Keywords :
Tamoxifen; Child; Humans; Tamoxifen/therapeutic use; Muscular Dystrophy, Duchenne/drug therapy; Muscular Dystrophy, Duchenne; Neurology (clinical)
Funding text :
Despite this disappointing result, two positive conclusions can be drawn from this trial. First, academic investigators, supported by a broad community, were able to fund and conduct a large international trial to investigate the effect of a drug with no potential for financial gain. This trial is a massive endeavour, and the effort should be acknowledged. Second, Henzi and colleagues 5 report these negative results. The reporting of trials with negative findings is of utmost importance. Over the past 15 years, several promising results from phase 1 and phase 2 trials could not be reproduced in double-blind, placebo-controlled studies. 6 The trials investigating the antisense oligonucleotide drisapersen, the coenzyme Q-derived idebenone, and the nuclear factor κ-light-chain-enhancer of activated B cells inhibitor edasalonexent are examples. 6 Blaming clinical trial design or clinical outcome for negative results in phase 3 trials ignores that no outcome measure or clinical trial design will make a non-efficacious drug efficacious.
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