Clinical trial NCT05883748: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants with EPP
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)
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE2
PHASE3
Conditions: Erythropoietic Protoporphyria
Keywords: EPP
DISC-1459
porphyria
RO4917838
Lead sponsor: Disc Medicine, Inc
Sponsor class: INDUSTRY
First posted: 2023-06-01
Enrollment: 200
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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


Eligibility criteria: 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 imminent suicidal risk identified by the C-SSRS as defined as suicidal ideation with intent (Grade 4 or 5) within the last year or any suicidal behavior within the last 5 years.
* Consumption of grapefruit/Seville orange and products containing these for 14 days prior to first dose of study drug and throughout the study

Primary outcomes:
measure: Incidence of treatment-emergent adverse events
timeFrame: up to 5 Years
measure: Incidence of clinically abnormal vital signs
timeFrame: up to 5 Years
measure: Incidence of clinically abnormal physical exam
timeFrame: up to 5 Years
measure: Incidence of abnormal laboratory test results
timeFrame: up to 5 Years
measure: Assessment of Patient Health Questionnaire (PHQ-8)
timeFrame: up to 5 Years
measure: Assessment of C-SSRS
timeFrame: up to 5 Years
Secondary outcomes:
measure: 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
timeFrame: up to 5 Years
measure: Change from baseline in whole blood metal-free PPIX levels
timeFrame: up to 5 Years
measure: Plasma Bitopertin Concentrations
timeFrame: up to 5 Years
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama Hospital
state: Alabama
city: San Francisco
country: United States
facility: University of California San Francisco
state: California
city: Miami
country: United States
facility: University of Miami Miller School of Medicine
state: Florida
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: New York
country: United States
facility: Mount Sinai Hospital
state: New York
city: Winston-Salem
country: United States
facility: Atrium Health Wake Forest Baptist
state: North Carolina
city: Philadelphia
country: United States
facility: Einstein Medical Center
state: Pennsylvania
city: Galveston
country: United States
facility: University of Texas
state: Texas
city: Seattle
country: United States
facility: Fred Hutchinson Cancer Center
state: Washington
city: Camperdown
country: Australia
facility: Royal Prince Alfred Hospital
state: New South Wales
city: Parkville
country: Australia
facility: The Royal Melbourne Hospital
state: Victoria
Study officials:
affiliation: Disc Medicine
name: Will Savage, MD PhD
role: STUDY_DIRECTOR
References: