A Phase 1 interventional study of ERX-963 and Placebo in Myotonic Dystrophy, Type 1 (DM1) and Myotonic Dystrophy, sponsored by Expansion Therapeutics, Inc.. Completed at 4 sites in United States. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2021-06-23.
Sponsored by Expansion Therapeutics, Inc. · Phase 1, Interventional, and Treatment
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.
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.
125 studies on the registry are indexed under Myotonic Dystrophy; 51 are open to participants now.
This study's enrollment of 12 is below the median of 41 across 66 interventional studies indexed under Myotonic Dystrophy.
Browse Myotonic Dystrophy studies →This is the only study on the registry with Expansion Therapeutics, Inc. as lead sponsor.
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Key Inclusion Criteria:
Key Exclusion Criteria:
Participants in this arm will receive ERX-963 followed by a washout period. After the washout period, participants will receive placebo.
Drug: ERX-963
Participants in this arm will receive placebo followed by a washout period. After the washout period, participants will receive ERX-963.
Drug: Placebo
Active medicine
Comparator
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.
Time frame: Adverse Events were collected from screening to the End of Study Visit, up to 57 days
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.
Time frame: 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
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.
Time frame: 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
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).
Time frame: 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)
Participants will be tested for their response time and number of lapses during the PVT.
Time frame: From dosing to approximately 2 hours
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.
Time frame: From dosing to approximately 2 hours
Between June 2019 and March 2020, 12 patients were enrolled and treated with ERX-963 at three sites in the United States (Stanford University Neurosciences Health Center, University of Iowa Hospitals and Clinics, and Sleep Specialists of South Florida).
| Milestone | Cohort 1: 1 mg ERX-963 | Cohort 2: 2 mg ERX-963 |
|---|---|---|
| Started | 7 | 5 |
| Completed | 7 | 5 |
| Not completed | 0 | 0 |
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.
| Participants | Cohort 1: 1 mg ERX-963 | Cohort 2: 2 mg ERX-963 |
|---|---|---|
| Serious Adverse Events | 0 | 0 |
| Drug-related AEs in Placebo period | 1 | 1 |
| Drug-related AEs in ERX-963 period | 0 | 0 |
| TEAEs in Placebo period | 0 | 2 |
| TEAEs in ERX-963 period | 1 | 1 |
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.
| score on a scale | Cohort 1: Placebo Period | Cohort 1: 1 mg ERX-963 Period | Cohort 2: Placebo Period | Cohort 2: 2 mg ERX-963 Period |
|---|---|---|---|---|
| baseline Stanford Sleepiness Score | 3.4 ± 0.79 | 3.4 ± 0.79 | 4.0 ± 2.12 | 4.2 ± 2.17 |
| 10 min. after end of infusion, SSS | 3.0 ± 0.82 | 2.7 ± 1.60 | 4.0 ± 1.87 | 3.8 ± 2.39 |
| 40 min. after end of infusion, SSS | 3.1 ± 1.57 | 2.9 ± 1.68 | 4.2 ± 1.92 | 4.0 ± 2.45 |
| 1 hr., 10 min. after end of infusion, SSS | 3.7 ± 1.98 | 3.1 ± 1.57 | 4.0 ± 1.73 | 5.0 ± 2.45 |
| 1 hr., 40 min. after end of infusion, SSS | 3.6 ± 1.40 | 3.3 ± 0.76 | 4.6 ± 1.82 | 5.6 ± 1.82 |
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.
| Score of a scale | Cohort 1: Placebo Period | Cohort 1: 1 mg ERX-963 Period | Cohort 2: Placebo Period | Cohort 2: 2 mg ERX-963 Period |
|---|---|---|---|---|
| Assess the Effect of ERX-963 on the Change in Patient Global Impression - Improvement Scale (PGI-I) Compared to Placebo | 3.4 ± 1.27 | 3.4 ± 1.13 | 4.0 ± 2.00 | 4.2 ± 1.92 |
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).
| Score on a scale | Cohort 1: Placebo Period | Cohort 1: 1 mg ERX-963 Period | Cohort 2: Placebo Period | Cohort 2: 2 mg ERX-963 Period |
|---|---|---|---|---|
| Assess the Effect of ERX-963 on the Clinical Global Impressment - Improvement (CGI-I) Scale Compared to Placebo | 3.7 ± 1.25 | 3.9 ± 0.90 | 4.0 ± 1.22 | 4.4 ± 1.67 |
Participants will be tested for their response time and number of lapses during the PVT.
No measurements were reported for this outcome.
Participants will be tested for the proportion of correct response to the One-back task.
No measurements were reported for this outcome.
Collected over Adverse Events were collected from screening to the End of Study Visit, which was a period of up to 57 days. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Cohort 1: Placebo Period | 0/7 (0%) | 0/7 (0%) | 0/7 (0%) |
| Cohort 1: 1 mg ERX-963 Period | 0/7 (0%) | 0/7 (0%) | 1/7 (14.3%) |
| Cohort 2: Placebo Period | 0/5 (0%) | 0/5 (0%) | 2/5 (40%) |
| Cohort 2: 2 mg ERX-963 Period | 0/5 (0%) | 0/5 (0%) | 1/5 (20%) |
| Event | Cohort 1: Placebo Period | Cohort 1: 1 mg ERX-963 Period | Cohort 2: Placebo Period | Cohort 2: 2 mg ERX-963 Period |
|---|---|---|---|---|
| DiplopiaEye disorders | 0/7 | 0/7 | 0/5 | 1/5 |
| Infusion site painGeneral disorders | 0/7 | 0/7 | 0/5 | 1/5 |
| ContusionInjury, poisoning and procedural complications | 0/7 | 0/7 | 1/5 | 0/5 |
| HypersomniaNervous system disorders | 0/7 | 0/7 | 1/5 | 0/5 |
| HeadacheNervous system disorders | 0/7 | 1/7 | 0/5 | 0/5 |
| Age, Continuous(years) | Cohort 1: 1 mg ERX-963 | Cohort 2: 2 mg ERX-963 | Total |
|---|---|---|---|
| Median | 54.0 (47 to 59) | 44.0 (29 to 56) | 51.5 (29 to 59) |
| Sex: Female, Male(Participants) | Cohort 1: 1 mg ERX-963 | Cohort 2: 2 mg ERX-963 | Total |
|---|---|---|---|
| Female | 5 | 1 | 6 |
| Male | 2 | 4 | 6 |
| Ethnicity (NIH/OMB)(Participants) | Cohort 1: 1 mg ERX-963 | Cohort 2: 2 mg ERX-963 | Total |
|---|---|---|---|
| Hispanic or Latino | 0 | 0 | 0 |
| Not Hispanic or Latino | 7 | 5 | 12 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | Cohort 1: 1 mg ERX-963 | Cohort 2: 2 mg ERX-963 | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 |
| White | 7 | 5 | 12 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Region of Enrollment(participants) | Cohort 1: 1 mg ERX-963 | Cohort 2: 2 mg ERX-963 | Total |
|---|---|---|---|
| United States | 7 | 5 | 12 |
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