Clinical trials: MYOPATHY
Propionic/Methylmalonic Acidemias, Maple Syrup Urine Disease
Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in Kazakhstan
Inborn errors of metabolism (IEM) are not have specific clinical signs, they masquerade as other diseases, and are difficult to diagnose using only clinical manifestations or routine laboratory tests.
- Conditions
- Propionic/Methylmalonic Acidemias, Maple Syrup Urine Disease, Citrullinemia, Argininosuccinic Aciduria
- Trial name
- NCT05910151
- Testing
- Obtaining Dry Blood Spots From Healthy Newborns (Aged 1-7 Days), Obtaining Dry Blood Spots From Healthy Children Aged 8 Days - 7 Years, Obtaining Dry Blood Spots From Healthy Children Aged 8-18 Years
- Where
- Kazakhstan
- Volunteers
- 2,250 people, healthy volunteers welcome
- Sponsor
- West Kazakhstan Medical University
Mitochondrial Myopathy
Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy
OBJECTIVES: I. Assess the efficacy of thioctic acid in treating a single patient with mitochondrial myopathy.
- Trial name
- NCT00004770
- Testing
- thioctic acid
- Volunteers
- 1 people
- Sponsor
- National Center for Research Resources (NCRR)
Cerebral Palsy, Spasticity
Power Wheelchair Joystick Use in Spastic Cerebral Palsy
There are over 750,000 individuals in the U.S. with Cerebral Palsy (CP). Up to 46% of adults with CP report limited mobility in their communities.
- Trial name
- NCT00337688
- Testing
- isometric joystick
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University of Pittsburgh
Deep Breathing Maneuver, PROGRESSIVE MUSCLE RELAXATION EXERCISES
SHORT-TERM EFFECTS OF DEEP BREATHING AND PROGRESSIVE MUSCLE RELAXATION EXERCISES ON MUSCULOSKELETAL PAIN AND WELL-BEING IN HIGH SCHOOL STUDENTS
Exam stress in high school students, long hours of studying in the static posture, and lack of physical activity habits can cause physical and psychological discomfort in them.
- Trial name
- Deep breathing (NCT06972173)
- Testing
- DEEP BREATHING AND PROGRESSIVE MUSCLE RELAXATION EXERCISES
- Where
- Turkey (Türkiye)
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- Kırklareli University
Spasticity, Muscle, Cerebral Palsy
Effect of Session Frequency of rESWT on Gastrocnemius Muscle Spasticity in Children With Spastic Type Cerebral Palsy
Spasticity, defined as a velocity dependent increase in tonic stretch reflexes, is one of the most prominent motor problems that occur in patients with cerebral palsy (CP).
- Conditions
- Spasticity, Muscle, Cerebral Palsy, Child; Spastic
- Trial name
- NCT04574622
- Testing
- radial Extracorporeal Shockwave Therapy
- Where
- Indonesia
- Volunteers
- 14 people
- Sponsor
- Rizky Kusuma Wardhani
Duchenne Muscular Dystrophy
Strength Training in Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a debilitating neuromuscular disease that causes muscle breakdown, weakness, and eventual death.
- Trial name
- NCT02421523
- Testing
- Aim 2 Exercise group, Aim 1 Exercise Dosing, Aim 2 Control group
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- University of Florida
Cardiomyopathy Associated With Myopathy and Sudden Death
Cardiac Arrhythmias and Sudden Death in Patients Affected With Laminopathies
This research study includes patients ages 1 to 25 years old with Lamin A/C related muscular dystrophy (LMNA-MD). The goal of this study is to evaluate how the heart is affected in children and teens with LMNA-MD.
- Trial name
- NCT02601066
- Testing
- Electrophysiological Study and ECG holter monitor
- Where
- Spain
- Volunteers
- 40 people
- Sponsor
- Hospital Sant Joan de Deu
Severe Spasticity
Safety Study of 3 mg/mL Baclofen Injection (Intrathecal) Using A Programmable Pump
Safety study to assess the 3 mg/mL baclofen injection (intrathecal) using a programmable pump
- Trial name
- NCT01520545
- Testing
- Gablofen® 3 mg/mL (baclofen injection), SynchroMed® II Programmable Pump
- Where
- 13 sites in United States
- Volunteers
- 153 people
- Sponsor
- Piramal Critical Care, Ltd.
HSP, Hereditary Spastic Paraplegia
Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)
Safety and Efficacy of AAV9/AP4B1 For Patients with AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47): A Phase 1/2 Single-Center, Open-Label Study of Stereotactic Intra-cisterna Magna Administration.
- Conditions
- HSP, Hereditary Spastic Paraplegia, Hereditary Spastic Paraparesis, Hereditary Spastic Paraplegia Type 50
- Trial name
- NCT06948019
- Testing
- BFB-101 (AAV9-CBh-AP4B1)
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- BlackfinBio Ltd
Duchenne Muscular Dystrophy, Duchenne Muscular Dystrophy-Associated Dilated Cardiomyopathy
Duchenne Muscular Dystrophy Heart Study
Retrospective cohort study including patients with genetically proven Duchenne muscular dystrophy, diagnosed from January 1993 to March 2020.
