Back to resultsA Eight-Week Study of NLS-2 (Mazindol Extended Release) in Participants With Narcolepsy Type 1 (AMAZE)
Primary Purpose
Narcolepsy Type 1, Narcolepsy With Cataplexy
Status
Not yet recruiting
Phase
Phase 3
Locations
Study Type
Interventional
Intervention
NLS-2
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Narcolepsy Type 1 focused on measuring Narcolepsy, Cataplexy, Narcolepsy Type 1, Narcolepsy with Cataplexy
Eligibility Criteria
Key Inclusion Criteria: Adults ≥ 18 years of age at the time of signing the informed consent. Documented primary diagnosis of Narcolepsy Type 1 (NT1) according to the International Classification of Sleep Disorders-Third Edition (ICSD-3) criteria. Willing and able to safely discontinue all prohibited medications, including medications for the treatment of narcolepsy. Body Mass Index (BMI) within the range of 18 - 40 kg/m2 (inclusive). Key Exclusion Criteria: Any other (besides narcolepsy) clinically relevant medical, behavioral, or psychiatric disorder that is associated with excessive daytime sleepiness or cataplexy. History of myocardial infarction or significant cardiovascular disease, structural cardiac abnormalities, cardiomyopathy, congestive heart failure, cardiac arrhythmias, coronary artery disease, cerebrovascular disease (transient ischemic attack or stroke), or any other significant cardiac problem. History of long QT Syndrome or Torsades de Pointes, or an immediate family history of sudden cardiac death. History of pulmonary hypertension and/or valvulopathy. History of epilepsy, convulsions, or seizures (excluding early childhood febrile seizures). Significant history of head injury or head trauma. Recent or active suicidal ideation or behavior Current, or within the past year, diagnosis of substance abuse or dependence disorder (SUD) including alcohol abuse. Narrow-angle glaucoma. Severe renal or hepatic insufficiency. Occupation that requires variable or nighttime shift work.
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
NLS-2
Placebo
Arm Description
Participants will receive a single NLS-2 tablet orally once daily from Day 1 to Day 56 (until the end of Week 8).
Participants will receive a single NLS-2 placebo matching tablet orally once daily from Day 1 to Day 56 (until the end of Week 8).
Outcomes
Primary Outcome Measures
Number of cataplexy episodes at Week 8
Cataplexy episodes is a clinical outcome measure that assesses the frequency of cataplexy episodes.
Secondary Outcome Measures
Patient Reported Outcomes Measurement Information System-Sleep Related Impairment (PROMIS-SRI): change from baseline
The Patient Reported Outcomes Measurement Information System-Sleep Related Impairment (PROMIS-SRI) scale consists of 8 items to evaluate daytime consequences of functioning on a 5-point Likert scale ranging from 1 to 5. The PROMIS-SRI measures self-reported perceptions of alertness, sleepiness, and tiredness during usual waking hours, and perceived functional impairments during wakefulness associated with sleep problems or impaired alertness. Each question has five response options ranging in value from 1 to 5. To find the total raw score with all questions answered, sum the values of the response to each question and total score ranges from 8 to 40. Higher scores indicates greater sleep impairment.
Patient Reported Outcomes Measurement Information System-Sleep Disturbance (PROMIS-SD): change from baseline
The Patient Reported Outcomes Measurement Information System-Sleep Disturbance (PROMIS-SD) consists of a static 8-item questionnaire. Using a recall period of the past 7 days, it assesses the concepts of sleep initiation (2 items), quality of sleep (3 items), early morning feelings (2 items) and worrying about sleep (1 item). Each question has 5 response options ranging in value from 1 to 5. To find the total raw score with all questions answered, sum the values of the response to each question and a total score ranges from 8 to 40. Lower scores indicate less sleep disturbance. Negative changes in scores indicate improvement.
Epworth Sleepiness Scale (ESS): change from baseline
The Epworth Sleepiness Scale (ESS) is a patient-reported outcome measure that assesses daytime sleepiness. It asks the participant to rate, on a 4-point scale (0-3), their usual chances of dozing off or falling asleep in 8 different situations or activities that are commonly met in daily life. The total ESS score, ranging from 0 to 24, is the sum of 8 item scores, with higher scores indicating greater daytime sleepiness.
