Clinical trials: Myotonic Dystrophy 1
HER2-positive Breast Cancer, Recurrent Breast Cancer
BYL719 + T-DM1 in HER2(+) Metastatic Breast Cancer Pts Who Progress on Prior Trastuzumab & Taxane Tx
The purpose of this study is to see whether a combination of two different drugs - trastuzumab-MCC-DM1 (T-DM1) and BYL719 is safe, and if it might be effective in treating metastatic breast cancer.
- Conditions
- HER2-positive Breast Cancer, Recurrent Breast Cancer, Stage IIIA Breast Cancer, Stage IIIB Breast Cancer
- Trial name
- NCT02038010
- Testing
- PI3K inhibitor BYL719, ado-trastuzumab emtansine, pharmacological study
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Northwestern University
Myotonic Dystrophy Type 1 (DM1)
A Registered Observational Cohort Study of Myotonic Dystrophy Type 1
Myotonic dystrophy type 1 (DM1) is the most common form of muscular dystrophy.There is little phenotype and genetic data for Chinese DM1 patients.
- Trial name
- NCT06979024
- Testing
- Triplet-primed PCR or Long-read sequencing
- Where
- China
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- First Affiliated Hospital of Fujian Medical University
Breast Neoplasm
A Study of Ado-trastuzumab Emtansine (T-DM1)-Induced Peripheral Neuropathy in HER2-positive Breast Cancer Patients
Researchers plan to study the natural history of ado-trastuzumab emtansine (T-DM1)-induced neuropathy both in patients without any history of neuropathy or previous neurotoxic agent use and in patients who have such a history.
- Trial name
- NCT05889988
- Where
- 21 sites in United States
- Volunteers
- 36 people
- Sponsor
- Mayo Clinic
DM1
Induction of Pluripotent Stem Cells From Human Fibroblasts of DM1 Patients
The objective of the study is the modelisation of human cardiomyocyts from pluripotent stem cells in order to study the cardiac alterations induced by CTG expansions.
- Trial name
- NCT02084407
- Testing
- Clinical examination, skin biopsy, blood and urine sampling
- Where
- France
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Institut de Myologie, France
Myotonic Dystrophy 1, Myotonic Dystrophy 2
Tracking the Brain in Myotonic Dystrophies: a 5-year Longitudinal Follow-up Study
The natural history of brain affection in myotonic dystrophy types 1 and 2 is still unknown. The investigators designed a 5-year longitudinal neuropsychological and neuroimaging follow-up study to address this issue.
- Trial name
- NCT02729597
- Testing
- medical history, neurological clinical examination, neuropsychological testing
- Volunteers
- 49 people, healthy volunteers welcome
- Sponsor
- University Hospital, Bonn
Locally Advanced Cancer, Metastatic Cancer
Pilot Study of [68Ga]Ga-ABY-025 Imaging in Patients Undergoing Treatment With HER2-targeted Therapy
The purpose of this study is to determine if the radiotracer, [68Ga]Ga-ABY-025, used for PET imaging can help us better identify and visualize lesions or tumors, in patients who are receiving standard of care therapy HER2+…
- Conditions
- Locally Advanced Cancer, Metastatic Cancer, HER2, HER2 + Breast Cancer, HER2 + Gastric Cancer, Squamous Cell Carcinoma of Head and Neck, Esophageal Squamous Cell Carcinoma
- Trial name
- NCT06828588
- Testing
- [68Ga]Ga-ABY-025
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Vanderbilt-Ingram Cancer Center
Gastric Cancer
A Study of Trastuzumab Emtansine Versus Taxane in Participants With Human Epidermal Growth Factor Receptor 2 (HER2)-Positive Advanced Gastric Cancer
This multicenter, randomized, adaptive Phase II/III study will evaluate the efficacy and safety of trastuzumab emtansine (T-DM1) compared to standard taxane (docetaxel or paclitaxel) treatment in participants with human epidermal…
- Trial name
- NCT01641939
- Testing
- Taxane, trastuzumab emtansine
- Where
- 148 sites in United States, Argentina and 26 more
- Volunteers
- 415 people
- Sponsor
- Hoffmann-La Roche
Spinal Muscular Atrophy (SMA), Charcot-Marie-Tooth
Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers
In recent years, knowledge of neuromuscular diseases has advanced considerably, and new therapeutic avenues are beginning to emerge.
