Clinical trials: Myotonic Dystrophy 1
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
Steinert's Disease
Compare Train and 3D-4D Left Ventricular Systolic Function in Subjects Suffering From Dystrophy and Healthy Subjects
Steinert's disease is an orphan disease. The prognosis of patients with this disease is conditioned by cardiac involvement. Search an early stage alterations in contractile function in subjects suffering from dystrophy would…
- Trial name
- NCT02930408
- Testing
- electrocardiogram, Echocardiography
- Volunteers
- 70 people
- Sponsor
- Poitiers University Hospital
Breast Cancer
A Study of Trastuzumab Emtansine (T-DM1) in Combination With Docetaxel, and Potentially Pertuzumab, in Participants With Advanced Breast Cancer
This is an open-label, multi-center, non-randomized study of the safety and tolerability of the combination of T-DM1 plus docetaxel for the treatment of participants with metastatic breast cancer (MBC) and of T-DM1 plus docetaxel…
- Trial name
- NCT00934856
- Testing
- Docetaxel, Pertuzumab, Trastuzumab emtansine
- Where
- 8 sites in United States, France and 2 more
- Volunteers
- 98 people
- Sponsor
- Hoffmann-La Roche
Diabete Mellitus
CGM to Aid Transition From Inpatient to Outpatient
A pilot study using the FreeStyle Libre to assess its potential benefits in patients with diabetes starting when they are inpatients and continuing during the transition of care to the outpatient setting to see if diabetes care…
- Trial name
- NCT03155594
- Testing
- Freestyle Libre
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Methodist Medical Center of Illinois
Inclusion Body Myositis, Myotonic Dystrophy
Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders
Currently, there are no standardised fall risk scores or guidelines on when to use appropriate assistive gait devices (AGDs) for people with neuromuscular disorders (NMDs).
- Conditions
- Inclusion Body Myositis, Myotonic Dystrophy, Limb-girdle and Facioscapulohumeral Muscular Dystrophies, Pompe Disease
- Trial name
- FBIndex (NCT06605612)
- Testing
- FBIndex
- Where
- Germany
- Volunteers
- 108 people
- Sponsor
- LMU Klinikum
Prostatic Neoplasms
MLN2704 in Subjects With Metastatic Androgen-Independent Prostate Cancer
The purpose of the study is to determine the highest dose of MLN2704 that can be given multiple times safely to patients with prostate cancer, and to identify any side effects associated with taking the drug.
- Trial name
- NCT00070837
- Testing
- MLN2704 (DM1 conjugated monoclonal antibody MLN591)
- Where
- 4 sites in United States
- Volunteers
- 46 people
- Sponsor
- Millennium Pharmaceuticals, Inc.
Breast Cancer
A Study to Evaluate the Safety and Effectiveness of Trastuzumab Emtansine (T-DM1) as Therapy in Chinese Participants With HER2 Positive Advanced Breast Cancer
This study is a post-marketing, observational, multicenter, prospective study. It will investigate the the safety and effectiveness of T-DM1 in the Chinese population in real-world clinical practice.
- Trial name
- NCT05945927
- Testing
- Trastuzumab emtansine
- Where
- 30 sites in China
- Volunteers
- 178 people
- Sponsor
- Hoffmann-La Roche
HER2-positive Breast Cancer, Breast Cancer Metastatic
Palbociclib in Estrogen Receptor Positive (ER+) Human Epidermal Growth Factor Receptor 2 Positive (HER2+) Metastatic Breast Cancer
This study will determine the recommend dose of palbociclib in combination with letrozole and another medication, Ado-trastuzumab emtansine (T-DM1).
- Trial name
- NCT03709082
- Testing
- Palbociclib 75mg, Letrozole 2.5mg, T-DM1
- Where
- 5 sites in United States
- Volunteers
- 3 people
- Sponsor
- University of Kansas Medical Center
Metastatic Breast Cancer
Corrected QT Interval Effects of Trastuzumab Emtansine (T-DM1) in Patients With Human Epidermal Growth Factor Receptor 2 (HER2)-Positive Locally Advanced or Metastatic Breast Cancer and the Safety and Tolerability of Combined T-DM1 and Pertuzumab in Patients With Early Disease Progression
This is a multicenter, open-label, single-arm Phase II study designed to evaluate the effect of T-DM1 on the duration of corrected QT (QTc) interval in patients with HER2-positive locally advanced or metastatic breast cancer and…
- Trial name
- NCT00943670
- Testing
- pertuzumab, Trastuzumab emtansine [Kadcyla]
- Volunteers
- 51 people
- Sponsor
- Genentech, Inc.
