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

PHASE2CompletedINTERVENTIONAL
Enrollment

75

Participants

Timeline

Start Date

October 31, 2022

Primary Completion Date

February 20, 2024

Study Completion Date

August 23, 2024

Conditions
Erythropoietic Protoporphyria
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

DRUG

DISC-1459

Oral dose level 1, once a day for up to 8 months

Trial Locations (9)

10029

Mount Sinai Hospital, New York

19141

Einstein Medical Center, Philadelphia

27157

Atrium Health Wake Forest Baptist, Winston-Salem

33136

University of Miami Miller School of Medicine, Miami

35233

University of Alabama Hospital, Birmingham

77550

University of Texas, Galveston

94117

University of California San Francisco, San Francisco

98109

Fred Hutchinson Cancer Center, Seattle

02114

Massachusetts General Hospital, Boston

Sponsors

Lead Sponsor

All Listed Sponsors
lead

Disc Medicine, Inc

INDUSTRY

NCT05308472 - Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Participants with EPP | Biotech Hunter | Biotech Hunter