Clinical trials: Mitochondria
Bronchiolitis Acute, Wheezing
The Role of Vitamin D and Vitamin D Receptor in Recurrent Wheezing in Children With Bronchiolitis: Immunological Association With T-lymphocyte Mitochondrial Mass
A prospective study will be conducted involving children under 2 years of age hospitalized for bronchiolitis during the period 2025-2026.
- Conditions
- Bronchiolitis Acute, Wheezing, RSV
- Trial name
- NCT07700134
- Testing
- No Intervention: Observational Cohort
- Where
- Greece
- Volunteers
- 130 people, healthy volunteers welcome
- Sponsor
- National and Kapodistrian University of Athens
MELAS Syndrome, Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS)
A Study to Assess TTI-0102 vs Placebo in MELAS Patients
This is a randomized, double-blind, placebo-controlled study. Prior to treatment, patients will undergo a screening visit. If eligible, each subject will return for a Day 1 visit and will receive their first dose of…
- Trial name
- NCT06644534
- Testing
- TTI-0102
- Where
- 2 sites in France, Netherlands
- Volunteers
- 9 people
- Sponsor
- Thiogenesis Therapeutics, Inc.
Friedreich Ataxia, Friedreich's Ataxia
A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia
To evaluate the efficacy and safety of subcutaneous nomlabofusp in adult and pediatric subjects with Friedreich's ataxia
- Trial name
- FORWARD-FA (NCT07778836)
- Testing
- Nomlabofusp
- Where
- 4 sites in United States
- Volunteers
- 150 people
- Sponsor
- Larimar Therapeutics, Inc.
Friedreich Ataxia
Evaluation of the Effects of Calcitriol's in the Neurological Symptoms of Friedreich's Ataxia Patients
Friedreich's Ataxia (FA) is an autosomal recessive disease the mutation of which leads to a deficiency of a protein called frataxin, which is responsible for the symptoms of the disease.
- Trial name
- CalcitriolFA (NCT04801303)
- Testing
- Calcitriol, Blood analysis for frataxin's level measurement, Blood analysis for hypercalcemia's control
- Where
- Spain
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Berta Alemany
Mitochondrial Diseases
EPI-743 for Mitochondrial Respiratory Chain Diseases
This study will evaluate the safety and efficacy of EPI-743 in participants with severe mitochondrial respiratory chain diseases who are considered to be within 90 days of end-of-life care.
- Trial name
- NCT01370447
- Testing
- EPI-743
- Where
- 16 sites in United States
- Sponsor
- PTC Therapeutics
Autism Spectrum Disorder, Autism
Mitochondrial Dysfunction in Autism Spectrum Disorder
Researchers at Arkansas Children's Hospital Research Institute are conducting a study about mitochondrial function in children. The study involves up to 5 visits to Arkansas Children's Hospital with fasting blood draws…
- Conditions
- Autism Spectrum Disorder, Autism, Mitochondrial Disease, Developmental Delay
- Trial name
- Mito (NCT02000284)
- Where
- United States
- Volunteers
- 297 people, healthy volunteers welcome
- Sponsor
- University of Arkansas
Obesity, Insulin Resistance
Mitochondrial Function in Pediatric Obesity
The prevalence of pediatric obesity is increasing at an unprecedented rate. Obese children are at risk for the development of insulin resistance, relative insulin deficiency and type 2 diabetes mellitus.
- Trial name
- NCT00577174
- Where
- United States
- Volunteers
- 110 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Friedreich's Ataxia
Cardiac Output and Fatigue in Friedreich's Ataxia
This is a clinical trial examining to impact of aerobic training plus omaveloxolone in FRDA. Thirty individuals with FRDA will be recruited; 20 individuals will be on omaveloxolone treatment whereas the other ten individuals will…
- Trial name
- NCT07444333
- Testing
- Aerobic Exercise
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Scott Barbuto
Mitochondrial Diseases, Pearson Syndrome
A Study to Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-BM-BLD in Pediatric Patients With Pearson Syndrome
Mitochondrial diseases are a genetically heterogeneous group of disorders caused by mutations or deletions in mitochondrial DNA (mtDNA) displaying a wide range of severity and phenotypes.
- Trial name
- NCT03384420
- Testing
- CD34+ cells enriched with MNV-BLD
- Where
- Israel
- Volunteers
- 7 people
- Sponsor
- Minovia Therapeutics Ltd.
Mitochondrial Myopathies, Mitochondrial DNA Mutation
Genomic Profiling of Mitochondrial Disease - Imaging Analysis for Precise Mitochondrial Medicine
This study is an observational longitudinal study involving the use of MRIs and video recordings taken at home of patients completing basic tasks.