Patient Global Impression of Severity (PGI-S): change from baseline
The Patient Global Impression of Severity (PGI-S) is a patient-reported outcome measure that assesses the participant's perception of the severity of their illness. The participant rates their condition on a point scale, from "no symptoms" to "extremely severe."
Clinician Global Impression of Severity (CGI-S): change from baseline
The Clinical Global Impressions of Severity (CGI-S) is a clinician-rated outcome measure that assesses the clinician's impression of the participant's current illness severity. The clinician rates the severity of the participant's condition on a point scale, from "normal, not at all ill" to "among the most extremely ill patients".
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05914194
Brief Title
A Eight-Week Study of NLS-2 (Mazindol Extended Release) in Participants With Narcolepsy Type 1
Acronym
AMAZE
Official Title
A Phase 3, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Trial to Evaluate the Efficacy and Safety of NLS-2 (Mazindol Extended-Release) Tablets in the Treatment of Narcolepsy Type 1
Study Type
Interventional
2. Study Status
Record Verification Date
June 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
July 31, 2023 (Anticipated)
Primary Completion Date
July 31, 2024 (Anticipated)
Study Completion Date
August 15, 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
NLS Pharmaceutics
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
The goal of this clinical trial is to see how NLS-2 (mazindol extended-release) works on symptoms of narcolepsy, including cataplexy and excessive daytime sleepiness.
Approximately 48 participants will take part in the study across the United States.
The study treatment (NLS-2 or placebo) will be administered for 8 weeks. After this treatment period, the participant may have the option to participate in a separate long-term extension study during which all participants will be treated with NLS-2.
Detailed Description
This is a Phase 3, double-blind, placebo-controlled, multicenter, randomized, parallel-group clinical trial. The primary goal of this study is to assess the efficacy and safety of NLS-2 (mazindol extended-release) tablets in treating Narcolepsy Type 1. The trial aims to determine the effects of NLS-2 on the improvement of narcolepsy symptoms, including cataplexy frequency and excessive daytime sleepiness.
Participants in this trial will receive either NLS-2 or a placebo for 8 weeks. The treatment assignment will be concealed from both the participant and the investigator throughout the trial (unless there is an urgent medical need).
Approximately 48 patients will be enrolled at multiple clinical sites across the United States. Following the completion of the trial, participants may be given the option to enroll in a separate long-term extension trial where all participants will receive NLS-2 treatment.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Narcolepsy Type 1, Narcolepsy With Cataplexy
Keywords
Narcolepsy, Cataplexy, Narcolepsy Type 1, Narcolepsy with Cataplexy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Model Description
Randomized, Double-Blind, Placebo-Controlled
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
48 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
NLS-2
Arm Type
Experimental
Arm Description
Participants will receive a single NLS-2 tablet orally once daily from Day 1 to Day 56 (until the end of Week 8).
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Participants will receive a single NLS-2 placebo matching tablet orally once daily from Day 1 to Day 56 (until the end of Week 8).
Intervention Type
Drug
Intervention Name(s)
NLS-2
Other Intervention Name(s)
mazindol extended-release (ER), mazindol controlled-release (CR)
Intervention Description
Participants will receive NLS-2 tablets orally.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Participants will receive NLS-2 matching placebo tablets orally.
Primary Outcome Measure Information:
Title
Number of cataplexy episodes at Week 8
Description
Cataplexy episodes is a clinical outcome measure that assesses the frequency of cataplexy episodes.
Time Frame
8 weeks
Secondary Outcome Measure Information:
Title
Patient Reported Outcomes Measurement Information System-Sleep Related Impairment (PROMIS-SRI): change from baseline
Description
The Patient Reported Outcomes Measurement Information System-Sleep Related Impairment (PROMIS-SRI) scale consists of 8 items to evaluate daytime consequences of functioning on a 5-point Likert scale ranging from 1 to 5. The PROMIS-SRI measures self-reported perceptions of alertness, sleepiness, and tiredness during usual waking hours, and perceived functional impairments during wakefulness associated with sleep problems or impaired alertness. Each question has five response options ranging in value from 1 to 5. To find the total raw score with all questions answered, sum the values of the response to each question and total score ranges from 8 to 40. Higher scores indicates greater sleep impairment.
