Clinical trials: Muscle Disorders
Spinal Muscular Atrophy Type I
Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1
The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of EXG001-307 as a treatment of spinal muscular atrophy Type 1 (SMN1).
- Trial name
- NCT05614531
- Testing
- EXG001-307 injection
- Where
- 2 sites in China
- Volunteers
- 12 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd
Duchenne Muscular Dystrophy
A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy
This is a first-in-human/first-in-patient, multi-center, open-label, non-randomized, ascending dose, safety and tolerability study of a single intravenous infusion of PF-06939926 in ambulatory and non-ambulatory subjects with…
- Trial name
- NCT03362502
- Testing
- PF-06939926
- Where
- 22 sites in United States
- Volunteers
- 23 people
- Sponsor
- Pfizer
Airway Obstruction, Asthma
Effects of Combined Respiratory Muscle Training and Steam Inhalation
The purpose of this investigation is to obtain more information on the efficacy and safety of respiratory training methods with WellO2 in patients with asthma and COPD.
- Conditions
- Airway Obstruction, Asthma, COPD
- Trial name
- NCT04584398
- Testing
- Respiratory muscle training with steam inhalation
- Where
- Finland
- Volunteers
- 60 people
- Sponsor
- WellO2 Oy
Bladder Cancer
Treatment of High-risk Non-muscle Invasive Bladder Cancer With IMUNO BGC Moreau RJ as Prophylaxis
Bladder cancer is the fourth reason of neoplasia in our environment, there are 25 new cases diagnosed per 100,000 inhabitants each year.
- Trial name
- NCT03982797
- Testing
- Instillation of IMUNO BCG Moreau RJ
- Where
- 15 sites in Spain
- Volunteers
- 306 people
- Sponsor
- Biofabri, S.L
Primary Mitochondrial Disease
Safety, Tolerability, Efficacy of MTP-131 for Treatment of Mitochondrial Disease in Subjects From the MMPOWER Study
This randomized, double-blind, placebo-controlled, crossover study screened 32 subjects with primary mitochondrial myopathy (PMM) to evaluate the safety, tolerability, pharmacokinetics (PK), and efficacy of subcutaneous…
- Trial name
- MMPOWER-2 (NCT02805790)
- Testing
- Elamipretide
- Where
- 4 sites in United States
- Volunteers
- 30 people
- Sponsor
- Stealth BioTherapeutics Inc.
Muscular Dystrophies, Spinal Muscular Atrophy
Schulze Muscular Dystrophy Ability Clinical Study
The primary objective of the Schulze study is to evaluate the function of the upper limbs of subjects diagnosed with neuromuscular disorders, with and without use of the Abilitech Assist device in the clinic and home…
- Conditions
- Muscular Dystrophies, Spinal Muscular Atrophy, Duchenne Muscular Dystrophy, Limb Girdle Muscular Dystrophy
- Trial name
- NCT05409079
- Testing
- Abilitech Assist
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- AbiliTech Medical Inc.
Duchenne Muscular Dystrophy
Long Term Follow-up for RGX-202
RGX-202-5101 is a long-term follow up study that evaluates the long-term safety and efficacy of RGX-202 in participants who have received RGX-202 (an investigative gene therapy designed to deliver a transgene for novel…
- Trial name
- NCT06491927
- Testing
- No Intervention
- Where
- 5 sites in United States
- Volunteers
- 66 people
- Sponsor
- REGENXBIO Inc.
Advanced or Metastatic Solid Tumor, Pancreatic Ductal Adenocarcinoma
AB122 Platform Study
This is a phase 1, non-randomized open-label, multicenter platform study designed to evaluate the tolerability and safety of AB122 in patients with malignancies specified in each cohort.
- Conditions
- Advanced or Metastatic Solid Tumor, Pancreatic Ductal Adenocarcinoma, Colorectal Cancer, Non-small Cell Lung Cancer
- Trial name
- NCT04999761
- Testing
- AB122, TAS-116, TAS-120
- Where
- 9 sites in Japan
- Volunteers
- 917 people
- Sponsor
- Taiho Pharmaceutical Co., Ltd.
Muscular Atrophy, Spinal
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2…
- Trial name
- HINALEA 1 (NCT05861986)
- Testing
- Risdiplam
- Where
- 16 sites in United States, Germany and 2 more
- Volunteers
- 28 people
- Sponsor
- Hoffmann-La Roche
Muscular Dystrophy (DMD)
Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD
The purpose of this study is to determine the safety and tolerability of RO7239361 in boys with Duchenne Muscular Dystrophy with any genetic mutation.
- Trial name
- NCT02515669
- Testing
- RO7239361
- Where
- 12 sites in United States, Canada
- Volunteers
- 43 people
- Sponsor
- Hoffmann-La Roche
Fibromyalgia
Safety and Efficacy Study of Xyrem® (Sodium Oxybate) in Subjects With Fibromyalgia.
The objective of this trial is to evaluate the safety and efficacy of Xyrem® in long term use.
