Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants with EPP

Disc Medicine

Status and phase: Invitation-only; Phase 3; Phase 2

Conditions: Erythropoietic Protoporphyria

Treatments: Drug: DISC-1459

Study type: Interventional

Funder types: Industry

Identifiers

NCT05883748

DISC-1459-501

Details and patient eligibility

About

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

Enrollment: 200 estimated patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Participants with diagnosis of EPP who are participating (or who have participated) in a prior Disc Medicine bitopertin study and who have completed the randomized treatment phase and End-of-Study visit
• Aged ≥12 years upon study consent
• Body weight ≥32 kg for participants <18 years of age and BMI ≥18.5 kg/m2 for adult participants

Exclusion criteria

• Participants who have an ongoing SAE from a clinical study that is assessed by the investigator as related to bitopertin
• Other medical or psychiatric condition or laboratory finding not specifically noted above that, in the judgement of the Investigator or Sponsor, would put the participant at unacceptable risk or otherwise preclude participation in the study
• 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
• Planned treatment with afamelanotide or dersimelagon during the study
• Planned use of any drugs or herbal remedies known to be strong inhibitors or inducers of cytochrome p450 (CYP)3A4 enzymes throughout the study
• If female, pregnant, or breastfeeding
• Participation in any other clinical protocol or investigational trial, other than Disc Medicine bitopertin trials, that involves administration of experimental therapy and/or therapeutic devices within 30 days of Day 1
• Score of PHQ-8 ≥10 at screening or imminent suicidal risk identified by the C-SSRS as defined as suicidal ideation with intent (Grade 4 or 5) within the last year or any suicidal behavior within the last 5 years.
• Consumption of grapefruit/Seville orange and products containing these for 14 days prior to first dose of study drug and throughout the study

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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200 participants in 2 patient groups

DISC-1459 Oral Dose Level 1

Experimental group

Description:

Oral dose, once a day

Treatment:

Drug: DISC-1459

Drug: DISC-1459

DISC-1459 Oral Dose Level 2

Experimental group

Description:

Oral dose, once a day

Treatment:

Drug: DISC-1459

Drug: DISC-1459