Expanded Access Program of Bitopertin For Participants With EPP or XLP

Available (expanded access)

Conditions studied: Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)

In brief

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.

Key facts

Study ID

NCT07603401

Run by

Disc Medicine, Inc

Last updated by the study team

2026-08-03

Who can join

Age: 12 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Written informed consent (and assent where applicable).
• Age ≥12 years at time of request.
• Diagnosis of erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP), confirmed by ferrochelatase (FECH) or aminolevulinic acid synthase 2 (ALAS2) genotyping or by biochemical porphyrin analysis. Prior confirmation is acceptable; re-testing is not required if documentation of prior diagnosis is available.
• Hemoglobin ≥10 g/dL at baseline.
• Females of childbearing potential must have a negative pregnancy test prior to enrollment.

You may not qualify if…

• Known hypersensitivity to bitopertin or its excipients.
• Patients with AST or ALT ≥3 × upper limit of normal (ULN), or total bilirubin ≥2 × ULN (unless due to Gilbert syndrome), or albumin below the lower limit of normal at screening.
• Patients with a history of liver transplantation or anticipated need for liver transplantation.
• Pregnancy, planned pregnancy during the program, or breastfeeding.
• Inability or unwillingness to comply with contraception requirements.
• Use of any of the following prohibited concomitant therapies:
• Strong CYP3A4 inhibitors
• Strong CYP3A4 inducers
• Grapefruit or Seville orange products
• Afamelanotide or dersimelagon
• Chronic opioid therapy (>7 consecutive days)
• Patients who are currently participating in a bitopertin clinical study.
• Patients who previously participated in a bitopertin study and discontinued the study due to an adverse event or for reasons that, in the judgement of the treating physician or Disc, would put the patient at unacceptable risk or otherwise preclude participation in the EAP.
• Patients who have received another investigational therapy within 30 days.
• History of suicidal ideation with intent (C-SSRS Grade 4 or 5) within the past year or suicidal behavior within the past 5 years.

Where it is running

• University of Miami Miller School of Medicine — Miami, Florida, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• MetroBoston Clinical Partners — Boston, Massachusetts, United States
• Mount Sinai Hospital — New York, New York, United States
• Wake Forest University — Winston-Salem, North Carolina, United States

Full record on ClinicalTrials.gov

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