Expanded Access Program of Bitopertin For Participants With EPP or XLP (NCT07603401) | Clinical Trial Compass
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Expanded Access Program of Bitopertin For Participants With EPP or XLP
United States
Plain-language summary
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.
Who can participate
Age range
12 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* 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.
Exclusion Criteria:
* 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…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.