Pearson SyndromePhase 2NCT02104336
Phase 2 Study of EPI-743 in Children With Pearson Syndrome
A Phase 2 study testing EPI-743 in people with Pearson Syndrome, run by Edison Pharmaceuticals Inc at 4 sites in United States.
Trial summary
Treatment of Pediatric Subjects with Pearson syndrome Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Pearson Syndrome
- What is being tested?
- EPI-743
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 4 sitesUnited States
- Study dates
- 31 Aug 2014to 29 Feb 2016
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Cleveland Clinic
Cleveland, Ohio
Baylor College of Medicine
Houston, Texas, 77030
Children's Hospital of Orange County
Orange, California, 82868
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
About this study
Official title: An Open-Label Phase 2 Safety and Efficacy Study of EPI-743 (VincerinoneTM) in Children With Pearson Syndrome
Treatment of Pediatric Subjects with Pearson syndrome
Read the 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.
- Conditions
- Pearson Syndrome
- Being tested
- EPI-743
- Keywords
- Pearson, EPI743, EPI-743, Vincerinone, Mitochondrial disorder, Mitochondrial disease
- Medical subject headings
- VLCAD deficiency, Mitochondrial Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EPI-743Drug
Also called Vincerinone (TM)
EPI-743 is the quinone oxidation product of alpha-tocotrienol
| Study group | Role | What they receive |
|---|---|---|
| EPI-743 | Experimental | 15 mg/kg EPI-743 to be administered three times per day for 1 year Receives: Drug: EPI-743 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Occurence of episodes of sepsis, metabolic crisis or hepatic faillure
Measured: 1 year
Secondary outcomes (10)
Transfusion avoidance
Measured: 1 year
Hematologic function
Measured: 1 year
Neuromuscular function
Neuromuscular function as assessed by Gross Motor Function Measure
Measured: 1 year
Disease severity
Disease severity as assessed by Newcastle Pediatric Mitochondrial Disease Scale
Measured: 1 year
Renal function
Measured: 1 year
Hepatic function
Measured: 1 year
Weight gain
Measured: 1 year
Hospitalizations
Measured: 1 year
Pancreatic function
Pancreatic function as assessed by insulin requirement and hemoglobin A1c
Measured: 1 year
Mortality
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Apr 2014 (estimated)
- Study start31 Aug 2014
- Primary completion30 Nov 2015
- Study completion29 Feb 2016
Why it stopped: Results from other studies did not support continuation of this trial
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pearson SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Edison Pharmaceuticals Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Matthew Klein, MD, FACS, Study chair, Edison Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02104336
- Sponsor's protocol ID
- EPI743-13-024
- Status last verified
- Nov 2020
- First submitted
- 2 Apr 2014
- Last updated
- 18 Nov 2020
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Phase 2 Safety and Efficacy Study of EPI-743 (VincerinoneTM) in Children With Pearson Syndrome
- ClinicalTrials.gov ID
- NCT02104336
- Acronym / sponsor ID
- EPI743-13-024
- Conditions
- Pearson Syndrome
- Keywords
- Pearson, EPI743, EPI-743, Vincerinone, Mitochondrial disorder, Mitochondrial disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Results from other studies did not support continuation of this trial
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Edison Pharmaceuticals IncIndustry (pharma, biotech, devices)
- Study start
- 31 Aug 2014 (actual)
- Primary completion
- 30 Nov 2015 (actual)
- Study completion
- 29 Feb 2016 (actual)
- First posted
- 4 Apr 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| EPI-743 | Experimental | Drug: EPI-74315 mg/kg EPI-743 to be administered three times per day for 1 year |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EPI-743Also: Vincerinone (TM)EPI-743 is the quinone oxidation product of alpha-tocotrienol | Drug | EPI-743 |
Primary outcome
Occurence of episodes of sepsis, metabolic crisis or hepatic faillure
Time frame: 1 year
10 secondary outcomes
Transfusion avoidance
Time frame: 1 year
Hematologic function
Time frame: 1 year
Neuromuscular function
Time frame: 1 year
Disease severity
Time frame: 1 year
Renal function
Time frame: 1 year
Hepatic function
Time frame: 1 year
Weight gain
Time frame: 1 year
Hospitalizations
Time frame: 1 year
Pancreatic function
Time frame: 1 year
Mortality
Time frame: 1 year
Results and safety
The study was stopped early (reason given: Results from other studies did not support continuation of this trial). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Nov 2020.
Related trials for Pearson Syndrome
Enrolling by invitation
Investigation of the Treatment of Patients With Mitochondrial Defects by Assisted Reproductive Technology
Pearson Syndrome, Mitochondria DNA Deletion
RecruitingPhase 2
Evaluate the Safety and Therapeutic Effects of a Single Intravenous Infusion (IV) of Autologous CD34+ Cells Enriched With Allogenic Placenta-derived Mitochondria in Patients With a Diagnosis of Pearson Syndrome (PS)
Mitochondrial Diseases, Pearson Syndrome
RecruitingObservational study
Global Registry and Natural History Study for Mitochondrial Disorders
Mitochondrial Diseases, Kearns-Sayre Syndrome
RecruitingObservational study
North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)
Mitochondrial Disorders, Mitochondrial Genetic Disorders
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Matthew Klein, MD, FACS study chair
Edison Pharmaceuticals
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing EPI-743 in people with Pearson Syndrome, run by Edison Pharmaceuticals Inc at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 18 Nov 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Results from other studies did not support continuation of this trial). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Occurence of episodes of sepsis, metabolic crisis or hepatic faillure (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Nov 2020.