Clinical trials: Mitochondrial Disorder
Cytomegalovirus Infections
Neonatal CMV-Ganciclovir Follow-up Study
The purpose of this study is to document the developments associated with puberty and determine if any of the children who previously participated in another research study have been diagnosed with cancer.
- Trial name
- NCT00031421
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Frailty, Aging
MItochondrial Intervention for Resilience in Older Adults With Coenzyme Q10
This randomized, double-blind, placebo-controlled clinical trial will investigate whether 10 weeks of CoQ10 supplementation can improve biological resilience and functional health in community-dwelling adults aged 65 years or…
- Conditions
- Frailty, Aging, Mitochondria Health, Skin Aging
- Trial name
- MIRO-Q (NCT07772336)
- Testing
- Q10Vital® (CoQ10, ubiquinone)
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- University Medical Centre Ljubljana
Friedreich Ataxia, Neuro-Degenerative Disease
FA Clinical Outcome Measures
This multicenter natural history study aims to expand the network of clinical research centers in FA, and to provide a framework for facilitating therapeutic interventions.
- Trial name
- FA-COMS (NCT03090789)
- Where
- 14 sites in United States, Australia and 3 more
- Volunteers
- 2,000 people, healthy volunteers welcome
- Sponsor
- Friedreich's Ataxia Research Alliance
Clinically Isolated Syndrome, Relapsing-Remitting Multiple Sclerosis
Mitochondrial Dysfunction and Disease Progression
While the last several years have seen great strides in the treatment of relapsing forms of MS, progressive MS, responsible for the majority of MS-related disability, lags far behind.
- Conditions
- Clinically Isolated Syndrome, Relapsing-Remitting Multiple Sclerosis, Secondary Progressive Multiple Sclerosis, Primary Progressive Multiple Sclerosis
- Trial name
- NCT02549703
- Where
- United States
- Volunteers
- 47 people
- Sponsor
- Icahn School of Medicine at Mount Sinai
Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia
Palliative Care Needs of Children With Rare Diseases and Their Families
The palliative care needs of family caregivers of children with rare diseases and their children are largely unmet, including the need for support to prepare for future medical decision making.
- Conditions
- Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome
- Trial name
- FACE-Rare (NCT06938542)
- Testing
- Family Centered pediatric palliative care for family caregivers of children with rare diseases.
- Where
- United States
- Volunteers
- 480 people, healthy volunteers welcome
- Sponsor
- Children's National Research Institute
Multiple Sclerosis (MS) - Relapsing-remitting, Multiple Sclerosis
Healthy Eating and Active Living for People Living With Multiple Sclerosis (HEAL MS): A Lifestyle Program for People With Multiple Sclerosis
The goal of this clinical trial is to learn whether a structured lifestyle program can improve health and wellbeing in people living with multiple sclerosis (MS).
- Trial name
- HEAL-MS (NCT07589062)
- Testing
- Multi-Domain Lifestyle Intervention
- Where
- United Arab Emirates
- Volunteers
- 30 people
- Sponsor
- New York University Abu Dhabi
Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency
Hepatic Histopathology in Urea Cycle Disorders
This is a multi-site, retrospective chart review as well as a prospective study to evaluate histopathologic findings in liver samples from individuals with any UCD diagnosis.
- Conditions
- Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency, Citrullinemia 1, ARGI Deficiency
- Trial name
- NCT04908319
- Where
- 2 sites in United States
- Volunteers
- 70 people
- Sponsor
- Baylor College of Medicine
Mitochondrial Myopathy, MELAS Syndrome
Study of the Metabolism of Pyruvate and Related Problems in Patients With Lactic Acidemia
OBJECTIVES: I. Study the metabolism of pyruvate and related problems in patients with lactic acidemia. II. Define the nature of the metabolic defect.
- Conditions
- Mitochondrial Myopathy, MELAS Syndrome, Lactic Acidosis
- Trial name
- NCT00004353
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- National Center for Research Resources (NCRR)
Cytomegalovirus Infections, HIV Infections
A Phase I Pharmacokinetic Study in HIV-Positive Subjects of Oral Ganciclovir and Concomitant Antiretroviral Zidovudine and Didanosine
To determine whether there is a pharmacokinetic drug interaction between oral ganciclovir and oral zidovudine (AZT) and between oral ganciclovir and oral didanosine (ddI).
