Essential3 study 2
22 evidence topics · 7 sources
Objective
Maintenance of response and durability
Location and study date
Reported initiation of the shared Essential3 program
| Milestone | Reported date |
|---|---|
| Program initiation | “November 2023” |
Exact first-participant and last-participant visit dates
No evidence found.
Study design
Blinded lead-in followed by randomized withdrawal
Eligibility criteria
Adult essential tremor population
| Criterion | Reported requirement |
|---|---|
| Age | “18–85 years” |
| Symptom duration | “≥3 years” |
| Stable background medication before screening | “≥28 days” |
Response required for randomized withdrawal
| Criterion | Reported threshold or assessment |
|---|---|
| mADL11 reduction for stable response | “3 points” |
| Visits averaged for response qualification | “Day 49 and 56” |
| Consecutive visits required to establish loss of response | “2 consecutive visits” |
| Deterioration from randomized-withdrawal baseline | “3 points or more” |
Treatment
Ulixacaltamide lead-in regimen
Study outcomes
Rationale for using mADL11 response maintenance as the primary endpoint
Primary response-maintenance outcome
Statistical analysis description
Number of participants (Planned and analyzed)
Original planned enrollment
| Study component | Planned participants |
|---|---|
| Study 2 randomized-withdrawal design | “200” |
Actual enrollment
| Population | Reported participants |
|---|---|
| Enrolled | “238” |
Description of analysis sets
Additional model specifications and missing-data procedures
No evidence found.
Results
Participant disposition
Overall study discontinuation
| Disposition | Ulixacaltamide, 231 participants in the safety population |
|---|---|
| Discontinued from the study | “88 (38.1%)” |
Disposition separately within each randomized-withdrawal treatment group
No evidence found.
Baseline characteristics
Baseline characteristics separately for the randomized-withdrawal treatment groups
No evidence found.
Efficacy results
Health-related quality of life and patient-reported outcomes
Generic health utility and preference-based quality-of-life outcomes
No evidence found.
Safety results
Drug-related TEAEs leading to discontinuation
Overall safety population
| Safety outcome | Ulixacaltamide, 231 participants |
|---|---|
| Any TEAE | “209 (90.5%)” |
| Severe TEAE | “17 (7.4%)” |
| Any serious adverse event | “4 (1.73%)” |
Safety separately within each randomized-withdrawal treatment group
No evidence found.
Study limitations
Summary
The randomized comparison included 80 selected responders from 238 enrolled participants and assessed withdrawal over four weeks. Its response-maintenance estimate therefore applies to participants who reached the response threshold after initial treatment, rather than all adults starting ulixacaltamide. The safety population included 231 participants; drug-related adverse events led to discontinuation in 28.1%. PGI-C and CGI-S comparisons during withdrawal did not reach statistical significance, with p-values of 0.087 and 0.055. The reported overall safety results should not be interpreted as adverse-event comparisons between the two randomized-withdrawal groups.