Clinical trials: Mitochondrial Diseases
Heart Failure，Congestive, Heart Failure New York Heart Association Class IV
Nicotinamide Riboside in LVAD Recipients
While preliminary data show that oral nicotinamide riboside (NR) supplementation increases myocardial levels of oxidized nicotine-adenine dinucleotide (NAD+) levels in mice, there has been no direct evidence that suggests oral NR…
- Conditions
- Heart Failure，Congestive, Heart Failure New York Heart Association Class IV, Mitochondrial Alteration
- Trial name
- PilotNR-LVAD (NCT03727646)
- Testing
- Nicotinamide riboside
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- University of Washington
Leber Hereditary Optic Neuropathy
Safety and Efficacy Study of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy (LHON)
This study is meant to assess the safety and efficacy of rAAV2-ND4 treatment of Leber hereditary optic neuropathy with 11778 LHON mutation.
- Trial name
- rAAV2-ND4 (NCT01267422)
- Testing
- rAAV2-ND4
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Bin Li
Autism Spectrum Disorders
University of California, San Diego (UCSD) Suramin Autism Treatment-1 (SAT1) Trial
This study is designed to test the safety and efficacy of a single, intravenous dose of suramin in autism spectrum disorders (ASD).
- Trial name
- SAT1 (NCT02508259)
- Testing
- Suramin, Saline
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- University of California, San Diego
Obesity
Effects of Roflumilast on Insulin and Blood Sugar Levels in Prediabetic Overweight and Obese Individuals
Background: Roflumilast is a drug used to treat chronic obstructive pulmonary disease (COPD). It is designed to help reduce lung inflammation.
- Trial name
- NCT01862029
- Testing
- Roflumilast
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Leber Congenital Amaurosis 10, Blindness
Extension Study to Study PQ-110-001 (NCT03140969)
Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders
- Trial name
- INSIGHT (NCT03913130)
- Testing
- QR-110
- Where
- 3 sites in United States, Belgium
- Volunteers
- 9 people
- Sponsor
- Laboratoires Thea
Friedreich Ataxia
NAD+ Precursor Supplementation in Friedreich's Ataxia
The primary objective is to test the safety and tolerability of short-term therapy with a nicotinamide adenine dinucleotide (NAD+) precursor (MIB-626) in adults with Friedreich's Ataxia (FA) without overt heart failure and with a…
- Trial name
- NCT04817111
- Testing
- MIB-626
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Metro International Biotech, LLC
Friedreich Ataxia
A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia
In this study, researchers are learning more about RTA 408, also known as omaveloxolone, BIIB141, or SKYCLARYS®. The main goal of this study is to learn more about the safety of RTA 408 and how it affects physical effort…
- Trial name
- NCT02255435
- Testing
- Omaveloxolone Capsules, 2.5 mg, Omaveloxolone Capsules, 5 mg, Omaveloxolone Capsules, 10 mg
- Where
- 11 sites in United States, Australia and 3 more
- Volunteers
- 172 people
- Sponsor
- Biogen
HIV, Aging
Angiotensin Receptors and Age Related Mitochondrial Decline in HIV Patients
This study is designed to evaluate specific factors in mitochondria that may precipitate premature aging and physical weakness in HIV patients. Angiotensin receptors 1 and 2 (AT1R and AT2R) are found in virtually every cell type.
- Conditions
- HIV, Aging, Sarcopenia, Angiotensin Receptor Antagonists
- Trial name
- RAS-HIV (NCT02606279)
- Testing
- valsartan
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Wake Forest University Health Sciences
Friedreich Ataxia
Interferon Gamma-1b in Friedreich Ataxia (FRDA)
Friedreich ataxia (FRDA) is a progressive neurodegenerative disease of children and adults for which there is presently no therapy.
