ESPE2022 Poster Category 1 Growth and Syndromes (85 abstracts)
Global Increlex® Registry, post-authorisation surveillance registry monitoring the long-term safety and effectiveness of mecasermin in children and adolescents with Severe Primary IGFD (SPIGFD): objectives and study design
Peter Bang 1 , Sarah Mazain 2 , Valérie Perrot 2 & Caroline Sert 2
1Division of Pediatrics, Department of Biomedical and Clinical Sciences, Faculty of Health Sciences, Linköping University, Linköping, Sweden; 2Ipsen Pharma, Boulogne-Billancourt, France
Background: Severe primary insulin-like growth factor-1 deficiency (SPIGFD) is a rare growth disorder characterised by severe short stature. Replacement therapy with recombinant human IGF-1 (rhIGF-1; mecasermin [Increlex®]) is currently the only available treatment for children/adolescents with SPIGFD. We describe the methodology of the Global Increlex Patient Registry (NCT00903110), designed to monitor long-term safety and effectiveness of mecasermin during treatment and for a minimum of five years following end of treatment and/or until adult height is reached according to locally approved product information.
Methods: The registry is a multicenter, observational, open-label study ongoing in Europe from 2008, and has transitioned to a global registry with the inclusion of USA study sites. Eligible patients receive 40-120 µg/kg (0.04-0.12 mg/kg) mecasermin, twice daily, as prescribed by their physician. Table 1 presents outcome measures. Target enrollement is 500 patients.
| Primary: |
| During the treatment period, up to 30 days after last dose: • Incidence of serious adverse events (SAEs)a, all adverse events (AEs), targeted AEs, deaths and withdrawals due to AEs |
| Secondary: |
| Within five years post treatment: • Incidence of SAEsa and all AEs, targeted AEs, deaths and withdrawals due to AEs in the overall population, and in children and adolescents exposed to mecasermin for at least three cumulative years excluding interruptions |
| During the treatment period, within five years post treatment: • Incidence of special situations and concomitant medications |
| From baseline at least up to 5 years or until final adult height (FAH) is achieved: • Changes in height Standard Deviation Score (HtSDS) • Height velocity • Bone age development • Body mass index • Pubertal stage • Estimated differences between predicted adult height and FAH • Modeling to identify predictive factors of HtSDS change, height velocity, FAH, pubertal (Tanner) stage and bone age development |
| Periodically assessed until treatment stop: • Dose of mecasermin administrated • Duration of mecasermin exposure |
| Throughout the study: • Biochemical assessments: baseline growth hormone, IGF-1 and IGF binding protein-3 levels and binding proteins • Presence/absence of gene deletion/mutation |
| At baseline, year one, end of treatment and at FAH: • Changes in quality of life assessment using EQ-5D in patients aged four and over |
| At different timeframes (e.g. first three years after treatment initiation, three-five years and over five years): • Description of neoplasia (benign/malignant) and hypoglycaemia |
| aIncluding AEs of special interest of neoplasia |
Conclusions: The registry will allow for characterisation of the risk-benefit profile of mecasermin in patients presenting with SPIGFD in real-life settings.
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