Clinical trials: Neuromuscular Disorder
X-Linked Myotubular Myopathy
Study of ASP2957 in Male Participants With X-linked Myotubular Myopathy Who Need Ventilators
X-linked myotubular myopathy (XLMTM) is a rare and serious condition present at birth where the muscles do not work properly. There are currently no approved therapies for XLMTM.
- Trial name
- NCT07052929
- Testing
- ASP2957, Methylprednisolone, Prednisolone
- Where
- 5 sites in United States, Canada
- Volunteers
- 9 people
- Sponsor
- Astellas Gene Therapies
Generalized Myasthenia Gravis (gMG), gMG
A Study to Evaluate the Impact of Efgartigimod on Overall Disease Experience of People Suffering From Generalized Myasthenia Gravis (gMG) in Italy
This study aims to generate real world evidence (RWE) from Italian clinical practice on the impact of efgartigimod alfa in gMG patients encompassing clinical outcomes and patient reported experiences.
- Trial name
- POEMA (NCT07595653)
- Where
- 30 sites in Italy
- Volunteers
- 100 people
- Sponsor
- argenx
Mitochondrial Diseases, Maternally Inherited Diabetes and Deafness (MIDD)
KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases
The KHENERFIN study aims to determine whether the study medicine, sonlicromanol, is able to reduce symptoms of fatigue and the impact of fatigue on daily life, and whether sonlicromanol is able to improve physical abilities of…
- Conditions
- Mitochondrial Diseases, Maternally Inherited Diabetes and Deafness (MIDD), Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS), Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
- Trial name
- KH176-301 (NCT06451757)
- Testing
- Sonlicromanol
- Where
- 10 sites in United States, Denmark and 5 more
- Volunteers
- 220 people
- Sponsor
- Khondrion BV
Healthy Volunteers
A Study of VRG50635 in Healthy Volunteers
The primary objectives of this study are to investigate the safety and tolerability of VRG50635 and to determine how VRG50635 is absorbed by the body.
- Trial name
- NCT06286475
- Testing
- VRG50635
- Where
- United States
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- Verge Genomics
Incurable Metastatic Colorectal Carcinoma, Incurable Progressive, Recurrent or Metastatic Squamous Cell Carcinoma of the Head and Neck
Phase 1 and 2 Study of PX-866 and Cetuximab
The purpose of this Phase 1/2 open-label study is to determine the safety and efficacy of a cetuximab and PX-866 combination treatment.
- Trial name
- NCT01252628
- Testing
- PX-866 (SCCHN), Cetuximab (SCCHN), PX-866 (CRC)
- Where
- 40 sites in United States, Canada
- Volunteers
- 178 people
- Sponsor
- Cascadian Therapeutics Inc.
Amyotrophic Lateral Sclerosis
A Biomarker Study to Evaluate MN-166 in Subjects With Amyotrophic Literal Sclerosis (ALS)
This is a multi-center, open-label study of MN-166 (ibudilast) in subjects with ALS. To be eligible subjects must meet the El Escorial criteria of possible, laboratory-supported probable, probable, or definite criteria for a…
- Trial name
- NCT02714036
- Testing
- ibudilast
- Where
- 2 sites in United States
- Volunteers
- 35 people
- Sponsor
- MediciNova
Diphtheria, Tetanus
Study of DTaP-IPV-Hep B-PRP~T Combined Vaccine Compared With PENTAXIM™ and ENGERIX B® PEDIATRICO in Argentinean Infants
Primary Objective: To demonstrate that the immune response of the DTaP-IPV-Hep B-PRP~T is non-inferior for all valences to those of the association of PENTAXIM™ and ENGERIX B® PEDIATRICO one month after a three-dose primary…
- Conditions
- Diphtheria, Tetanus, Pertussis, Haemophilus Influenzae Type b
- Trial name
- NCT00831311
- Testing
- DTaP-IPV-HB-PRP~T, DTaP-IPV//PRP~T combined vaccine & Recombinant hep B vaccine
- Where
- Argentina
- Volunteers
- 624 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company
Tetraplegia/Tetraparesis, Quadriplegia
GB-PRIME: An Early Feasibility Study of a Precise Robotically Implanted Brain-Computer Interface for the Control of External Devices
The GB-PRIME Study is an early feasibility study designed to assess the clinical safety and functionality of the Neuralink N1 Implant and R1 Robot.
- Conditions
- Tetraplegia/Tetraparesis, Quadriplegia, Quadriplegia/Tetraplegia, Cervical Spinal Cord Injury
- Trial name
- GB-PRIME (NCT07127172)
- Testing
- N1 Implant, R1 Robot
- Where
- 2 sites in United Kingdom
- Volunteers
- 7 people
- Sponsor
- Neuralink Corp
Duchenne Muscular Dystrophy
Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or…
- Trial name
- NCT01247207
- Testing
- Ataluren
- Where
- 34 sites in United States, Canada
- Volunteers
- 265 people
- Sponsor
- PTC Therapeutics
Pain, Pain Syndrome
Efficacy and Tolerability of AP707 in Patients With Chronic Pain Due to Traumatic or Post-operative Peripheral Neuropathy
Over the last years a rising medical need for treatment of chronic pain was identified. Based on previous findings indicating the pain modulating effects of cannabinoids in chronic pain disorders, this clinical trial investigates…
- Conditions
- Pain, Pain Syndrome, Pain, Chronic, Chronic Pain
- Trial name
- NCT06071936
- Testing
- Adezunap (AP707)
- Where
- Germany
- Volunteers
- 558 people
- Sponsor
- Apurano Pharmaceuticals GmbH
Peripheral Neuropathy
BlueWind Medical Reprieve System for the Treatment of PNP
The current study will evaluate the safety and performance of the BlueWind Medical Reprieve System for the treatment of Chronic Painful Peripheral Neuropathy.
