Clinical trials: Neuromuscular Disorder
Duchenne Muscular Dystrophy
Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to see whether PRO044 is safe and effective to use as medication for Duchenne Muscular Dystrophy (DMD) patients with a mutation around location 44 in the DNA for the dystrophin protein.
- Trial name
- NCT02329769
- Testing
- PRO044 SC 6 mg/kg, PRO044 IV 6 mg/kg, PRO044 IV 9 mg/kg
- Where
- 5 sites in Belgium, Italy and 2 more
- Volunteers
- 15 people
- Sponsor
- BioMarin Pharmaceutical
Neuropathic Pain
Safety, Tolerability, and Pharmacokinetics of Multiple Ascending Doses of REL-1017 (d-Methadone)
This study evaluated the safety, tolerance, and pharmacokinetics (PK) of d-methadone in a limited dose range, in multiple administrations in humans.
- Trial name
- NCT03638869
- Testing
- REL-1017
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Relmada Therapeutics, Inc.
Refractive Errors
Clinical Evaluation of Acuity 200 (Fluoroxyfocon A) RGP Contact Lens
The objective of this clinical investigation is to collect valid scientific safety and effectiveness data on the Acuity 200 (fluoroxyfocon A) Rigid Gas Permeable Contact Lens, manufactured from a newly developed rigid gas…
- Trial name
- Acuity 200 (NCT03971227)
- Testing
- Acuity 200 (Fluoroxyfocon A) contact lens, Acuity 100 (Hexafocon A) contact lens
- Where
- 3 sites in United States
- Volunteers
- 74 people, healthy volunteers welcome
- Sponsor
- Acuity Polymers, Inc.
Fibromyalgia
Safety and Efficacy of AGN 203818 for Pain Associated With Fibromyalgia Syndrome
This study will explore the safety and effectiveness of different doses of AGN 203818 in treating the pain associated with fibromyalgia syndrome. The study is being conducted in 2 parts.
- Trial name
- NCT00445705
- Testing
- AGN 203818
- Where
- 3 sites in United States, Switzerland and 1 more
- Volunteers
- 211 people
- Sponsor
- Allergan
Spinal Muscular Atrophy
A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).
The primary objective of Part 1 of this study is to assess the safety and tolerability of Nusinersen in participants with SMA who are not eligible to participate in the clinical studies ISIS 396443-CS3B (NCT02193074) or ISIS…
- Trial name
- EMBRACE (NCT02462759)
- Testing
- Nusinersen
- Where
- 7 sites in United States, Germany
- Volunteers
- 21 people
- Sponsor
- Biogen
Nerve Pain
A Study to Investigate the Analgesic Efficacy of AZD2423 Compared With Placebo After 28 Days Treatment in Patients With Posttraumatic Neuralgia..
A study to investigate the analgesic efficacy of AZD2423 compared with placebo after 28 days treatment in patients with posttraumatic neuralgia.
- Trial name
- NCT01200524
- Testing
- AZD2423
- Where
- 25 sites in Bulgaria, Denmark and 5 more
- Volunteers
- 133 people
- Sponsor
- AstraZeneca
Generalized Myasthenia Gravis
A Study of Efgartigimod PH20 SC in Children Between 2 and Less Than 18 Years of Age With Generalized Myasthenia Gravis
The purpose of this study is to measure the pharmacokinetics (PK), pharmacodynamics (PD), safety, tolerability, and immunogenicity of efgartigimod PH20 SC in pediatric participants with gMG aged 2 to <18 years.
- Trial name
- ADAPT Jr SC (NCT06392386)
- Testing
- Efgartigimod PH20 SC
- Where
- 22 sites in United States, Australia and 12 more
- Volunteers
- 12 people
- Sponsor
- argenx
Duchenne Muscular Dystrophy
A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)
The LYNX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics and biomarkers in children with Duchenne muscular dystrophy including a randomized, double-blind, placebo-controlled part A, followed by an…
- Trial name
- NCT05540860
- Testing
- Sevasemten Dose 1, Sevasemten Dose 2, Sevasemten Dose 3
- Where
- 14 sites in United States
- Volunteers
- 76 people
- Sponsor
- Edgewise Therapeutics, Inc.
Fibromyalgia
A Study to Evaluate Lu AG06466 in Participants With Fibromyalgia
The purpose of this study is to find out the effect of Lu AG06466 on the body in participants with fibromyalgia by assessing pain levels, brain signal changes, and psychiatric (mental) assessments.
- Trial name
- NCT04974359
- Testing
- Lu AG06466
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- H. Lundbeck A/S
Group B Streptococcus (GBS) Disease
A Study to Assess the Persistence of a GBS Antibody in Women Previously Immunized With a GBS Vaccine
This study looks at the body's immune responses to a GBS vaccine 2 years after the orginal vaccine(s) were given in the V98P1 study. Blood will be drawn and evaluated for GBS antibody levels.
