Clinical trials: Muscular Diseases
Urinary Bladder Neoplasms
A Study of Stimulator of Interferon Genes (STING) Agonist E7766 in Non-muscle Invasive Bladder Cancer (NMIBC) Including Participants Unresponsive to Bacillus Calmette-Guerin (BCG) Therapy, INPUT-102
This is an open label, multicenter, phase 1/1b study to assess safety/tolerability and preliminary clinical activity of E7766 as a single agent administered intravesically in participants with NMIBC.
- Trial name
- NCT04109092
- Testing
- E7766
- Where
- 9 sites in United States
- Sponsor
- Eisai Inc.
Spinal Muscular Atrophy Type I
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
GB221 is a gene therapy that delivers a working SMN1 gene to the motor neurons of people with spinal muscular atrophy (SMA) Type 1. This study will evaluate the safety, tolerability and efficacy of GB221 in two groups: 1.
- Trial name
- NCT07070999
- Testing
- GB221
- Where
- Brazil
- Volunteers
- 22 people
- Sponsor
- Gemma Biotherapeutics
Duchenne Muscular Dystrophy
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)
RGX-202 is a gene therapy designed to deliver a transgene for a novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain.
- Trial name
- NCT05693142
- Testing
- RGX-202
- Where
- 23 sites in United States, Canada
- Volunteers
- 65 people
- Sponsor
- REGENXBIO Inc.
Appendicitis Acute, Appendicitis
Safety & Feasibility of PERIsign System in Demonstrating Involuntary Muscle Defense as a Sign of Peritonitis in Subjects With Appendicitis
This will be an explorative, proof-of-principle, open, multi-center investigation. The investigation aims to investigate the safety of the PERIsign system and the ability of physicians to differentiate between healthy individuals…
- Conditions
- Appendicitis Acute, Appendicitis, Abdominal Pain, Peritonitis
- Trial name
- PeriSaFe01 (NCT06296953)
- Testing
- PERIsign examination
- Where
- 2 sites in Sweden
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- STB INN AB
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.
Post-Stroke Spasticity, Cerebrovascular Accident
Study Of GSK1358820 In Patients With Post-Stroke Upper Limb Spasticity
This is a study to confirm the superior efficacy of a single treatment of GSK1358820 over placebo in patients with post-stroke upper limb spasticity of both the wrist and finger flexors using the Modified Ashworth Scale (MAS)…
- Trial name
- NCT00460564
- Testing
- GSK1358820
- Where
- 18 sites in Japan
- Volunteers
- 109 people
- Sponsor
- GlaxoSmithKline
FSHD - Facioscapulohumeral Muscular Dystrophy
Safety and Preliminary Efficacy of ULSC in Facioscapulohumeral Muscular Dystrophy (FSHD)
The goal of this clinical trial is to learn about how an umbilical cord lining-derived stem cell product (ULSC) performs when treating Facioscapulohumeral Muscular Dystrophy (FSHD) 1 or 2.
- Trial name
- NCT07086521
- Testing
- ULSC
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Restem, LLC.
Duchenne Muscular Dystrophy
Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting
The primary objective of this study is to evaluate acute liver injury (ALI) rates associated with ELEVIDYS with the addition of sirolimus as an adjunct prophylactic immunosuppression agent.
- Trial name
- ENHANCE (NCT07542314)
- Testing
- ELEVIDYS, Sirolimus, Glucocorticoids
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Sarepta Therapeutics, Inc.
Bladder Cancer
A Study of IAP0971 in Combination With Bacillus Calmette Guerin in High Risk Non-muscular Invasive Bladder Cancer
The efficacy and safety of IAP0971 single drug or combined with BCG intravesical instillation in the treatment of high-risk non muscle invasive bladder cancer with BCG treatment failure.
- Trial name
- NCT06255964
- Testing
- IAP0971, IAP0971＋BCG
- Volunteers
- 236 people
- Sponsor
- SUNHO（China）BioPharmaceutical CO., Ltd.
Muscular Atrophy, Spinal
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene…
- Trial name
- HINALEA 2 (NCT05861999)
- Testing
- Risdiplam
- Where
- 17 sites in United States, Germany and 4 more
- Volunteers
- 19 people
- Sponsor
- Hoffmann-La Roche
Fibromyalgia
Treatment of Fibromyalgia Using Deep Shaped-Field Transcranial Magnetic Stimulation a Clinical Feasibility Study
Deep shaped-field transcranial magnetic stimulation is a new type of transcranial magnetic stimulation technology which may provide a drug-free method for treating fibromyalgia.
