Clinical Trials on Pharmacogenic Myopathy

Total 412 results

Centre Hospitalier Universitaire Vaudois

Not yet recruiting

Pharmacogenetic Testing at Community Pharmacy (OffiGene)

Pharmacogenetic Testing
University of Florida
National Center for Advancing Translational Sciences (NCATS)

Completed

Pilot of Preemptive Pharmacogenetics in Medically Underserved Patients

Pharmacogenetic Testing

United States
Newcastle-upon-Tyne Hospitals NHS Trust

Recruiting

Myotubular and Centronuclear Myopathy Patient Registry

X-linked Myotubular Myopathy | Centronuclear Myopathy | Myotubular Myopathy | Myotubular Myopathy 1 | Myotubular (Centronuclear) Myopathy | Centronuclear Myopathy, X-Linked

United Kingdom
Instituto de Investigación Hospital Universitario...
Instituto de Salud Carlos III

Not yet recruiting

Efficacy, Safety and Cost-efficacy of a Pre-emptive Genotyping Strategy in Patients Receiving Statins (PREVESTATGx)

Cardiovascular Diseases | Dyslipidemias | Statin Adverse Reaction | Pharmacogenic Myopathy

Spain
Clinic for Special Children

Recruiting

WiTNNess - TNNT1 Myopathy Natural History Study (WiTNNess)

Myopathy | Myopathy; Hereditary | Myopathies, Nemaline | TNNT1-associated Myopathy | Infantile-onset Nemaline Rod Myopathy | Myopathy, Rod | Amish Nemaline Myopathy | Nemaline Myopathy 5 | NEM5 | Genetic Muscle Disease | Recessive Hereditary Disorder (Autosomal) | ANM

United States
Awenydd GmbH

Unknown

Medication Optimisation for Reducing Events in a Private Practice Setting (MORE)

Pharmacogenetic Analysis to Reduce Events.

Germany
Vastra Gotaland Region

Enrolling by invitation

Changes of Motor Function Tests in Congenital Myopathy Subjects Treated With Oral Salbutamol as Compared to no Treatment (COMPIS)

Musculoskeletal Diseases | Neuromuscular Diseases | Centronuclear Myopathy | Nemaline Myopathy | Congenital Myopathy | Myosin Storage Myopathy

Sweden
Data Collection Analysis Business Management
PAS Research Services

Unknown

Pharmacogenomics Results Affect Patients' Plan of Care and Changes in Medication Prescription(s)

Pharmacogenomic

United States
Ultragenyx Pharmaceutical Inc

Terminated

A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER; UX001) Tablets in Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy [HIBM]) Patients With Severe Ambulatory Impairment

Hereditary Inclusion Body Myopathy | GNE Myopathy | Inclusion Body Myopathy 2 | Distal Myopathy With Rimmed Vacuoles | Distal Myopathy, Nonaka Type | Quadriceps Sparing Myopathy

Canada, United States, Bulgaria
James Dowling
Canadian Institutes of Health Research (CIHR); Cures Within Reach; The Joshua... and other collaborators

Recruiting

Tamoxifen Therapy for Myotubular Myopathy (TAM4MTM)

X Linked Myotubular Myopathy

United Kingdom, Canada, United States
Radboud University Medical Center
UMC Utrecht

Not yet recruiting

The Natural History and Muscle Fatigability of Patients With Congenital Myopathies. (READYCOM)

Centronuclear Myopathy | Nemaline Myopathy | Central Core Disease | Multi-Minicore Disease

Netherlands
Seattle Institute for Biomedical and Clinical Research
Genomind, LLC

Completed

Medication Optimization Using Pharmacogenetic Testing and the G-DIG to Reduce Polypharmacy in a Mental Health Population (MedOPT)

Pharmacogenetic Testing

United States
Ain Shams University

Completed

Effect of Glucuronosyltransferase (UGT) Genetic Variation on Pharmacokinetics of Empagliflozin

Pharmacokinetics | Pharmacogenomic

Egypt
Duke University
National Institute of General Medical Sciences (NIGMS)

Completed

Pharmacogenetic Testing in Primary Care

Pharmacogenetic Testing

United States
Ultragenyx Pharmaceutical Inc

Terminated

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)

