Clinical trials: DM1
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
Myopathy
Efficacy and Tolerance of AVAPS Mode in Myotonic Dystrophy
Myotonic dystrophy type 1 is a myopathy with complex respiratory pattern and at risk to develop respiratory failure. Classical mode of ventilation are sometimes not tolerated or ineffective in this population.
- Trial name
- NCT01530841
- Testing
- Nocturnal ventilation
- Where
- France
- Volunteers
- 32 people
- Sponsor
- Centre d'Investigation Clinique et Technologique 805
Myotonic Dystrophy 1
Evaluation of Cognitive Functions in 20 Patients With Type 1 Myotonic Dystrophy With Virtual Reality Approach
Type 1 myotonic dystrophy (MD1) is a genetic and hereditary disease that primarily affects muscle tissue, resulting in myotonia (difficulty relaxing after contraction) and atrophy (progressive muscle weakening with decreased…
- Trial name
- GoodDiagNMD (NCT06270186)
- Testing
- Type 1 myotonic dystrophy
- Sponsor
- Centre Hospitalier Universitaire de Nice
Breast Cancer
A Prospective, Randomized Multicenter, Open-label Comparison of Preoperative Trastuzumab Emtansine (T-DM1) With or Without Standard Endocrine Therapy vs. Trastuzumab With Standard Endocrine Therapy Given for Twelve Weeks in Patients With Operable HER2+/HR+ Breast Cancer Within the ADAPT Protocol.
Trial to optimize neoadjuvant therapy for HER overexpression and co-expressing of hormone receptors(ER and/or PR) breast cancer (HEr2+/HR+).
- Trial name
- ADAPT; T-DM1 (NCT01745965)
- Testing
- T-DM1, Trastuzumab
- Where
- 2 sites in Germany
- Volunteers
- 380 people
- Sponsor
- Women's Cancer Study Group GmbH
Metastatic Breast Cancer
Trastuzumab & Pertuzumab Followed by T-DM1 in MBC
In HER2-positive metastatic breast cancer, trastuzumab based treatment is the standard of care as long as there are no contraindications to trastuzumab.
- Trial name
- NCT01835236
- Testing
- Trastuzumab, Pertuzumab, Paclitaxel
- Where
- 71 sites in France, Germany and 2 more
- Volunteers
- 208 people
- Sponsor
- Swiss Cancer Institute
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
Steinert's Disease, Myotonic Dystrophy 1
Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease)
The study team hypothesize that non-diabetic patients with Myotonic dystrophy type I (DM1) will improve their symptoms, especially their motor deficit which is the main feature of the disease, because of the splicing defect…
- Conditions
- Steinert's Disease, Myotonic Dystrophy 1, Metformin
- Trial name
- METFORMYO (NCT05532813)
- Testing
- Treatment taken
- Where
- France
- Volunteers
- 142 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Myotonia Congenita, Paramyotonia Congenita
Study of Ranolazine in Myotonia Congenita, Paramyotonia Congenita and Myotonic Dystrophy Type 1
The purpose of this study is to gather preliminary data to determine if ranolazine is a safe and effective treatment for the symptoms of myotonia congenital, paramyotonia congenita, and myotonic dystrophy type 1.
- Conditions
- Myotonia Congenita, Paramyotonia Congenita, Myotonic Dystrophy 1
- Trial name
- NCT02251457
- Testing
- Ranolazine
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Ohio State University
Advanced Breast Cancer
Phase 1b Study of PD-0332991 in Combination With T-DM1(Trastuzumab-DM1)
Standard of care: Treatment with Trastuzumab Experimental: 21-Day Cycle of Combination therapy with T-DM1 intravenously on Day 1 and oral PD-0332991 on Days 5-18 Study Design and Methodology: This is a phase 1B inter-patient dose…
- Trial name
- NCT01976169
- Testing
- PD-0332991 and T-DM1
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- University of Texas Southwestern Medical Center
Congenital Myotonic Dystrophy, Childhood Myotonic Dystrophy
Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension
This is a natural history study to improve the types of assessments and biological samples that will be used in clinical drug trials in both congenital myotonic dystrophy and childhood myotonic dystrophy.
