Please note, throughout this website, when discussing “SMA”, we are referring to 5q SMA, the most common form of the disease.
What is NURTURE1
STUDY DESCRIPTION 1,3
NURTURE (Study CS5) is a Phase II, open-label study to evaluate SPINRAZA in pre-symptomatic infants genetically diagnosed with SMA. Patients in this study were deemed most likely to develop Type I or Type II SMA and were ≤ 6 weeks of age at first dose.
In the March 2019 interim analysis, the median age at first dose was 22 days.
The median time on study at last visit was 33.9 months. 25 patients were involved in the analysis; 15 had 2 SMN2 copies and 10 had 3 SMN2 copies. The primary endpoint was time to death or respiratory intervention (≥6 hours /day continuously for ≥7 consecutive days or tracheostomy). The median time to respiratory intervention could not be estimated due to too few events. Four (16%) infants (all with two SMN2 copies) utilised respiratory intervention for ≥6 hours per day continuously for ≥7 days, all of whom initiated respiratory intervention during an acute, reversible illness.
What are the key findings from the NURTURE
interim analysis (March 2019)1
interim analysis (March 2019)1
In the NURTURE interim analysis (March 2019):
References
1. De Vivo DC et al., Neuromuscular Disorders https://doi.org/10.1016/j.nmd.2019.09.007
2. NICE. Managed Access Agreement (Draft). Nusinersen SPINRAZA) for the treatment of 5s spinal muscular atrophy. Available from: https://www.nice.org.uk/guidance/ta588/documents/committee-papers-5 [Last accessed October 2019]
3. SPINRAZA Summary of Product Characteristics.
Adverse events should be reported. For Ireland, reporting forms and information can be found at www.hpra.ie. For the UK, reporting forms and information can be found at yellowcard.mhra.gov.uk or via the Yellow Card app available from the Apple App Store or Google Play Store. Adverse events should also be reported to Biogen Idec Ltd on 1800 812 719 in Ireland and 0800 008 7401 in the UK.