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

Erythropoietic Protoporphyria Clinical Trial

Official title:

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT05883748
• Other study ID #: DISC-1459-501
• Status: Enrolling by invitation
• Phase: Phase 2/Phase 3
• Start date: August 31, 2023
• Est. completion date: June 2028

Study information

• Verified date: February 2024
• Source: Disc Medicine, Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 200
• Est. completion date: June 2028
• Est. primary completion date: June 2028
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

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

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 any response of "yes" on the C-SSRS

• Grapefruit/Seville orange and products containing these for 14 days prior to first dose of study drug and throughout the stud

Related Conditions & MeSH terms

• Erythropoietic Protoporphyria

Intervention

Drug:
• DISC-1459
DISC-1459 dose level 1
• DISC-1459
DISC-1459 dose level 2

Locations

Country	Name	City	State
Australia	Royal Prince Alfred Hospital	Camperdown	New South Wales
Australia	The Royal Melbourne Hospital	Parkville	Victoria
United States	University of Alabama Hospital	Birmingham	Alabama
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	University of Texas	Galveston	Texas
United States	University of Miami Miller School of Medicine	Miami	Florida
United States	Mount Sinai Hospital	New York	New York
United States	Einstein Medical Center	Philadelphia	Pennsylvania
United States	University of California San Francisco	San Francisco	California
United States	Fred Hutchinson Cancer Center	Seattle	Washington
United States	Atrium Health Wake Forest Baptist	Winston-Salem	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Disc Medicine, Inc

Countries where clinical trial is conducted

United States,  Australia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of treatment-emergent adverse events		up to 5 Years
Primary	Incidence of clinically abnormal vital signs		up to 5 Years
Primary	Incidence of clinically abnormal physical exam		up to 5 Years
Primary	Incidence of abnormal laboratory test results		up to 5 Years
Primary	Assessment of Patient Health Questionnaire (PHQ-8)	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.	up to 5 Years
Primary	Assessment of C-SSRS	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.	up to 5 Years
Secondary	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
Secondary	Change from baseline in whole blood metal-free PPIX levels		up to 5 Years
Secondary	Plasma Bitopertin Concentrations		up to 5 Years