Clinical Trials in Italy / NCT05883748
Enrolling by invitation
Phase 2/3
HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP
NCT05883748 · tracked via the Priya Life Science Italy tracker
Condition(s) studied
Erythropoietic Protoporphyria
Investigational drug(s) / intervention(s)
DISC-1459: DISC-1459 dose level 1
DISC-1459: DISC-1459 dose level 2
Study 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
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
* Willing to practice highly effective methods of birth control (both males who have partners of childbearing potential and females of childbearing potential during the study, while taking study drug, and for at least 30 days after the last dose of study drug.
* Negative urine or serum pregnancy test (females of childbearing potential).
* Able to understand the study aims, procedures, and requirements, and provide written informed consent (and assent if necessary).
* Able to comply with all study procedures.
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 outcome measure(s)
- Incidence of treatment-emergent adverse events — up to 5 Years
- Incidence of clinically abnormal vital signs — up to 5 Years
- Incidence of clinically abnormal physical exam — up to 5 Years
- Incidence of abnormal laboratory test results — up to 5 Years
- Assessment of Patient Health Questionnaire (PHQ-8) — up to 5 Years
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. - Assessment of C-SSRS — up to 5 Years
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.
Trial sites (26)
| Facility | City | Region | Status |
|---|---|---|---|
| University of Alabama Hospital | Birmingham | Alabama | |
| University of California San Francisco | San Francisco | California | |
| University of Miami Miller School of Medicine | Miami | Florida | |
| Massachusetts General Hospital | Boston | Massachusetts | |
| MetroBoston Clinical Partners | Boston | Massachusetts | |
| Mount Sinai Hospital | New York | New York | |
| Atrium Health Wake Forest Baptist | Winston-Salem | North Carolina | |
| Remington-Davis Clinical Research | Columbus | Ohio | |
| Einstein Medical Center | Philadelphia | Pennsylvania | |
| University of Texas | Galveston | Texas | |
| Fred Hutchinson Cancer Center | Seattle | Washington | |
| Royal Prince Alfred Hospital | Camperdown | New South Wales | |
| The Royal Melbourne Hospital | Parkville | Victoria | |
| UZ Leuven | Leuven | Belgium | |
| CHU de Nantes - Hôtel Dieu, Service de dermatologie | Nantes | France | |
| Centre d'Investigation Clinique (CIC) Hôpital Bichat - Claude-Bernard | Paris | France | |
| Charité - Universitätsmedizin Berlin, Institute of Allergology | Berlin | Germany | |
| Klinikum Chemnitz gGmbH | Chemnitz | Saxony | |
| Children's Health Ireland (CHI) | Dublin | Ireland | |
| Instituto Dermatologico San Gallicano Istituti Fisioterapici Ospitalieri IRCCS | Roma | Italy | |
| Erasmus MC | Rotterdam | Netherlands | |
| Hospital Clinic de Barcelona | Barcelona | Spain | |
| Karolinska University Hospital | Stockholm | Sweden | |
| Guy's and St Thomas' NHS Foundation Trust | London | England | |
| Clinical Research Centre, Ninewells Hospital & Medical School , NHS Tayside | Dundee | Scotland | |
| Photobiology Unit, Salford Royal Hospital | Salford | United Kingdom |
More Disc Medicine, Inc trials in Italy
Study of Bitopertin in Participants With EPP or XLP (APOLLO)
Active, not recruiting
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT05883748 on ClinicalTrials.gov ↗ ← All trials in Italy