Clinical trials: Neuromuscular Disorder
Pain, Weakness
Clinical Trial of CoQ10 for Mild-to-Moderate Statin-Associated Muscle Symptoms
This study will evaluate whether a dietary supplement, coenzyme Q10 (CoQ10), will improve muscle symptoms, such as muscle aches, pains, cramps, and/or weakness, which are experienced by some individuals who use statin medications.
- Conditions
- Pain, Weakness, Cramps, Myalgia or Myositis Nos
- Trial name
- NCT01032993
- Testing
- Coenzyme Q10
- Where
- United States
- Volunteers
- 68 people, healthy volunteers welcome
- Sponsor
- Catherine Buettner
Post-polio Syndrome
Study of the Efficacy and Safety of Immune Globulin Intravenous (Human) Flebogamma® 5% Dual Inactivation and Filtration (DIF) in Participants With Post-polio Syndrome
This was a multicenter, prospective, randomized, placebo-controlled, double-blind, parallel group clinical trial with adaptive dose selection in participants with post-polio syndrome (PPS).
- Trial name
- FORCE (NCT02176863)
- Testing
- Flebogamma® 5% DIF
- Where
- 21 sites in United States, Canada and 7 more
- Volunteers
- 191 people
- Sponsor
- Instituto Grifols, S.A.
FAP, Familial Amyloid Polyneuropathy
Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)
This study evaluates the safety and tolerability of extended dosing with IONIS-TTR Rx in patients with Familial Amyloid Polyneuropathy.
- Conditions
- FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin
- Trial name
- NCT02175004
- Testing
- Inotersen
- Where
- 22 sites in United States, Argentina and 7 more
- Volunteers
- 135 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Muscular Dystrophy, Duchenne
A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy
This is a multicenter, double blind, placebo controlled, multiple dose study to examine the safety and efficacy of MNK-1411 in male subjects 4 to 8 years of age (inclusive) with Duchenne Muscular Dystrophy (DMD).
- Trial name
- BRAVE (NCT03400852)
- Testing
- MNK-1411
- Where
- 16 sites in United States, Bulgaria and 6 more
- Volunteers
- 44 people
- Sponsor
- Mallinckrodt ARD LLC
Amyotrophic Lateral Sclerosis
A Study to Assess the Safety, Tolerability, and Effect on Disease Progression of BIIB105 in Participants With Amyotrophic Lateral Sclerosis (ALS) and Participants With the ALS Ataxin-2 (ATXN2) Genetic Mutation
The ALSpire Study is a clinical trial evaluating the investigational drug BIIB105 in adults living with amyotrophic lateral sclerosis (ALS). The ALSpire Study consists of two parts: Part 1: 6-month placebo-controlled study.
- Trial name
- ALSpire (NCT04494256)
- Testing
- BIIB105
- Where
- 17 sites in United States, Canada and 2 more
- Volunteers
- 99 people
- Sponsor
- Biogen
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
Finding the Optimum Regimen for Duchenne Muscular Dystrophy
The Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR DMD) study will compare three ways of giving corticosteroids to boys with Duchenne muscular dystrophy (DMD) to determine which of the three ways increases…
- Trial name
- FOR-DMD (NCT01603407)
- Testing
- Prednisone, Deflazacort
- Where
- 32 sites in United States, Canada and 3 more
- Volunteers
- 196 people
- Sponsor
- University of Rochester
Gastrointestinal Diseases, Bone Diseases
Virtual Path Planning for Image-guided Needle Interventions
Philips Healthcare has added a virtual path planner to the current commercially available XperGuide software platform and that has the potential to significantly reduce dose during image-guided needle interventions.
- Conditions
- Gastrointestinal Diseases, Bone Diseases, Muscular Diseases, Spinal Diseases
- Trial name
- NCT02021071
- Testing
- XperGuide, XperGuide with virtual path planning
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Philips Clinical & Medical Affairs Global
Duchenne Muscular Dystrophy
A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.
This is an 48-week open-label study to determine the efficacy and safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 for the treatment of boys with duchenne muscular dystrophy who have a single exon duplication of either exon 45, 51 or…
- Trial name
- NCT04179409
- Testing
- Amondys 45, Exondys 51, Vyondys 53
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Kevin Flanigan
Duchenne Muscular Dystrophy (DMD)
A Study to Assess Safety/Tolerability, pk, Effects on Histology, Clinical Parameters of Givinostat in Children With DMD
The primary objective of Parts 1 and 2 of the study were to establish the histologic effects of givinostat administered chronically at the selected daily dose.
- Trial name
- NCT01761292
- Testing
- Givinostat
- Where
- 4 sites in Italy
- Volunteers
- 20 people
- Sponsor
- Italfarmaco
Frailty Syndrome
Ghrelin Plus Strength Training in Frail Elderly Study
Frailty is a geriatric syndrome leading to physical deterioration including muscle wasting (sarcopenia) and unintentional weight loss. There are currently no approved therapies for frailty.
