Clinical trials: Atrophy
Duchenne's Muscular Dystrophy, Scoliosis
Test-Retest Reliability of Pulmonary Function Tests in Patients With Duchenne's Muscular Dystrophy
Nearly all patients with Duchenne's Muscular Dystrophy (DMD) have scoliosis. Posterior instrumented spinal fusion, which is a surgery to correct scoliosis, has been shown to improve quality of life and satisfaction of both…
- Trial name
- NCT00207857
- Where
- United States
- Sponsor
- Children's Healthcare of Atlanta
Muscular Dystrophy
Clinical Trial Readiness for the Dystroglycanopathies
The purpose of the study is to describe the early signs and symptoms of the dystroglycanopathies, and to gather information that will be required for future clinical trials.
- Trial name
- NCT00313677
- Where
- United States
- Volunteers
- 190 people
- Sponsor
- Katherine Mathews
Spinal Muscular Atrophy
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)
The primary objective of this study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally to participants with later-onset Spinal Muscular Atrophy (SMA).
- Trial name
- CHERISH (NCT02292537)
- Testing
- Nusinersen
- Where
- 24 sites in United States, Canada and 8 more
- Volunteers
- 126 people
- Sponsor
- Biogen
Duchenne Muscular Dystrophy (DMD), Cardiomyopathy
Cardiomyopathy in DMD: Lisinopril vs. Losartan
This trial is a double-blind randomized clinical trial of lisinopril versus losartan for the treatment of cardiomyopathy in Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT01982695
- Testing
- Losartan, Lisinopril
- Where
- 6 sites in United States
- Volunteers
- 23 people
- Sponsor
- Nationwide Children's Hospital
Facioscapulohumeral Muscular Dystrophy
A Multicenter Collaborative Study on the Clinical Features, Expression Profiling, and Quality of Life of Infantile Onset FSHD
This study is an observational study that aims to advance our knowledge on infantile onset FSHD. The study will include 50 participants of all ages who have presented with symptoms of FSHD between birth and 10 years of age.
- Trial name
- FSHD (NCT01437345)
- Where
- 12 sites in United States, Australia and 3 more
- Volunteers
- 53 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Muscular Atrophy, Spinal
A Study of the Safety of Nusinersen (Spinraza®) and Its Effects on Motor Function in Koreans Living With SMA Treated During Their Usual Medical Care
In this study, researchers will learn more about the effects of nusinersen, also known as Spinraza®, in Korean participants with spinal muscular atrophy, also known as SMA.
- Trial name
- STANDARD (NCT04317794)
- Testing
- Nusinersen Sodium Injection
- Where
- 16 sites in South Korea
- Volunteers
- 74 people
- Sponsor
- Biogen
Coeliac Disease
Diagnostic Accuracy of IgA Anti-transglutaminase Antibodies Measured by CLIA for Predicting Villous Atrophy in Coeliac Disease
Celiac disease (CD) is an autoimmune disorder in which ingestion of gluten leads to immune-mediated damage of the small intestine. Diagnosis has traditionally required histological confirmation of duodenal villous atrophy.
- Trial name
- NCT07364461
- Where
- Spain
- Volunteers
- 765 people, healthy volunteers welcome
- Sponsor
- Hospital Mutua de Terrassa
Spinal Muscular Atrophy
An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)
This objectives of this study are to evaluate the safety, tolerability, and pharmacokinetics of a single dose of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA).
- Trial name
- SMNRx (NCT01494701)
- Testing
- nusinersen
- Where
- 4 sites in United States
- Volunteers
- 28 people
- Sponsor
- Biogen
Sarcopenia
Identification of Risk Phenotypes for Sarcopenia Using DXA WBC, Anthropometric and MA Among Young Adults
This cross-sectional study aims to identify risk phenotypes for sarcopenia among young adults using Dual-energy X-ray Absorptiometry (DXA) for whole-body composition analysis, along with anthropometric and musculoskeletal…
- Trial name
- NCT06739850
- Testing
- Identification of Risk Phenotypes of Sarcopenia
- Where
- Pakistan
- Volunteers
- 221 people
- Sponsor
- Superior University
Muscular Dystrophy, Duchenne, Muscular Dystrophies
An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
The primary objective of this study is to obtain long term safety data of ataluren in male participants with nonsense mutation dystrophinopathy (who participated and completed a previous Phase 3 study of ataluren…
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- NCT02090959
- Testing
- Ataluren
- Where
- 58 sites in United States, Australia and 17 more
- Volunteers
- 219 people
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy
Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients
The specific aim of this Phase I/II study is to assess the safety of intravenous administered Morpholino oligomer directed against exon 51 (AVI-4658 PMO).
