Clinical trials: Muscular Atrophy
SMA - Spinal Muscular Atrophy, Gene Therapy
Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
Phase 3 pivotal US trial studying open-label intravenous administration of onasemnogene abeparvovec-xioi in spinal muscular atrophy (SMA) Type 1 participants.
- Trial name
- STR1VE (NCT03306277)
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 16 sites in United States
- Volunteers
- 22 people
- Sponsor
- Novartis Gene Therapies
Spinal Muscular Atrophy
An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy
This is an interventional study to evaluate safety and efficacy of AAV-hSMN1 in spinal muscular atrophy patients.
- Trial name
- NCT06288230
- Testing
- vesemnogene lantuparvovec
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Lantu Biopharma
Duchenne Muscular Dystrophy
Evaluation of Brain Dysfunction in Patients with Duchene Muscular Dystrophy
To study the cognitive function \& psychiatric aspects of patients with DMD by specific psychometry tests \& its relation to quality of life of the patients To evaluate brain structural changes using MRI and correlate them to the…
- Trial name
- NCT06732011
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- Assiut University
Duchenne Muscular Dystrophy
Upper Extremity Muscle Strength, Balance and Functional Skills in DMD
This study aims to examine the relationship between upper extremity muscle strength, balance and functional skills of children with DMD.
- Trial name
- NCT06379906
- Testing
- Assessment
- Where
- Turkey (Türkiye)
- Volunteers
- 32 people
- Sponsor
- Halic University
Muscular Atrophy, Spinal
A Real-world Study of Disease-modifying Therapy Treatment Outcomes in Patients With Spinal Muscular Atrophy
The aim of this study was to evaluate treatment patterns, SMA-related complications and services, and all-cause medical encounters of patients with SMA receiving multiple DMTs in the real world.
- Trial name
- NCT07403214
- Where
- United States
- Volunteers
- 4,805 people
- Sponsor
- Novartis Pharmaceuticals
Muscular Dystrophies
A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess Effect, Safety, Tolerability and PK of Multiple SC Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for IV Dosing as an Alternative Route of Administration
The purpose of the extension phase of this study is to determine whether Drisapersen is effective in the treatment of boys with Duchenne muscular dystrophy resulting from a mutation thought to be corrected by exon 51 skipping.
- Trial name
- NCT01910649
- Testing
- Drisapersen
- Volunteers
- 12 people
- Sponsor
- BioMarin Pharmaceutical
Muscular Dystrophy, Duchenne, Upper Extremity
The Effects of Trunk Exercises on Upper Extremity and Respiratory Functions in DMD
The aim of this study was to investigate the effects of trunk training on trunk control, upper extremity, and pulmonary function in children with Duchenne muscular dystrophy (DMD).
- Conditions
- Muscular Dystrophy, Duchenne, Upper Extremity, Respiratory Functions
- Trial name
- NCT04669847
- Testing
- Trunk-oriented exercise program, Conventional exercise program
- Where
- Turkey (Türkiye)
- Volunteers
- 26 people
- Sponsor
- Gokce Yagmur Gunes Gencer
Duchenne Muscular Dystrophy
Efficacy of Umbilical Cord Mesenchymal Stem Cells in Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a genetic disorder caused by an absence of dystrophin and characterized by progressive muscle degeneration.
- Trial name
- NCT02285673
- Testing
- Umbilical Cord Mesenchymal Stem Cell
- Where
- Turkey (Türkiye)
- Volunteers
- 10 people
- Sponsor
- Acibadem University
Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy)
A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping
A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping
- Conditions
- Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Muscular Disorders, Atrophic, Muscular Disease
- Trial name
- SAFARI44 (NCT07587242)
- Testing
- AOC 1044
- Where
- 18 sites in Australia, Belgium and 6 more
- Volunteers
- 70 people
- Sponsor
- Avidity Biosciences, Inc.
Spinal Muscular Atrophy (SMA), Cerebral Palsy (CP)
Gut Peptides and Bone Remodeling in Children With Neuromuscular Disorders
Both GIP and GLP-2 reduce bone resorption (measured as CTX) in healthy adult individuals. In this study, we will investigate whether GIP and GLP-2 reduce CTX in children with spinal muscular atrophy, duchenne muscular dystrophy…
- Conditions
- Spinal Muscular Atrophy (SMA), Cerebral Palsy (CP), Duchenne Muscular Dystrophy (DMD)
- Trial name
- NCT07254988
- Testing
- Meal test, GIP + GLP-2
- Where
- Denmark
- Volunteers
- 3 people
- Sponsor
- University of Copenhagen
Prostate Cancer, Radiotherapy Side Effect
PROstate CAncer Radiotherapy - Bowel Quality of Life (PROCAR-BQ)
This is a prospective pilot study to evaluate the mean increase of bowel symptoms after pelvic radiotherapy (RT) in prostate cancer (PCa) patient using the validated \& newly translated EORTC-QLQ PRT20 module.
