Please select the outcome measures that correspond to the clinical trial below.
Title: A Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Infantile-Onset Spinal Muscular Atrophy
Brief Summary: The primary objective is to examine the clinical efficacy of multiple doses of nusinersen (ISIS 396443) administered intrathecally to participants with Infantile-Onset Spinal Muscular Atrophy (SMA). The secondary objectives are to examine the safety and tolerability of multiple doses of nusinersen administered intrathecally to participants with infantile-onset SMA and to examine the cerebral spinal fluid (CSF) and plasma Pharmacokinetics (PK) of multiple doses of nusinersen administered intrathecally to participants with infantile-onset SMA.


Options:
A: Number of participants that experience Adverse Events (AEs) and Serious Adverse Events (Up to 88 Days); Number of participants with clinically significant neurological examination abnormalities (Up to 88 Days); Number of participants with clinically significant vital sign abnormalities (Up to 88 Days); Number of participants with clinically significant physical examination abnormalities (Up to 88 Days); Number of participants with clinically significant weight abnormalities (Up to 88 Days); Number of participants with clinically significant laboratory parameters (Up to 88 Days); Number or participants with clinically significant cerbrospinal fluid (CSF) laboratory parameters (Up to 88 Days); Number of participants with clinically significant electrocardiograms (ECGs) abnormalities (Up to 88 Days); Number of participants who use concomitant medications (Up to 88 Days); PK parameters of nusinersen (ISIS 396443): Maximum observed plasma drug concentration (Cmax) (Plasma at 1, 2, 4 and 20 hours after dosing); PK parameters of nusinersen: Time to reach maximum observed concentration (Tmax) (Plasma at 1, 2, 4 and 20 hours after dosing); PK parameters of nusinersen: Area under the plasma concentrations time curve from the time of the intrathecal (IT) dose to the last collected sample (AUCinf) (Plasma at 1, 2, 4 and 20 hours after dosing); PK parameters of nusinersen (ISIS 396443): Apparent terminal elimination half-life (t1/2), if possible (Plasma at 1, 2, 4 and 20 hours after dosing)
B: Number of participants that experience Adverse Events (AEs) and Serious Adverse Events (Up to 24 Weeks); Number of participants with clinically significant neurological examination abnormalities (Up tp 24 Weeks); Number of participants with clinically significant vital sign abnormalities (Up to 24 Weeks); Number of participants with clinically significant physical examination abnormalities (Up to 24 Weeks); Number of participants with clinically significant weight abnormalities (Up to 24 Weeks); Number of participants with clinically significant laboratory parameters (Up to 24 Weeks); Number of participants with clinically significant electrocardiograms (ECGs) abnormalities (Up to 24 Weeks); Number of participants who use concomitant medications (Up to 24 Weeks)
C: Percent of Participants Who Achieved Improvement in Motor Milestones as Assessed by Section 2 of the HINE at the Last Visit (Day 1352 or Early Termination)
D: Time to death or respiratory intervention (Up to Day 2891)

Answer: C