Clinical trials: Myopathies
Renal Impairment, Hemodialysis
Study to Evaluate the Pharmacokinetics, Safety, and Pharmacodynamics of INCB000928 in Participants With Impaired Renal Function and Hemodialysis
This is a multicenter, open-label parallel-group to evaluate single oral doses of INCB000928 in participants with varying level of renal function or impairment.
- Trial name
- NCT05099445
- Testing
- INCB000928
- Where
- 5 sites in United States
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Incyte Corporation
Duchenne Muscular Dystrophy
First in Human SAD/MAD Safety and PK Study With Adult DMD Safety and PK Cohort
This is a first-in-human (FIH), Phase 1 study of orally administered SAT-3247 in healthy adult volunteers (HVs) and adult participants with DMD to determine safety, tolerability, pharmacokinetics and pharmacodynamics.
- Trial name
- NCT06565208
- Testing
- SAT-3247
- Where
- 2 sites in Australia
- Volunteers
- 77 people, healthy volunteers welcome
- Sponsor
- Satellos Bioscience, Inc.
Spasticity
Phase II Study to Assess Safety, Pharmacokinetics and Efficacy of SL-1002 for Limb Spasticity
This is a randomized, double-blind, placebo-controlled single ascending dose escalation study intended to assess the safety, pharmacokinetics and efficacy of single treatment of SL-1002 in patients with mild to severe limb…
- Trial name
- RAISE (NCT05311215)
- Testing
- SL-1002
- Where
- 2 sites in United States
- Volunteers
- 32 people
- Sponsor
- Saol Therapeutics Inc
Primary Mitochondrial Disease
An Observational Study of Patients With Primary Mitochondrial Disease (SPIMM-300)
This is an observational study of patients with Primary Mitochondrial Disease with either signs or symptoms suggestive of myopathy.
- Trial name
- RePOWER (NCT03048617)
- Where
- 33 sites in United States, Australia and 7 more
- Volunteers
- 215 people
- Sponsor
- Stealth BioTherapeutics Inc.
Oculopharyngeal Muscular Dystrophy
A Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of BB-301 Administered to Subjects With Oculopharyngeal Muscular Dystrophy With Dysphagia
Subjects who have enrolled in the oculopharyngeal muscular dystrophy (OPMD) natural history study (Study BNTC-OPMD-NH-001) and have completed at least 6 months of follow up in Study BNTC-OPMD-NH-001 may be eligible to participate…
- Trial name
- NCT06185673
- Testing
- BB-301: Dose escalation phase 1b cohort 1, BB-301: Dose escalation phase 1b cohort 2, BB-301: Dose escalation phase 1b cohort 3
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Benitec Biopharma, Inc.
Hereditary Inclusion Body Myopathy, Distal Myopathy With Rimmed Vacuoles
Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
The objective of this study is to evaluate the long-term safety and efficacy of Ace-ER treatment in subjects with GNEM.
- Conditions
- Hereditary Inclusion Body Myopathy, Distal Myopathy With Rimmed Vacuoles, Distal Myopathy, Nonaka Type, GNE Myopathy
- Trial name
- NCT02736188
- Testing
- Aceneuramic Acid Extended-Release Tablets
- Where
- 14 sites in United States, Bulgaria and 5 more
- Volunteers
- 143 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Long Chain Fatty Acid Oxidation Disorders
Expanded Access to Triheptanoin
Expanded access may be provided for qualified patients who have limited treatment options and are not eligible for a clinical trial.
- Trial name
- NCT03773770
- Testing
- Triheptanoin
- Sponsor
- Ultragenyx Pharmaceutical Inc
GNE Myopathy
First-in-human Study of UX016 in GNEM
The goal of this study is to assess the safety and dose of UX016 and its impact on muscle strength in adults with GNE Myopathy (GNEM).
- Trial name
- NCT07511556
- Testing
- UX016
- Where
- 2 sites in United States
- Volunteers
- 24 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Fibromyalgia
Growth Hormone in Neuroendocrine Dysfunction With Severe Fibromyalgia Syndrome
The purpose of this study is to evaluate the efficacy of recombinant human growth hormone (r-hGH) treatment in severe fibromyalgia subjects with growth axis dysfunction.
- Trial name
- NCT00933686
- Testing
- Saizen®
- Where
- Spain
- Volunteers
- 113 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Idiopathic Inflammatory Myopathies
Phase 2 Study of Rapcabtagene Autoleucel in Myositis
A Phase 2, randomized, open-label, controlled study to evaluate the efficacy and safety of rapcabtagene autoleucel versus comparator in participants with severe refractory idiopathic inflammatory myopathies (IIM)
- Trial name
- NCT06665256
- Testing
- Rapcabtagene autoleucel, Active Comparator Option
- Where
- 68 sites in United States, Australia and 13 more
- Volunteers
- 21 people
- Sponsor
- Novartis Pharmaceuticals
Healthy Volunteer, Muscular Dystrophies
Absorption, Metabolism, Excretion and Absolute Bioavailability of EDG-5506 in Healthy Volunteers
This is a Phase 1 2-part, single-center, open-label study in healthy male volunteers. Part A will assess the absorption, metabolism, excretion, and pharmacokinetics of one oral dose of radiolabeled EDG-5506.
