Clinical trials: Atrophy
Spinal Muscular Atrophy
A Registry of Cases of Spinal Muscular Atrophy
Because no long-term record of prenatal Spinal Muscular Atrophy (SMA) treatment exists, this study will address an important gap in the understanding of the safety and benefit of treatment while in the womb.
- Trial name
- NCT07805278
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- St. Jude Children's Research Hospital
Muscular Dystrophy, Facioscapulohumeral
Digital Biotyping of FSHD Patients and Controls
Facioscapulohumeral muscular dystrophy (FSHD) is a devastating progressive muscle dystrophy. There is no treatment. FSHD is generally characterized by asymmetrical weakness and wasting of facial, shoulder girdle and upper arm…
- Trial name
- NCT04999735
- Testing
- CHDR Monitoring Remotely (MORE), Withings Steel HR, Withings Body+ scale
- Where
- Netherlands
- Volunteers
- 58 people, healthy volunteers welcome
- Sponsor
- Centre for Human Drug Research, Netherlands
Myotonic Dystrophy, Duchenne Muscular Dystrophy
Biomarker Development for Muscular Dystrophies
Current methods of measuring the response to new treatments for muscular dystrophies involve the examination of small pieces of muscle tissue called biopsies.
- Conditions
- Myotonic Dystrophy, Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy
- Trial name
- NCT05019625
- Where
- 5 sites in United States
- Volunteers
- 465 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Sarcopenia, Physical Fitness
Swedish Physical Activity and Fitness Longitudinal Study
Aging means loss of strength, which can affect the ability to lead an active life and be independent. Physical activity can counteract the loss of strength to some extent, but individual differences are large and the mechanisms…
- Conditions
- Sarcopenia, Physical Fitness, Muscle Strength, Dynapenia
- Trial name
- SPAF (NCT06496204)
- Where
- Sweden
- Volunteers
- 429 people, healthy volunteers welcome
- Sponsor
- Karolinska Institutet
Duchenne Muscular Dystrophy
A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
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.
- Trial name
- NCT07160634
- Testing
- SGT-003
- Where
- 7 sites in United States, Australia and 1 more
- Volunteers
- 80 people
- Sponsor
- Solid Biosciences Inc.
Duchenne Muscular Dystrophy, Cardiomyopathy
HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne)
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.
- Trial name
- HOPE (NCT02485938)
- Testing
- Allogeneic Cardiosphere-Derived Cells (CAP-1002)
- Where
- 3 sites in United States
- Volunteers
- 25 people
- Sponsor
- Capricor Inc.
Eye Diseases Hereditary, Retinal Disease
Inherited Retinal Degenerative Disease Registry
The My Retina Tracker® Registry is sponsored by the Foundation Fighting Blindness and is for people affected by one of the rare inherited retinal degenerative diseases studied by the Foundation.
- Conditions
- Eye Diseases Hereditary, Retinal Disease, Achromatopsia, Bardet-Biedl Syndrome
- Trial name
- MRTR (NCT02435940)
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Foundation Fighting Blindness
Duchenne Muscular Dystrophy, Upper Extremity Problem
Reliability and Validity of the Turkish Version of the Upper Limb Short Questionnaire in Duchenne Muscular Dystrophy
Purpose: This study aimed to evaluate the construct validity and reliability of the Turkish version of the Upper Limb Short Questionnaire (ULSQ) in Duchenne muscular dystrophy (DMD).
- Trial name
- NCT05274555
- Testing
- Assessment
- Where
- Turkey (Türkiye)
- Volunteers
- 41 people
- Sponsor
- Dokuz Eylul University
Duchenne Muscular Dystrophy
Peak Cough Flow and Cough Clearance in Patients With Muscular Dystrophy
This study is to determine whether physiologic measures (peak cough flow, measures of respiratory muscle strength including MIP, MEP ,SNIP, and spirometry) can predict spontaneous cough clearance (as measured by a nuclear…
- Trial name
- NCT02034305
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- University of Pittsburgh
Spinal Muscular Atrophy, Neuromuscular Diseases
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
This trial will study the efficacy and safety of taldefgrobep alfa as an adjunctive therapy for participants who are already taking a stable dose of nusinersen and/or risdiplam and/or have a history of onasemnogene abeparvovec…
- Conditions
- Spinal Muscular Atrophy, Neuromuscular Diseases, SMA
- Trial name
- RESILIENT (NCT05337553)
- Testing
- taldefgrobep alfa
- Where
- 53 sites in United States, Belgium and 7 more
- Volunteers
- 269 people
- Sponsor
- Biohaven Pharmaceuticals, Inc.
Muscular Dystrophies
Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
The purpose of this study is to explore long-term safety, tolerability and efficacy of GSK2402968 in DMD subjects who previously participated in either DMD114117 or DMD114044.
