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HELIOS: Long-term study of DISC-1459 (or Bitopertin) in patients with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP).
EU CTIS ID: 2025-523275-27-00
What this study is testing
To assess the long-term safety and tolerability of bitopertin in participants with EPP (any FECH and ALAS2 genotypes) or X-linked protoporphyria (XLP).
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. Participants with diagnosis of EPP or XLP who are participating (or who have participated) in a prior Disc bitopertin study and who have completed the randomized treatment phase and End-of- Study visit.
- 2. Aged ≥12 years upon study consent.
- 3. Body weight ≥32 kg for participants <18 years of age and BMI ≥18.5 kg/m2 for adult participants.
- 4. 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.
- 5. Negative urine or serum pregnancy test (females of childbearing potential).
- 6. Able to understand the study aims, procedures, and requirements, and provide written informed consent (and assent if necessary).
You likely can't join if
- 1. Participants who have an ongoing serious adverse event (SAE) from a clinical study that is assessed by the investigator as related to bitopertin.
- 10. Consumption of grapefruit/Seville orange and products containing these for 14 days prior to first dose of study drug and throughout the study.
- 2. 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.
- 3. 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.
- 4. Planned treatment with afamelanotide or dersimelagon during the study.
- 5. Planned use of any drugs or herbal remedies known to be strong or moderate inhibitors or inducers of cytochrome P450 (CYP)3A4 enzymes throughout the study.
See the full eligibility criteria
- 1. Participants with diagnosis of EPP or XLP who are participating (or who have participated) in a prior Disc bitopertin study and who have completed the randomized treatment phase and End-of- Study visit.
- 2. Aged ≥12 years upon study consent.
- 3. Body weight ≥32 kg for participants <18 years of age and BMI ≥18.5 kg/m2 for adult participants.
- 4. 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.
- 5. Negative urine or serum pregnancy test (females of childbearing potential).
- 6. Able to understand the study aims, procedures, and requirements, and provide written informed consent (and assent if necessary).
- 7. Able to comply with all study procedures.
- 1. Participants who have an ongoing serious adverse event (SAE) from a clinical study that is assessed by the investigator as related to bitopertin.
- 10. Consumption of grapefruit/Seville orange and products containing these for 14 days prior to first dose of study drug and throughout the study.
- 2. 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.
- 3. 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.
- 4. Planned treatment with afamelanotide or dersimelagon during the study.
- 5. Planned use of any drugs or herbal remedies known to be strong or moderate inhibitors or inducers of cytochrome P450 (CYP)3A4 enzymes throughout the study.
- 6. Current or planned treatment with antipsychotic medication.
- 7. If female, pregnant, planning to become pregnant, or breastfeeding.
- 8. Participation in any other clinical protocol or investigational study, other than Disc bitopertin studies, that involves administration of experimental therapy and/or therapeutic devices within 30 days of Day 1.
- 9. 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.
The study team makes the final eligibility decision.
Where it's taking place
- Canada
- Australia
- United Kingdom
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Canada; Australia; United Kingdom; United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.