Clinical trials: Myopathies
Alopecia Areata, Depressive Episode
The Epidemiology, Management, and the Associated Burden of Related Conditions in Alopecia Areata
This study series consists of four related studies and aims to explore and describe many important elements of alopecia areata over three key areas: (1) the current epidemiology of alopecia areata, (2) the prevalence and…
- Conditions
- Alopecia Areata, Depressive Episode, Recurrent Depressive Disorder, Anxiety Disorders
- Trial name
- NCT04239521
- Testing
- Exposure of interest (studies 2 & 3).
- Where
- United Kingdom
- Volunteers
- 51,955 people
- Sponsor
- Momentum Data
Mitochondrial Diseases
A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease
A long-term extension study to assess the safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) in children with inherited mitochondrial diseases who previously enrolled into study…
- Trial name
- NCT02473445
- Testing
- Cysteamine Bitartrate
- Where
- 5 sites in United States
- Volunteers
- 22 people
- Sponsor
- Amgen
Centronuclear Myopathy
Early Phase Human Drug Trial to Investigate DYN101 in Participants 2 to 17 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 and tolerability as well as pharmacokinetics (PK), pharmacodynamics (PD) and…
- Trial name
- DyNaMic (NCT04743557)
- Testing
- DYN101
- Where
- 2 sites in France, Germany
- Sponsor
- Dynacure
Tendinopathy, Insertional Achilles Tendinopathy
Evaluation of Collagen-based Medical Device Treatment Combined With Physiotherapy in Subjects With Achilles Tendinopathy.
Achilles tendinopathy is a condition characterized by inflammation of the Achilles tendon. Achilles tendinopathies are classified into insertional tendinitis and noninsertional tendinitis.
- Conditions
- Tendinopathy, Insertional Achilles Tendinopathy, Achilles Tendinopathy, Peritendinitis
- Trial name
- AKITENMED (NCT05464498)
- Testing
- MD-Tissue Collagen Medical Device, eccentric strengthening protocol
- Where
- Italy
- Volunteers
- 72 people
- Sponsor
- Guna S.p.a
Septic Shock, Acute Respiratory Distress Syndrome
CytOSorb TreatMent Of Critically Ill PatientS Registry
Registry intended to provide a data repository and reporting infrastructure for the surveillance of CytoSorb device use in real-world critical care settings, and to serve as an objective, comprehensive, and scientifically-based…
- Conditions
- Septic Shock, Acute Respiratory Distress Syndrome, Trauma, Rhabdomyolysis
- Trial name
- COSMOS (NCT05146336)
- Testing
- CytoSorb
- Where
- 28 sites in Austria, Germany and 4 more
- Volunteers
- 3,000 people
- Sponsor
- CytoSorbents, Inc
Fibromyalgia
Occipital Nerve (C2) Stimulation in the Treatment of Fibromyalgia
The study is designed as a prospective, randomized, double-blind, single center study with a 6 month duration post permanent system implantation. Forty patients will be implanted.
- Trial name
- NCT01298609
- Testing
- Occipital Stimulation
- Where
- Belgium
- Volunteers
- 40 people
- Sponsor
- Abbott Medical Devices
Charcot-Marie-Tooth Disease
A Study to Assess the Safety and Effectiveness of FLX-787 in Subjects With Charcot-Marie-Tooth Disease Experiencing Muscle Cramps.
The COMMIT Study will assess the safety and effectiveness of FLX-787 in men and women with Charcot-Marie-Tooth disease (CMT) experiencing muscle cramps.
- Trial name
- COMMIT (NCT03254199)
- Testing
- FLX-787-ODT (orally disintegrating tablet)
- Where
- 20 sites in United States
- Volunteers
- 27 people
- Sponsor
- Flex Pharma, Inc.
ICU Acquired Weakness
Safety and Efficacy of the MyokinE100 (CL-EMS Alpha) Device in Inducing Visible Muscle Contraction in Healthy Volunteers
Every patient admitted to the Intensive Care Unit (ICU) is at risk of developing "Intensive Care Unit Acquired Weakness" (ICUAW).
- Trial name
- NCT06023511
- Testing
- Electrical muscle stimulation
- Where
- United States
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Health Discovery Labs
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
Osteoarthritis, Fibromyalgia
Pilot Study of the Efficacy & Safety of Natural Eggshell Membrane (NEM) for Joint & Connective Tissue Disorders
The purpose of this study was to evaluate the use of the dietary supplement Natural Eggshell Membrane (NEM) for the treatment of joint and connective tissue discomfort, pain, and flexibility.
