A Long-term Follow-up Study in Participants Who Received CTX001
This study is called 'A Long-term Follow-up Study in Participants Who Received CTX001'. It's a new follow-up study for people who have already had a special treatment called CTX001. This treatment is for serious blood conditions like beta-thalassemia and sickle cell disease. It includes both children aged two and older, and adults. The main goal of this study is to see how safe CTX001 is over a long time and how well it continues to work. It's an 'open-label' study, which means everyone involved knows they are receiving the treatment. If you've previously received CTX001 in one of the earlier studies (like 111, 141, 161 for beta-thalassemia or 121, 151, 161 for sickle cell disease), this study is for you.
At a glance
What is this study about?
This study is a follow-up for people who have already received a special treatment called CTX001. This treatment is designed for serious inherited blood conditions such as beta-thalassemia and sickle cell disease. Both conditions affect the red blood cells, which are responsible for carrying oxygen around the body. Beta-thalassemia can cause severe anaemia and often needs regular blood transfusions. Sickle cell disease causes pain, infections, and other serious health problems.
Researchers want to understand how safe CTX001 is over many years and how well it continues to help people who have already received it. Because CTX001 is a newer type of treatment, it's important to keep a close eye on people who have had it to learn more about its long-term effects. This particular study is called an 'open-label rollover study', which simply means it's a long-term follow-up that extends from earlier studies, and everyone involved knows they are part of the treatment group.
By carefully watching people who have already been treated with CTX001, doctors can learn vital information. This information helps them understand the full benefits and any potential long-term side effects, ensuring the treatment is as safe and effective as possible for future patients. This study will gather important details about how people's health changes over time after receiving CTX001.
Key takeaways
- This is a long-term follow-up study for CTX001, a treatment for serious blood conditions.
- It's only for people who have already received CTX001 in a previous study.
- The study aims to check the safety and effectiveness of CTX001 over many years.
- Both children (age 2+) and adults can participate.
- Participation involves regular health check-ups and monitoring.
- You can withdraw from the study at any time.
Who may be eligible?
To be able to join this study, you must have already received the CTX001 treatment in one of the earlier studies. It doesn't matter if you got the treatment for beta-thalassemia or sickle cell disease in those previous studies.
Also, if you are able to make your own decisions, you will need to read and sign a consent form, which explains everything about the study. If you are a child or unable to give consent yourself, your parent, guardian, or legal representative would need to sign this form on your behalf. There might also be another form for children to sign, called an 'assent form', which shows they understand and agree to take part.
This study is for both children (from two years old) and adults of any age. There are no other reasons that would prevent you from taking part, as long as you meet the main requirement of having already received CTX001.
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 have already received CTX001 treatment in one of the earlier studies.
- I have a condition like beta-thalassemia or sickle cell disease.
- I am aged two years or older.
- I (or my legal guardian) am willing to sign consent forms.
What does participation involve?
Since this is a long-term follow-up study, you would mainly need to attend regular check-up appointments at your study clinic. During these visits, the study team will monitor your general health, conduct some routine physical exams, and may take blood samples to check how the CTX001 treatment is working and if there are any changes over time. You won't be receiving a new dose of CTX001 in this study; instead, the focus is on observing the effects of the treatment you've already had.
The specific schedule and number of visits will be explained by the study team. They will also tell you how long they expect you to be part of this follow-up study. You won't be given any new medication as part of this study, but your doctors will continue to look after your health needs related to your condition. The total duration of your involvement could be several years, as the aim is to gather long-term information.
Potential risks and benefits
Locations (20)
- Lucile Packard Children's HospitalVerified postcodePalo Alto, United States
- Ann & Robert H. Lurie Children's Hospital of Chicago - HematologyVerified postcodeChicago, United States
- Herbert Irving Pavilion - HematologyVerified postcodeNew York, United States
- New York Presbyterian Hospital - Morgan Stanley Children's HospitalVerified postcodeNew York, United States
- Levine Children's Hospital - HematologyVerified postcodeCharlotte, United States
- The Children's Hospital of Philadelphia - HematologyVerified postcodePhiladelphia, United States
- St. Jude Children's Research HospitalVerified postcodeMemphis, United States
- TriStar Medical Group Children's Specialists - Pediatric OncologyVerified postcodeNashville, United States
- Methodist Healthcare System of San Antonio, Methodist Hospital, Methodist Children's HospitalVerified postcodeSan Antonio, United States
- Hopital Universitaire des Enfants Reine Fabiola (HUDERF) - HematologyVerified postcodeBrussels, Belgium
- Hospital for Sick Children - HematologyVerified postcodeToronto, Canada
- Toronto General Hospital - HematologyVerified postcodeToronto, Canada
Common questions
What is CTX001?
CTX001 is a special treatment for serious inherited blood conditions like beta-thalassemia and sickle cell disease.
Who can join this study?
You can join this study if you have already received the CTX001 treatment in one of the earlier studies for beta-thalassemia or sickle cell disease.
How long will I be in the study?
This is a long-term study, so it will involve regular check-ups over several years to understand the lasting effects of the treatment you've already had.
Do I get more CTX001 treatment in this study?
No, this study is about following up on how the CTX001 treatment you've already received is working over time. You won't get a new dose.
Can children join this study?
Yes, children from the age of two who have already received CTX001 in a previous study can take part, with their parent's or guardian's permission.
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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