Clinical trials · Philadelphia, PA
Beta-Thalassemia clinical trials in Philadelphia
We’re tracking 7 recruiting beta-thalassemia studies with a site in or near Philadelphia — including Phase 1/Phase 2 and Phase 3 studies, each written for real people, not researchers.
Recruiting studies near Philadelphia
- NCT06363760Enrolling by invitation
Long-term follow-up after EDIT-301 gene therapy
This long-term follow-up study monitors people with sickle cell disease or transfusion-dependent beta-thalassemia who have already received the gene-editing therapy EDIT-301. It helps researchers understand how safe and effective the treatment is over time.
Philadelphia, PennsylvaniaAges 12–50 - NCT06271512Recruiting
Follow-up study for beta-thalassemia patients treated with beti-cel
This study is for people with beta-thalassemia who have already received the gene therapy betibeglogene autotemcel (beti-cel). It collects information to learn how patients are doing after this treatment in real-world settings.
Philadelphia, PennsylvaniaAges Any age - NCT04064060RecruitingPhase 3
Long-term safety follow-up for luspatercept study participants
This trial studies the long-term safety of luspatercept (ACE-536) in people who already took part in an earlier luspatercept study. It mainly helps doctors better understand ongoing side effects and overall safety over time.
Philadelphia, PennsylvaniaAges 18 years+ - NCT06364774RecruitingPhase 1/Phase 2
Gene therapy for transfusion-dependent beta thalassemia
This gene therapy trial uses a modified virus to deliver a healthy gene into your own stem cells. The goal is to help your body make normal red blood cells so you may need fewer or no transfusions.
Philadelphia, PennsylvaniaAges 18–40 - NCT04208529Enrolling by invitationPhase 3
Long-term follow-up after receiving CTX001 infusion
This study follows people for a long time after they received CTX001 in an earlier (parent) study. It helps researchers learn about long-term safety and outcomes of the treatment.
Philadelphia, PennsylvaniaAges 2 years+ - NCT07517133RecruitingPhase 3
Mitapivat for Children With Non-Transfusion-Dependent Thalassemia
This study tests a medicine called mitapivat in children ages 1–17 with thalassemia who don't need frequent blood transfusions. The medicine may help their bodies make more healthy red blood cells and reduce anemia symptoms.
Philadelphia, PennsylvaniaAges 1–17 - NCT07506863RecruitingPhase 3
Mitapivat for children with transfusion-dependent thalassemia
This study tests a study pill called mitapivat for children aged 1 to 17 with transfusion-dependent thalassemia. The goal is to see if mitapivat can reduce the need for blood transfusions and improve blood counts.
Philadelphia, PennsylvaniaAges 1–17
Hear when a new Beta-Thalassemia trial opens near Philadelphia, PA
We’ll email you when one opens near Philadelphia, PA — at most once a week, no account needed, unsubscribe anytime.
Beta-Thalassemia trials in other cities
Where these studies are running near Philadelphia
Institutions with a site for the recruiting beta-thalassemia studies listed above.
- Children's Hospital of Philadelphia · 6 studies
- Local Institution
What taking part in a beta-thalassemia study involves
A screening visit first
Before anything else, the study team checks whether you fit — usually a visit with some tests. You can stop at any point, and screening is typically free.
Care at a nearby site
Study visits happen at a clinic or hospital taking part. Many studies cover the cost of the study treatment and related visits, and some reimburse travel.
You stay in control
Taking part is voluntary and you can leave a study at any time, for any reason, without affecting your regular care.
The team decides eligibility
Our fit check is a helpful first read, not a decision. The study team makes the final call after reviewing your health history.
Looking wider? See all recruiting beta-thalassemia studies in Pennsylvania.
Common questions
- Are there beta-thalassemia clinical trials near Philadelphia?
- Yes. We're currently tracking 7 recruiting beta-thalassemia studies with a site near Philadelphia, each rewritten in plain language so you can see what it's testing and who it's for.
- How do I find out if I qualify for a beta-thalassemia study near Philadelphia?
- Each study lists its eligibility rules — age, diagnosis, prior treatments. On every trial page we explain these in plain language and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Do I have to live in Philadelphia to take part?
- Not necessarily. These studies have a site near Philadelphia, but eligibility is about your health, not your address — some people travel to take part, and a few studies reimburse travel. The study team can tell you what's required.
- Does it cost anything to join a beta-thalassemia trial?
- Using Clin2 is always free. Many trials cover the study treatment and related visits; some reimburse travel. The study team explains exactly what's covered before you decide.
Not the right time?
New beta-thalassemia studies open near Philadelphia regularly. Set up a health profile and we’ll quietly watch for studies that fit you and email you when one opens.