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

Part of paid clinical trials in Birmingham, Alabama.

Sponsor
Disc Medicine, Inc
Study ID
NCT05883748
Phase
PHASE2/PHASE3
Status
Enrolling By Invitation

Conditions

  • Erythropoietic Protoporphyria

Eligibility Criteria

Sex
ALL
Age
12 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • DISC-1459 — DRUG
    DISC-1459 dose level 1
  • DISC-1459 — DRUG
    DISC-1459 dose level 2

Study Details

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

Key Dates

Start date
Aug 31, 2023
Status verified
Dec 2025
Primary completion
Jun 30, 2028
Completion
Jun 30, 2028

Study Design

Enrollment
230 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: DISC-1459 Oral Dose Level 1
    Oral dose, once a day
  • Experimental: DISC-1459 Oral Dose Level 2
    Oral dose, once a day

Primary Outcome Measure

Incidence of treatment-emergent adverse events [ Time Frame: up to 5 Years ]

Locations (11)

FacilityCityStateZIPSite coordinators
University of Alabama HospitalBirminghamAlabama35233-
University of California San FranciscoSan FranciscoCalifornia94117-
University of Miami Miller School of MedicineMiamiFlorida33136-
Massachusetts General HospitalBostonMassachusetts02114-
MetroBoston Clinical PartnersBostonMassachusetts02135-
Mount Sinai HospitalNew YorkNew York10029-
Atrium Health Wake Forest BaptistWinston-SalemNorth Carolina27157-
Remington-Davis Clinical ResearchColumbusOhio43215-
Einstein Medical CenterPhiladelphiaPennsylvania19141-
University of TexasGalvestonTexas77550-
Fred Hutchinson Cancer CenterSeattleWashington98109-

Find similar trials in Birmingham, AL

By research site
University of Alabama Hospital· Birmingham, ALUniversity of California San Francisco· San Francisco, CAUniversity of Miami Miller School of Medicine· Miami, FLMassachusetts General Hospital· Boston, MAMetroBoston Clinical Partners· Boston, MAMount Sinai Hospital· New York, NY