Clinical trials: Steinert Disease
HER2-positive Breast Cancer, Metastatic Breast Cancer
Copanlisib in Combination With T-DM1 in Pretreated Unresectable Locally Advanced or Metastatic HER2-positive Breast Cancer
This study is a Phase 1b open label, single arm, adaptive multi-centre trial of copanlisib in combination with trastuzumab emtansine (T-DM1) in pretreated locally advanced or metastatic HER2-positive breast cancer.
- Conditions
- HER2-positive Breast Cancer, Metastatic Breast Cancer, Locally Advanced Breast Cancer, Unresectable Breast Cancer
- Trial name
- Panthera (NCT04042051)
- Testing
- Copanlisib, Trastuzumab emtansine
- Where
- 3 sites in Ireland, Spain
- Volunteers
- 2 people
- Sponsor
- Cancer Trials Ireland
Myotonic Dystrophy
Sleep Breathing Disorders, a Main Trigger for Cardiac ARythmias in Type I Myotonic Dystrophy ?
Up to one-third of patients with myotonic dystrophy type 1 die suddenly mainly from arrhythmias. Sleep apnea is prevalent in myotonic dystrophy (DM1) patients.
- Trial name
- STAR (NCT02375087)
- Where
- France
- Volunteers
- 73 people
- Sponsor
- University Hospital, Grenoble
Nemaline Myopathy Type 6, Myotonic Dystrophy Type 2
Muscle Relaxation in Myopathies With Positive Muscle Phenomena
The aim of this study is to quantify muscle relaxation properties of the finger flexor muscles in patients with different myopathies.
- Conditions
- Nemaline Myopathy Type 6, Myotonic Dystrophy Type 2, McArdle Disease
- Trial name
- NCT03211923
- Testing
- Transcranial magnetic stimulation (TMS)
- Volunteers
- 30 people
- Sponsor
- Radboud University Medical Center
Myotonic Dystrophy Type 1 (DM1)
A Remote Physical Activity Program in the Population Suffering from Type 1 Myotonic Dystrophy
The COVID-19 pandemic exacerbates health problems by reducing access to adapted and advanced physical rehabilitation for several people who need rehabilitation services, including the population with myotonic dystrophy type 1…
- Trial name
- NCT05072288
- Testing
- Remote activity program
- Where
- 2 sites in Canada
- Volunteers
- 40 people
- Sponsor
- Université du Québec à Chicoutimi
Salivary Gland Cancer, HER2 Gene Mutation
Post-op T-DM1 in HER-2+ Salivary Gland Carcinomas
This research is being done to see how safe and effective the use of the study drug Ado-trastuzumab (T) emtansine (DM1), T-DM1, and standard of care chemoradiation are when used together in treating HER2-positive salivary gland…
- Trial name
- NCT04620187
- Testing
- Ado-trastuzumab (T) emtansine (T-DM1), Standard of Care Radiotherapy, Standard of Care Chemotherapy
- Where
- 13 sites in United States
- Volunteers
- 37 people
- Sponsor
- Dana-Farber Cancer Institute
Myopathy
Efficacy and Tolerance of AVAPS Mode in Myotonic Dystrophy
Myotonic dystrophy type 1 is a myopathy with complex respiratory pattern and at risk to develop respiratory failure. Classical mode of ventilation are sometimes not tolerated or ineffective in this population.
- Trial name
- NCT01530841
- Testing
- Nocturnal ventilation
- Where
- France
- Volunteers
- 32 people
- Sponsor
- Centre d'Investigation Clinique et Technologique 805
Myotonic Dystrophy Type 1
Safety and Efficacy Study of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) In Myotonic Dystrophy Type 1
To investigate the effects of rhIGF-I/rhIGFBP-3 treatment for 24 weeks on endurance, ambulation, cognitive functioning, insulin resistance, lipid levels, muscle function and strength, pain, gastrointestinal functioning, and…
- Trial name
- NCT00577577
- Testing
- rhIGF-I/rhIGFBP-3
- Where
- 12 sites in United States
- Volunteers
- 69 people
- Sponsor
- Insmed Incorporated
Metastatic Breast Cancer
Liquid Biopsy: Intercepting Mutational Trajectories of HER2 (Human Epidermal Growth Factor Receptor 2) Breast Cancer (GIM21 Trial)
This is an open, interventional, non-pharmacological, prospective study. Patients will receive trastuzumab emtansine (T-DM1) at 3.6 mg/kg intravenously every 21 days, as per Summary of Product Characteristics (SmPC).
- Trial name
- GIM21 (NCT05735392)
- Testing
- Both blood and tissues collection for patients with metastatic breast cancer HER2+ pretreated with no more than one line of anti-HER2 therapy for advanced breast cancer.
- Where
- 9 sites in Italy
- Volunteers
- 48 people
- Sponsor
- Consorzio Oncotech
Myotonic Dystrophy
Postural Spirometry Changes in Ambulatory Myotonic Dystrophy Patients
Myotonic dystrophy Type 1 (MD1, Steinert's disease), an autosomal dominant multisystem disease, is of the most common muscular dystrophies in adults, with a European prevalence of 3-15/100 000.
