Clinical trials: Muscle Disorders
Neuroblastoma, Rhabdomyosarcoma
Study to Find a Safe Dose and Show Early Clinical Activity of Weekly Nab-paclitaxel in Pediatric Patients With Recurrent/ Refractory Solid Tumors
The purpose of this study is to find the safe dose of nab-paclitaxel in children with solid tumors, and to see if it works to treat these solid tumors in children and young adults (in Phase 1 ≤ 18 years old and in Phase 2 ≤ 24…
- Conditions
- Neuroblastoma, Rhabdomyosarcoma, Ewing's Sarcoma, Ewing's Tumor
- Trial name
- NCT01962103
- Testing
- nab-paclitaxel
- Where
- 20 sites in United States, Canada and 5 more
- Volunteers
- 107 people
- Sponsor
- Celgene
Spinal Muscular Atrophy
Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen
Risdiplam Exchange (RISE) is a study of spinal muscular atrophy (SMA) patients who crossover to 36 months of open-label risdiplam monotherapy following a comparable period of nusinersen treatment.
- Trial name
- RISE (NCT05522361)
- Testing
- Risdiplam
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Clinic for Special Children
Muscular Atrophy, Spinal
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2…
- Trial name
- HINALEA 1 (NCT05861986)
- Testing
- Risdiplam
- Where
- 16 sites in United States, Germany and 2 more
- Volunteers
- 28 people
- Sponsor
- Hoffmann-La Roche
Duchenne Muscular Dystrophy (DMD)
Digital Monitoring of Upper Limb Function in Non-Ambulant DMD
Duchenne Muscular Dystrophy (DMD) is a rare genetic disorder caused by the absence of dystrophin, leading to progressive muscle degeneration.
- Trial name
- Acti-nRoll (NCT07664124)
- Testing
- Performance of the Upper Limb (PUL) Test, Brook test, Dynamometric measurements of muscle strength
- Where
- Belgium
- Volunteers
- 50 people
- Sponsor
- Centre Hospitalier Universitaire de Liege
Contractures, Scars
Preexpanded Perforator Flaps in Children
Contractures and broad scars of the axilla, anterior chest wall and neck have detrimental effects on functional, physical and psychological development of children.
- Trial name
- NCT01700595
- Testing
- new technical intervention
- Where
- Turkey (Türkiye)
- Volunteers
- 7 people
- Sponsor
- Istanbul University
Duchenne Muscular Dystrophy (DMD)
Evaluation of the Safety and Efficacy of BBM-D101 to Treat Patients with Duchenne Muscular Dystrophy
The purpose of the study is to assess the safety, tolerability, and efficacy of BBM-D101 to treat patients with Duchenne Muscular Dystrophy.
- Trial name
- NCT06641895
- Testing
- BBM-D101
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Shanghai Jiao Tong University School of Medicine
Spastic Cerebral Palsy, Dysarthria
Lateral Cord Magnetic Stimulation For Refractory Spastic Cerebral Palsy
Lateral cord stimulation (LCS) was discovered by the author as the producer of an average threshold increase for abnormal muscle contraction responses, in experimental pathological conditions, as described and referred.
- Trial name
- NCT03676439
- Testing
- Magnetic Spinal Stimulation
- Where
- Argentina
- Volunteers
- 20 people
- Sponsor
- Sociedad Argenttina de Neuromodulación
COPD
Does Muscle Wasting Always Mean Muscle Weakness? A Prevalence Study in COPD
Peripheral muscle mass and strength are relevant indicators of COPD survival. Current guidelines recommend to assess muscle strength only in muscle wasted patients.
- Trial name
- M2W (NCT02080936)
- Where
- France
- Volunteers
- 138 people
- Sponsor
- 5 Santé
Shoulder Dislocation or Subluxation
Rotofunc - Non-traumatic Shoulder Instability and Total Rotator Cuff Function
The goal of this clinical trial is to test the effect of a novel assessment and treatment strategy for young adults with severely disabling non-traumatic shoulder instability.
- Trial name
- ROTOFUNC (NCT06261645)
- Testing
- Early Physiotherapist selected active exercises, Delayed Physiotherapist selected active exercises
- Where
- Sweden
- Volunteers
- 100 people
- Sponsor
- Sahlgrenska University Hospital
Metabolic Myopathy
Metabolomic Approach During Exercise Testing in Myalgia Induced by eXercise
Metabolic myopathies form a group of pathologies related to a deficit of muscle energy production (enzymatic deficit) by disorder of the metabolism of carbohydrates, lipids, purines, or mitochondrial involvement related to…
- Trial name
- Matmix (NCT05413681)
- Testing
- a supra-maximal exercise test on a cycloergometer., handgrip muscle activity test.
- Where
- France
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- University Hospital, Clermont-Ferrand
Peritoneal Neoplasms, Retroperitoneal Neoplasms
Continuous Hyperthermic Peritoneal Perfusion (CHPP) With Cisplatin for Children With Peritoneal Cancer
There has been no successful treatment of diffuse peritoneal metastasis or carcinomatosis, in childhood tumors. Once this advanced stage of disease is evident, survival is measured in weeks.
