Clinical trials: Skeletal Abnormalities
Congenital Heart Disease, Single Ventricle
Cardiovascular, Pulmonary and Skeletal Muscle Evaluation Postoperative in Fontan Patients: Effects of Exercise Training
Introduction: The Fontan operation is the surgical treatment in most patients with either anatomic or functional single ventricles.
- Conditions
- Congenital Heart Disease, Single Ventricle, Univentricular Heart
- Trial name
- NCT02283255
- Testing
- Aerobic Exercise Training, Inspiratory Muscle Training, No Exercise Training
- Where
- Brazil
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo
Dentofacial Abnormalities, Dentofacial Deformities
Orthognathic Speech Pathology: Phonetic Contrasts of Patients With Dental Discrepancies Pre- and Post-Treatment Analyses
The investigators are studying how speech is effected by jaw and tooth position in jaw surgery patients. Eighty percent of our jaw surgery patients have speech pathologies, compared to five percent of the general population, but…
- Conditions
- Dentofacial Abnormalities, Dentofacial Deformities, Dentofacial Anomalies, Including Malocclusion, Malocclusion
- Trial name
- OSP (NCT04117360)
- Testing
- Orthognathic surgery and Orthodontics
- Where
- United States
- Volunteers
- 180 people
- Sponsor
- University of North Carolina, Chapel Hill
Pulmonary Artery Hypertension
Skeletal Muscle Mitochondrial Abnormalities and the Metabolic Syndrome in PAH
Pulmonary arterial hypertension (PAH) is characterized by the progressive increase in pulmonary vascular resistance ultimately leading to right ventricular (RV) failure.
- Trial name
- NCT03979482
- Where
- Canada
- Volunteers
- 30 people
- Sponsor
- Laval University
Hereditary Multiple Osteochondromas
Functional and Morphological Characterization of Multiple Osteochondromas Disorder
The main purpose of the study is the characterization of functional and morphological alterations due to multiple osteochondromas in a pediatric and adult population through the identification of relevant anthropometric and…
- Trial name
- MAMBO (NCT06703736)
- Where
- Italy
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Istituto Ortopedico Rizzoli
Chronic Obstructive Pulmonary Disease
Effects of Pulmonary Rehabilitation on Skeletal Muscle in COPD Patients
In a former study, the investigator observed significant differences in the response to pulmonary rehabilitation between COPD patients with the "normal" genetic variant of alpha-1 antitrypsin (PiMM) and those with a homozygous…
- Trial name
- NCT02915614
- Testing
- Pulmonary rehabilitation
- Where
- Germany
- Volunteers
- 40 people
- Sponsor
- Schön Klinik Berchtesgadener Land
Three Dimensional, Dento-Skeletal
Three Dimensional Evaluation of Dento-Skeletal Effects of Leaf Self Expander in Cleft Lip and Palate Patients
This study aims to evaluate the dento-alveolar changes after maxillary expansion in cleft lip and palate patients by Leaf Self Expander, as compared to conventional Rapid Maxillary Expander using digital cast analysis and…
- Conditions
- Three Dimensional, Dento-Skeletal, Leaf Self Expander, Cleft Lip
- Trial name
- NCT07384338
- Testing
- Rapid maxillary expansion, Leaf Self Expander
- Where
- Egypt
- Volunteers
- 20 people
- Sponsor
- Al-Azhar University
Danon Disease
Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B
This is a non-randomized open-label Phase 1 study to evaluate the safety and toxicity of gene therapy using a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2…
- Trial name
- NCT03882437
- Testing
- RP-A501
- Where
- 3 sites in United States
- Volunteers
- 7 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Staphylococcus
Efficacy of a Short-term Sequential Therapy in Non-complicated Catheter Related Bacteremia by Methicillin- Susceptible S.Aureus.
Evaluate the efficacy of a sequential regimen of 14 days in patients with catheter-related bacteremia by S. aureus methicillin-susceptible, selected based on a pre-established clinical and microbiological criteria.
