Clinical trials: Mitochondrial Disorder
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's Ataxia
A Study to Learn More About Friedrich's Ataxia in People Living in the Gulf Cooperation Council (GCC) Region of the World
In this study, researchers will learn more about a disease called Friedrich's Ataxia, also known as FA. This is known as an "observational" study, which collects health information about study participants without changing their…
- Trial name
- NCT07887828
- Volunteers
- 40 people
- Sponsor
- Biogen
Idiopathic Infertility, Infertility Unexplained
Biological Aging Hallmarks-Guided Integrative TCM and Conventional Medicine in Post-Treatment Unexplained Female Infertility
The goal of this clinical trial is to determine whether telomere profiling and other biological aging hallmarks can help identify underlying mechanisms of persistent infertility in women with post-treatment unexplained…
- Conditions
- Idiopathic Infertility, Infertility Unexplained, Infertility (IVF Patients), Infertility Assisted Reproductive Technology
- Trial name
- TECMI (NCT07404969)
- Testing
- 1. Aging Hallmarks Biomarker Profiling (Telomere-Based), 2. Integrative Botanical and Nutraceutical Intervention, 3. Natural Conception or Assisted Reproductive Technologies
- Where
- Switzerland
- Volunteers
- 15 people
- Sponsor
- BEYOND GENOMiX SA, AG, Ltd
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
Amyotrophic Lateral Sclerosis
GM604 Phase 2A Randomized Double-blind Placebo Controlled Pilot Trial in Amyotrophic Lateral Disease (ALS)
GM604 is an endogenous human embryonic stage neural regulatory and signaling peptide that controls the development, monitoring and correction of the human nervous system.
- Trial name
- GALS (NCT01854294)
- Testing
- GM604
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Genervon Biopharmaceuticals, LLC
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
A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
The primary objective of the study is to assess the pharmacokinetics (PK) and safety of vatiquinone administered in participants with Friedreich ataxia (FA) younger than 7 years.
- Trial name
- NCT05485987
- Testing
- Vatiquinone
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- PTC 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.
Friedreich Ataxia
A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors
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 Friedreich's Ataxia, also known as FA.
- Trial name
- NCT06623890
- Testing
- Omaveloxolone
- Where
- 14 sites in United States, Austria and 5 more
- Volunteers
- 300 people
- Sponsor
- Biogen
Mitochondrial Myopathy
Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy
Phase 1/2, multi-center, randomized, double-blind, multiple ascending dose, placebo-controlled study that enrolled 36 subjects with mitochondrial myopathy associated with genetically confirmed mitochondrial disease to evaluate…
- Trial name
- MMPOWER (NCT02367014)
- Testing
- elamipretide (low dose), elamipretide (intermediate dose), elamipretide (high dose)
- Where
- 4 sites in United States
- Volunteers
- 36 people
- Sponsor
- Stealth BioTherapeutics Inc.
Friedreich Ataxia
A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia
In this study, researchers will learn more about omaveloxolone, also known as BIIB141 or SKYCLARYS®. Omaveloxolone is already approved for people with Friedreich's Ataxia (FA) who are 16 years of age or older.
- Trial name
- BRAVE (NCT06953583)
- Testing
- Omaveloxolone
- Where
- 34 sites in United States, Australia and 14 more
- Volunteers
- 255 people
- Sponsor
- Biogen
Mitochondrial Diseases, Mitochondrial Myopathies
The KHENERGYZE Study
Mitochondrial diseases, estimated prevalence 1 in 4,300 adults, is caused by pathogenic mutations in genes finally encoding for mitochondrial proteins of the various enzyme complexes of the OXPHOS.
- Conditions
- Mitochondrial Diseases, Mitochondrial Myopathies, Mitochondrial Encephalomyopathies, MELAS Syndrome
- Trial name
- NCT04165239
- Testing
- KH176
- Where
- 4 sites in Denmark, Germany and 2 more
- Volunteers
- 27 people
- Sponsor
- Khondrion BV
Mitochondrial Diseases
EPI-743 for Mitochondrial Respiratory Chain Diseases
This study will evaluate the safety and efficacy of EPI-743 in participants with severe mitochondrial respiratory chain diseases who are considered to be within 90 days of end-of-life care.
- Trial name
- NCT01370447
- Testing
- EPI-743
- Where
- 16 sites in United States
- Sponsor
- PTC Therapeutics
Neuromuscular Disease
Safety and Efficacy Study of A0001 in Patients With the A3243G Mitochondrial DNA Point Mutation
This is a phase 2a, double-blind, placebo-controlled, single-center study. Twenty-one patients who qualify for the study will be randomly assigned to either active drug or placebo.
- Trial name
- NCT01074359
- Testing
- A0001 (alpha-tocopherolquinone)
- Where
- United Kingdom
- Volunteers
- 30 people
- Sponsor
- Penwest Pharmaceuticals Co.
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
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
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
Leber's Congenital Amaurosis
Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene
The purpose of this study is to evaluate the safety and tolerability of QR-110 administered via intravitreal injection in subjects with LCA due to the CEP290 p.Cys998X mutation.
- Trial name
- NCT03140969
- Testing
- QR-110
- Where
- 3 sites in United States, Belgium
- Volunteers
- 11 people
- Sponsor
- Laboratoires Thea
Mitochondrial Diseases, Pearson Syndrome
Evaluate the Safety and Therapeutic Effects of a Single Intravenous Infusion (IV) of Autologous CD34+ Cells Enriched With Allogenic Placenta-derived Mitochondria in Patients With a Diagnosis of Pearson Syndrome (PS)
Primary Mitochondrial diseases are a clinically and genetically heterogeneous group of disorders caused by mutations in genes encoded by nuclear Deoxyribonucleic Acid (DNA) or by mutations and/or deletions in the mitochondrial…
- Trial name
- NCT06017869
- Testing
- MNV-201
- Where
- Israel
- Volunteers
- 6 people
- Sponsor
- Minovia Therapeutics Ltd.
OPA1 Gene Mutation, Autosomal Dominant Optic Atrophy
SAD of IVT PYC-001 in OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy (Sundew)
A First-in-Human multi-centre, prospective, Phase1a, Single Ascending Dose (SAD) interventional study of PYC-001 in participants with confirmed OPA1 mutation (haploinsufficiency) associated ADOA.
- Conditions
- OPA1 Gene Mutation, Autosomal Dominant Optic Atrophy, Hereditary Optic Atrophies, Kjer Optic Atrophy
- Trial name
- NCT06461286
- Testing
- PYC-001
- Where
- 2 sites in Australia
- Volunteers
- 18 people
- Sponsor
- PYC Therapeutics