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Leigh Disease clinical trials

View clinical trials related to Leigh Disease.

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NCT ID: NCT05554835 Recruiting - MDS Clinical Trials

Global Registry and Natural History Study for Mitochondrial Disorders

Start date: February 1, 2009
Phase:
Study type: Observational [Patient Registry]

The main goal of the project is provision of a global registry for mitochondrial disorders to harmonize previous national registries, enable world-wide participation and facilitate natural history studies, definition of outcome measures and conduction of clinical trials.

NCT ID: NCT05277363 Withdrawn - Leigh Syndrome Clinical Trials

A Study of the Natural Course of SURF1 Deficiency

Start date: May 4, 2022
Phase:
Study type: Observational

The purpose of the study is to prospectively and systematically collect standardized clinical information, to describe important features of the disease course of SURF1 deficiency. These include but are not limited to symptomatology, clinical course, and risk factors for severe disease and complications.

NCT ID: NCT04846036 Active, not recruiting - Clinical trials for Mitochondrial Diseases

The KHENERGYC Study

Start date: February 1, 2021
Phase: Phase 2
Study type: Interventional

This a randomized placebo controlled, double-blind phase II study to explore the pharmacokinetics, safety and efficacy of sonlicromanol in children (from birth to 17 years) with genetically confirmed mitochondrial disease of which the gene defect is known to decrease one or more oxidative phosphorylation system enzymes and who suffer from motor symptoms ("KHENERGYC").

NCT ID: NCT04378075 Completed - Clinical trials for Mitochondrial Diseases

A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy

MIT-E
Start date: September 28, 2020
Phase: Phase 2/Phase 3
Study type: Interventional

This is a parallel-arm, double-blind, placebo-controlled study with a screening phase that includes a 28-day run-in phase to establish baseline seizure frequency, followed by a 24-week, randomized, placebo-controlled phase. After completion of the randomized, placebo-controlled phase, participants may enter a 48-week, long-term, extension phase during which they will receive open-label treatment with vatiquinone.

NCT ID: NCT03747328 Withdrawn - Leigh Syndrome Clinical Trials

ABI-009 (Nab-sirolimus) in Patients With Genetically-confirmed Leigh or Leigh-like Syndrome

Start date: April 30, 2022
Phase: Phase 2
Study type: Interventional

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

NCT ID: NCT03137355 Recruiting - Leigh Syndrome Clinical Trials

The International Registry for Leigh Syndrome

Start date: June 17, 2015
Phase:
Study type: Observational [Patient Registry]

The purpose of this study is to develop a database containing clinical and laboratory information for patients with Leigh syndrome. The goal is to provide a greater understanding of Leigh syndrome allowing further characterization of this disease.

NCT ID: NCT02544217 Completed - Clinical trials for Mitochondrial Disease

A Dose-escalating Clinical Trial With KH176

Start date: May 2015
Phase: Phase 1
Study type: Interventional

Mitochondrial Diseases are rare progressive, multi-system, often early fatal disorders affecting both children and adults. KH176 is a novel chemical entity currently under development for the treatment of inherited mitochondrial diseases, including MELAS (Mitochondrial Encephalomyopathy, Lactic acidosis, and Stroke-like episodes), Leigh's Disease and Leber's Hereditary Optic Neuropathy (LHON). KH176 is a potent intracellular redox modulating agent targeting the reactive oxygen species which are important in the pathogenesis of disorders of mitochondrial oxidative phosphorylation. After demonstrating a favourable safety profile in the pre-clinical testing, the safety, tolerability and pharmacokinetic and pharmacodynamic characteristics of the compound will now be evaluated in healthy male subjects in this trial

NCT ID: NCT02352896 Completed - Leigh Syndrome Clinical Trials

Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome

Start date: January 31, 2014
Phase: Phase 2
Study type: Interventional

EPI-743 in Leigh syndrome participants that participated in previous EPI743-12-002 (NCT01721733) study.

NCT ID: NCT02023866 Completed - Clinical trials for Inherited Mitochondrial Disease, Including Leigh Syndrome

Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease

MITO-001
Start date: May 2014
Phase: Phase 2
Study type: Interventional

To evaluate safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) administered up to 1.3 g/m²/day in two divided doses, every 12 hours, for up to 6 months in patients with inherited mitochondrial disease.

NCT ID: NCT01803906 Enrolling by invitation - Clinical trials for Mitochondrial Disease

Tissue Sample Study for Mitochondrial Disorders

Start date: February 2012
Phase:
Study type: Observational

The investigators are studying patients with undefined mitochondrial diseases to identify genetic mutations in nuclear or mitochondrial Deoxyribonucleic Acid (DNA). Most patients with suspected or known mitochondrial diseases have no genetic confirmation. The investigators expect that evaluating tissue samples from patients with mitochondrial disorders will lead us to discover mutations in new or known genes causing mitochondrial dysfunction.