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NCT07543302
The intended investigation is a pilot study to evaluate the safety and efficacy of a novel transcutaneous auricular vagus nerve stimulator system, termed TRAVAGUS ONE, to reduce systemic levels of inflammatory mediators in patients with Duchenne muscular dystrophy (DMD). Electrical vagus nerve stimulation is an investigational anti-inflammatory therapy targeting the nervous system to modulate dysregulated inflammation. DMD is a severe genetic disorder characterized by progressive muscle degeneration and weakness due to the alterations of a protein named dystrophin that helps keep muscle cells intact. The disease affects male children, and the symptom onset is in early childhood. In addition to the muscle degeneration all patients suffer from severe systemic inflammation and express increased systemic levels of proinflammatory molecules, which can be quantified in peripheral blood samples. Daily, systemic corticosteroid therapy with high doses is the standard of care in DMD to control symptoms and to slow disease progression through potent anti-inflammatory activity. Unfortunately, high dosage and long-term use of corticosteroids are typically also accompanied by severe adverse effects that reduce the quality of life in DMD patients. There is thus a great need for improved anti-inflammatory treatment with less severe adverse effects. In the planned pilot study involving 20 DMD patients aged 5-17 years, the investigators intend to treat each patient for one week in their home environment using transcutaneous auricular vagus nerve stimulation (taVNS) with a novel device named Travagus One to find out whether this intervention is safe and may reduce systemic levels of proinflammatory molecules. Venous blood samples will be collected at three different time points before and after the taVNS treatment period. Note: This study relates to an FDA-nonregulated Device. There are no U.S. Locations for the study. The study was approved by the Swedish Medical Products Agency.
NCT06817382
The primary objective of this study is to evaluate the safety and tolerability of a single dose of INS1201 via IT administration in ambulatory male participants with DMD.
NCT06839469
The purpose of this research is (1) to identify disease specific walking-related digital biomarkers of disease severity, and (2) monitor longitudinal changes in natural environments, for extended periods of time, in DMD and SMA.
NCT06491927
RGX-202-5101 is a long-term follow up study that evaluates the long-term safety and efficacy of RGX-202 in participants who have received RGX-202 (an investigative gene therapy designed to deliver a transgene for novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain) in a separate parent study.
NCT07429240
The purpose of this Phase 1/2a trial is to evaluate the safety, tolerability, and preliminary efficacy of PBGENE-DMD in patients with DMD harboring mutations amenable to excision of exons 45-55. Given the limitations of existing therapeutic strategies, PBGENE-DMD represents a novel, innovative approach with the potential for a one-time, durable correction of the underlying genetic defect in the largest molecular subset of patients with DMD.
NCT07037862
This is a study of the investigational medicine ENTR-601-44 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition. The researchers want to: Test how safe ENTR-601-44 is, learn about any side effects, and look at the potential positive effects of ENTR-601-44, compared to placebo. Placebo looks like the investigational medicine but does not contain any active ingredient. In this summary ENTR-601-44 and placebo are both called study treatments. The study has 2 parts: * Part A * A Double-Blind Period, to evaluate if ENTR-601-44 is safe and to determine the best dose of ENTR-601-44 for Part B. * Following the Double-Blind period, participants will roll into an open-label treatment period during which the safety and efficacy of extended dosing will be evaluated. * Part B * To further evaluate the effect and safety of ENTR-601-44 at the dose determined in Part A. Participants will: * Receive study treatment in the form of multiple intravenous (IV) infusions (slow injection) into a vein over the course of several weeks in Part A and in Part B * Visit the clinic regularly for checkups and tests such as: blood and urine tests, physical examinations, questionnaires, and exercise tests. Participants will have a muscle biopsy at the beginning of their participation and after their last dose to allow researchers to compare whether there have been changes in the muscle as a result of the study drug. Participants are allowed to continue receiving their standard of care therapy for DMD during the study, as long as their health remains stable.
