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

NCT05883748 · Status: ENROLLING_BY_INVITATION · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 230

Last updated 2025-12-18

No results posted yet for this study

Summary

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

Conditions

Interventions

DRUG

DISC-1459

DISC-1459 dose level 1

DRUG

DISC-1459

DISC-1459 dose level 2

Sponsors & Collaborators

  • Disc Medicine, Inc

    lead INDUSTRY

Principal Investigators

  • Will Savage, MD PhD · Disc Medicine

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-08-31
Primary Completion
2028-06-30
Completion
2028-06-30
FDA Drug
Yes

Countries

  • United States
  • Australia

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05883748 on ClinicalTrials.gov