A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study
This study is designed for people who have been taking a medication called mitapivat as part of a previous clinical trial sponsored by a company called Agios. If your doctor believes that mitapivat is helping you with your condition – such as Pyruvate Kinase Deficiency, certain types of anaemia like Hemolytic Anaemia, Sickle Cell Disease, or Thalassemia – this study allows you to keep receiving the treatment. It's important because it ensures that patients who are already getting good results from the medication don't have to stop taking it once their original study finishes. You would continue on the same dose you were taking before. The main goal is to provide ongoing access to this treatment for patients who are clearly benefiting.
At a glance
What is this study about?
This study is set up for people who have already been taking a specific medication called mitapivat through an earlier research study. Sometimes, when a research study finishes, patients might have to stop taking a drug even if it's helping them. This 'access study' is designed to prevent that, offering a way for you to continue treatment.
The main purpose is to make sure that if you, and your doctor, both feel that mitapivat is really making a difference to your health, you can carry on receiving it. This is especially important for conditions like Pyruvate Kinase Deficiency, certain types of anaemia (where your body doesn't have enough healthy red blood cells), Sickle Cell Disease, or Thalassemia. The study wants to make sure people benefiting from the drug can keep taking it safely.
Because this is an extension study, you wouldn't be starting a new treatment or a new dose. You would simply carry on with the same amount of mitapivat you were taking at the end of your previous study. This ongoing access is carefully managed by your doctor to ensure it continues to be the right choice for you.
Key takeaways
- This study is for people already taking mitapivat in a previous trial.
- It allows you to continue treatment if your doctor thinks it's helping.
- You will stay on the same dose of mitapivat you were taking before.
- It aims to give ongoing access to a helpful medication.
- You must be 18 or older to participate.
- You can withdraw from the study at any time.
Who may be eligible?
To be able to join this study, you must already be taking the drug mitapivat as part of a previous study sponsored by Agios, and your doctor must believe it's helping you with your condition. You need to be 18 years old or older, and it's open to both men and women.
If you are a woman who could become pregnant, you'll need to either agree not to have sex that could lead to pregnancy, or use two effective forms of contraception (birth control) throughout the study and for 28 days afterwards. One of these must be highly effective, like a contraceptive pill, implant, or IUD, and the second can be a barrier method like a condom.
There are also reasons why you might not be able to join. For example, if you are allergic to mitapivat or any of its ingredients. Also, if you have any other serious health conditions – including mental health issues – or are receiving other treatments that your doctor feels could make the study unsafe for you, or make it hard to understand the results, then you wouldn't be able to take part. People who are in prison or under court order in an institution are also not eligible.
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 currently taking mitapivat as part of an Agios-sponsored study?
- Does your doctor believe mitapivat is currently helping your condition?
- Are you 18 years old or older?
- Are you able to use effective birth control if you are a woman who could become pregnant?
- Do you have any known allergies to mitapivat or its ingredients?
- Are you willing to avoid joining other clinical studies while in this one?
What does participation involve?
If you decide to take part, you'll continue to take the same dose of mitapivat that you were receiving at the very end of your previous study. This isn't a study where you'd be trying a new dose or a different treatment.
Your participation will involve regular visits to your clinic or hospital for check-ups. During these visits, your medical team will monitor your health, ask about any side effects, and make sure the medication is still helping you. They might also do blood tests or other assessments to keep track of your condition.
The total length of time you'll be in the study will depend on how long your doctor believes you are benefiting from the treatment and if the medication becomes available more widely. Throughout this time, you must agree not to join any other clinical studies, especially those testing other drugs, to ensure your health can be properly monitored.
Potential risks and benefits
Locations (23)
- Hôpital ErasmeVerified postcodeAnderlecht, Belgium
- Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS)Verified postcodePorto Alegre, Brazil
- Hospital de Clínicas da UnicampVerified postcodeCampinas, Brazil
- HEMORIO Instituto Nacional de HematologiaVerified postcodeRio de Janeiro, Brazil
- Praxis Pesquisa MedicaVerified postcodeSanto André, Brazil
- GSH Banco de Sangue de São PauloVerified postcodeSão Paulo, Brazil
- Hospital das Clínicas da Faculdade de Medicina da Universidad de São PauloVerified postcodeSão Paulo, Brazil
- Hospital Das Clínicas da Faculdade de Medicina de Ribeirão Preto - USPVerified postcodeSão Paulo, Brazil
- McMaster Centre for Transfusion Medicine McMaster UniversityVerified postcodeHamilton, Canada
- University Health NetworkVerified postcodeToronto, Canada
- Hopitaux de La TimoneVerified postcodeMarseille, France
- CHU de Bordeaux - Hôpital PellegrinVerified postcodeBordeau, France
Common questions
What is mitapivat?
Mitapivat is a medication being studied for conditions like Pyruvate Kinase Deficiency, Sickle Cell Disease, and Thalassemia, which affect red blood cells.
Why is this study happening?
This study allows patients who are already benefiting from mitapivat in a previous trial to keep receiving the medication, ensuring continued access to treatment.
Will I get a different dose of the medication?
No, you will continue on the exact same dose of mitapivat that you were taking at the end of your previous study.
How long will I be in this study?
The length of your participation will depend on how long your doctor believes you are benefiting from the treatment and when the drug might become more widely available.
Can I stop taking part at any time?
Yes, you have the right to leave the study at any point, for any reason, and this will not affect your future 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?
Discussion
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