- Conditions
- Spinal Muscular Atrophy (SMA), Charcot-Marie-Tooth, Muscular Dystrophy, Myotonic Dystrophy
- Trial name
- Myokinesis (NCT07321977)
- Testing
- Gait analysis via video capture
- Where
- France
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Institut de Myologie, France
Myotonic Dystrophy 2
Investigating Exercise in Myotonic Dystrophy Type 2 (DM2)
An exercise regimen (PRIME: Proximal Resistance In-House Movement Exercise) has been designed for patients with myotonic dystrophy type 2 (DM2).
- Trial name
- DM2 (NCT06716931)
- Testing
- Exercise
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Massachusetts General Hospital
LGMD1B, LGMD1C
Long-Term Development of Muscular Dystrophy Outcome Assessments
This is a 24-month, observational study of up to 1000 participants with Limb Girdle Muscular Dystrophy (LGMD), Myotonic Dystrophy Type 2 (DM2), and late onset Pompe disease (LOPD).
- Conditions
- LGMD1B, LGMD1C, LGMD1D, LGMD1E
- Trial name
- GRASP-01-005 (NCT05989620)
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- Virginia Commonwealth University
All Diagnosed Health Conditions, ADD/ADHD
At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions
We are the missing link in clinical trials, connecting patients and researchers seamlessly and conveniently using a mobile health platform to advance medical research.
- Conditions
- All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis
- Trial name
- NCT01931644
- Where
- United States
- Volunteers
- 17,667 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
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
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
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
Glycogen Storage Disease Type 2, Dystrophia Myotonica
An MRI Study on Muscular Diseases -Pompe Disease and Dystrophia Myotonica-
The aim of the project is to develop new Magnetic Resonance (MR) imaging techniques for better diagnosis and monitoring of patients with muscular disorders.
- Trial name
- NCT02708784
- Where
- 2 sites in Denmark
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University of Aarhus
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 Versus T-DM1 for Human Epidermal Growth Factor Receptor 2 (HER2)-Positive, Unresectable and/or Metastatic Breast Cancer Previously Treated With Trastuzumab and Taxane [DESTINY-Breast03]
This study is designed to compare the anti-tumor activity as well as the safety and efficacy of DS-8201a versus T-DM1 in HER2-positive, unresectable and/or metastatic breast cancer subjects previously treated with trastuzumab and…
- Trial name
- NCT03529110
- Testing
- Trastuzumab deruxtecan (T-DXd), Ado-trastuzumab emtansine (T-DM1)
- Where
- 164 sites in United States, Australia and 13 more
- Volunteers
- 524 people
- Sponsor
- Daiichi Sankyo
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
FS-1502 Versus T-DM1 for HER2-Positive Unresectable Locally Advanced or Metastatic Breast Cancer
This study is designed to compare the anti-tumor activity as well as the safety and efficacy of FS-1502 versus T-DM1 in HER2-positive, unresectable locally advanced or metastatic breast cancer subjects previously treated with…
- Trial name
- NCT05755048
- Testing
- Investigational drug: Recombinant Anti-HER2 Humanized Monoclonal Antibody - Monomethyl Auristatin F Conjugates for Injection (FS-1502), Control drug: Trastuzumab emtansine (Kadcyla, T-DM1)
- Where
- 56 sites in China
- Volunteers
- 314 people
- Sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
Type 1 Diabetes
SweMaMi DMI, Microbiome
Dysbiosis is associated with an increased risk of preeclampsia during pregnancy. The microbiome differs between those with and without type 1 diabetes and the cause of type 1 diabetes has been associated with dysbiosis.
- Trial name
- NCT04346524
- Testing
- sampling of microbiome
- Where
- 2 sites in Sweden
- Volunteers
- 100 people
- Sponsor
- Karolinska Institutet