- Conditions
- Prostate Cancer, Radiotherapy Side Effect, Bowel Dysfunction, Quality of Life
- Trial name
- PROCAR-BQ (NCT05880446)
- Testing
- Bowel symptoms & QoL assessment
- Where
- Belgium
- Volunteers
- 41 people
- Sponsor
- Jules Bordet Institute
Lumbar Disc Herniation, Lumbar Degenerative Disease
Multivariate Analysis and Risk Factors Were Used to Predict the Short-term Postoperative Pain of Degenerative Lumbar Spine
The objective of this retrospective clinical study wasto study the risk factors for short-term low back and leg pain after PLIF in patients with lumbar Degenerative disease, and to establish a risk prediction model for short-term…
- Conditions
- Lumbar Disc Herniation, Lumbar Degenerative Disease, Sarcopenia
- Trial name
- NCT06628583
- Testing
- Magnetic Resonance Imaging with Contrast
- Where
- China
- Volunteers
- 936 people, healthy volunteers welcome
- Sponsor
- Hao Liu
Stroke, Hemiplegia Following Ischemic Stroke
Improving STEP in Stroke Patients
Stroke is the leading cause of acquired disability in adults. Stroke causes death in 10% of patients, disability and functional handicap in 60% of cases.
- Conditions
- Stroke, Hemiplegia Following Ischemic Stroke, Disability Physical, Spasticity as Sequela of Stroke
- Trial name
- I-STEP (NCT07403149)
- Testing
- REMARCHE® shoes
- Volunteers
- 58 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Muscle Tone Poor, Spinal Muscular Atrophy
Myotonometer Intra- and Inter Examiner Reliability on Spinal Muscular Atrophy
The primary aim of the study was to measure the intra-rater and inter-rater reliability of MyotonPRO in measuring postural muscle tone and mechanical properties in individuals with spinal muscular atrophy (SMA).
- Trial name
- NCT05521217
- Testing
- MyotonPRO
- Where
- 2 sites in Turkey (Türkiye)
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Istanbul Medipol University Hospital
Duchenne Muscular Dystrophy
Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy
The intent of this protocol is to provide continued access to vamorolone for subjects in the United States and Canada who have completed the VBP15-LTE, VBP15- 004, or VBP15-006 protocols (and are thereby ineligible to enroll in…
- Trial name
- NCT03863119
- Testing
- Vamorolone
- Where
- 12 sites in United States, Canada and 1 more
- Sponsor
- Santhera Pharmaceuticals
Duchenne or Severe Becker Myopathy
IRM Cardiaque en Respiration Libre Pour Des Patients Atteints de Dystrophinopathie sévère
Clinical, prospective and monocentric study aiming at assessing the feasibility of fibrosis detection and quantification (and of function assessment) during MRI without breath-holds in a population of adults and children with…
- Trial name
- NCT02078076
- Testing
- Magnetic Resonance Cardiac Imaging (with Gadolinium)
- Where
- France
- Volunteers
- 22 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Duchenne Muscular Dystrophy (DMD)
An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy
This open-label extension study aims to evaluate the long-term safety and tolerability of weekly BMN 351 infusions, as well as to assess the effect of BMN 351 on physical function, in participants with DMD who participated in the…
- Trial name
- NCT07573631
- Testing
- BMN 351
- Where
- 6 sites in Italy, Netherlands and 3 more
- Volunteers
- 18 people
- Sponsor
- BioMarin Pharmaceutical
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
DMD/BMD is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function.
- Trial name
- NCT00592553
- Testing
- Ataluren
- Where
- 37 sites in United States, Australia and 9 more
- Volunteers
- 174 people
- Sponsor
- PTC Therapeutics
Myotonic Dystrophy Type 1
Virtual Reality-training in Theory of Mind in the Childhood Form of Myotonic Dystrophy Type 1
The ability of an individual to conceive some alternative representations and to behave in a flexible manner would emerge from preschool age and drastically improve between the ages of 3 and 5 (Doebel and Zelazo, 2013).
- Trial name
- TOM-VR-DM1 (NCT05916677)
- Testing
- Virtual Reality-training in Theory of Mind, Traditional training in Theory of Mind
- Where
- France
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Institut de Myologie, France
Duchenne Muscular Dystrophy (DMD)
A Study to Evaluate the Safety and Tolerability of GEN6050X in Duchenne Muscular Dystrophy.
The study will evaluate the safety and tolerability of GEN6050X gene therapy in Duchenne muscular dystrophy (DMD) patients amenable to exon 50 skipping.
- Trial name
- GEN6050XIIT (NCT06392724)
- Testing
- GEN6050X intravenous injection
- Where
- China
- Volunteers
- 3 people
- Sponsor
- Peking Union Medical College Hospital