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Table 1 Statistically significant results from clinical studies

From: 10th European Conference on Rare Diseases & Orphan Products (ECRD 2020)

Outcome measureAuthor, yearInterventionResults (P-value)
RSBQPercy, et al. (2017) [5]TFT 200 mg/kg vs PLCClinical benefit was observed for patients treated by TFT (0.042T)
CGIPercy, et al. (2017) [5]TFT 200 mg/kg vs PLCClinical benefit was observed for patients treated by TFT (0.029T)
VASPercy, et al. (2017) [5]TFT 200 mg/kg vs PLCClinical benefit was observed for patients treated by TFT (0.025T)
O’Leary, et al. (2018) [6]PLC-MCS vs MCS-PLCWorsening of symptoms for patients treated by PLC-MCS (0.0211T; 0.0111W)
ADAMSO’Leary, et al. (2018) [6]PLC-MCS vs MCS-PLCWorsening of symptoms for patients treated by PLC-MCS (0.5535T; 0.0272W)
EEGO’Leary, et al. (2018) [6]PLC-MCS vs MCS-PLCWorsening of symptoms for patients treated by PLC-MCS (0.0208T; 0.0110W)
Gorbachevskaya, et al. (2001) [7]CL vs UTClinical benefit was observed for patients treated with CL: lower value of LRP in alpha and beta bands (< 0.001T; < 0.01T), higher levels of LRP in the theta band (< 0.001T)
Gorbachevskaya, et al. (2001) [7]Before CL vs after CLImprovement of the brain functional stage after treatment with CL: decrease of theta LRP in central and frontal regions (< 0.05T; < 0.01T), increase of beta activity LRP in the parietal region (< 0.05T), restoration of occipital alpha rhythm (< 0.05T)
ECGGuideri, et al. (2005) [8]ALC at BS vs ALC after 6 monthsClinical benefit was observed in patients treated with ALC: increase of total power (0.01T), VLF (0.01T), and LF (0.009T)
Guideri, et al. (2005) [8]UT at BS vs UT after 6 monthsDecrease in heart rate variability was observed in UT: decrease of total power (0.04T) and LF (0.05T), and increase of QTcD (0.01T)
Respiratory functionPercy, et al. (1994) [9]NLT vs PLCPositive effect of NLT was observed: higher awake min. O2 saturation value (0.03T), less % time spent with disorganized breathing (0.02T), higher end tidal carbon dioxide value (0.02T)
Djukic, et al. (2016) [10]Before glatiramer acetate 20 mg vs afterImprovement of respiratory function: decrease of breath hold index (0.004T; 0.03W) and breath hold time (0.007T; 0.004W)
Khwaja, et al. (2014) [11]Pre MAD MCS vs post OLEImprovement of respiratory function: improvement of apnea (0.012T)
CSSMaffei, et al. (2014) [12]ω-3 PUFAs at BS vs ω-3 PUFAs after 6 monthsSignificant improvements were observed: decrease in score for CSS (< 0.005A), ambulation (0.02A), hand use (0.002A), motor (0.009A), non-verbal communication (0.002A), and respiratory dysfunction (< 0.0001A)
  1. A, Analysis of variance; ADAMS, Anxiety, Depression, and Mood Scale; ALC, acetyl-L-carnitine; BS, baseline; CGI, Clinical Global Impression; CL, cerebrolysin; CSS, Clinical Severity Scale; ECG, electrocardiogram; EEG, electroencephalogram; LF, low-frequency component (range: 0.04–0.15 Hz); LRP, logarithm of relative spectral power; MAD, multiple ascending dose; MCS, mecasermin; MCS-PLC, mecasermin for the first treatment period, placebo for the second; NLT, naltrexone; OLE, open-label extension; PLC, placebo; PLC-MCS, placebo for the first treatment period, mecasermin for the second; PUFA, polyunsaturated fatty acids; QTcD, QTc dispersion (difference between the min. and max. heart rate-adjusted QT interval among the 12 ECG leads); RSBQ, Rett Syndrome Behavioural Questionnaire; T, student’s t-test; TFT, trofinetide; UT, untreated; VAS, visual analog scale; VLF, very low-frequency component (< 0.04 Hz); W, Wilcoxon signed-rank test