Clinical trials: Myopathies
Fibrodysplasia Ossificans Progressiva (FOP)
A Rollover Study to Further Evaluate the Safety and Efficacy of Palovarotene Capsules in Male and Female Participants Aged ≥14 Years With Fibrodysplasia Ossificans Progressiva (FOP) Who Have Completed the Relevant Parent Studies.
The main objective of this study is to further evaluate the safety and efficacy of palovarotene in adult and paediatric participants with FOP.
- Trial name
- PIVOINE (NCT05027802)
- Testing
- Palovarotene
- Where
- 13 sites in United States, Argentina and 8 more
- Volunteers
- 61 people
- Sponsor
- Ipsen
Pulmonary Disease, Chronic Obstructive, Amyotrophic Lateral Sclerosis
European Home Mechanical Ventilation Registry
The European Home Mechanical Ventilation Registry (EHMVR) will enable a thorough evaluation of HMV by documenting the characteristics of HMV patients and their treatment.
- Conditions
- Pulmonary Disease, Chronic Obstructive, Amyotrophic Lateral Sclerosis, Spinal Cord Injury, Muscular Dystrophies
- Trial name
- EHMVR (NCT02315339)
- Where
- Germany
- Volunteers
- 37 people
- Sponsor
- ResMed
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
Myositis, Active Idiopathic Inflammatory Myopathy
A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Active Idiopathic Inflammatory Myopathy
The main purpose of this study is to measure the long-term safety and tolerability of efgartigimod PH20 SC in adult participants with Idiopathic Inflammatory Myopathy (IIM) who previously participated in ARGX-113-2007.
- Conditions
- Myositis, Active Idiopathic Inflammatory Myopathy, Dermatomyositis, Polymyositis
- Trial name
- ALKIVIA+ (NCT05979441)
- Testing
- Efgartigimod PH20 SC
- Where
- 92 sites in United States, Argentina and 27 more
- Volunteers
- 240 people
- Sponsor
- argenx
Parkinsons Disease
First-in-Human Study of ALN-SNCA in Adult Participants With Early Parkinson's Disease (PD)
This study is researching an experimental drug called ALN-SNCA (called "study drug"). The study is focused on people with early Parkinson's disease, a disorder of the nervous system that affects movement.
- Trial name
- NCT07216066
- Testing
- ALN-SNCA
- Where
- 3 sites in Canada, Netherlands
- Volunteers
- 46 people
- Sponsor
- Regeneron Pharmaceuticals
Fibromyalgia
A Proof-of-concept Study to Evaluate the Efficacy and Safety of Rozanolixizumab to Treat Adult Study Participants With Severe Fibromyalgia Syndrome
The purpose of the study is to evaluate efficacy and safety of rozanolixizumab to treat adult study participants with severe fibromyalgia syndrome (FMS).
- Trial name
- NCT05643794
- Testing
- rozanolixizumab
- Where
- 7 sites in United Kingdom
- Volunteers
- 63 people
- Sponsor
- UCB Biopharma SRL
Advanced Malignant Neoplasm, Pigmented Villonodular Synovitis
Study of Vimseltinib (DCC-3014) in Patients With Advanced Tumors and Tenosynovial Giant Cell Tumor
This is a multicenter, open-label Phase 1/2 study of vimseltinib in patients with malignant solid tumors and tenosynovial giant cell tumor (TGCT).
- Conditions
- Advanced Malignant Neoplasm, Pigmented Villonodular Synovitis, Giant Cell Tumor of Tendon Sheath, Tenosynovial Giant Cell Tumor
- Trial name
- NCT03069469
- Testing
- Vimseltinib
- Where
- 24 sites in United States, Australia and 7 more
- Volunteers
- 120 people
- Sponsor
- Deciphera Pharmaceuticals, LLC
Dupuytren's Contracture
Post Approval Commitment Study
An agreement has been made to conduct a post-approval commitment study that provides supplementary information relating to the use of Xiapex® as well as other non-pharmacological treatments for Dupuytren's contracture by health…
- Trial name
- NCT01444729
- Testing
- Xiapex, surgery
- Where
- 7 sites in Norway, Spain
- Volunteers
- 305 people
- Sponsor
- Endo USA Inc., a Keenova Therapeutics Company
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
Fibromyalgia
Randomized, Double-Blind, Placebo-Controlled Study Of Pregabalin In Patients With Fibromyalgia
This study, will compare pregabalin with placebo for the duration of 15 weeks to evaluate the efficacy and safety of pregabalin in patients with fibromyalgia.
