Clinical trials: MYOPATHY
Systemic Sclerosis, Idiopathic Inflammatory Myopathies
A Study of AZD0120 in Autoimmune Diseases
This trial is a Phase 1b/2, open-label, multi-center, clinical study of AZD0120, a BCMA/CD19 dual targeting CAR+ T-cell therapy, to evaluate the safety and tolerability in adult participants with systemic sclerosis (SSc)…
- Conditions
- Systemic Sclerosis, Idiopathic Inflammatory Myopathies, Rheumatoid Arthritis
- Trial name
- AURORA (NCT07295847)
- Testing
- AZD0120, SOC
- Where
- 25 sites in United States, Australia and 4 more
- Volunteers
- 27 people
- Sponsor
- AstraZeneca
Compartment Syndrome
Double Blinded, Randomized Clinical Trial of the Twin Star ECSPRESS Monitoring System for the Reduction of the Incidence of Fasciotomy
The proposed study is a double-blinded, randomized clinical trial of tissue pressure monitoring only (Control group) vs. tissue pressure monitoring plus tissue ultrafiltration (Treatment group) in patients with tibia fractures…
- Trial name
- ECSPRESS (NCT01879696)
- Testing
- ECSPRESS catheter with active vacuum
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- Twin Star Medical, Inc.
Fibromyalgia
Study Evaluating the Efficacy of DVS-233 in Fibromyalgia
The purpose of this study is to determine if DVS-233 is safe and effective in the treatment of pain and other symptoms of fibromyalgia syndrome.
- Trial name
- NCT00301431
- Testing
- DVS-233
- Where
- 59 sites in United States
- Volunteers
- 1,050 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Severe Spasticity
SISTERS: Spasticity In Stroke Study - Randomized Study
To demonstrate that Intrathecal Baclofen (ITB) Therapy, compared to Best Medical Treatment (BMT), has superior efficacy in the treatment of severe spasticity in adult post-stroke patients with generalized spastic hypertonia who…
- Trial name
- SISTERS (NCT01032239)
- Testing
- intrathecal baclofen
- Where
- 25 sites in United States, Austria and 7 more
- Volunteers
- 61 people
- Sponsor
- MedtronicNeuro
Plantar Fibromatosis
Long-Term Safety, Efficacy, and Durability of EN3835 in the Treatment and Retreatment of Plantar Fibromatosis
Assess the long-term safety and efficacy of EN3835 in participants who have participated in a parent placebo-controlled study of EN3835 (EN3835-222, NCT05152173) and assess the efficacy and safety of EN3835 in the treatment and…
- Trial name
- NCT05254457
- Testing
- EN3835
- Where
- 27 sites in United States
- Volunteers
- 145 people
- Sponsor
- Endo USA Inc., a Keenova Therapeutics Company
Duchenne Muscular Dystrophy
Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy
The purpose of the study is to see whether PRO045 is safe and effective to use as medication for Duchenne Muscular Dystrophy (DMD) patients with a mutation around location 45 in the DNA for the dystrophin protein.
- Trial name
- NCT01826474
- Testing
- PRO045, 0.15 mg/kg/week, PRO045, 1.0 mg/kg/week, PRO045, 3.0 mg/kg/week
- Where
- 6 sites in Belgium, France and 3 more
- Volunteers
- 15 people
- Sponsor
- BioMarin Pharmaceutical
Duchenne Muscular Dystrophy
Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)
The VILT-502 study is Non-interventional Study(United States)/Low-intervention Clinical Trial (Canada) of Viltolarsen administered intravenously once weekly for 10 years to boys with DMD who complete the NS-065/NCNP-01-202 study.
- Trial name
- NCT04687020
- Testing
- Viltolarsen
- Where
- 5 sites in United States, Canada
- Volunteers
- 9 people
- Sponsor
- NS Pharma, Inc.
Temporomandibular Joint Disorders
Exploratory Study To Assess the Efficacy and Safety of a Triple Combination for Acute Pain Treatment in Patients With Temporomandibular Joint Disorders (TMJD).
