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New drug offers hope for patients who Can't tolerate sunlight

NCT ID NCT05308472

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

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.

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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

  • 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.