Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

HepaStem Long-Term Safety Registry – Registry for Patients who have been administered HepaStem

This study is gathering information on people who have previously received a treatment called HepaStem. HepaStem is a type of cell therapy that was given to patients in earlier clinical studies for conditions like urea cycle disorders, Crigler-Najjar syndrome, and certain inflammatory liver diseases. This particular study isn't giving new treatment; instead, it's a long-term follow-up registry. The main goal is to carefully record any important health events that happen after receiving HepaStem. This includes checking for serious health problems, the need for organ transplants, any new growths or cancers, infections, or any other health issues that might be linked to the HepaStem treatment. It's a way for doctors to keep track of patients' health over time and learn more about the treatment's long-term effects.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Cellaïon SA

Enrolment target

Start

03 Feb 2023

What is this study about?

Imagine you've been part of a study where you received a special cell treatment called HepaStem for a liver problem or a genetic condition like urea cycle disorders or Crigler-Najjar syndrome. Now that the main treatment study has finished, doctors want to make sure everyone who received HepaStem is doing well in the long term. This new study, called the HepaStem Long-Term Safety Registry, is a way to do just that.

It's not about giving you more HepaStem or trying a new treatment. Instead, it's like a long-term check-up where researchers will keep track of your health. They're especially interested in noticing any important changes or new health issues that might come up over time. This helps them understand the full picture of how HepaStem affects people's health in the years after treatment.

The main purpose is to learn about how safe HepaStem is over a longer period. By carefully recording any significant health events – good or bad – the researchers can gather important information. This information is crucial for understanding whether HepaStem has any long-term effects and helps doctors decide if it could be a helpful treatment for others in the future.

Key takeaways

This study is about long-term health monitoring, not new treatment.
It's only for adults who have already had HepaStem for liver or genetic conditions.
Researchers want to track any serious health changes over time.
Your regular doctor will help provide health updates.
Your participation helps doctors learn about HepaStem's long-term safety.

Who may be eligible?

This study is specifically for adults who have already received the HepaStem treatment in a previous study carried out by Promethera Biosciences or Promethera Therapeutics. You cannot join this study if you haven't had HepaStem before.

It includes people who received HepaStem for a variety of conditions, such as problems with how the body processes waste (urea cycle disorders), a rare liver condition affecting how the body handles a substance called bilirubin (Crigler-Najjar syndrome), and certain liver diseases that involve inflammation.

Essentially, if you're 18 or older and were one of the patients who bravely took part in an earlier HepaStem treatment study, then you'd be eligible for this long-term follow-up.

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 18 years old or older.
I have previously received HepaStem treatment.
My HepaStem treatment was part of a study by Promethera Biosciences or Promethera Therapeutics.
I received HepaStem for a condition like urea cycle disorder, Crigler-Najjar, or an inflammatory liver disease.

Answer every question to see your result.

What does participation involve?

If you join this study, you won't be given any new medication or treatment. Your participation will mainly involve sharing information about your health over time. This might include attending regular check-ups with your doctor, who will then share updates with the study team. You might also be asked about your overall well-being and any health changes you've experienced. The study will track important health events, such as hospital stays, new diagnoses, or any other serious health issues. The total duration of your participation will depend on how long the study continues to monitor patients who received HepaStem.

Potential risks and benefits

The main benefit of taking part is contributing to a better understanding of HepaStem's long-term safety, which can help future patients. As this is an observational study and no new treatment is given, the direct medical risks are very low. However, sharing your medical information, even if anonymised, carries a small risk to privacy. You are free to withdraw from the study at any time without giving a reason, and this will not affect your usual medical care.

Locations (5)

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

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Unverified
Spain
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Unverified
Belgium
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Unverified
France
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Unverified
Bulgaria
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Unverified
Poland

Common questions

What is HepaStem?

HepaStem is an experimental cell therapy that was previously given to patients in earlier studies for various liver-related health problems.

Will I receive more HepaStem treatment in this study?

No, this study is only for people who have already received HepaStem in a previous clinical trial. You will not get any new treatment.

Why is this study happening?

Doctors want to carefully track the long-term health of people who received HepaStem to learn more about its effects over time, especially any important health changes.

What health information will the study collect?

The study will record information about any serious health events, such as needing an organ transplant, new growths, infections, or other health problems that might be linked to HepaStem.

Does this study involve many doctor's visits?

Your involvement typically won't mean extra doctor's visits beyond your usual care. Your regular doctor will help share your health updates with the study team.