APOLLO: A Randomized, Double-Blind, Placebo-Controlled Study of Bitopertin to Evaluate the Efficacy, Safety, and Tolerability in Participants with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)
Erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) are rare genetic conditions that make skin painfully sensitive to sunlight. Even short exposure can cause intense burning, stinging, and swelling due to a chemical build-up in the blood called protoporphyrin. The APOLLO study is testing an investigational medicine called bitopertin. Researchers hope it will lower these chemical levels and help people spend more time outside without discomfort. Participants will take either bitopertin or an inactive dummy pill (placebo) once a day for six months. Neither the participants nor the study team will know who receives which pill. During the trial, doctors will track how much pain-free time participants spend in daylight between 10:00 and 18:00, monitor their blood levels, and check for side effects. The study is open to adults aged 18 and older who have been diagnosed with EPP or XLP.
At a glance
What is this study about?
Living with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP) often means planning your life around avoiding the sun. In both conditions, the body produces too much of a natural compound called protoporphyrin. When sunlight hits the skin, it reacts with this compound, causing severe and long-lasting burning pain. Currently, treatment options to prevent this pain are very limited.
The APOLLO trial is testing a new medicine called bitopertin. Bitopertin works by slowing down the production of protoporphyrin in the body. If the level of this compound drops, sunlight should cause less damage and pain. This is a Phase 3 clinical trial, which means bitopertin has already shown promise in earlier research and is now being tested in a larger group of people to confirm how well it works and how safe it is.
To ensure fair results, the trial is 'double-blind' and 'placebo-controlled'. Half of the participants will receive bitopertin tablets, while the other half will receive a matching placebo tablet with no active medicine. A computer decides who gets what at random, and neither you nor your study doctor will know which one you are taking. Over a six-month period, doctors will compare the two groups to see if bitopertin genuinely gives people more pain-free hours in the sun.
Key takeaways
- The APOLLO trial tests bitopertin, a potential new treatment for adults with EPP or XLP.
- The main aim is to increase the amount of time people can spend in sunlight without severe pain.
- Participants take a daily tablet for six months, receiving either bitopertin or a dummy pill (placebo).
- Taking part involves regular clinic visits, blood tests, and keeping a daily diary of sun exposure.
- Participation is completely voluntary and you can stop at any time.
Who may be eligible?
This trial is open to adults aged 18 and over who have a confirmed medical diagnosis of either EPP or XLP. Both men and women are welcome to apply.
To take part, you must be able to attend scheduled visits at a study clinic, have regular blood tests, and complete a daily diary recording the time you spend outdoors and any symptoms you feel.
Certain people may not be able to join. For example, you may not be eligible if you have certain other medical conditions, take specific medicines that could interfere with the study, or are pregnant or breastfeeding. The study team will carry out health checks and review your full medical history before you join.
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 am aged 18 or older.
- I have been diagnosed with EPP or XLP.
- I experience painful skin reactions when exposed to daylight.
- I can take a daily tablet and attend regular clinic visits for six months.
- I am able to keep a daily log of my time in the sun and any symptoms.
What does participation involve?
Taking part in the APOLLO trial lasts for about six months (24 weeks). You will be asked to take your study tablets once every day. Throughout the study, you will need to visit the hospital clinic regularly for health check-ups. At these visits, doctors will check your general wellbeing, ask about any side effects, and take blood samples to measure your protoporphyrin levels. You will also use an electronic diary to track how much time you spend in the daylight between 10:00 and 18:00, and note down any early signs of skin reactions, such as stinging, itching, or burning.
Potential risks and benefits
Locations (9)
- —UnverifiedGermany
- —UnverifiedIreland
- —UnverifiedSpain
- —UnverifiedFrance
- —UnverifiedNorway
- —UnverifiedNetherlands
- —UnverifiedSweden
- —UnverifiedBelgium
- —UnverifiedItaly
Common questions
What is a placebo?
A placebo is a dummy tablet that looks identical to the real medicine but contains no active ingredient. It helps doctors see whether improvements are genuinely caused by bitopertin.
Can I choose to get bitopertin instead of the placebo?
No. A computer randomly decides which treatment you receive to make sure the study results are completely unbiased.
Will taking part cost me anything?
No. The study tablets and clinic visits are provided free of charge, and you may be reimbursed for reasonable travel costs to and from the hospital.
Do I have to intentionally go out into the bright sun?
No. You should not deliberately burn yourself. The study measures your normal, day-to-day sun exposure and asks you to record when you first start feeling symptoms.
What happens if I change my mind after starting?
You are free to leave the study at any point. Your decision will not change how your regular doctors treat you or your condition.
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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