The primary objectives of this study are to determine the safety, tolerability and efficacy of human fibroblast growth factor 1 (FGF-1) when administered intranasally to four (4) subjects with idiopathic, stage III or IV Parkinson's disease (Hoehn and Yahr staging criteria). This is an open-label, pilot study to study two dose levels of human FGF-1. A low dose of 450 µg FGF-1 (6 µg/kg for a 75 kg subject) and a high dose of 900 µg FGF-1 (12 µg/kg for a 75 kg subject) will be studied sequentially. Subjects will be screened up to 14 days before administration of the investigational drug product (FGF-1). If the subject complies with all inclusion/exclusion criteria, they will be enrolled in the study.~The proposed daily intranasal dosing regimen for the four subjects is as follows:~Week 1: 450 µg FGF-1 Week 2: 450 µg FGF-1 Week 3: No patient dosing, but safety and efficacy testing will be performed. If deemed safe, but a documented improvement in efficacy is not observed, the four subjects will proceed to administration of a higher dose of FGF-1.~Week 4: daily dosing with 900 µg FGF-1 Weeks 5-12: Follow-up weekly visits; no drug dosing; only safety and efficacy testing will be performed.~Prior to delivering the drug, the subject's vital signs will be taken. Following the intranasal delivery of the FGF-1 dose by the ViaNase® Electronic Atomizer (this device, which the U.S. FDA has authorized for use in humans, releases a metered dose of a drug into each nostril of the subject's nose, which then is inhaled by breathing evenly over a 2-minute period). Vital signs will be measured after the two-minute breathing period and then again in 15 minutes and 30 minutes. Following the final dosing period, follow-up visits for safety testing and efficacy evaluations will be given at weekly intervals for the following 7 weeks.~Safety and tolerability will be assessed by evaluating adverse events (AEs) (including a battery of laboratory evaluations, vital signs, physical examination findings, eye exams, and electrocardiograms [ECGs]). Efficacy will be assessed with the U.S. Movement Disorder Society-Sponsored Revision of the Uniﬁed Parkinson's Disease Rating Scale (MDS-UPDRS). This is a validated questionnaire that takes 30 minutes for a trained medical professional to administer to patients and measures the longitudinal progression of Parkinson's disease, including a section on motor skill testing.~Expected outcomes from this study include establishing that human FGF-1 is safe and tolerable when administered intranasally to subjects with Parkinson's disease and that a clinical benefit is observed in these patients, who currently have very limited medical options.
This is an open-label, pilot study to evaluate the safety, tolerability and efficacy of two dose levels of human FGF-1 administered intranasally to subjects with Parkinson's disease. A low dose of 450 µg FGF-1 (6 µg/kg for a 75 kg subject) and a high dose of 900 µg FGF-1 (12 µg/kg for a 75 kg subject) will be studied sequentially.