Clinical trials: Myotonic Dystrophy 1
Myotonic Dystrophy 1
Effects of a 12-week Strength Training Program in Women With Myotonic Dystrophy Type 1
20 women with myotonic dystrophy type 1 (DM1) will complete a 12-week lower-limb strength training program. The training program consist of 3 series of 6 to 8 maximal repetitions of 5 different exercises: Leg extension, leg…
- Trial name
- NCT05400629
- Testing
- Training program
- Where
- Canada
- Volunteers
- 12 people
- Sponsor
- Université de Sherbrooke
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
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.
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
Metastatic Breast Cancer
A Study of Trastuzumab Emtansine, Paclitaxel, and Pertuzumab in Patients With HER2-Positive, Locally Advanced or Metastatic Breast Cancer
This Phase Ib-IIa, multi-institutional, open-label, dose-escalation study is designed to evaluate the safety, tolerability, pharmacokinetics and feasibility of trastuzumab emtansine (T-DM1) administered by intravenous (IV)…
- Trial name
- NCT00951665
- Testing
- paclitaxel, pertuzumab [Perjeta], trastuzumab emtansine [Kadcyla]
- Where
- 5 sites in United States
- Volunteers
- 107 people
- Sponsor
- Genentech, Inc.
Metastatic Breast Cancer
A Study of Trastuzumab-Mcc-DM1 Administered Intravenously to Patients With HER2-Positive Metastatic Breast Cancer
Study of trastuzumab emtansine (T-DM1) administered to patients with human epidermal growth factor receptor 2 (HER2)-positive metastatic breast cancer.
- Trial name
- NCT00679211
- Testing
- Trastuzumab emtansine [Kadcyla]
- Volunteers
- 110 people
- Sponsor
- Genentech, Inc.
Neuromuscular Diseases, Obesity (Disorder)
Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology
The ActiLiège-Adult study is a prospective, longitudinal, observational study designed to collect natural history data on adult patients with neurological or metabolic diseases affecting movement.
- Conditions
- Neuromuscular Diseases, Obesity (Disorder), Myotonic Dystrophy 1, Myasthenic Syndrome
- Trial name
- Acti-Adult (NCT07136844)
- Testing
- Syde, Dynamometric measurements of muscle strength, The 6-minute walk test (6MWT)
- Where
- Belgium
- Volunteers
- 300 people
- Sponsor
- Centre Hospitalier Universitaire de Liege
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
Myotonic Dystrophy Type 2, Myotonic Dystrophy Type 1
Brain Structure and Clinical Endpoints in Myotonic Dystrophy Type 2
Nearly two-third of patients with myotonic dystrophy type 2 (DM2) report that impaired cognition is among the most disabling symptoms and deeply affects their quality of life.
- Trial name
- BraCE-DM2 (NCT05854433)
- Testing
- Non-interventional study
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Wake Forest University Health Sciences
Myotonic Dystrophy
The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2
A Randomized, Double-blind, Placebo-Controlled, Multi-Center Study to Investigate the Efficacy and Safety of Once Daily Mexiletine PR During 26 Weeks of Treatment in Patients with Myotonic Dystrophy Type 1 and Type 2 (HERCULES…
- Trial name
- NCT06523400
- Testing
- Mexiletine granules for prolonged-release oral suspension
- Where
- 7 sites in Belgium, Denmark and 4 more
- Volunteers
- 176 people
- Sponsor
- Lupin Ltd.
Advanced Breast Cancer
Unraveling KAdcyla Resistance In Human Epidermal Growth Factor Receptor 2(HER2) Positive Advanced Breast Cancer (KATIA)
The study aim is to perform a comprehensive and integrated characterization of mechanisms of primary and acquired resistance to Kadcyla in a prospective cohort of progressive/recurrent HER2-positive breast cancer patients.
- Trial name
- KATIA (NCT03829306)
- Where
- 10 sites in Spain
- Volunteers
- 32 people
- Sponsor
- Spanish Breast Cancer Research Group
Myotonic Dystrophy 1, Diabetes
Myotonic Dystrophy - Vascular and Cognition
The cognitive disorders of adult forms of myotonic dystrophies type 1 are heterogeneous (impairment of executive functions, visio construction and theory of the mind, which can progress to the stage of dementia).
- Conditions
- Myotonic Dystrophy 1, Diabetes, Carbohydrate Intolerance
- Trial name
- DM-VASCOG (NCT04656210)
- Testing
- MRI, neuropsychological tests
- Where
- France
- Volunteers
- 85 people
- Sponsor
- University Hospital, Lille
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
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
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
Myotonic Dystrophy 1
Low-frequency Repetitive Nerve Stimulation in Myotonic Dystrophy Type 1
The study design is a prospective cohort study. It aims to evaluate the neuromuscular junction in dystrophic myotonia 1 (DM 1) using low-frequency repetitive nerve stimulation (RNS) on several nerve-muscle pairs of the one side…
- Trial name
- NCT05662150
- Testing
- Low frequency repetitive stimulation
- Where
- Belgium
- Volunteers
- 9 people
- Sponsor
- Vrije Universiteit Brussel
HER2-positive Breast Cancer
Patients With Brain Metastases From HER2-positive Breast Cancer
The purpose of this study is to determine the optimal sequences of combined trastuzumab emtansine (T-DM1) and whole-brain radiotherapy in patients presenting brain metastases from HER2-positive breast cancer in terms of acute…
- Trial name
- BIRTH (NCT02135159)
- Testing
- TDM1, Brain Sequential RT
- Where
- France
- Volunteers
- 36 people
- Sponsor
- Institut du Cancer de Montpellier - Val d'Aurelle
Myotonic Dystrophy 1
Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy
This is a phase 1/2a double-blinded, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of single and multiple ascending doses of SRP-1003 compared to…
- Trial name
- NCT06138743
- Testing
- SRP-1003 IV Infusion, SRP-1003 SC Injection
- Where
- 35 sites in Australia, Belgium and 9 more
- Volunteers
- 78 people
- Sponsor
- Sarepta Therapeutics, Inc.
Early Breast Cancer
Chemotherapy-Free pCR-Guided Strategy With Trastuzumab-pertuzumab and T-DM1 in HER2-positive Early Breast Cancer
This is a multicenter, open-label, single-arm, one-stage, phase II study to assess the efficacy of a chemotherapy-free pathological complete response (pCR)-guided strategy with trastuzumab and pertuzumab (given as a subcutaneous…
- Trial name
- PHERGAIN-2 (NCT04733118)
- Testing
- Trastuzumab and Pertuzumab (FDC SC) and T-DM1
- Where
- 41 sites in Bulgaria, Germany and 3 more
- Volunteers
- 393 people
- Sponsor
- MedSIR