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Objective  To measure the efficacy of mecasermin (recombinant human insulin‐like growth factor 1, rh IGF ‐1), for treating symptoms of Rett syndrome ( RTT ) in a pediatric population using a double‐blind crossover study design.    Methods  Thirty girls with classic  RTT  in postregression stage were randomly assigned to placebo or rh IGF ‐1 in treatment period 1 and crossed over to the opposite assignment for period 2 (both 20 weeks), separated by a 28‐week washout period. The primary endpoints were as follows: Anxiety Depression and Mood Scale ( ADAMS ) Social Avoidance subscale, Rett Syndrome Behaviour Questionnaire ( RSBQ ) Fear/Anxiety subscale, Parent Target Symptom Visual Analog Scale ( PTSVAS ) top three concerns, Clinical Global Impression ( CGI ), Parent Global Impression ( PGI ), and the Kerr severity scale. Cardiorespiratory‐ and electroencephalography ( EEG )‐based biomarkers were also analyzed.    Results  There were no significant differences between randomization groups. The majority of  AE s were mild to moderate, although 12 episodes of serious  AE s occurred. The Kerr severity scale,  ADAMS  Depressed Mood subscale, Visual Analog Scale Hyperventilation, and delta average power change scores significantly increased, implying worsening of symptoms. Electroencephalography ( EEG ) parameters also deteriorated. A secondary analysis of subjects who were not involved in a placebo recall confirmed most of these findings. However, it also revealed improvements on a measure of stereotypic behavior and another of social communication.    Interpretation  As in the phase 1 trial, rh IGF ‐1 was safe; however, the drug did not reveal significant improvement, and some parameters worsened.

Placebo‐controlled crossover assessment of mecasermin for the treatment of Rett syndrome