Clinical trials: Mitochondrial Diseases
Friedreich Ataxia
A Study to Learn How BIIB141 (Omaveloxolone) Affects the Health of Participants With Friedrich's Ataxia Who Took it During Pregnancy and/or During Breastfeeding and About the Health of Their Babies
In this study, researchers will learn more about the safety of BIIB141, also known as omaveloxolone or SKYCLARYS. This is a drug available for doctors to prescribe for people with Friedrich's Ataxia, also known as FA.
- Trial name
- NCT06628687
- Testing
- Omaveloxolone
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Biogen
Leigh Syndrome
Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome
The purpose of this study is to evaluate the effects of EPI-743 in children with Leigh syndrome on disease severity, neuromuscular function, respiratory function, disease morbidity and mortality and disease associated biomarkers.
- Trial name
- NCT01721733
- Testing
- EPI-743 15 mg/kg, EPI-743 5 mg/kg
- Where
- 4 sites in United States
- Volunteers
- 35 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
Friedreich Ataxia
An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia
An open label study designed to evaluate the safety, PK, PD, and clinical effects of long-term daily administration of CTI-1601 enrolling adolescent and adult patients with FRDA who have participated in a prior clinical study of…
- Trial name
- NCT06447025
- Testing
- CTI-1601
- Where
- 10 sites in United States
- Volunteers
- 85 people
- Sponsor
- Larimar Therapeutics, Inc.
Leber's Hereditary Optic Neuropathy
Study to Assess Efficacy,Safety and Tolerability of Idebenone in the Treatment of Leber's Hereditary Optic Neuropathy
This study is meant to assess the effectiveness of idebenone on visual function measures in patients with Leber's Hereditary Optic Neuropathy over a 6 months period.
- Trial name
- RHODOS (NCT00747487)
- Testing
- Idebenone
- Where
- 3 sites in Canada, Germany and 1 more
- Volunteers
- 85 people
- Sponsor
- Santhera Pharmaceuticals
Friedreich Ataxia, Cardiomyopathy
Clinical Course Of Disease In Participants With FA-CM
Characteristics and clinical course of disease In participants with cardiomyopathy associated with Friedreich Ataxia (CLARITY-FA)
- Trial name
- NCT06865482
- Where
- 19 sites in United States, Brazil and 6 more
- Volunteers
- 65 people
- Sponsor
- Lexeo Therapeutics
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.
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
Frailty, Sarcopenia
Impact of Urolithin A (Mitopure) on Mitochondrial Quality in Muscle of Frail Older Adults
The study is a single-center, randomized, placebo-control double-blind study in frail older adults over 65yrs. to investigate the impact of Mitopure (Urolithin A) supplementation on muscle mitochondrial quality in frail older…
- Conditions
- Frailty, Sarcopenia, Aging, Muscle Atrophy
- Trial name
- MitoEM (NCT06556706)
- Testing
- Mitopure (Urolithin A)
- Where
- Canada
- Volunteers
- 26 people
- Sponsor
- Amazentis SA
Head and Neck Squamous Cell Carcinoma, Oral Mucositis
MIT-001 for Prevention of CCRT-Induced OM in HNSCC Patients
The proposed study in patients with previously untreated locally advanced head and neck squamous cell carcinoma (HNSCC) is designed to evaluate the efficacy and safety of three different doses of MIT-001 compared to the placebo…
- Trial name
- MIT-001 (NCT04651634)
- Testing
- MIT-001 plus CCRT
- Where
- 16 sites in United States, South Korea
- Volunteers
- 60 people
- Sponsor
- MitoImmune Therapeutics
Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)
An Open-label Extension Safety Study of MELAS Patients Who Completed TIS6463-203 (PRIZM)
The goal of this clinical trial is to evaluate the long-term safety and tolerability of zagociguat in patients with MELAS who completed study medication treatment in the lead-in study TIS6463-203.
- Trial name
- NCT06961344
- Testing
- zagociguat 15mg
- Where
- 16 sites in United States, Australia and 3 more
- Volunteers
- 37 people
- Sponsor
- Tisento Therapeutics
Diabete Type 2, Obesity
Ph 2a Study of HU6 on Energy Metabolism, Muscle and Liver Substrate Metabolism, and Mitochondrial Function in Subjects Who Are Overweight or Obese With Type 2 Diabetes
This is a randomized, double-blind, placebo-controlled parallel group study of HU6 and placebo in subjects who are overweight or obese with T2D. The study will be conducted in 4 stages.
