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Clinical Trials in the USA / NCT06910358
Active, not recruiting Phase 3

Study of Bitopertin in Participants With EPP or XLP (APOLLO)

NCT06910358 · tracked via the Priya Life Science USA tracker
Sponsor
Disc Medicine, Inc
Phase
Phase 3
Started
2025-04-04
Last updated
2026-09-02

Condition(s) studied

Erythropoietic Protoporphyria (EPP)X-Linked Protoporphyria (XLP)

Investigational drug(s) / intervention(s)

PlaceboDISC-1459

Placebo: Oral dose, once a day for 24 weeks

DISC-1459: Oral dose, once a day for 24 weeks

Study summary

The goal of this clinical trial is to learn if bitopertin works and is safe to treat EPP or XLP in participants 12 years or older. The main questions it aims to answer are:

* Whether bitopertin increases pain-free sunlight exposure after 6 months of treatment in participants with EPP or XLP.
* How PPIX concentration levels change from before bitopertin treatment to after 6 months of treatment.

Researchers will compare bitopertin to a placebo look-alike substance that contains no drug.

Participants will complete daily questionnaires and attend study visits for assessments.

Eligibility

Sex
ALL
Min age
12 Years
Max age
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (27)

FacilityCityRegionStatus
Marvel Clinical Research Huntington Beach California
University of California San Francisco San Francisco California
University of Miami Miller School of Medicine Miami Florida
Massachusetts General Hospital Boston Massachusetts
MetroBoston Clinical Partners Boston Massachusetts
Henry Ford Health System Detroit Michigan
Mount Sinai Hospital New York New York
Wake Forest University Winston-Salem North Carolina
Remington-Davis Clinical Research Columbus Ohio
University of Texas Medical Branch Galveston Texas
University of Washington Seattle Washington
Royal Prince Alfred Hospital Camperdown New South Wales
The Royal Melbourne Hospital Parkville Victoria
UZ Leuven Leuven Belgium
University of Alberta Edmonton Alberta
CHU de Nantes - Hôtel Dieu, Service de dermatologie Nantes France
Centre d'Investigation Clinique (CIC) Hôpital Bichat - Claude-Bernard Paris France
Charité - Universitätsmedizin Berlin, Institute of Allergology Berlin Germany
Klinikum Chemnitz gGmbH Chemnitz Saxony
Children's Health Ireland (CHI) Dublin Ireland
Instituto Dermatologico San Gallicano Istituti Fisioterapici Ospitalieri IRCCS Roma Italy
Erasmus MC Rotterdam The Netherlands
Hospital Clinic de Barcelona Barcelona Spain
Karolinska University Hospital Stockholm Sweden
Guy's and St Thomas' NHS Foundation Trust London England
Clinical Research Centre, Ninewells Hospital & Medical School , NHS Tayside Dundee Scotland
Photobiology Unit, Salford Royal Hospital Salford United Kingdom

More Disc Medicine, Inc trials in the USA

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06910358 on ClinicalTrials.gov ↗ ← All trials in the USA