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New pill could let Sun-Sensitive patients enjoy the outdoors

NCT ID NCT06910358

Disease control Sponsor: Disc Medicine, Inc Source: ClinicalTrials.gov ↗

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 This study
Running, but no longer taking on new participants.
Completed
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Sep 03, 2026 · Updated 12 times

Summary

This Phase 3 trial tests whether bitopertin, a daily pill, can help people with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP) spend more time in sunlight without pain. About 183 participants aged 12 and older will take bitopertin or a placebo for 6 months. The study measures pain-free sun exposure and changes in blood porphyrin levels.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Bitopertin
What this could lead to
If it works, this could provide a daily pill that allows people with EPP or XLP to spend more time in sunlight without pain.
What could go wrong
This is a Phase 3 trial, but results are not yet known. The drug may not work better than placebo, and side effects are possible.
Why investors are watching

Disc Medicine is running a Phase 3 trial of bitopertin in patients with the rare light-sensitivity diseases EPP and XLP. The trial tests whether the drug lets patients spend more time in sunlight without pain, which is the main symptom of these conditions. For a small company, this readout is a major event because it could determine whether the drug reaches the market.

If it works: If the trial succeeds, Disc Medicine could have a new treatment for a condition with few options, which would be a meaningful step for the company. A positive result could also support regulatory approval and make the drug a commercial product.

If it fails: If the trial fails or is delayed, Disc Medicine would face a setback with no approved product from this program. Clinical trials often fail, so investors should expect that possibility.

AI-written from the trial record. Speculative, and not investment advice.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

183 people

The number who actually took part.

Started

Apr 2025

Expected to finish

Oct 2026

An estimate. End dates often move.

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

12 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 12 years or older at the time of study consent. 2. Diagnosis of EPP or XLP, based on medical history by ferrochelatase (FECH) or aminolevulinic acid synthase 2 (ALAS2) genotyping or by biochemical porphyrin analysis. 3. Minimum daily Sun Exposure Diary compliance ≥85% on Days -14 through Day -1, inclusive, during screening, and at least 1 successfully completed Sun Exposure Challenge (adults only, as this assessment is optional for adolescents) or historical recall of time to prodrome 4. Body weight ≥32 kg (ages 12 to \<18 years), body mass index ≥18.5 kg/m2 (ages ≥18 years) at screening. 5. Washout of at least 2 months prior to screening of afamelanotide and dersimelagon, if applicable. 6. Aspartate aminotransferase and alanine transaminase \<3× upper limit of normal (ULN)and total bilirubin \<2× ULN (unless documented Gilbert syndrome) at screening. Albumin \>lower limit of normal (LLN). 7. Willing to practice highly effective methods of birth control (both males who have partners of childbearing potential and females of childbearing potential during screening, while taking study drug, and for at least 30 days after the last dose of study drug). Exclusion Criteria: 1. Major surgery within 8 weeks before screening or incomplete recovery from any previous surgery. 2. Other than EPP or XLP, an inherited intrinsic or extrinsic red cell disease associated with anemia. 3. Known hypersensitivity to any component of the study drug. 4. History of liver transplantation or anticipated need for liver transplantation. 5. History of alcohol dependence or excessive alcohol consumption, as assessed by the Investigator. 6. Active 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. Prior exposure to bitopertin. 10. Concurrent or planned treatment with afamelanotide or dersimelagon during the study period. 11. 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. 12. New treatment for anemia, including initiation of iron supplementation, within 1 month of screening. 13. Current or planned use of any drugs or herbal remedies known to be strong or moderate inhibitors or inducers of cytochrome P450 (CYP)3A4 enzymes for 28 days prior to the first dose and throughout the study. 14. Current or planned treatment with antipsychotic medication. Laboratory Exclusions: 15. Hemoglobin \<10 g/dL at screening. Miscellaneous: 16. Participation in other interventional clinical studies within 30 days prior to screening. 17. If female, pregnant or breastfeeding.

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Conditions

The condition(s) this trial relates to.

erythropoietic protoporphyria porphyria X-linked erythropoietic protoporphyria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Erythropoietic protoporphyria (EPP) X-linked protoporphyria (XLP)

Contacts and locations

Locations

  • CHU de Nantes - Hôtel Dieu, Service de dermatologie

    Nantes, France, 44093, France

  • Centre d'Investigation Clinique (CIC) Hôpital Bichat - Claude-Bernard

    Paris, France, 75018, France

  • Charité - Universitätsmedizin Berlin, Institute of Allergology

    Berlin, Germany, 12203, Germany

  • Children's Health Ireland (CHI)

    Dublin, D12N512, Ireland

  • Clinical Research Centre, Ninewells Hospital & Medical School , NHS Tayside

    Dundee, Scotland, DD1 9SY, United Kingdom

  • Erasmus MC

    Rotterdam, The Netherlands, 3015 GD, Netherlands

  • Guy's and St Thomas' NHS Foundation Trust

    London, England, SE1 9RT, United Kingdom

  • Henry Ford Health System

    Detroit, Michigan, 48202, United States

  • Hospital Clinic de Barcelona

    Barcelona, Spain, 08036, Spain

  • Instituto Dermatologico San Gallicano Istituti Fisioterapici Ospitalieri IRCCS

    Roma, 53-00144, Italy

  • Karolinska University Hospital

    Stockholm, Sweden, 141 86, Sweden

  • Klinikum Chemnitz gGmbH

    Chemnitz, Saxony, 09116, Germany

  • Marvel Clinical Research

    Huntington Beach, California, 92647, United States

  • Massachusetts General Hospital

    Boston, Massachusetts, 02115, United States

  • MetroBoston Clinical Partners

    Boston, Massachusetts, 02135, United States

  • Mount Sinai Hospital

    New York, New York, 10029, United States

  • Photobiology Unit, Salford Royal Hospital

    Salford, M6 8HD, United Kingdom

  • Remington-Davis Clinical Research

    Columbus, Ohio, 43215, United States

  • Royal Prince Alfred Hospital

    Camperdown, New South Wales, 2050, Australia

  • The Royal Melbourne Hospital

    Parkville, Victoria, 3050, Australia

  • UZ Leuven

    Leuven, 3000, Belgium

  • University of Alberta

    Edmonton, Alberta, T6G 2R3, Canada

  • University of California San Francisco

    San Francisco, California, 94143, United States

  • University of Miami Miller School of Medicine

    Miami, Florida, 33146, United States

  • University of Texas Medical Branch

    Galveston, Texas, 77550, United States

  • University of Washington

    Seattle, Washington, 98195, United States

  • Wake Forest University

    Winston-Salem, North Carolina, 27157, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.