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DISC-1459-301

EPP | XLP

This study is testing a drug called bitopertin to see if it can help people with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP) spend more time in sunlight without skin pain or burning. These rare conditions cause a substance called protoporphyrin IX to build up in the blood, which can lead to painful skin reactions to sunlight and whcih may affect the liver. Bitopertin may lower this substance in the body. Participants will receive either bitopertin or a placebo (a look-alike pill with no active medicine) to compare results. The study will also look at how participants feel on the treatment and how well they tolerate any side effects.

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Conditions de participation

  • Sexe:

    Female, Any, Male

  • Âges admissibles:

    18 to 90

Critères de participation

Inclusion Criteria:
1.Aged 18 years or older.
2.Diagnosis of EPP or XLP confirmed by medical history, genetic testing, or lab analysis.
3.At least 85% completion of a Sun Exposure Diary during the screening period, and,
4.either a completed Sun Exposure Challenge or a recalled time to early sun-related symptoms (prodrome). The challenge is optional for adolescents.
5.Body weight of at least 32 kilograms.
6.No use of afamelanotide or dersimelagon in the 2 months prior to screening.
7.Liver enzyme levels (AST and ALT) within the normal range.
8.Willingness to use highly effective birth control methods during the study and for at least 30 days after the last dose, for both males with partners of childbearing potential and females of childbearing potential.
9.Negative pregnancy test at screening and before the first dose (for females of childbearing potential).
10.Ability to understand the study and provide signed informed consent (and assent if underage).
11.Ability to follow all study procedures.
Exclusion Criteria:
1.Had major surgery recently or hasn’t recovered from past surgery.
2.Has a blood disorder (other than EPP/XLP), like G6PD or sickle cell disease.
3.Allergic to anything in the study drug or has had a liver transplant.
4.Has alcohol dependence or active HIV, hepatitis B, or C.
5.Shows signs of depression or has had suicidal thoughts or behavior.
6.Has a serious health or mental condition that could make participation unsafe.
7.Has taken bitopertin before or is using other treatments like afamelanotide.
8.Recently used or may need strong pain meds (opioids) or anemia treatments.
9.Taking medications or supplements that interfere with drug processing.
10.Is pregnant, breastfeeding, or has low blood count (hemoglobin <10 g/dL).

Lieu de l'étude

University of Alberta
University of Alberta
Edmonton, Alberta
Canada

Contactez l'équipe d'étude

Étude parrainée par
University of Alberta
Participants recherchés
Plus d'informations
ID de l'étude: Pro00155144