Clinical trials: MYOPATHY
Centronuclear Myopathy
Early Phase Human Drug Trial to Investigate Dynamin 101 (DYN101) in Patients ≥ 16 Years With Centronuclear Myopathies
There are no available treatments aside from supportive care for patients with Centronuclear myopathy (CNM). This trial will assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD)/preliminary efficacy of…
- Trial name
- Unite-CNM (NCT04033159)
- Testing
- DYN101
- Where
- 8 sites in Belgium, Denmark and 4 more
- Volunteers
- 14 people
- Sponsor
- Dynacure
Duchenne Muscular Dystrophy
Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy
This is a Phase 2/3, multicenter, randomized, double-blind, placebo-controlled study with an open-label extension period to evaluate the safety and efficacy of WVE-210201 (suvodirsen) in ambulatory male pediatric patients with…
- Trial name
- DYSTANCE 51 (NCT03907072)
- Testing
- WVE-210201 (suvodirsen)
- Where
- 22 sites in United States, Belgium and 6 more
- Volunteers
- 6 people
- Sponsor
- Wave Life Sciences USA, Inc.
Muscular Dystrophy, Duchenne
Effect of EPA and DHA in the Inflammation and Metabolic Disorders in DMD/DMB Patients
The purpose of this study is to evaluate the effect of docosahexaenoic fatty acid and eicosapentaenoic fatty acid supplementation for six months on the inflammation state as well as the process of muscular regeneration and the…
- Trial name
- NCT01826422
- Testing
- EPA and DHA
- Where
- Mexico
- Volunteers
- 40 people
- Sponsor
- Coordinación de Investigación en Salud, Mexico
Very Long-chain Acyl-CoA Dehydrogenase Deficiency, Trifunctional Protein Deficiency
High Protein Diet in Patients With Long-chain Fatty Acid Oxidation Disorders
The study also determines if eating a diet higher in protein alters body composition, energy balance and metabolic control among patients with a long-chain fatty acid oxidation disorder.
- Conditions
- Very Long-chain Acyl-CoA Dehydrogenase Deficiency, Trifunctional Protein Deficiency, Carnitine Palmitoyltransferase 2 Deficiency, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency
- Trial name
- NCT01494051
- Testing
- Diet counseling
- Volunteers
- 13 people
- Sponsor
- Oregon Health and Science University
Duchenne Muscular Dystrophy
Stacking Exercises Aid the Decline in FVC and Sick Time
Duchenne Muscular Dystrophy is complicated by weak breathing muscles and lung infections. "Lung volume recruitment" is a technique performed using a face mask or mouthpiece and a hand-held resuscitation bag to stack breaths…
- Trial name
- STEADFAST (NCT01999075)
- Testing
- Lung Volume Recruitment (LVR), Conventional Treatment
- Where
- 9 sites in Canada
- Volunteers
- 70 people
- Sponsor
- Children's Hospital of Eastern Ontario
Duchenne Muscular Dystrophy
Open-label Extension of the HOPE-2 Trial
This Phase 2, multi-center, open-label extension trial will provide deramiocel (CAP-1002) to subjects that were enrolled in the HOPE-2 trial and completed 12 months of follow-up.
- Trial name
- HOPE-2-OLE (NCT04428476)
- Testing
- Deramiocel (CAP-1002)
- Where
- 5 sites in United States
- Volunteers
- 13 people
- Sponsor
- Capricor Inc.
Myotonic Dystrophy 1
Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy
The purpose of this study is to determine whether Tideglusib is safe and efficacious in the treatment of adolescents and adults with congenital and juvenile-onset Myotonic Dystrophy.
- Trial name
- NCT02858908
- Testing
- Tideglusib
- Where
- United Kingdom
- Volunteers
- 16 people
- Sponsor
- AMO Pharma Limited
Type 1 Diabetes
The Summer Camp Study: Blood Glucose Control With a Bi-Hormonal Bionic Endocrine Pancreas
This study will test the hypothesis that a wearable automated bionic pancreas system that automatically delivers both insulin and glucagon can improved glycemic control vs.
- Trial name
- NCT01833988
- Testing
- Bi-hormonal Bionic Pancreas, Usual Care
- Where
- United States
- Volunteers
- 32 people
- Sponsor
- Massachusetts General Hospital
Limb-Girdle Muscular Dystrophy, Type 2D
A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9004 (Patidistrogene Bexoparvovec) in Participants With Limb-Girdle Muscular Dystrophy, Type 2D (LGMD2D)
This is an open-label, dose escalation gene transfer therapy study evaluating the safety of SRP-9004 (patidistrogene bexoparvovec) via isolated limb infusion (ILI) administration in approximately 6 participants with LGMD2D.
- Trial name
- NCT01976091
- Testing
- SRP-9004
- Volunteers
- 6 people
- Sponsor
- Sarepta Therapeutics, Inc.