- Trial name
- NCT01229852
- Testing
- DSF-rTMS
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Cervel Neurotech, Inc.
Stress Urinary Incontinence, Mixed Urinary Incontinence
Comparing Use of a Digital Health System of Pelvic Floor Exercise Program to Kegel Exercises in Stress Urinary Incontinence
A randomized controlled trial for patients with stress urinary incontinence or stress-dominant mixed incontinence. Subjects will be randomized to 8-weeks of home kegel exercises or 8 weeks of pelvic floor muscle exercises using…
- Trial name
- NCT03632447
- Testing
- Leva, PFDx
- Where
- 7 sites in United States
- Volunteers
- 77 people
- Sponsor
- Renovia, Inc.
Non-muscle Invasive Bladder Cancer
A Study of Sasanlimab in People With Non-muscle Invasive Bladder Cancer
The purpose of this study is to learn about the safety and effects of the study medicine (sasanlimab) in people with non-muscle invasive bladder cancer.
- Trial name
- CREST (NCT04165317)
- Testing
- PF-06801591, Bacillus Calmette-Guerin
- Where
- 196 sites in United States, Australia and 12 more
- Volunteers
- 1,068 people
- Sponsor
- Pfizer
Tennis Elbow
ALGRX 4975 in the Treatment of Tennis Elbow
Tennis elbow is a painful condition that results from repetitive arm movement with overuse of muscles, e.g. when playing tennis. A single injection of ALGRX 4975 has the potential to reduce pain for a period of weeks to months.
- Trial name
- NCT00130949
- Testing
- ALGRX 4975
- Where
- Slovakia
- Volunteers
- 45 people
- Sponsor
- AlgoRx Pharmaceuticals
FSHD, FSHD1
Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Participants with…
- Conditions
- FSHD, FSHD1, FSHD2, FMD
- Trial name
- FORTITUDE (NCT05747924)
- Testing
- AOC 1020
- Where
- 17 sites in United States, Canada and 1 more
- Volunteers
- 90 people
- Sponsor
- Avidity Biosciences, Inc.
Duchenne Muscular Dystrophy
Proof of Concept Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With Duchenne Muscular Dystrophy (DMD)
To Assess the Activity and Safety of SMT C1100 (Ezutromid) in Paediatric Male Participants with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT02858362
- Testing
- Ezutromid
- Where
- 16 sites in United States, United Kingdom
- Volunteers
- 43 people
- Sponsor
- Summit Therapeutics
Spinal Muscular Atrophy
Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2
To evaluate the safety and efficacy of intravenous onasemnogene abeparvovec-xioi in pre-symptomatic patients with SMA and 2 or 3 copies SMN2
- Trial name
- SPR1NT (NCT03505099)
- Testing
- onasemnogene abeparvovec-xioi
- Where
- 16 sites in United States, Australia and 4 more
- Volunteers
- 30 people
- Sponsor
- Novartis Gene Therapies
Fibromyalgia
Tool for Fibromyalgia Diagnosis and Effect of Extra Virgin Olive Oil
PRONACERA THERAPEUTICS S.L. is a young biotechnological company focused on the development of genetic diagnostic tools and treatments for pathologies in different medical areas such as reproduction, fibromyalgia and rare diseases…
- Trial name
- FIBROKIT (NCT05921409)
- Testing
- Extra Virgin Olive oil-supplemented Mediterranean Diet Intervention, Olive oil-supplemented Mediterranean Diet Intervention
- Where
- Spain
- Volunteers
- 250 people, healthy volunteers welcome
- Sponsor
- Pronacera Therapeutics SL
Airway Obstruction, Asthma
Effects of Combined Respiratory Muscle Training and Steam Inhalation
The purpose of this investigation is to obtain more information on the efficacy and safety of respiratory training methods with WellO2 in patients with asthma and COPD.
- Conditions
- Airway Obstruction, Asthma, COPD
- Trial name
- NCT04584398
- Testing
- Respiratory muscle training with steam inhalation
- Where
- Finland
- Volunteers
- 60 people
- Sponsor
- WellO2 Oy
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring Program
The objectives of this study are to characterize the disease impact, the disease management and resource utilization, and provide benefits to the LC-FAOD community, by use of a convenient online platform for participants (or…
- Trial name
- NCT04812106
- Testing
- No Intervention
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
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