Funded Studies
Objective/Rationale:
Uncontrolled saliva or drooling (sialorrhea) is a socially debilitating condition with high prevalence in Parkinson’s disease (PD) patients. The NH004 treatment to control sialorrhea uses a known drug (tropicamide) that has been approved for another medical use and has now been formulated in a postage stamp-sized thin film. This thin film is placed in and slowly dissolves in the mouth to maximize the local drug delivery. This project builds off a 2007 grant funded by MJFF.
Project Description:
A small study conducted in a physician’s office demonstrated that a single use of NH004 shows promise in reducing the symptoms of sialorrhea. Investigators will now conduct a clinical study in a home setting testing NH004 over a two-week period in PD patients with sialorrhea. The study will be conducted in a “blinded” fashion with half the people receiving the test treatment and half receiving an identical looking placebo film.
Relevance to Treatment of Parkinson’s Disease:
NH004 is designed to provide relief for PD patients suffering from sialorrhea.
Anticipated Outcome:
Researchers expect to prove that the NH004 films are an effective treatment to provide relief from sialorrhea for PD patients in a home-based setting.
PARTNERING PROGRAM
This grant was selected by The Michael J. Fox Foundation staff to be highlighted via the Foundation’s Partnering Program.
Final Outcome
The safety profile of the NH004 was acceptable, with relatively few, and no serious, side effects. The use of intra-orally dissolving thin films appears to be an acceptable drug delivery means for PD patients.
Unfortunately, analysis of the results from this clinical trial did not demonstrate that NH004 thin films reduce sialorrhea (excessive drooling) in PD patients over placebo, which may have been due to the unanticipated and considerable variability in the outcome measures at baseline.
A post-hoc analysis indicated a non-significant trend in a secondary outcome measure in favor of tropicamide NH004 films over placebo.
Presentations & Publications
Farber NM, Perez-Lloret S, Gamzu, ER. Design and Development of a Novel Supportive Care Product for the Treatment of Sialorrhea in Parkinson’s Disease. Current Topics in Medicinal Chemistry. 2015;15:939-954.
Lloret SP. Nano G, Carrosella A, Gamzu E, Merello M. A Double-blind, Placebo-controlled, Randomized, Crossover Pilot Study of the Safety and Efficacy of Multiple Doses of Intra-oral Tropicamide Films for the Short-term Relief of Sialorrhea Symptoms in Parkinson's Disease Patients. Journal of Neurological Sciences. 310:248-250.
October 2016