After a year of unprecedented and transformative adaptation in clinical research, on February 5th 2021, industry experts came together to share their collective reflection on decentralized clinical trial (DCT) modalities and the role they will play in securing the advancement of tomorrow’s research. Join us in this first part of the conversation as we discuss remote modalities across the lifecycle of a trial and cover the following topics:
- Effective DCT feasibility and planning, moderated by Edye Edens
- Remote patient recruitment and inclusion of diverse patient populations, moderated by Raymond Nomizu
- Modalities and thoughts for obtaining informed consent remotely, moderated by Maya Zlatanova
- QA/QC and remote monitoring, moderated by Daniel Perez
Daniel Perez is the Chief Operating Officer at MACRO Trials. MACRO is a Site Optimization Organization (SOO) dedicated to providing the institutional infrastructure to perform the highest quality clinical trials across all specialties and private settings. At MACRO, Daniel combines his operational acumen with research and regulatory expertise to help bring its vision into a tangible reality. He leads MACRO’s operations by constantly challenging the status quo, and continuously evaluating the clinical trial process through the lens of the patient. Daniel also serves as a founding board member for Clinical Trials in Color, a 501(c)(3) non-profit foundation with a mission to improve health outcomes in communities of color by increasing diversity and access to clinical trials.
Edye T. Edens, JD, MA, CIP, CCRP is the Senior Research Compliance Consultant at First Class Solutions focusing on research compliance and life sciences. Edye is a licensed attorney with an international human rights, research ethics, and health law background. Her consulting services include research administration, healthcare compliance, grants/contracts to IRB, COI, education, privacy, HIPAA, multiple areas of FDA compliance related to drugs, devices, and food; AAHRPP, misconduct, and site-level compliance work as it relates to QA, monitoring, and auditing (particularly oncology).
Maya Zlatanova is a clinical research entrepreneur and the co-founder of FindMeCure Ltd. The company has been focusing on data intelligence tools to support operational teams to better plan clinical trials closer to patients. FindMeCure’s platform – TrialHub, is the first one to connect sites with experience in remote support to patients with potential decentralized trials and supports the decision-making process around technology deployment in 70 countries. Mrs. Zlatanova has been also advocating for improving patient recruitment through easier access of patients to clinical trials information and direct-to-patient strategies.
Raymond Nomizu is the co-founder of Clinical Research IO, a site-centred electronic Source and CTMS solution. Clinical Research IO allows research sites to collect data accurately and contemporaneously and sponsors to view the data in real-time, without costly source data verification. Prior to founding CRIO, Raymond owned and operated an independent clinical research site, co-founded a real estate data analytics firm, and served as a management consultant focusing on process improvement. Raymond has AB and JD degrees from Harvard University.
David Lazar, MD, Ophthalmologist, Retina and Macular Disease Specialist and Clinical Investigator at MACRO Trials in Los Angeles, discusses clinical trials for rare inherited retinal diseases (IRD) retinitis pigmentosa, Stargardt disease and choroideremia.
Question: Currently, October 2020, listed on clinicaltrials.gov there are only 2 choroideremia, 9 Stargardt disease and about 50 retinitis pigmentosa studies being recruited for throughout the world. Why are there so few clinical trials for these inherited retinal diseases?
Dr. Lazar: These inherited eye diseases are very rare. Even the most common of the three, retinitis pigmentosa, affects only 1 in 3,500 to 4,000 in the U.S. and Europe. Stargardt disease affects about 1 in 8,000 to 10,000 and choroideremia is estimated to affect 1 in 50,000 to 100,000.
As a vitreoretinal specialist in a populous city such as Los Angeles, I see patients with these retinal diseases; however, a clinical trial for one of these rare diseases, will include several geographic trial locations to recruit the number of patients necessary to conduct a robust trial.
Question: Can those three inherited retinal diseases be studied in one clinical trial?