- Trial name
- NCT06104358
- Testing
- HU6
- Where
- United States
- Sponsor
- Rivus Pharmaceuticals, Inc.
Reperfusion Injury, Post Cardiac Arrest Brain Injury
Light Utilization COX-Inhibitory Device Therapy for Infant Cardiac Arrest (LUTICA Study). The LUCID Device is Used in the Treatment of Ischemic Brain Reperfusion Injury Caused by Cardiac Arrest in Pediatric Patients.
Approximately 15,200 children receive cardiopulmonary resuscitation (CPR) for in-hospital cardiac arrest (IHCA) each year in the United States. Of these, about 60% are less than one year of age.
- Trial name
- LUTICA (NCT07556939)
- Testing
- LUTICIA - Light Utilization COX-Inhibitory Device)
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Mitovation, Inc
Intermediate AMD
A Clinical Trial Evaluating the Safety and Efficacy of a New Light Combination Therapy Addressing Intermediate AMD
The proposed clinical investigation wants primary to validate the safety of the innovative light therapy approach and in second priority provide insight and confirmations on therapeutic effect.
- Trial name
- retinaSEES-PoC (NCT06557369)
- Testing
- reSEES
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- Oculox Technologies SA
Myelodysplastic Syndromes
A Study to Evaluate the MNV-201 in Patients With Low Risk MDS
Myelodysplastic syndromes (MDS) are a group of bone marrow failures that occur when the blood-forming cells in the bone marrow become abnormal leading to an abnormal differentiation and production of one or more blood cell types.
- Trial name
- NCT06465160
- Testing
- MNV-201 (Autologous CD34+ Cells Enriched with allogenic Placenta Derived Mitochondria)
- Where
- Israel
- Volunteers
- 15 people
- Sponsor
- Minovia Therapeutics Ltd.
Barth Syndrome
Clinical Trial in Patients With Barth Syndrome- 4TAZPower
Phase 3b/4, randomized, double-blind, parallel-group, placebo-controlled clinical trial to evaluate the efficacy, safety, and pharmacokinetics of a once daily SC injection of elamipretide in subjects with genetically confirmed…
- Trial name
- 4TAZPower (NCT07531251)
- Testing
- Elamipretide
- Where
- 3 sites in United States, Canada and 1 more
- Volunteers
- 48 people
- Sponsor
- Stealth BioTherapeutics 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.
Cytomegalovirus Infections, Cytomegalovirus Retinitis
Open Label Ganciclovir Therapy for Sight- or Life-Threatening Cytomegalovirus Disease in the Immunocompromised Patient
To make intravenous (IV) ganciclovir available to immunocompromised patients with life-threatening or sight-threatening Cytomegalovirus (CMV) infection, where the symptoms of the disease are too severe to allow admission to a…
- Conditions
- Cytomegalovirus Infections, Cytomegalovirus Retinitis, HIV Infections
- Trial name
- NCT00002025
- Testing
- Ganciclovir
- Where
- United States
- Sponsor
- Hoffmann-La Roche
Friedreich Ataxia, Cardiomyopathy, Secondary
Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy
The purpose of Study LX2006-03, a multicenter, Phase 2, open-label, randomized, controlled study, is to evaluate the efficacy and safety of LX2006 gene therapy in participants with Friedreich ataxia (FA) cardiomyopathy (CM).
- Trial name
- SUNRISE-FA 2 (NCT07721025)
- Testing
- LX2006, Usual Care
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- Lexeo Therapeutics
Cytomegalovirus Retinitis, HIV Infections
An Open-Label Safety Study of Oral Ganciclovir Maintenance Treatment of CMV Retinitis in People With Limited Venous Access
To provide oral ganciclovir to patients who require maintenance for control of cytomegalovirus (CMV) retinitis, but who lack patent permanent central venous access for long-term administration of intravenous drugs.
- Trial name
- NCT00002135
- Testing
- Ganciclovir
- Where
- United States
- Sponsor
- Hoffmann-La Roche