Clinical trials: Neuromuscular Disorder
Polyneuropathy, Inflammatory Demyelinating, Chronic
Real-world Study of Treatment Outcomes in Chronic Inflammatory Demyelinating Polyneuropathy/Polyradiculoneuropathy (CIDP)
This study is an observational, ambispective, descriptive, non-interventional study of people with a chronic inflammatory demyelinating polyneuropathy/polyradiculoneuropathy (CIDP) diagnosis in the United States with residual…
- Trial name
- ORBIT-CIDP (NCT06968975)
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Sanofi
Duchenne Muscular Dystrophy
Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping
This is a multicenter, open-label, dose-escalation study to evaluate the safety, tolerability, and PK of once-weekly IV infusions of eteplirsen in approximately 12 male participants, ages 6 months to 48 months (inclusive), who…
- Trial name
- NCT03218995
- Testing
- Eteplirsen
- Where
- 4 sites in Belgium, France and 2 more
- Volunteers
- 15 people
- Sponsor
- Sarepta Therapeutics, Inc.
Diphtheria, Tetanus
Study of the Effect of Pneumococcal Conjugate Vaccine (PCV) on Immunogenicity of Pentacel™
This study is designed to evaluate in a controlled manner the effect of Prevnar® on the immune responses of Pentacel™ Primary Objective - Stage I: To compare the immune responses elicited by an infant series of Pentacel™ when…
- Conditions
- Diphtheria, Tetanus, Haemophilus Infection, Pertussis
- Trial name
- NCT00772928
- Testing
- Pentacel™: HCPDT-IPV//PRP-T Vaccine and Prevnar®
- Where
- 23 sites in United States
- Volunteers
- 1,167 people, healthy volunteers welcome
- Sponsor
- Sanofi
Duchenne Muscular Dystrophy
An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy
This is a multicenter, prospective, observational Phase 4 study including a post marketing safety requirement, designed to collect both medical history data and prospective data on Duchenne muscular dystrophy (DMD) treatment…
- Trial name
- ENDURE (NCT06270719)
- Testing
- Delandistrogene Moxeparvovec, Standard of Care
- Where
- 29 sites in United States
- Volunteers
- 500 people
- Sponsor
- Sarepta Therapeutics, Inc.
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
Amyotrophic Lateral Sclerosis
Intermediate-Sized Expanded Access Study
This expanded access protocol is to provide access to the investigational product, SLS-005, to participants with ALS who are not eligible to participate in clinical trials.
- Trial name
- NCT05597436
- Testing
- Trehalose
- Where
- 21 sites in United States
- Sponsor
- Seelos Therapeutics, Inc.
Fibromyalgia
A Study to Test the Effectiveness and Safety of Fremanezumab on Participants With Fibromyalgia
The primary objective of the study is to estimate the treatment effect of fremanezumab administered subcutaneously (SC) in reducing pain in adult participants with fibromyalgia (FM).
- Trial name
- NCT03965091
- Testing
- Fremanezumab
- Where
- 49 sites in United States
- Volunteers
- 189 people
- Sponsor
- Teva Branded Pharmaceutical Products R&D, Inc.
Dermatomyositis, Polymyositis, Sjogren's, SLE, SSc
A Phase 1 Study of MEDI7734 in Type I Interferon-Mediated Autoimmune Diseases
To evaluate the safety and tolerability of escalating, single subcutaneous doses of MEDI7734 in adult subjects with type I interferon-mediated autoimmune diseases.
- Trial name
- NCT02780674
- Testing
- MEDI7734
- Where
- 7 sites in United States
- Volunteers
- 36 people
- Sponsor
- Amgen
Vitamin D Deficiency
A Study to Assess the Effectiveness of an Investigational Drug in Elderly Subjects With Low Vitamin D Levels (0822A-009)
This study will assess the effectiveness of an investigational drug in elderly subjects with low vitamin D levels.
- Trial name
- NCT00242476
- Testing
- MK0822A
- Volunteers
- 216 people, healthy volunteers welcome
- Sponsor
- Merck Sharp & Dohme LLC
Amyotrophic Lateral Sclerosis, ALS
Radicava® (Edaravone) Findings in Biomarkers From ALS (REFINE-ALS)
REFINE-ALS is a prospective, observational, longitudinal, multicenter study designed to identify biomarkers to serve as quantifiable biological non-clinical measures of Edaravone effects in ALS.
- Trial name
- NCT04259255
- Testing
- Edaravone (Radicava®/Radicava ORS®)
- Where
- 20 sites in United States, Canada
- Volunteers
- 74 people
- Sponsor
- Shionogi
Duchene Muscular Dystrophy
A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy
This is a prospective observational study conducted to evaluate safety, tolerability, and functional outcomes of patients with DMD newly initiating oral givinostat or having started therapy within 6 months as part of routine…
- Trial name
- PROVIDUS (NCT07127978)
- Where
- 9 sites in United States
- Volunteers
- 300 people
- Sponsor
- ITF Therapeutics LLC
Generalized Myasthenia Gravis
Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis
The purpose of this study is to evaluate the safety and efficacy of ALXN1720 for the treatment of generalized MG (gMG) in adults with autoantibodies against acetylcholine receptor (AChR).
