A Randomized, Double-Blind, to Evaluate the Efficacy and Safety of Staccato Apomorphine (AZ-009) in Patients With Parkinson's Disease Experiencing OFF Episodes
Overview
- Phase
- Phase 2
- Intervention
- Apomorphine Cartridge
- Conditions
- Parkinson Disease
- Sponsor
- Alexza Pharmaceuticals, Inc.
- Enrollment
- 8
- Locations
- 13
- Primary Endpoint
- MDS-Unified Parkinson's Disease Rating Scale
- Status
- Terminated
- Last Updated
- 6 months ago
Overview
Brief Summary
This study will be conducted with In-clinic visits and treatment at home for each patient with established Parkinson's disease (PD) experiencing daily OFF episodes.
Detailed Description
The purpose of this protocol is to establish the safety, tolerability, and efficacy of AZ-009 compared to placebo in patients with established PD experiencing daily OFF episodes. Patients between the ages of 30 and 85 with a clinical diagnosis of established PD who experience motor fluctuation and have recognizable OFF periods are eligible for participation in this study. The patients will be classified as Modified Hoehn \& Yahr stage II-IV in the ON state and have clear, self-described daily motor fluctuations while on oral l-dopa or Carbidopa (with or without adjunctive PD therapy). This study will be conducted in approximately 50 patients, with up to 8 Open-Label titration in-clinic visits with treatment at home between visits. This is followed by Double Blind which includes a 13 day treatment at home period, and an in-clinic visit and an End of Study/Early Termination visit for each patient with established Parkinson's disease (PD) experiencing daily OFF episodes.
Investigators
Eligibility Criteria
Inclusion Criteria
- •1\. Understand and voluntarily sign an informed consent document prior to any study related assessments/procedures being conducted.
- •2\. Willing and able to travel to the clinical research center and adhere to the overall study visit schedule, procedures, and other protocol requirements.
- •3\. Male or female between the ages of 30 and 85 (inclusive).
- •Body weight ≥ 50 kg.
- •Willing to abstain from alcohol for 6 hours prior to a study visit and minimize alcohol use throughout the study duration.
- •6\. Have a clinical diagnosis of PD; with fulfillment of Steps 1 and 2 of the UK Parkinson's Disease Brain Bank Criteria.
- •7\. Optimized and stabilized on oral dopaminergic therapy including levodopa at least 3 times daily and in combination with decarboxylase inhibitor at least 30 days prior to screening.
- •8\. Classified as Modified Hoehn \& Yahr stage II-IV in the ON state at Visit
- •Have an MDS-UPDRS III score of at least 30 in the OFF state prior to the L-dopa challenge at Visit
- •10\. Experience self-described motor fluctuations (confirmed by the Motor Fluctuation Questionnaire at Screening) with recognizable OFF periods while on optimized oral l-dopa or dopamine agonist therapy.
Exclusion Criteria
- •Subjects eligible for enrollment in the study must meet all of the following inclusion criteria and none of the exclusion criteria.
- •Inclusion Criteria:
- •Understand and voluntarily sign an informed consent document prior to any study related assessments/procedures being conducted.
- •Willing and able to travel to the clinical research center and adhere to the overall study visit schedule, procedures, and other protocol requirements.
- •Male or female between the ages of 30 and 85 (inclusive).
- •Body weight ≥ 50 kg.
- •Willing to abstain from alcohol for 6 hours prior to a study visit and minimize alcohol use throughout the study duration.
- •Have a clinical diagnosis of PD; with fulfillment of Steps 1 and 2 of the UK Parkinson's Disease Brain Bank Criteria.
- •Optimized and stabilized on oral dopaminergic therapy including levodopa at least 3 times daily and in combination with decarboxylase inhibitor at least 30 days prior to screening.
- •Classified as Modified Hoehn \& Yahr stage II-IV in the ON state at Visit
Arms & Interventions
Staccato Apomorphine
1mg, 2mg, 3mg, 4mg
Intervention: Apomorphine Cartridge
Staccato Placebo
Placebo
Intervention: Apomorphine Cartridge
Outcomes
Primary Outcomes
MDS-Unified Parkinson's Disease Rating Scale
Time Frame: at 10, 20, 30 and 45 minutes
The MDS-UPDRS is a revision of the Unified Parkinson's Disease Rating Scale (UPDRS) and was developed to evaluate various aspects of Parkinson's disease including non-motor and motor experiences of daily living and motor complications.