Expanded Access Program of Bitopertin For Participants With EPP or XLP
Trial statusAvailable
Trial phaseNot listed
Trial typeExpanded Access
Biological sexAll
Age12+
SponsorDisc Medicine, Inc
About this trial
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.
Eligibility criteria
Qualifiers
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.
Disqualifiers
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.
Trial design
Treatments tested in this trial
- DISC-1459
Treatment groups
No treatment groups listed
Sponsors and collaborators
Source ClinicalTrials.gov