Clinical trials: Myopathies
Fibrodysplasia Ossificans Progressiva
A Study to Assess Safety, Tolerability and Efficacy of Garetosmab Versus Placebo Administered Intravenously (IV) in Adult Participants With Fibrodysplasia Ossificans Progressiva (FOP)
This study is researching an experimental drug called garetosmab. The study is focused on adult patients with fibrodysplasia ossificans progressiva (FOP).
- Trial name
- OPTIMA (NCT05394116)
- Testing
- Garetosmab
- Where
- 22 sites in United States, Australia and 16 more
- Volunteers
- 63 people
- Sponsor
- Regeneron Pharmaceuticals
Spasticity
Study Assessing Adult Subjects Affected With Upper and/or Lower Limb Spasticity Treated With Botulinum Neurotoxin Type A
The aim of the study is to describe the long term use of Botulinum Neurotoxin Type A (BoNT-A) in adult subjects affected with upper and/or lower limb spasticity who received treatment with BoNT-A for a minimum of three injections…
- Trial name
- RESULT (NCT04340336)
- Where
- 3 sites in Italy
- Volunteers
- 150 people
- Sponsor
- Ipsen
Myotonic Dystrophy Type 1
A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1
This study will test the safety, tolerability, and pharmacokinetics of multiple escalating doses of ISIS-DMPKRx administered subcutaneously to adult patients with DM1.
- Trial name
- NCT02312011
- Testing
- IONIS-DMPKRx
- Where
- 8 sites in United States
- Volunteers
- 48 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Muscle Weakness
Combination Treatment With BTL-899 and HPM-6000UF Devices II
This study will evaluate the clinical efficacy of the BTL-899 and HPM-6000UF devices in combination for non-invasive strengthening of the core muscles and overall improvement in quality of life in female patients after childbirth.
- Trial name
- NCT05713864
- Testing
- BTL-899, HPM-6000UF
- Where
- 3 sites in United States
- Volunteers
- 33 people, healthy volunteers welcome
- Sponsor
- BTL Industries Ltd.
Healthy, CVA
Development of Automated Analysis to Electroencephelogram (EEG) Data in Patients Treated at the Sagol Hyperbaric Medicine and Research Center at the Years 2017-2019.
DELPhI software developed for the analysis of EEG recordings in response to magnetic stimulation in relation to clinical data.
- Conditions
- Healthy, CVA, Hemorrhage, TBI (Traumatic Brain Injury)
- Trial name
- NCT04095377
- Testing
- No intervention, this is a retrospective study.
- Where
- Israel
- Volunteers
- 875 people, healthy volunteers welcome
- Sponsor
- QuantalX Neuroscience
Hereditary Inclusion Body Myopathy, Distal Myopathy With Rimmed Vacuoles
Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
The primary objective of this study is to evaluate the effect of 6 g/day aceneuramic acid extended-release (Ace-ER) treatment of participants with GNEM on upper extremity muscle strength (upper extremity composite [UEC] score) as…
- Conditions
- Hereditary Inclusion Body Myopathy, Distal Myopathy With Rimmed Vacuoles, Distal Myopathy, Nonaka Type, GNE Myopathy
- Trial name
- GNEM (NCT02377921)
- Testing
- aceneuramic acid extended-release (Ace-ER)
- Where
- 13 sites in United States, Bulgaria and 5 more
- Volunteers
- 89 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Duchenne Muscular Dystrophy
A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995)
The purpose of this extension study is to evaluate the ongoing safety and tolerability of additional treatment with eteplirsen administered once weekly by intravenous (IV) infusion in male participants with DMD who have…
- Trial name
- NCT03985878
- Testing
- Eteplirsen
- Where
- 4 sites in Belgium, France and 2 more
- Volunteers
- 15 people
- Sponsor
- Sarepta Therapeutics, Inc.
Myotonic Dystrophy Type 1 (DM1)
A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I
The purpose of the study is to evaluate the long-term safety and tolerability, efficacy and pharmacokinetics of VX-670 in participants with Myotonic Dystrophy Type I (DM1).
- Trial name
- NCT06926621
- Testing
- VX-670
- Where
- 16 sites in United States, Australia and 6 more
- Volunteers
- 44 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Autosomal Dominant Hypocalcemia
ADH1 and ADH2 Disease Monitoring Study (DMS)
A global, multi-center, Disease Monitoring Study (DMS) in participants with Autosomal Dominant Hypocalcemia Type 1 (ADH1) or Autosomal Dominant Hypocalcemia Type 2 (ADH2) designed to characterize ADH1 and ADH2 disease…
- Trial name
- CLARIFY (NCT05227287)
- Where
- 27 sites in United States, Australia and 11 more
- Volunteers
- 95 people
- Sponsor
- Calcilytix Therapeutics, Inc., a BridgeBio company
Muscle Spasticity
The Efficacy and Safety of MEDITOXIN® in Children With Cerebral Palsy
The purpose of this study is to evaluate the efficacy and safety of Meditoxin® in the treatment of children with cerebral palsy.
- Trial name
- NCT01256021
- Testing
- Botulinum Toxin Type A
- Where
- 4 sites in South Korea
- Volunteers
- 212 people
- Sponsor
- Medy-Tox
Muscle Spasticity, Stroke
OnabotulinumtoxinA Treatment in Adult Patients With Upper Limb Spasticity
This is a safety and efficacy study of onabotulinumtoxinA in poststroke patients with upper limb spasticity.
