Narcolepsy Type 1Phase 3AMAZENCT05914194
A Eight-Week Study of NLS-2 (Mazindol Extended Release) in Participants With Narcolepsy Type 1
A Phase 3 study testing NLS-2 in people with Narcolepsy Type 1 and Narcolepsy With Cataplexy, run by NLS Pharmaceutics.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Narcolepsy Type 1, Narcolepsy With Cataplexy
- What is being tested?
- NLS-2
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 48 participants
- Where
- No sites listed yet
- Study dates
- 15 Jul 2024to 30 Jul 2025
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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).
Who cannot join
- 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.
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- 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.
Trial locations and contacts
No sites are listed yet.
About this study
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
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.
Read the 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.
- Conditions
- Narcolepsy Type 1, Narcolepsy With Cataplexy
- Being tested
- NLS-2, Placebo
- Keywords
- Narcolepsy, Cataplexy, Narcolepsy Type 1, Narcolepsy with Cataplexy
- Medical subject headings
- Narcolepsy, Cataplexy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NLS-2Drug
Also called mazindol extended-release (ER), mazindol controlled-release (CR)
Participants will receive NLS-2 tablets orally.
PlaceboDrug
Participants will receive NLS-2 matching placebo tablets orally.
| Study group | Role | What they receive |
|---|---|---|
| NLS-2 | Experimental | Participants will receive a single NLS-2 tablet orally once daily from Day 1 to Day 56 (until the end of Week 8). Receives: Drug: NLS-2 |
| Placebo | Placebo comparator | 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). Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of cataplexy episodes at Week 8
Cataplexy episodes is a clinical outcome measure that assesses the frequency of cataplexy episodes.
Measured: 8 weeks
Secondary outcomes (5)
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.
Measured: 8 weeks
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.
Measured: 8 weeks
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.
Measured: 8 weeks
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."
Measured: 8 weeks
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".
Measured: 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jun 2023
- Study start15 Jul 2024 (estimated)
- Primary completion30 Jul 2025 (estimated)
- Study completion30 Jul 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Narcolepsy Type 1Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Randomized, Double-Blind, Placebo-Controlled
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 48 participants (estimated)
Sponsor and investigators
- Lead sponsor
- NLS Pharmaceutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- George Apostol, MD, MS, Study chair, NLS Pharmaceutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05914194
- Sponsor's protocol ID
- NLS-1031
- Acronym
- AMAZE
- Status last verified
- May 2024
- First submitted
- 13 Jun 2023
- Last updated
- 21 May 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05914194
- Acronym / sponsor ID
- AMAZE / NLS-1031
- Conditions
- Narcolepsy Type 1, Narcolepsy With Cataplexy
- Keywords
- Narcolepsy, Cataplexy, Narcolepsy Type 1, Narcolepsy with Cataplexy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 48 participants (estimated)
- Lead sponsor
- NLS PharmaceuticsIndustry (pharma, biotech, devices)
- Study start
- 15 Jul 2024 (estimated)
- Primary completion
- 30 Jul 2025 (estimated)
- Study completion
- 30 Jul 2025 (estimated)
- First posted
- 22 Jun 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
- Model description
- Randomized, Double-Blind, Placebo-Controlled
| Arm | Type | Receives |
|---|---|---|
| NLS-2 | Experimental | Drug: NLS-2Participants will receive a single NLS-2 tablet orally once daily from Day 1 to Day 56 (until the end of Week 8). |
| Placebo | Placebo comparator | Drug: PlaceboParticipants will receive a single NLS-2 placebo matching tablet orally once daily from Day 1 to Day 56 (until the end of Week 8). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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 (11 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NLS-2Also: mazindol extended-release (ER), mazindol controlled-release (CR)Participants will receive NLS-2 tablets orally. | Drug | NLS-2 |
| PlaceboParticipants will receive NLS-2 matching placebo tablets orally. | Drug | Placebo |
Primary outcome
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.
5 secondary outcomes
Patient Reported Outcomes Measurement Information System-Sleep Related Impairment (PROMIS-SRI): change from baseline
Time frame: 8 weeks
Patient Reported Outcomes Measurement Information System-Sleep Disturbance (PROMIS-SD): change from baseline
Time frame: 8 weeks
Epworth Sleepiness Scale (ESS): change from baseline
Time frame: 8 weeks
Patient Global Impression of Severity (PGI-S): change from baseline
Time frame: 8 weeks
Clinician Global Impression of Severity (CGI-S): change from baseline
Time frame: 8 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 May 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
George Apostol, MD, MS study chair
NLS Pharmaceutics
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing NLS-2 in people with Narcolepsy Type 1 and Narcolepsy With Cataplexy, run by NLS Pharmaceutics.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 21 May 2024. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Jeff Bernier (617-905-2282, amaze@nls-pharma.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of cataplexy episodes at Week 8 (time frame: 8 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 May 2024.