This is an open-label, long-term extension study to investigate the safety, tolerability and efficacy of DISC-1459 in participants with EPP.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
230
University of Alabama Hospital
Birmingham, Alabama, United States
University of California San Francisco
San Francisco, California, United States
University of Miami Miller School of Medicine
Miami, Florida, United States
Incidence of treatment-emergent adverse events
Time frame: up to 5 Years
Incidence of clinically abnormal vital signs
Time frame: up to 5 Years
Incidence of clinically abnormal physical exam
Time frame: up to 5 Years
Incidence of abnormal laboratory test results
Time frame: up to 5 Years
Assessment of Patient Health Questionnaire (PHQ-8)
The Patient Health Questionnaire (PHQ-8), an 8-item participant-report measure for screening for depression and for establishing depression severity. The total score ranges from 0-24, with a higher score indicating greater depression symptom severity.
Time frame: up to 5 Years
Assessment of C-SSRS
The C-SSRS is a clinician-rated scale that assesses suicidality from ideation to behaviors and monitors the potential emergence of suicidality in clinical studies.
Time frame: up to 5 Years
Change from baseline in daily daylight tolerance, as assessed by total hours spent in the sunlight without pain and average time to first prodromal syndrome in sunlight
Time frame: up to 5 Years
Change from baseline in whole blood metal-free PPIX levels
Time frame: up to 5 Years
Plasma Bitopertin Concentrations
Time frame: up to 5 Years
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Massachusetts General Hospital
Boston, Massachusetts, United States
MetroBoston Clinical Partners
Boston, Massachusetts, United States
Mount Sinai Hospital
New York, New York, United States
Atrium Health Wake Forest Baptist
Winston-Salem, North Carolina, United States
Remington-Davis Clinical Research
Columbus, Ohio, United States
Einstein Medical Center
Philadelphia, Pennsylvania, United States
University of Texas
Galveston, Texas, United States
...and 3 more locations