Clinical trials: Atrophy
Muscular Dystrophy, Duchenne
The Efficacy and Safety of Metoprolol as add-on Treatment to Standard of Care in Preventing Cardiomyopathy in Patients With DMD
The study includes 150 patients with DMD diagnosis confirmed by genetic testing, 8-16 years old (≥8 and <17) at the study entry with a follow-up of up to 5 years.
- Trial name
- MeDMD (NCT05066633)
- Testing
- Metoprolol Succinate
- Where
- Poland
- Volunteers
- 150 people
- Sponsor
- Medical University of Gdansk
Cerebral Palsy, Arthrogryposis
Effects of Power Mobility on Young Children With Severe Motor Impairments
The purpose this study is to determine the effects of power mobility on the development and function of young children of young children whose severe physical disabilities limit their exploratory behaviors and may unnecessarily…
- Conditions
- Cerebral Palsy, Arthrogryposis, Spinal Muscular Atrophy, Other Central Nervous System or Musculoskeletal Disorders
- Trial name
- NCT01028833
- Testing
- Power mobility
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- University of Oklahoma
Leber Congenital Amaurosis 10, Blindness
A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)
The purpose of this double-masked, randomized, controlled, multiple-dose study is to evaluate the efficacy, safety, tolerability and systemic exposure of sepofarsen (QR-110) administered via intravitreal injection in subjects…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders
- Trial name
- ILLUMINATE (NCT03913143)
- Testing
- sepofarsen
- Where
- 14 sites in United States, Belgium and 7 more
- Volunteers
- 36 people
- Sponsor
- ProQR Therapeutics
Muscular Dystrophies, Muscular Dystrophy
Muscle Tissue Bank for Muscular Dystrophy
This protocol proposes to establish gene expression profiles of muscular dystrophies for correct diagnosis and for development of experimental therapies for these diseases.
- Trial name
- NCT01950897
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- Wake Forest University Health Sciences
Spinal Muscular Atrophy
A Study of CK-2127107 in Patients With Spinal Muscular Atrophy
This study will evaluate the pharmacodynamic (PD) effect of CK-2127107 (hereafter referred to as reldesemtiv) versus placebo on measures of skeletal muscle function or fatigability in patients with Type II, III, or IV spinal…
- Trial name
- NCT02644668
- Testing
- Reldesemtiv 150 mg, Reldesemtiv 450 mg
- Where
- 19 sites in United States, Canada
- Volunteers
- 70 people
- Sponsor
- Cytokinetics
Alveolar Bone Loss, Periodontitis
Intraoral Ultrasound Imaging of Tooth-periodontium Complex
Misaligned teeth are very common in the population and are commonly known as malocclusion. This happens when the upper teeth do not align properly with the lower teeth.
- Trial name
- NCT04971642
- Testing
- Intraoral ultrasound
- Where
- Canada
- Volunteers
- 64 people, healthy volunteers welcome
- Sponsor
- University of Alberta
Muscular Atrophy, Spinal
Natural History of SMA
This is an investigator initiated observational study with the aim to record several aspects of function, care and adverse events in a large cohort of SMA patients followed longitudinally by using a structured academic disease…
- Trial name
- iSMAR (NCT05755451)
- Where
- 5 sites in Italy
- Volunteers
- 1,200 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Stomach Neoplasms
Lifestyle Intervention for Prevention of Gastric Neoplasm
The purpose of this study is to determine whether positive lifestyle interventions (diet modification and smoking cessation) are effective in the prevention of gastric pre-cancer and cancer occurrences and reccurence of gastric…
- Trial name
- NCT02679495
- Testing
- Active measures of lifestyle modification
- Where
- China
- Volunteers
- 350 people
- Sponsor
- Shandong University
Trapezius Muscle Atrophy
Trapezius Muscle Activities in the Natural Head Posture and Corrected Head Posture During Wall-Slide Exercise
The goal of this observational study is to investigate the effect of head posture on trapezius muscle activity and ratios during wall slide exercise.
- Trial name
- NCT06872957
- Testing
- Surface Electromyography Noraxon MiniDTS system
- Where
- Turkey (Türkiye)
- Volunteers
- 19 people, healthy volunteers welcome
- Sponsor
- Ankara Yildirim Beyazıt University
Optic Pathway Glioma
Safety and Efficacy of the PAINLESS Nerve Growth Factor CHF6467 in Optic Pathway Glioma (OPG)
Infantile optic pathway glioma (OPG) is generally benign and slow-growing, but due to infiltration and compression of sensitive neuronal structures in the optical pathways, progressive visual loss is a frequent and highly…
- Trial name
- NGF-OPG (NCT05733572)
- Testing
- CHF6467
- Where
- Italy
- Volunteers
- 36 people
- Sponsor
- Benedetto Falsini
Leber Hereditary Optic Neuropathy (LHON), Leber's Hereditary Optic Neuropathy (LHON)
Pilot Study of the Efficacy of Nicotinamide (Vitamin B3) in Leber's Hereditary Optic Neuropathy
Leber Hereditary Optic Neuropathy (LHON) is a rare genetic disease that causes sudden and severe vision loss, usually in young adults.