- Conditions
- Healthy Volunteer, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- NCT05730842
- Testing
- EDG-5506 Tablet, Radiolabeled EDG-5506 Suspension, Radiolabeled EDG-5506 Intravenous
- Where
- United States
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- Edgewise Therapeutics, Inc.
Fibromyalgia
The Role of Ribose in Patients Diagnosed With Fibromyalgia
The purose of this research study is to determine the potential benefit of D-ribose, a nutritional supplement (a sugar), versus a placebo (another sugar) in people with fibromyalgia.
- Trial name
- NCT00566514
- Testing
- D-ribose, dextrose
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Bioenergy Life Science, Inc.
Fibrodysplasia Ossificans Progressiva
A Study of Andecaliximab in Participants With Fibrodysplasia Ossificans Progressiva (FOP)
This study is researching an experimental drug called andecaliximab. The study will include pediatric and adult patients with fibrodysplasia ossificans progressiva (FOP).
- Trial name
- ANDECAL (NCT06508021)
- Testing
- Andecaliximab
- Where
- 3 sites in United States
- Volunteers
- 92 people
- Sponsor
- Ashibio Inc
Fibrodysplasia Ossificans Progressiva
A Study to Document and to Further Describe Long-term Safety and Effectiveness of Palovarotene in Participants With Fibrodysplasia Ossificans Progressiva (FOP)
The participants in this registry study will have fibrodysplasia ossificans progressiva (FOP). FOP is an ultra-rare, severely disabling disease characterized by new bone formation in areas of the body where bone is not normally…
- Trial name
- FOPal (NCT06089616)
- Where
- 5 sites in United States, Canada
- Volunteers
- 100 people
- Sponsor
- Ipsen
ANCA-Associated Vasculitis (AAV), Idiopathic Inflammatory Myositis (IIM)
FT839 in Autoimmune Diseases
The primary objectives of this trial are to evaluate the safety and tolerability and to determine the maximum tolerated dose (MTD) or recommended Phase 2 dose of FT839 with or without rituximab and/or background therapy and/or…
- Conditions
- ANCA-Associated Vasculitis (AAV), Idiopathic Inflammatory Myositis (IIM), Rheumatoid Arthritis (RA), Systemic Lupus Erythematosus (SLE)
- Trial name
- NCT07800871
- Testing
- FT839
- Where
- United States
- Volunteers
- 446 people
- Sponsor
- Fate Therapeutics
Sporadic Inclusion Body Myositis
Efficacy and Safety of Bimagrumab/BYM338 at 52 Weeks on Physical Function, Muscle Strength, Mobility in sIBM Patients
This study evaluated the efficacy, safety and tolerability of multiple doses of bimagrumab/BYM338 vs placebo, when administered intravenously (i.v.), on physical function, muscle strength, and mobility in patients with sporadic…
- Trial name
- RESILIENT (NCT01925209)
- Testing
- BYM338/bimagrumab
- Where
- 38 sites in United States, Australia and 8 more
- Volunteers
- 251 people
- Sponsor
- Novartis Pharmaceuticals
Muscle Damage, Muscle Strength
HIFEM and Radiofrequency for Muscular System Function Improvement
The goal of this clinical trial is to investigate the effect of the BTL-899M device on muscular system function in adult subjects seeking treatment for improving their muscular system function in the lower extremities.
- Conditions
- Muscle Damage, Muscle Strength, Muscle Weakness
- Trial name
- NCT06703749
- Testing
- Treatment with BTL-899M, Treatment with BTL-899M with intensities below therapeutic threshold
- Where
- 3 sites in United States
- Volunteers
- 33 people, healthy volunteers welcome
- Sponsor
- BTL Industries Ltd.
Intermediate AMD
A Clinical Trial Evaluating the Safety and Efficacy of a New Light Combination Therapy Addressing Intermediate AMD
The proposed clinical investigation wants primary to validate the safety of the innovative light therapy approach and in second priority provide insight and confirmations on therapeutic effect.
- Trial name
- retinaSEES-PoC (NCT06557369)
- Testing
- reSEES
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- Oculox Technologies SA
Blepharospasm, Hemifacial Spasm
Post Marketing Surveillance Study of Dysport
The purpose of this study is to provide further information regarding the risks and benefits of Dysport in marketed indications.
- Conditions
- Blepharospasm, Hemifacial Spasm, Cervical Dystonia, Spasmodic Torticollis
- Trial name
- NCT00210431
- Where
- 36 sites in France, Germany and 1 more
- Volunteers
- 783 people
- Sponsor
- Ipsen
Fibromyalgia
A Long-Term Study To Evaluate Safety And Efficacy Of Pregabalin In Patients With Fibromyalgia
This study will assess the safety and efficacy of the long-term use of pregabalin at doses up to 450 mg/day in patients with fibromyalgia who have completed 16 weeks of dosing in Study A0081208 (NCT00830167).
- Trial name
- NCT00830128
- Testing
- pregabalin (Lyrica)
- Where
- 19 sites in Japan
- Volunteers
- 106 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.