Clinical trials: Inherited Neuropathies
Heart Transplantation, Dilated Cardiomyopathy (DCM)
Multimodal Analysis of Endomyocardial Biopsies
The goal of this observational study is to pursue a multimodal approach to identify the molecular signatures and immune signalling molecules of various myocardial diseases and thereby contribute to improving diagnosis and…
- Conditions
- Heart Transplantation, Dilated Cardiomyopathy (DCM), Hypertrophic Cardiomyopathy (HCM), Myocarditis, Pericarditis
- Trial name
- NCT07359690
- Where
- Germany
- Volunteers
- 216 people
- Sponsor
- University Hospital, Essen
Burns
Determination of Nerve Growth Factor (NGF) Expression in Pediatric Patients With Severe Burns: a Pilot Study
The goal of this observational study is to determine the blood values of NGF in severely burned patients, to describe any changes over time and finally to correlate the expression of NGF to the severity of the burn.
- Trial name
- NCT05751174
- Testing
- pediatric patients affected by severe burns
- Where
- Italy
- Volunteers
- 12 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Leber Hereditary Optic Neuropathy
Safety and Efficacy Study of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy (LHON)
This study is meant to assess the safety and efficacy of rAAV2-ND4 treatment of Leber hereditary optic neuropathy with 11778 LHON mutation.
- Trial name
- rAAV2-ND4 (NCT01267422)
- Testing
- rAAV2-ND4
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Bin Li
Diabetes Mellitus, Type 2, Diabetes Mellitus, Type 1
Prevalence and Risk Evaluation of Diabetic Complications of the Foot in A Large Canadian Population
The study aims to assess foot complications among patients with diabetes in Canada, using patient data collected during diabetes foot assessments performed by the LMC Chiropody Team between February 27, 2018 and April 17, 2019.
- Conditions
- Diabetes Mellitus, Type 2, Diabetes Mellitus, Type 1, Foot Deformities, Onychauxis
- Trial name
- PEDAL (NCT04327622)
- Where
- Canada
- Volunteers
- 5,084 people
- Sponsor
- LMC Diabetes & Endocrinology Ltd.
Aortic Stenosis Symptomatic, Amyloidosis Cardiac
ATTR Amyloidosis in Elderly Patients With Aortic Stenosis
Severe aortic stenosis is defined with a mean transvalvular pressure gradient (MTPG) > 40mmHg and a calculated aortic valve area of < 1cm2.
- Trial name
- NCT04061213
- Testing
- Tc-99m-DPD scintigraphy
- Where
- Switzerland
- Volunteers
- 489 people
- Sponsor
- Insel Gruppe AG, University Hospital Bern
Glenohumeral Subluxation, Neonatal Brachial Plexus Palsy
Investigation of the Relationship Between Shoulder Alpha Angle and Humeral Head Displacement Percentage Measured by Ultrasonography in Neonatal Brachial Plexus Palsy Infants With Clinical Parameters
This study will examine infants with a birth-related nerve injury called neonatal brachial plexus palsy, which can affect movement in the arm and sometimes cause the shoulder joint to shift out of its normal position…
- Conditions
- Glenohumeral Subluxation, Neonatal Brachial Plexus Palsy, Brachial Plexus Injuries
- Trial name
- NCT07768904
- Testing
- Shoulder Ultrasonography, Standardized Clinical Examination
- Where
- Turkey (Türkiye)
- Volunteers
- 19 people
- Sponsor
- Fatih Sultan Mehmet Training and Research Hospital
Safety Issues, Efficacy, Self
Trial of Befizal® 200 mg for the Treatment of Leber Hereditary Optic Neuropathy
Study of the efficiency of Béfizal® 200 mg in 14 adult patients with a LHON that occurred for less than 5 years. Patient must have certain specific mutations
- Trial name
- Béfinohl (NCT04561466)
- Testing
- Béfizal
- Where
- France
- Volunteers
- 14 people
- Sponsor
- Hôpital Necker-Enfants Malades
Advanced Solid Tumor
A Study to Evaluate ARV-6723 Alone and With Pembrolizumab in Participants With Advanced Solid Tumors
This is a study to evaluate the safety and potential anti-tumor activity of an investigational drug called ARV-6723, in participants with advanced solid tumors.
- Trial name
- NCT07749586
- Testing
- ARV-6723, Pembrolizumab, SOC
- Where
- 4 sites in United States
- Volunteers
- 499 people
- Sponsor
- Arvinas Inc.
Heart Failure NYHA Class II, Heart Failure NYHA Class III
CMR T1 Mapping for Diagnosis of Cardiac Amyloidosis
The study aims to test the diagnostic accuracy of T1 mapping for the diagnosis of cardiac amyloidosis prospectively. The hypothesis is that T1 mapping in older patients with symptomatic heart failure, increased LV wall thickness…
- Conditions
- Heart Failure NYHA Class II, Heart Failure NYHA Class III, Heart Failure NYHA Class IV, Heart Failure With Preserved Ejection Fraction
- Trial name
- CMR for CA (NCT04862273)
- Testing
- Native T1 CMR, Web-based ATTR probability estimator (Pfizer, New York), 99mTc-DPD scintigraphy
- Where
- Germany
- Volunteers
- 112 people
- Sponsor
- University of Leipzig
Cardiac Amyloidosis
AI Echocardiographic Screening of Cardiac Amyloidosis
Recent advances in machine learning and image processing techniques have shown that machine learning models can identify features unrecognized by human experts and accurately assess common measurements made in clinical practice.
