a randomized, double-blind, placebo-controlled study.
- Trial name
- NCT01717534
- Where
- 2 sites in Mexico
- Volunteers
- 374 people, healthy volunteers welcome
- Sponsor
- Société des Produits Nestlé (SPN)
Infant Growth
Growth and Health Efficacy of Novel Infant Formula : A Randomized Trial
Randomized, controlled study consisting of three parallel arms to investigate the effects of novel infant formula on growth, gut health, immune function, and sleep status in healthy infants and young children aged 12-18 months.
- Trial name
- NCT07534735
- Testing
- Investigational Formula 1, Investigational Formula 2
- Where
- China
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Biostime (Changsha) Nutrition Foods Limited
Lafora Disease
Natural History and Functional Status Study of Patients With Lafora Disease
A natural history and functional status study to characterize the clinical disease course in Lafora disease patients using standardized, quantitative evaluations and to identify useful biomarkers and clinical outcome measures for…
- Trial name
- NCT03876522
- Where
- 4 sites in United States, Italy and 1 more
- Volunteers
- 33 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Myotubular Myopathy
Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM)
This is a prospective, non-interventional, longitudinal study of the natural history and function of approximately 60 patients with MTM from the United States, Canada and Europe.
- Trial name
- MTM (NCT02057705)
- Where
- 13 sites in United States, Belgium and 5 more
- Volunteers
- 48 people
- Sponsor
- Valerion Therapeutics, LLC
Congenital Bleeding Disorder, Haemophilia A With Inhibitors
A Long-term Assessment of Physical Activity, Range of Motion, and Functional Status Following Elective Orthopedic Surgery in Hemophilia Patients With Inhibitors
This study is conducted in the United States of America (USA). The aim of this study is to assess the long-term (5+ years) postoperative functional outcomes of elective orthopaedic surgery (EOS) patients from previously reported…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- Trial name
- NCT01830712
- Testing
- eptacog alfa (activated)
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Novo Nordisk A/S