Clinical trials: Muscle Disorders
Duchenne Muscular Dystrophy
HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02
This study, HALO-DMD-03, is a follow-on study to HALO-DMD-01 and HALO-DMD-02, and allows continued open-label access to HT-100 for subjects who have completed these studies.
- Trial name
- NCT02525302
- Testing
- HT-100
- Where
- 5 sites in United States
- Volunteers
- 10 people
- Sponsor
- Akashi Therapeutics
Fatigue Syndrome, Chronic, Myalgic Encephalomyelitis
Oral Melatonin Plus Zinc Supplementation in Chronic Fatigue Syndrome/Myalgic Encephalomyelitis (CFS/ME)
The purpose of this study is to evaluate the effects of oral melatonin plus zinc supplementation in relieving self-reported fatigue in CFS/ME
- Trial name
- MELATOZINC (NCT03000777)
- Testing
- melatonin plus zinc
- Where
- Spain
- Volunteers
- 60 people
- Sponsor
- Laboratorios Viñas, S.A.
Spinal Muscular Atrophy
An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)
This objectives of this study are to evaluate the safety, tolerability, and pharmacokinetics of a single dose of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA).
- Trial name
- SMNRx (NCT01494701)
- Testing
- nusinersen
- Where
- 4 sites in United States
- Volunteers
- 28 people
- Sponsor
- Biogen
Oculopharyngeal Muscular Dystrophy
Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients
The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.
- Trial name
- HOPEMD (NCT02015481)
- Testing
- Cabaletta
- Where
- 4 sites in United States, Canada and 1 more
- Volunteers
- 25 people
- Sponsor
- Bioblast Pharma Ltd.
Heart Diseases, ASD - Atrial Septal Defect
Abbott Structural Heart Device Registry
The Abbott Structural Heart (SH) Registry is being conducted to confirm the safety and performance of Abbott's SH devices in a post-market, real-world setting.
- Conditions
- Heart Diseases, ASD - Atrial Septal Defect, VSD - Muscular Ventricular Septal Defect, PFO - Patent Foramen Ovale
- Trial name
- SH-Registry (NCT06590467)
- Testing
- Amplatzer™ Occlusion Devices, Epic™ Surgical Tissue Heart Valve devices
- Where
- 36 sites in United States, Denmark and 8 more
- Volunteers
- 2,500 people
- Sponsor
- Abbott Medical Devices
Muscular Dystrophy, Duchenne
Safety Study of BLS-M22 in Healthy Volunteers
BLS-M22 is being developed as an anti-myostatin agent for the treatment of Duchenne Muscular Dystrophy (Muscular Dystrophy). A total of 37 subjects participated in this study to confirm the safety of BLS-M22.
- Trial name
- NCT03789734
- Testing
- BLS-M22
- Where
- South Korea
- Volunteers
- 37 people, healthy volunteers welcome
- Sponsor
- BioLeaders Corporation
Uterine Leiomyomas
Therapeutic MRI Guided High Intensity Focused Ultrasound Ablation of Uterine Fibroids
This study is to collect supplementary safety and technical effectiveness data of Philips MRI guided High Intensity Focused Ultrasound (HIFU) in ablating uterine tissue associated with symptomatic fibroids in a 3T MRI scanner.
- Trial name
- HIFU (NCT01141062)
- Testing
- Philips MR-guided HIFU
- Where
- 2 sites in Canada
- Volunteers
- 15 people
- Sponsor
- Philips Healthcare
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
MELAS Syndrome, Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS)
A Study to Assess TTI-0102 vs Placebo in MELAS Patients
This is a randomized, double-blind, placebo-controlled study. Prior to treatment, patients will undergo a screening visit. If eligible, each subject will return for a Day 1 visit and will receive their first dose of…
- Trial name
- NCT06644534
- Testing
- TTI-0102
- Where
- 2 sites in France, Netherlands
- Volunteers
- 9 people
- Sponsor
- Thiogenesis Therapeutics, Inc.
Uterine Fibroids (UF)
A Study to Evaluate Changes in Hair in Adult Participants Taking Oral Oriahnn Capsules With Heavy Menstrual Bleeding (HMB) Associated With Uterine Fibroids (UF)
Uterine Fibroids (UF) are benign smooth muscle neoplasms of the uterus that affect women of reproductive age. UFs are one of the leading causes of hospitalizations for gynecological disorders and often lead to hysterectomy.
