Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

New drug bitopertin offers hope for sunlight pain relief in rare blood disorders

NCT ID NCT05883748

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 This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Sep 11, 2026 · Updated 11 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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

About 230 people

The number the study aims to enrol. It can still change while the study runs.

Started

Aug 2023

Expected to finish

Jun 2028

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: * Participants with diagnosis of EPP who are participating (or who have participated) in a prior Disc Medicine bitopertin study and who have completed the randomized treatment phase and End-of-Study visit * Aged ≥12 years upon study consent * Body weight ≥32 kg for participants \<18 years of age and BMI ≥18.5 kg/m2 for adult participants * Willing to practice highly effective methods of birth control (both males who have partners of childbearing potential and females of childbearing potential during the study, while taking study drug, and for at least 30 days after the last dose of study drug. * Negative urine or serum pregnancy test (females of childbearing potential). * Able to understand the study aims, procedures, and requirements, and provide written informed consent (and assent if necessary). * Able to comply with all study procedures. Exclusion Criteria: * Participants who have an ongoing SAE from a clinical study that is assessed by the investigator as related to bitopertin * Other medical or psychiatric condition or laboratory finding not specifically noted above that, in the judgement of the Investigator or Sponsor, would put the participant at unacceptable risk or otherwise preclude participation in the study * 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 * Planned treatment with afamelanotide or dersimelagon during the study * Planned use of any drugs or herbal remedies known to be strong inhibitors or inducers of cytochrome p450 (CYP)3A4 enzymes throughout the study * If female, pregnant, or breastfeeding * Participation in any other clinical protocol or investigational trial, other than Disc Medicine bitopertin trials, that involves administration of experimental therapy and/or therapeutic devices within 30 days of Day 1 * Score of PHQ-8 ≥10 at screening or imminent suicidal risk identified by the C-SSRS as defined as suicidal ideation with intent (Grade 4 or 5) within the last year or any suicidal behavior within the last 5 years. * Consumption of grapefruit/Seville orange and products containing these for 14 days prior to first dose of study drug and throughout the study

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.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    26 sites in 11 countries. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Atrium Health Wake Forest Baptist

    Winston-Salem, North Carolina, 27157, United States

  • 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

  • Einstein Medical Center

    Philadelphia, Pennsylvania, 19141, United States

  • Erasmus MC

    Rotterdam, 3015 GD, Netherlands

  • Fred Hutchinson Cancer Center

    Seattle, Washington, 98109, United States

  • Guy's and St Thomas' NHS Foundation Trust

    London, England, SE1 9RT, United Kingdom

  • 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

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, 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 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.