Clinical trials: Steinert Disease
Duchenne Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy
Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next Study
The objective of the ActiLiège Next study is to collect longitudinal data from patients and control subjects using a wearable magneto-inertial device.
- Conditions
- Duchenne Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy 1, Charcot-Marie-Tooth
- Trial name
- NCT05982119
- Testing
- ActiMyo/Syde
- Where
- 8 sites in Belgium, Czechia and 5 more
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Myotonic Dystrophy 1, DM1
The Spanish National Registry for Myotonic Dystrophy Type 1
Myotonic Dystrophy Type 1 (DM1) is a rare genetic neuromuscular condition that can affect multiple organs and varies widely in how it presents.
- Conditions
- Myotonic Dystrophy 1, DM1, Myotonic Dystrophy Type 1, Myotonic Dystrophy, Congenital
- Trial name
- DM1-Hub (NCT07385443)
- Testing
- Patient Registry
- Where
- 8 sites in Spain
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Fundació Institut Germans Trias i Pujol
Non Invasive Prenatal Diagnosis
NIPD on CFTC for Triplet Repeat Diseases
The purpose of this study is to develop and validate an analytical and clinical NIPD test for triplet repeat diseases by isolated circulating fetal trophoblastic cells (CFTC) analysis from maternal blood, searching for the…
- Trial name
- DIACCIMEX (NCT03087526)
- Testing
- Non invasive prenatal diagnosis
- Where
- 8 sites in France
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University Hospital, Montpellier
Inclusion Body Myositis, Myotonic Dystrophy 1
The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.
The planned project is an intervention study to assess the risk of falling after adaptation of an assistive gait devices in patients with the following neuromuscular diseases: Inclusion body myositis, myotonic dystrophy, limb…
- Conditions
- Inclusion Body Myositis, Myotonic Dystrophy 1, Myotonic Dystrophy 2, Facio-Scapulo-Humeral Dystrophy
- Trial name
- Risk of fall (NCT07072676)
- Testing
- Assistive gait devices combined with physiotherapy
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- LMU Klinikum
Metastatic Breast Cancer
A Study of Trastuzumab Emtansine (Trastuzumab-MCC-DM1, T-DM1) in Combination With Pertuzumab Administered to Patients With Human Epidermal Growth Factor Receptor-2 (HER2)-Positive Locally Advanced or Metastatic Breast Cancer Who Have Previously Received Trastuzumab
This was a multi-institutional, multinational, open-label, single-arm Phase Ib/II study designed to evaluate the safety, tolerability, pharmacokinetics, and efficacy of trastuzumab emtansine (trastuzumab-MCC-DM1) administered by…
- Trial name
- NCT00875979
- Testing
- Trastuzumab emtansine [Kadcyla] 3.0 mg/kg, Trastuzumab emtansine [Kadcyla] 3.6 mg/kg, Pertuzumab 420 mg
- Where
- 20 sites in United States, Belgium and 5 more
- Volunteers
- 67 people
- Sponsor
- Hoffmann-La Roche
Myotonic Dystrophy Type 1, Congenital DM1
Remote Assessments and Genetic Determinants of Congenital and Childhood Myotonic Dystrophy
Myotonic dystrophy type 1 (DM1) can affect people in many different ways, even in the same family. The symptoms that children experience can be different and more severe than adults.
- Conditions
- Myotonic Dystrophy Type 1, Congenital DM1, Juvenile DM1, Childhood DM1
- Trial name
- REACH-DM KIDS (NCT07630389)
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- University of Rochester
Myotonic Dystrophy Type 1 (DM1), DM1
Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment basivarsen (DYNE-101) for the treatment of myotonic dystrophy 1 (DM1).
- Conditions
- Myotonic Dystrophy Type 1 (DM1), DM1, Myotonic Dystrophy, Steinert Disease
- Trial name
- NCT07486934
- Testing
- zeleciment basivarsen (DYNE-101)
- Where
- 43 sites in United States, Australia and 9 more
- Volunteers
- 150 people
- Sponsor
- Dyne Therapeutics
Pulmonary Disease, Chronic Obstructive, Amyotrophic Lateral Sclerosis
European Home Mechanical Ventilation Registry
The European Home Mechanical Ventilation Registry (EHMVR) will enable a thorough evaluation of HMV by documenting the characteristics of HMV patients and their treatment.
