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

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ClinicalTrials.gov Identifier: NCT02104336
Recruitment Status : Terminated (Results from other studies did not support continuation of this trial)
First Posted : April 4, 2014
Last Update Posted : July 25, 2018
Sponsor:
Information provided by (Responsible Party):
Edison Pharmaceuticals Inc

Brief Summary:
Treatment of Pediatric Subjects with Pearson syndrome

Condition or disease Intervention/treatment Phase
Pearson Syndrome Drug: EPI-743 Phase 2

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.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 2 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Phase 2 Safety and Efficacy Study of EPI-743 (VincerinoneTM) in Children With Pearson Syndrome
Study Start Date : August 2014
Actual Primary Completion Date : November 2015
Actual Study Completion Date : February 2016


Arm Intervention/treatment
Experimental: EPI-743
15 mg/kg EPI-743 to be administered three times per day for 1 year
Drug: EPI-743
EPI-743 is the quinone oxidation product of alpha-tocotrienol
Other Name: Vincerinone (TM)




Primary Outcome Measures :
  1. Occurrence of episodes of sepsis, metabolic crisis or hepatic failure [ Time Frame: 1 year ]

Secondary Outcome Measures :
  1. Transfusion avoidance [ Time Frame: 1 year ]
  2. Hematologic function [ Time Frame: 1 year ]
  3. Neuromuscular function [ Time Frame: 1 year ]
    Neuromuscular function as assessed by Gross Motor Function Measure

  4. Disease severity [ Time Frame: 1 year ]
    Disease severity as assessed by Newcastle Pediatric Mitochondrial Disease Scale

  5. Renal function [ Time Frame: 1 year ]
  6. Hepatic function [ Time Frame: 1 year ]
  7. Weight gain [ Time Frame: 1 year ]
  8. Hospitalizations [ Time Frame: 1 year ]
  9. Pancreatic function [ Time Frame: 1 year ]
    Pancreatic function as assessed by insulin requirement and hemoglobin A1c

  10. Mortality [ Time Frame: 1 year ]

Other Outcome Measures:
  1. Number of Dose Limiting SAEs [ Time Frame: 1 year ]
    Any adverse event greater than or equal to grade 3 that is deemed related to treatment with EPI-743 and not related to underlying disease



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Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02104336


Locations
United States, California
Children's Hospital of Orange County
Orange, California, United States, 82868
United States, Ohio
Cleveland Clinic
Cleveland, Ohio, United States
United States, Pennsylvania
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
United States, Texas
Baylor College of Medicine
Houston, Texas, United States, 77030
Sponsors and Collaborators
Edison Pharmaceuticals Inc
Investigators
Study Chair: Matthew Klein, MD, FACS Edison Pharmaceuticals

Responsible Party: Edison Pharmaceuticals Inc
ClinicalTrials.gov Identifier: NCT02104336     History of Changes
Other Study ID Numbers: EPI743-13-024
First Posted: April 4, 2014    Key Record Dates
Last Update Posted: July 25, 2018
Last Verified: July 2018

Keywords provided by Edison Pharmaceuticals Inc:
Pearson
EPI743
EPI-743
Vincerinone
Mitochondrial disorder
Mitochondrial disease

Additional relevant MeSH terms:
Syndrome
Lipid Metabolism, Inborn Errors
Muscular Diseases
Mitochondrial Diseases
Disease
Pathologic Processes
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipid Metabolism Disorders
Metabolic Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Ubiquinone
Micronutrients
Growth Substances
Physiological Effects of Drugs