Clinical trials: Atrophy
Duchenne Muscular Dystrophy
Heart Rate Variability in Individuals With Duchenne Muscular Dystrophy
A cross-sectional study was carried out, in which 40 boys, aged 11 to 18 years, were evaluated. The recruitment of groups was carried out at the neuromuscular disease outpatient clinic of the Federal University of São Paulo…
- Trial name
- NCT04740554
- Testing
- Duchenne Muscular Dystrophy group with Deflazacort, Duchenne Muscular Dystrophy group with Prednisone/Predisolone, Duchenne Muscular Dystrophy group without Corticosteroids therapy
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo
Duchenne Muscular Dystrophy
Phase 1/2a for Safety, PK and PD of SQY51 in Paediatric and Adult Patients Duchenne Muscular Dystrophy
This is a Phase 1/2a, monocentric, open label study to evaluate the safety, pharmacokinetics, and pharmacodynamics of SQY51 in patients with Duchenne muscular dystrophy
- Trial name
- AVANCE1 (NCT05753462)
- Testing
- Phase 1, SQY51, Phase 2a, SQY51 (cohort 1), Phase 2a, SQY51 (cohort 2)
- Where
- France
- Volunteers
- 12 people
- Sponsor
- Sqy Therapeutics
Spinal Muscular Atrophy
Implementation of FEES in Spinal Muscle Atrophy
The major aim of this project is to assess comprehensively frequency and extent of dysphagia and bulbar dysfunction in SMA1, 2, and 3 patients by applying FEES and validated dysphagia scores.
- Trial name
- DYS-SMA (NCT04773470)
- Testing
- Flexible Endoscopic Evaluation of Swallowing
- Where
- Germany
- Volunteers
- 79 people
- Sponsor
- University of Giessen
Myotonic Dystrophy 1
An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE)
The purpose of this study is to learn about the long-term safety and tolerability of PGN-EDODM1 in participants with myotonic dystrophy type 1 (DM1) who have completed a prior study with PGN-EDODM1.
- Trial name
- NCT07220603
- Testing
- PGN-EDODM1
- Where
- 3 sites in Canada
- Volunteers
- 48 people
- Sponsor
- PepGen Inc
Muscular Atrophy, Spinal
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene…
- Trial name
- HINALEA 2 (NCT05861999)
- Testing
- Risdiplam
- Where
- 17 sites in United States, Germany and 4 more
- Volunteers
- 19 people
- Sponsor
- Hoffmann-La Roche
Duchenne Muscular Dystrophy
Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
This is a multicenter, open-label, long-term, extension, phase 2 study to evaluate the safety and efficacy of long-term treatment with DS-5141b in patients with DMD who have completed DS5141-A-J101.
- Trial name
- NCT04433234
- Testing
- DS-5141b
- Where
- 2 sites in Japan
- Volunteers
- 8 people
- Sponsor
- Daiichi Sankyo Co., Ltd.
Dystrophinopathy, Duchenne Muscular Dystrophy
Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on Corticosteroids
The purpose of the study is to assess the safety, tolerability, and effects of L-Arginine on muscles in boys with dystrophinopathy on corticosteroids.
- Conditions
- Dystrophinopathy, Duchenne Muscular Dystrophy, Becker's Muscular Dystrophy
- Trial name
- NCT01388764
- Testing
- L-arginine
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Massachusetts General Hospital
Neuro-Ophthalmology, Optic Neuropathies
Optic Nerve Head Evaluation Through Multimodal Blood Flow Analysis: a Prospective Observational, Multi-center Study
OCT-A and eco-colordoppler are technologies which enable optic nerve head (ONH) blood perfusion detection. The aim of the study is to assess ONH vascular and structural components in optic neuropathies with and without ONH edema…
- Conditions
- Neuro-Ophthalmology, Optic Neuropathies, Optic Atrophy, Hereditary, Leber, Optic Neuropathy, Anterior Ischemic
- Trial name
- FlowONH (NCT07075887)
- Volunteers
- 70 people
- Sponsor
- Barboni
Muscular Dystrophy, Duchenne
Double Push Acoustic Radiation Force (DP ARF) Ultrasound for Monitoring Degeneration in Duchenne Muscular Dystrophy
This is a pilot clinical trial to assess the ability of a new ultrasound-based imaging method, Double-Push Acoustic Radiation Force (DP ARF) ultrasound, to monitor the progression of Duchenne muscular dystrophy.