- Trial name
- NCT01480245
- Testing
- GSK2402968
- Where
- 59 sites in Argentina, Australia and 22 more
- Volunteers
- 233 people
- Sponsor
- GlaxoSmithKline
Duchenne Muscular Dystrophy (DMD)
Virtual Reality and Whole Body Vibration on Muscle Strength and Balance in Children With DMD
we measure balance and quadriceps strength in Duchenne muscular dystrophy children after applying virtual reality and whole body vibration
- Trial name
- NCT07338812
- Testing
- virtual reality, whole body vibration
- Where
- 2 sites in Egypt
- Volunteers
- 50 people
- Sponsor
- Cairo University
Duchenne Muscular Dystrophin (DMD)
An Open-label, Multidose Dose-escalation Study to Understand the Safety of CRISPR Gene-editing Therapy and Its Long-Lasting Effects in DMD Patients (MUSCLE)
Duchenne muscular dystrophin (DMD) is an X-linked, fatal muscle-wasting disease caused by mutations in the DMD gene encoding the dystrophin proteins, with symptom onset before age of 6 years in boys.
- Trial name
- MUSCLE (NCT06594094)
- Testing
- HG302
- Where
- China
- Volunteers
- 4 people
- Sponsor
- HuidaGene Therapeutics Co., Ltd.
Age Related Macular Degeneration
Survival of Monocytes Collected From Patients With Atrophic AMD in Retinal Pigmented Epithelium Explants
Age-related macular degeneration (AMD) affects 2 million people in France and is the main cause of irreversible blindness in France. All patients initially have an early form of the disease.
- Trial name
- SURViVOR (NCT04697095)
- Testing
- Blood samples
- Where
- 2 sites in France
- Volunteers
- 80 people
- Sponsor
- Hospices Civils de Lyon
Type 2 Spinal Muscular Atrophy, Type 3 Spinal Muscular Atrophy
Prospective Study of the Natural History of Patients With Type 2 and 3 Spinal Muscular Atrophy
NatHis-SMA is a prospective, longitudinal and interventional study of the natural history of patients with type 2 and 3 Spinal Muscular Atrophy (SMA).
- Trial name
- NatHis-SMA (NCT02391831)
- Testing
- Strength, function and activity measurements, Muscle MRI, Electrophysiology measurements
- Where
- 9 sites in Belgium, France and 1 more
- Volunteers
- 81 people
- Sponsor
- Institut de Myologie, France
Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy
Registry for Duchenne and Becker Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is an X-linked, recessive, progressive, and degenerative neuromuscular disorder that affects approximately one in 5,000 newborn boys.
- Conditions
- Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy, Dystrophinopathy Symptomatic Female Carrier
- Trial name
- FAIR-DMD (NCT07402122)
- Where
- 30 sites in Austria, Germany
- Volunteers
- 1,500 people
- Sponsor
- Dr. Andreas Ziegler
Spinal Muscular Atrophy
Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
A multi-center, longitudinal, prospective, non-comparative study to investigate the long-term safety and effectiveness of risdiplam, prescribed based on clinician judgment as per the Evrysdi® U.S.
- Trial name
- WeSMA (NCT05232929)
- Testing
- Risdiplam
- Where
- 38 sites in United States, Puerto Rico
- Volunteers
- 403 people
- Sponsor
- Genentech, Inc.
Spinal Muscular Atrophy Type I
CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I
This is a multi-center trial to test safety and evaluate early treatment intervention with valproic acid and carnitine in moderating SMA symptoms of Type I infants.
- Trial name
- NCT00661453
- Testing
- Valproic Acid and Levocarnitine
- Where
- 8 sites in United States, Canada and 1 more
- Volunteers
- 40 people
- Sponsor
- University of Utah
Sarcopenia, Muscle Loss
Comparison of Computed Tomography Data With Routine Measurements Concerning Bone and Muscle Health of Aged Individuals
This study focuses on researching sarcopenia and bone loss (osteoporosis), aiming to develop early and effective methods for diagnosis and treatment.
- Conditions
- Sarcopenia, Muscle Loss, Osteoporosis
- Trial name
- SARCATAR (NCT06488872)
- Testing
- comparison of CT scan with routinely assessed methods of muscle mass and strength
- Where
- Switzerland
- Volunteers
- 300 people
- Sponsor
- University Department of Geriatric Medicine FELIX PLATTER
Atypical Parkinsonism, Dementia With Lewy Bodies
Focused Ultrasound Thalamotomy for Tremor Relief in Atypical Parkinsonism
The goal of this retrospective observational study is to describe the efficacy of focused ultrasound ventral-intermediate nucleus thalamotomy in patients with atypical parkinsonism.
- Conditions
- Atypical Parkinsonism, Dementia With Lewy Bodies, Multiple System Atrophy, MSA
- Trial name
- FUSAP (NCT06204575)
- Testing
- Data collection
- Where
- Israel
- Volunteers
- 8 people
- Sponsor
- Rambam Health Care Campus
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.