Clinical trials: Atrophy
Limb Girdle Muscular Dystrophy, Type 2I, Limb Girdle Muscular Dystrophy
Limb-Girdle Muscular Dystrophy Type 2I in Norway
Key goals are to establish the natural history of limb-girdle muscular dystrophy type 2I (LGMD 2I) and identify feasible and sensitive tools and biomarkers to measure disease affection and progression, determine the Norwegian…
- Conditions
- Limb Girdle Muscular Dystrophy, Type 2I, Limb Girdle Muscular Dystrophy, Muscular Dystrophies, Limb Girdle Muscular Dystrophy R9 FKRP-related
- Trial name
- NCT03930628
- Where
- Norway
- Volunteers
- 106 people
- Sponsor
- University Hospital of North Norway
Duchenne Muscular Dystrophy
Halt cardiomyOPathy progrEssion in Duchenne (HOPE-OLE)
This Phase 2, multi-center, open-label extension trial will provide CAP-1002 to participants who were randomized to the Usual Care treatment group of the HOPE-Duchenne study (NCT02485938) and completed 12 months of follow-up.
- Trial name
- HOPE-OLE (NCT06304064)
- Testing
- Allogeneic Cardiosphere-Derived Cells (CAP-1002)
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Capricor Inc.
Spinal Muscular Atrophy (SMA)
Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).
The study objective is to determine the real-world safety and effectiveness of Vesemnogene lantuparvovec for the treatment of SMA.
- Trial name
- NCT07265232
- Testing
- vesemnogene lantuparvovec
- Where
- Indonesia
- Sponsor
- Lantu Biopharma
Spinal Muscular Atrophy
A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy
This is a multi-center, exploratory, non-comparative, and open-label study to investigate the safety, tolerability, PK, and PK/PD relationship of risdiplam in adults, children and infants with Spinal Muscular Atrophy (SMA)…
- Trial name
- Jewelfish (NCT03032172)
- Testing
- Risdiplam
- Where
- 24 sites in United States, Belgium and 7 more
- Volunteers
- 174 people
- Sponsor
- Hoffmann-La Roche
Scars, Insulin
Microneedling With Regular Insulin Versus Microneedling Alone in Treatment of Atrophic Scars
Recently, few studies have attempted to test the regenerative effects of human insulin application by microneedling on atrophic scars versus other topical preparations. However, the scars were limited etiologically to acne scars.
- Trial name
- NCT06021275
- Testing
- Microneedling with topical application of regular insulin, Microneedling with topical application of saline
- Where
- Egypt
- Volunteers
- 40 people
- Sponsor
- Alexandria University
Multiple System Atrophy (MSA)
Expanded Access Protocol of Verdiperstat in Patients With Multiple System Atrophy (MSA)
The purpose of this expanded access program is to provide access to the investigational drug verdiperstat in patients with Multiple System Atrophy (MSA).
- Trial name
- NCT05086094
- Testing
- BHV3241, verdiperstat
- Where
- United States
- Sponsor
- Biohaven Pharmaceuticals, Inc.
Stargardt Disease
A Natural History of the Progression of Stargardt Disease: Retrospective and Prospective Studies
Stargardt disease is currently an incurable and untreatable macular dystrophy that causes severe visual loss in children and young adults, thereby causing enormous morbidity with economic, psychological, emotional, and social…
- Trial name
- ProgSTAR (NCT01977846)
- Where
- 9 sites in United States, France and 2 more
- Volunteers
- 259 people
- Sponsor
- Foundation Fighting Blindness
Duchenne Muscular Dystrophy
Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to see whether PRO044 is safe and effective to use as medication for Duchenne Muscular Dystrophy (DMD) patients with a mutation around location 44 in the DNA for the dystrophin protein.
- Trial name
- NCT02329769
- Testing
- PRO044 SC 6 mg/kg, PRO044 IV 6 mg/kg, PRO044 IV 9 mg/kg
- Where
- 5 sites in Belgium, Italy and 2 more
- Volunteers
- 15 people
- Sponsor
- BioMarin Pharmaceutical
Spinal Muscular Atrophy 1
A Clinical Study Evaluating the Safety and Efficacy of SKG0201 Injection in Patients With Spinal Muscular Atrophy Type 1
This is a clinical study to evaluate the safety and efficacy of gene therapy drug SKG0201 Injection in patients with spinal muscular atrophy Type 1 (SMA 1).