NCT07209332
This is a Phase 2 open-label extension (OLE) study to evaluate the long-term safety, tolerability, efficacy, pharmacokinetics, and the pharmacodynamics (PD) through potential exploratory biomarker(s) of intravenous (IV) WVE-N531 in patients with DMD who participated in another study of WVE-N531. All patients will have rolled over from a previous study of WVE-N531.
NCT03368742
This is a controlled, open-label, single-ascending dose study to evaluate the safety and tolerability of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Participants will receive a single intravenous (IV) infusion of SGT-001 and will be followed for approximately 5 years. The protocol was amended to drop the control arm after 4 participants were dosed.
NCT07160634
This is a Phase 3, double-blind, placebo-controlled study with the primary objective of evaluating the efficacy of a single IV infusion of SGT-003 in pediatric ambulant male participants with DMD. The secondary objectives include the evaluation of additional efficacy and safety outcomes. The study will be divided into 2 parts. Participants will be randomized 1:1 to either SGT-003 in Part 1 followed by placebo in Part 2 or to placebo in Part 1 followed by SGT-003 in Part 2. Participants will continue to be monitored in long term follow up (LTFU) for at least 5 years from their SGT-003 dosing date.
NCT06450639
The purpose of this study is to assess the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of satralizumab, a humanized anti-interleukin-6 receptor (aIL-6R) monoclonal antibody, in ambulatory and non-ambulatory participants with DMD age ≥ 8 to \< 18 years old receiving corticosteroid therapy.
NCT07437378
Duchenne Muscular Dystrophy (DMD) is a progressive X-linked neuromuscular disorder characterized by muscle degeneration, pseudohypertrophy, and declining functional mobility. This cross-sectional observational study investigates the relationship between gastrocnemius muscle architecture and functional ability in ambulatory children with DMD. Muscle thickness and fascicle length were assessed using ultrasonography and correlated with motor function and ankle plantarflexion during gait.
NCT07423026
Every year, 100 boys are born in the UK with a rare muscle disease called Duchenne muscular dystrophy. These boys cannot make an important muscle protein called dystrophin. They become weaker as they get older and lose the ability to walk as teenagers. This is a life-limiting condition. There is no cure, but medicines are being made that could help these boys make dystrophin. These medicines are most likely to work best in toddlers, before their muscles become damaged. There is no way of testing these medicines in children under four. In older children, it is possible to measure how well and how quickly a child can do movements like sitting up, standing up, and running. Unfortunately, these tests are not suitable for toddlers as they often struggle to listen and do what they are asked to do. Tiredness and mood can also affect their scores. Luckily, there is a new way of testing how well children move. They can wear special watch-like devices on their ankles that record information about their steps as they go about their normal lives. This is a good way of testing how well a child walks. It is now used to test medicines in children over four years old. Our aim is to test whether this device works well in children under four. This study will invite 30 boys with DMD (and their parent/caregiver) and 30 boys without DMD aged 1-3 years old from across the country to join the study. There are no hospital visits. Children will receive the watch-like devices to wear for three blocks of 28-days over six months during their normal daily activities. At the start and end of the study, a physiotherapist will visit the homes of boys with DMD. They will check their movements using other tests. The investigators will find out 1) if young boys are happy to wear the device, 2) how it compares to other tests, and 3) if it can detect changes in walking ability. This study could give us a way to test medicines in younger children. Wearable devices could cut down the travel and stress of tests for boys and their families. Children with learning or behavioural difficulties, and children living far from research centres could now also take part in studies of new medicines. This study could bring us a step closer to treating this life-limiting disease.
NCT06128564
This open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks.
