Table 2 Treatment-emergent adverse events of special interest (safety population)

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

Category of AESI preferred term

SPR1NT two-copy cohort (n = 14b) (%)

Hepatotoxicity

Any TEAE

3 (21)

Aspartate aminotransferase increased

3 (21)

Alanine aminotransferase increased

1 (7)

Gamma-glutamyltransferase increased

1 (7)

Thrombocytopenia

Any TEAE

3 (21)

Thrombocytopenia

1 (7)

Vessel puncture site bruise

1 (7)

Platelet count decreased

1 (7)

Cardiac adverse events

Any TEAE

2 (14)

Blood creatine phosphokinase MB increased

1 (7)

Blood creatine phosphokinase increased

1 (7)

Troponin increased

1 (7)

Sensory abnormalities suggestive of ganglionopathy

Any TEAE

3 (21)

Areflexia

2 (14)

Hyporeflexia

1 (7)

Thrombotic microangiopathya

Any TEAE

2 (14)

Thrombocytopenia

1 (7)

Platelet count decreased

1 (7)

  1. AESI, adverse event of special interest; TEAE, treatment-emergent adverse event.
  2. No TEAE representing thrombotic microangiopathy was identified.
  3. aThrombocytopenia and platelet count decreased are TEAEs that also fall under the thrombotic microangiopathy AESI category.
  4. bSafety population, n = 4 males and n = 10 females; mean (s.d.) age at dosing, 20.6 (7.9) days.
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