- Trial name
- PNP (NCT02062398)
- Testing
- The Reprieve System
- Where
- 4 sites in Belgium, Poland
- Volunteers
- 13 people
- Sponsor
- BlueWind Medical
Amyotrophic Lateral Sclerosis (ALS)
Phase 2, Randomized, Double Blind, Placebo Controlled Multicenter Study of Autologous MSC-NTF Cells in Patients With ALS
This is a multi-center, randomized, double blind, placebo controlled study to evaluate the safety and efficacy of autologous (self) transplantation of Neurotrophic factors-secreting Mesenchymal Stromal Cells (MSC-NTF, NurOwn™) in…
- Trial name
- NurOwn (NCT02017912)
- Testing
- Nurown MSC-NTF cells
- Where
- 3 sites in United States
- Volunteers
- 48 people
- Sponsor
- Brainstorm-Cell Therapeutics
Stroke, Wound Heal
Study of gekoTM Interaction With Cardiac Pacemakers
The gekoTM device is a small disposable battery powered device that attaches to the lower leg near the knee and stimulates the common peroneal nerve, causing muscle contraction of the lower leg.
- Conditions
- Stroke, Wound Heal, Edema Leg, Cardiac Pacemaker Electrical Interference
- Trial name
- NCT04391257
- Testing
- geko™
- Where
- 2 sites in United Kingdom
- Volunteers
- 28 people
- Sponsor
- Firstkind Ltd
Healthy Volunteers
A Study to Determine the Relative Bioavailability of Capsule and Tablet Formulations of GDC-0941 and the Effect of Food and Proton Pump Inhibition on GDC-0941 Tablet Pharmacokinetics in Healthy Volunteers
This is a Phase I, randomized, two-part, single-center pharmacokinetics (PK) study to assess the relative bioavailability of capsule and tablet formulations of GDC-0941 in the fasted state (Part 1) and to determine the effect of…
- Trial name
- NCT00999128
- Testing
- GDC-0941, rabeprazole
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Genentech, Inc.
Hepatitis B, Polio
Comparison of a DTaP-IPV-HB-PRP~T Combined Vaccine to Infanrix™-Hexa, When Administered With Prevnar® in Thai Infants
The purpose of the study is to provide immunogenicity and safety data of the investigational hexavalent vaccine when it is given concomitantly (the same day at separate injection sites) with Prevnar, according to the 2-4-6 month…
- Conditions
- Hepatitis B, Polio, Diphtheria, Pertussis
- Trial name
- NCT00401531
- Testing
- DTaP-IPV-HB-PRP~T and Pneumococcal polysaccharide vaccines, DTaP-HB-IPV and Pneumococcal polysaccharide vaccines
- Where
- 2 sites in Thailand
- Volunteers
- 412 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company
Juvenile Dermatomyositis
Clinical Trial of CD19-targeted CAR-T Therapy for Refractory Juvenile Dermatomyositis
This is a Phase I clinical trial to evaluate the efficacy and safety of CD19-targeted CAR-T in the treatment of refractory juvenile dermatomyositis (RJDM).The experiment was divided into two phases: dose exploration (Part A) and…
- Trial name
- NCT06569472
- Testing
- CD19-targetd CAR-T
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Chongqing Precision Biotech Co., Ltd
Stroke, Muscle Spasticity
Anti-Gravity Treadmill and Electromagnetic Stimulation in Stroke Rehabilitation
The goal of this clinical trial is to learn if adding new technologies to standard therapy helps people recover after a stroke. It looks at adults aged 18 to 74 who recently had a stroke and need help with moving and walking.
- Conditions
- Stroke, Muscle Spasticity, Stroke Rehabilitation
- Trial name
- NCT07698639
- Testing
- Anti-Gravity Treadmill, High-Intensity Electromagnetic Stimulation, Standard Rehabilitation
- Where
- Russia
- Volunteers
- 105 people
- Sponsor
- BTL Industries Ltd.
Duchenne Muscular Dystrophy
Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)
The purpose of the study is to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study.
- Trial name
- SIDEROS-E (NCT03603288)
- Testing
- idebenone 150 mg film-coated tablets
- Where
- 39 sites in United States, Austria and 7 more
- Volunteers
- 161 people
- Sponsor
- Santhera Pharmaceuticals
Muscle Spasm
Low Intensity Ultrasound Therapy for Upper Back Pain Relief
The purpose of this study is to measure the effectiveness of a wearable therapeutic ultrasound device for relief of pain associated with the trapezius muscle of the upper back over a 4-week period.
- Trial name
- NCT02135094
- Testing
- Active ultrasound therapy device
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- ZetrOZ, Inc.
Haematologic Neoplasms
A Study of AZD2962, an IRAK4 Inhibitor (IRAK4 [a Body Protein] Blocker), in Participants With Haematologic Neoplasms (Blood Cancers)
The purpose of the study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics, and preliminary efficacy of AZD2962, an Interleukin-1 Receptor-Associated Kinase 4 (IRAK4) inhibitor, as monotherapy and in…
- Trial name
- NCT07064122
- Testing
- AZD2962
- Where
- 21 sites in United States, Australia and 5 more
- Volunteers
- 72 people
- Sponsor
- AstraZeneca
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