Clinical trials: Dystrophia Myotonica
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
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
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
Myotonic Dystrophy Type 1
Children's Health Research Institute(CHRI), Stanford Lucile Packard Children Hospital (LPCH) Protocol on Myotonic Dystrophy
Study to focus on the defining and managing the neuropsychological abnormalities of myotonic dystrophy and to find out if the neuropsychological abnormalities have any correlation with changes seen on Magnetic Resonance Imaging.
- Trial name
- CHRI (NCT02269865)
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Stanford University
Breast Cancer
Tolerability and Efficacy of Adjuvant T-DM1 in Patients with HER2 Positive Breast Cancer After Incomplete Pathological Response to Neoadjuvant Chemotherapy Including Anti-HER2 Agents.
Multicenter, retrospective-prospective, real-world observational study, with the aim of evaluating tolerability and efficacy in a population of patients treated according to clinical practice outside of studies randomized.
- Trial name
- ATD (NCT06272799)
- Testing
- T-DM1 adjuvant
- Where
- Italy
- Volunteers
- 160 people
- Sponsor
- Regina Elena Cancer Institute
Breast Cancer
T-DM1 vs Paclitaxel/Trastuzumab for Breast (ATEMPT Trial)
This research study is a Phase II clinical trial. Phase II clinical trials test the effectiveness of an investigational drug to learn whether the drug works in treating a specific cancer.
- Trial name
- NCT01853748
- Testing
- Trastuzumab, Paclitaxel, Trastuzumab emtansine
- Where
- 85 sites in United States
- Volunteers
- 512 people
- Sponsor
- Dana-Farber Cancer Institute
HER2 Positive Breast Cancers
A Study of Tucatinib (ONT-380) Combined With Ado-trastuzumab Emtansine (T-DM1) in Patients With HER2+ Breast Cancer
The purpose of this study is to determine the maximal tolerated dose (MTD) or recommended dose (RD) and to assess the safety and tolerability of tucatinib (ONT-380) combined with ado-trastuzumab emtansine (T-DM1) in patients with…
- Trial name
- NCT01983501
- Testing
- Tucatinib (ONT-380), T-DM1
- Where
- 11 sites in United States, Canada
- Volunteers
- 57 people
- Sponsor
- Seagen Inc.
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
Myotonic Dystrophy 1
Symptoms and Outcome Measures for Upper- Limb Function in Myotonic Dystrophy Type 1
Myotonic Dystrophy type 1 (DM1) is a multisystem disease that causes muscle weakness and myotonia. As a result upper limb function might become impaired.
- Trial name
- SOUL-DM1 (NCT05006924)
- Where
- 3 sites in Norway
- Volunteers
- 89 people
- Sponsor
- Oslo University Hospital
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
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
Poor Neck Proprioception May Cause Balance Deficits in Myotonic Dystrophy 1
Impairment of balance and gait are frequent complaints in patients with myotonic dystrophy type 1 (DM1). In these persons, there is an increased risk for stumbles and falls when compared to normal subjects.
- Trial name
- CABLAMYD (NCT04712422)
- Testing
- Healthy subjects, Pathologic group
- Where
- 2 sites in Italy
- Volunteers
- 42 people
- Sponsor
- Istituto Auxologico Italiano
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
Breast Cancer, Brain Metastasis
T-DM1 Alone Versus T-DM1 and Metronomic Temozolomide in Secondary Prevention of HER2-Positive Breast Cancer Brain Metastases Following Stereotactic Radiosurgery
Background: Sometimes breast cancer spreads (metastasizes) to the brain. Researchers want to study new treatments for brain metastases. The drug Temozolomide is approved to treat brain tumors.
- Conditions
- Breast Cancer, Brain Metastasis, Brain Cancer
- Trial name
- NCT03190967
- Testing
- T-DM1, TMZ
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- National Cancer Institute (NCI)
HER2-positive Breast Cancer
A Study of Tucatinib vs. Placebo in Combination With Ado-trastuzumab Emtansine (T-DM1) for Patients With Advanced or Metastatic HER2+ Breast Cancer
This study is being done to see if tucatinib with ado-trastuzumab emtansine (T-DM1) works better than T-DM1 alone to help patients who have a specific type of breast cancer called HER2 positive breast carcinoma.
- Trial name
- NCT03975647
- Testing
- tucatinib, T-DM1
- Where
- 490 sites in United States, Australia and 18 more
- Volunteers
- 466 people
- Sponsor
- Seagen, a wholly owned subsidiary of Pfizer
Myotonic Dystrophy Type 1
Virtual Reality-training in Theory of Mind in the Childhood Form of Myotonic Dystrophy Type 1
The ability of an individual to conceive some alternative representations and to behave in a flexible manner would emerge from preschool age and drastically improve between the ages of 3 and 5 (Doebel and Zelazo, 2013).
- Trial name
- TOM-VR-DM1 (NCT05916677)
- Testing
- Virtual Reality-training in Theory of Mind, Traditional training in Theory of Mind
- Where
- France
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Institut de Myologie, France
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
Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1
The purpose of this study is to investigate the effects of mexiletine treatment for 6 months on ambulation, myotonia, muscle function and strength, pain, gastrointestinal functioning, cardiac conduction, and quality of life in…
- Trial name
- NCT01406873
- Testing
- Mexiletine
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- University of Rochester
Myotonic Dystrophy Type 1, Urinary Incontinence
Pelvic Floor Muscle Training for Women with Myotonic Dystrophy
Myotonic dystrophy type 1 (DM1) is a neuromuscular disease characterized by multisystem manifestations. DM1 can affect the urinary system through the impact of the pelvic floor muscles (PFM).
- Trial name
- NCT06316778
- Testing
- Pelvic floor muscle training
- Where
- Canada
- Volunteers
- 12 people
- Sponsor
- Université de Sherbrooke
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