Juvenile Dermatomyositis
Five-year Actively Controlled Clinical Trial in New Onset Juvenile Dermatomyositis
This is a 5-year project, involving 185 partners from 46 countries ((110 in 21 European Union (EU) States and 75 in 25 extra-EU States)), with a randomised clinical trials (RCT) in juvenile dermatomyositis (JDM): 5-year phase III…
- Trial name
- PRINTOJDMTR (NCT00323960)
- Testing
- 3 MPDN pulse + PDN, 3 MPDN pulse + PDN + CSA, 3 MPDN pulse + PDN + MTX
- Where
- Italy
- Volunteers
- 139 people
- Sponsor
- Istituto Giannina Gaslini
Intra-Abdominal Hypertension
Intra-Abdominal Hypertension in Neonatal Intensive Care Patients
The observational study is designed to determine the feasibility of performing Intra-Abdominal Pressure (IAP) via bladder pressure measurements and of tracking IAP in neonates who require bladder catheterization.
- Trial name
- NCT00747552
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of Utah
Cerebral Palsy, Muscle Spasticity
Dysport® Pediatric Lower Limb Spasticity Follow-on Study
The purpose of this research study was to determine the long term safety and efficacy of repeated treatments with Dysport® used in the treatment of lower limb spasticity in children with dynamic equinus foot deformity due to…
- Conditions
- Cerebral Palsy, Muscle Spasticity, Children
- Trial name
- NCT01251380
- Testing
- Botulinum toxin type A
- Where
- 27 sites in United States, Chile and 4 more
- Volunteers
- 216 people
- Sponsor
- Ipsen
Fibrodysplasia Ossificans Progressiva
An Efficacy and Safety Study of Palovarotene to Treat Preosseous Flare-ups in FOP Subjects
Fibrodysplasia ossificans progressiva (FOP) is a rare, severely disabling disease characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) that result in abnormal bone formation in muscles, tendons, and…
- Trial name
- NCT02190747
- Testing
- Palovarotene
- Where
- 4 sites in United States, France and 1 more
- Volunteers
- 40 people
- Sponsor
- Clementia Pharmaceuticals Inc.
Blepharospasm, Torticollis
MDs on Botox Utility (MOBILITY)
The MOBILITY Project is a prospective, non-randomized, observational, multi-centre evaluation of Health Utility via the SF-12® Health Survey Scores and the SF-6D in patients receiving BOTOX® for therapeutic use.
- Conditions
- Blepharospasm, Torticollis, Strabismus, Muscle Spasticity
- Trial name
- NCT00535938
- Testing
- botulinum toxin type A
- Where
- Canada
- Volunteers
- 1,372 people
- Sponsor
- Allergan
Irritable Bowel Syndrome (IBS), Functional Abdominal Pain
Peer Mentorship: An Intervention To Promote Effective Pain Self-Management In Adolescents
This protocol matches child subjects with peer mentors of similar age who have learned to function successfully with a chronic pain disorder.
- Conditions
- Irritable Bowel Syndrome (IBS), Functional Abdominal Pain, Fibromyalgia, Complex Regional Pain Syndrome (CRPS)
- Trial name
- NCT01118988
- Testing
- Mentorship
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- University of California, Los Angeles
Pain Associated With Fibromyalgia
Special Investigation of Pregabalin for Fibromyalgia (Regulatory Post Marketing Commitment Plan)
To collect the efficacy and safety information of Pregabalin on Fibromyalgia patients related to their appropriate use in daily practice.
- Trial name
- NCT01773993
- Testing
- Pregabalin
- Volunteers
- 534 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Duchenne Muscular Dystrophy
A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy
This is a multicenter, prospective, single cohort study designed to describe the natural history of DMD in Chinese male patients.
- Trial name
- NCT03760029
- Testing
- Visit frequency
- Where
- 8 sites in China
- Volunteers
- 312 people
- Sponsor
- Pfizer
Duchenne Muscular Dystrophy (DMD)
Twice Weekly Steroids and Exercise as Therapy for DMD
The study team will determine the potential of low dose twice weekly prednisone and whether exercise training can synergize to delay disease progression and improve muscle strength/physical function in boys with Duchenne muscular…
- Trial name
- NCT04322357
- Testing
- Prednisone, Exercise Training on standard steroid regimen, Exercise training on twice-weekly steroids
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- University of Florida
Duchenne Muscular Dystrophy
Phase I/II 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 DMD patients with a mutation around location 44 in the DNA for the dystrophin protein.
- Trial name
- NCT01037309
- Testing
- PRO044 SC, PRO044 IV
- Where
- 4 sites in Belgium, Italy and 2 more
- Volunteers
- 18 people
- Sponsor
- BioMarin Pharmaceutical
Pediatrics, Muscle Spasticity
BOTOX® Treatment in Pediatric Lower Limb Spasticity
This study will evaluate the safety and efficacy of BOTOX® (botulinum toxin Type A) in pediatric patients with lower limb spasticity.
- Conditions
- Pediatrics, Muscle Spasticity, Cerebral Palsy
- Trial name
- NCT01603628
- Testing
- botulinum toxin Type A
- Where
- 53 sites in United States, Hungary and 7 more
- Volunteers
- 384 people
- Sponsor
- Allergan