- Trial name
- DMD-HS (NCT03443115)
- Where
- France
- Volunteers
- 700 people
- Sponsor
- Hôpital Cochin
Spastic Cerebral Palsy (sCP)
Botulinum Neurotoxin for Children With CP: a Delicate Balance Between Clinical Benefits and Muscular Harm
A cross-sectional design study will be carried out to explore the morphological muscle properties and alterations in muscle composition on a macroscopic level in children with spastic cerebral palsy (CP).
- Trial name
- Bo-Balance (NCT06991725)
- Where
- Belgium
- Volunteers
- 51 people, healthy volunteers welcome
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
Sjogren-Larsson Syndrome (SLS)
Sjogren-Larsson Syndrome: Natural History, Clinical Variation and Evaluation of Biochemical Markers
Sjogren-Larsson syndrome (SLS) is a rare genetic disease in which patients typically exhibit ichthyosis (dry, scaly skin), intellectual disability, spasticity, seizures and a distinctive maculopathy.
- Trial name
- SLS (NCT01971957)
- Where
- 2 sites in United States
- Volunteers
- 20 people
- Sponsor
- University of Nebraska
Cerebral Palsy, Excessive Crying
Excessive Crying in Children With Cerebral Palsy and Communication Deficits
Management of excessive crying in children with cerebral palsy and communication deficits [ECCCPCD] was guided by the associated clinical findings and investigations.
- Conditions
- Cerebral Palsy, Excessive Crying, Pain
- Trial name
- ECCCPCD (NCT04523935)
- Testing
- Baclofen, Diazepam, Clonazepam, Trihexyphenidyl, Tetrabenazine, Gabapentin, Topiramate, Lamotrigine, Amitriptyline.
- Volunteers
- 131 people
- Sponsor
- Sathbhavana Brain Clinic
Post-COVID-19 Condition, Ulcerative Colitis
Emerging Indications for Hyperbaric Oxygen Treatment
This study will evaluate the effectiveness of hyperbaric oxygen therapy (HBOT) on treating emerging indications (i.e., conditions that have shown to potentially benefit from HBOT) using the Multicenter Registry for Hyperbaric…
- Conditions
- Post-COVID-19 Condition, Ulcerative Colitis, Crohn Disease, Calciphylaxis
- Trial name
- NCT07240649
- Testing
- Hyperbaric oxygen treatment (HBOT), 100% Oxygen
- Volunteers
- 100 people
- Sponsor
- Jay C. Buckey Jr.
Panniculitis, Dermatomyositis
Panniculitis in Dermatomyositis
Adipose tissue involvement is rare in dermatomyositis. The occurrence of partial or diffuse lipodystrophy is a rare but well-characterized manifestation, particularly in juvenile forms of dermatomyositis.
- Trial name
- PANNICUL (NCT07345949)
- Where
- France
- Volunteers
- 20 people
- Sponsor
- University Hospital, Strasbourg, France
Muscular Dystrophies, Muscular Dystrophy, Duchenne
A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
HOPE-3 is a two cohort, Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of a cell therapy called deramiocel (CAP-1002) in study participants with Duchenne…
- Conditions
- Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- HOPE-3 (NCT05126758)
- Testing
- Deramiocel (CAP-1002)
- Where
- 20 sites in United States
- Volunteers
- 106 people
- Sponsor
- Capricor Inc.
Myotonic Dystrophy Type 1
Children's Health Research Institute(CHRI), Stanford Lucile Packard Children Hospital (LPCH) Protocol on Myotonic Dystrophy
Study to focus on the defining and managing the neuropsychological abnormalities of myotonic dystrophy and to find out if the neuropsychological abnormalities have any correlation with changes seen on Magnetic Resonance Imaging.
- Trial name
- CHRI (NCT02269865)
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Stanford University
Injury, Knee, Injury, Ankle
Prevention of Patellar and Achilles Tendinopathies in Youth Basketball
This study seeks to determine the prevalence of knee and ankle tendinopathies in youth basketball players, as well as determine the efficacy of a Basketball specific Neuromuscular training warm-up in reducing injuries.
- Conditions
- Injury, Knee, Injury, Ankle, Tendinopathy, Sports Injuries in Children
- Trial name
- NCT04363398
- Testing
- Comprehensive Follow-up, Standard Follow-up
- Where
- Canada
- Volunteers
- 441 people, healthy volunteers welcome
- Sponsor
- Sport Injury Prevention Research Centre
Spinal Cord Injuries, Muscle Spasticity
Cyproheptadine and Chlorpromazine Effects on Spasticity
The main goal of this research is to understand the neuronal mechanisms that mediate the development of spasticity and motor dysfunction after spinal cord injury.
- Trial name
- NCT01509885
- Where
- Canada
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- University of Alberta
Muscular Dystrophies
Device for Breathing Frequency Monitoring in Muscular Dystrophy
Patients at risk of developing respiratory dysfunctions, such as patients with severe forms of muscular dystrophy, need a careful respiratory assessment, and periodic follow-up visits to monitor the progression of the disease.
- Trial name
- NCT04313595
- Testing
- RespirHó
- Where
- Italy
- Volunteers
- 15 people
- Sponsor
- IRCCS Eugenio Medea
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