A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
This study is a long-term check-up for people who have received a new treatment called EDIT-301. This treatment was given to individuals with severe sickle cell disease or transfusion-dependent beta-thalassaemia, which are conditions affecting red blood cells. The main goal is to understand how safe and well this EDIT-301 treatment continues to work over many years. Researchers will gather information from participants who were part of earlier studies involving EDIT-301. It's important to note that no new medicine will be given in this particular follow-up study. Instead, it's about carefully observing and recording how participants are doing after their initial treatment to learn more about its lasting effects.
At a glance
What is this study about?
This study is carefully looking at the long-term effects of a treatment called EDIT-301. This treatment was given in previous studies to people living with either severe sickle cell disease or transfusion-dependent beta-thalassaemia. Both of these are blood conditions where the body doesn't make healthy red blood cells properly. Sickle cell disease causes red blood cells to become stiff and C-shaped, which can block blood flow and cause pain and organ damage. Transfusion-dependent beta-thalassaemia means someone needs regular blood transfusions to stay healthy.
The main aim of this follow-up study is to see how safe and effective the EDIT-301 treatment has been for these individuals over many years. This helps doctors and scientists understand the lasting benefits and any potential long-term concerns of the treatment. By gathering this information, they can learn more about how EDIT-301 helps people manage their conditions and improve their quality of life.
It’s important to know that this study is purely for observation. No new medicine will be given. Instead, researchers will collect information from people who have already had the EDIT-301 treatment in earlier studies. This ongoing observation is crucial for building a complete picture of the treatment's success and safety over time, helping to make future treatments even better for others with similar conditions.
Key takeaways
- It's about long-term check-ups for an existing treatment (EDIT-301).
- No new medicine or treatment is given in this study.
- Aims to understand how safe and effective EDIT-301 is over many years.
- For people who previously received EDIT-301 for sickle cell disease or beta-thalassaemia.
- Participation involves regular health checks and sharing health information.
- Your involvement helps improve future treatments for others.
Who may be eligible?
To be able to take part in this study, you must have already received an infusion of the EDIT-301 treatment as part of a previous research study. This means you were a participant in an earlier clinical trial testing EDIT-301 for sickle cell disease or transfusion-dependent beta-thalassaemia.
You also need to agree to join this specific long-term follow-up study. If you are under 16, a parent or legal guardian will need to give their permission for you to take part. This ensures everyone understands what the study involves and agrees to participate.
If you are still actively receiving treatment in one of the initial EDIT-301 studies and haven't yet reached the point where you would normally join a long-term follow-up study, you wouldn't be able to join this one yet. This study is specifically for those who have completed their initial treatment phase.
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.
- Have I already received the EDIT-301 treatment in a past study?
- Am I willing to have regular health check-ups and share my health information?
- Am I or my guardian willing to sign a consent form for this follow-up study?
- Am I aged between 12 and 50 years old?
- Am I not currently active in an earlier EDIT-301 treatment study?
What does participation involve?
If you decide to take part, you won't be given any new medicine. This study is all about checking up on you over time. You will have regular visits to a clinic where the study team will carry out health checks, which might include blood tests, physical examinations, and asking you about your general health and how you're feeling. These assessments are to track your progress and see how the EDIT-301 treatment is working in the long run.
The study will follow you for a long period, typically several years, to understand the lasting effects of the treatment. The exact number of visits and tests will be explained to you by the study team. You always have the right to stop participating in the study at any time, for any reason, without it affecting your medical care.
Potential risks and benefits
Locations (18)
- UCSF Benioff Children's HospitalVerified postcodeOakland, United States
- Children's Hospital ColoradoVerified postcodeAurora, United States
- Smilow Cancer HospitalVerified postcodeNew Haven, United States
- Johns Hopkins All Children's HospitalVerified postcodeSt. Petersburg, United States
- Children's Healthcare of AtlantaVerified postcodeAtlanta, United States
- University of MinnesotaVerified postcodeMinneapolis, United States
- Columbia University Medical Center - Department of PediatricsVerified postcodeNew York, United States
- Columbia University Medical CenterVerified postcodeNew York, United States
- Atrium HealthVerified postcodeCharlotte, United States
- Cleveland ClinicVerified postcodeCleveland, United States
- Nationwide Children's HospitalVerified postcodeColumbus, United States
- Children's Hospital of PhiladelphiaVerified postcodePhiladelphia, United States
Common questions
What is the main goal of this study?
The main goal is to check how safe and well the EDIT-301 treatment has worked over a long time for people who received it previously.
Will I be given any new medicine in this study?
No, you will not receive any new medicine. This study is focused on monitoring those who have already had the EDIT-301 treatment.
What medical conditions are being looked at?
The study is for people who have severe sickle cell disease or a type of thalassaemia called transfusion-dependent beta-thalassaemia.
How long will I need to be in the study?
The study will follow participants for a long time, typically several years, to understand the lasting effects of the treatment.
Can anyone join this study?
To join, you must have already received the EDIT-301 treatment in a previous study and agree to participate in this long-term follow-up.
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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