Clinical trials: Mitochondrial Disorders
Traumatic Brain Injury
Copenhagen Head Injury Ciclosporin (CHIC) Study
This is an open label study on the pharmacokinetics and safety of ciclosporin in patients with severe traumatic brain injury, who require intensive care unit admission and monitoring of intracranial pressure via a ventricular…
- Trial name
- CHIC (NCT01825044)
- Testing
- NeuroSTAT 5 mg/kg/day, NeuroSTAT 10 mg/kg/day
- Where
- Denmark
- Volunteers
- 16 people
- Sponsor
- NeuroVive Pharmaceutical AB
Cytomegalovirus Retinitis, HIV Infections
A Study of the Safety and Tolerance of Long-Term Therapy With Intravenous Cytovene (Ganciclovir Sodium) for Cytomegalovirus Retinitis in Persons With AIDS
To evaluate the safety and tolerance of long-term ganciclovir (DHPG) therapy for newly diagnosed macular threatening Cytomegalovirus (CMV) retinitis in AIDS patients.
- Trial name
- NCT00002034
- Testing
- Zidovudine, Ganciclovir
- Where
- 9 sites in United States, Canada
- Volunteers
- 100 people
- Sponsor
- Hoffmann-La Roche
Parkinson's Disease (PD)
A Phase 2 Study to Assess the Effects of SUL-238 on High Energy Phosphates With ³¹P-MRS in Patients With Early, Untreated Parkinson's Disease
The main goal of the study is to investigate how well the new drug SUL-238 works in Parkinson's Disease (PD). This is done by means of an MRS scan. An MRS scan is similar to a regular MRI scan.
- Trial name
- SHEPHERD (NCT07322887)
- Testing
- SUL-238 (1500 mg t.i.d.) film-coated tablets, SUL-238 (500 mg t.i.d.) film-coated tablets
- Where
- Netherlands
- Volunteers
- 45 people
- Sponsor
- GEN İlaç ve Sağlık Ürünleri A.Ş.
Leber Hereditary Optic Neuropathy (LHON)
Leber Hereditary Optic Neuropathy (LHON) Historical Case Record Survey
The objective of this survey is to establish the clinical course of vision loss and recovery in patients with a genetically confirmed diagnosis of Leber Hereditary Optic Neuropathy (LHON).
- Trial name
- NCT01892943
- Where
- 8 sites in Belgium, Denmark and 3 more
- Volunteers
- 306 people
- Sponsor
- Santhera Pharmaceuticals
Friedreich Ataxia
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
The primary objective of the study is to evaluate the efficacy (using the modified Friedreich Ataxia Rating Scale [mFARS]) and safety of vatiquinone in participants with Friedreich ataxia (FA).
- Trial name
- MOVE-FA (NCT04577352)
- Testing
- Vatiquinone
- Where
- 14 sites in United States, Australia and 7 more
- Volunteers
- 146 people
- Sponsor
- PTC Therapeutics
Friedreich's Ataxia
GV101 for Friedreich's Ataxia (FA) Treatment
This is a dose-ranging, randomized, double-blind, placebo-controlled 12-week Phase 2a trial with an up to two week loading dose period.
- Trial name
- NCT07870590
- Testing
- GV101
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Graviton Bioscience Corporation
Mitochondrial Disease, Chronic Kidney Disease
Expanded Access Program for R007 (Probucol) Tablets in Adults With Mitochondrial Disease and Chronic Kidney Disease
The purpose of this expanded access program is to enable access to R007 (probucol) tablets for the compassionate treatment of adults with mitochondrial disease who also have chronic kidney disease.
- Trial name
- NCT06931834
- Testing
- Probucol
- Where
- United States
- Sponsor
- RiboNova Inc.
Cytomegalovirus Retinitis, HIV Infections
A Randomized Study Comparing the Safety and Efficacy of Two Regimens of Oral Ganciclovir to Intravenous Ganciclovir Maintenance Therapy for Cytomegalovirus Retinitis in People With AIDS Who Have Received Prior Ganciclovir Therapy
To compare the safety and tolerance of oral ganciclovir at a double dose 3 times/day or a single dose 6 times/day to IV ganciclovir given for 20 weeks of maintenance therapy.
- Trial name
- NCT00002247
- Testing
- Ganciclovir
- Where
- 20 sites in United States, Canada
- Volunteers
- 225 people
- Sponsor
- Hoffmann-La Roche
Friedreich Ataxia, Healthy Volunteers
Determine Range of Tissue Frataxin Concentrations and Other Potential Biomarkers
To examine range of tissue frataxin (FXN) concentrations, specific ribonucleic acids, other proteins and specialized lipids in the buccal cells, blood, and skin cells of normal healthy volunteers without Friedreich's ataxia…
- Trial name
- NCT05028764
- Testing
- Buccal Swabs, Blood Draws and Skin Punch Biopsy
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Larimar Therapeutics, Inc.
Alzheimer's Disease
Safety and Efficacy Study Evaluating Dimebon in Patients With Mild to Moderate Alzheimer's Disease on Donepezil
The purpose of this study is to determine if Dimebon is safe and effective in patients with mild to moderate Alzheimer's disease on Donepezil.
