Clinical Trials/NCT05883748

NCT05883748

Enrolling by Invitation

Phase 2

HELIOS: An Open-Label, Long-Term Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)

Disc Medicine, Inc13 sites in 2 countries230 target enrollmentAugust 31, 2023

ConditionsErythropoietic Protoporphyria

InterventionsDISC-1459

DrugsDISC-1459

Overview

Phase: Phase 2
Intervention: DISC-1459
Conditions: Erythropoietic Protoporphyria
Sponsor: Disc Medicine, Inc
Enrollment: 230
Locations: 13
Primary Endpoint: Incidence of clinically abnormal vital signs
Status: Enrolling by Invitation
Last Updated: 4 months ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

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

Registry

clinicaltrials.gov

Start Date

August 31, 2023

End Date

June 1, 2028

Last Updated

4 months ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Disc Medicine, Inc

Responsible Party

Sponsor

Eligibility Criteria

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
•Willing to practice highly effective methods of birth control (both males who have partners of childbearing potential and females of childbearing potential during the study, while taking study drug, and for at least 30 days after the last dose of study drug.
•Negative urine or serum pregnancy test (females of childbearing potential).
•Able to understand the study aims, procedures, and requirements, and provide written informed consent (and assent if necessary).
•Able to comply with all study procedures.

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

Arms & Interventions

DISC-1459 Oral Dose Level 2

Oral dose, once a day

Intervention: DISC-1459

DISC-1459 Oral Dose Level 1

Oral dose, once a day

Intervention: DISC-1459

Outcomes

Primary Outcomes

Incidence of clinically abnormal vital signs

Time Frame: up to 5 Years

Incidence of clinically abnormal physical exam

Time Frame: up to 5 Years

Incidence of abnormal laboratory test results

Time Frame: up to 5 Years

Incidence of treatment-emergent adverse events

Time Frame: up to 5 Years

Assessment of Patient Health Questionnaire (PHQ-8)

Time Frame: up to 5 Years

The Patient Health Questionnaire (PHQ-8), an 8-item participant-report measure for screening for depression and for establishing depression severity. The total score ranges from 0-24, with a higher score indicating greater depression symptom severity.

Assessment of C-SSRS

Time Frame: up to 5 Years

The C-SSRS is a clinician-rated scale that assesses suicidality from ideation to behaviors and monitors the potential emergence of suicidality in clinical studies.

Secondary Outcomes

Change from baseline in daily daylight tolerance, as assessed by total hours spent in the sunlight without pain and average time to first prodromal syndrome in sunlight(up to 5 Years)
Plasma Bitopertin Concentrations(up to 5 Years)
Change from baseline in whole blood metal-free PPIX levels(up to 5 Years)