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

Conditions

• Erythropoietic Protoporphyria (EPP)
• X-Linked Porphyria (XLP)

Eligibility Criteria

Sex

ALL

Age

12 Years - N/A

Healthy Volunteers

Not accepted

Study Details

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.

Key Dates

Start date

Apr 30, 2026

Status verified

Apr 2026

Primary completion

Mar 1, 2027

Completion

Apr 1, 2027

Study Design

Enrollment

50 participants (estimated)

Primary Outcome Measure

Plasma Protoporphyrin IX (PPIX) Levels Over Time [ Time Frame: Screening; Baseline; Weeks 4, 8, 12, 16, 20, 24 ]

Central Contacts

• GondolaBio Inquiries
  650-249-2108
  [email protected]

Locations (5)

Find similar trials in Boston, MA

Related Studies

• GATEWAY: A Phase 2a Study of PORT-77 in Adults With Erythropoietic Protoporphyria
  PHASE2 · Enrolling By Invitation · Portal Therapeutics, Inc. · Tempe, Arizona