- Conditions
- Peritoneal Neoplasms, Retroperitoneal Neoplasms, Gastrointestinal Neoplasms, Adenocarcinoma
- Trial name
- NCT00436657
- Testing
- CHPP of Cisplatin, Abdominal Surgery
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- M.D. Anderson Cancer Center
Duchenne Muscular Dystrophy
An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
The main purposes of this study are to see if it is safe to use a new medication called vamorolone for more than two weeks in children with Duchenne muscular dystrophy (DMD), to see if vamorolone works for the treatment for DMD…
- Trial name
- NCT02760277
- Testing
- Vamorolone 0.25 mg/day/day, Vamorolone 0.75 mg/day/day, Vamorolone 2.0 mg/day/day
- Where
- 12 sites in United States, Australia and 4 more
- Volunteers
- 48 people
- Sponsor
- ReveraGen BioPharma, Inc.
Duchenne Muscular Dystrophy, Gait Disorders in Children
Development of a New Instrument to Evaluate Gait Characteristics of Individuals With Duchenne Muscular Dystrophy
The aim of this study was to develop a gait assessment instrument for Duchenne Muscular Dystrophy patients (DMD-GAS), and investigate its validity and reliability.The scale was developed considering the expert opinions which…
- Conditions
- Duchenne Muscular Dystrophy, Gait Disorders in Children, Gait Disorders, Neurologic
- Trial name
- NCT05244395
- Testing
- Duchenne Muscular Dystrophy Gait Assessment Scale
- Where
- Turkey (Türkiye)
- Volunteers
- 56 people
- Sponsor
- Hacettepe University
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
Hypotension
Avoiding Hypotension in Preterm Neonates
Objectives Firstly to examine in preterm neonates, whether it is possible to reduce number of hypotensive episodes and use of catecholamines by applying cerebral/peripheral near infrared spectroscopy in combination with…
- Trial name
- AHIP (NCT01910467)
- Testing
- NIRS visible and predefined interventions, NIRS not visible and treatment as usual
- Where
- Austria
- Volunteers
- 98 people
- Sponsor
- Medical University of Graz
Dystonic Cerebral Palsy
Intrathecal Baclofen and Pediatric Dystonia
The goal of this study is to better understand outcomes in children with dystonic cerebral palsy (CP) receiving intrathecal baclofen (ITB), ventral dorsal rhizotomy (VDR), or deep brain stimulation (DBS) as part of standard…
- Trial name
- PREDICT-ITB (NCT06606574)
- Testing
- ITB Titration Protocol, Ventral Dorsal Rhizotomy, Deep Brain Stimulation
- Where
- United States
- Volunteers
- 65 people
- Sponsor
- Baylor College of Medicine
Spinal Muscular Atrophy
A Review of the Management and Outcomes of Children With SMA in the West Midlands During 2017-2022
Spinal Muscular Atrophy (SMA) is a rare neuromuscular condition, characterised by loss of motor neurons as a result of a mutation in the survival motor neuron gene.
- Trial name
- NCT05994950
- Where
- 2 sites in United Kingdom
- Volunteers
- 30 people
- Sponsor
- Robert Jones and Agnes Hunt Orthopaedic and District NHS Trust
Duchenne Muscular Dystrophy
A Study of ATL1102 or Placebo in Participants With Non-ambulatory Duchenne Muscular Dystrophy
This Phase IIb study is a two part, multicenter study to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of ATL1102 in non-ambulant boys with Duchenne Muscular Dystrophy aged 10 to <18 years old.
- Trial name
- NCT05938023
- Testing
- ATL1102 25mg, ATL1102 50mg
- Where
- 13 sites in Australia, Bulgaria and 3 more
- Volunteers
- 48 people
- Sponsor
- Percheron Therapeutics
Liposarcoma, Malignant Peripheral Nerve Sheath Tumors
Selinexor in Combination With Ixazomib for the Treatment of Advanced Sarcoma
The main purpose of this study is to establish a safe and tolerable dose combination (the "maximum tolerated dose") of selinexor and ixazomib when used together for the treatment of patients with certain types of advanced…
- Conditions
- Liposarcoma, Malignant Peripheral Nerve Sheath Tumors, Alveolar Soft Part Sarcoma, Ewing Sarcoma
- Trial name
- NCT03880123
- Testing
- Selinexor, Ixazomib
- Where
- United States
- Sponsor
- Matthew Ingham
Duchenne Muscular Dystrophy
Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy
While it has been known for many years that corticosteroid use benefits boys with Duchenne Muscular dystrophy (DMD), most clinicians do not consider treating until after age 3 or 4 years of age.
- Trial name
- NCT02167217
- Testing
- Prednisolone
- Where
- 6 sites in United States
- Volunteers
- 25 people
- Sponsor
- Washington University School of Medicine