Active — Not RecruitingPhase 3

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

United States183 participantsStarted 2025-04-04

Plain-language 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.

Who can participate

Age range12 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Aged 12 years or older at the time of study consent.
✓. Diagnosis of EPP or XLP, based on medical history by ferrochelatase (FECH) or aminolevulinic acid synthase 2 (ALAS2) genotyping or by biochemical porphyrin analysis.
✓. 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
✓. Body weight ≥32 kg (ages 12 to \<18 years), body mass index ≥18.5 kg/m2 (ages ≥18 years) at screening.
✓. Washout of at least 2 months prior to screening of afamelanotide and dersimelagon, if applicable.
✓. 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).
✓. 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

What they're measuring

Average monthly total time in sunlight on days without pain from a phototoxic reaction between 10:00 to 18:00 (10:00 AM to 6:00 PM) after 6 months (24 weeks) of treatment

Timeframe: 24 weeks

Percent change from baseline in whole-blood metal-free PPIX levels at 6 months

Timeframe: 24 weeks

Safety and tolerability, as assessed by adverse events (AEs) and laboratory results, over the 6-month treatment period

Timeframe: 24 weeks

Trial details

NCT IDNCT06910358

SponsorDisc Medicine, Inc

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-09

View on ClinicalTrials.gov More Erythropoietic Protoporphyria (EPP) trials

Plain-language summary

Who can participate

Age range12 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Aged 12 years or older at the time of study consent.
✓. Diagnosis of EPP or XLP, based on medical history by ferrochelatase (FECH) or aminolevulinic acid synthase 2 (ALAS2) genotyping or by biochemical porphyrin analysis.
✓. 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
✓. Body weight ≥32 kg (ages 12 to \<18 years), body mass index ≥18.5 kg/m2 (ages ≥18 years) at screening.
✓. Washout of at least 2 months prior to screening of afamelanotide and dersimelagon, if applicable.
✓. 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).
✓. 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

What they're measuring

Average monthly total time in sunlight on days without pain from a phototoxic reaction between 10:00 to 18:00 (10:00 AM to 6:00 PM) after 6 months (24 weeks) of treatment

Timeframe: 24 weeks

Percent change from baseline in whole-blood metal-free PPIX levels at 6 months

Timeframe: 24 weeks

Safety and tolerability, as assessed by adverse events (AEs) and laboratory results, over the 6-month treatment period

Timeframe: 24 weeks