- Trial name
- Pink Orchid (NCT06058728)
- Where
- United States
- Sponsor
- AbbVie
Fibrodysplasia Ossificans Progressiva
In-Home Evaluation of Episodic Administration of Palovarotene in Fibrodysplasia Ossificans Progressiva (FOP) Subjects
Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) that result in abnormal bone formation (heterotopic ossification…
- Trial name
- NCT02521792
- Testing
- Palovarotene
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Clementia Pharmaceuticals Inc.
Fibromyalgia
Investigation of AVACEN Thermal Exchange System for Fibromyalgia Pain
Hypothesis: Daily use of the AVACEN Thermal Exchange System for 4 weeks will show improved Fibromyalgia pain and functioning markers, as assessed by biomarkers and clinical and psychological assessment.
- Trial name
- AVACEN: TES (NCT01619579)
- Testing
- AVACEN Thermal Exchange System
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Avacen, Inc.
Aging, Healthy Aging
Biological Effects in Consumers of Resveratrol-enriched Wine
The objective of this clinical trial is to compare the health conditions of the participating population before and after an intervention with Resveratrol enriched wine. The main questions it aims to answer are: 1.
- Conditions
- Aging, Healthy Aging, Sarcopenia, Obesity
- Trial name
- NCT05981053
- Testing
- Wine enriched with Resveratrol
- Where
- Argentina
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- PINER Pastor Manfredi Winery
Congenital Myotonic Dystrophy
Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy
This is an open-label phase 2/3 study for individuals with Congenital Myotonic Dystrophy (Congenital DM1) who participated in the preceding AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who…
- Trial name
- REACH CDM X (NCT05004129)
- Testing
- Tideglusib
- Where
- 14 sites in United States, Australia and 2 more
- Volunteers
- 76 people
- Sponsor
- AMO Pharma Limited
Spinal Muscular Atrophy (SMA)
A Study of the Safety and Preliminary Efficacy of NKG001 in Pediatric Patients With Type 1 or Type 2 Spinal Muscular Atrophy
This study is a single-center, open-label, single-arm, non-randomized, single-dose, dose-escalation investigator-initiated trial (IIT) designed to evaluate the safety, tolerability, and preliminary efficacy of NKG001 Injection…
- Trial name
- NCT07617779
- Testing
- NKG001
- Where
- China
- Volunteers
- 21 people
- Sponsor
- Nikegen Pharmaceutical (Hangzhou) Company Limited
Stress Urinary Incontinence
Pelvic Floor Muscle Training With a Digital Therapeutic Device to Standard Exercises for Stress Urinary Incontinence
A virtual prospective randomized controlled study to evaluate the efficacy of using the leva® Pelvic Digital Health System (PDHS) to perform PFMT compared to a standard home exercise home program for the treatment of SUI/SMUI.
- Trial name
- NCT04508153
- Testing
- Leva Pelvic Digital Health System, Kegel exercises
- Where
- 3 sites in United States
- Volunteers
- 369 people
- Sponsor
- Renovia, Inc.
Duchenne Muscular Dystrophy (DMD)
An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy
This open-label extension study aims to evaluate the long-term safety and tolerability of weekly BMN 351 infusions, as well as to assess the effect of BMN 351 on physical function, in participants with DMD who participated in the…
- Trial name
- NCT07573631
- Testing
- BMN 351
- Where
- 6 sites in Italy, Netherlands and 3 more
- Volunteers
- 18 people
- Sponsor
- BioMarin Pharmaceutical
Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2
A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).
- Conditions
- Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2, LGMD2I, Muscular Dystrophy
- Trial name
- NCT05230459
- Testing
- AB-1003 dose level 1, AB-1003 dose level 2
- Where
- 6 sites in United States
- Volunteers
- 9 people
- Sponsor
- AskBio Inc
Cerebral Palsy
Individual Patient Expanded Access IND of Hope Biosciences Autologous Adipose-derived Mesenchymal Stem Cells for the Treatment of Cerebral Palsy
This is an Individual Patient Expanded Access Protocol of Autologous HB-adMSCs for the Treatment of Cerebral Palsy (CP) with the primary goal of treating 1 individual with CP who has exhausted all treatment options, his condition…
- Trial name
- NCT04029896
- Testing
- HB-adMSCs
- Where
- United States
- Sponsor
- Hope Biosciences Research Foundation
Muscle Spasticity, Cerebrovascular Accident
Asian Botulinum Clinical Trial Designed for Early Stroke Spasticity
The aim of this clinical study is to investigate the efficacy and safety of Dysport® in patients with early onset of upper limb spasticity within 2-12 weeks after stroke.
- Trial name
- NCT00234546
- Testing
- Botulinum toxin type A
- Where
- 5 sites in Hong Kong, Malaysia and 3 more
- Volunteers
- 162 people
- Sponsor
- Ipsen
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