Placebo-Controlled, Double-Blind, Study to Determine the Safety and Efficacy of SDX in Patients With IH

NCT05668754 | Completed Phase 2 FDA Drug

• 1 other identifier: KP1077.D01
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 41

Brief Summary

This is a study of the safety, efficacy and pharmacokinetics (PK) of Serdexmethylphenidate (SDX) compared to placebo in subjects with Idiopathic Hypersomnia (IH).

Conditions

Idiopathic Hypersomnia

Keywords

Disorders of Excessive Somnolence; Sleep Disorders; Intrinsic Dyssomnias; Sleep Wake Disorders; Nervous System Diseases; Mental Disorders

Outcome Measures

Primary Outcomes (6)

• Safety parameters - TEAEs

  Type and frequency of Treatment-Emergent Adverse Events

  Time Frame: Start of OLTP to end of DBWP (7 weeks)

• Safety parameter - Heart Rate

  Change from baseline in heart rate (beats/minute).

  Time Frame: Start of OLTP to end of DBWP (7 weeks)

• Safety parameter - Blood Pressure

  Change from baseline in blood pressure (mmHg).

  Time Frame: Start of OLTP to end of DBWP (7 weeks)

• Safety parameter - Laboratory Tests

  Clinical significant change from baseline in clinical laboratory tests.

  Time Frame: Start of OLTP to end of DBWP (7 weeks)

• Safety parameter - ECG

  Clinical significant change from baseline in electrocardiogram (ECG).

  Time Frame: Start of OLTP to end of DBWP (7 weeks)

• Safety parameter - PSQI

  Change from baseline in Pittsburg Sleep Quality Index Question #6. Sleep quality score ranging from very good (0) to very bad (4).

  Time Frame: Start of OLTP to end of DBWP (7 weeks)

Secondary Outcomes (9)

• Change from baseline in Epworth Sleepiness Scale (ESS) score

  Start to end of DBWP (2 weeks)

• Change from baseline in Brain Fog score

  Time Frame: Start to end of DBWP (2 weeks)

• Percentage of participants with increase (worsening) of 2 points or more from baseline in the Clinical Global Impression of Severity (CGI-S)

  Start to end of DBWP (2 weeks)

• Percentage of participants with increase (worsening) of 2 points or more baseline in the Patient Global Impression of Severity (PGI-S).

  Start to end of DBWP (2 weeks)

• Change from baseline in Total Score of the Idiopathic Hypersomnia Severity Hypersomnia Scale (IHSS)

  Start to end of DBWP (2 weeks)

Study Arms (2)

Experimental: SDX

EXPERIMENTAL

SDX capsules at the optimized daily dose, once in the evening daily (qd pm) or twice per day (bid), for 2 weeks (DBWP)

Drug: Serdexmethylphenidate

Active Comparator

PLACEBO COMPARATOR

Placebo capsules once in the evening daily (qd pm) or twice per day (bid), for 2 weeks (DBWP)

Drug: Serdexmethylphenidate; Other: Placebo

Interventions

Serdexmethylphenidate DRUG

Participants randomized to active drug will receive their optimized dose according to a dosing regimen set by randomization at the start of the OLTP. The 4 possible oral SDX doses are 80, 160, 240, or 320 mg/day. The optimal SDX dose will be determined during the 5-week OLTP preceding the 2-week DBWP.

Also known as: SDX

Active Comparator; Experimental: SDX

Placebo OTHER

Participants randomized to placebo will receive matching placebo capsules to the optimized dose established at the end of the OLTP, according to a dosing regimen set by randomization at the start of the OLTP.

Active Comparator

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• At least 18 years of age at the time of consent
• Body Mass Index (BMI) ≤35 kg/m2
• Documented primary diagnosis of IH according to the International Classification of Sleep Disorders (ICSD-3) criteria
• At the Screening Visit and Baseline Visit (start of OLTP), Epworth Sleepiness Scale (ESS) scores ≥11
• Average nightly Total Sleep Time (TST) of ≥7 hours, per subject history and confirmed during screening.
• Subject must be in general good health defined as the absence of any clinically relevant abnormalities as determined by the Investigator based on physical and neurological examinations, vital signs, ECGs, medical history, and clinical laboratory values (hematology, chemistry, and urinalysis) at Screening.
• If currently treated with nicotine replacement therapy, must have been taking the same regimen and dose for at least 2 months prior to screening and must agree to take the same dose during the study.
• Have used a medically acceptable method of contraception for at least 2 months prior to the first dose of study drug and consent to use a medically acceptable method of contraception from the first dose of study drug, throughout the entire study period, and for 90 days after the last dose of study drug.

You may not qualify if:

• Hypersomnia due to another medical, behavioral, or psychiatric disorder condition (eg, narcolepsy, depression disorders, multiple sclerosis, Parkinson's disease, stroke).
• Clinically significant sleep-related breathing disorders, including sleep apnea, treatment with Continuous Positive Airway Pressure (CPAP) therapy, Obstructive Apnea Hypopnea Index (AHI) \>15 episodes per hour, or hypoventilation.
• Clinically significant parasomnias (eg, sleep walking, rapid eye movement \[REM\] sleep behavior disorder, etc).
• Periodic Limb Movement Disorder (PLMD) Arousal Index (PLMA-I) \>15 during Screening PSG, a historical diagnosis of PLMD (last 10 years), or a PLMD diagnosis older than 10 years with current (last 60 days) treatment or symptoms of rhythmic movements involving one or both legs during sleep.
• Occupation requiring nighttime shift work or variable shift work with early work start times (before 6 AM), if this occurs more than once per week.
• Planned travel during the study that includes more than 3 time zones, or planned travel that includes 3 time zones on more than 2 occasions during the study.
• Going to sleep for the night later than 1 AM at a frequency of more than once per week.
• Current or past (within 1 year) major depressive episode according to DSM-5 criteria.
• Any history of attempted suicide (lifetime) or clinically significant suicidal ideation, in the opinion of the Investigator, based on the C-SSRS assessment at Screening.
• Any clinically significant unstable medical abnormality, chronic disease (eg, asthma or diabetes), or a history of a clinically significant abnormality of the cardiovascular, central nervous system,
• Any of the following out-of-range vital signs at Screening: systolic blood pressure outside 90-145 mmHg; diastolic blood pressure outside 50-90 mmHg; resting heart rate outside 40-100 beats per minute.
• History or presence of abnormal ECGs, which in the Investigator's opinion is clinically significant, including the following:
• ECG findings of ischemia or infarct
• Complete bundle branch blocks
• Symptomatic arrhythmias as ventricular arrhythmias (non- sustained ventricular tachycardia (VT), multifocal or frequent premature ventricular contractions), bundle branch block, axis deviation, or abnormal or any predominantly non-sinus- conducted rhythm.