The objetive of this study is to explore the efficacy and safety of a triple combination of ibuprofen, magnesium and ascorbic acid for acute pain treatment in TMJD patients
- Trial name
- NCT02602483
- Testing
- Ibuprofen, Magnesium, Ascorbic Acid
- Where
- 4 sites in Spain
- Volunteers
- 96 people
- Sponsor
- Spherium Biomed
Spinal Muscular Atrophy
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)
To evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene weighing ≥…
- Trial name
- SMART (NCT04851873)
- Testing
- OAV101
- Where
- 13 sites in United States, Australia and 7 more
- Volunteers
- 24 people
- Sponsor
- Novartis Pharmaceuticals
Fibromyalgia
Prospective Evaluation of RINCE to Reduce Fibromyalgia Effects - St. Joseph Mercy Oakland
The purpose of this study is to test the safety and efficacy of a noninvasive cortical electrostimulation therapy known as "Reduced Impedance Noninvasive Cortical Electrostimulation" (RINCE) in the management of fibromyalgia.
- Trial name
- PERRFECT-SJMO (NCT01825954)
- Testing
- RINCE
- Where
- United States
- Volunteers
- 46 people
- Sponsor
- Cerephex Corporation
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
Duchenne Muscular Dystrophy
Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)
The aim of this study is to provide continuing access to BMN 044 treatment for subjects previously treated with BMN 044. The information gained from this study is expected to further characterize the efficacy and safety of BMN…
- Trial name
- NCT02958202
- Testing
- BMN 044 IV 6 mg/kg, BMN 044 IV 9 mg/kg, BMN 044 SC 6 mg/kg
- Where
- 5 sites in Belgium, Italy and 2 more
- Volunteers
- 7 people
- Sponsor
- BioMarin Pharmaceutical
Primary Mitochondrial Myopathy
A Study to Evaluate ASP0367 in Participants With Primary Mitochondrial Myopathy
The purpose of this study was to evaluate the dose response of Bocidelpar on functional improvement relative to placebo, safety, and tolerability in participants with Primary Mitochondrial Myopathy.
- Trial name
- MOUNTAINSIDE (NCT04641962)
- Testing
- Bocidelpar
- Where
- 11 sites in United States
- Volunteers
- 34 people
- Sponsor
- Astellas Pharma Inc
Myotonic Dystrophy 1
Effect of MYODM on Quality of Life, Fatigue and Hypersomnia in Patients With Myotonic Dystrophy Type 1
The purpose of this study is to determine whether MYODM (formulated composition containing Theobroma cacao supplemented with caffeine (caffeine/theobromine ratio1/1.85, w/w) is effective in the treatment of excessive daytime…
- Trial name
- NCT04634682
- Testing
- MYODM
- Where
- Spain
- Volunteers
- 30 people
- Sponsor
- Myogem Health Company, S.L.
Physical Activity, Muscle Recovery
Effect of NMN on Muscle Recovery and Physical Capacity in Healthy Volunteers With Moderate Physical Activity
The purpose of this study is to evaluate the effect of NMN supplementation (250 and 500 mg/day over 38 days) compared to placebo in healthy volunteers with moderate physical activity on muscle recovery, physical capacity…
- Trial name
- NCT04664361
- Testing
- Nicotinamide mononucleotide
- Where
- France
- Volunteers
- 131 people, healthy volunteers welcome
- Sponsor
- Seneque SA
Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy)
A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping
A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping
- Conditions
- Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Muscular Disorders, Atrophic, Muscular Disease
- Trial name
- SAFARI44 (NCT07587242)
- Testing
- AOC 1044
- Where
- 18 sites in Australia, Belgium and 6 more
- Volunteers
- 70 people
- Sponsor
- Avidity Biosciences, Inc.
Mitochondrial Diseases, Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
The KHENERGYC Study
This a randomized placebo controlled, double-blind phase II study to explore the pharmacokinetics, safety and efficacy of sonlicromanol in children (from birth to 17 years) with genetically confirmed mitochondrial disease of…
- Conditions
- Mitochondrial Diseases, Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation, MELAS, Subacute Necrotizing Encephalomyelopathy
- Trial name
- NCT04846036
- Testing
- Sonlicromanol
- Where
- Netherlands
- Volunteers
- 24 people
- Sponsor
- Khondrion BV
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
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.
Myotonic Dystrophy Type 1 (DM1)
A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.
- Trial name
- Galileo (NCT06185764)
- Testing
- VX-670
- Where
- 26 sites in United States, Australia and 8 more
- Volunteers
- 47 people
- Sponsor
- Vertex Pharmaceuticals Incorporated