Trial · NCT01838941

Betaine and Peroxisome Biogenesis Disorders

NCT01838941, Phase 3, Completed, McGill University Health Centre/Research Institute of the McGill University Health Centre; results first posted 2016-06-28

3 sourcestrials-2026-11-12-0.5.0

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ConductScience Library. Betaine and Peroxisome Biogenesis Disorders. Trials, record NCT01838941. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01838941

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Trial

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

Title: Betaine and Peroxisome Biogenesis Disorders
NCT number: NCT01838941
Status: Completed
Phases: Phase 3
Enrollment: 12 participants, actual
Lead sponsor: McGill University Health Centre/Research Institute of the McGill University Health Centre
Conditions: Peroxisome Biogenesis Disorders
Interventions: Betaine
First posted: 2013-04-24
Last updated: 2016-06-28
Results first posted: 2016-06-28
Limitations and caveats: Large intra- and inter-individual variations in peroxisome functions, variation in phenotype severity, variation in age, number of participants; blood metabolite markers may be less sensitive than direct measurements of cell responses.

Source ClinicalTrials.gov

Registered outcomes2

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

List	Outcome	Time frame	Description
Primary	Peroxisome Biochemical Functions as Measured by Plasma Very Long Chain Fatty Acid	6 months	C26/C22 ratio in plasma is a recognized biomarker for very long chain fatty acid (normal range: 0.002-0.018). It was measured twice before the beginning of treatment and measured once at the end.
Secondary	Developmental Status	6 months	Denver Developmental Screening Test expressed in years and months.

Source ClinicalTrials.gov

Outcomes2

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Peroxisome Biochemical Functions as Measured by Plasma Very Long Chain Fatty Acid	6 months		ratio	MEAN
SECONDARY	Developmental Status	6 months	The Denver Developmental Screening Test was not performed.

Source ClinicalTrials.gov

Results by group2

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

Outcome	Group	Category	Value	Spread	Units
Peroxisome Biochemical Functions as Measured by Plasma Very Long Chain Fatty Acid	Betaine	Baseline	0.180	Full Range 0.027 to 0.301	ratio
Peroxisome Biochemical Functions as Measured by Plasma Very Long Chain Fatty Acid	Betaine	At 6 months	0.188	Full Range 0.059 to 0.286	ratio

Source ClinicalTrials.gov

Participant flow3

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

Period	Milestone	Group	Participants
Overall Study	STARTED	Betaine	12
Overall Study	COMPLETED	Betaine	10
Overall Study	NOT COMPLETED	Betaine	2

Source ClinicalTrials.gov

Adverse events17

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	Diarrhea	Gastrointestinal disorders	Betaine	2	12	3
other	Change in body odor (skin, urine, and/or breath)	Skin and subcutaneous tissue disorders	Betaine	3	12	4
other	Dry mouth	General disorders	Betaine	1	12	1
other	Croup	Respiratory, thoracic and mediastinal disorders	Betaine	1	12	1
other	Fatigue	General disorders	Betaine	3	12	3
other	Respiratory tract infection	Respiratory, thoracic and mediastinal disorders	Betaine	2	12	2
other	Gas	Gastrointestinal disorders	Betaine	1	12	1
other	Constipation	Gastrointestinal disorders	Betaine	4	12	4
other	Vomiting	Gastrointestinal disorders	Betaine	1	12	1
other	Ear infection/Otitis media	Ear and labyrinth disorders	Betaine	2	12	9

1 to 10 of 17Source ClinicalTrials.gov

See all 17 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Betaine
Total, other adverse events	Betaine	12	12
Total, serious adverse events	Betaine	0	12

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
20212125	BACKGROUND

Source 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 comparisons0

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

None in this release.

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.

For software

Read this trial from software.

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

GET https://conductscience.org/api/trials/v1/NCT01838941

GET https://conductscience.org/api/trials/v1/NCT01838941/registered

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