Tier 2 — strong

A therapeutic trial of pro-methylation dietary supplements in Angelman syndrome

Lynne M Bird, Wen-Hann Tan, Carlos A Bacino, Sarika U Peters, Steven A Skinner, Irina Anselm, Rene Barbieri-Welge, Astrid Bauer-Carlin, Jennifer K Gentile, Daniel G Glaze, Lucia T Horowitz, K Naga Mohan, Mark P Nespeca, Trilochan Sahoo, Dean Sarco, Susan E Waisbren, Arthur L Beaudet
American journal of medical genetics. Part A 2011 155A(12):2956-2963

Bibliography

PubMed
PMID 22002941
PubMed Central
PMC3222728
DOI
10.1002/ajmg.a.34297
Funding
Reported funding/support: National Institute of Child Health & Human Development : NICHDU54 HD061222-08 || HDNational Institute of Child Health & Human Development : NICHDU54 HD061222-06 || HD
Competing interests
A competing-interest declaration was not available in the indexed record or accessible disclosure metadata.

Study snapshot

DesignOpen-label trial with historical controls
ModelHuman participants
Sample90 enrolled children; 65 completed
InterventionBetaine, metafolin, creatine and vitamin B12
Duration1 year
EndpointsDevelopment; DNA methylation; Adverse events

What the study showed, in plain terms

Published in 2011, this source examined brain health and combination interventions. Design: Open-label trial with historical controls. Sample/model: 90 enrolled children; 65 completed. The exposure or intervention was Betaine, metafolin, creatine and vitamin B12. Follow-up or treatment period: 1 year.

Developmental performance and measured methylation did not significantly improve. Ten adverse events led to seven withdrawals.

A multi-supplement regimen and historical control. Withdrawal events mean the study should not be summarized as proving universal safety.

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Key findings

  • Developmental performance and measured methylation did not significantly improve.
  • Ten adverse events led to seven withdrawals.
  • The recorded outcome domains were Development; DNA methylation; Adverse events.
  • The result applies to the tested exposure: Betaine, metafolin, creatine and vitamin B12; 1 year.

What this study can and cannot tell us

  • A multi-supplement regimen and historical control. Withdrawal events mean the study should not be summarized as proving universal safety.
  • Without a sufficiently blinded concurrent control, time effects, expectations, co-interventions and regression toward the mean can explain part of an apparent improvement.
  • Source basis: Accessible full-text extract plus indexed bibliographic data. Missing method details, funding information or participant denominators are not inferred.

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Editorial review

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Dr. Khaled Mahmoud, MBBChCritical Care Medicine, Longevity Science & Editorial Direction Dr. Amr Yasser, PhDCardiometabolic, Musculoskeletal, Gut & Bioavailability Dr. Abdulla Mostafa, PhDCellular Senescence, Neurological, Mitochondrial & Brain-Ageing Biology