- Trial name
- NCT01052935
- Testing
- Group B streptococcus (GBS) vaccine
- Where
- Switzerland
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Novartis
Amyotrophic Lateral Sclerosis (ALS)
Humanitarian Device Exemption Post-Approval Study of NeuRx Diaphragm Pacing System for Amyotrophic Lateral Sclerosis
This post-approval study will follow 60 participants who have ALS, documented chronic hypoventilation, and bilateral phrenic nerve function, and who undergo the surgical implantation procedure to receive the NeuRx Diaphragm…
- Trial name
- NCT01605006
- Testing
- NeuRx Diaphragm Pacing System (DPS)
- Where
- 11 sites in United States
- Volunteers
- 97 people
- Sponsor
- Synapse Biomedical
Neuromuscular Blockade, Arthroplasty, Replacement, Hip
Reversal of Neuromuscular Blockade With Sugammadex or Usual Care in Hip Fracture Surgery or Joint (Hip/Knee) Replacement (P07038)
This study will assess the effect of reversal of neuromuscular blockade with sugammadex compared with reversal according to usual care (neostigmine or spontaneous reversal) on the incidence of post-surgical bleeding events and on…
- Conditions
- Neuromuscular Blockade, Arthroplasty, Replacement, Hip, Arthroplasty, Replacement, Knee, Blood Coagulation
- Trial name
- NCT01422304
- Testing
- Sugammadex, neostigmine and glycopyrrolate or atropine
- Volunteers
- 1,198 people
- Sponsor
- Merck Sharp & Dohme LLC
Spinal Muscular Atrophy (SMA)
Virtual Reality for the Mitigation of Anxiety During Intrathecal Administration in Participants With Spinal Muscular Atrophy
The primary objective of the study is to evaluate anxiety level during intrathecal administration (IT) under standard of care (SOC) and virtual reality (VR) conditions using a reliable self-rating scale.
- Trial name
- REALITY (NCT05354414)
- Testing
- Virtual reality, Standard of Care
- Where
- 12 sites in France
- Volunteers
- 63 people
- Sponsor
- Biogen
Spasticity, Multiple Sclerosis
One Year Extension Study To Protocol C2/5/TZ:MS-05
Open label, one year extension study to evaluate the clinical efficacy and safety of 12 mg sublingual tizanidine administered once nightly in MS patients who successfully completed Phase I/II protocol C2/5/TZ:MS-05 at the Tel…
- Trial name
- NCT00464958
- Testing
- sublingual tizanidine 12 mg
- Where
- Israel
- Volunteers
- 10 people
- Sponsor
- Teva GTC
Fibromyalgia
Study of the Symptomatic Improvement in Patients With Fibromyalgia by Means of a Food Supplement
Fibromyalgia is a syndrome (set of symptoms) of unknown etiology and characterized by diffuse musculoskeletal pain and chronic fatigue.
- Trial name
- FIBROFIN (NCT05718908)
- Testing
- Food supplement
- Where
- Spain
- Volunteers
- 30 people
- Sponsor
- Arafarma Group, S.A.
Amyotrophic Lateral Sclerosis
A Substudy of LY4256984 in Participants With Sporadic Amyotrophic Lateral Sclerosis
The main purpose of this study is to assess the long-term safety and tolerability of LY4256984 in participants with Amyotrophic Lateral Sclerosis (ALS).
- Trial name
- NCT07571174
- Testing
- LY4256984
- Where
- 11 sites in Belgium, Canada and 3 more
- Volunteers
- 32 people
- Sponsor
- Eli Lilly and Company
HFpEF - Heart Failure With Preserved Ejection Fraction, HFmrEF
US Benchmarking Clinical Study
The goal of this observational study is to measure shear wave velocity (SWV) in patients with non-reduced left ventricular ejection fraction (LVEF) heart failure and left ventricular hypertrophy, with or without transthyretin…
- Conditions
- HFpEF - Heart Failure With Preserved Ejection Fraction, HFmrEF, ATTR-CM (Transthyretin Amyloid Cardiomyopathy), Left Ventricular Hypertrophy
- Trial name
- EMS-CT-1 (NCT07215715)
- Where
- 3 sites in United States
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- eMyosound SAS
Fibromyalgia
Adolescent Fibromyalgia Study
This study will compare pregabalin with placebo over a 15 week period in adolescents with fibromyalgia aged 12-17 years to evaluate the safety and efficacy of pregabalin.
- Trial name
- NCT01020474
- Testing
- pregabalin (Lyrica)
- Where
- 36 sites in United States, Czechia and 2 more
- Volunteers
- 107 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Pain, Delayed Onset Muscle Soreness
Phase II Study of AP0302 5% Versus a Vehicle Comparator
This study is designed to evaluate the analgesic safety and efficacy of study drug (AP0302) applied topically every 6 hours as compared to a vehicle, in subjects experiencing delayed onset muscle soreness.
- Trial name
- NCT02324985
- Testing
- S-Ibuprofen Topical Gel 5%, Vehicle Topical Gel
- Where
- United States
- Volunteers
- 147 people, healthy volunteers welcome
- Sponsor
- Aponia Laboratories, Inc.
Hemoglobinopathies, Myelodysplastic Syndromes
Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload
This study will evaluate the change in cardiac iron load over a 53 week period measured by MRI in 2 cohorts of patients
- Conditions
- Hemoglobinopathies, Myelodysplastic Syndromes, Other Inherited or Acquired Anaemia, MPD Syndrome
- Trial name
- NCT00673608
- Testing
- deferasirox
- Where
- 4 sites in Australia
- Volunteers
- 118 people
- Sponsor
- Novartis
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