NCT06887491
Duchenne Muscular Dystrophy (DMD) is an X-linked recessive disorder characterized by progressive muscle degeneration and frequent developmental, cognitive and behavioral impairments, occurring in one in 5000 live births of boys. DMD is caused by a deficiency of the protein dystrophin, which maintains the structure and functionality of muscle cells. The absence of dystrophin leads to the weakening and eventual death of muscle cells, resulting in reduced muscle strength and impaired motor function. In early childhood, children with DMD may experience delays in basic motor skills such as walking and standing. Later in life, the disease leads to more severe motor dysfunctions, including loss of muscle strength, problems with balance and coordination, and an increased risk of falls. DMD can also negatively affect cognitive function. Dystrophin is found not only in muscles but also in the brain, and its deficiency in the brain can lead to cognitive problems such as learning disabilities, attention deficits and impaired executive function. These cognitive impairments can affect the academic performance of children with DMD and their functional abilities in everyday life. In this project, the potential effects of a dual task program designed for children with DMD on physical and cognitive functioning will be examined. Dual task training aims to increase children's capacity to perform two different tasks simultaneously, which may improve the integration of cognitive and motor functions. For example, activities such as counting while walking or answering a question while carrying an object in the hand require children to use both motor and cognitive skills simultaneously. Such exercises can increase coordination between cognitive and motor functions and improve the independence of children with DMD in activities of daily living and their overall quality of life. The main goals of the program are to produce positive effects on motor skills and cognitive functions, improve balance and coordination, and enable children to move more independently in activities of daily living. Furthermore, this study highlights the value of a multidisciplinary approach, providing important insights into how rehabilitation approaches can be developed for individuals with a special condition such as DMD. Collaboration between physiotherapists and occupational therapists plays a critical role in providing comprehensive care for these children. The methodology of this study included boys with DMD who were admitted to Lokman Hekim University Muscular and Nerve Diseases Application Center. The children will be randomly assigned to the intervention and control groups, and the children in the intervention group will be enrolled in a dual task performance program for two days a week, one session a day, 45 minutes each session, for eight weeks, with at least two days in between. The effectiveness of the program will be measured using various motor and cognitive assessment tools. The hypotheses of this study are that dual task training will positively affect motor and cognitive and physical functions in children with DMD, improve balance and coordination, and increase the level of independence of these children in activities of daily living. In conclusion, this project aims to contribute to the development of innovative approaches in the treatment of children with DMD. This approach can improve the overall quality of life of children, as well as support their social participation and educational achievement.
NCT03863119
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 another trial of vamorolone therapy), during the time a new drug application for vamorolone is under preparation and review.
NCT05683379
This is an observational screening study to evaluate the prevalence of anti-adeno-associated serotype 8 (AAV8) antibodies in participants with Duchenne muscular dystrophy (DMD).
NCT05016908
Current methods of measuring the response to new treatments for muscular dystrophies involve the examination of small pieces of muscle tissue called biopsies. The investigators are interested in finding less invasive methods that reduce the need for muscle biopsies. The purpose of this research is to learn about the possibility of detecting and measuring the activity and severity of muscular dystrophies by examining a urine sample and a blood sample.
NCT06682585
Brief Summary The goal of this clinical trial is to evaluate the impact of a disease-specific, individualized diet on the nutritional status and functional abilities of Duchenne muscular dystrophy (DMD) patients. The study will focus on children aged 4-8 years residing in Ankara, Turkey. Key questions the investigators aim to answer: Can a tailored dietary intervention improve the nutritional status of DMD patients? Does a specialized diet positively impact the functional abilities of DMD patients, as measured by the North Star Ambulation Assessment (NSAA)? Participants will undergo a comprehensive nutritional assessment, including anthropometric measurements, and will receive individualized dietary counseling. The intervention will focus on optimizing energy, protein, calcium, and fluid intake, as well as addressing the potential side effects of corticosteroid therapy. The primary outcome measure will be changes in nutritional status, as assessed by anthropometric measurements. Secondary outcome measures will include changes in functional abilities as measured by the NSAA and quality of life assessments.
NCT01648634
The objective is to determine whether nebivolol, a beta-blockade drug, can prevent the development of heart disease in patients with Duchenne muscular dystrophy aged 10 to 15 year-old.
NCT05558813
The purpose of this study is to describe the progression of tissular and functional myocardial abnormalities in patients with Duchenne muscular dystrophy using cardiac magnetic resonance imaging and blood biomarkers assays.