- Trial name
- NCT00830167
- Testing
- Pregabalin
- Where
- 35 sites in Japan
- Volunteers
- 498 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Primary Mitochondrial Myopathy
An Efficacy and Safety Study of 24 Week Treatment With Mavodelpar (REN001) in Primary Mitochondrial Myopathy Patients
This is a randomized, double-blind, placebo-controlled, parallel group, multi-centre, study designed to investigate the efficacy and safety of REN001 administered once daily over a 24-week period to patients with PMM.
- Trial name
- STRIDE (NCT04535609)
- Testing
- Mavodelpar
- Where
- 41 sites in United States, Australia and 13 more
- Volunteers
- 213 people
- Sponsor
- Reneo Pharma Ltd
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
Chronic Pain, Anxiety
Nu-V3 Non-Invasive Nerve Stimulation Device Trial for Chronic Pain, Anxiety, Depression, Sleeplessness
The Nu-V3 Clinical Study is a prospective, single-arm, open-label, multi-center study using the Nu-V3 cranial nerve stimulation treatment device in patients with chronic pain, anxiety, depression, and/or sleeplessness.
- Conditions
- Chronic Pain, Anxiety, Depression, Sleeplessness
- Trial name
- Nu-V3 (NCT05335980)
- Testing
- Nu-V3
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Nu-Life Solutions
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)
The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study.
- Trial name
- ENVISION (NCT05881408)
- Testing
- delandistrogene moxeparvovec
- Where
- 46 sites in United States, Australia and 12 more
- Volunteers
- 148 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
The study will evaluate the safety and efficacy of gene therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study with two thirds of participants assigned to gene therapy.
- Trial name
- NCT04281485
- Testing
- PF-06939926
- Where
- 53 sites in United States, Australia and 13 more
- Volunteers
- 114 people
- Sponsor
- Pfizer
Spasticity, Stroke
A Randomized, Double Blind, Multi-center, Active Drug Controlled, Phase III Clinical Trial to Compare the Safety and Efficacy of DWP450 Versus Botox® in Treatment of Post Stroke Upper Limb Spasticity
Purpose: This study is a Randomized, Double Blind, Multi-center, Active Drug Controlled, Phase III Clinical Trial to Compare the Safety and Efficacy of DWP450 Versus Botox® in Treatment of Post Stroke Upper Limb(wrist, finger…
- Trial name
- NCT01945684
- Testing
- Botulinum toxin type A
- Where
- South Korea
- Volunteers
- 197 people
- Sponsor
- Daewoong Pharmaceutical Co. LTD.
Spasticity With Multiple Sclerosis
Evaluation of the Effect of Nabiximols Oromucosal Spray on Clinical Measures of Spasticity in Participants With Multiple Sclerosis
This study will be conducted to evaluate the effect of multiple doses of nabiximols compared with placebo on a clinical measure of velocity-dependent muscle tone in the lower limbs (Lower Limb Muscle Tone-6 [LLMT-6]) in…
- Trial name
- RELEASE MSS5 (NCT04984278)
- Testing
- Nabiximols
- Where
- 30 sites in United States, Czechia and 3 more
- Volunteers
- 56 people
- Sponsor
- Jazz Pharmaceuticals
Hereditary Inclusion Body Myopathy, GNE Myopathy
GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)
HIBM is a severe progressive myopathy that typically presents in early adulthood as weakness in the distal muscles of the lower extremities and progresses proximally, leading to a loss of muscle strength and function, and…
- Conditions
- Hereditary Inclusion Body Myopathy, GNE Myopathy, Nonaka Disease, Quadriceps Sparing Myopathy (QSM)
- Trial name
- NCT01784679
- Where
- 5 sites in United States, Bulgaria and 3 more
- Volunteers
- 319 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Fibromyalgia, Myofascial Pain Syndromes
A Study to Evaluate eFFIcacy and Safety of Sublingual TNX-102 SL Tablet Taken at Bedtime in Patients With fibRoMyalgia
The use of low-dose CBP dosed nightly at bedtime for FM was supported by the results of Tonix' TNX-CY-F202 Phase 2b study (also referred to as the BESTFIT Study).
- Conditions
- Fibromyalgia, Myofascial Pain Syndromes, Muscular Diseases, Musculoskeletal Diseases
- Trial name
- AFFIRM (NCT02436096)
- Testing
- TNX-102 SL Tablet, 2.8mg
- Where
- 35 sites in United States
- Volunteers
- 519 people
- Sponsor
- Tonix Pharmaceuticals, Inc.
Muscle Weakness
Functional Change With MMS
Evaluate the safety and efficacy of magnetic muscle stimulation (MMS) of abdominal muscle.
- Trial name
- FMS (NCT04506502)
- Testing
- The ZELTIQ System
- Where
- United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Zeltiq Aesthetics