Clinical trials: Neuromuscular Diseases
Painful Diabetic Neuropathy
Safety, Tolerability & Pharmacokinetics Study of CNTX-6016 in Healthy Subjects and Subjects With PDN
A Phase 1b study to evaluate the safety, tolerability and pharmacokinetics of multiple ascending doses of CNTX-6016 in healthy subjects and a single cohort to evaluate painful diabetic neuropathy.
- Trial name
- NCT04857957
- Where
- United States
- Volunteers
- 46 people, healthy volunteers welcome
- Sponsor
- Centrexion Therapeutics
Low Muscle Strength, Malnutrition
Efficacy of Oral Nutritional Supplement in Community-Dwelling Adults Following Acute Care
The study aims to determine the effects of Oral Nutritional Supplement (ONS) added to usual care on body weight compared with usual care alone in community-dwelling adults aged 55 years and older who are at risk of malnutrition…
- Trial name
- NCT07753239
- Testing
- ONS, Standard of Care (SOC)
- Where
- 7 sites in Thailand
- Volunteers
- 304 people
- Sponsor
- Abbott Nutrition
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
ALS, Amyotrophic Lateral Sclerosis
A Phase 1 Study to Investigate the Safety and Pharmacokinetics of Fosigotifator in Patients With Amyotrophic Lateral Sclerosis
Fosigotifator is an investigational drug being researched for the treatment of Amyotrophic Lateral Sclerosis. This is an up to 156-week, 2-part study.
- Trial name
- NCT04948645
- Testing
- Fosigotifator
- Where
- 14 sites in United States, Canada
- Volunteers
- 31 people
- Sponsor
- AbbVie
Amyotrophic Lateral Sclerosis
Creation of a Bank of Fibroblast From Patients With Amyotrophic Lateral Sclerosis: Pilot Study
Patients with ALS will be included in the reference center for ALS in hospital La Pitié Salpêtrière, Paris. The study proposes to investigate the pathophysiology of ALS by setting up a fibroblast bank for studying molecular…
- Trial name
- ALSCELL (NCT01639391)
- Testing
- skin biopsy
- Where
- France
- Volunteers
- 19 people
- Sponsor
- Institut Pasteur
Amyotrophic Lateral Sclerosis (ALS)
PHENOGENE-1A (Cromolyn) Treatment in Patients With Mild to Moderate ALS
The purpose of this study is to test the effects of PHENOGENE-1A, which is the treatment under investigation in this study. This research will investigate if PHENOGENE-1A can help people with ALS by measuring their function using…
- Trial name
- NCT07142291
- Testing
- Cromolyn Sodium (34.2 mg BID), Cromolyn Sodium (17.1 mg BID), Riluzole (100 mg)
- Where
- 22 sites in United States, Czechia and 4 more
- Volunteers
- 105 people
- Sponsor
- PhenoNet, Inc.
Fibromyalgia, Painful Diabetic Peripheral Neuropathy
Examination of Pregabalin Access for Treatment of Indicated Pain Disorders: the ExPAND Study
The investigators hypothesize that fibromyalgia (FM) and painful diabetic peripheral neuropathy (pDPN) patients with access restrictions on pregabalin will lead to higher healthcare resource use and cost compared to patients…
- Trial name
- NCT01280747
- Testing
- prior authorization, no prior authorization
- Where
- 17 sites in United States
- Volunteers
- 42 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
ALS, ALS (Amyotrophic Lateral Sclerosis)
Systems Biology of Amyotrophic Lateral Sclerosis (ALS)
The goal of this observational study is to identify molecular features in multiple clinical samples and/or the patient environment that are associated with ALS.
- Trial name
- NCT07007390
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Viome
Fibromyalgia
Micronized and Ultramicronized Palmitoylethanolamide in Fibromyalgia Patients
The onset of chronic Fibromyalgia symptomatology is due to central alterations, together with peripheral neuroimmune modifications.
- Trial name
- NCT04488926
- Testing
- micronized and ultra-micronized Palmitoylethanolamide (mPEA and umPEA, 300mg + 600mg) microgranules, Standard Therapy, Rescue Drug
- Where
- Italy
- Volunteers
- 21 people
- Sponsor
- Epitech Group SpA
Hereditary Transthyretin Amyloidosis (ATTRv), Polyneuropathy
Retrospective Study Collecting Neurological Follow-up of Hereditary Transthyretin Amyloidosis (ATTRv) Patients Included in B3461028 and B3461045.
A study of patients with hereditary transthyretin amyloidosis (ATTRv) and wild-type transthyretin amyloidosis (ATTRwt) that have been enrolled in B3461028 and B3461045 studies in Spain - exposed to tafamidis 61mg for ≥12 months…
- Trial name
- TRAMA (NCT05560555)
- Testing
- Tafamidis
- Where
- 3 sites in Spain
- Volunteers
- 5 people
- Sponsor
- Pfizer
BRAF or NRAS Mutant Metastatic Melanoma
A Phase II Study of Single Agent MEK162 in Patients With Advanced Melanoma
The study will assess the safety and efficacy of single-agent MEK162 in adult patients with locally advanced and unresectable or metastatic malignant cutaneous melanoma, harboring BRAFV600E or NRAS mutations.
