Clinical trials: Steinert Disease
HER2-positive Breast Cancer, Recurrent Breast Cancer
BYL719 + T-DM1 in HER2(+) Metastatic Breast Cancer Pts Who Progress on Prior Trastuzumab & Taxane Tx
The purpose of this study is to see whether a combination of two different drugs - trastuzumab-MCC-DM1 (T-DM1) and BYL719 is safe, and if it might be effective in treating metastatic breast cancer.
- Conditions
- HER2-positive Breast Cancer, Recurrent Breast Cancer, Stage IIIA Breast Cancer, Stage IIIB Breast Cancer
- Trial name
- NCT02038010
- Testing
- PI3K inhibitor BYL719, ado-trastuzumab emtansine, pharmacological study
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Northwestern University
Neuromuscular Diseases (NMD), Charcot Marie Tooth Disease (CMT)
Personalized Training for People With Rare Neuromuscular Disorders
The goal of this study is to investigate the effects of personalized exercise treatment on dynamic balance and physical function in comparison with regular follow-up in adults with rare-neuromuscular disorders…
- Conditions
- Neuromuscular Diseases (NMD), Charcot Marie Tooth Disease (CMT), Facioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy Type 1 (DM1)
- Trial name
- PETRA-NMD (NCT06708468)
- Testing
- Exercise, Usual Care
- Where
- 5 sites in Norway
- Volunteers
- 120 people
- Sponsor
- Oslo University Hospital
Breast Cancer
FS-1502 Versus T-DM1 for HER2-Positive Unresectable Locally Advanced or Metastatic Breast Cancer
This study is designed to compare the anti-tumor activity as well as the safety and efficacy of FS-1502 versus T-DM1 in HER2-positive, unresectable locally advanced or metastatic breast cancer subjects previously treated with…
- Trial name
- NCT05755048
- Testing
- Investigational drug: Recombinant Anti-HER2 Humanized Monoclonal Antibody - Monomethyl Auristatin F Conjugates for Injection (FS-1502), Control drug: Trastuzumab emtansine (Kadcyla, T-DM1)
- Where
- 56 sites in China
- Volunteers
- 314 people
- Sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. 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
Steinert's Disease
Compare Train and 3D-4D Left Ventricular Systolic Function in Subjects Suffering From Dystrophy and Healthy Subjects
Steinert's disease is an orphan disease. The prognosis of patients with this disease is conditioned by cardiac involvement. Search an early stage alterations in contractile function in subjects suffering from dystrophy would…
- Trial name
- NCT02930408
- Testing
- electrocardiogram, Echocardiography
- Volunteers
- 70 people
- Sponsor
- Poitiers University Hospital
Diastolic Dysfunction
Diastolic Function in Myotonic Dystrophy Type 1
Myotonic dystrophy type 1 (DM1) is a neuromuscular disorder in relation with an unstable expansion of CTG repeat. Patients with DM1 are at risk of arrhythmia and conduction disorders.
- Trial name
- NCT06029192
- Where
- France
- Volunteers
- 500 people
- Sponsor
- Centre d'Investigation Clinique et Technologique 805
Spinal Muscular Atrophy (SMA), Charcot-Marie-Tooth
Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers
In recent years, knowledge of neuromuscular diseases has advanced considerably, and new therapeutic avenues are beginning to emerge.
- Conditions
- Spinal Muscular Atrophy (SMA), Charcot-Marie-Tooth, Muscular Dystrophy, Myotonic Dystrophy
- Trial name
- Myokinesis (NCT07321977)
- Testing
- Gait analysis via video capture
- Where
- France
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Institut de Myologie, France
Non-small Cell Lung Cancer
Deciphering Antitumour Response and Resistance With INtratumour Heterogeneity
DARWIN II is a multi-arm non-randomised phase II trial, Eligible patient will be those who relapse with NSCLC (clinical trials.gov ref. NCT02183883). .
- Trial name
- DARWINII (NCT02314481)
- Testing
- MPDL3280A, Vemurafenib, Alectinib
- Where
- United Kingdom
- Volunteers
- 50 people
- Sponsor
- University College, London
Type 1 Diabetes, Adolescent Development
Emoticare: Serious Game for the Adaptation to the Disease of Adolescents With Type 1 Diabetes Mellitus.
The intervention program targets adolescents with chronic Type 1 Diabetes Mellitus. Given the critical developmental stage and life transitions, necessitating robust personal resources, psychological support becomes imperative.
- Trial name
- NCT06331429
- Testing
- EmoTiCare
- Where
- Spain
- Volunteers
- 200 people
- Sponsor
- University of Valencia
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.
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
Myotonic Dystrophy 1, Sudden Cardiac Death
Sudden Cardiac Death Stratification in Myotonic Dystrophy Type 1 Patients
The aim of the study is to evaluate if the electrophysiological study (EPS) guided therapy, including the prophylactic implantation of implantable cardioverter defibrillator (ICD), in inducible patients, is able to improve…
- Trial name
- ACADEMY 1 (NCT03784586)
- Where
- Italy
- Volunteers
- 70 people
- Sponsor
- University of Campania Luigi Vanvitelli
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
Elderly Metastatic Breast Cancer Population
Elderly Metastatic Breast Cancer: Pertuzumab-Herceptin vs Pertuzumab-Herceptin-Metronomic Chemotherapy, Followed by T-DM1
Chemotherapy and HER2 targeted agents can improve survival significantly in metastatic breast cancer. Chemotherapy however is associated with significant side-effects and can impact on Quality of Life and functionality in older…
- Trial name
- NCT01597414
- Testing
- Pertuzumab + trastuzumab, Pertuzumab + trastuzumab + metronomic chemotherapy
- Where
- 30 sites in Belgium, France and 6 more
- Volunteers
- 80 people
- Sponsor
- European Organisation for Research and Treatment of Cancer - EORTC
Myotonic Dystrophy
A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1
This is a Phase 1/Phase 2 open-label single arm, multicenter, and multinational study with SAR446268 for treatment of male and female participants 10 to 55 years old with non-congenital myotonic dystrophy (DM) type 1 (DM1).
- Trial name
- BrAAVe (NCT06844214)
- Testing
- SAR446268
- Where
- 10 sites in United States, Argentina and 4 more
- Volunteers
- 32 people
- Sponsor
- Sanofi
DM1, Myotonic Dystrophy
Global Study of Del-desiran for the Treatment of DM1
A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy…
- Conditions
- DM1, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonia
- Trial name
- HARBOR (NCT06411288)
- Testing
- AOC 1001 (del-desiran)
- Where
- 34 sites in United States, Canada and 8 more
- Volunteers
- 159 people
- Sponsor
- Avidity Biosciences, Inc.
Diabete Mellitus
CGM to Aid Transition From Inpatient to Outpatient
A pilot study using the FreeStyle Libre to assess its potential benefits in patients with diabetes starting when they are inpatients and continuing during the transition of care to the outpatient setting to see if diabetes care…
- Trial name
- NCT03155594
- Testing
- Freestyle Libre
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Methodist Medical Center of Illinois
Breast Cancer
A Study of Poziotinib in Combination With T-DM1 in HER2-Positive Breast Cancer
This is a Phase 1b, open-label, multicenter study to determine the Maximum Tolerated Dose (MTD) or Maximum Administered Dose (MAD) of poziotinib when administered with standard dosing of T-DM1 (3.6 mg/kg IV on Day 1 of each…
- Trial name
- NCT03429101
- Testing
- Poziotinib, T-DM1
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Spectrum Pharmaceuticals, Inc
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