- Trial name
- NCT01242007
- Testing
- Supine spirometry
- Where
- France
- Volunteers
- 58 people
- Sponsor
- University of Nancy
Cancer of Breast, Trastuzumab Emtansine
Trastuzumab Emtansine(T-DM1) After Trastuzumab Deruxtecan(T-DXd) in HER2-Positive Breast Cancer in Korea
Prospective observational study of trastuzumab emtansine immediately after trastuzumab deruxtecan in patients with advanced HER2-positive breast cancer in Korea
- Conditions
- Cancer of Breast, Trastuzumab Emtansine, Trastuzumab Deruxtecan
- Trial name
- NCT06928818
- Where
- South Korea
- Volunteers
- 100 people
- Sponsor
- Seoul National University Hospital
Inherited Cardiac Arrythmias, Long QT Syndrome (LQTS)
Derivation of Human Induced Pluripotent Stem (iPS) Cells to Heritable Cardiac Arrhythmias
Human induced pluripotent stem cells (hiPSCs) have driven a paradigm shift in the modeling of human disease; the ability to reprogram patient-specific cells holds the promise of an enhanced understanding of disease mechanisms and…
- Conditions
- Inherited Cardiac Arrythmias, Long QT Syndrome (LQTS), Brugada Syndrome (BrS), Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
- Trial name
- NCT02413450
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Johns Hopkins University
Prostate Cancer
A Trial of MLN2704 in Subjects With Metastatic Androgen Independent Prostate Cancer
This is the first study of MLN2704 administered to humans. The purpose of the study is to determine the highest dose of MLN2704 that can be given safely to patients with prostate cancer, and to identify any side effects…
- Trial name
- NCT00052000
- Testing
- MLN2704 (DM1 conjugated monoclonal antibody MLN591)
- Where
- 2 sites in United States
- Volunteers
- 29 people
- Sponsor
- Millennium Pharmaceuticals, Inc.
Breast Cancer
Tyrosine Kinase Inhibitors (TKIs) in Trastuzumab Emtansine (T-DM1) Resistant HER2-positive Metastatic Breast Cancer: A Real-world Study
This is a real world study to evaluate the efficacy and safety of tyrosine kinase inhibitors after T-DM1 progression.
- Trial name
- NCT05231863
- Testing
- TKIs
- Where
- China
- Volunteers
- 100 people
- Sponsor
- The First Affiliated Hospital with Nanjing Medical University
Breast Cancer
DS-8201a in Pre-treated HER2 Breast Cancer That Cannot be Surgically Removed or Has Spread [DESTINY-Breast02]
This study will compare DS 8201a to standard treatment. Participants must have HER2 breast cancer that has been treated before. Their cancer: cannot be removed by an operation has spread to other parts of the body
- Trial name
- NCT03523585
- Testing
- Trastuzumab deruxtecan, Capecitabine, Lapatinib
- Where
- 227 sites in United States, Australia and 13 more
- Volunteers
- 608 people
- Sponsor
- Daiichi Sankyo
Duchenne / Becker Muscular Dystrophy, Dystrophia Myotonica 1
Evaluation of the Role of miR-1 in the Pathogenesis and as a Biomarker in Muscular Dystrophies and Congenital Myopathies
The study aims to find out if a specific blood molecule called miR-1, can be used as a biomarker to track the health of patients with certain muscle diseases.
- Conditions
- Duchenne / Becker Muscular Dystrophy, Dystrophia Myotonica 1, Congenital Myopathies, Healthy Participants
- Trial name
- Dystro-miR1 (NCT07415837)
- Testing
- dosage of blood biomarker miR1
- Where
- France
- Volunteers
- 104 people, healthy volunteers welcome
- Sponsor
- University Hospital, Clermont-Ferrand
Myotonic Dystrophy Type 2
Clinical Outcome Measures in Myotonic Dystrophy Type 2
A monocentric, longitudinal, observational case-control study in patients with Myotonic Dystrophy type 2 (DM2). At least 60 DM2 will be evaluated through a battery of patients reported Outcomes (PROs) and clinical Outcome…
- Trial name
- COMEDY-2 (NCT03603171)
- Testing
- DM1-ActivC, R-PAct, Beck depression inventory
- Where
- Germany
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Prof. Dr. Benedikt Schoser
Myotonia Congenita, Paramyotonia Congenita
Study of Ranolazine in Myotonia Congenita, Paramyotonia Congenita and Myotonic Dystrophy Type 1
The purpose of this study is to gather preliminary data to determine if ranolazine is a safe and effective treatment for the symptoms of myotonia congenital, paramyotonia congenita, and myotonic dystrophy type 1.
- Conditions
- Myotonia Congenita, Paramyotonia Congenita, Myotonic Dystrophy 1
- Trial name
- NCT02251457
- Testing
- Ranolazine
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Ohio State University
Myotonic Dystrophy
Efficacy and Safety of DHEA for Myotonic Dystrophy
To test the efficacy and safety of two doses of dehydroepiandrosterone (DHEA) in adults with myotonic dystrophy
- Trial name
- NCT00167609
- Testing
- dehydroepiandrosterone 100 and 400 mg
- Where
- 10 sites in France
- Volunteers
- 75 people
- Sponsor
- University of Versailles
Myotonic Dystrophy 1
Myotonic Dystrophy Type 1 Congenital and Juvenile Form: From Diagnosis to Rehabilitation [MDCJ-NeuBeRe]
The rationale of the study is to collect structured data in the neuropsychological, clinical neuroradiologic and neurorehabilitation fields in children/young people affected by congenital and juvenile myotonic dystrophy.
- Trial name
- MDCJ-NeuBeRe (NCT06378216)
- Testing
- neurocognitive evaluations
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- IRCCS Eugenio Medea
Myotonic Dystrophy 1
Home-based Training and Supplementation in DM1 Patients
Myotonic dystrophy type 1 (DM1) is a rare genetic disease that affects about 1 in 2100 people. Patients diagnosed with DM1 present with many symptoms, however, their muscles are mainly affected.
- Trial name
- DM1HBET (NCT05848830)
- Testing
- Mult-ingredient supplement, Concurrent exercise training
- Where
- Canada
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- McMaster University