- Trial name
- NCT01506518
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- University of North Carolina, Chapel Hill
Muscular Dystrophy, Duchenne
An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen)
The purpose of this extension study is to evaluate the safety, tolerability, and pharmacokinetics of repeat administrations of SRP-5051 (vesleteplirsen) in participants with Duchenne muscular dystrophy (DMD) who participated in…
- Trial name
- NCT03675126
- Testing
- SRP-5051
- Where
- 8 sites in United States, Canada
- Volunteers
- 15 people
- Sponsor
- Sarepta Therapeutics, Inc.
Muscular Dystrophies
LARA: Use of JACO2 Controlled by Joystick or by Voice Control System, in Muscular Dystrophy
As part of the EMPATIA @ Lecco project, task 2.9 and task 4.5 respectively provide for the refinement and testing of robotic solutions on the market oriented towards patient empowerment.
- Trial name
- NCT04313049
- Testing
- JACO2, VOCAL CONTROL
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- IRCCS Eugenio Medea
Gyrate Atrophy, Gyrata of Choroid and Retina; Atrophy
Gyrate Atrophy Ocular and Systemic Study
The Gyrate Atrophy Ocular and Systemic Study characterizes the natural history of ornithine levels and retinal degeneration (RD) associated with disease-causing OAT variants in the presence of standard care dietary treatment…
- Conditions
- Gyrate Atrophy, Gyrata of Choroid and Retina; Atrophy, Ornithine-δ-aminotransferase, OAT
- Trial name
- GYROS (NCT05312736)
- Where
- 11 sites in United States, Brazil and 6 more
- Volunteers
- 46 people
- Sponsor
- Jaeb Center for Health Research
Vaginitis Atropic, Pelvic Organ Prolapse
Vaginal Estrogens Comparative Trial on Pelvic Organ Prolapse Patients
To evaluate the difference of three vaginal estrogens creams comparative with placebo on improvement of hormonal cytology, local and systemic climacteric complaints, as well as its endometrial security.
- Conditions
- Vaginitis Atropic, Pelvic Organ Prolapse, Endometrial Hyperplasia
- Trial name
- NCT03032848
- Testing
- Conjugated Estrogen, Promestriene, Estriol
- Volunteers
- 69 people
- Sponsor
- Universidade Federal do Paraná
Infantile-onset Spinal Muscular Atrophy
Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)
To provide access to nusinersen to eligible patients with Infantile-onset Spinal Muscular Atrophy (SMA) (consistent with Type 1) to address a high-unmet medical need.
- Trial name
- NCT02865109
- Testing
- Nusinersen
- Where
- 7 sites in Colombia, New Zealand and 1 more
- Sponsor
- Biogen
Muscular Dystrophy, Duchenne
Study of Genotype and Phenotype Characterization in Duchenne Muscular Dystrophy With Small Mutations
Improved standards of care and the regular early use of glucocorticoid treatment have changed the natural history of Duchenne muscular dystrophy (DMD), affecting both survival and time of loss of functional milestones.
- Trial name
- NCT05833633
- Testing
- MUSCLE MAGNETIC RESONANCE IMAGING (MRI)
- Where
- Italy
- Volunteers
- 17 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Atopic Dermatitis
Opzelura Experience Study
Atopic dermatitis is a common multifactorial skin disease. Topicals are the mainstay of treatment, but adherence to topicals is often abysmal, possibly worsened by the complexity of recommended treatment algorithms.
- Trial name
- NCT07661979
- Testing
- ruxolitinib, ruxolitinib and an Eczema Action Plan
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Wake Forest University Health Sciences
Limb-girdle Muscular Dystrophy
A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4
This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4).
- Trial name
- EMERGENE (NCT06246513)
- Testing
- SRP-9003, Glucocorticoid
- Where
- 10 sites in United States, Belgium and 4 more
- Volunteers
- 17 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping
We will utilize the Cooperative International Neuromuscular Research Group (CINRG) network to collect and store tissue and blood from patients with Duchenne muscular dystrophy (DMD) with specific genetic mutations within the…
- Trial name
- NCT01772043
- Where
- 9 sites in United States, Canada
- Volunteers
- 53 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Spinal Muscular Atrophy
A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND)
In this study, researchers will learn more about the use of a higher dose of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA).
- Trial name
- ASCEND (NCT05067790)
- Testing
- Nusinersen
- Where
- 22 sites in United States, Germany and 3 more
- Volunteers
- 45 people
- Sponsor
- Biogen
SMA
Study to Evaluate the Efficacy of Riluzole in Children and Young Adults With Spinal Muscular Atrophy (SMA)
This is a multicentric, randomized, double-blind study versus placebo, with two parallel groups treated to evaluate the efficacy and the tolerance of Riluzole in children and young adults (6 to 20 years of age) with SMA.
- Trial name
- ASIRI (NCT00774423)
- Testing
- Riluzole
- Where
- France
- Volunteers
- 141 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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