Back in June, Oxford BioMedica announced
that it had voluntarily paused recruitment for its clinical trials for wet AMD (RetinoStat Phase I), Stargardt’s Disease (StarGen Phase I/IIa) and Usher’s Syndrome (UshStat Phase I/IIa). The company had halted recruitment of the aforementioned studies, as a precautionary measure, while it investigated the detection of very low concentrations of a potential impurity in its clinical trial material derived from a third party raw material.
Oxford has since performed extensive characterization studies using its newly developed, state-of-the-art analytical methods to identify the impurity as highly fragmented DNA derived from fetal bovine serum (FBS), the most widely-used growth supplement for cell culture media. In light of these findings, Oxford remains convinced of the safety, integrity and quality of its LentiVector platform products and no safety concerns relating to any of the ocular products have been identified in any pre-clinical and clinical data generated to date.
Today, the company announced
that following the submission of a comprehensive data package to the FDA and the French regulatory agency, ANSM, it has received agreement from both agencies to resume recruitment into its ocular clinical trials using the existing clinical trial material. The company will continue to use highly sensitive, state-of-the-art analytical methods to ensure the quality and integrity of its lentiviral vector products and will work with FDA and ANSM to define the necessary specifications for future batches of clinical trial material.
Oxford is now working closely with the clinical trial centers to obtain the necessary ethics committee approvals in order to resume recruitment into the clinical studies.
(For a list of the clinical site centers in the U.S. and France involved in the three studies, please take a look at my Gene Therapy Ongoing Clinical Trial Table
John Dawson, Chief Executive Officer of Oxford BioMedica, said: "We value our relationships with the regulatory authorities and are pleased that, on the basis of our extensive technical investigations to demonstrate the integrity of our products, FDA and ANSM agree with our proposal to resume treating patients in our ocular trials as soon as possible.
"We place the highest importance on safety, and our analytical methods and quality assurance processes are continuously evolving to ensure that we remain at the forefront of gene therapy development and manufacture. I am confident that, with significant opportunities ahead such as the recently-announced AMSCI project win, Oxford BioMedica will continue to lead the way in delivering novel gene therapies to patients."
For your information, Oxford BioMedica has reported that 9 of the 18 patients to be treated in the wet AMD clinical trial had been treated; 12 of the 28 patients in the Stargardt’s trial; and 3 of 18 patients in the Usher Syndrome trial had been treated prior to the halt in recruitment in June.
Coincidently, Genzyme, who is also running a gene therapy clinical trial to treat the wet form of AMD, also announced a halt in recruitment for its trial in July. No reason for the stoppage has been given and all attempts to determine why the halt in recruitment occurred have been rebuffed. As of the last time I had obtained reliable information about the Genzyme trial, 6 of 34 patients to be treated had been treated.
Genzyme Update – October 25, 2013
After many attempts to determine why Genzyme halted its clinical trial recruitment, I have finally received the answer. Here is the statement received from a spokesperson from Genzyme:
“We enrolled 19 patients in this clinical trial, all of whom have been treated. The protocol stated that we would enroll "up to 34" patients, but that number accounted for the possibility of replacing patients who withdrew early from the trial. Since no patients withdrew from the trial, we did not need to recruit 34 patients in order to meet the target enrollment numbers. The protocol specified that we planned to enroll 12 patients in the dose escalation part of the trial, and 10 patients in the second part of the trial, for a total of 22 patients. We stopped enrollment at 19 (three short of this target) purely because our clinical material was coming to the end of its stability protocol. There were no safety or product quality issues. We continue to monitor the 19 patients who were treated in our trial.”
Thank you Genzyme for providing this update.