Sponsors & Collaborators

• Zevra Therapeutics: lead
• Rho, Inc.: collaborator

Study Sites (41)

Sleep Disorders Center Of Alabama

Birmingham, Alabama, 35201, United States

Location

Amr Daphne

Daphne, Alabama, 36526, United States

Location

Lakeview Clinical Research

Guntersville, Alabama, 35976, United States

Location

SOCAL Clinical Research

Huntington Beach, California, 92647, United States

Location

Stanford University

Redwood City, California, 94063, United States

Location

Sleep Medicine Specialists of California

San Ramon, California, 94583, United States

Location

SDS Clinical Trials, Inc

Santa Ana, California, 92701, United States

Location

Delta Waves, Inc.

Colorado Springs, Colorado, 80903, United States

Location

Saint Francis Sleep Allergy and Lung Institute LLC

Clearwater, Florida, 33755, United States

Location

New Generation of Medical Trials

Hialeah, Florida, 33010, United States

Location

Angels Clinical Research

Miami, Florida, 33122, United States

Location

Ivetmar Medical Group

Miami, Florida, 33130, United States

Location

Somnology Research Associates

Miami, Florida, 33130, United States

Location

Clinical Trial Services, Corp

Miami, Florida, 33144, United States

Location

Pasadena Center for Medical Research

St. Petersburg, Florida, 33707, United States

Location

Clinical Site Partners, LLC - Winter Park

Winter Park, Florida, 32789, United States

Location

Global Research Associates

Atlanta, Georgia, 30303, United States

Location

Neurotrials Research, Inc

Atlanta, Georgia, 30303, United States

Location

Clinical Research Institute - Stockbridge

Stockbridge, Georgia, 30281, United States

Location

The University of Kansas Medical Center Research Institution Inc.

Kansas City, Kansas, 64106, United States

Location

Mid-Atlantic Epilepsy and Sleep Center - Bethesda

Bethesda, Maryland, 20817, United States

Location

Neurocare, Inc.

Newton, Massachusetts, 02459, United States

Location

Western Michigan University Homer Stryker Md School of Medicine

Kalamazoo, Michigan, 49007, United States

Location

Henry Ford Health - Columbus

Novi, Michigan, 48375, United States

Location

Clinical Neurophysiology Services PC

Sterling Heights, Michigan, 48313, United States

Location

University of Missouri School Of Medicine

Columbia, Missouri, 65211, United States

Location

Clayton Sleep Institute, Llc

St Louis, Missouri, 63123, United States

Location

Barrett Clinic

La Vista, Nebraska, 68128, United States

Location

Global Medical Institutes LLC- Princeton Medical Institute

Lawrence, New Jersey, 08648, United States

Location

Clinical Research of Gastonia (CRG)

Gastonia, North Carolina, 28052, United States

Location

Intrepid Research

Cincinnati, Ohio, 45202, United States

Location

Ohio Sleep Medicine Institute

Dublin, Ohio, 43017, United States

Location

Brian Abaluck, LLC

Malvern, Pennsylvania, 19355, United States

Location

Thomas Jefferson University

Philadelphia, Pennsylvania, 19107, United States

Location

Medical University Of South Carolina (MUSC) - Institute Of Psychiatry (IOP)

Charleston, South Carolina, 29401, United States

Location

Bogan Sleep Consultants

Columbia, South Carolina, 29201, United States

Location

Futuresearch Trials Of Neurology

Austin, Texas, 78701, United States

Location

Dfw Clinical Research Associates

Fort Worth, Texas, 76244, United States

Location

Houston Clinical Research Associates

Houston, Texas, 77002, United States

Location

Sleep Therapy & Research Center

San Antonio, Texas, 78205, United States

Location

TPMG Clinical Research - Williamsburg

Williamsburg, Virginia, 23185, United States

Location

MeSH Terms

Conditions

Idiopathic Hypersomnia; Disorders of Excessive Somnolence; Sleep Wake Disorders; Nervous System Diseases; Mental Disorders

Interventions

serdexmethylphenidate and dexmethylphenidate

Condition Hierarchy (Ancestors)

Sleep Disorders, Intrinsic; Dyssomnias; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Christopher Drake, PhD

  Investigator

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: Open-Label Titration Period with a Double-Blind Withdrawal Period
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Phase 2, placebo-controlled, double-blind, randomized withdrawal study to determine the safety and efficacy of oral SDX in patients with Idiopathic Hypersomnia (IH).

Study Record Dates

• First Submitted: December 19, 2022
• First Posted: December 30, 2022
• Study Start: December 28, 2022
• Primary Completion: February 28, 2024
• Study Completion: March 21, 2024
• Last Updated: February 3, 2025

Record last verified: 2025-01

Locations

US (41)