New hope for rare sun allergy: bitopertin offered to patients with no options
NCT ID NCT07603401
First seen Jun 25, 2026 · Last updated Jul 01, 2026 · Updated 3 times
Summary
This expanded access program provides bitopertin (DISC-1459) to patients aged 12 and older with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP) who have no satisfactory treatment options in the US. The goal is to offer access and gather safety information. Participants take a 60 mg oral dose once daily.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
bitopertin (DISC-1459)
What this could lead to
If successful, this could provide a treatment option for people with EPP and XLP who currently have no satisfactory therapies.
What could go wrong
This is an expanded access program, not a formal trial, so data on effectiveness is limited. It may not work for everyone, and side effects are possible.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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MetroBoston Clinical Partners
AVAILABLEBoston, Massachusetts, 02135, United States
Contact
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Mount Sinai Hospital
AVAILABLENew York, New York, 10029, United States
Contact
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University of Miami Miller School of Medicine
AVAILABLEMiami, Florida, 33146, United States
Contact