Biogen Idec has announced plans to run a phase 2 clinical study of the experimental drug ISIS-SMNRx in infants with spinal muscular atrophy (SMA)who are less than 6 weeks old and have a genetic diagnosis of the disorder but are not yet showing symptoms.
ISIS-SMNRx is being developed by Isis Pharmaceuticals in collaboration with Biogen Idec. Developed using a gene-modifying strategy known as antisense, it targets the SMN2 gene and encourages production of the full-length SMN protein that is needed but deficient in SMA-affected cells. It is hoped that administering this drug very early in life may prevent the development of SMA-associated weakness. MDA-funded laboratory research contributed to the development of ISIS-SMNRx.
ISIS-SMNRx is also being tested in ongoing, open Phase 3 trials in infants with SMA who are up to 7 months old and showing symptoms (ENDEAR), and in children with SMA who are 2-12 years old (CHERISH).
The new phase 2 exploratory clinical study – dubbed NURTURE – is being conducted in presymptomatic infants with a genetic SMA diagnosis to see whether early treatment with ISIS-SMNRx, before signs of SMA are evident, could delay or even prevent the development of the disease, as well as to further investigate the safety and tolerability of the drug.
In October, Isis announced positive results from phase 2 clinical trials of ISIS-SMNRx in infants and children with SMA
About the ISIS-SMNRx trial in infants without symptoms
All trial participants will receive the drug, which will be administered via a thin needle into the fluid surrounding the spinal cord ("intrathecally") in a procedure commonly referred to as a lumbar puncture. The babies will receive one injection every four months over approximately 2.5 years, for a total of about 10 injections. They'll be required to stay overnight in the hospital on the night of the first injection and to return to the study center the day after each later injection.
Participants will also need to return to the study center periodically during the course of the trial so that their health, safety and development can be monitored. In addition, study team members will contact families on a monthly basis to check on their child's health.
The trial is expected to include up to 25 infants from around the world, with participating sites at centers in the U.S., Taiwan and other countries throughout the world.
It may be possible to take part in this trial by relocating to a country where there is a study site, but legal or regulatory barriers to doing so may exist in some situations. Biogen Idec strongly recommends that families contact the desired study site to discuss their specific circumstances and potential eligibility before making any decisions about relocation.
Participants in the trial of ISIS-SMNRx for presymptomatic infants must:
Participants must not:
Note: This is not a complete list of study criteria.
For more information
For details and contact information for the presymptomatic infant study of ISIS-SMNRX (NURTURE) , see A Study of Multiple Doses of ISIS-SMNRx Delivered to Infants with Genetically Diagnosed and Presymptomatic SMA, or enter NCT02386553in the search box at ClinicalTrials.gov. For information about the symptomatic infant trial (ENDEAR), see A Study to Assess the Efficacy and Safety of ISIS-SMNRx in Infants With SMA, or enter NCT02193074 in the search box at ClinicalTrials.gov. For information about the trial in children ages 2-12 (CHERISH) , see A Study to Assess the Efficacy and Safety of ISIS-SMNRx in Patients with Later-Onset SMA, or enter NCT02292537 in the search box at ClinicalTrials.gov.
For more about ISIS-SMNRx, see ISIS-SMNRx on the Isis site.