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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.
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.
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.
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.
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.
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.
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.
NCT07172737
Dushenne muscular dystrophy (DMD), which is characterized by the deficiency of dystrophin protein, prevents the muscle from maintaining its normal activity, causing progressive damage to the heart, respiratory muscles and skeletal muscles. When studies on patients with DMD are examined in the literature, very few studies are found investigating the effectiveness of pure respiratory muscle training, while no studies are found investigating the effectiveness of functional respiratory muscle training.
NCT06597656
This is a gene transfer therapy study evaluating the safety of and delandistrogene moxeparvovec dystrophin protein expression from delandistrogene moxeparvovec following therapeutic plasma exchange (plasmapheresis) in ambulatory male participants with DMD and pre-existing antibodies to AAVrh74 over a period of 59 weeks.
NCT07129954
Primary objectives WP1: Evaluate the prevalence of FOF in the study population and how this varies over time. Evaluate whether there are relationships between the variables investigated (clinical, motor, cognitive, psychological) and the presence of FOF. WP2: To evaluate, among those who presented disabling FOF, the effects of two different therapeutic approaches: motor rehabilitation vs. motor rehabilitation plus cognitive-behavioral psychotherapy. Secondary objectives WP1: To evaluate whether different profiles defined by specific clinical, motor, cognitive, psychological, and personological characteristics can be characterized among patients with dystrophy and FOF and how these impact functionality, activity, participation, and quality of life. WP2: Evaluate the effects of cognitive-behavioral therapy (CBT) and a motor treatment on cognitive and psychological aspects, the frequency of falls, and the functional validity.
NCT06392724
The study will evaluate the safety and tolerability of GEN6050X gene therapy in Duchenne muscular dystrophy (DMD) patients amenable to exon 50 skipping.
NCT06290713
Examining two strategies as potential adjuvant therapies for Duchenne muscular dystrophy (DMD); aerobic exercise training (to induce adaptations in skeletal muscle and improve cardiovascular health) and tadalafil, an FDA-approved vasodilator (to optimize blood flow and muscle perfusion which is impaired and often overlooked in DMD). Target: improved muscle function, vascular health, and DMD treatment.
NCT06833931
The study consists of 3 periods: A Screening Period (up to 45 days), a double-blind, placebo-controlled Multiple Ascending Dose (MAD) Period (28 weeks), and a Long-Term Extension (LTE) Period (108 weeks). The primary purpose of the MAD period is to evaluate the safety and tolerability and levels of dystrophin after multiple ascending intravenous (IV) doses of PGN-EDO51 administered to participants with Duchenne muscular dystrophy (DMD). During the MAD period, participants will be randomized to either receive PGN-EDO51 or placebo in a 3:1 fashion, meaning that participants have a 75% chance of receiving PGN-EDO51 and a 25% chance of receiving placebo during this period. The primary purpose of the open-label LTE period is to evaluate the long-term safety and tolerability of PGN-EDO51 in participants who have completed the MAD period. All participants who roll-over into the LTE will receive PGN-EDO51 (no placebo in the LTE).
NCT06925269
The purpose of this study is to understand DMD functional losses or abilities and their association with independence and quality of life from the perspective of individuals with DMD and/or and their caregivers. This is a qualitative interview study in which individuals with DMD and/or their caregivers will be asked to participate in a semi-structured, approximately 60- minute interview. Interviews will focus on functional abilities and independence. Caregivers and boys with DMD will be interviewed. This study includes no treatment nor intervention; however, some participants are being treated by a drug that is approved in the U.S. and the U.K. and under investigation in other geographies.
NCT06900049
The purpose of this study is to evaluate the safety, tolerability, and efficacy of LE051 intravenous therapy in DMD patients treated with exon 51 skipping therapy.
NCT06412328
Having and caring for a child with disabilities brings emotional, social and economic difficulties for many families. Families may experience many physiological and psychological problems due to the stress and anxiety they experience. In addition, it is seen that families with children with disabilities give up their existing roles, reduce their participation in social activities, and reach stagnation in their social lives. Mothers are affected psychologically more than fathers and feel lonely. It is stated that mothers believe that they cannot afford everything in the face of the responsibilities they carry and accordingly, they experience emotional and psychological problems such as stress, anxiety, depression, absent-mindedness, forgetfulness and tantrums. Living with a child with a disability causes family members to experience different emotions as mentioned above; families may frequently experience fear, anxiety, guilt, anger and depression. It is reported that mothers of children with DMD experience depression, anxiety about the future and uncertainty more than mothers of healthy children. Families of children with DMD reported that they felt tired and fatigued during the process of caring for the child and had difficulties in participating in social activities and allocating time for themselves. Most of these families stated that they needed psychological and social support. Therefore, it is important to address the psychiatric aspects of families with children with DMD during the disease process. Parental health contributes positively to the health and adaptation of the family in general. Examining the psychiatric symptoms caused by the problems experienced by families related to DMD and how they cope with this stress will be useful in evaluating and addressing these families. In addition, the social support that families with children with disabilities receive from their immediate environment and institutions is also an important issue. It has been reported that social support from relatives, friends, neighbors, organizations and communities increases the psychological resilience levels of families, they feel that they are not alone in the face of problems, and their anxiety levels decrease. In the literature, it is generally mentioned that when the culture of pediatric care is supportive and family-oriented, the care of the patient will undergo a change when transitioning from pediatric care to the adult period. However, studies evaluating the problems experienced by families in the care of patients with DMD, psychiatric symptoms, ways of coping with stress and perceived social support are insufficient. It is important to evaluate the problems experienced by parents in the families of children with DMD in developing skills to cope with the disease process and disease-related problems, and then to provide training in these areas. Because if parents, who are in the role of caregivers, are equipped with knowledge and skills in this context, they will provide better care and be more useful to their children with DMD. In line with this information, the aim of this study was to evaluate the problems experienced by parents of children with DMD, psychiatric symptoms, coping skills with stress and the level of social support they perceive and to implement a psychosocial support-based psychoeducation program related to these areas.
NCT06641895
The purpose of the study is to assess the safety, tolerability, and efficacy of BBM-D101 to treat patients with Duchenne Muscular Dystrophy.
NCT02485938
Male participants with cardiomyopathy secondary to Duchenne muscular dystrophy (DMD) meeting all inclusion and no exclusion criteria will be randomized. All participants will be at least 12 years of age. They will be randomized in a 1:1 manner to either intracoronary infusion of CAP-1002 in three coronary arteries supplying the three major cardiac territories of the left ventricle of the heart (anterior, lateral, inferior/posterior) or usual care. In the active treatment arm, all three major cardiac territories will be treated (infused) during a single procedure in an open-label fashion.