Hereditary Inclusion Body Myopathy | GNE Myopathy | Distal Myopathy With Rimmed Vacuoles | Distal Myopathy, Nonaka Type | Quadriceps Sparing Myopathy

United States, Canada, United Kingdom, Israel, Italy, Bulgaria, France
Astellas Gene Therapies

Completed

A Clinical Assessment Study in X-Linked Myotubular Myopathy (XLMTM) Subjects (INCEPTUS)

X-linked Myotubular Myopathy

United States, France, United Kingdom, Canada, Germany
University of Florida
Audentes Therapeutics

Completed

Respiratory Muscle Function in Untreated X-Linked Myotubular Myopathy (XLMTM)

X-linked Myotubular Myopathy

United States
Rigshospitalet, Denmark

Active, not recruiting

Contractile Cross Sectional Areas and Muscle Strength in Patients With Congenital Myopathies

Congenital Myopathy | Inherited Muscle Diseases | RYR1-myopathy

Denmark
Ultragenyx Pharmaceutical Inc

Completed

Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) (GNEM)

Hereditary Inclusion Body Myopathy | GNE Myopathy | Distal Myopathy With Rimmed Vacuoles | Distal Myopathy, Nonaka Type

United States, Canada, United Kingdom, Israel, Italy, Bulgaria, France
Assiut University

Not yet recruiting

Magnetic Resonance Imaging and Ultrasonography in Evaluation of Muscle Diseases

Myositis | Congenital Myopathy | Acquired Myopathy
Boston Children's Hospital
Muscular Dystrophy Association

Recruiting

Molecular and Genetic Studies of Congenital Myopathies

Centronuclear Myopathy | Myotubular Myopathy | Nemaline Myopathy | Central Core Disease | Congenital Fiber Type Disproportion | Multiminicore Disease | Rigid Spine Muscular Dystrophy | Undefined Congenital Myopathy

United States
Geisinger Clinic

Enrolling by invitation

Preemptive Pharmacogenomics Testing Among Geriatric Patients

Polypharmacy | Pharmacogenomic Testing

United States
Astellas Gene Therapies

Completed

A Medical Chart Review of Patients With X-Linked Myotubular Myopathy (XLMTM)

Males With X-linked Myotubular Myopathy (XLMTM)

United States, United Kingdom
Cure CMD
University of Michigan; Congenital Muscle Disease International Registr

Completed

Myotubular Myopathy Event Study (MTMES)

X-linked Myotubular Myopathy

United States
Nobelpharma

Completed

Efficacy Confirmation Study of NPC-09

GNE Myopathy | Nonaka Disease | Distal Myopathy With Rimmed Vacuoles (DMRV) | Hereditary Inclusion Body Myopathy (hIBM)

Japan
Lawson Health Research Institute

Not yet recruiting

Personalized Pharmacotherapy Using Pharmacogenetics in Veterans (PGx)

Pharmacogenetic Testing to Determine Pharmacological Treatment in PTSD
Ra Pharmaceuticals

Terminated

Safety and Efficacy Study of Zilucoplan in Subjects With Immune-Mediated Necrotizing Myopathy

Immune Mediated Necrotizing Myopathy

United States, Netherlands, France, United Kingdom
Ultragenyx Pharmaceutical Inc
Newcastle University

Completed

GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)

Hereditary Inclusion Body Myopathy | GNE Myopathy | Nonaka Disease | Quadriceps Sparing Myopathy (QSM) | Distal Myopathy With Rimmed Vacuoles (DMRV)

United States, Bulgaria, Canada, France, United Kingdom
Yale University
Eunice Kennedy Shriver National Institute of Child Health and Human Development...

