Clinical trials: Atrophy
Spinal Muscular Atrophy
Evaluation of the Muscle Strength and Motor Ability in Children With Spinal Muscle Atrophy(SMA) Treated With Valproic Acid
The purpose of this study is to determine if the treatment with valproic acid can increase the muscle strength and motor ability of children with spinal muscular atrophy.
- Trial name
- NCT01033331
- Where
- Brazil
- Volunteers
- 22 people
- Sponsor
- University of Sao Paulo General Hospital
Spinal Muscular Atrophy
Metabolomics of Children With SMA
The aim of the proposed project is to evaluate whether the metabolome of patients with spinal muscular atrophy (SMA) before the initiation of treatment with nusinersen differs from the metabolome of healthy individuals and…
- Trial name
- NCT04587492
- Testing
- Nusinersen
- Where
- 2 sites in Slovenia
- Volunteers
- 35 people
- Sponsor
- University Medical Centre Ljubljana
Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy Clinical Trial
The primary objective of this investigation is to assess the effectiveness of transcutaneous electrical nerve stimulation applied using VECTTOR to reduce the symptoms of Duchenne Muscular Dystrophy and reduce the impact of DMD…
- Trial name
- DMD (NCT01874275)
- Testing
- VECTTOR
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Alan Neuromedical Technologies, LLC
Scoliosis Neuromuscular, Duchenne Muscular Dystrophy
Prevention of Scoliosis in Patients With Duchenne Muscular Dystrophy Using Portable Seat Device
This study will be conducted without blind method. The portable seat device devised to maintain lumbar lordosis will be made within 1 year after the loss of ambulation in the participants with Duchenne muscular dystrophy with…
- Conditions
- Scoliosis Neuromuscular, Duchenne Muscular Dystrophy, Lordosis Lumbar
- Trial name
- NCT03611244
- Testing
- Portable seat device devised to maintain lumbar lordosis
- Where
- 2 sites in South Korea
- Volunteers
- 98 people
- Sponsor
- Seoul National University Hospital
Age Related Macular Degeneration, Intermediate AMD
A Collaborative Resource of Heidelberg Multimodal Imaging of Intermediate and Early Atrophic AMD Cases to Study Prediction of Disease Progression
This is a multicentre retrospective and prospective cohort study with the goal to develop a well-characterised multimodal image database of eyes with intermediate AMD with and without early atrophy. The main objectives are: 1.
- Conditions
- Age Related Macular Degeneration, Intermediate AMD, Atrophy, Prediction
- Trial name
- INTERCEPT-AMD (NCT05698316)
- Testing
- Collection of both retrospective and prospective data collection in 3 visits.
- Where
- 25 sites in France, Germany and 7 more
- Volunteers
- 1,000 people
- Sponsor
- Association for Innovation and Biomedical Research on Light and Image
Duchenne Muscular Dystrophy
Relationship Between PFTs and Pdi in DMD
A cross-sectional study to explore the relationship between clinically assessed pulmonary function test (PFT) measures and transdiaphragmatic (Pdi) measures in Duchenne muscular dystrophy (DMD) as well as to explore the…
- Trial name
- NCT03335384
- Testing
- Esophageal Balloon, Gastric Balloon, Nasal Pressure Transducer
- Sponsor
- University of Minnesota
Duchenne Muscular Dystrophy, Duchenne
Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients
Phase 2a trial of SAT-3247 in ambulatory DMD patients aged ≥ 7 and < 10 years. The trial has two parts. In Part 1, the trial will study two doses of SAT-3247 in a randomized, double-blind, placebo-controlled weekday regimen for…
- Conditions
- Duchenne Muscular Dystrophy, Duchenne, DMD, Neuromuscular Diseases
- Trial name
- BASECAMP (NCT07287189)
- Testing
- SAT-3247
- Where
- 20 sites in United States, Australia and 6 more
- Volunteers
- 51 people
- Sponsor
- Satellos Bioscience, Inc.
Duchenne Muscular Dystrophy
Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy
The proposed clinical trial study of rAAVrh74.MCK.GALGT2 for duchenne muscular dystrophy (DMD) patients. There will be a modified intravascular limb infusion (ILI) procedure that will be used to sequentially deliver vector to…
- Trial name
- NCT03333590
- Testing
- rAAVrh74.MCK.GALGT2
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Kevin Flanigan
Tarui Disease, Debrancher Deficiency
The Effect of Triheptanoin on Fatty Acid Oxidation and Exercise Tolerance in Patients With Glycogenoses
The aim of this study is to investigate the effect of 14 days of treatment with the dietary oil-supplement Triheptanoin on fat metabolism and exercise tolerance in patients with Phosphofructokinase deficiency, Debrancher…
- Conditions
- Tarui Disease, Debrancher Deficiency, GYG1 DEFICIENCY
- Trial name
- NCT03642860
- Testing
- Triheptanoin
- Where
- Denmark
- Volunteers
- 3 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Neuromuscular Disease, Duchenne Muscular Dystrophy
Feasibility of Virtual Reality in Children With Neuromuscular Disease, Effectiveness of Virtual Reality and Biofeedback
Our study is a randomized controlled study and the subjects included in the study will be divided into three groups as virtual reality training, biofeedback training, and conventional rehabilitation.