- Trial name
- NCT06664866
- Testing
- EchoNet-LVH Assessment
- Where
- 4 sites in United States
- Volunteers
- 500 people
- Sponsor
- Cedars-Sinai Medical Center
Amyloid Cardiomyopathy
Unmasking the Prevalence of AC in an Unselected Echocardiographic Population
This study will investigate the prevalence of echocardiographic red-flags of amyloid cardiomyopathy (AC) in patients undergoing clinically-indicated echocardiography (observational phase) and the prevalence of AC among…
- Trial name
- AC-TIVE (NCT04738266)
- Testing
- Amyloid Cardiomyopathy
- Where
- 16 sites in Italy
- Volunteers
- 381 people
- Sponsor
- University of Trieste
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Efficacy and Safety of AG10 in Subjects with Transthyretin Amyloid Polyneurophathy
See updated study design under NCT04882735. Phase 3 efficacy and safety of AG10 compared with placebo in subjects with symptomatic Transthyretin Amyloid Polyneuropathy (ATTR-PN)
- Trial name
- ATTRibute-PN (NCT04418024)
- Testing
- AG10
- Where
- United States
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Charcot-Marie-Tooth Disease, Type IA
Biomarkers and Validation of Selected Outcome Measures (CMTBiomarker)
CMT is a rare disease for which novel treatments are being developed. Evaluation of intervention efficacy is hampered by slow progression and lack of sensitive outcome measures.
- Trial name
- NCT03386266
- Where
- Germany
- Volunteers
- 156 people
- Sponsor
- University Medical Center Goettingen
Arthritis, Osteoarthritis
Tanezumab In Osteoarthritis Of The Knee (2)
The purpose of this study is to test the efficacy and safety of 2 doses of tanezumab compared with naproxen and placebo in patients with osteoarthritis.
- Trial name
- NCT00830063
- Testing
- tanezumab 10 mg, tanezumab 5 mg, naproxen
- Where
- 94 sites in United States
- Volunteers
- 832 people
- Sponsor
- Pfizer
Piriformis Syndrome, Pes Planus
Investigation of the Relationship Between Pes Planus and Piriformis Syndrome: A Case-Control Study
The aim of this study is to investigate the relationship between Pes Planus and Piriformis Syndrome. The study will be conducted on individuals aged 18-65 years who are native Turkish speakers, have Turkish reading and writing…
- Trial name
- Piriformis (NCT07674433)
- Where
- Turkey (Türkiye)
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Kahramanmaras Sutcu Imam University
Charcot-Marie-Tooth Disease Type 1A
Clinical Trial Evaluating the Impact of an Intensive Rehabilitation Program Combined With Tendon Vibratory Stimulation on Functional Balance in Individuals With Charcot-Marie-Tooth Disease Type 1A
Charcot-Marie-Tooth (CMT) disease is caused by a genetic abnormality involving the PMP22 gene, resulting in demyelination of the peripheral nerves.
- Trial name
- EQUIVIB-CMT (NCT07726043)
- Testing
- Multidisciplinary rehabilitation program
- Where
- France
- Volunteers
- 30 people
- Sponsor
- UGECAM PACA et Corse
Leber's Hereditary Optic Neuropathy (LHON)
Evaluation of Impact of Disease and Visual Disability on Quality of Life and Loss of Independence of Patients Living in France With Leber's Hereditary Optic Neuropathy (LHON) Through Qualitative and Quantitative Data Collection
Leber's hereditary optic neuropathy (LHON) is a rare mitochondrial genetic disorder characterized by optic nerve atrophy due to the degeneration of retinal ganglion cells, which leads to acute visual loss.
- Trial name
- NCT05555784
- Where
- France
- Volunteers
- 12 people
- Sponsor
- Argo Sante
Amyloidosis; Systemic, Amyloidosis Cardiac
Essen Amyloidosis Registry
The Essen Amyloidosis Registry (EAR) is a prospective, observational registry designed to collect comprehensive clinical data on patients diagnosed with systemic amyloidosis.
- Trial name
- EAR (NCT06887283)
- Where
- Germany
- Volunteers
- 400 people, healthy volunteers welcome
- Sponsor
- University Hospital, Essen
Neuromuscular Diseases, Obesity (Disorder)
Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology
The ActiLiège-Adult study is a prospective, longitudinal, observational study designed to collect natural history data on adult patients with neurological or metabolic diseases affecting movement.
- Conditions
- Neuromuscular Diseases, Obesity (Disorder), Myotonic Dystrophy 1, Myasthenic Syndrome
- Trial name
- Acti-Adult (NCT07136844)
- Testing
- Syde, Dynamometric measurements of muscle strength, The 6-minute walk test (6MWT)
- Where
- Belgium
- Volunteers
- 300 people
- Sponsor
- Centre Hospitalier Universitaire de Liege
Cardiac Amyloidosis
Identification of Prognostic Parameters in Patients With " Senile " Amyloid Cardiomyopathy
The aging of the population is a reality in our society, with a strong increase in the number of elderly patients hospitalized for heart failure in our institutions.
- Trial name
- EVAMYLOSE (NCT04225754)
- Where
- France
- Volunteers
- 142 people
- Sponsor
- University Hospital, Grenoble