Clinical trials: Steinert Disease
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
Myotonic Dystrophy Type 1 (DM1)
A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I
The purpose of the study is to evaluate the long-term safety and tolerability, efficacy and pharmacokinetics of VX-670 in participants with Myotonic Dystrophy Type I (DM1).
- Trial name
- NCT06926621
- Testing
- VX-670
- Where
- 16 sites in United States, Australia and 6 more
- Volunteers
- 44 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
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
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
Congenital Myotonic Dystrophy
Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy
This is an open-label phase 2/3 study for individuals with Congenital Myotonic Dystrophy (Congenital DM1) who participated in the preceding AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who…
- Trial name
- REACH CDM X (NCT05004129)
- Testing
- Tideglusib
- Where
- 14 sites in United States, Australia and 2 more
- Volunteers
- 76 people
- Sponsor
- AMO Pharma Limited
Myotonic Dystrophy Type 1
Multicenter Observational Study of Myotonic Dystrophy Type 1
The purpose of the study is to determine the best ways to assess how people are affected by myotonic dystrophy type 1 (DM1). The study will assess walking speed, muscle strength, muscle size, myotonia, heart rhythm, mental…
- Trial name
- MOS-DM1/POP (NCT02308657)
- Where
- 6 sites in United States
- Volunteers
- 120 people
- Sponsor
- University of Rochester
Myotonic Dystrophy Type 1
PhenoDM1 (Myotonic Dystrophy Type 1 Natural History Study)
PhenoDM1 will use patient reported outcomes to assess levels of pain, fatigue and quality of life in this cohort. Clinical and functional outcomes will look at muscle wasting and levels of myotonia.
- Trial name
- PhenoDM1 (NCT02831504)
- Where
- 2 sites in United Kingdom
- Volunteers
- 213 people
- Sponsor
- Newcastle-upon-Tyne Hospitals NHS Trust
Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne
Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks to ambulatory Duchenne muscular dystrophy (DMD) participants, 4 to 18…
- Conditions
- Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne, Muscular Dystrophy (DMD), DMD
- Trial name
- FORZETTO (NCT07608432)
- Testing
- Zeleciment Rostudirsen (DYNE-251)
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- Dyne Therapeutics
Myotonic Dystrophy 1
Venous Thromboembolism in Myotonic Dystrophy Type 1
Investigators identified a high risk of deep vein thrombosis and pulmonary embolism in patients presenting myotonic dystrophy type 1 treated in our hospital, 10 times higher than general population matched on age and sex.
- Trial name
- DM1-VTE (NCT03424460)
- Testing
- Haemostasis tests, Monocytes and megacaryocytes culture and RNA extraction, RNA extraction
- Where
- France
- Volunteers
- 130 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Myotonic Dystrophy 1
Effects of a 12-week Strength Training Program in Men With Myotonic Dystrophy Type 1
Eleven men with myotonic dystrophy type 1 (DM1) underwent a 12-week lower-limb strength training program. The training program consisted of 3 series of 6 to 8 maximal repetitions of 5 different exercises: Leg extension, leg…
- Trial name
- NCT04018820
- Testing
- Training program
- Where
- Canada
- Volunteers
- 11 people
- Sponsor
- Élise Duchesne
Dystrophia Myotonica Type 1, Myotonia Congenita
Lamotrigine as Treatment of Myotonia
Myotonia is a functional limiting symptom where the muscle stiffens on action leading to arrest of movement. Pharmacological treatment may make the difference between a physically restricted and a normal life.
- Conditions
- Dystrophia Myotonica Type 1, Myotonia Congenita, Paramyotonia Congenita, Hyperkalemic Periodic Paralysis
- Trial name
- NCT01939561
- Testing
- Lamotrigine
- Where
- Denmark
- Volunteers
- 27 people
- Sponsor
- Grete Andersen, MD
Diabetes Mellitus, Type 1, Metabolic Disorder
A Telematic Program for Optimization of Metabolic Control in Diabetes Mellitus Type 1 (DM1) Patients
A greater visit frequency between the diabetes mellitus 1 (DM1) patient and the medical team increases the possibilities to improve metabolic control. The support of telematic visits can support the patient and the health system.
- Trial name
- NCT01337141
- Testing
- Control, Telecare system
- Where
- 5 sites in Spain
- Volunteers
- 154 people
- Sponsor
- Spanish Biomedical Research Centre in Diabetes and Associated Metabolic Disorders
Myotonic Dystrophy 1, DM1
Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)
Building on previous work of the Myotonic Dystrophy Clinical Research Network (DMCRN), the present study seeks to overcome insufficient data on natural history; lack of reliable biomarkers; and incomplete characterization and…
- Trial name
- NCT03981575
- Where
- 17 sites in United States, Canada and 5 more
- Volunteers
- 700 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
Breast Cancer Metastatic
DZD1516 in Combination With Trastuzumab and Capecitabine, or in Combination With T-DM1, in Patients With Metastatic HER2 Positive Breast Cancer
DZD1516 is an oral, blood brain barrier penetrable, selective HER2 tyrosine kinase inhibitor. This study is designed to evaluate the safety and tolerability of DZD1516 in patients with metastatic HER2 positive breast cancer who…
- Trial name
- NCT04509596
- Testing
- DZD1516 mono therapy in Part A, DZD1516 in combination with trastuzumab and/or capecitabine in Part B, DZD1516 in combination with T-DM1 in Part C
- Where
- 3 sites in United States, China
- Volunteers
- 23 people
- Sponsor
- Dizal Pharmaceuticals
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
Myotonic Dystrophy 1, Steinert Disease
Ventilatory Response After Non Invasive Ventilation in Type 1 Myotonic Dystrophy
It has been suggested that patients with Myotonic Dystrophy type 1 have primary altered ventilatory response to chemical stimuli and chronic hypoventilation is related not always to muscle weakness.
- Trial name
- NCT02880735
- Testing
- Non Invasive Ventilation.
- Where
- Mexico
- Volunteers
- 27 people
- Sponsor
- National Institute of Respiratory Diseases, Mexico
Myotonic Dystrophy 1, Excessive Daytime Sleepiness
Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1
The primary objective of this study is to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness (EDS) in patients with Myotonic Dystrophy Type 1 ages 18 to 65 years.
- Trial name
- NCT04886518
- Testing
- Pitolisant Oral Tablet
- Where
- 13 sites in United States, Canada
- Volunteers
- 30 people
- Sponsor
- Harmony Biosciences Management, Inc.
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Cell-free fetal DNA (cffDNA) is present in the maternal blood from the early first trimester of gestation and makes up 5%-20% of the total circulating cell-free DNA (cfDNA) in maternal plasma.
- Conditions
- Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease, Cystic Fibrosis, Fragile X Syndrome
- Trial name
- DANNIgene (NCT06147414)
- Testing
- Blood sample
- Where
- France
- Volunteers
- 550 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
DM1, Muscular Dystrophies
Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
AOC 1001-CS2 (MARINA-OLE) (NCT05479981) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) (NCT05027269) study to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of multiple-doses of AOC 1001…
- Conditions
- DM1, Muscular Dystrophies, Myotonic Dystrophy, Myotonic Dystrophy 1
- Trial name
- MARINA-OLE (NCT05479981)
- Testing
- AOC 1001
- Where
- 8 sites in United States
- Volunteers
- 37 people
- Sponsor
- Avidity Biosciences, Inc.