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Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Participants With EPP

NCT05308472 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 75

Last updated 2026-01-08

Study results available
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Summary

This is a Phase 2, multi-center, double-blind, placebo-controlled, parallel group study of bitopertin to evaluate the safety, tolerability, efficacy, and PPIX concentration change in participants with EPP. Participants may roll over to an open label extension portion after completing the double-blind treatment period.

Conditions

Interventions

DRUG

DISC-1459

Oral dose level 1, once a day for 120 days

DRUG

DISC-1459

Oral dose level 2, once a day for 120 days

DRUG

Placebo

Oral dose, once a day for 120 days

Sponsors & Collaborators

  • Disc Medicine, Inc

    lead INDUSTRY

Principal Investigators

  • Will Savage, MD PhD · Disc Medicine

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-10-31
Primary Completion
2024-02-20
Completion
2024-08-23
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Drugs
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 NCT05308472 on ClinicalTrials.gov