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This is an open-label, long-term extension study to investigate the safety, tolerability and efficacy of DISC-1459 in participants with EPP.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| DISC-1459 Oral Dose Level 1 | Experimental | Oral dose, once a day |
|
| DISC-1459 Oral Dose Level 2 | Experimental | Oral dose, once a day |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| DISC-1459 | Drug | DISC-1459 dose level 1 |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of treatment-emergent adverse events | up to 5 Years | |
| 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 | |
| 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. | 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. | up to 5 Years |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | |
| Change from baseline in whole blood metal-free PPIX levels | up to 5 Years |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Will Savage, MD PhD | Disc Medicine | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama Hospital | Birmingham | Alabama | 35233 | United States | ||
| University of California San Francisco |
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| DISC-1459 | Drug | DISC-1459 dose level 2 |
|
|
| Plasma Bitopertin Concentrations | up to 5 Years |
| San Francisco |
| California |
| 94117 |
| United States |
| University of Miami Miller School of Medicine | Miami | Florida | 33136 | United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| MetroBoston Clinical Partners | Boston | Massachusetts | 02135 | United States |
| Mount Sinai Hospital | New York | New York | 10029 | United States |
| Atrium Health Wake Forest Baptist | Winston-Salem | North Carolina | 27157 | United States |
| Remington-Davis Clinical Research | Columbus | Ohio | 43215 | United States |
| Einstein Medical Center | Philadelphia | Pennsylvania | 19141 | United States |
| University of Texas | Galveston | Texas | 77550 | United States |
| Fred Hutchinson Cancer Center | Seattle | Washington | 98109 | United States |
| Royal Prince Alfred Hospital | Camperdown | New South Wales | 2050 | Australia |
| The Royal Melbourne Hospital | Parkville | Victoria | 3050 | Australia |
| CHU de Nantes - Hôtel Dieu, Service de dermatologie | Nantes | France | 44093 | France |
| Centre d'Investigation Clinique (CIC) Hôpital Bichat - Claude-Bernard | Paris | France | 75018 | France |
| Charité - Universitätsmedizin Berlin, Institute of Allergology | Berlin | Germany | 12203 | Germany |
| Erasmus MC | Rotterdam | 3015 GD | Netherlands |
| Hospital Clinic de Barcelona | Barcelona | Spain | 08036 | Spain |
| Guy's and St Thomas' NHS Foundation Trust | London | England | SE1 9RT | United Kingdom |
| ID | Term |
|---|---|
| D046351 | Protoporphyria, Erythropoietic |
| D011164 | Porphyrias |
| ID | Term |
|---|---|
| D017094 | Porphyrias, Hepatic |
| D008107 | Liver Diseases |
| D004066 | Digestive System Diseases |
| D012873 | Skin Diseases, Genetic |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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| ID | Term |
|---|---|
| C550631 | (4-(3-fluoro-5-trifluoromethylpyridin-2-yl)piperazin-1-yl)(5-methanesulfonyl-2-(2,2,2-trifluoro-1-methylethoxy)phenyl)methanone |
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