- Trial name
- NCT00423605
- Testing
- Xyrem®
- Where
- 91 sites in United States, France and 5 more
- Volunteers
- 560 people
- Sponsor
- Jazz Pharmaceuticals
Tendon Injuries, Musculoskeletal Pain
Long Duration Therapeutic Ultrasound on Tendon Injuries
Human clinical trial to measure the effect of long duration therapeutic ultrasound on tendon injuries. The hypothesis is that use of long duration ultrasound will relieve pain, increase tendon strength, and improve quality of…
- Trial name
- NCT02340273
- Testing
- Therapeutic ultrasound
- Where
- 2 sites in United States
- Volunteers
- 13 people
- Sponsor
- ZetrOZ, Inc.
Pediatrics, Muscle Spasticity
BOTOX® Open-Label Treatment in Pediatric Lower Limb Spasticity
This study will evaluate the long-term safety of BOTOX® (botulinum toxin Type A) for the treatment of pediatric lower limb spasticity.
- Conditions
- Pediatrics, Muscle Spasticity, Cerebral Palsy
- Trial name
- NCT01603641
- Testing
- Botulinum Toxin Type A
- Where
- 56 sites in United States, Hungary and 7 more
- Volunteers
- 370 people
- Sponsor
- Allergan
Myotonic Dystrophy 1
A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
The purpose of this study is to learn about the effects of an investigational medicine, PGN-EDODM1, to see how safe and tolerable multiple administrations of PGN-EDODM1 are for people with myotonic dystrophy type 1 (DM1) compared…
- Trial name
- FREEDOM2-DM1 (NCT06667453)
- Testing
- PGN-EDODM1
- Where
- 12 sites in Canada, New Zealand and 2 more
- Volunteers
- 24 people
- Sponsor
- PepGen Inc
Neuromuscular Diseases, Cerebral Palsy
Rehabilitative Effect of the Use of a Gait Exoskeleton in Patients With Neuromuscular Disease or Cerebral Palsy
The purpose of this multicentric study is to assess the rehabilitative effect derived from the use of the ATLAS exoskeleton in children with neuromuscular diseases or cerebral palsy and with a level of less than 3 or less in the…
- Conditions
- Neuromuscular Diseases, Cerebral Palsy, SMA II
- Trial name
- NCT04813601
- Testing
- ATLAS 2030
- Where
- Spain
- Volunteers
- 10 people
- Sponsor
- MarsiBionics
Tendinopathy, Tendon Tear
NeoPatch Used as Adjunct in Foot and Ankle Tendon Surgery
This study is a prospective, single center, postmarket study to evaluate the safety and effectiveness of NeoPatch used as a tissue cover in foot and ankle tendon revision and repair surgery.
- Conditions
- Tendinopathy, Tendon Tear, Tendon Injuries, Foot Ankle Injuries
- Trial name
- NCT03307499
- Testing
- NeoPatch
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- CryoLife, Inc.
Uterine Fibroids, Endometriosis
A Phase 3B Study to Evaluate Bone Mineral Density With Long-Term Use of Relugolix Combination Tablet in Women With Uterine Fibroids or Endometriosis
The purpose of this clinical trial to characterize changes in bone mineral density during continuous treatment with relugolix combination tablet for up to 48 months (4 years) and 1 year of post-treatment follow-up in…
- Trial name
- NCT05862272
- Testing
- Relugolix Combination Tablet
- Where
- 120 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
Leiomyoma, Uterine Hemorrhage
A Study of the Efficacy and Safety of a 3-month Treatment Course of Ulipristal Acetate for the Treatment of Abnormal Uterine Bleeding Associated With Leiomyomas
This study will evaluate the superiority of ulipristal acetate versus placebo for the treatment of abnormal uterine bleeding associated with uterine fibroids
- Trial name
- NCT02147197
- Testing
- Ulipristal acetate (UPA)
- Where
- 28 sites in United States
- Volunteers
- 157 people
- Sponsor
- Allergan
Hyperlipidemia
Goal Achievement After Utilizing an Anti-PCSK9 Antibody in Statin Intolerant Subjects-3
The primary objective of this study was to evaluate the effect of 24 weeks of evolocumab administered subcutaneously (SC) every month, compared with ezetimibe, on low-density lipoprotein cholesterol (LDL-C) levels in adults with…
- Trial name
- GAUSS-3 (NCT01984424)
- Testing
- Atorvastatin, Ezetimibe
- Where
- 58 sites in United States, Australia and 11 more
- Volunteers
- 511 people
- Sponsor
- Amgen
Musculoskeletal Pain
Study of Epicutaneously Applied Ketoprofen Transfersome® Gel With or Without Combination With Oral Celecoxib for the Treatment of Muscle Pain Induced by Eccentric Exercise
1. Comparison of the effect of ketoprofen Transfersome® gel (KTG) to placebo gel (PG) on muscle pain of the calf caused by eccentric contractions 2.
- Trial name
- NCT01020279
- Testing
- Celecoxib, Ketoprofen, Ketoprofen
- Where
- Germany
- Volunteers
- 82 people, healthy volunteers welcome
- Sponsor
- X-pert Med GmbH
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