Dr. Lazar: Each of those inherited retinal diseases occurs due to a particular gene mutation or damage. With choroideremia, there is an issue with the CHM gene responsible for signaling the production of a protein called REP-1 that is required for retinal health. Stargardt disease is most frequently due to a mutation in the ABCA4 gene that prevents the ABCA4 protein from removing toxins from photoreceptor cells. Retinitis pigmentosa can occur when there is harm to one or more of 50 genes that transport instructions for making photoreceptors. After the Human Genome Project was completed in April 2003 and science was able to identify the specific gene for each condition, the door opened to the possibility of novel therapies.
Genetic testing makes it possible to provide a definitive diagnosis for a patient and helps to enroll patients in clinical trials specific to their condition. My desire is to ensure that patients participate in all clinical trials available to them. My website at https://irdclinicaltrials.com/understanding-irds/ is dedicated to helping patients understand and navigate clinical trials. In addition, the site features a secure and confidential sign-up form for patients or their guardians who wish to be notified when a clinical trial becomes available.
Question: How would a clinical trial for an inherited retinal disease be performed?
Dr. Lazar: I have served as a co-investigator in several national clinical trials for the treatment of dry and wet macular degeneration. The therapies that are available and helping patients with macular degeneration today were not available before 2005. Patients diagnosed with retinitis pigmentosa, Stargardt disease or choroideremia have likely been told that their condition is incurable, leads to blindness and that there are no available treatments. Novel clinical trials are just beginning for these diseases in 2020. My goal is to give inherited retinal disease patients the hope and opportunity experienced by patients with macular degeneration by enrolling them in and performing clinical trials to discover treatments.
As principal investigator, I would recruit, administer and track inherited retinal disease patients over the course of the clinical trial. Patients who fulfill the trial parameters would be enrolled in Phase III of an FDA-approved study. I would administer the therapeutic, such as perform surgery, administer injection or prescribe medications. Tracking the patient during the course of the trial and noting outcomes ensure patient safety and treatment efficacy. Ideally, a study would result in a novel therapy that becomes an ongoing protocol.
Question: Why focus on choroideremia, Stargardt disease and retinitis pigmentosa clinical trials?
Dr. Lazar: Regarding those particular inherited retinal diseases, there are no treatments available. Nothing. Any new discovery could be a game changer for IRD patients. Generally, patients diagnosed with these conditions are young and looking to medical research to help with a discovery to improve or prolong their vision. I am motivated by how motivated patients are to be involved in helping not just themselves, but others who share their conditions.
Twenty years after the completion of the Human Genome Project, science is becoming more focused on targeted gene therapy, which appears promising for potential treatments for retinitis pigmentosa, Stargardt disease and choroideremia, which are all caused by genetic defects. The anatomy of the retina makes it especially conducive for translational gene therapy. As a physician and researcher, helping to facilitate clinical trials for patients with IRDs is extremely rewarding. Clinical trials lead the medical community to life-changing treatments, and being at the forefront of IRD research provides my patients the possibility to early access of novel therapeutics.
About Dr. David Lazar
David Lazar, MD, is a board-certified ophthalmologist with subspecialty training in medical and surgical diseases of the retina. As a vitreoretinal specialist, Dr. Lazar is well known for his surgical skill and expertise in diagnosing and treating issues that affect the retina and the vitreous body. As a practitioner and researcher, he continually seeks the best treatments available for conditions that affect eye health and threaten vision.
With a database of numerous IRD patients, including those at his West Los Angeles practice, Dr. Lazar seeks to perform clinical trials to develop novel therapies specifically for retinitis pigmentosa, Stargardt disease and choroideremia. Contact Dr. Lazar at 866-773-8462, or learn more about his practice at https://lazarretina.com and clinical trials at https://irdclinicaltrials.com/understanding-irds/.
About MACRO Trials
MACRO Trials (MACRO) was founded in 2009 by physician colleagues passionate about performing high-quality research within their specialties. After establishing a record of conducting industry-sponsored and investigator-initiated trials in the fields of aesthetics, dermatology and plastic surgery, MACRO applied their unique research framework to support other principal investigators in private practice. Working closely with sponsors and contract research organizations, MACRO went on to facilitate and optimize research without the associated administrative and regulatory headaches. With a mission to change the culture of clinical research, and an eye toward a technologically adaptive research paradigm, MACRO is evolving to continue to incorporate best practices in clinical trial optimization.