Active not recruitingPHASE3INTERVENTIONAL

Study of Bitopertin in Participants With EPP or XLP (APOLLO)

The APOLLO study is testing a medication called bitopertin for people aged 12 and over who have Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP). These conditions make skin very sensitive to sunlight. The main aim is to find out if bitopertin can increase the amount of time people with EPP or XLP can spend in the sun without pain after six months of treatment. Researchers will also look at how levels of a chemical called PPIX in the blood change. Some participants will receive bitopertin, while others will get a 'dummy' pill called a placebo to compare its effects. Participants will complete daily questionnaires and attend regular clinic visits.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Disc Medicine, Inc

Enrolment target

183

Start

04 Apr 2025

Estimated completion

01 Oct 2026

Erythropoietic Protoporphyria (EPP)X-Linked Protoporphyria (XLP)

What is this study about?

The APOLLO study is a clinical trial looking into a new medication called bitopertin. It’s for people who have conditions called Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP). These conditions are rare and make a person's skin very sensitive to sunlight, causing pain, burning, and itching, sometimes very quickly after exposure. This study wants to find out if bitopertin is safe to use and if it can help people with EPP or XLP live more comfortably, particularly when they are exposed to the sun.

The main things the researchers want to learn are: can bitopertin help people spend more time in the sun without feeling pain? They will measure this after six months of taking the medication. They also want to see if bitopertin changes the levels of a substance in the blood called PPIX, which is often high in people with EPP and XLP. By understanding these effects, the study hopes to see if bitopertin could be a helpful new treatment option for people with these challenging conditions.

To see if bitopertin really works, some people in the study will take the actual medication, while others will take a 'placebo'. A placebo looks exactly like the study medication but contains no active drug. This comparison helps researchers be sure that any changes noticed are due to the bitopertin and not just other factors. This is a common and important part of how new medications are tested to make sure results are reliable and accurate.

Key takeaways

This study evaluates a new drug, bitopertin, for EPP and XLP.
It aims to see if bitopertin increases pain-free sun exposure.
Participants will take either bitopertin or a placebo.
Daily questionnaires and clinic visits are required fieldwork.
The study involves people aged 12 and older with EPP or XLP.
You can withdraw from the study at any time.

Who may be eligible?

To join this study, you must be at least 12 years old and have a confirmed diagnosis of EPP or XLP. You'll also need to have kept a diary of your sun exposure in the days just before starting the study, showing you’re good at tracking details. There are also some general health checks, like having a certain body weight if you're under 18, and a minimum body mass index if you're 18 or older.

There are also some reasons why you might not be able to join. For example, if you've had major surgery recently or haven't fully recovered from a previous surgery. You also can't have certain other health problems like a history of liver transplant, alcohol dependence, or specific serious infections like HIV or hepatitis. If you're pregnant, planning to become pregnant, or breastfeeding, you also won't be able to take part.

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.

Are you 12 years old or older?
Have you been diagnosed with EPP or XLP?
Can you easily track details like daily sun exposure?
Do you have good general health, without severe liver issues, HIV, or hepatitis?
Are you able to use effective birth control if applicable?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will either receive the study medication (bitopertin) or a placebo (a dummy pill with no drug). You will need to take this medication regularly. Throughout the study, you'll be asked to fill out daily questionnaires, for example, about your sun exposure and any pain you experience. You will also need to attend regular visits to the clinic. During these visits, the study team will carry out health checks, take blood samples, and ask you about your symptoms and how you're feeling. They will monitor your health closely to make sure the treatment is safe and to see how well it's working. The exact length of your participation would be explained by the study team.

Potential risks and benefits

Taking part in a clinical trial has potential benefits and risks. You might benefit from the new medication if it proves to be effective for your condition, or you could contribute important information that helps others in the future. However, there's also the chance you might receive the placebo and not the active drug. As with any medication, there could be side effects, some of which might not be known yet. The study team will explain all known risks and monitor you closely. Remember, you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (27)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Marvel Clinical Research
Verified postcode
Huntington Beach, United States
University of California San Francisco
Verified postcode
San Francisco, United States
University of Miami Miller School of Medicine
Verified postcode
Miami, United States
Massachusetts General Hospital
Verified postcode
Boston, United States
MetroBoston Clinical Partners
Verified postcode
Boston, United States
Henry Ford Health System
Verified postcode
Detroit, United States
Mount Sinai Hospital
Verified postcode
New York, United States
Wake Forest University
Verified postcode
Winston-Salem, United States
Remington-Davis Clinical Research
Verified postcode
Columbus, United States
University of Texas Medical Branch
Verified postcode
Galveston, United States
University of Washington
Verified postcode
Seattle, United States
Royal Prince Alfred Hospital
Verified postcode
Camperdown, Australia

Common questions

What is EPP or XLP?

They are rare conditions that make your skin extremely sensitive to sunlight, causing pain and burning after even short exposure.

What is a 'placebo'?

A placebo is a pill that looks exactly like the study medication but does not contain any active drug. It helps researchers compare if the real medication is effective.

How long will I be in the study?

The study aims to see changes after 6 months of treatment, but the full duration of your participation, including follow-up, will be explained by the study team.

Will I know if I'm getting the real medication or the placebo?

To ensure fair results, neither you nor the study team will know until after the study is over. This is called 'blinding'.

What happens if I want to stop participating?

You are free to leave the study at any time, for any reason, and it will not affect your ongoing medical care.