Not yet recruiting

Oral Contraceptive Pill (OCP) Pharmacogenomics

Contraception | Pharmacogenomic Drug Interaction

United States
Texas Tech University Health Sciences Center

Recruiting

Annatto-derived GG for Statin-associated Myopathy (GG-statin)

Myopathy; Primary

United States
Astellas Gene Therapies

Active, not recruiting

Gene Transfer Clinical Study in X-Linked Myotubular Myopathy (ASPIRO)

X-Linked Myotubular Myopathy

United States, Canada, France, Germany
Reneo Pharma Ltd

Terminated

An Open Label, Long Term Safety Study of REN001 in Primary Mitochondrial Myopathy Patients (Stride Ahead)

Primary Mitochondrial Myopathy

Spain, Australia, Belgium, Denmark, Canada, United Kingdom, France, Germany, Hungary, Italy, Netherlands, New Zealand
Lawson Health Research Institute

Completed

Evaluation of Patients With Statin Myopathy Using an N of 1 Trial Design

Statin Myopathy

Canada
The Emmes Company, LLC
United Mitochondrial Disease Foundation; PolG Foundation Mito Australia

Recruiting

Mitochondrial Video Assessment Source Material Study

PolG, Primary Mitochondrial Myopathy

United States
ClinLogic LLC

Recruiting

Reduction of Adverse Drug Events and Readmissions (RADAR-PGX)

Pharmacogenomic Testing for Medication Management

United States
Reneo Pharma Ltd

Completed

An Efficacy and Safety Study of 24 Week Treatment With Mavodelpar (REN001) in Primary Mitochondrial Myopathy Patients (STRIDE)

Primary Mitochondrial Myopathy

United States, Spain, Australia, France, United Kingdom, New Zealand, Denmark, Norway, Czechia, Belgium, Canada, Germany, Hungary, Italy, Netherlands
Stealth BioTherapeutics Inc.

Terminated

A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension (MMPOWER-3)

Primary Mitochondrial Myopathy

United States, Germany, Canada, Italy, United Kingdom, Denmark, Hungary
Reneo Pharma Ltd

Terminated

A Study of the Safety of REN001 in Patients With Primary Mitochondrial Myopathy

Primary Mitochondrial Myopathy

United Kingdom
Armgo Pharma, Inc.
National Institute of Nursing Research (NINR); National Institute of Neurological... and other collaborators

Completed

S 48168 (ARM 210) for the Treatment of RYR1-related Myopathies (RYR1-RM)

RYR-1 Myopathy

United States
Rockefeller University

Terminated

Metabolic Features of Post-Myopathy Patients Associated With Statin Treatment

Myopathy (Statin Associated)

United States
Oregon Health and Science University

Completed

Neurosciences-Intensive Care Unit Electrical Stimulation

Intensive Care (ICU) Myopathy

United States
University of Melbourne
Melbourne Health; National University Hospital, Singapore; Flinders Medical Centre and other collaborators

Completed

Physical Function in Critical Care (PaciFIC) (PaciFIC)

Intensive Care (ICU) Myopathy

Singapore, Australia, Brazil
University of Florida
National Human Genome Research Institute (NHGRI)

Recruiting

Trial of Preemptive Pharmacogenetics in Underserved Patients (ToPP-UP)

Pharmacogenetic Testing | Use of Prescription Medications

United States
Vanderbilt University Medical Center

Recruiting

VICE-MPRINT: Maternal and Pediatric Pharmacogenetics Survey (MPRINT)

Pharmacogenomic Testing

United States
Rigshospitalet, Denmark

Completed

Aerobic Training in Patients With Congenital Myopathies

Congenital Myopathy

Denmark
Ruth Salim

Unknown

MR in Patients With Collagen VI Related Myopathies

Bethlem Myopathy | Ullrich Disease

Denmark
Universidade Estadual do Oeste do Paraná

Completed

Correlation Between Spirometry and Functional Independence in Adult Post-ICU Patients

Intensive Care (ICU) Myopathy | Functional Independence
Hartford Hospital

Unknown

CoEnzyme Q10 in Statin Myopathy

Statin Myopathy

United States
Sándor Beniczky
University of Aarhus; Danish Council for Independent Research; Søster og Verner...

Completed

Understanding the Mechanisms of Critical Illness Myopathy by Use of a Novel Electrophysiological Method - MVRCs

Critical Illness Myopathy | Myopathy Critical Illness

Denmark
Ultragenyx Pharmaceutical Inc

Completed

An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy

GNE Myopathy | Hereditary Inclusion Body Myopathy (HIBM)

United States, Israel

Clinical Trials on Pharmacogenic Myopathy

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CROs in Dominican Republic

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Dietary Supplements

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