A Eight-Week Study of NLS-2 (Mazindol Extended Release) in Participants With Narcolepsy Type 1 (AMAZE)

May 19, 2024 updated by: NLS Pharmaceutics

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

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.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Estimated)

48

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: NLS-2
Participants will receive a single NLS-2 tablet orally once daily from Day 1 to Day 56 (until the end of Week 8).
Drug: NLS-2
Participants will receive NLS-2 tablets orally.
Other Names:
  • mazindol extended-release (ER), mazindol controlled-release (CR)
Placebo Comparator: Placebo
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).
Drug: Placebo
Participants will receive NLS-2 matching placebo tablets orally.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of cataplexy episodes at Week 8
Time Frame: 8 weeks
Cataplexy episodes is a clinical outcome measure that assesses the frequency of cataplexy episodes.
8 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Epworth Sleepiness Scale (ESS): change from baseline
Time Frame: 8 weeks
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.
8 weeks
Patient Global Impression of Severity (PGI-S): change from baseline
Time Frame: 8 weeks
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."
8 weeks
Clinician Global Impression of Severity (CGI-S): change from baseline
Time Frame: 8 weeks
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".
8 weeks
Patient Reported Outcomes Measurement Information System-Sleep Related Impairment (PROMIS-SRI): change from baseline
Time Frame: 8 weeks
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.
8 weeks
Patient Reported Outcomes Measurement Information System-Sleep Disturbance (PROMIS-SD): change from baseline
Time Frame: 8 weeks
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.
8 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

NLS Pharmaceutics

Investigators

  • Study Chair: George Apostol, MD, MS, NLS Pharmaceutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

July 15, 2024

Primary Completion (Estimated)

July 30, 2025

Study Completion (Estimated)

July 30, 2025

Study Registration Dates

First Submitted

June 13, 2023

First Submitted That Met QC Criteria

June 13, 2023

First Posted (Actual)

June 22, 2023

Study Record Updates

Last Update Posted (Actual)

May 21, 2024

Last Update Submitted That Met QC Criteria

May 19, 2024

Last Verified

May 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NLS-1031

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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