Chop intend motor function

The childrens hospital of philadelphia infant test of neuromuscular disorders chop intend may be used to evaluate the motor skills of infants with sma 10,11. Mar 24, 2020 in str1veus, patients achieved rapid and sustained improvement in motor function unseen in natural history. The chopintend was the obvious motor outcome for avxs101 study due to its published reliability and. The chop intend is a reliable measure of motor skills in patients with smai and neuromuscular disorders presenting in infancy. Table 4 relative treatment effects with avxs101 versus nusinersen regarding motor function i. Chop intend was developed by evaluating infants n26 with type i sma, mean age 11. Pdf the childrens hospital of philadelphia infant test.

Singledose genereplacement therapy for spinal muscular. The chop intend is a reliable measure of motor skills in patients with smai. Subjects were evaluated as part of a multicenter natural history study. Genereplacement therapy for sma1 may necessitate new rating. New data presented at mda clinical conference show benefit in. The childrens hospital of philadelphia infant test of neuromuscular disorders chop intend may be used to evaluate the motor skills of infants with sma10. Biogen presents data at 2019 aan annual meeting affirming. The chop infant test of neuromuscular disorders chop intend 3536 gross motor function measure gmfm38,39 timed tests of function time to walkrun 30 feet or 10 meters. Evaluate the utility of the infant test of neuromuscular development chopintend to quantify change in infants participating in avxs101 gene replacement therapy trial nct021229521.

Risdiplam keeps showing survival, motor function improvements. Singledose genereplacement therapy for spinal muscular atrophy. Treatment of infantileonset spinal muscular atrophy with. Impact of age and motor function in a phase 12a study of infants. Baseline day 1 to month 12 and 24 proportion of participants who achieve a score of 40 or higher, 50 or higher and 64 in the chop intend motor function scale time frame. Results showed that treatment with risdiplam rg7916 in patients with spinal muscular atrophy were able to improve scores in a scale that measures motor function known as chopintend.

Avxs101, the food and drug administrationapproved therapy for spinal muscular atrophy sma, yields rapid, sustained improvements in chop intend scores, better survival, and motor function improvements at longterm followup, according to an analysis presented at the annual meeting of the child neurology society. A score of 40 or greater is rarely observed in untreated infants. Chicagothe childrens hospital of philadelphia infant test of neuromuscular disorders chopintend was designed to track motor function in patients with spinal muscular atrophy type 1 sma1, but the test may not be sensitive to advances in motor function made by patients who receive an investigational genereplacement therapy, researchers said at the 47th annual meeting of the child neurology society. Oct 07, 2019 chopintend, a measure for assessing motor skills of sma type 1 infants, uses a 064point scale, in which higher scores indicate better motor function. Chop intendchildrens hospital of philadelphia infant test of neuromuscular disorders a highdose cohort n12 in start. By six months, essentially no untreated patients with sma1 achieve a score of 40 or greater points on the chopintend, and scores may decrease between age 6 and 12 months. Motor function treatment effect was assessed by a chopintend response, defined as an increase of. The test was developed following the evaluation of 26 infants with smai mean age 11. Childrens hospital of philadelphia infant test of neuromuscular disorders source. Assessment of function in sma spinal muscular atrophy. The chop intend was assessed until a participant reached a maximum score of 64, after which it was not assessed. Many continue to improve and maintain these scores beyond a point in time at which untreated individuals with type 1 sma would experience a significant decline.

The motor skills of patients with spinal muscular atrophy, type i. The face validity of the chop intend is supported by the use of an expert panel in item selection. The childrens hospital of philadelphia infant test of. The longterm effects of spinraza were evaluated in cs2cs12 over a 3year period 1050 days in 28 individuals with lateronset types 2 and 3 sma ranging from 2 to 16 years old at the time of first dose. Head in midline with visual stimulation supine head midline visual stimulation is given with toy. Here we demonstrate its potential utility of a sub set of chop intend items for a series of individuals with type 2 sma with scores on the expanded hammersmith functional motor scale hfmse of 2 or less. Nusinersen versus sham control in infantileonset spinal.

