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Phase 2 Study of EPI-743 in Children With Pearson Syndrome

An Open-Label Phase 2 Safety and Efficacy Study of EPI-743 (VincerinoneTM) in Children With Pearson Syndrome

Status
Terminated
Phases
Phase 2
Study type
Interventional
Source
ClinicalTrials.gov
Registry ID
NCT02104336
Enrollment
2
Registered
2014-04-04
Start date
2014-08-31
Completion date
2016-02-29
Last updated
2020-11-18

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Pearson Syndrome

Keywords

Pearson, EPI743, EPI-743, Vincerinone, Mitochondrial disorder, Mitochondrial disease

Brief summary

Treatment of Pediatric Subjects with Pearson syndrome

Detailed description

If effective, treatment with EPI-743 should result in a significant reduction in occurrence of sepsis, metabolic crisis, hepatic failure and transfusion dependence.

Interventions

DRUGEPI-743

EPI-743 is the quinone oxidation product of alpha-tocotrienol

Sponsors

Edison Pharmaceuticals Inc
Lead SponsorINDUSTRY

Study design

Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE

Eligibility

Sex/Gender
ALL
Age
No minimum to 18 Years
Healthy volunteers
No

Inclusion criteria

* Genetically confirmed diagnosis of Pearson syndrome * Age less than 18 * Availability of medical history for 12 months prior to enrollment * Abstention from use of CoQ10, vitamin E, lipoic acid and Idebenone 14 days prior to treatment with EPI-743

Exclusion criteria

* Allergy to EPI-743, sesame oil or vitamin E * Clinical history of bleeding/ abnormal PT/PTT * Concurrent inborn errors of metabolism * Use of anticoagulant medications * Participation in any interventional study within 30 days of treatment * Use of erythropoietin 30 days prior to trial enrollment

Design outcomes

Primary

MeasureTime frame
Occurence of episodes of sepsis, metabolic crisis or hepatic faillure1 year

Secondary

MeasureTime frameDescription
Neuromuscular function1 yearNeuromuscular function as assessed by Gross Motor Function Measure
Disease severity1 yearDisease severity as assessed by Newcastle Pediatric Mitochondrial Disease Scale
Renal function1 year—
Hepatic function1 year—
Hematologic function1 year—
Hospitalizations1 year—
Pancreatic function1 yearPancreatic function as assessed by insulin requirement and hemoglobin A1c
Mortality1 year—
Transfusion avoidance1 year—
Weight gain1 year—

Other

MeasureTime frameDescription
Number of Dose Limiting SAEs1 yearAny adverse event greater than or equal to grade 3 that is deemed related to treatment with EPI-743 and not related to underlying disease

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026