Time Frame
8 weeks
Title
Patient Reported Outcomes Measurement Information System-Sleep Disturbance (PROMIS-SD): change from baseline
Description
The Patient Reported Outcomes Measurement Information System-Sleep Disturbance (PROMIS-SD) consists of a static 8-item questionnaire. Using a recall period of the past 7 days, it assesses the concepts of sleep initiation (2 items), quality of sleep (3 items), early morning feelings (2 items) and worrying about sleep (1 item). Each question has 5 response options ranging in value from 1 to 5. To find the total raw score with all questions answered, sum the values of the response to each question and a total score ranges from 8 to 40. Lower scores indicate less sleep disturbance. Negative changes in scores indicate improvement.
Time Frame
8 weeks
Title
Epworth Sleepiness Scale (ESS): change from baseline
Description
The Epworth Sleepiness Scale (ESS) is a patient-reported outcome measure that assesses daytime sleepiness. It asks the participant to rate, on a 4-point scale (0-3), their usual chances of dozing off or falling asleep in 8 different situations or activities that are commonly met in daily life. The total ESS score, ranging from 0 to 24, is the sum of 8 item scores, with higher scores indicating greater daytime sleepiness.
Time Frame
8 weeks
Title
Patient Global Impression of Severity (PGI-S): change from baseline
Description
The Patient Global Impression of Severity (PGI-S) is a patient-reported outcome measure that assesses the participant's perception of the severity of their illness. The participant rates their condition on a point scale, from "no symptoms" to "extremely severe."
Time Frame
8 weeks
Title
Clinician Global Impression of Severity (CGI-S): change from baseline
Description
The Clinical Global Impressions of Severity (CGI-S) is a clinician-rated outcome measure that assesses the clinician's impression of the participant's current illness severity. The clinician rates the severity of the participant's condition on a point scale, from "normal, not at all ill" to "among the most extremely ill patients".
Time Frame
8 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria:
Adults ≥ 18 years of age at the time of signing the informed consent.
Documented primary diagnosis of Narcolepsy Type 1 (NT1) according to the International Classification of Sleep Disorders-Third Edition (ICSD-3) criteria.
Willing and able to safely discontinue all prohibited medications, including medications for the treatment of narcolepsy.
Body Mass Index (BMI) within the range of 18 - 40 kg/m2 (inclusive).
Key Exclusion Criteria:
Any other (besides narcolepsy) clinically relevant medical, behavioral, or psychiatric disorder that is associated with excessive daytime sleepiness or cataplexy.
History of myocardial infarction or significant cardiovascular disease, structural cardiac abnormalities, cardiomyopathy, congestive heart failure, cardiac arrhythmias, coronary artery disease, cerebrovascular disease (transient ischemic attack or stroke), or any other significant cardiac problem.
History of long QT Syndrome or Torsades de Pointes, or an immediate family history of sudden cardiac death.
History of pulmonary hypertension and/or valvulopathy.
History of epilepsy, convulsions, or seizures (excluding early childhood febrile seizures).
Significant history of head injury or head trauma.
Recent or active suicidal ideation or behavior
Current, or within the past year, diagnosis of substance abuse or dependence disorder (SUD) including alcohol abuse.
Narrow-angle glaucoma.
Severe renal or hepatic insufficiency.
Occupation that requires variable or nighttime shift work.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Jeff Bernier
Phone
617-905-2282
Email
amaze@nls-pharma.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
George Apostol, MD, MS
Organizational Affiliation
NLS Pharmaceutics
Official's Role
Study Chair
First Name & Middle Initial & Last Name & Degree
Jennifer Franco
Organizational Affiliation
NLS Pharmaceutics
Official's Role
Study Director
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
A Eight-Week Study of NLS-2 (Mazindol Extended Release) in Participants With Narcolepsy Type 1
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