New drug offers hope for patients who Can't tolerate sunlight
NCT ID NCT05308472
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested a drug called bitopertin in 75 adults with erythropoietic protoporphyria (EPP), a rare condition that causes severe pain from sunlight exposure. The goal was to see if the drug is safe and can lower levels of a harmful substance in the blood. Participants received either bitopertin or a placebo, and some later joined an open-label extension.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
-
75 people
The number who actually took part.
- Started
-
Oct 2022
- Finished
-
Aug 2024
- Lead sponsor
-
A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 years and older
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Aged 18 years or older at the time of signing the informed consent form (ICF). 2. Diagnosis of EPP, based on medical history by ferrochelatase ( FECH) genotyping or by biochemical porphyrin analysis. 3. Body weight ≥50 kg. 4. Washout of at least 2 months prior to Screening of afamelanotide and dersimelagon, if applicable. 5. Aspartate aminotransferase (AST) and alanine transaminase (ALT) \<2× upper limit of normal (ULN) and total bilirubin \<ULN (unless documented Gilbert syndrome) at Screening. Albumin \>lower limit of normal (LLN). Exclusion Criteria: Medical History: 1. Major surgery within 8 weeks before Screening or incomplete recovery from any previous surgery. 2. Other than EPP, an inherited or acquired red cell disease associated with anemia. 3. A history or known allergic reaction to any investigational product excipients or history of anaphylaxis to any food or drug. 4. History of liver transplantation. 5. History of alcohol dependence or excessive alcohol consumption, as assessed by the Investigator. 6. Human immunodeficiency virus (HIV), active Hepatitis B, or C. 7. Other medical or psychiatric condition or laboratory finding not specifically noted above that, in the judgment of the Investigator or Sponsor, would put the participant at unacceptable risk or otherwise preclude the participant from participating in the study 8. Condition or concomitant medication that would confound the ability to interpret clinical, clinical laboratory, or participant diary data, including a major psychiatric condition that has had an exacerbation or required hospitalization in the last 6 months. Treatment History: 9. Concurrent or planned treatment with afamelanotide or dersimelagon during the study period. 10. Treatment with opioids for any period \>7 days in the 2 months prior to screening or anticipated to require opioid use for \>7 days at any point during the study. 11. New treatment for anemia, including initiation of iron supplementation, in the 2 months prior to Screening. 12. Current or planned use of any drugs or herbal remedies known to be strong inhibitors or inducers of CYP3A4 enzymes for 28 days prior to the first dose and throughout the study. Laboratory Exclusions: 13. Hemoglobin \<10 g/dL at Screening.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Erythropoietic protoporphyria are added.
Genom att skicka in godkänner du våra Användarvillkor
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
-
Atrium Health Wake Forest Baptist
Winston-Salem, North Carolina, 27157, United States
-
Einstein Medical Center
Philadelphia, Pennsylvania, 19141, United States
-
Fred Hutchinson Cancer Center
Seattle, Washington, 98109, United States
-
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
-
Mount Sinai Hospital
New York, New York, 10029, United States
-
University of Alabama Hospital
Birmingham, Alabama, 35233, United States
-
University of California San Francisco
San Francisco, California, 94117, United States
-
University of Miami Miller School of Medicine
Miami, Florida, 33136, United States
-
University of Texas
Galveston, Texas, 77550, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.