- Trial name
- CONCERT (NCT00829374)
- Testing
- Dimebon
- Where
- 101 sites in United States, Australia and 8 more
- Volunteers
- 1,003 people
- Sponsor
- Medivation, Inc.
Colitis, HIV Infections
A Study of Ganciclovir in the Treatment of Cytomegalovirus of the Large Intestine in Patients With AIDS
To attempt to demonstrate the efficacy of ganciclovir (DHPG) treatment of cytomegalovirus (CMV) colitis in AIDS patients by evaluating both clinical and virologic parameters.
- Trial name
- NCT00002273
- Testing
- Ganciclovir
- Where
- United States
- Sponsor
- Hoffmann-La Roche
Friedreich Ataxia, Cardiomyopathy
A Study of ASP2016 in Adults Who Have Heart Disease Associated With Friedreich Ataxia
Friedreich Ataxia is a rare condition that causes damage to the nervous system and muscles. People with Friedreich Ataxia have difficulty walking, lose sensation in their arms and legs, and have slurred speech.
- Trial name
- NCT06483802
- Testing
- ASP2016, Prednisolone
- Sponsor
- Astellas Gene Therapies
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.
Leber Hereditary Optic Disease
Efficacy and Safety Study of Bilateral IVT Injection of GS010 at Two Dose Levels in LHON Patients
The goal of this Clinical trial is to assess the safety and efficacy of GS010 at two dose levels on visual acuity and retinal mitochondrial activity in patients affected with ND4 Leber Hereditary Optic Neuropathy (LHON)
- Trial name
- REVISE (NCT07303296)
- Testing
- GS010 High dose, GS010 Low dose
- Where
- France
- Volunteers
- 14 people
- Sponsor
- GenSight Biologics
Inherited Mitochondrial Disease
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
The primary objective of this study is to assess the safety of vatiquinone in participants with inherited mitochondrial disease who had prior exposure to vatiquinone in a PTC/BioElectron sponsored (previously Edison) clinical…
- Trial name
- NCT05218655
- Testing
- Vatiquinone
- Where
- 26 sites in United States, France and 5 more
- Volunteers
- 101 people
- Sponsor
- PTC Therapeutics
Mitochondrial Diseases
RG2133 (2',3',5'-Tri-O-Acetyluridine) in Mitochondrial Disease
The objective of the study is to determine the safety and tolerability of RG2133 in patients with Mitochondrial Disease.
- Trial name
- NCT00060515
- Testing
- RG2133 (2',3',5'-tri-O-acetyluridine)
- Volunteers
- 12 people
- Sponsor
- Repligen Corporation
Cytomegalovirus Retinitis, HIV Infections
A Phase I Study of the Safety of Proleukin (Aldesleukin) in Combination With Ganciclovir and Antiretroviral Therapy in HIV Seropositive Patients With Cytomegalovirus (CMV) Retinitis
To determine the MTD and dose-limiting toxicities of a regimen of therapeutic ganciclovir, antiretroviral therapy, and recombinant interleukin-2 (aldesleukin; Proleukin) as an immune adjuvant in HIV-seropositive patients.
- Trial name
- NCT00002321
- Testing
- Aldesleukin
- Where
- United States
- Sponsor
- Chiron Corporation
Aging, Cognitive Decline
Impact of a Novel Brain Longevity Supplement Containing the Postbiotic Urolithin A (Mitopure) on Cognitive Function and Related Health Outcomes
This is a parallel group, randomized, double-blind, placebo-controlled clinical study assessing the safety and effects of a novel nutritional product on self-reported cognitive function and health in adults that reside in the…
- Conditions
- Aging, Cognitive Decline, Brain Fog, Cognitive Function
- Trial name
- CLARITY (NCT07060898)
- Testing
- Active Product
- Where
- United States
- Volunteers
- 650 people, healthy volunteers welcome
- Sponsor
- Amazentis SA
Autism Spectrum Disorder
An Exploratory Open Label Study of EPI-743 (Vincerinone TM) in Children With Autism Spectrum Disorder
The investigators hypothesize that EPI-743 may provide clinical benefit to children with Autism Spectrum Disorder.
- Trial name
- Autism (NCT02226458)
- Testing
- EPI-743
- Sponsor
- Edison Pharmaceuticals Inc
Primary Mitochondrial Diseases
A First in Human Study to Evaluate the Safety of Infusion of MNV-BM-PLC (Autologous CD34+ Cells Enriched With Placenta Derived Allogeneic Mitochondria) in Patients With Primary Mitochondrial Diseases Associated With Mitochondrial DNA Mutation or Deletion
The study objectives are to evaluate the safety of a single intravenous (IV) infusion of autologous CD34+ cells enriched with placenta-derived allogeneic mitochondria in participant with primary mitochondrial disease associated…
- Trial name
- NCT04548843
- Testing
- Bone Marrow mobilization, Apheresis, MNV-BM-PLC infusion
- Where
- Israel
- Sponsor
- Minovia Therapeutics Ltd.