- Trial name
- NCT00844597
- Testing
- AVI-4658 for Injection
- Where
- 2 sites in United Kingdom
- Volunteers
- 19 people
- Sponsor
- Sarepta Therapeutics, Inc.
Myotonic Dystrophy 1, Huntington Disease
NIPD on cffDNA for Triplet Repeat Diseases
The purprose of this study is to develop and validate an analytical NIPD test for triplet repeat disesases by NGS analysis from maternal blood, searching for the familial mutation in families at risk of having one of the…
- Conditions
- Myotonic Dystrophy 1, Huntington Disease, Fragile X Syndrome
- Trial name
- NCT04698551
- Testing
- Non invasive prenatal diagnosis
- Where
- France
- Volunteers
- 36 people
- Sponsor
- University Hospital, Montpellier
Duchenne Muscular Dystrophy (DMD)
E-monitoring of PULMonary Function in Patients With Duchenne Muscular Dystrophy at Home"
Duchenne muscular dystrophy (DMD) is the most common, progressive, irreversible muscular dystrophy. The pulmonary function is crucial for the duration of life in this disease.
- Trial name
- E-PULMoDMD (NCT05516745)
- Testing
- AioCare spirometer, telerehabiliation of the respiratory system
- Where
- Poland
- Volunteers
- 200 people
- Sponsor
- Medical University of Gdansk
Muscular Dystrophies
Device for Breathing Frequency Monitoring in Muscular Dystrophy
Patients at risk of developing respiratory dysfunctions, such as patients with severe forms of muscular dystrophy, need a careful respiratory assessment, and periodic follow-up visits to monitor the progression of the disease.
- Trial name
- NCT04313595
- Testing
- RespirHó
- Where
- Italy
- Volunteers
- 15 people
- Sponsor
- IRCCS Eugenio Medea
Stargardt Disease
The Natural History of the Progression of Atrophy Secondary to Stargardt Disease Type 4: PROM1-Related Macular Dystrophy
While a fair amount of clinical data on Stargardt disease type 1 (STGD1) have been published, very little is known about Stargardt disease type 4 (STGD4).
- Trial name
- ProgStar-4 (NCT02410122)
- Where
- 5 sites in United States, Germany and 1 more
- Volunteers
- 15 people
- Sponsor
- Johns Hopkins University
Gingival Recession, Wounds and Injuries
The Incidence of Gingival Fissures _ a Crossover Single-blinded Randomized Clinical Trial
The objective of this study is to evaluate the incidence of gingival fissures according to the type of brush used, soft and medium.
- Trial name
- NCT01636830
- Testing
- Toothbrush type
- Where
- Brazil
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Federal University of Rio Grande do Sul
Duchenne Muscular Dystrophy (DMD)
A Study of PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
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).
- Trial name
- CONNECT2-EDO51 (NCT06833931)
- Testing
- IV infusion
- Sponsor
- PepGen Inc
Increased Lordosis/Scoliosis, Hyporeflexia
Biomarker for Duchenne Muscular Dystrophy
International, multicenter, observational, longitudinal study to identify biomarker/s for Duchenne Muscular Dystropy (DMD) and to explore the clinical robustness, specificity, and long-term variability of these biomarker/s.
- Conditions
- Increased Lordosis/Scoliosis, Hyporeflexia, Duchenne Muscular Dystrophy, Red-Green Color Blindness
- Trial name
- BioDuchenne (NCT02994030)
- Where
- 12 sites in Albania, Egypt and 6 more
- Volunteers
- 103 people
- Sponsor
- CENTOGENE GmbH Rostock
Muscular Atrophy, Spinal
A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)
In this PIERRE-PK study, researchers will learn how the body processes nusinersen when it is given through the ThecaFlex DRx™ System, compared to when nusinersen is given by lumbar puncture (LP).
- Trial name
- PIERRE-PK (NCT06555419)
- Testing
- Nusinersen, ThecaFlex DRx System
- Where
- 21 sites in United States, France and 5 more
- Volunteers
- 78 people
- Sponsor
- Biogen
Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy
Duchenne Electronic Health Record Study
This study aims to collect retrospective and prospective, long-term data of patients with dystrophinopathy (including Duchenne, Becker, and female carriers) through electronic transfer.
- Conditions
- Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy, Dystrophinopathy, Dystrophinopathy Symptomatic Female Carrier
- Trial name
- NCT07609394
- Testing
- Observational study with patients who may be treated with various disease-modifying therapies
- Where
- 11 sites in United States
- Volunteers
- 2,500 people
- Sponsor
- The Duchenne Registry
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