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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Bibliography
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Mendell, JR, Sahenk, Z, Lehman, K, et al. Assessment of systemic delivery of rAAVrh74.MHCK7.micro-dystrophin in children with Duchenne muscular dystrophy: a nonrandomized controlled trial. JAMA Neurol 77 (2020), 1122–1131.
Botti, V, Menzel, O, Staedler, D, A state-of-the-art review of tamoxifen as a potential therapeutic for Duchenne muscular dystrophy. Front Pharmacol, 13, 2022, 1030785.
Tsabari, R, Simchovitz, E, Lavi, E, et al. Safety and clinical outcome of tamoxifen in Duchenne muscular dystrophy. Neuromuscul Disord 31 (2021), 803–813.
Henzi, BC, Schmidt, S, Nagy, S, et al. Safety and efficacy of tamoxifen in Duchenne muscular dystrophy (TAMDMD): a multicentre, randomised, doucle-blind, placebo-controlled, phase 3 trial. Lancet Neurol 22 (2023), 890–899.
Markati, T, De Waele, L, Schara-Schmidt, U, et al. Lessons Learned from discontinued clinical developments in Duchenne muscular dystrophy. Front Pharmacol, 12, 2021, 735912.
Livingstone, A, Servais, L, Wilkinson, DJC, Crowdfunding for neuromuscular disease treatment: the ethical implications. Lancet Neurol 20 (2021), 788–789.
Muntoni, F, Signorovitch, J, Sajeev, G, et al. Real-world and natural history data for drug evaluation in Duchenne muscular dystrophy: suitability of the North Star Ambulatory Assessment for comparisons with external controls. Neuromuscul Disord 32 (2022), 271–283.
Wang, S, Kidwell, KM, Roychoudhury, S, Dynamic enrichment of Bayesian small-sample, sequential, multiple assignment randomized trial design using natural history data: a case study from Duchenne muscular dystrophy. Biometrics, 2023 published online June 15. https://doi.org/10.1111/biom.13887.
Servais, L, Yen, K, Guridi, M, et al. Stride velocity 95th centile: insights into gaining regulatory qualification of the first wearable-derived digital endpoint for use in Duchenne muscular dystrophy. Trials J Neuromuscul Dis 9 (2022), 335–346.
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