- Trial name
- NCT02145676
- Testing
- onabotulinumtoxinA
- Where
- 2 sites in United States, Canada
- Volunteers
- 53 people
- Sponsor
- Allergan
Primary Mitochondrial Disease
Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM)
This is a Phase 2 Open-Label extension study to evaluate the long term safety and tolerability of daily elamipretide injections in patients with genetically confirmed Primary Mitochondrial Disease who previously participated in…
- Trial name
- NCT02976038
- Testing
- elamipretide
- Where
- 4 sites in United States
- Volunteers
- 28 people
- Sponsor
- Stealth BioTherapeutics Inc.
Muscular Dystrophies
A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects
The purpose of this study is investigate the pharmacokinetics, safety and tolerability of single subcutaneous administration of GSK2402968 in non-ambulant boys with Duchenne muscular dystrophy
- Trial name
- DEMAND I (NCT01128855)
- Testing
- 3 mg/kg GSK2402968, 6 mg/kg GSK2402968, 9 mg/kg GSK2402968
- Where
- 2 sites in United States, France
- Volunteers
- 20 people
- Sponsor
- GlaxoSmithKline
Tendonitis, Bursitis
Ketoprofen Topical Patch 20% in the Treatment of Pain Associated With Shoulder, Elbow or Knee Tendonitis or Bursitis
The purpose of this study is to evaluate the effect of a ketoprofen topical patch on the pain associated with tendonitis or bursitis of the shoulder, elbow or knee.
- Trial name
- NCT00352625
- Testing
- Ketoprofen Topical Patch 20%
- Where
- United States
- Volunteers
- 330 people
- Sponsor
- Alkemira SA
Pain
Study to Evaluate the Efficacy and Safety of OraVescent Fentanyl Citrate Treatment for the Management of Breakthrough Pain in Opioid-Tolerant Patients With Noncancer-Related Chronic Pain
The purpose of the study is to evaluate the efficacy and safety of OraVescent fentanyl treatment compared to placebo treatment monthly over a 12-week treatment period in alleviating breakthrough pain (BTP) in opioid-tolerant…
- Trial name
- NCT00343733
- Testing
- OraVescent Fentanyl
- Where
- 21 sites in United States
- Volunteers
- 120 people
- Sponsor
- Cephalon
Duchenne Muscular Dystrophy
Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
This long-term extension study is an open-label, multiple-dose study to evaluate the long-term safety, tolerability, efficacy and PD of vamorolone administered once daily by liquid oral suspension over a Treatment Period of 24…
- Trial name
- NCT03038399
- Testing
- Vamorolone 0.25 mg/day/day, Vamorolone 0.75 mg/day/day, Vamorolone 2.0 mg/day/day
- Where
- 12 sites in United States, Australia and 4 more
- Volunteers
- 46 people
- Sponsor
- ReveraGen BioPharma, Inc.
Polymyositis, Dermatomyositis
A Study to Investigate the Efficacy and Safety of Anifrolumab Administered as Subcutaneous Injection and Added to Standard of Care Compared With Placebo Added to Standard of Care in Adult Participants With Idiopathic Inflammatory Myopathies (Polymyositis and Dermatomyositis)
The purpose of this multicenter, randomized, placebo-controlled and double-blind study is to evaluate the efficacy and safety of subcutaneous anifrolumab compared with placebo on the overall disease activity in participants with…
- Trial name
- JASMINE (NCT06455449)
- Testing
- Anifrolumab (blinded), Anifrolumab (unblinded, open label)
- Where
- 237 sites in United States, Argentina and 27 more
- Volunteers
- 240 people
- Sponsor
- AstraZeneca
Fibromyalgia
Discontinuation Study of the Durability of Effect of Milnacipran for the Treatment of Fibromyalgia
The purpose of this study is to evaluate the durability of effect of milnacipran for the treatment of fibromyalgia in patients receiving long-term milnacipran treatment and to characterize the effects of milnacipran on multiple…
- Trial name
- NCT01014585
- Testing
- Milnacipran
- Where
- 58 sites in United States
- Volunteers
- 340 people
- Sponsor
- Forest Laboratories
Spasticity, Multiple Sclerosis
Evaluate the Maintenance of Effect After Long-term Treatment With Sativex® in Subjects With Symptoms of Spasticity Due to Multiple Sclerosis
The purpose of this study is to evaluate the maintenance of effect after long-term treatment with Sativex® in subjects with symptoms of spasticity due to Multiple Sclerosis (MS) who have been receiving long-term benefit from…
- Trial name
- NCT00702468
- Testing
- Sativex
- Where
- United Kingdom
- Volunteers
- 36 people
- Sponsor
- Jazz Pharmaceuticals
Stroke, TBI
Characterization of the DELPhI System in Assessing Brain's Functionality in Different Neurological Disorders
We use Transcranial magnetic stimulation (TMS), combined with simultaneous registration of electroencephalograph (EEG),for examining human cortical functionality.
- Conditions
- Stroke, TBI, Fibromyalgia, PDD
- Trial name
- NCT03832413
- Testing
- DELPhI (TMS-EEG analysis)
- Where
- Israel
- Volunteers
- 193 people, healthy volunteers welcome
- Sponsor
- QuantalX Neuroscience