If your study involves the use of a new drug, or the new use of an existing drug, the IRB process isn’t the only hurdle you’ll have to jump.  In order to conduct a trial using one of these, the FDA requires approval via an Investigational New Drug filing (IND), and the IRB must have the IND number before approving the protocol.  Hurdles like this can make the starting point of enrolling your first research subject seem far away, but the good news is  that you don’t have to do it alone. 

Dr. Helen Mintz-Hittner is the Clinical Professor holding the Alfred W. Lasher, III Professorship of Ophthalmology, and is the PI of a pivotal study to treat Retinopathy of Prematurity.  Since her research subjects are younger than four weeks old, they qualify as a vulnerable population and require a more complex protocol submission to the IRB that focuses on proving safety and minimizing risks.  The investigational treatment in the protocol uses bevacizumab, also known as Avastin, which is an approved drug for treating cancer in adults.  Although Dr. Hittner had already begun her IRB submission on her own- because this was off-label use in a vulnerable population, it became obvious that creating the IND filing would require outside help. 

When Dr. Hittner called the CCTS’s Regulatory Component, she spoke with Sr. Regulatory Affairs Specialist, Gena Monroe, who was very encouraging about the otherwise daunting process.  Monroe led her through the needed steps of her over 700+ page IND filing, which was successfully submitted and approved with no additional requirements made by the FDA’s reviewing division.  The process was long and detailed, but their collaborations proved worthwhile.  Dr. Hittner enjoyed working with Gena because “her guidance was so perfect that there were no unanswered questions or lacking forms.  This was indeed a miracle for a novice like me to obtain an IND so quickly.”  Gena also enjoyed working with Dr. Hittner, noting that this “model PI” is “very motivated and a dedicated researcher and doctor. She’s also exceptionally organized and detail oriented, which is what helps make FDA filings like this so successful.” Monroe also guided Dr. Hittner through the registration of the trial on clinicaltrials.gov, which is FDA required for such studies. “She’s one of the best researchers we have listed in the registry- Dr. Hittner regularly updates her study while I have to keep reminding other investigators just to register!”

The next steps were to process the protocol through the UTHSC-H IRB and Memorial Hermann Hospital, where approvals were granted.  When other hospitals both in and outside of Texas began to take notice that Dr. Hittner’s study had “navigated through shark infested regulations” and survived, they started asking to be included in the trial. Dr. Hittner then had new regulatory challenges: coordinating the addition of multiple sites operating under her IND, and maintaining compliance as the Sponsor Investigator.   Monroe provided guidance for this and also contacted both the Legal and Sponsored Projects departments of UTHSC-H, who then drafted the many contracts with the other hospitals.  This is now a nation-wide study, and may even become international, if Dr. Hittner decides to allow several South American sites to be included.  Each of the research sites have to go through their own institutional IRBs, but the burden of those processes has been greatly reduced because of the prior preparations and guidance provided by Gena Monroe.

The study will be enrolling at least 150 infants (they have 17 so far) with a 12 month period of follow up.  A very encouraged Dr. Hittner says, “Provided there is no toxicity, and we have not encountered any so far, Avastin therapy will change the standard of care for the treatment of Retinopathy of Prematurity in this country and throughout the world.” From the first phone call to effectively and compliantly becoming a multi-center study, the Regulatory Office has been invaluable to Dr. Hittner, who definitely recommends contacting the Regulatory Component of the Center for Clinical and Translational Sciences to other researchers.

Gena Monroe can be contacted at the Center for Clinical and Translational Sciences at (713) 500-7903 or by email at Gena.Monroe@uth.tmc.edu.

Jeanette Podbielski, R.N. is the study coordinator of a trial, in which the subjects are trauma patients in the ER.  The purpose of the study is to assess if trauma patients who require massive transfusion can be accurately predicted early after they arrive in the emergency department.  They will also assess if the use of whole blood during initial resuscitation will reduce the transfusion needs as compared to component therapy.  The component therapy is the current practice, which involves the use of packed red blood cells, fresh frozen plasma, and platelets.  With the possibility of a blood transfusion as opposed to the components, the hopeful outcome of the study is to improve the patient’s outcome. 

Because the study will be involving subjects who may not be able to give informed consent due to their traumatic and possibly unconscious state, the protocol allows for the use of a waiver of consent.  The waiver of consent is allowed when the conditions are life threatening, when there is a possibility for a direct benefit, when nothing is denied that would otherwise be done, and when a previous community consultation has occurred.  Podbielski will be coordinating these consultations with the community.  Once the study begins, she will be coordinating the 24-hour coverage to assure the data is obtained on the patients who will fit the criteria for the study.  There will also be medical students assisting with collecting the data.

Although Podbielski has been working with research at UTHSC-H for seventeen years, she has only recently begun working with the trauma surgery team.  When asked about the most challenging aspect of this study as the coordinator, she replied “I am not sure if I would call it challenging; however, the whole concept of waiver of consent and the process of getting community consent to start the study is very interesting.”