The FDA has lifted its temporary pause on bluebird bio’s gene therapy trials for adult, pediatric, and adolescent patients of sickle cell disease.
In February of this year, the FDA placed a hold on bluebird bio’s gene therapy trials of LentiGlobin for sickle cell disease (SCD). The pause came after two patients in the LentiGlobin trials developed blood cancer following their gene therapy treatment. Both patients experienced a suspected unexpected serious adverse reaction (SUSAR), one developing acute myeloid leukemia, and the other myelodysplastic syndrome.
Further review in March found that the AML SUSAR was “very unlikely” to have an association with the BB305 lentiviral vector. In April, additional analysis found that the MDS SUSAR had actually developed transfusion-dependent anemia, and a revised diagnosis was required.
The company is still in collaboration with trial investigators and sites to ensure a safe and effective restart to the studies.
The FDA has also lifted the hold in place on the company’s Phase III Northstar-2 and Northstar-3 trials of the related betibeglogene autotemcel gene therapy.
In a statement regarding the continuation of the studies, Andrew Obenshain of bluebird said: “we have gained deeper knowledge and understanding of the pathophysiology of sickle cell disease that will allow us to better serve patients and the broader community.”
Want to learn more?
In addition to reporting clinical trial news and stories from within the patient community, we operate Citrus, a fully integrated recruitment and retention software. Citrus fits any clinical trial and simplifies the implementation of patient engagement in your clinical trial. Whether you are already working with CTMS systems or not, Citrus is the perfect add-on to make patient interaction effective.
Since 2015, our team has been hard at work raising awareness of clinical trials across the globe. A Forbes ‘30 Under 30’ company and a graduate of the renowned Techstar program in New York, Citruslabs is at the forefront of patient recruitment and retention technologies. With over 3 million patients now recorded on our databases, our patient recruitment dashboard is fast becoming the most efficient method of trial recruitment.
Now a #1 health app in 17 countries, our Mindmate app is helping to revolutionize the way we think about healthcare. Mindmate “[takes] senior care into the digital age” (Forbes, 2018) by providing people with the cognitive decline the essential tools to help them rebuild and maintain strong mental health. Trusted by millions of users, our health community continues to grow as the content available expands to the needs of individuals today.
With more than 200 research sites now using the Citrus platform to screen thousands of patients every month, researchers are now well on their way to finding new treatments for hundreds of different health conditions - something that just would not be possible without a technological solution. Make sure that your clinical trial is in the lucky 12% and turn that luck into guaranteed results with Citrus.