- Conditions
- Mitochondrial Myopathies, Mitochondrial DNA Mutation, Mitochondrial Diseases, Chronic Progressive External Ophthalmoplegia With Myopathy
- Trial name
- NCT05012358
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Mayo Clinic
Rare Diseases, Genetic Predisposition
Mitochondrial Diseases - Long-read Genome and Transcriptome Sequencing in Cases Unresolved After Short-read Genomics
The MiDiSeq project will enroll 20 unresolved index patients with suspected mitochondrial disease prioritized for genomic analysis.
- Trial name
- NCT03962452
- Testing
- Next Generation Sequencing (NGS)
- Where
- Germany
- Volunteers
- 20 people
- Sponsor
- University Hospital Tuebingen
Mitochondrial Disease
Anesthetic Effects in Mitochondrial Disease
Summary. At the present, the investigators do not have the perfect anesthetic for mitochondrial patients. When possible, consideration should be given to the use of local anesthetics in small amounts.
- Trial name
- NCT01001585
- Testing
- sevoflurane
- Where
- United States
- Volunteers
- 55 people
- Sponsor
- d sessler
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
Thymidine Kinase 2 Deficiency
An Open-Label Study of Continuation Treatment With Combination Pyrimidine Nucleosides in Patients With TK2 Deficiency
This is a Phase 2 prospective open-label treatment study of the safety and efficacy of doxecitine and doxribtimine in study participants with thymidine kinase 2 (TK2) deficiency who participated in the retrospective study…
- Trial name
- Continuation (NCT03845712)
- Testing
- doxecitine and doxribtimine
- Where
- 8 sites in United States, Israel and 1 more
- Volunteers
- 47 people
- Sponsor
- UCB BIOSCIENCES, Inc.
Friedreich Ataxia
Patient-reported, Health Economic and Psychosocial Outcomes in Friedreich Ataxia
The PROFA study is an international, multi-centric observational and validation study to assess the patient-reported, psychosocial and economic outcomes of patients with Friedreich Ataxia (FA).
- Trial name
- PROFA (NCT05943002)
- Where
- 6 sites in Austria, France and 1 more
- Volunteers
- 200 people
- Sponsor
- German Center for Neurodegenerative Diseases (DZNE)
Mitochondrial Diseases, Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
The KHENERGYC Study
This a randomized placebo controlled, double-blind phase II study to explore the pharmacokinetics, safety and efficacy of sonlicromanol in children (from birth to 17 years) with genetically confirmed mitochondrial disease of…
- Conditions
- Mitochondrial Diseases, Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation, MELAS, Subacute Necrotizing Encephalomyelopathy
- Trial name
- NCT04846036
- Testing
- Sonlicromanol
- Where
- Netherlands
- Volunteers
- 24 people
- Sponsor
- Khondrion BV
Cytomegalovirus Infections, HIV Infections
A Randomized, Double-Blind Study of the Efficacy and Safety of Oral Ganciclovir for the Prevention of CMV Disease in People Infected With the Human Immunodeficiency Virus
To evaluate the efficacy of oral ganciclovir in preventing progression to cytomegalovirus (CMV) disease (e.g., CMV retinitis, gastrointestinal CMV disease) in CMV-infected people with HIV infection and CD4 lymphocyte counts <=…
- Trial name
- NCT00002095
- Testing
- Ganciclovir
- Where
- 19 sites in United States
- Volunteers
- 700 people
- Sponsor
- Hoffmann-La Roche
MPAN
Mitochondrial Membrane Protein Neurodegeneration (MPAN)
The purpose of this case study was to investigate the effects of therapeutic intervention in 9- and 12-year-old brothers with MPAN.
- Trial name
- NCT05678790
- Testing
- Passive Stretching, Mobilization, Muscle strengthening
- Where
- Turkey (Türkiye)
- Volunteers
- 2 people
- Sponsor
- Kirsehir Ahi Evran Universitesi
Mitochondrial Disease
Vatiquinone Expanded Access Protocol
The purpose of this study is to provide participants who successfully completed the study titled "An Open-label, Safety Study for Previously Treated Vatiquinone (PTC743) Subjects With Inherited Mitochondrial Disease" with liquid…
- Trial name
- EAP (NCT07159139)
- Testing
- Vatiquinone
- Sponsor
- Medical University of South Carolina
Friedreich's Ataxia
Patient Reported Outcomes in Friedreich's Ataxia Patients After Withdrawal From Treatment With Idebenone (PROTI)
This is a Phase IIIb Double-Blind, Randomised, Placebo-Controlled Study. The aim is to further investigate the effects of idebenone in patients with Friedreich's ataxia.
- Trial name
- PROTI (NCT01303406)
- Testing
- Idebenone
- Where
- 6 sites in Austria, Germany and 2 more
- Volunteers
- 29 people
- Sponsor
- Santhera Pharmaceuticals