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Intracerebroventricular delivery of vascular endothelial growth factor in patients with amyotrophic lateral sclerosis, a phase I study
| Content Provider | Oxford Academic |
|---|---|
| Author | Van Damme, Philip Tilkin, Petra Mercer, Katarina Jansson Terryn, Joke D’Hondt, Ann Herne, Nina Tousseyn, Thomas Claeys, Kristl G Thal, Dietmar R Zachrisson, Olof Almqvist, Per Nuttin, Bart Jerling, Markus Bernadotte, Folke Haegerstrand, Anders Robberecht, Wim |
| Copyright Year | 2020 |
| Abstract | We studied the feasibility, safety, tolerability and pharmacokinetics of intracerebroventricular delivery of recombinant human vascular endothelial growth factor in patients with amyotrophic lateral sclerosis. In this phase I study in patients with amyotrophic lateral sclerosis, the study drug was delivered using an implantable programmable pump connected to a catheter inserted in the frontal horn of the lateral cerebral ventricle. A first cohort received open label vascular endothelial growth factor (0.2, 0.8 and 2 µg/day), a second cohort received placebo, 0.8 or 2 µg/day of study dug. After the 3-month study period, all patients could participate in an open label extension study. In total, 18 patients with amyotrophic lateral sclerosis, seen at the University Hospitals in Leuven were included. The surgical procedure was well tolerated in most patients. One patient had transient postoperative seizures, due to an ischemic lesion along the catheter tract. The first 3-month study period was completed by 15/18 patients. Administration of 2 µg/day vascular endothelial growth factor resulted in sustained detectable levels in cerebrospinal fluid. A pulmonary embolus occurred in 3 patients, in 1 patient in the first 3-month study, and in 2 patients during the open label extension study. The study drug was well tolerated in the other patients, for up to 6 years in the open label extension study. Our study shows that intracerebroventricular administration of 2 µg/day of vascular endothelial growth factor to patients with amyotrophic lateral sclerosis is feasible, results in detectable cerebrospinal fluid levels and is well tolerated in most patients. The most common serious adverse event was a pulmonary embolus. |
| Related Links | https://academic.oup.com/braincomms/article-pdf/2/2/fcaa160/37828245/fcaa160.pdf |
| File Format | |
| e-ISSN | 26321297 |
| DOI | 10.1093/braincomms/fcaa160 |
| Journal | Brain Communications |
| Issue Number | 2 |
| Volume Number | 2 |
| Language | English |
| Publisher | Oxford Academic |
| Publisher Date | 2020-07-01 |
| Access Restriction | Open |
| Subject Keyword | Clinical Medicine Medicine and Health Neurology Neuroscience Science and Mathematics Vascular Endothelial Growth Factor a Amyotrophic Lateral Sclerosis Phase 1 Clinical Trials Safety Als Vegf Intracerebroventricular Synchromed® Ii Programmable Pump |
| Content Type | Text |
| Resource Type | Article |
| Subject | Psychiatry and Mental Health Neurology Cellular and Molecular Neuroscience Biological Psychiatry |
