Micallef 2009.
Methods | Randomised, placebo‐controlled, double‐blind clinical trial | |
Participants | 179 participants with CMT1A aged 34 to 58 years, genetically confirmed, both sexes | |
Interventions | 1 g/day ascorbic acid (N = 56), 3 g/day ascorbic acid (N = 61), versus matching placebo (N = 62) for 12 months | |
Outcomes | Primary: change in CMTNS after 12 months Secondary: change in CMTES, quantitative motor testing, SF‐36, ODSS, time to walk 10‐m, VAS, Clinical Global Impression Severity scale, ascorbic acid blood concentration, nerve conduction studies after 12 months |
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Funding sources | "French Ministry of Health (National PHRC 2004) and Association Française Contre les Myopathies." (p. 1) | |
Conflicts of interest | "Ascorbic acid for the treatment of CMT1A is under patent and its application has been registered by INSERM, AFM, and Marseille II University. MF is one of the inventors of the patent, which has been licensed to Murigenetics. MF and OB participate in the activities of Murigenetics as scientific advisers and are each 15% shareholders in the company. The other authors have no conflict of interest." (p. 8) | |
Notes | Date study was performed: recruitment from September 2005 to September 2007 | |
Risk of bias | ||
Bias | Authors' judgement | Support for judgement |
Random sequence generation (selection bias) | Low risk | "The randomisation sequence was generated using randomization.com" |
Allocation concealment (selection bias) | Unclear risk | "Active treatment and placebo were prepared by CLIPA Clinical Packaging." |
Blinding of participants and personnel (performance bias) All outcomes | Low risk | "Both patients and investigators were unaware of the treatment allocation." |
Blinding of outcome assessment (detection bias) All outcomes | Low risk | "Both patients and investigators were unaware of the treatment allocation." |
Incomplete outcome data (attrition bias) All outcomes | Low risk | "Analyses were done on the intention to treat population." |
Selective reporting (reporting bias) | Unclear risk | Study protocol not available |
Other bias | Low risk | None identified |