- Trial name
- NCT00750230
- Testing
- NEM
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- ESM Technologies, LLC
Muscular Dystrophy, Duchenne
Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy
The study will evaluate the safety and dystrophin expression following gene therapy in boys with Duchenne Muscular Dystrophy (DMD). It is a single-arm, non-randomized, open-label study
- Trial name
- NCT05429372
- Testing
- PF-06939926
- Where
- 13 sites in United States, Australia
- Volunteers
- 10 people
- Sponsor
- Pfizer
Systemic Lupus Erythematosus (SLE), Sjogren's Syndrome (SS)
A Clinical Study of TI-0032-III Injection in Patients With Relapsed and Refractory Autoimmune Diseases
This is an open-label, dose escalation study in patients with relapsed and refractory autoimmune diseases. Study drug, TI-0032-III injection, is composed of lipid nanoparticles (LNPs) targeting T cells that encapsulate circular…
- Conditions
- Systemic Lupus Erythematosus (SLE), Sjogren's Syndrome (SS), Systemic Sclerosis (SSc), Inflammatory Myopathy
- Trial name
- in vivo CAR-T (NCT07413341)
- Testing
- TI-0032-III injection
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Therorna
Stroke, Muscle Spasticity
Safety Study of Botulinum Toxin Type A for the Treatment of Focal Upper Limb Poststroke Spasticity
The purpose of this study is to evaluate the safety of repeated doses of Botulinum Toxin Type A for the treatment of focal upper limb poststroke spasticity
- Trial name
- NCT00651729
- Testing
- Botulinum Toxin Type A
- Where
- 2 sites in United States, Canada
- Volunteers
- 279 people
- Sponsor
- Allergan
Fibromyalgia
Long-term OL Study of [S,S]-RBX in Patients With Fibromyalgia
To evaluate the lon-term safety and tolerability of [S,S]-reboxetine in patients with fibromyalgia
- Trial name
- NCT00607256
- Testing
- [S,S]-reboxetine
- Where
- 79 sites in United States, Belgium and 9 more
- Volunteers
- 500 people
- Sponsor
- Pfizer
Myotubular Myopathy
Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM)
This is a prospective, non-interventional, longitudinal study of the natural history and function of approximately 60 patients with MTM from the United States, Canada and Europe.
- Trial name
- MTM (NCT02057705)
- Where
- 13 sites in United States, Belgium and 5 more
- Volunteers
- 48 people
- Sponsor
- Valerion Therapeutics, LLC
Nociceptive Pain, Musculoskeletal Pain
Clinical Trial of the invisaRED® IR REHAB Device When Used as an Adjunctive Therapy for Nociceptive Musculoskeletal Pain
This is a trial to measure the effectiveness and safety of the invisa-RED® IR REHAB device. The researchers will compare the muscle and joint pain relief of participants who are treated with the real machine, to those treated…
- Trial name
- NCT06393088
- Testing
- IR REHAB
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- IR Technology, LLC
Duchenne Muscular Dystrophy (DMD)
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44
This is a study of the investigational medicine ENTR-601-44 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition.
- Trial name
- ELEVATE-44 (NCT07037862)
- Testing
- ENTR-601-44
- Where
- 14 sites in Belgium, Italy and 2 more
- Volunteers
- 24 people
- Sponsor
- Entrada Therapeutics, Inc.
Traumatic Brain Injury
Pilot, Proof-of-Concept Study of Sublingual Tizanidine in Children With Chronic Traumatic Brain Injury (TBI)
Nightly administration of a unique, sublingual (under the tongue) formulation of tizanidine, a known anti-spasticity medication, has been shown in a previous study to improve sleep and next-day functioning in CP (cerebral palsy)…
- Trial name
- NCT00287157
- Testing
- Sublingual Tizanidine HCl
- Where
- Israel
- Volunteers
- 5 people
- Sponsor
- Teva GTC
Spasticity, Cerebrovascular Accident
Efficacy and Safety Study of MYOBLOC® in the Treatment of Adult Upper Limb Spasticity
Phase 2/3, randomized, double-blind, placebo-controlled, single-treatment, multicenter trial assessing the efficacy and safety of MYOBLOC for the treatment of upper limb spasticity in adults followed by an open-label extension…
- Conditions
- Spasticity, Cerebrovascular Accident, Multiple Sclerosis, Traumatic Brain Injury
- Trial name
- NCT04815967
- Testing
- Phase 2; Low Dose MYOBLOC (10,000 Units), Phase 2; High Dose MYOBLOC (20,000 Units), Phase 3; MYOBLOC
- Where
- 9 sites in United States, Hungary and 1 more
- Volunteers
- 32 people
- Sponsor
- Solstice Neurosciences, LLC, a subsidiary of MDD US Operations, LLC
Multiple Sclerosis, Spasticity, Muscle
Performance and Long-term Safety of FlowOx2.0™ in Patients With Multiple Sclerosis - Impact on Spasticity and Pain
The study is a 4-week double-blind, randomized, controlled, parallel design investigation to investigate the impact of intermittent negative pressure on spasticity and pain in people with multiple sclerosis (pwMS).
- Conditions
- Multiple Sclerosis, Spasticity, Muscle, Pain, Chronic
- Trial name
- FO-NP002 (NCT05562453)
- Testing
- FlowOx 2.0
- Where
- Sweden
- Volunteers
- 56 people
- Sponsor
- Otivio AS