Clinical trials: MNGIE
Mitochondrial Neurogastrointestinal Encephalomyopathy
Natural History Study of MNGIE
Non-interventional, prospective, multicenter, natural history study of patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE)
- Trial name
- NCT04245917
- Testing
- Non-interventional
- Where
- 5 sites in United States, Turkey (Türkiye)
- Volunteers
- 60 people
- Sponsor
- Entrada Therapeutics, Inc.
MNGIE, Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE)
MNGIE Natural History Study
The MNGIE Retrospective Natural History Study is a collaborative study between the University of Cambridge and the University of Bologna.
- Trial name
- NCT07627217
- Where
- United Kingdom
- Volunteers
- 50 people
- Sponsor
- University of Cambridge
Mitochondrial Disorders, Mitochondrial Disease
Tissue Sample Study for Mitochondrial Disorders
The investigators are studying patients with undefined mitochondrial diseases to identify genetic mutations in nuclear or mitochondrial Deoxyribonucleic Acid (DNA).
- Conditions
- Mitochondrial Disorders, Mitochondrial Disease, Melas, Kearns Sayer
- Trial name
- NCT01803906
- Where
- United States
- Volunteers
- 6,900 people, healthy volunteers welcome
- Sponsor
- Columbia University
Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE)
MNGIE Allogeneic Hematopoietic Stem Cell Transplant Safety Study
The purpose of this study is to find out if a stem cell transplant is safe for patients with a very rare disease. The stem cell transplant is called AHSCT (for "allogeneic hematopoetic stem cell transplantation").
- Trial name
- MASS (NCT02427178)
- Testing
- Hematopoietic Allogeneic Stem Cells
- Sponsor
- Michio Hirano, MD
Mitochondrial Disorders, Mitochondrial Genetic Disorders
North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)
The North American Mitochondrial Disease Consortium (NAMDC) maintains a patient contact registry and tissue biorepository for patients with mitochondrial disorders.
- Conditions
- Mitochondrial Disorders, Mitochondrial Genetic Disorders, Mitochondrial Diseases, Disorder of Mitochondrial Respiratory Chain Complexes
- Trial name
- NAMDC (NCT01694940)
- Where
- 17 sites in United States, Canada
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Columbia University
Mucopolysaccharidosis Disorders, Hurler Syndrome
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) using busulfan- and fludarabine-based…
- Conditions
- Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome
- Trial name
- NCT02171104
- Testing
- Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen
- Where
- United States
- Volunteers
- 149 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Mitochondrial Diseases, Kearns-Sayre Syndrome
Global Registry and Natural History Study for Mitochondrial Disorders
The main goal of the project is provision of a global registry for mitochondrial disorders to harmonize previous national registries, enable world-wide participation and facilitate natural history studies, definition of outcome…
- Conditions
- Mitochondrial Diseases, Kearns-Sayre Syndrome, MIDD, SANDO
- Trial name
- GENOMIT (NCT05554835)
- Where
- 34 sites in Austria, Germany and 1 more
- Volunteers
- 6,000 people
- Sponsor
- LMU Klinikum
Mitochondrial Diseases
A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease
A long-term extension study to assess the safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) in children with inherited mitochondrial diseases who previously enrolled into study…
- Trial name
- NCT02473445
- Testing
- Cysteamine Bitartrate
- Where
- 5 sites in United States
- Volunteers
- 22 people
- Sponsor
- Amgen
Mitochondrial Neurogastrointestinal Encephalopathy Disease
Evolution of Nutritional Status and Intestinal Function in Patients With MNGIE
Mitochondrial neurogastrointestinal encephalomyopathy syndrome (MNGIE) is a rare disease characterized by severe gastrointestinal and neurological dysfunction.
- Trial name
- SN-FI_MNGIE (NCT06784453)
- Where
- Italy
- Volunteers
- 8 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
Mitochondrial NeuroGastroIntestinal Encephalopathy (MNGIE)
The Natural History Study of Mitochondrial NeuroGastroIntestinal Encephalopathy (MNGIE)
This is a multi-center natural history study of Mitochondrial NeurogastroIntestinal Encephalopathy (MNGIE). Patients will be followed over time to assess clinical symptoms.
- Trial name
- NAHIM (NCT01694953)
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Columbia University
MNGIE, Dysmotility
Digestive Dysmotility in Mitochondrial Neurogastrointestinal Encephalomyopathy
Mitochondrial neurogastrointestinal encephalopathy (MNGIE) is an ultra-rare mitochondrial disease caused by mutations of the gen that codifies the enzyme thymidine phosphorylase The genetic defect results in systemic accumulation…
- Trial name
- NCT05658822
- Where
- Spain
- Volunteers
- 6 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Mitochondrial Diseases, Metabolic Disease
Trial of Erythrocyte Encapsulated Thymidine Phosphorylase In Mitochondrial Neurogastrointestinal Encephalomyopathy
The purpose of this study is to determine the safety, tolerability, action and effectiveness of repeated doses of Erythrocyte Encapsulated Thymidine Phosphorylase (EE-TP) for the treatment of patients with Mitochondrial…
- Conditions
- Mitochondrial Diseases, Metabolic Disease, Inborn Errors of Metabolism
- Trial name
- TEETPIM (NCT03866954)
- Testing
- EE-TP
- Sponsor
- St George's, University of London