Clinical Trials

The Cooley’s Anemia Foundation provides these links for informational purposes only. The Foundation is not endorsing any one trial over others.

Click on the trials listed below for more information.  Go to and search for “thalassemia” for a more complete list of trials.


Alicia Somma, who has thalassemia, recently participated in a clinical trial at Memorial Sloan-Kettering (#NCT00658385, “Assess the Feasibility and Safety of GCSF Mobilization of CD34+ Hematopoietic Progenitor Cells in Patients With Betathalassemia Major,” which is examining whether the use of GCSF will increase the number of stem cells produced; this is important in preparing for future gene therapy trials.) Below, Alicia answers questions about her experience.

Why did you decide to participate in this trial?
I decided to participate in this study because it is, to me, the most exciting thing we have been offered as thalassemia patients. So many great things have been accomplished in recent years due to scientific research, so when asked to be a part of it, I couldn’t see myself saying no. If there had been no one willing to participate in past studies, Exjade wouldn’t be offered to us now, and look what that has done for the quality of life for patients!
This study is an important one that will give us information that may help with gene therapy, which will help to improve our lives even more, and I certainly couldn’t see myself opting out. A better life is great, but I want the best life to be able to be offered to thal patients of the future. I believe this study can help accomplish that.

What was involved in the trial?
The trial was completed in 6 days. The first four days I was given a subcutaneous shot of GCSF, which is commonly used for other purposes in medicine, and is used in the study to help stimulate the production of stem cells. Although I am plenty used to giving myself sub-q needles from using Deserfal, basically my entire life, and it was offered to me to do the shots myself at home, I had the nurses at Sloan-Kettering administer the GCSF because I wanted to see Dr. Boulad, who was guiding me in the study, on a daily basis.
The actual process to remove my stem cells was done through a process on day 5 called leukapheresis. The procedure takes about 3 hours (duration varies depending on the weight
of the person undergoing the pheresis), and was relatively comfortable and not complicated. Whole blood is taken and separated in a machine, and what is not needed is given back simultaneously-this is all done through two IVs.
This study’s goal is to collect 8 million stem cells, which is very ambitious, and so most participants need to undergo leukapheresis for both days 5 and 6.

How did you feel about the experience?
As I said in my reasons to participate in the study in the first place, I was just excited. Although the GCS-F shots had some side effects of joint pain, and the pheresis wasn’t the most fun way to spend my mornings, when all was said and done, everything was worth the mild inconvenience. The entire week I was just so happy to be helping these scientists who have spent years and dedicated so much work to helping my friends and me. It was a wonderful experience.

What would you say to encourage others to think about participating in a clinical trial for thalassemia?
If I haven’t already said enough good things about participating in studies, let me continue!
I think that some younger thal patients, like myself, may not realize all that the older patients have done for us to help our lives be as easy as they are. We take Desferal for granted, and yet there were so many brave patients who tried it for the first time and that is what made it so avaiable and safe for us our entire lives.
Thalassemia patients need to look out for one another, and this is the easiest way to do that. Get involved, and donate your time so that the next generation born with this disease will have an even better quality of life than you. If you’re not going to help “future you’s,” why should anyone else?

Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Severe Variants of Beta 0/+Thalassemia, THALLO

Glutamine Therapy for Hemolysis-Associated Pulmonary Hypertension

Iron Balance Study of Deferasirox, Deferoxamine and the Combination of Both

Multi-Center Study of Iron Overload: Pilot and Survey Study (MCSIO) (Study is no longer recruiting patients)

An Open Label, Multi-center, Efficacy and Safety Study of Deferasirox in Iron Overloaded Patients With Non-transfusion Dependent Thalassemia (MACS1935)



A Study Evaluating the Safety and Efficacy of the LentiGlobin® BB305 Drug Product in Beta-Thalassemia Major Patients

Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)-thalassemia

Study of ACE-536 to Evaluate the Effects of ACE-536 in Patients With Beta-Thalassemia Intermedia

Zinc and Diabetes in Patients with Thalassemia: A Pilot Study

The following information on informed consent is taken from the web page on “Understanding Clinical Trials.” Click here to view the entire page.
What is informed consent?
Informed consent is the process of learning the key facts about a clinical trial before deciding whether or not to participate. It is also a continuing process throughout the study to provide information for participants. To help someone decide whether or not to participate, the doctors and nurses involved in the trial explain the details of the study. If the participant’s native language is not English, translation assistance can be provided. Then the research team provides an informed consent document that includes details about the study, such as its purpose, duration, required procedures, and key contacts.
Risks and potential benefits are explained in the informed consent document. The participant then decides whether or not to sign the document. Informed consent is not a contract, and the participant may withdraw from the trial at any time.
What kind of preparation should a potential participant make for the meeting with the research coordinator or doctor?
Plan ahead and write down possible questions to ask.
Ask a friend or relative to come along for support and to hear the responses to the questions.
Bring a tape recorder to record the discussion to replay later.
Every clinical trial in the U.S. must be approved and monitored by an Institutional Review Board (IRB) to make sure the risks are as low as possible and are worth any potential benefits. An IRB is an independent committee of physicians, statisticians, community advocates, and others that ensures that a clinical trial is ethical and the rights of study participants are protected. All institutions that conduct or support biomedical research involving people must, by federal regulation, have an IRB that initially approves and periodically reviews the research.
What should people consider before participating in a trial?

People should know as much as possible about the clinical trial and feel comfortable asking the members of the health care team questions about it, the care expected while in a trial, and the cost of the trial. The following questions might be helpful for the participant to discuss with the health care team. Some of the answers to these questions are found in the informed consent document.

What is the purpose of the study?
Who is going to be in the study?
Why do researchers believe the experimental treatment being tested may be effective? Has it been tested before?
What kinds of tests and experimental treatments are involved?
How do the possible risks, side effects, and benefits in the study compare with my current treatment?
How might this trial affect my daily life?
How long will the trial last?
Will hospitalization be required?
Who will pay for the experimental treatment?
Will I be reimbursed for other expenses?
What type of long-term follow up care is part of this study?
How will I know that the experimental treatment is working? Will results of the trials be provided to me?
Who will be in charge of my care?