Unrespectable Locally Advanced and or Metastatic HER2 Positive Breast Cancer Participants
JSKN003 Versus Trastuzumab Emtansine (T-DM1) for HER2-Positive, Advanced Breast Cancer
This study is designed to compare the safety and efficacy of JSKN003 versus T-DM1 in unrespectable locally advanced and/or metastatic HER2-positive breast cancer participants previously treated with trastuzumab and taxane.
- Trial name
- NCT06846437
- Testing
- JSKN003, Trastuzumab emtansine (T-DM1)
- Where
- China
- Volunteers
- 228 people
- Sponsor
- Shanghai JMT-Bio Inc.
Breast Cancer
A Study of Trastuzumab Emtansine Versus Capecitabine + Lapatinib in Participants With HER2-positive Locally Advanced or Metastatic Breast Cancer
This is a Phase III, randomized, multicenter, international, 2-arm, open-label clinical trial designed to compare the safety and efficacy of trastuzumab emtansine (T-DM1) with that of capecitabine + lapatinib in participants with…
- Trial name
- EMILIA (NCT00829166)
- Testing
- Trastuzumab emtansine, Lapatinib, Capecitabine
- Where
- 323 sites in United States, Bosnia and Herzegovina and 25 more
- Volunteers
- 991 people
- Sponsor
- Hoffmann-La Roche
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 Type 1, Urinary Incontinence
Pelvic Floor Muscle Training for Women with Myotonic Dystrophy
Myotonic dystrophy type 1 (DM1) is a neuromuscular disease characterized by multisystem manifestations. DM1 can affect the urinary system through the impact of the pelvic floor muscles (PFM).
- Trial name
- NCT06316778
- Testing
- Pelvic floor muscle training
- Where
- Canada
- Volunteers
- 12 people
- Sponsor
- Université de Sherbrooke
Myotonic Dystrophy
The United Kingdom National Registry for Myotonic Dystrophy
Myotonic dystrophy (dystrophia myotonica - DM) exists in two forms, usually referred to as DM1 (type 1) and DM2 (type 2). Both conditions are genetic disorders but each affects a different gene.
- Trial name
- NCT04003363
- Testing
- Patient Registry
- Where
- United Kingdom
- Volunteers
- 900 people
- Sponsor
- Newcastle University
Breast Cancer, HER2-positive Breast Cancer
ATEMPT 2.0: Adjuvant T-DM1 vs TH
This research study is studying how well newly diagnosed breast cancer that has tested positive for a protein called HER2 responds using one of two different combination of HER2-directed therapies as a treatment after surgery.
- Trial name
- NCT04893109
- Testing
- trastuzumab-emtansine, Trastuzumab SC, Paclitaxel
- Where
- 53 sites in United States
- Volunteers
- 500 people
- Sponsor
- Dana-Farber Cancer Institute
Myotonic Dystrophy Type 1 (DM1)
A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.
- Trial name
- Galileo (NCT06185764)
- Testing
- VX-670
- Where
- 26 sites in United States, Australia and 8 more
- Volunteers
- 47 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Metastatic HER2-positive Breast Cancer With Brain Metastasis
Kadcyla In pAtients With bRAin Metastasis
Women with breast cancer often develop metastases in the brain. Currently, treatment of these metastases is difficult and relies on radiotherapy or surgery which often fail.
- Trial name
- KIARA (NCT03203616)
- Testing
- KADCYLA 160 MG Injection
- Sponsor
- Jules Bordet Institute
Breast Cancer
An Open-Label, Phase Ib/II Clinical Trial Of Cdk 4/6 Inhibitor, Ribociclib (Lee011), In Combination With Trastuzumab Or T-Dm1 For Advanced/Metastatic Her2-Positive Breast Cancer.
Participants that have breast cancer that has spread to other parts of the body, is positive for a protein called HER2, and has not responded to standard treatment.
- Trial name
- NCT02657343
- Testing
- Ribociclib, T-DM1, Trastuzumab
- Where
- 2 sites in United States
- Volunteers
- 25 people
- Sponsor
- Dana-Farber Cancer Institute