- Trial name
- NCT01875263
- Testing
- Cloxacilin, Cloxacillin, Levofloxacin
- Where
- 11 sites in Spain
- Volunteers
- 1 people
- Sponsor
- Fundación Pública Andaluza Progreso y Salud
Skeletal Class II Malocclusion, Mandibular Retrognathia
Fixed Versus Conventional Twin Block Appliances for Skeletal Class II Malocclusion
This study compares two types of dental appliances a fixed Twin Block appliance and a conventional (removable) Twin Block appliance used to treat a bite problem called skeletal Class II malocclusion in growing children and…
- Trial name
- NCT07687927
- Testing
- Conventional Twin Block Appliance
- Where
- Egypt
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Al-Azhar University
Polycystic Kidney Diseases, Polycystic Liver Disease
The Association Between Skeletal Muscle Mass and Severity of Polycystic Liver Disease and Polycystic Kidney Disease
Primary sarcopenia is used to describe aging and progressed with the physiologic decline. Secondary sarcopenia is associated many chronic disease, including acquired immune deficiency syndrome, cancer, chronic heart failure…
- Conditions
- Polycystic Kidney Diseases, Polycystic Liver Disease, Muscle Loss
- Trial name
- NCT05215964
- Testing
- Renal artery embolization
- Where
- Taiwan
- Volunteers
- 600 people
- Sponsor
- National Taiwan University Hospital
Prader-Willi Syndrome, Sarcopenia
Ultrasound to Assess Sarcopenia in Prader Willi Syndrome
The aim of the study is to examine the clinical applicability of ultrasound as a diagnostic tool for sarcopenia in individuals with PWS by investigating the association between ultrasound-derived measurements, DXA-derived…
- Conditions
- Prader-Willi Syndrome, Sarcopenia, Metabolic Syndrome
- Trial name
- PWS (NCT06448871)
- Testing
- Ultrasound assessment of the right lower extremity muscles, Dual-energy X-ray absorptiometry (DXA) scan
- Where
- Taiwan
- Volunteers
- 49 people
- Sponsor
- Taipei Tzu Chi Hospital, Buddhist Tzu Chi Medical Foundation
Achondroplasia
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
This is a multicenter, multinational, randomized, active-controlled, operationally seamless Phase 2/3 study of BMN 333 in treatment-naïve pediatric participants with achondroplasia (ACH).
- Trial name
- ASPEN (NCT07441876)
- Testing
- BMN 333, Vosoritide Injection [Voxzogo]
- Where
- 28 sites in United States, Australia and 7 more
- Volunteers
- 160 people
- Sponsor
- BioMarin Pharmaceutical
Hypochondroplasia
Efficacy and Safety Evaluation of Recombinant Human Growth Hormone (r-hGH), Saizen®, on a Population of Children With Hypochondroplasia, Treated at Least 3 Years or Until Near Final Height, When Applicable, in Comparison With a Historic Cohort of Non-treated Children
This study is conducted to describe the efficacy and safety of recombinant human growth hormone (r-hGH) treatment Saizen® on children with hypochondroplasia.
- Trial name
- NCT01111019
- Testing
- Recombinant human growth hormone (r-hGH)
- Where
- France
- Volunteers
- 19 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Fontan Circulation
Muscle Function in Patients With Single-chamber Heart
Groundbreaking cardiac surgery has significantly improved survival rates for patients with single-chamber hearts. However, despite these life-saving interventions, the long-term prognosis remains concerning, with an increased…
- Trial name
- Muscle in TCPC (NCT07420660)
- Where
- Sweden
- Volunteers
- 34 people
- Sponsor
- Karolinska Institutet
Achondroplasia
A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia
Approximately 63 participants will be randomized to one of three doses to receive Recifercept either Low Dose Medium Dose High Dose Participants will will attend the clinic at baseline and at Day 1, 4, 8, 15, 29 \& then Month 2…
- Trial name
- NCT04638153
- Testing
- Recifercept
- Where
- 16 sites in United States, Australia and 6 more
- Volunteers
- 60 people
- Sponsor
- Pfizer
Statin Adverse Reaction
Exercise Intensity and Muscle Recovery in Statin Users
This study aims to investigate how different types and intensities of exercise affect muscle recovery in individuals taking statin medications compared to healthy controls.
- Trial name
- STN-EX (NCT07563452)
- Testing
- Dose-Escalation Exercise Protocol
- Where
- Austria
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Raffaele Mazzolari
Cleft Lip and Palate
Transverse Dento-skeletal Effects of Two Rapid Palatal Expansion Appliances in Cleft Lip and Palate Patients
Aim of the study: to compare the effect of Hyrax appliance and the Haas appliance improving the transverse skeletal and dental dimensions in cleft lip and palate patients during mixed dentition.
- Trial name
- NCT04236466
- Testing
- hyrax appliance, haas appliance
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Cairo University
Cleft Lip and Palate, Maxillary Expansion
Effectiveness of Two Maxillary Expanders in Improving the Transverse Skeletal and Dental Dimensions in Patients With Cleft Lip and Palate
This randomized controlled trial is designed to compare between quadlelix appliance vs hyrax expander regrading the their effectiveness in maxillary expansion in patients with cleft.
- Trial name
- NCT04331977
- Testing
- Maxillary Expansion
- Where
- Egypt
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Cairo University
Duchenne Muscular Dystrophy (DMD)
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
The primary objective of this study is to evaluate the safety and tolerability of SPOT-03 administered by intravenous (IV) infusion to DMD patients.
- Trial name
- NCT07188012
- Testing
- SPOT-03
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Shanghai Siponuoyin Biotechnology Co Ltd
Sickle Cell Hemoglobin C Disease, Hemoglobin S Disease
Evaluation of a Training Program for Homozygous Sickle Cell Disease Patients
Sickle cell disease (SCD) is the most frequent inherited disease in the world. Literature reports that SCD patients display intolerance to exercise, important muscle weakness and profound remodeling of skeletal muscle including…
- Trial name
- EXDRE (NCT02571088)
- Testing
- Training Program
- Where
- 9 sites in France
- Volunteers
- 40 people
- Sponsor
- Centre Hospitalier Universitaire de Saint Etienne