- Conditions
- Neuromuscular Disease, Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, Virtual Reality
- Trial name
- NCT03689660
- Testing
- Virtual Reality Training, Biofeedback Training, Conventional rehabilitation
- Where
- Turkey (Türkiye)
- Volunteers
- 24 people
- Sponsor
- Merve Kurt
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.
Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III Non Ambulant
Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Years SMA Patients.
Assess the efficacy and the safety of olesoxime in SMA type 2 or type 3 non ambulant patients aged 3-25 years
- Trial name
- NCT01302600
- Testing
- Olesoxime
- Where
- 22 sites in Belgium, France and 5 more
- Volunteers
- 165 people
- Sponsor
- Hoffmann-La Roche
Leber Hereditary Optic Neuropathy
RESCUE and REVERSE Long-term Follow-up
The goal of this clinical trial is to assess the long-term safety and efficacy of GS010, a gene therapy, and assess the quality of life in subjects with LHON due to the G11778A ND4 mitochondrial mutation and who were treated in…
- Trial name
- RESTORE (NCT03406104)
- Testing
- GS010
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 62 people
- Sponsor
- GenSight Biologics
Duchenne Muscular Dystrophy, Cardiomyopathy, Dilated
Bisoprolol in DMD Early Cardiomyopathy
This study aimed to use cardiac magnetic resonance imaging (CMR) to evaluate the efficacy and safety of bisoprolol therapy for boys with Duchenne muscular dystrophy(DMD) and preserved ejection fraction.
- Trial name
- NCT03779646
- Testing
- Bisoprolol Fumarate
- Where
- China
- Volunteers
- 42 people
- Sponsor
- Peking Union Medical College Hospital
Spinal Muscular Atrophy 1
Long-Term Follow-up Study for Patients From AVXS-101-CL-101
This is a long term, safety follow up study of patients in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering onasemnogene abeparvovec-xioi.
- Trial name
- START (NCT03421977)
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Novartis Gene Therapies
Spinal Muscular Atrophy (SMA)
Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy (SMA) is a neurogenetic disorder caused by a loss or mutation in the survival motor neuron 1 gene (SMN1) on chromosome 5q13, which leads to reduced SMN protein levels and a selective dysfunction of motor…
- Trial name
- NCT04174157
- Testing
- Prospective observational registry, Zolgensma
- Where
- 99 sites in United States, Greece and 9 more
- Volunteers
- 700 people
- Sponsor
- Novartis Pharmaceuticals
Duchenne Muscular Dystrophy (DMD)
An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy
This open-label extension study aims to evaluate the long-term safety and tolerability of weekly BMN 351 infusions, as well as to assess the effect of BMN 351 on physical function, in participants with DMD who participated in the…
- Trial name
- NCT07573631
- Testing
- BMN 351
- Where
- 6 sites in Italy, Netherlands and 3 more
- Volunteers
- 18 people
- Sponsor
- BioMarin Pharmaceutical
Duchenne Muscular Dystrophy
Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular Dystrophy
This study is single arm, single center trial to study the safety and efficacy of bone marrow-derived autologous specific populations of stem cells and mesenchymal stem cells for the treatment of Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT03067831
- Testing
- Stem Cells
- Where
- Jordan
- Volunteers
- 20 people
- Sponsor
- Stem Cells Arabia
Periodontitis, Periodontal Attachment Loss
T-PRF Provides Advantages on Periodontal Healing: A Split Mouth Clinical Study
It was aimed to evaluate the contributions of T-PRF treatment combined with open flap debridement on biological markers in GCF and periodontal outcomes in chronic periodontitis patients.
- Trial name
- NCT03027050
- Testing
- T-PRF, Open Flap Debridement alone
- Volunteers
- 33 people, healthy volunteers welcome
- Sponsor
- Ataturk University
Gingival Recession
Effect of Suturing Protocols on Coronally Advanced Flap for Root Coverage Outcomes
The purpose of this retrospective study is to investigate whether suturing protocols (suture removal timing) influence root coverage outcomes in recession defects treated with a coronally advanced flap (CAF) procedure.
- Trial name
- NCT05472233
- Testing
- Suture removal after Coronally Positioned Flap
- Where
- Italy
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- University of Cagliari
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