What question did this study set out to answer?

To assess the efficacy and safety of onasemnogene abeparvovec in presymptomatic infants genetically at risk for spinal muscular atrophy type 1.

June 17, 2022Nature MedicineOpen Access

Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial

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Authors

KSKevin A. StraussClinic for Special Children MFMichelle A. FarrarBoston Children's Hospital FMFrancesco MuntoniUniversity of London

Discussion

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Implication

Phase III trial demonstrates independent sitting and survival in presymptomatic infants with spinal muscular atrophy, highlighting the urgency of universal newborn screening.

Key Points

To assess the efficacy and safety of onasemnogene abeparvovec in presymptomatic infants genetically at risk for spinal muscular atrophy type 1.
Phase III, multicenter, single-arm interventional trial (SPR1NT; NCT03505099) evaluating onasemnogene abeparvovec in presymptomatic infants (N = 14) with biallelic SMN1 mutations and two copies of SMN2 treated at ≤6 weeks of life.
Outcomes were compared with a historical control cohort of untreated infants (n = 23) from the Pediatric Neuromuscular Clinical Research natural-history study.
All 14 infants (100%) achieved independent sitting for ≥30 seconds at any visit up to 18 months (Bayley-III item #26; P < 0.001), with 11 doing so within the normal developmental age window.
All 14 infants (100%) met the endpoint of survival without permanent ventilation at 14 months, 13 maintained body weight at or above the 3rd WHO percentile through 18 months, and none required respiratory or nutritional support.
No serious adverse events were considered related to the study treatment by investigators.

Cite This Study

Strauss et al. (2022) studied this question.

synapsesocial.com/papers/69ffdd7eda5c1eb07f2d8d51 https://doi.org/10.1038/s41591-022-01866-4

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June 17, 2022Nature MedicineOpen Access

Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial

View Full Paper

Ask AI

Bookmark

Authors

KSKevin A. StraussClinic for Special Children MFMichelle A. FarrarBoston Children's Hospital FMFrancesco MuntoniUniversity of London

Discussion

Member takes

Implication

Phase III trial demonstrates independent sitting and survival in presymptomatic infants with spinal muscular atrophy, highlighting the urgency of universal newborn screening.

Key Points

To assess the efficacy and safety of onasemnogene abeparvovec in presymptomatic infants genetically at risk for spinal muscular atrophy type 1.
Phase III, multicenter, single-arm interventional trial (SPR1NT; NCT03505099) evaluating onasemnogene abeparvovec in presymptomatic infants (N = 14) with biallelic SMN1 mutations and two copies of SMN2 treated at ≤6 weeks of life.
Outcomes were compared with a historical control cohort of untreated infants (n = 23) from the Pediatric Neuromuscular Clinical Research natural-history study.
All 14 infants (100%) achieved independent sitting for ≥30 seconds at any visit up to 18 months (Bayley-III item #26; P < 0.001), with 11 doing so within the normal developmental age window.
All 14 infants (100%) met the endpoint of survival without permanent ventilation at 14 months, 13 maintained body weight at or above the 3rd WHO percentile through 18 months, and none required respiratory or nutritional support.
No serious adverse events were considered related to the study treatment by investigators.

Cite This Study

Strauss et al. (2022) studied this question.

synapsesocial.com/papers/69ffdd7eda5c1eb07f2d8d51 https://doi.org/10.1038/s41591-022-01866-4

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