- Trial name
- NCT01320085
- Testing
- MEK162
- Where
- 25 sites in United States, Germany and 3 more
- Volunteers
- 183 people
- Sponsor
- Pfizer
Duchenne Muscular Dystrophy (DMD)
A Study of PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
The study consists of 3 periods: A Screening Period (up to 45 days), a double-blind, placebo-controlled Multiple Ascending Dose (MAD) Period (28 weeks), and a Long-Term Extension (LTE) Period (108 weeks).
- Trial name
- CONNECT2-EDO51 (NCT06833931)
- Testing
- IV infusion
- Sponsor
- PepGen Inc
Radiating Pain, Myelopathy
Interbody Fusion Devices in the Treatment of Cervicobrachial Syndrome
This study randomizes neck and arm pain patients being treated with discectomy and anterior interbody fusion into two groups: one to receive a new ceramic implant and a control group with a more traditional plastic implant.
- Conditions
- Radiating Pain, Myelopathy, Cervicobrachial Syndrome
- Trial name
- CASCADE (NCT01511445)
- Testing
- Anterior cervical discectomy and fusion (ACDF) with PEEK Cage, Anterior cervical discectomy and fusion (ACDF) with a Valeo CSC Cage
- Where
- Netherlands
- Volunteers
- 100 people
- Sponsor
- Amedica Corporation
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II
Therapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in Spain
An observational, retrospective, cross-sectional, multicenter study. Real-world data were obtained from medical records of Spanish public hospitals (9 hospitals).
- Trial name
- NCT06632730
- Where
- United States
- Volunteers
- 69 people
- Sponsor
- Novartis
Dermatomyositis, Polymyositis
Study of M5049 in DM and PM Participants (NEPTUNIA)
The purpose of this study is to evaluate the efficacy and safety of orally administered M5049 in idiopathic inflammatory myopathies, specifically dermatomyositis (DM) and polymyositis (PM) participants for 24 weeks.
- Trial name
- NCT05650567
- Testing
- M5049 dose
- Where
- 31 sites in United States, Czechia and 5 more
- Volunteers
- 20 people
- Sponsor
- EMD Serono Research & Development Institute, Inc.
Hepatitis B, Poliomyelitis
Post-marketing Safety Study of GSK Biological's Pediarix™ Vaccine
Pre-licensure studies of GSK Biologicals' PEDIARIX vaccine have shown it to be generally safe and unlikely to result in vaccine-associated serious adverse events.
- Conditions
- Hepatitis B, Poliomyelitis, Diphtheria, Tetanus
- Trial name
- NCT00146835
- Testing
- Pediarix, Licensed DTPa containing vaccine, Prevnar®: (Wyeth)
- Volunteers
- 120,794 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Malnutrition, Calorie, Sarcopenia
Malnutrition Screening Study
This is a non-interventional, cross-sectional, non-randomized, observational study to screen for the risk of malnutrition and sarcopenia in outpatients aged 18 and over who attend hospital clinics for any reason in Turkey.
- Trial name
- NCT07256925
- Where
- 69 sites in Turkey (Türkiye)
- Volunteers
- 7,500 people
- Sponsor
- Abbott Nutrition
Spasticity
A Study to Assess the Safety and Efficacy of IPN10200 in Adult Participants With Upper Limb Spasticity.
The purpose of the study is to assess the safety and efficacy of increasing doses of Corabotase (also known as IPN10200) with the aim to evaluate the Pharmacodynamics (PD) profile of Corabotase and to establish the total…
- Trial name
- LANTIMA (NCT04752774)
- Testing
- Corabotase, Dysport
- Where
- 81 sites in United States, Austria and 12 more
- Volunteers
- 240 people
- Sponsor
- Ipsen
Transthyretin (TTR) Amyloid Cardiomyopathy
Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy
This Phase 3 study will investigate the efficacy, safety and tolerability of an oral daily dose of 20 mg or 80 mg tafamidis meglumine capsules compared to placebo in subjects with either transthyretin genetic variants or…
- Trial name
- ATTR-ACT (NCT01994889)
- Testing
- Tafamidis
- Where
- 80 sites in United States, Belgium and 11 more
- Volunteers
- 441 people
- Sponsor
- Pfizer
Fibromyalgia
An Extension Study of Duloxetine in Fibromyalgia (Extension of F1J-JE-HMGZ, NCT01552057)
The purpose of the study is to assess the safety and efficacy of duloxetine in participants with fibromyalgia at long-term use.
- Trial name
- NCT01621191
- Testing
- Duloxetine
- Where
- Japan
- Volunteers
- 149 people
- Sponsor
- Eli Lilly and Company
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