Trial · NCT01642056

EPI-743 for Metabolism or Mitochondrial Disorders

NCT01642056, Phase 1, Phase 2, Completed, National Human Genome Research Institute (NHGRI); results first posted 2021-04-14

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ConductScience Library. EPI-743 for Metabolism or Mitochondrial Disorders. Trials, record NCT01642056. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01642056

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: EPI-743 for Metabolism or Mitochondrial Disorders
NCT number: NCT01642056
Status: Completed
Phases: Phase 1, Phase 2
Enrollment: 20 participants, actual
Lead sponsor: National Human Genome Research Institute (NHGRI)
Conditions: Mitochondrial Disease, Neurology, Myopathy
Interventions: EPI-743, Placebo
First posted: 2012-07-17
Last updated: 2021-04-14
Results first posted: 2021-04-14
Limitations and caveats: All 20 randomized patients were included in the model to assess baseline characteristics. Of the 20 randomized, 19 provided 6-month outcomes (10 placebo, 9 treatment), who then crossed over their treatments and provided 8-month baseline scores. Of these, 16 provided 14-month outcomes (8 who had crossed over to treatment, and 8 who had crossed over to placebo).

Source ClinicalTrials.gov

Registered outcomes4

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Part I-III, Baseline	Baseline - Day 0	NPMDS is a semi-quantitative clinical rating scale that evaluates the progression of mitochondrial disease in pediatric patients. The NPMDS scale provides an assessment of the progression of mitochondrial disease. Scores from Parts I-III are added to get a value between 0 and 130, with higher scores representing more severe disease.
Primary	Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Part I-III, 6 Months	6 months	NPMDS is a semi-quantitative clinical rating scale that evaluates the progression of mitochondrial disease in pediatric patients. The NPMDS scale provides an assessment of the progression of mitochondrial disease. Scores from Parts I-III are added to get a value between 0 and 130, with higher scores representing more severe disease.
Primary	Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Part I-III, Post Washout	8 month - Post washout	NPMDS is a semi-quantitative clinical rating scale that evaluates the progression of mitochondrial disease in pediatric patients. The NPMDS scale provides an assessment of the progression of mitochondrial disease. Scores from Parts I-III are added to get a value between 0 and 130, with higher scores representing more severe disease.
Primary	Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Part I-III, 14 Months	14 months	NPMDS is a semi-quantitative clinical rating scale that evaluates the progression of mitochondrial disease in pediatric patients. The NPMDS scale provides an assessment of the progression of mitochondrial disease. Scores from Parts I-III are added to get a value between 0 and 130, with higher scores representing more severe disease.

Source ClinicalTrials.gov

Outcomes4

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Part I-III, Baseline	Baseline - Day 0	Participants who who had treatment intervention in phase I. The outcome measures were analyzed separately by arm and phase, not by combining results by intervention across phases, due to the progressive nature of the underlying disease.	Units on a scale	MEAN
PRIMARY	Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Part I-III, 6 Months	6 months	(Participants who who had treatment intervention in phase I. The outcome measures were analyzed separately by arm and phase, not by combining results by intervention across phases, due to the progressive nature of the underlying disease.	Units on a scale	MEAN
PRIMARY	Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Part I-III, Post Washout	8 month - Post washout	Participants who received treatment in phase II. The outcome measures were analyzed separately by arm and phase, not by combining results by intervention across phases, due to the progressive nature of the underlying disease.	Units on a scale	MEAN
PRIMARY	Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Part I-III, 14 Months	14 months	Participants who received treatment in phase II. The outcome measures were analyzed separately by arm and phase, not by combining results by intervention across phases, due to the progressive nature of the underlying disease.	Units on a scale	MEAN

Source ClinicalTrials.gov

Results by group8

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Part I-III, Baseline	EPI-743, Then Placebo	23.9	Standard Error 2.8	Units on a scale
Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Part I-III, Baseline	Placebo, Then EPI-743	20.4	Standard Error 3.7	Units on a scale
Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Part I-III, 6 Months	EPI-743, Then Placebo	27.1	Standard Error 2.7	Units on a scale
Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Part I-III, 6 Months	Placebo, Then EPI-743	20.2	Standard Error 3.8	Units on a scale
Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Part I-III, Post Washout	EPI-743, Then Placebo	27.1	Standard Error 3.5	Units on a scale
Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Part I-III, Post Washout	Placebo, Then EPI-743	18.3	Standard Error 3.6	Units on a scale
Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Part I-III, 14 Months	EPI-743, Then Placebo	26	Standard Error 3.3	Units on a scale
Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Part I-III, 14 Months	Placebo, Then EPI-743	15.8	Standard Error 3.7	Units on a scale

Source ClinicalTrials.gov

Participant flow12

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
First Intervention	STARTED	EPI-743, Then Placebo	10
First Intervention	STARTED	Placebo, Then EPI-743	10
First Intervention	COMPLETED	EPI-743, Then Placebo	9
First Intervention	COMPLETED	Placebo, Then EPI-743	10
First Intervention	NOT COMPLETED	EPI-743, Then Placebo	1
First Intervention	NOT COMPLETED	Placebo, Then EPI-743	0
Second Intervention	STARTED	EPI-743, Then Placebo	9
Second Intervention	STARTED	Placebo, Then EPI-743	10
Second Intervention	COMPLETED	EPI-743, Then Placebo	8
Second Intervention	COMPLETED	Placebo, Then EPI-743	8

1 to 10 of 12Source ClinicalTrials.gov

See all 12 participant flow

Adverse events108

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Anaemia	Blood and lymphatic system disorders	EPI-743	0	20
other	Anaemia	Blood and lymphatic system disorders	Placebo	2	19	3
other	Polycythaemia	Blood and lymphatic system disorders	EPI-743	2	20	3
other	Polycythaemia	Blood and lymphatic system disorders	Placebo	5	19	5
other	Tinnitus	Ear and labyrinth disorders	EPI-743	0	20
other	Tinnitus	Ear and labyrinth disorders	Placebo	1	19	1
other	Adrenal insufficiency	Endocrine disorders	EPI-743	1	20	1
other	Adrenal insufficiency	Endocrine disorders	Placebo	0	19
other	Goitre	Endocrine disorders	EPI-743	1	20	1
other	Goitre	Endocrine disorders	Placebo	0	19

1 to 10 of 108Source ClinicalTrials.gov

See all 108 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	EPI-743	1	20
Total, all-cause mortality	Placebo	0	19
Total, other adverse events	EPI-743	17	20
Total, other adverse events	Placebo	18	19
Total, serious adverse events	EPI-743	3	20
Total, serious adverse events	Placebo	4	19

Source ClinicalTrials.gov

Papers2

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
21600768	BACKGROUND	PubMed
22410442	BACKGROUND	PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
EPI-743 vs Placebo: deaths from any cause	Trial comparisons
EPI-743 vs Placebo: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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The record and each of its lists are JSON, twenty rows a page. No key.

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