- Conditions
- Pulmonary Disease, Chronic Obstructive, Amyotrophic Lateral Sclerosis, Spinal Cord Injury, Muscular Dystrophies
- Trial name
- EHMVR (NCT02315339)
- Where
- Germany
- Volunteers
- 37 people
- Sponsor
- ResMed
Inclusion Body Myositis, Myotonic Dystrophy
The Risk of Falls Index for Patients With Neuromuscular Disorders
The combination of short quantitatively assessing muscular function and balance in combination with short clinical scores, can be a new valid approach to evaluate the patient risk of fall and help to create a quick checkup test…
- Conditions
- Inclusion Body Myositis, Myotonic Dystrophy, Facioscapulohumeral Muscular Dystrophies, Myasthenia Gravis
- Trial name
- NCT05890833
- Testing
- The risk of falls
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- LMU Klinikum
Breast Cancer
A Dose-Escalation Study Evaluating the Combination of Trastuzumab Emtansine (T-DM1) With Neratinib in Women With Metastatic HER2-Positive Breast Cancer
This study is being done for the following reasons: The study has two parts. The purpose of the first part (Phase I) of the study is to find out the highest dose of neratinib that can be given safely with T-DM1.
- Trial name
- NCT02236000
- Testing
- Neratinib, T-DM1
- Where
- 15 sites in United States
- Volunteers
- 49 people
- Sponsor
- NSABP Foundation Inc
Breast Cancer
A Study Of Pembrolizumab In Combination With Trastuzumab-DM1
This research study is studying a combination of drugs as a possible treatment for metastatic breast cancer. The interventions involved in this study are: Pembrolizumab Trastuzumab emtansine (also called T-DM1)
- Trial name
- NCT03032107
- Testing
- T-DM1, Pembrolizumab
- Where
- 2 sites in United States
- Volunteers
- 20 people
- Sponsor
- Dana-Farber Cancer Institute
HER2-positive Breast Cancer
Primary Prevention of Thrombocytopenia Associated With T-DM1 Therapy in HER2 Positive Breast Cancer With Herombopag
Ado-trastuzumab emtansine (T-DM1) demonstrates favorable efficacy in breast cancer treatment but is frequently associated with thrombocytopenia.
- Trial name
- NCT07198672
- Testing
- Herombopag
- Volunteers
- 45 people
- Sponsor
- Zhenzhen Liu
HER2-positive Breast Cancer, Brain Metastases
A Study of HER2+ Breast Cancer Patients With Active Brain Metastases Treated With Afatinib & T-DM1 vs. T-DM1 Alone
This study is being done for the following reasons: The study has two parts. The purpose of the first part (Phase I) of the study is to find out the highest dose of Afatinib that can be given safely with T-DM1.
- Trial name
- HER2BAT (NCT04158947)
- Testing
- Afatinib, T-DM1
- Where
- 4 sites in China
- Volunteers
- 130 people
- Sponsor
- xuexin he
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
Myotonic Dystrophy 1
Effect of MYODM on Quality of Life, Fatigue and Hypersomnia in Patients With Myotonic Dystrophy Type 1
The purpose of this study is to determine whether MYODM (formulated composition containing Theobroma cacao supplemented with caffeine (caffeine/theobromine ratio1/1.85, w/w) is effective in the treatment of excessive daytime…
- Trial name
- NCT04634682
- Testing
- MYODM
- Where
- Spain
- Volunteers
- 30 people
- Sponsor
- Myogem Health Company, S.L.
Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy)
A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping
A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping
- Conditions
- Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Muscular Disorders, Atrophic, Muscular Disease
- Trial name
- SAFARI44 (NCT07587242)
- Testing
- AOC 1044
- Where
- 18 sites in Australia, Belgium and 6 more
- Volunteers
- 70 people
- Sponsor
- Avidity Biosciences, Inc.
Breast Cancer
T-DM1 in HER2-positive Metastatic/Relapsed Breast Cancer
This is a retrospective, multicenter, non-interventional study for the evaluation of real-world efficacy and safety of T-DM1 in metastatic/relapsed HER2-positive breast cancer as part of the establishment of hospital medical…
- Trial name
- NCT04202328
- Where
- 60 sites in South Korea
- Volunteers
- 1,000 people
- Sponsor
- Samsung Medical Center
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
Myotonic Dystrophy 1
Calcium Channel Blocker in Myotonic Dystrophy Type 1
This is a Phase 1 clinical trial designed to evaluate the safety and tolerability of amlodipine, a calcium channel blocker, in adults with Myotonic Dystrophy Type 1 (DM1).
- Trial name
- CAP DM1 (NCT07075965)
- Testing
- Amlodipine
- Volunteers
- 20 people
- Sponsor
- University of Rochester
Myopathy, Muscular Weakness
Efficacy and Tolerance of Early Launching of Nocturnal Non Invasive
This is a multicenter randomized controlled open labeled study testing efficacy and tolerance of early launching of night non invasive ventilation in patients with myotonic dystrophy type 1(DM1).
- Conditions
- Myopathy, Muscular Weakness, Respiratory Insufficiency
- Trial name
- DYVINE (NCT01225614)
- Testing
- Bilevel pressure ventilator
- Where
- France
- Volunteers
- 77 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris