A Study to Assess the Safety, Tolerability, and Pharmacokinetics of Multiple Doses of IONIS 396443 Delivered Intrathecally to Patients With Infantile-Onset Spinal Muscular Atrophy
This study will test the safety, tolerability, and pharmacokinetics of multiple doses of IONIS-SMNRx administered into the spinal fluid three times over the duration of the trial, in patients with Infantile-Onset Spinal Muscular Atrophy.
Stanford is now accepting new patients for this trial.
- drug : IONIS-SMNRx
Phase: Phase 2
Ages Eligible For Study:
- Genetic documentation of 5q SMA (homozygous gene deletion or mutation) - Onset of clinical signs and symptoms consistent with SMA at ? 21 days and <6 months (180 days) of age - At study entry, receiving adequate nutrition and hydration (with or without gastrostomy), in the opinion of the Site Investigator - Body weight >5th percentile for age using CDC guidelines - Medical care meets and is expected to continue to meet guidelines set out in the Consensus Statement for Standard of Care in SMA (Wang et al. 2007), in the opinion of the Site Investigator - Gestational age of 35 to 42 weeks and gestation body weight ?2 kg - Reside within approximately 9 hours ground-travel distance from a participating study center for the duration of the study. Residence >2 hours ground-travel distance from a study center must obtain clearance from the Site Investigator and the study Medical Monitor - Able to complete all study procedures, measurements and visits and parent or guardian/subject has adequately supportive psychosocial circumstances, in the opinion of the Site Investigator