- Trial name
- NCT06191354
- Testing
- SKG0201 Injection
- Where
- 3 sites in China
- Volunteers
- 12 people
- Sponsor
- Kun Sun
Duchenne Muscular Dystrophy
Digestive Events in Duchenne Muscular Dystrophy Patients
Relation between clinical and genetic features and acute digestive events in Duchenne muscular dystrophy patients
- Trial name
- DIGD (NCT04384354)
- Where
- France
- Volunteers
- 200 people
- Sponsor
- Centre d'Investigation Clinique et Technologique 805
Spinal Muscular Dystrophy, Neuromuscular Disability
Effects of Standing on Non-Ambulatory Children With Spinal Muscular Atrophy
Non-ambulatory children with a neuromuscular disability such as spinal muscular atrophy (SMA) are at significant risk for poor bone health as defined by low bone mineral density (BMD) and increased propensity to fracture.
- Trial name
- NCT02341053
- Testing
- Assisted Standing Treatment Program
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Gillette Children's Specialty Healthcare
Spinal Muscular Atrophy
Mechanisms and Treatment of Exercise Intolerance and Persistent Fatigue in Spinal Muscular Atrophy
This study will focus on the pathophysiological underpinnings of reduced exercise capacity and fatigue in ambulatory patients with spinal muscular atrophy (SMA).
- Trial name
- NCT05518773
- Testing
- Observational
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- Columbia University
Duchenne Muscular Dystrophy (DMD)
DMD Voice: Qualitative Interviews With Patients and Caregivers
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.
- Trial name
- NCT06925269
- Where
- United States
- Volunteers
- 68 people
- Sponsor
- Red Nucleus Enterprise Solutions, LLC
Duchenne Muscular Dystrophy
Tamoxifen in Duchenne Muscular Dystrophy
A randomised, double blind, placebo controlled, 48-week clinical trial with a core population (group A) of 79 ambulant 6.5 to 12 years old Duchenne's muscular dystrophy (DMD) patients that are under stable standard treatment of…
- Trial name
- TAMDMD (NCT03354039)
- Testing
- Tamoxifen
- Where
- 11 sites in France, Germany and 4 more
- Volunteers
- 93 people
- Sponsor
- University Hospital, Basel, Switzerland
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
SMA - Spinal Muscular Atrophy
Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase III
This trial employs a multicenter, randomized, open-label, standard-of-care-controlled design and plans to enroll 50 patients with Type 2 SMA aged 2 to 12 years who have previously received nusinersen.
- Trial name
- NCT06971094
- Testing
- GC101 adeno-associated virus injection
- Where
- 7 sites in China
- Volunteers
- 50 people
- Sponsor
- GeneCradle Inc
Duchenne Muscular Dystrophy
Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD)
To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids in participants with non-ambulatory Duchenne muscular dystrophy (age 12 years and older).
- Trial name
- LELANTOS-1 (NCT04371666)
- Testing
- Pamrevlumab, Corticosteroids
- Where
- 53 sites in United States, Australia and 12 more
- Volunteers
- 98 people
- Sponsor
- Kyntra Bio
Bone Loss, Alveolar, Tooth Loss
Tooth Autotransplantation and Bone Dimension Changes
Tooth autotransplantation is a treatment option to replace tooth with imposible prognosis. In several cases the receipt site has a reduced bone dimension.
- Conditions
- Bone Loss, Alveolar, Tooth Loss, Bone Atrophy, Alveolar
- Trial name
- NCT03927937
- Testing
- AUTOTRANSPLANTATION TOOTH
- Where
- Spain
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Universidad de Murcia
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, Becker Muscular Dystrophy
Brain Involvement in Dystrophinopathies Part 2
The objective of this study is to understand the relationship between DMD and BMD brain comorbidities, and the location of the gene mutation which causes the disease.
- Trial name
- NCT04668716
- Where
- 7 sites in Denmark, France and 4 more
- Volunteers
- 339 people, healthy volunteers welcome
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
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