SMA Clinical Trial NCT02268552
Spinal Muscular Atrophy (SMA) is a rare genetic disease. It affects approximately 1 in 6,000 to 10,000 babies born worldwide each year. Novartis Pharma AG is sponsoring a clinical research trial for babies diagnosed with SMA type I, the most common and most severe form of SMA. The research study is conducted by study physicians in over four countries and currently needs volunteer patients.
The investigational medication, LMI070, is being studied as a possible treatment for newly diagnosed SMA type I patients. LMI070 has so far only been tested in animals. It is currently unknown whether LMI070 is effective. This study is part of the process of learning whether or not LMI070 is an effective treatment in babies. The study involves doses of LMI070 over an initially planned thirteen-week period.
Patients who participate in this trial:
- Are up to 8 months of age at enrolment
- Have SMA type I (confirmed by a genetic test)
- Have 2 copies of the SMN2 gene
- Were born after the 32nd week of gestation
- Are able to lift forearms actively off bed
- Live within 2 hours drive of the study site (other distances should be discussed with the study site)
- Have not participated in other studies where a trial medication was received in the last 14 days.
Patients enrolled in this trial:
- Will attend investigator visits, at least weekly, for a minimum of 13 weeks
- Extended treatment may be available if the study doctor and Novartis feel it is safe and in the best interest of your baby
- Will participate at no additional cost
- May withdraw from the trial at any time.
If you are interested in learning more about this research study, please contact the nearest study location.
Additional study physicians may be listed in the future. You can also share this information via the links at the top of the page.