Clinical trials: Mitochondria
Primary Mitochondrial Diseases
Collection of Blood and Skin Samples From Patients With Primary Mitochondrial Diseases and Healthy Volunteers
Mitochondrial diseases are a genetically diverse group of disorders, some of which are caused by mutations or deletions in the mitochondrial DNA (mtDNA) and which display a wide range of severity and phenotypes.
- Trial name
- NCT06474104
- Where
- Israel
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Minovia Therapeutics Ltd.
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.
Cytomegalovirus Infections, HIV Infections
Ganciclovir: Compassionate Use in Patients With Serious or Life-Threatening Cytomegalovirus Infections
To provide ganciclovir on a compassionate use basis to immunocompromised patients with serious cytomegalovirus (CMV) infections and to study safety and efficacy in this patient population.
- Trial name
- NCT00002024
- Testing
- Ganciclovir
- Where
- United States
- Sponsor
- Hoffmann-La Roche
Friedreich Ataxia
A Study to Assess Nomlabofusp in Adolescents and Children With Friedreich's Ataxia
The goal of this clinical trial is to evaluate the safety and tolerability of nomlabofusp (CTI-1601) in adolescents and children with Friedreich's ataxia (FRDA).
- Trial name
- NCT06681766
- Testing
- Nomlabofusp
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Larimar Therapeutics, Inc.
Solid Tumor
Study to Assess Safety and Efficacy of the Second Mitochondrial-derived Activator of Caspases (SMAC) Mimetic Debio 1143
Part A (dose-optimization)- to determine the recommended phase 2 dose (RP2D) taking into account dose-limiting toxicity (DLT/s) in Cycle 1, overall safety/tolerability and pharmacokinetic (PK), by optimizing doses of Debio 1143…
- Trial name
- SMARTPLUS-106 (NCT04122625)
- Testing
- Debio 1143, Nivolumab
- Where
- 17 sites in United States, France and 1 more
- Volunteers
- 46 people
- Sponsor
- Debiopharm International SA
Friedreich Ataxia
Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of multiple doses of intravenous DT-216 in adult patients with Friedrich Ataxia.
- Trial name
- NCT05573698
- Testing
- DT-216
- Where
- United States
- Volunteers
- 32 people
- Sponsor
- Design Therapeutics, Inc.
Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)
A Phase 2b Study of Zagociguat in Patients With MELAS
PRIZM is a Phase 2b randomized, double-blind, placebo-controlled, 3-treatment, 2-period, crossover study evaluating the efficacy and safety of oral zagociguat 15 and 30 mg vs.
- Trial name
- PRIZM (NCT06402123)
- Testing
- zagociguat 15mg, zagociguat 30mg
- Where
- 20 sites in United States, Australia and 4 more
- Volunteers
- 43 people
- Sponsor
- Tisento Therapeutics
Friedreich Ataxia, Cardiomyopathy, Secondary
Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia
This is a Phase 1/2, open-label, dose-ascending, multicenter study of the safety and efficacy of LX2006 for participants who have Friedreich's Ataxia with evidence of cardiomyopathy.
- Trial name
- NCT05445323
- Testing
- Low dose LX2006, Mid Dose LX2006, High Dose LX2006
- Where
- 3 sites in United States
- Volunteers
- 8 people
- Sponsor
- Lexeo Therapeutics
Parkinson's Disease
Safety and Biomarker Study of PTC-589 in Participants With Parkinson's Disease
Open-label study with 30-day run-in phase and adaptive design component to include more participants if deemed appropriate by investigators.
- Trial name
- NCT02462603
- Testing
- PTC-589
- Where
- 5 sites in United States, Germany and 1 more
- Volunteers
- 44 people
- Sponsor
- Edison Pharmaceuticals 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
Leber Hereditary Optic Neuropathy
Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year
The goal of this clinical trial is to assess the efficacy and safety of GS010 gene therapy - (lenadogene nolparvovec) in subjects with LHON due to the G11778A ND4 mitochondrial mutation with a vision loss up to one year.
- Trial name
- REFLECT (NCT03293524)
- Testing
- GS010
- Where
- 13 sites in United States, Belgium and 5 more
- Volunteers
- 98 people
- Sponsor
- GenSight Biologics
Friedreich Ataxia, Cardiomyopathies
A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ATI-105 in Healthy Adults
The goal of this clinical trial is to learn how ATI-105 administered to healthy participants at works in the body at different doses and with or without food.
- Conditions
- Friedreich Ataxia, Cardiomyopathies, Central Nervous System Diseases, Neuromuscular Diseases
- Trial name
- NCT07869654
- Testing
- ATI-105
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Adjuvia Therapeutics, Inc.
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
Mitochondrial Diseases
Open Label Extension to Assess Long Term Safety and Efficacy of KL1333 in Patients With Primary Mitochondrial Disease
The purpose of this study is to investigate if the study medicine, KL1333, is safe, well-tolerated and effective long-term in improving the symptoms of fatigue and impacts on daily living and functional capacity (physical…
- Trial name
- Falcon-OLE (NCT07514338)
- Testing
- Napazimone
- Where
- 23 sites in United States, Belgium and 7 more
- Volunteers
- 140 people
- Sponsor
- Pharming Technologies B.V.
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.
Leber's Hereditary Optic Neuropathy
A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic Neuropathy
This is a Phase 2, prospective, randomized, double-masked, vehicle controlled, single-center study in approximately 12 subjects with LHON to evaluate safety, tolerability and efficacy of elamipretide (MTP-131) topical ophthalmic…
- Trial name
- NCT02693119
- Testing
- elamipretide (MTP-131) 1% topical ophthalmic solution, Vehicle topical ophthalmic solution
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Stealth BioTherapeutics Inc.
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
Leigh Syndrome
ABI-009 (Nab-sirolimus) in Patients With Genetically-confirmed Leigh or Leigh-like Syndrome
A phase 2a, open-label study to evaluate the safety, tolerability, and clinical activity of ABI-009 (nab-sirolimus) in patients with genetically-confirmed Leigh or Leigh-like syndrome
- Trial name
- NCT03747328
- Testing
- ABI-009
- Sponsor
- Aadi Bioscience, Inc.
Primary Mitochondrial Disease
OMT-28 in Patients With Primary Mitochondrial Disease (PMD) (PMD-OPTION)
The goal of this clinical trial is to learn about the treatment effects of the investigational new drug OMT-28 in patients with Primary Mitochondrial Disease.
- Trial name
- PMD-OPTION (NCT05972954)
- Testing
- OMT-28
- Where
- 9 sites in Germany, Italy and 1 more
- Volunteers
- 28 people
- Sponsor
- Omeicos Therapeutics GmbH