Erythropoietic Protoporphyria (EPP)

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Review clinical trials related to Erythropoietic Protoporphyria (EPP). Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Available

Expanded Access Program of Bitopertin For Participants With EPP or XLP

The goal of this expanded access program is to provide bitopertin to patients with EPP and XLP who have no satisfactory treatment options available in the US and to learn if bitopertin is safe to treat EPP and XLP.

Trial details
Age: 12+Biological sex: AllType: Expanded AccessSponsor: Disc Medicine, IncUpdated: Jun 30, 2026Locations: 3
Eligibility criteria

Written informed consent (and assent where applicable). [+4]

Known hypersensitivity to bitopertin or its excipients. [+13]

Status: Recruiting

Observational Study of Adults and Adolescents With Erythropoietic Protoporphyria (EPP) and X-linked Porphyria (XLP)

The purpose of this study is to collect information about the effects of EPP/XLP in adults and adolescents. This is an observational study in which participants will not receive any treatment. Study details include: * The study duration will be up to 6 months for each participant. * After Screening, participants will have a Baseline (Day 1) visit and return for visits every 4 weeks through 24 weeks. * Assessments to evaluate EPP/XLP will include clinical laboratory tests, physical exams, vital signs measurement, blood draws for PPIX concentration, reporting of EPP/XLP-related symptoms (including completion of a daily diary), light dosimetry.

Participants needed: 50
Trial details
Age: 12+Biological sex: AllType: ObservationalSponsor: Portal Therapeutics, Inc.Updated: May 5, 2026Locations: 19
Eligibility criteria

Aged 12 years and older. [+10]

Diagnosis of another porphyria or another photodermatosis that may confound the... [+3]