- Trial name
- NCT01898611
- Testing
- Ghrelin
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- University of Pennsylvania
Diabetes Complications, Diabetes Mellitus
The Efficacy Of Intravenous Immunoglobulin Therapy In Treatment Induced Neuropathy Of Diabetes
The goal of this pilot study is to obtain preliminary data on the magnitude of the treatment effect of IVIG on the neuropathic pain and neuropathy severity associated with treatment induced neuropathy (TIND).
- Conditions
- Diabetes Complications, Diabetes Mellitus, Diabetic Neuropathies
- Trial name
- NCT02915263
- Testing
- IGIV-C, 0.9% Sodium Chloride
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Beth Israel Deaconess Medical Center
Primary Familial Dilated Cardiomyopathy
Exploratory Study of Danicamtiv in Patients With Primary Dilated Cardiomyopathy (DCM) Due to Genetic Variants or Other Causalities
The purpose of this Phase 2a study is to establish safety and preliminary efficacy of treatment with danicamtiv in patients with primary dilated cardiomyopathy (DCM) due to MYH7 or TTN variants or other causalities.
- Trial name
- NCT04572893
- Testing
- danicamtiv
- Where
- 19 sites in United States, Germany and 2 more
- Volunteers
- 41 people
- Sponsor
- Bristol-Myers Squibb
Fibromyalgia
Study of Gralise to Treat Fibromyalgia Patients
To determine Gralise in treating fibromyalgia pain: efficacy safety
- Trial name
- Gralise (NCT02052414)
- Testing
- Gabapentin ER
- Where
- United States
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- The Center for Clinical Research, Winston-Salem, NC
Fibromyalgia
Combined Behavioral and Analgesic Trial for Fibromyalgia
This is a randomized control study that involves a combination of a drug treatment, behavioral health, and placebo controls. Some participants will receive a medication approved by the Food and Drug Administration for treatment…
- Trial name
- COMBAT-FM (NCT01598753)
- Testing
- Tramadol, Cognitive Behavior Therapy for FM, Health Education
- Where
- 2 sites in United States
- Volunteers
- 134 people
- Sponsor
- University of Washington
Amyloid Cardiomyopathy, Transthyretin-Related
Screening for Cardiac Amyloidosis With Nuclear Imaging for Minority Populations
In this study, the investigators recruited a cohort of elderly Black and Hispanic patients with heart failure to define the number of patients who have cardiac amyloidosis by utilizing highly sensitive heart imaging and blood…
- Trial name
- SCAN-MP (NCT03812172)
- Testing
- 99mTc-PYP or 99m Tc-HDP
- Where
- 4 sites in United States
- Volunteers
- 646 people
- Sponsor
- Mathew S. Maurer, MD
Neuropathic Pain
Effect of Eslicarbazepine Acetate on the Pharmacokinetics of Metformin in Healthy Volunteers
The primary objective was to investigate whether multiple-dose administration of eslicarbazepine acetate affects the pharmacokinetics of metformin.
- Trial name
- NCT00971295
- Testing
- Metformin, Eslicarbazepine acetate
- Where
- Portugal
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Bial - Portela C S.A.
Muscular Dystrophies
A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
The purpose of this study is to determine whether GSK2402968 is effective in the treatment of ambulant boys with Duchenne muscular dystrophy resulting from a mutation thought to be corrected by exon 51 skipping.
- Trial name
- DMD114044 (NCT01254019)
- Testing
- GSK2402968 6mg/kg/week
- Where
- 47 sites in Argentina, Belgium and 18 more
- Volunteers
- 186 people
- Sponsor
- GlaxoSmithKline
Tuberous Sclerosis Complex (TSC), Lymphangioleiomyomatosis (LAM)
Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)
This study will evaluate the safety and efficacy of RAD001 in treating patients with Angiomyolipoma associated with Tuberous Sclerosis Complex or Sporadic Lymphangioleiomyomatosis.
- Trial name
- EXIST-2 (NCT00790400)
- Testing
- Everolimus (RAD001)
- Where
- 25 sites in United States, Canada and 9 more
- Volunteers
- 118 people
- Sponsor
- Novartis Pharmaceuticals
Fibromyalgia
Use of Repetitive Transcranial Magnetic Stimulation to Augment Hypnotic Analgesia
The investigators plan to use functional neuroimaging (fMRI) to understand the brain systems affected when hypnosis and hypnotic analgesia are augmented with repetitive transcranial magnetic stimulation (rTMS), a form of…
- Trial name
- NCT02969707
- Testing
- MagPro TMS system (MagVenture, Denmark), Hypnosis
- Where
- United States
- Volunteers
- 101 people
- Sponsor
- Stanford University
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