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

Myotonic Dystrophy Clinical Trial

Official title:

Double-Blind, Placebo-Controlled, Dose-Range-Finding, Crossover Trial of Single Day Administration of ERX-963 in Adults With Myotonic Dystrophy Type 1

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03959189
• Other study ID #: ERX-963-001
• Status: Completed
• Phase: Phase 1
• Start date: June 17, 2019
• Est. completion date: April 30, 2020

Study information

• Verified date: May 2021
• Source: Expansion Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

This study is evaluating single administration of two dose levels of ERX-963 to explore the relationship between dose, safety, tolerability, exposure and clinical benefit. This is a multi-center, randomized, double-blind, placebo-controlled, two-treatment period crossover study in two cohorts of participants with DM1. Participants who have consented and meet eligibility criteria will receive two treatments, placebo and ERX-963, in a randomized crossover fashion with a washout period between the treatments. On treatment days, participants will receive treatment followed by repeated blood collection for pharmacokinetic analysis and administration of a battery of outcome measures relevant to sleep and cognition.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 12
• Est. completion date: April 30, 2020
• Est. primary completion date: March 31, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

Key Inclusion Criteria:

• 18 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

Key Exclusion Criteria:

• 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

Related Conditions & MeSH terms

• Myotonic Dystrophy
• Myotonic Dystrophy, Type 1 (DM1)

Intervention

Drug:
• ERX-963
Active medicine
• Placebo
Comparator

Locations

Country	Name	City	State
United States	The Center for Sleep & Wake Disorders	Chevy Chase	Maryland
United States	University of Iowa	Iowa City	Iowa
United States	Sleep Medicine Specialists of South Florida	Miami	Florida
United States	Stanford Neurosciences Health Center	Palo Alto	California

Sponsors (1)

Lead Sponsor	Collaborator
Expansion Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Assess the Effect of ERX-963 on the Stanford Sleepiness Scale Score Compared to the Effect of Placebo	Participants will self-report their level of sleepiness by self-rated questionnaire "Stanford Sleepiness Scale" (SSS). This is a single item questionnaire on a 7-point scale (1-7). Higher values indicate worse outcome.	From dosing to approximately 2 hours
Secondary	Assess the Effect of ERX-963 on the Change in Patient Global Impression - Improvement Scale (PGI-I) Compared to Placebo	The PGI-I is a 7-point rating system used by the patient to rate their overall clinical condition after intervention relative to before intervention where 1=very much improved; 2=much improved; 3=minimally improved; 4=no change; 5=minimally worse; 6=much worse, and 7=very much worse.	Administered at the end of the dosing visit day, upon completion of the other outcome measures. Approximately 2 hours after the end of infusion.
Secondary	Assess the Effect of ERX-963 on the Clinical Global Impressment - Improvement (CGI-I) Scale Compared to Placebo	The CGI-I is a 7-point rating system used by the clinician or investigator to compare the patient's overall clinical condition after intervention relative to before intervention where 1=very much improved; 2=much improved; 3=minimally improved; 4=no change; 5=minimally worse; 6=much worse, and 7=very much worse (Guy, 1976; Busner, 2007).	Administered at the end of the dosing visit day, upon completion of the other outcome measures. Approximately 2 hours after the end of infusion.
Secondary	Assess the Effect of ERX-963 on the Psychomotor Vigilance Task (PVT)	Participants will be tested for their response time and number of lapses during the PVT.	From dosing to approximately 2 hours
Secondary	Assess the Effect of ERX-963 on the One-back Task	Participants will be tested for the proportion of correct response to the One-back task.	From dosing to approximately 2 hours

External Links

•   Expansion Therapeutics Corporate Website