- Trial name
- NCT00002096
- Testing
- Zidovudine, Didanosine, Ganciclovir
- Where
- 3 sites in United States
- Volunteers
- 24 people
- Sponsor
- Hoffmann-La Roche
Primary Mitochondrial Disease
Natural History Study of Mitochondrial Myopathy
The goal of this observational study is to develop and validate tools to measure disease course in patients with primary mitochondrial myopathy (PMM).
- Trial name
- NCT05250375
- Where
- United States
- Volunteers
- 1,300 people, healthy volunteers welcome
- Sponsor
- Children's Hospital of Philadelphia
Diabetes
Changes in Mitochondrial Uncoupling Protein 2 (UCP2) Messenger RNA(mRNA) in Type 2 Diabetes (T2DM) Patients
Many patients with Diabetes find difficulties in achieving glycemic control. Hemoglobin A1c(HgBA1c) is used as a marker for glycemic control.
- Trial name
- Diabetes (NCT01781754)
- Testing
- Blood withdrawal
- Where
- Israel
- Volunteers
- 50 people
- Sponsor
- Tel-Aviv Sourasky Medical Center
HIV Infections, Lipodystrophy
Observational Study of Fat Loss in HIV Infected Adults Taking Nucleoside Reverse Transcriptase Inhibitors (NRTIs)
Nucleoside reverse transcriptase inhibitors (NRTIs) are a class of anti-HIV (Human Immunodeficiency Virus) drug that can be an important part of an HIV treatment regimen.
- Conditions
- HIV Infections, Lipodystrophy, Metabolic Diseases, Nutrition Disorders
- Trial name
- NCT00119405
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Optic, Atrophy, Hereditary, Leber
Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation
The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation…
- Trial name
- RESCUE (NCT02652767)
- Testing
- GS010
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 39 people
- Sponsor
- GenSight Biologics
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
Mitochondrial Function, Glucose Intolerance
Dose-response Relationship Between Exercise and Health Outcomes
In this study 11 healthy volunteers will perform high intensity exercise training during 4 weeks. The exercise frequency will increase progressively during the first three weeks, followed by a fourth week with lower exercise…
- Trial name
- NCT04753021
- Testing
- Exercise
- Where
- Sweden
- Volunteers
- 11 people, healthy volunteers welcome
- Sponsor
- The Swedish School of Sport and Health Sciences
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
Myopathy, HMG COA Reductase Inhibitor Adverse Reaction
Evaluation of Ubiquinol on Mitochondrial Oxidative Capacity in Statin Patients Using 31PMRS
It is our primary hypothesis that statin drugs impair skeletal muscle mitochondrial function and that ubiquinol (the reduced active form of CoQ10) supplementation will block impairment of PCr recovery kinetics in patients using…
- Conditions
- Myopathy, HMG COA Reductase Inhibitor Adverse Reaction, Hypercholesterolemia
- Trial name
- NCT01702987
- Testing
- ubiquinol, statin
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Beth Israel Deaconess Medical Center
Mitochondrial Myopathies, Healthy Controls
Diagnostic Screening Tests and Potential Biomarkers in Mitochondrial Myopathies
The purpose of this study is to develop simple diagnostic screeningtests and investigate potential biomarkers for identifying patients with abnormalities of mitochondrial function, which also can be used as outcome measures in…
- Trial name
- NCT03513835
- Testing
- Diagnostic screenings tests
- Where
- Denmark
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Insulin Resistance
Mechanisms of Insulin Resistance and Exercise in South Asians
This study determines the effect of aerobic and resistance exercise training on whole-body and skeletal muscle insulin sensitivity in south Asians and evaluate the mechanisms which contribute to improvements in insulin…
- Trial name
- NCT04007926
- Testing
- Aerobic exercise programme, Resistance exercise programme
- Where
- United Kingdom
- Volunteers
- 66 people, healthy volunteers welcome
- Sponsor
- University of Glasgow
Human Immunodeficiency Virus (HIV), Mitochondrial Diseases
Mitochondria in HIV and Aging (MITO+)
Older adults with human immunodeficiency virus (HIV) and a long history of antiretroviral therapy have more mitochondrial dysfunction- the cells that help them make energy.
- Trial name
- (MITO+) (NCT03489421)
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- University of Colorado, Denver