New drug bitopertin offers hope for sunlight pain relief in rare blood disorders
NCT ID NCT05883748
First seen Jun 27, 2026 · Last updated Aug 13, 2026 · Updated 6 times
Summary
This long-term study tests the safety and effectiveness of bitopertin (DISC-1459) in people with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP), rare conditions that cause severe pain from sunlight exposure. About 230 participants who previously took bitopertin will continue treatment at one of two dose levels. The goal is to see if the drug can increase time spent in sunlight without pain and reduce related symptoms.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Bitopertin (also called DISC-1459)
- What this could lead to
- If it works, this could provide a long-term treatment option for people with EPP or XLP, allowing them to spend more time in sunlight without pain.
- What could go wrong
- This is an open-label extension study, so there is no placebo group for comparison. Side effects and long-term safety are still being evaluated, and the drug may not work for everyone.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
Locations
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Atrium Health Wake Forest Baptist
Winston-Salem, North Carolina, 27157, United States
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CHU de Nantes - Hôtel Dieu, Service de dermatologie
Nantes, France, 44093, France
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Centre d'Investigation Clinique (CIC) Hôpital Bichat - Claude-Bernard
Paris, France, 75018, France
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Charité - Universitätsmedizin Berlin, Institute of Allergology
Berlin, Germany, 12203, Germany
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Einstein Medical Center
Philadelphia, Pennsylvania, 19141, United States
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Erasmus MC
Rotterdam, 3015 GD, Netherlands
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Fred Hutchinson Cancer Center
Seattle, Washington, 98109, United States
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Guy's and St Thomas' NHS Foundation Trust
London, England, SE1 9RT, United Kingdom
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Hospital Clinic de Barcelona
Barcelona, Spain, 08036, Spain
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Klinikum Chemnitz gGmbH
Chemnitz, Saxony, 09116, Germany
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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MetroBoston Clinical Partners
Boston, Massachusetts, 02135, United States
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Mount Sinai Hospital
New York, New York, 10029, United States
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Photobiology Unit, Salford Royal Hospital
Salford, M6 8HD, United Kingdom
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Remington-Davis Clinical Research
Columbus, Ohio, 43215, United States
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Royal Prince Alfred Hospital
Camperdown, New South Wales, 2050, Australia
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The Royal Melbourne Hospital
Parkville, Victoria, 3050, Australia
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UZ Leuven
Leuven, 3000, Belgium
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University of Alabama Hospital
Birmingham, Alabama, 35233, United States
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University of California San Francisco
San Francisco, California, 94117, United States
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University of Miami Miller School of Medicine
Miami, Florida, 33136, United States
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University of Texas
Galveston, Texas, 77550, United States
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