Clinical trials: Functional Deformity
Charcot-Marie-Tooth Disease
Observational Study of Neuromuscular Function in CMT Type 1&2 and Healthy Controls
The primary aim of this study is to discover whether patients with CMT type 1 and 2 have neuromuscular junction transmission deficits.
- Trial name
- ESTABLISH (NCT04980807)
- Where
- 2 sites in United States, Denmark
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- NMD Pharma A/S
Leber Congenital Amaurosis (LCA), Retinitis Pigmentosa (RP)
Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT
To evaluate the natural history of visual function in subjects with IRD phenotypically diagnosed as Leber congenital amaurosis (LCA) or retinitis pigmentosa (RP) caused by RPE65 or LRAT gene mutations.
- Trial name
- NCT02575430
- Testing
- No treatment: retrospective chart review
- Where
- 9 sites in United States, Canada and 5 more
- Volunteers
- 59 people
- Sponsor
- QLT Inc.
Abdominal Pain, Functional Abdominal Pain
Epidemiological Analysis for Hereditary Angioedema Disease
An international, multicenter, epidemiological, observational study investigating the prevalence of Hereditary Angioedema (HAE) disease among participants with recurrent episodes of abdominal pain of no obvious etiology.
- Trial name
- EHA (NCT03558009)
- Where
- 35 sites in Germany, Italy and 4 more
- Volunteers
- 2,318 people
- Sponsor
- CENTOGENE GmbH Rostock
Hypertension, Hyperhomocysteinemia
CSPPT- Chronic Kidney Diseases Study
The purpose of this trial is to confirm that enalapril maleate and folic acid tablets is more effective in preventing renal function decline among the patients with primary hypertension when compared to enalapril maleate.
- Trial name
- CSPPT-CKD (NCT01871740)
- Testing
- Enalapril maleate and folic acid tablets, Enalapril maleate
- Where
- 2 sites in China
- Sponsor
- Shenzhen Ausa Pharmed Co.,Ltd
Pulmonary Hypertension
Phase 2 Study of Ambrisentan for Liver Function Test Rescue in Pulmonary Arterial Hypertension
This Phase 2 study was to determine the incidence of increased serum aminotransferase concentrations (alanine aminotransferase [ALT] and/or aspartate aminotransferase [AST]), as well as the overall safety and tolerability of…
- Trial name
- NCT00423592
- Testing
- ambrisentan
- Volunteers
- 36 people
- Sponsor
- Gilead Sciences
Functional Gastrointestinal Disorders
Evolution of Functional Gastrointestinal Disorders in Infants Fed With a New Infant Formula
This study aims to assess the evolution of functional gastrointestinal disorders (FGIDs) in infants fed with a new infant formula, using the Gastrointestinal (GI) and gastroesophageal reflux (GER) (GIGER) scale through an…
- Trial name
- NCT06488274
- Testing
- New infant formula
- Where
- 4 sites in France
- Volunteers
- 139 people
- Sponsor
- United Pharmaceuticals
Hypochondroplasia
Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia
Phase 2, multicenter, OLE study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR (fibroblast growth factor receptor) 1-3-selective tyrosine kinase inhibitor, in participants with…
- Trial name
- NCT07393373
- Testing
- Infigratinib
- Where
- 24 sites in United States, Australia and 8 more
- Volunteers
- 135 people
- Sponsor
- QED Therapeutics, a BridgeBio company