Safety, Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1

NCT03959189 | Completed Phase 1 Results FDA Drug

• 1 other identifier: ERX-963-001
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 4

Brief Summary

Participants in this study will receive two treatments, placebo and ERX-963, on different days in a randomized fashion. The primary purpose of this study is to investigate the safety and tolerability of ERX-963 in participants diagnosed with Myotonic Dystrophy, Type 1 (DM1). The secondary purpose is to evaluate the potential of ERX-963 treatment to reduce excessive daytime sleepiness / hypersomnia and improve cognitive function in DM1 participants compared to placebo treatment.

Conditions

Myotonic Dystrophy, Type 1 (DM1); Myotonic Dystrophy

Keywords

Excessive Daytime Sleepiness; hypersomnia

Outcome Measures

Primary Outcomes (1)

• Incidence of Adverse Events, Serious Adverse Events, and Drug-related Adverse Events [Safety and Tolerability] After a Single Dose of ERX-963 vs. Placebo

  An adverse event (AE) was defined as any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study intervention. Treatment-emergent AEs were AEs which started between the date and time of study drug dosing and through Study Day 2, within each period. Drug-related AEs were assessed by the investigator to determine the relationship (related or unrelated) between the study intervention and each AE occurrence.

  Adverse Events were collected from screening to the End of Study Visit, up to 57 days

Secondary Outcomes (5)

• Assess the Effect of ERX-963 on the Stanford Sleepiness Scale Score Compared to the Effect of Placebo

  From dosing to approximately 2 hours

• Assess the Effect of ERX-963 on the Change in Patient Global Impression - Improvement Scale (PGI-I) Compared to Placebo

  Administered at the end of the dosing visit day, upon completion of the other outcome measures. Approximately 2 hours after the end of infusion.

• Assess the Effect of ERX-963 on the Clinical Global Impressment - Improvement (CGI-I) Scale Compared to Placebo

  Administered at the end of the dosing visit day, upon completion of the other outcome measures. Approximately 2 hours after the end of infusion.

• Assess the Effect of ERX-963 on the Psychomotor Vigilance Task (PVT)

  From dosing to approximately 2 hours

• Assess the Effect of ERX-963 on the One-back Task

  From dosing to approximately 2 hours

Study Arms (2)

ERX-963 then placebo

EXPERIMENTAL

Participants in this arm will receive ERX-963 followed by a washout period. After the washout period, participants will receive placebo.

Drug: ERX-963

Placebo then ERX-963

EXPERIMENTAL

Participants in this arm will receive placebo followed by a washout period. After the washout period, participants will receive ERX-963.

Drug: Placebo

Interventions

ERX-963 DRUG

Active medicine

ERX-963 then placebo

Placebo DRUG

Comparator

Placebo then ERX-963

Eligibility Criteria

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

You may qualify if:

• to 65 years of age
• DM1 defined by genetic testing or clinical-confirmation
• Epworth Sleepiness Scale (ESS) of \> 11 or participants who have long sleep periods of an average of \> 10 hours a day
• Age of onset of DM1 greater than 16 years

You may not qualify if:

• Significant respiratory compromise
• Significant cardiac disease
• Diagnosis of symptomatic Restless Leg Syndrome or significant untreated nocturnal hypoxias
• Significant moderate to severe hepatic insufficiency
• Clinically active depression, anxiety, or other medical condition that, in the investigator's opinion, would interfere with the safety and efficacy assessments
• History of seizures
• History of panic disorders

Sponsors & Collaborators

• Expansion Therapeutics, Inc.: lead

Study Sites (4)

Stanford Neurosciences Health Center

Palo Alto, California, 94305, United States

Location

Sleep Medicine Specialists of South Florida

Miami, Florida, 33126, United States

Location

University of Iowa

Iowa City, Iowa, 52242, United States

Location

The Center for Sleep & Wake Disorders

Chevy Chase, Maryland, 20815, United States

Location

Related Links

• Expansion Therapeutics Corporate Website

MeSH Terms

Conditions

Myotonic Dystrophy; Disorders of Excessive Somnolence

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Myotonic Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Sleep Disorders, Intrinsic; Dyssomnias; Sleep Wake Disorders; Mental Disorders

Limitations and Caveats

The 0.5 mg dose level was not tested in this study. The analyses of the effect of ERX-963 on the Psychomotor Vigilance Task and the One-Back task were removed from the final analysis and documented accordingly in a Statistical Analysis Plan memo.

Results Point of Contact

• Title: Chief Medical Officer
• Organization: Expansion Therapeutics, Inc.

info@expansionrx.com

858-764-4290

Study Officials

• Elliot Ehrich, MD

  Chief Medical Officer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 16, 2019
• First Posted: May 22, 2019
• Study Start: June 17, 2019
• Primary Completion: March 31, 2020
• Study Completion: April 30, 2020
• Last Updated: June 23, 2021
• Results First Posted: June 23, 2021

Record last verified: 2021-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)