- Conditions
- Leber Hereditary Optic Neuropathy (LHON), Leber's Hereditary Optic Neuropathy (LHON), Mitochondrial Disease, Optic Nerve Disease
- Trial name
- NICOLHON (NCT07258667)
- Testing
- Nicotinamide treatment
- Where
- France
- Volunteers
- 13 people
- Sponsor
- University Hospital, Angers
Spinal Muscular Atrophy
Measuring Levels of SMN in Blood Samples of SMA Patients
Spinal muscular atrophy (SMA) is a disorder that affects the motor neurons. SMA is caused by a mutation in a part of the DNA called the survival motor neuron (SMN1) gene, which normally produces a protein called SMN.
- Trial name
- NCT00061607
- Where
- United States
- Volunteers
- 73 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Spinal Muscular Atrophy
A Trial of Hydroxyurea in Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is an autosomal recessive disorder in humans which results in the loss of motor neurons. It is caused by reduced levels of the survival motor neuron (SMN) protein as a result of loss or mutation of…
- Trial name
- NCT00485511
- Testing
- Hydroxyurea
- Where
- Taiwan
- Sponsor
- Kaohsiung Medical University Chung-Ho Memorial Hospital
RT II Gingival Recession
Hyaluronic Acid and Polynucleotides With Connective Tissue Graft and Coronally Advanced Flap for RT II Gingival Recession
This case series will evaluate the effectiveness of injectable hyaluronic acid combined with polynucleotides as an adjunct to coronally advanced flap and connective tissue graft in patients with RT II gingival recession.
- Trial name
- NCT07771244
- Testing
- Coronally Advanced Flap with Connective Tissue Graft and Hyaluronic Acid/Polynucleotides
- Where
- Egypt
- Volunteers
- 12 people
- Sponsor
- Cairo University
Spinal Muscular Atrophy (SMA), Duchenne Muscular Dystrophy (DMD)
Active NBS Study: Decentralised Monitoring Motor Development in Children With Duchenne Muscular Dystrophy or Spinal Muscular Atrophy Identified by Newborn Screening
The Active NBS Liege study is a monocentric, academic, fully remote, observational study designed to validate digital measures of motor development in children with spinal muscular atrophy (SMA) or Duchenne muscular dystrophy…
- Trial name
- Active-NBS BE (NCT07286565)
- Testing
- MAIJU, Syde, Questionnaires
- Where
- Belgium
- Volunteers
- 100 people
- Sponsor
- Centre Hospitalier Universitaire de Liege
Duchenne Muscular Dystrophy
NS-050/NCNP-03 in Boys With DMD (Meteor50)
This is a Phase 1/2 study of Multiple-Ascending Dose (MAD) levels for 12 weeks of treatment followed by 24 weeks of open-label treatment with a selected dose of NS-050/NCNP-03 administered once weekly to ambulant boys with DMD…
- Trial name
- NCT06053814
- Testing
- NS-050/NCNP-03
- Where
- 19 sites in United States, Canada and 3 more
- Volunteers
- 20 people
- Sponsor
- NS Pharma, Inc.
Duchenne Muscular Dystrophy
A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995)
The purpose of this extension study is to evaluate the ongoing safety and tolerability of additional treatment with eteplirsen administered once weekly by intravenous (IV) infusion in male participants with DMD who have…
- Trial name
- NCT03985878
- Testing
- Eteplirsen
- Where
- 4 sites in Belgium, France and 2 more
- Volunteers
- 15 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
Improved Muscle Function in Duchenne Muscular Dystrophy Through L-Arginine and Metformin
The purpose of the study is to show that the intake of L-arginine and metformin improves muscle function and delays disease progression in patients with Duchenne's muscular dystrophy.
- Trial name
- NCT02516085
- Testing
- Metformin, L-Arginine
- Volunteers
- 5 people
- Sponsor
- University Hospital, Basel, Switzerland
Muscular Dystrophy, Duchenne
Study of Genotype and Phenotype Characterization in Duchenne Muscular Dystrophy With Small Mutations
Improved standards of care and the regular early use of glucocorticoid treatment have changed the natural history of Duchenne muscular dystrophy (DMD), affecting both survival and time of loss of functional milestones.
- Trial name
- NCT05833633
- Testing
- MUSCLE MAGNETIC RESONANCE IMAGING (MRI)
- Where
- Italy
- Volunteers
- 17 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Leber Congenital Amaurosis 10, Blindness
Extension Study to Study PQ-110-001 (NCT03140969)
Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders
- Trial name
- INSIGHT (NCT03913130)
- Testing
- QR-110
- Where
- 3 sites in United States, Belgium
- Volunteers
- 9 people
- Sponsor
- Laboratoires Thea
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