- Trial name
- NCT05556096
- Testing
- ALXN1720
- Where
- 132 sites in United States, Argentina and 20 more
- Volunteers
- 260 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Dermatomyositis, Polymyositis
A Study to Understand How the Study Medicine Dazukibart Works in People With Idiopathic Inflammatory Myopathies
The purpose of this study is to understand how the study medicine, dazukibart, works in people with active idiopathic inflammatory myopathies (dermatomyositis [DM] or polymyositis [PM]).
- Trial name
- NCT06698796
- Testing
- Dazukibart
- Where
- 26 sites in United States, Argentina and 10 more
- Volunteers
- 211 people
- Sponsor
- Pfizer
Generalized Myasthenia Gravis
A Safety and Tolerability Study of ARGX-113 in Patients With Myasthenia Gravis Who Have Generalized Muscle Weakness.
This is a Long-Term, Single-Arm, Open-Label, Multicenter Phase 3 follow-on trial of the ARGX-113-1704 study to evaluate the safety and tolerability of ARGX-113 in patients with gMG.
- Trial name
- ADAPT+ (NCT03770403)
- Testing
- ARGX-113
- Where
- 52 sites in United States, Belgium and 13 more
- Volunteers
- 151 people
- Sponsor
- argenx
Amyotrophic Lateral Sclerosis
Treatment Continuation Study for Patients With ALS/MND Who Completed Study CMD-2019-001
Provides up to six months treatment with CuATSM for subjects who have successfully completed study CMD-2019-001
- Trial name
- NCT04313166
- Testing
- Cu(II)ATSM
- Where
- Australia
- Volunteers
- 55 people
- Sponsor
- Collaborative Medicinal Development Pty Limited
Facioscapulohumeral Muscular Dystrophy (FSHD)
A Study to Evaluate RO7204239 in Participants With Facioscapulohumeral Muscular Dystrophy
The purpose of this study is to evaluate the pharmacodynamics, safety, tolerability, pharmacokinetics, and efficacy of RO7204239, a humanized monoclonal antibody that binds to human latent myostatin, in ambulant adult…
- Trial name
- MANOEUVRE (NCT05548556)
- Testing
- RO7204239
- Where
- 9 sites in United States, Denmark and 2 more
- Volunteers
- 51 people
- Sponsor
- Hoffmann-La Roche
Generalized Myasthenia Gravis
An Extension Study of MOM-M281-004 to Evaluate the Safety, Tolerability, and Efficacy of M281 Administered to Patients With Generalized Myasthenia Gravis
The purpose of this study is to evaluate the long-term safety and tolerability of M281 in participants with generalized myasthenia gravis (gMG)
- Trial name
- NCT03896295
- Testing
- M281
- Where
- 54 sites in United States, Belgium and 6 more
- Volunteers
- 37 people
- Sponsor
- Momenta Pharmaceuticals, Inc.
Spasticity, Multiple Sclerosis
Efficacy Study of Arbaclofen to Treat Spasticity in Multiple Sclerosis
This is a multicenter, randomized (1:1:1), double-blind, active and placebo controlled, parallel group study to evaluate safety, tolerability and efficacy of oral arbaclofen in MS patients with spasticity.
- Trial name
- NCT01743651
- Testing
- arbaclofen, baclofen
- Where
- 48 sites in United States, Russia and 1 more
- Volunteers
- 353 people
- Sponsor
- RVL Pharmaceuticals, Inc.
PreDiabetes, Older Adult
Examining the Effects of Vivo on Physical Function and Glycemic Level in Sedentary Older With Prediabetes.
This is a 12-week randomized, controlled trial that seeks to examine the effect of Vivo on 1) change in lower extremity strength defined as number of chair stands done in 30 seconds and 2) change in average glycemic level (HbA1c)…
- Conditions
- PreDiabetes, Older Adult, Muscle Loss, Muscle Weakness
- Trial name
- NCT06458530
- Testing
- Vivo
- Where
- United States
- Volunteers
- 74 people, healthy volunteers welcome
- Sponsor
- Impactiv, Inc.
Hepatitis B, Diphtheria
Immunogenicity and Safety of DTPa-HBV-IPV/Hib Compared to DTPa-IPV/Hib and HBV Administered Concomitantly
This study will assess the immunogenicity of GlaxoSmithKline (GSK) Biologicals' (formerly SmithKline Beecham Biologicals') DTPa-HBV-IPV/Hib (Infanrix hexa™) vaccine compared to the separate administration of DTPa-HBV-IPV…
- Conditions
- Hepatitis B, Diphtheria, Haemophilus Influenzae Type b (Hib), Poliomyelitis
- Trial name
- NCT01457495
- Testing
- DTPa-HBV-IPV/Hib (Infanrix-hexa™), DTPa-IPV/Hib (Infanrix-IPV/Hib™), HBV (Engerix™-B)
- Volunteers
- 312 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
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