May 06, 2019 interim results from the endearshine openlabel extension study of infants n89 followed for up to four years demonstrated that treatment with spinraza resulted in additional or new motor function improvements on the childrens hospital of philadelphia infant test of neuromuscular disorders chop intend. The chop intend can provide a useful measure of motor skill in this population both for clinical monitoring and for research trials. Genereplacement therapy for sma1 may necessitate new. Chop intend scores and age were significantly correlated r. Table 4 survival, motor function, and motor milestones. Oct 17, 2018 chopintend uses a 064point scale where higher scores indicate better motor function. Disorders chopintend was designed to track motor function in. Chop intend can be used for a number of neuromuscular disorders, including sma. May, 2019 to investigate whether age at dosing and baseline motor function were associated with any difference in motor function improvements, we characterized the chopintend changes in infants participating in the avxs101 gene replacement therapy trial, in particular the response of patients with low motor function when dosed at an early age. While it can assess motor function in older people with sma, it was designed for infants with less functional ability. Validated, 16 items, 64point scale shown reliable in sma type i subjects derived in part from timp test of infant motor performance designed to measure motor function in. Mar 12, 2018 nurture participants also achieved a mean chop intend score, which measures general motor function among infants with sma, of 58. Validation of the childrens hospital of philadelphia infant. Motor function test reliability during the neuronext spinal.

Adapted from the test of infant motor performance, campbell, sk. Expanded hammersmith functional motor scale33,34 the test of infant motor performance screening items timpsi35 the chop infant test of neuromuscular disorders chop intend3536 gross motor function measure gmfm38,39 timed tests of function time to walkrun 30 feet or 10 meters. Understand the childrens hospital of philadelphia infant test of neuromuscular disorders chop intend chop intend was specifically developed to assess motor function in patients with sma. Place head in maximum available rotation and provide visual stimulation to encourage midline head falls to side, no attempts to regain midline. The chop intend may be more sensitive to changes in gross motor function and muscle strength in individuals with type 2 sma due to a floor effect on hfmse, and may be a reliable measure for longterm follow up. Currently, the test of infant motor performance timp and the alberta infant. Items for the chop intend were selected by an expert panel based on item mean and. Dec 19, 2018 change from baseline score in the childrens hospital of philadelphia infant test of neuromuscular disorders chop intend motor function scale time frame. Jun 19, 2018 results showed that treatment with risdiplam rg7916 in patients with spinal muscular atrophy were able to improve scores in a scale that measures motor function known as chopintend.

Comparison of avxs101 relative to nusinersen for the treatment of infants with spinal muscular atrophy type 1. Motor function tests selected for the sma infant biomarker study included the timpsi, chopintend, and the aims. Chop intend is a 16item, 64point motor assessment developed to evaluate motor skills in infants with sma type i, with higher scores indicating better motor function 30,31. Further research is needed to establish the sensitivity of the measure to change over time and to further establish its validity as a measure of motor function in the target population. Impact of age and motor function in a phase 12a study of. Designed to measure motor function in weak infants. Zolgensma data shows rapid, significant, clinically. Clinical efficacy assessments included event free survival, and change from baseline of two assessments of motor function. Jan 30, 2018 read how infants with spinal muscular atrophy have improved motor function after longer spinraza treatment, and spend less time hospitalized, according to data presented at a jan. Patients with sma1 also do not achieve major milestones in motor function and have a decline in function, as measured on the chop intend childrens hospital of philadelphia infant test of. Sma1 is a neurodegenerative disease that results in motor function decline.

Assessing upper limb function in nonambulant sma patients. Mar 16, 2019 despite some differences in clinical trial design and study outcomes, clinical trials of avxs101 and nusinersen in symptomatic infants with sma type 1 both assessed eventfree survival i. All three mfts assess spontaneous movement, postural control, and gravity eliminated and antigravity strength in a variety of postures and positions. Chop intend manual of procedures testing environment. Avxs101 may result in longterm motor improvements in sma. The chopintend is a functional scale shown to reliably assess motor function in patients with sma1 and other neuromuscular disorders presenting in infancy. The definitions of response with respect to hine2 and chop intend were selected to measure deviations from the expected trajectory of motor function decline that was established in published. Mfm has been used to evaluate patients from 260 years old. A study of risdiplam in infants with genetically diagnosed. Apr 10, 2018 the chop intend is a validated motor outcome measure developed for weak infants with type 1 sma.

Infant motor function improves with longer spinraza treatment. Nusinersen initiated in infants during the presymptomatic. Pdf the childrens hospital of philadelphia infant test of. Longitudinal assessment to evaluate the sensitivity of a subset of the chop intend to detect change. Ptc therapeutics surges on positive outcome from rare disease. Respiratory support and chop intend scores were correlated r. In str1veus, patients achieved rapid and sustained improvement in motor function unseen in natural history. Ptc therapeutics surges on positive outcome from rare.