Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants with EPP
- Registration Number
- NCT05883748
- Lead Sponsor
- Disc Medicine, Inc
- Brief Summary
This is an open-label, long-term extension study to investigate the safety, tolerability and efficacy of DISC-1459 in participants with EPP.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- All
- Target Recruitment
- 200
- Participants with diagnosis of EPP who are participating (or who have participated) in a prior Disc Medicine bitopertin study and who have completed the randomized treatment phase and End-of-Study visit
- Aged ≥12 years upon study consent
- Body weight ≥32 kg for participants <18 years of age and BMI ≥18.5 kg/m2 for adult participants
- Participants who have an ongoing SAE from a clinical study that is assessed by the investigator as related to bitopertin
- Other medical or psychiatric condition or laboratory finding not specifically noted above that, in the judgement of the Investigator or Sponsor, would put the participant at unacceptable risk or otherwise preclude participation in the study
- Condition or concomitant medication that would confound the ability to interpret clinical, clinical laboratory, or participant diary data, including a major psychiatric condition that has had an exacerbation or required hospitalization in the last 6 months
- Planned treatment with afamelanotide or dersimelagon during the study
- Planned use of any drugs or herbal remedies known to be strong inhibitors or inducers of cytochrome p450 (CYP)3A4 enzymes throughout the study
- If female, pregnant, or breastfeeding
- Participation in any other clinical protocol or investigational trial, other than Disc Medicine bitopertin trials, that involves administration of experimental therapy and/or therapeutic devices within 30 days of Day 1
- Score of PHQ-8 ≥10 at screening or imminent suicidal risk identified by the C-SSRS as defined as suicidal ideation with intent (Grade 4 or 5) within the last year or any suicidal behavior within the last 5 years.
- Consumption of grapefruit/Seville orange and products containing these for 14 days prior to first dose of study drug and throughout the study
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description DISC-1459 Oral Dose Level 1 DISC-1459 Oral dose, once a day DISC-1459 Oral Dose Level 2 DISC-1459 Oral dose, once a day
- Primary Outcome Measures
Name Time Method Incidence of clinically abnormal vital signs up to 5 Years Incidence of clinically abnormal physical exam up to 5 Years Incidence of abnormal laboratory test results up to 5 Years Incidence of treatment-emergent adverse events up to 5 Years Assessment of Patient Health Questionnaire (PHQ-8) up to 5 Years 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.
Assessment of C-SSRS up to 5 Years 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.
- Secondary Outcome Measures
Name Time Method Change from baseline in whole blood metal-free PPIX levels 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 up to 5 Years Plasma Bitopertin Concentrations up to 5 Years
Trial Locations
- Locations (11)
University of Miami Miller School of Medicine
🇺🇸Miami, Florida, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Einstein Medical Center
🇺🇸Philadelphia, Pennsylvania, United States
University of Texas
🇺🇸Galveston, Texas, United States
Fred Hutchinson Cancer Center
🇺🇸Seattle, Washington, United States
Royal Prince Alfred Hospital
🇦🇺Camperdown, New South Wales, Australia
The Royal Melbourne Hospital
🇦🇺Parkville, Victoria, Australia
University of Alabama Hospital
🇺🇸Birmingham, Alabama, United States
University of California San Francisco
🇺🇸San Francisco, California, United States
Atrium Health Wake Forest Baptist
🇺🇸Winston-Salem, North Carolina, United States
Mount Sinai Hospital
🇺🇸New York, New York, United States