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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT05883748
Registration number
NCT05883748
Ethics application status
Date submitted
22/05/2023
Date registered
1/06/2023
Date last updated
22/02/2024
Titles & IDs
Public title
Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP
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Scientific title
An Open-Label, Long-Term Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With Erythropoietic Protoporphyria (EPP)
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Secondary ID [1]
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DISC-1459-501
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Erythropoietic Protoporphyria
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Condition category
Condition code
Skin
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Other skin conditions
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Blood
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Other blood disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - DISC-1459
Treatment: Drugs - DISC-1459
Experimental: DISC-1459 Oral Dose Level 1 - Oral dose, once a day
Experimental: DISC-1459 Oral Dose Level 2 - Oral dose, once a day
Treatment: Drugs: DISC-1459
DISC-1459 dose level 1
Treatment: Drugs: DISC-1459
DISC-1459 dose level 2
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Incidence of treatment-emergent adverse events
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Assessment method [1]
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Timepoint [1]
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up to 5 Years
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Primary outcome [2]
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Incidence of clinically abnormal vital signs
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Assessment method [2]
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Timepoint [2]
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up to 5 Years
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Primary outcome [3]
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Incidence of clinically abnormal physical exam
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Assessment method [3]
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Timepoint [3]
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up to 5 Years
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Primary outcome [4]
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Incidence of abnormal laboratory test results
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Assessment method [4]
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Timepoint [4]
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up to 5 Years
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Primary outcome [5]
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Assessment of Patient Health Questionnaire (PHQ-8)
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Assessment method [5]
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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.
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Timepoint [5]
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up to 5 Years
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Primary outcome [6]
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Assessment of C-SSRS
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Assessment method [6]
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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.
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Timepoint [6]
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up to 5 Years
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Secondary outcome [1]
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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
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Assessment method [1]
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Timepoint [1]
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up to 5 Years
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Secondary outcome [2]
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Change from baseline in whole blood metal-free PPIX levels
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Assessment method [2]
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Timepoint [2]
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up to 5 Years
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Secondary outcome [3]
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Plasma Bitopertin Concentrations
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Assessment method [3]
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Timepoint [3]
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up to 5 Years
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Eligibility
Key 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
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Minimum age
12
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2/Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
31/08/2023
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/06/2028
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Actual
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Sample size
Target
200
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
NSW,VIC
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Recruitment hospital [1]
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Royal Prince Alfred Hospital - Camperdown
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Recruitment hospital [2]
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The Royal Melbourne Hospital - Parkville
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Recruitment postcode(s) [1]
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2050 - Camperdown
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Recruitment postcode(s) [2]
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3050 - Parkville
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Alabama
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Country [2]
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United States of America
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State/province [2]
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California
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Country [3]
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United States of America
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State/province [3]
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Florida
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Country [4]
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United States of America
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State/province [4]
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Massachusetts
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Country [5]
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United States of America
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State/province [5]
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New York
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Country [6]
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United States of America
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State/province [6]
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North Carolina
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Country [7]
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United States of America
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State/province [7]
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Pennsylvania
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Country [8]
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United States of America
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State/province [8]
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Texas
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Country [9]
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United States of America
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State/province [9]
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Washington
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Disc Medicine, Inc
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Address
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Country
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT05883748
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Will Savage, MD PhD
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Address
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Disc Medicine
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05883748
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