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Study of Bitopertin in Participants With EPP or XLP (APOLLO) | Clinical Trials | Clareo Health

RECRUITINGPHASE3

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

APOLLO: A Randomized, Double-Blind, Placebo-Controlled Study of Bitopertin to Evaluate the Efficacy, Safety, and Tolerability in Participants With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)

NCT06910358•Disc Medicine, Inc

View on ClinicalTrials.gov

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

Age

12 - No limit years

Sex

ALL

Healthy Volunteers

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 more criteria

Locations (23)

Marvel Clinical Research

Huntington Beach, California, United States

University of California San Francisco

San Francisco, California, United States

University of Miami Miller School of Medicine

Miami, Florida, United States

Massachusetts General Hospital

Boston, Massachusetts, United States

MetroBoston Clinical Partners

Boston, Massachusetts, United States

Henry Ford Health System

Detroit, Michigan, United States

Mount Sinai Hospital

New York, New York, United States

Wake Forest University

Winston-Salem, North Carolina, United States

Remington-Davis Clinical Research

Columbus, Ohio, United States

University of Texas Medical Branch

Galveston, Texas, United States

Key Dates

Start Date

April 4, 2025

Primary Completion Date

October 1, 2026

Completion Date

November 1, 2026

Last Updated

February 12, 2026

Enrollment

150

ESTIMATED participants

Conditions

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

Interventions

Placebo

DRUG

DISC-1459

DRUG

Contacts

Disc Medicine Clinical Trials

CONTACT

(617) 674 9274 clinicaltrials@discmedicine.com

GATEWAY: A Phase 2a Study of PORT-77 in Adults With Erythropoietic Protoporphyria

NCT06971900

Erythropoietic Protoporphyria (EPP)

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TERMINATEDNA

Effect of Isoniazid on Protoporphyrin Levels in Erythropoietic Protoporphyria

NCT01550705

Erythropoietic Protoporphyria (EPP)X Linked Erythropoietic Protoporphyria

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 12, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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