A Dose-escalating Clinical Trial With KH176

Khondrion

Status and phase: Completed; Phase 1

Conditions: LHON; Leigh Syndrome; Mitochondrial Disease; MELAS; Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation

Treatments: Drug: placebo; Drug: KH176

Study type: Interventional

Funder types: Industry; Other

Identifiers

NCT02544217

KH176-101

Details and patient eligibility

About

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

Enrollment: 32 patients

Sex: Male

Ages: 18 to 55 years old

Volunteers: Accepts Healthy Volunteers

Inclusion criteria

• Healthy as assessed by medical history, physical examination, Vital Signs, Clinical Laboratory, ECG

Exclusion criteria

• Allergies,
• Concomitant medication,
• concomitant disease,
• relevant surgery,
• recent blood donation

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Double Blind

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32 participants in 2 patient groups

Single Escalating

Experimental group

Description:

2 alternating groups receiving escalating single doses of active/placebo

Treatment:

Drug: placebo

Drug: KH176

Multiple Escalating

Experimental group

Description:

3 multiple escalating groups, receiving active/placebo

Treatment:

Drug: placebo

Drug: KH176