Table 1. Some of the current clinical trials in mitochondrial disorders initiated in 2023.

Interventions	Mechanism	Status	Conditions	Phase	Location	Purpose of Study	Subjects	Trial Number
Coenzyme Q10	Antioxidant	Not yet recruiting	Prader-Willi Syndrome	Phase 2	Canada	To clarify the influence of Coenzyme Q10 on cellular metabolism in individuals with PWS.	N= 14 Age= childI, adultII Sex= female and male	NCT03831425
Omaveloxolone (SKYCLARYS)	Enhances mitochondrial biogenesis	Not yet recruiting	Friedreich Ataxia	Phase 1	United States	To assess the tolerability, safety, and pharmacokinetics of omaveloxolone in pediatric patients suffering from Friedreich's Ataxia	N=20 Age= child Sex= female and male	NCT06054893
N-Acetylcysteine	Antioxidant/restoring nitric oxide production	Recruiting	Mitochondrial Disease	Phase 1	United States	To identify the safest and most effective dose of N-Acetylcysteine.	N=18 Age= adult, older adultIII Sex= female and male	NCT05241262
Nicotinamide	Enhances mitochondrial biogenesis	Not yet recruiting	Friedreich Ataxia	Phase 2	Austria/ France/ Germany/ Italy/ Spain/ United Kingdom	To present clinical evidence supporting the effectiveness and safety of nicotinamide in individuals with Friedreich ataxia.	N= 225 Age= adult, older adult Sex= female and male	NCT03761511
Nicotinamide Riboside (NR)	Precursor of NAD+/increases mitochondrial number and mitochondrial function	Recruiting	Mitochondrial Myopathy	Phase 2	United States	To investigate the impact of Nicotinamide Riboside on adult-onset symptoms of mitochondrial myopathy	N=34 Age= adult, older adult Sex= female and male	NCT05590468
OMT-28	Enhance mitochondrial function	Recruiting	Primary Mitochondrial Disease	Phase 2	Germany/ Italy	To study the effects of OMT-28 on patients with primary mitochondrial disease	N=32 Age= adult Sex= female and male	NCT05972954
NR082 Injection	Gene Therapy	Recruiting	Leber Hereditary Optic Neuropathy	Phase 1/ Phase 2	United States	To assess the effectiveness and safety of NR082 in treating LHON associated with ND4 mutations	N=18 Age= adult, older adult Sex= female and male	NCT05293626
NFS-02 Injection	Gene Therapy	Recruiting	Leber Hereditary Optic Neuropathy	Phase 1/ Phase 2	United States/ China	To assess how safe and well-tolerated NFS-02 is and to look at its early effectiveness in treating LHON that is caused by a mutation in the mitochondrial ND1 gene	N=18 Adult, Age= older adult Sex= female and male	NCT05820152
MNV-201	Cell therapy produced by MAT	Recruiting	Mitochondrial Diseases/Pearson Syndrome	Phase 1	Israel	To assess the safety and therapeutic benefits of transplanting MNV-201 in children suffering from Pearson Syndrome	N= 5 Age= child, adult Sex= female and male	NCT06017869
Autologous USC (urine-derived stem cell) mitochondrial oocyte transplantation	MAT and mitochondrial transplantation	Enrolling by invitation	Female Infertility / Mitochondrial disorder	-	China	To investigate the effectiveness and safety of autologous mitochondrial transplantation of stem cells derived from urine in elderly women with a poor prognosis	N= 40 Age= adult, older adult Sex= female only	NCT06020742

Note:^IChild= birth – 17 years old.

^IIAdult= 18 – 64 years old.

^IIIOlder adult= above 65 years old.