RECRUITING

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

Conditions

Erythropoietic Protoporphyria (EPP)X-Linked Protoporphyria (XLP)EPP XLP DISC-1459 RO4917838 porphyria

Quick Navigation

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

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)

Quick Facts

Study Start:2025-04

Study Completion:2026-11

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06910358

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:12 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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).	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. 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. 15. Hemoglobin \<10 g/dL at screening. 16. Participation in other interventional clinical studies within 30 days prior to screening. 17. If female, pregnant or breastfeeding.

Inclusion Criteria

Exclusion 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).

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.
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.
15. Hemoglobin \<10 g/dL at screening.
16. Participation in other interventional clinical studies within 30 days prior to screening.
17. If female, pregnant or breastfeeding.

Contacts and Locations

Study Contact

Disc Medicine Clinical Trials

CONTACT

(617) 674 9274

clinicaltrials@discmedicine.com

Principal Investigator

Will Savage, MD, PhD

STUDY_DIRECTOR

Disc Medicine

Study Locations (Sites)

Marvel Clinical Research

Huntington Beach, California, 92647

United States

MetroBoston Clinical Partners

Boston, Massachusetts, 02135

United States

Collaborators and Investigators

Sponsor: Disc Medicine, Inc

Will Savage, MD, PhD, STUDY_DIRECTOR, Disc Medicine

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-04

Study Completion Date2026-11

Study Record Updates

Study Start Date2025-04

Study Completion Date2026-11

Terms related to this study

Keywords Provided by Researchers

EPP
XLP
DISC-1459
RO4917838
porphyria

Additional Relevant MeSH Terms

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