Bitopertin for EPP

Recruiting at 11 trial locations

Overseen By Disc Medicine Clinical Trials

Age: Any Age

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Disc Medicine, Inc

Must be taking: Bitopertin

Must not be taking: Afamelanotide, Dersimelagon, CYP3A4 inhibitors

Disqualifiers: Pregnancy, Suicidal risk, Major psychiatric, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: up to 5 years

200 Participants Needed

Trial Summary

What is the purpose of this trial?

This is an open-label, long-term extension study to investigate the safety, tolerability and efficacy of DISC-1459 in participants with EPP.

Do I have to stop taking my current medications for the trial?

The trial requires that you do not take certain medications that affect liver enzymes (CYP3A4) during the study. If you are on such medications, you may need to stop them. It's best to discuss your current medications with the study team to see if any changes are needed.

Research Team

Will Savage, MD PhD

Principal Investigator

Disc Medicine

Eligibility Criteria

This trial is for individuals aged ≥12 years with a confirmed diagnosis of Erythropoietic Protoporphyria (EPP) who have previously participated in a bitopertin study. Adults must have a BMI ≥18.5 kg/m2, and minors must weigh at least 32 kg. Exclusions include ongoing severe side effects from previous studies related to bitopertin, certain medical or psychiatric conditions, pregnancy, breastfeeding, recent participation in other clinical trials, and use of specific drugs.

Inclusion Criteria

I am 12 years old or older.

I have EPP and have completed a prior Disc Medicine bitopertin study.

Exclusion Criteria

I will be treated with afamelanotide or dersimelagon in the study.

I am not pregnant or breastfeeding.

I am experiencing a severe side effect from bitopertin in another study.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive DISC-1459 (Bitopertin) orally once a day

up to 5 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Long-term study to investigate the safety, tolerability, and efficacy of DISC-1459 in participants with EPP

up to 5 years

Treatment Details

Interventions

• DISC-1459

Trial Overview The trial is testing the long-term safety and effectiveness of DISC-1459 (Bitopertin) for treating EPP. It's an open-label extension study which means everyone knows they are receiving Bitopertin without any placebo control.

Participant Groups

Group I: DISC-1459 Oral Dose Level 2 Experimental Treatment 1 Intervention

Oral dose, once a day

Group II: DISC-1459 Oral Dose Level 1 Experimental Treatment 1 Intervention

Oral dose, once a day

Who Is Running the Clinical Trial?

Disc Medicine, Inc

Lead Sponsor

Trials

6

Recruited

580+