- Conditions
- Congenital Myotonic Dystrophy, Childhood Myotonic Dystrophy, Myotonic Dystrophy
- Trial name
- TREAT-EXT (NCT06747884)
- Where
- 4 sites in United States, Brazil
- Volunteers
- 200 people
- Sponsor
- Virginia Commonwealth University
Breast Cancer, Breast Cancer Stage I
Adjuvant Chemoradiation and Biomarkers of Response in High-risk Breast Cancer
The goal of this study is to evaluate the safety and effectiveness of adjuvant chemoradiation therapy in high-risk breast cancer patients who had received neoadjuvant chemotherapy before their lumpectomy and/or mastectomy and…
- Conditions
- Breast Cancer, Breast Cancer Stage I, Breast Cancer Stage II, Breast Cancer Stage III
- Trial name
- Breast53 (NCT05288777)
- Testing
- T-DM1, Capecitabine, External Beam Radiation Therapy 0
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- University of Virginia
HER-2 Positive Breast Cancer, Breast Cancer
T-DM1+Pertuzumab in Pre-OP Early-Stage HER2+ BRCA
This research study is studying a combination of drugs as a possible treatment for breast cancer that has tested positive for a protein called HER2.
- Conditions
- HER-2 Positive Breast Cancer, Breast Cancer, Stage II Breast Cancer, Stage III Breast Cancer
- Trial name
- NCT02326974
- Testing
- T-DM1, Pertuzumab, Excision of tumor/mastectomy
- Where
- 4 sites in United States
- Volunteers
- 164 people
- Sponsor
- Dana-Farber Cancer Institute
Breast Cancer
ATOP TRIAL: T-DM1 in HER2 Positive Breast Cancer
This research study is studying an investigational drug as a possible treatment for breast cancer that is positive for the protein Human Epidermal Growth Factor Receptor 2, also known as HER2-positive breast cancer.
- Trial name
- NCT03587740
- Testing
- T-DM1
- Where
- 14 sites in United States
- Volunteers
- 111 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 Type 1 (DM1)
Technology Assisted Rehabilitation for Upper Limb Function in Myotonic Dystrophy Type 1
Myotonic Dystrophy type 1 (DM1) is a genetic multisystem disease causing muscle weakness and myotonia. As a result, upper limb function might become impaired.
- Trial name
- Technorehab (NCT05560438)
- Testing
- Tyromotion Amadeo and Armeo Senso
- Where
- Norway
- Volunteers
- 6 people
- Sponsor
- University of Oslo
Myotonic Dystrophy 1
DM-IMT - Controlled, Randomized, Three-arm Intervention Study on the Safety and Efficacy of Regular Respiratory Muscle Training in Patients With Myotonic Dystrophy Type 1
For some diseases, regular respiratory muscle training could delay the start of ventilation. For DM1, however, there are no clinically high-quality studies.
- Trial name
- DM-IMT (NCT04052958)
- Testing
- IMT - inspiratory muscle training
- Where
- Germany
- Volunteers
- 45 people
- Sponsor
- LMU Klinikum
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.
Breast Cancer
A Study of Trastuzumab Emtansine (T-DM1) Sequentially With Anthracycline-based Chemotherapy, as Adjuvant or Neoadjuvant Therapy for Patients With Early Stage Herceptin (HER)2-positive Breast Cancer
This single-arm open-label study assessed the safety, feasibility, and efficacy of trastuzumab emtansine (T-DM1) after the completion of anthracycline-based adjuvant/neoadjuvant chemotherapy in patients with early HER2-positive…
- Trial name
- NCT01196052
- Testing
- Trastuzumab emtansine
- Where
- 44 sites in United States, Belgium and 6 more
- Volunteers
- 153 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.
Wheelchair Mobility, Manual Wheelchair Skills Training
Wheelchair Skills Training for People with ARSACS and DM1
Wheelchairs (WC) are often provided to people with ARSACS and MD1 when they are not able to walk anymore. However, giving someone a MWC alone does not guarantee they will use it safely or properly.
- Conditions
- Wheelchair Mobility, Manual Wheelchair Skills Training, ARSACS, Myotonic Dystrophy Type 1
- Trial name
- NCT06596850
- Testing
- Wheelchair Skills Training Program
- Where
- Canada
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Laval University