- Trial name
- NCT01965327
- Testing
- Interferon Gamma-1b
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Children's Hospital of Philadelphia
Type 1 Diabetes
Effect of Metformin on Vascular and Mitochondrial Function in Type 1 Diabetes
Insulin resistance (IR) is an important contributor to increased cardiovascular disease risk in type 1 diabetes (T1D). The purpose of this study is to measure the effect of metformin on insulin sensitivity, vascular function and…
- Trial name
- MeT1 (NCT01813929)
- Testing
- Metformin
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- University of Colorado, Denver
Pulmonary Arterial Hypertension, Interstitial Lung Disease
A Study of Treprostinil to Treat Interstitial Lung Disease Pulmonary Hypertension
The purpose of this research study is to find out more about the drug treprostinil via inhaler and the mechanisms of why patients with pulmonary arterial hypertension related to Interstitial Lung disease (PAH-ILD) have…
- Conditions
- Pulmonary Arterial Hypertension, Interstitial Lung Disease, Healthy Adults
- Trial name
- NCT05564637
- Testing
- Treprostinil, Right Heart Catheterization (RHC) while exercising, Optional Muscle Biopsy
- Where
- United States
- Volunteers
- 29 people, healthy volunteers welcome
- Sponsor
- Mayo Clinic
Functional Gastrointestinal Disorders, Irritable Bowel Syndrome
Does Improving Vagal Tone Increase Mitochondrial Bioenergetics
This study evaluates the effect of auricular neurostimulation on mitochondrial bioenergetics and inflammation through vagal nerve modulation via non-invasive percutaneous electrical nerve field stimulator in children with…
- Conditions
- Functional Gastrointestinal Disorders, Irritable Bowel Syndrome, Dyspepsia, Functional Abdominal Pain Syndrome
- Trial name
- NCT03931330
- Testing
- Percutaneous neurostimulation
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Medical College of Wisconsin
Leber's Hereditary Optic Neuropathy (LHON)
Historical Case Record Survey of Visual Acuity Data From Patients With Leber's Hereditary Optic Neuropathy (LHON)
The purpose of this survey is to collect visual acuity data from patients with LHON in order to establish the clinical course (natural history) and visual acuity outcomes in patients with a genetically confirmed diagnosis of…
- Trial name
- NCT02796274
- Where
- 21 sites in Belgium, France and 5 more
- Volunteers
- 219 people
- Sponsor
- Santhera Pharmaceuticals
Chronic Kidney Diseases
Effects of GLP1-RA on Ectopic Fat Deposition in Chronic Kidney Disease
Chronic kidney disease (CKD) is a burden of morbidity and mortality. Increased protein breakdown in skeletal muscle (wasting) and ectopic fat deposition are important determinants of poor clinical outcome in patient with CKD.
- Trial name
- GLIMP (NCT05254418)
- Testing
- dulaglutide injection
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Vanderbilt University Medical Center
Leber's Hereditary Optic Neuropathy
Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy
The study is a dose-escalation study, phase 1. The objective of this proposed clinical trial is to evaluate the safety of mitochondrially targeted ND4 gene therapy with the adeno-associated viral vector in appropriate LHON…
- Trial name
- LHON (NCT02161380)
- Testing
- injection of scAAV2-P1ND4v2 1.18x10e9 vg (Low),, injection of scAAV2-P1ND4v2 5.81 X10e9 vg (Med), injection of scAAV2-P1ND4v2 2.4 X10e10vg (High)
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Byron Lam
HIV
Effect of Gender and HIV Infection on Zidovudine and Lamivudine Pharmacokinetics
This study evaluated the blood and blood cell concentrations of zidovudine and lamivudine in men versus women and in those with versus without HIV infection.
- Trial name
- NCT01386970
- Testing
- zidovudine 300mg and lamivudine 150mg as Combivir
- Where
- United States
- Volunteers
- 43 people, healthy volunteers welcome
- Sponsor
- University of Colorado, Denver
Hemodialysis-Induced Symptom, Mitochondrial Diseases
Mitochondria and Chronic Kidney Disease
The overarching goal of this study is to determine the role of chronic kidney disease and the activation of the kallikrein-kinin system during hemodialysis on the development of mitochondrial dysfunction; the investigators will…
- Trial name
- MitoCKD (NCT03177798)
- Testing
- Icatibant
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Vanderbilt University Medical Center
Friedreich's Ataxia
Long-Term Safety Extension Study of ACTIMMUNE® (Interferon γ-1b) in Children and Young Adults With Friedreich's Ataxia
The purpose of this long term extension study is to evaluate the long-term safety of ACTIMMUNE® (interferon-γ 1b) in participants with Friedreich's Ataxia (FA).
- Trial name
- STEADFAST (NCT02797080)
- Testing
- interferon γ-1b
- Where
- 4 sites in United States
- Volunteers
- 38 people
- Sponsor
- Amgen
HIV Infections
Metabolic Effects of Non-Thymidine Analogue Anti-HIV Medications
The purpose of this study is to observe the effects of certain anti-HIV medications on mitochondrial activity and fat cell death.
- Trial name
- NCT00433992
- Where
- United States
- Volunteers
- 56 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Heart Failure
The Effect of Nicotinamide Riboside on Skeletal Muscle Function in Heart Failure Subjects
Background: People are living longer and are more likely to survive a heart attack if they have one. Longer life expectancy is good but it also means more people get chronic heart failure over time.
- Trial name
- NCT03565328
- Testing
- Nicotinamide Riboside
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)