Close
A Phase III, trial of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Muscular Atrophy
| Sponsor / funder | ISIS PHARMACEUTICALS, INC. |
| CI | Professor Volker Straub, Professor Francesco Muntoni |
| sites | Newcastle, London GOSH |
| Contact |
Newcastle: becky.davis@newcastle.ac.uk |
| More information on the trial |
This is a randomised, double-blind, sham-procedure controlled study of intrathecal
administration of the drug (ISIS 396443) over 13 months.
Primary objective
To assess the clinical efficacy, safety, tolerability, and pharmacokinetics.
Recruitment
About 111 subjects randomized in a 2:1 ratio
- 74 patients to receive the drug by intrathecal lumbar puncture
- 37 control to receive a sham-procedure control.
A scaled equivalent dose of 12 mg of the drug will be given at each of 6 times (i.e., on Study Days 1, 15, 29, 64, 183, and 302).
Primary endpoint:
- time to death
- permanent ventilation: ≥16 hours ventilation/day continuously for >21 days in the absence of an acute reversible event OR tracheostomy