Expanded Access Program of Bitopertin For Participants With EPP or XLP
NCT07603401 · Status: TEMPORARILY_NOT_AVAILABLE · Type: EXPANDED_ACCESS
Last updated 2026-05-22
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.
Conditions
- Erythropoietic Protoporphyria (EPP)
- X-Linked Protoporphyria (XLP)
Interventions
- DRUG
-
DISC-1459
DISC-1459, 60 mg oral dose once a day
Sponsors & Collaborators
-
Disc Medicine, Inc
lead INDUSTRY
Principal Investigators
-
Will Savage, MD, PhD · Disc Medicine
Eligibility
- Min Age
- 12 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
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