HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP
The HELIOS study is a long-term research project focusing on a medicine called DISC-1459, also known as Bitopertin. It's designed for people who have a rare blood condition called Erythropoietic Protoporphyria, or EPP. This condition makes people very sensitive to sunlight. The main goal of this study is to keep a close eye on the medicine's safety, how well people can tolerate it, and how effective it is over a longer period. Participants in this study would have already taken part in previous Bitopertin studies. By continuing to follow these patients, researchers hope to gather more important information to help understand if this medicine could be a good long-term treatment option for EPP.
At a glance
What is this study about?
The HELIOS study is an important next step for people living with Erythropoietic Protoporphyria (EPP). EPP is a condition that makes your skin extremely sensitive to light, often causing painful blisters and burns even after short sun exposure. This study is looking at a new medicine called DISC-1459, also known as Bitopertin, which is hoped to help manage this condition.
This study is for people who have already been involved in earlier studies looking at Bitopertin. It's called an "open-label, long-term extension study." "Open-label" means that both you and your study doctor will know if you are receiving the study medicine. "Long-term extension" means that it's designed to continue checking on people over a longer period, building on what we've already learned. The main goal is to understand how safe the medicine is when taken for a long time, how well people tolerate it (meaning, if it causes side effects), and if it continues to be helpful in treating EPP.
By continuing to gather information from people who have already started treatment, researchers can get a much clearer picture of the medicine's overall effects. This long-term data is crucial for understanding if Bitopertin could become a widely available treatment in the future to help people with EPP live more comfortably and safely.
Key takeaways
- The HELIOS study is a long-term follow-up for people with EPP who have already been in a Bitopertin trial.
- It aims to check the safety, how well the medicine is tolerated, and its effectiveness over an extended period.
- Participants will receive the study medicine, DISC-1459 (Bitopertin), and have regular health checks.
- It's open to people aged 12 and older who meet specific health and prior study requirements.
- This research helps gather crucial information for potentially making this medicine available in the future.
Who may be eligible?
To join the HELIOS study, you must have EPP and have already taken part in a previous study of DISC-1459 (Bitopertin). You need to be at least 12 years old. If you're under 18, you should weigh at least 32 kg, and if you're an adult, your Body Mass Index (BMI) should be 18.5 or higher.
It's important that you are able to understand what the study involves and are willing to follow all the study instructions. For both men and women who could have children, it's necessary to use effective birth control during the study and for a period afterwards. Women who could have children will also need a negative pregnancy test before starting.
There are some reasons why you might not be able to join. For example, if you've had serious side effects from Bitopertin in a previous study that your doctor thinks are still affecting you. Also, if you have other health issues or are taking certain medications that might interfere with the study's results or put you at risk, you might not be eligible. This includes certain mental health conditions, planned treatments with specific other medications for EPP, or if you are pregnant or breastfeeding.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- I have Erythropoietic Protoporphyria (EPP).
- I have already taken part in a previous Bitopertin study.
- I am at least 12 years old.
- I can understand and follow study instructions.
- I am willing to use effective birth control if applicable.
What does participation involve?
If you decide to take part in the HELIOS study, it means you'll continue receiving the study medicine, DISC-1459 (Bitopertin), as instructed by the study team. You will have regular visits to the clinic so doctors can monitor your health, check for any side effects, and see how well the medicine is working for your EPP. These visits will likely involve checking your vital signs, taking blood and urine samples, and answering questions about your health and how you're feeling.
You'll be asked to keep a record of certain information between visits, like a diary of your symptoms or sun exposure. The study will likely last for a significant period, as it's designed for long-term follow-up, but the exact duration will be explained by the study team. All scheduled appointments, tests, and medicines related to the study will be provided to you.
Potential risks and benefits
Locations (19)
- University of Alabama HospitalVerified postcodeBirmingham, United States
- University of California San FranciscoVerified postcodeSan Francisco, United States
- University of Miami Miller School of MedicineVerified postcodeMiami, United States
- Massachusetts General HospitalVerified postcodeBoston, United States
- MetroBoston Clinical PartnersVerified postcodeBoston, United States
- Mount Sinai HospitalVerified postcodeNew York, United States
- Atrium Health Wake Forest BaptistVerified postcodeWinston-Salem, United States
- Remington-Davis Clinical ResearchVerified postcodeColumbus, United States
- Einstein Medical CenterVerified postcodePhiladelphia, United States
- University of TexasVerified postcodeGalveston, United States
- Fred Hutchinson Cancer CenterVerified postcodeSeattle, United States
- Royal Prince Alfred HospitalVerified postcodeCamperdown, Australia
Common questions
What is EPP?
EPP, or Erythropoietic Protoporphyria, is a rare inherited condition that makes your skin highly sensitive to light, leading to pain, redness, and swelling, often within minutes of sun exposure.
What is DISC-1459 (Bitopertin)?
DISC-1459 (Bitopertin) is an experimental medicine being tested to see if it can help people with EPP. It's not yet approved for general use.
Will I get a placebo in this study?
No, this is an 'open-label' study, which means everyone will receive the study medicine, DISC-1459. There is no placebo group.
How long will I be in the study?
This is a long-term study, so your participation could last for an extended period. The exact length will be discussed with you by the study team.
Can I leave the study if I want to?
Yes, you can choose to leave the study at any time, for any reason, without it affecting your regular medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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