Clinical News Roundup: HHS To Provide More Info To Patients

By Ed Miseta, Chief Editor, Clinical Leader

In an effort to make information about clinical trials widely available to the public, the U.S. Department of Health and Human Services (HHS) has issued a final rule that outlines requirements for registering certain clinical trials and submitting summary results information to ClinicalTrials.gov. The new rule expands the legal requirements for submitting registration and results information for clinical trials involving Food and Drug Administration (FDA)-regulated drug, biological and device products. At the same time, the National Institutes of Health (NIH) has issued a complementary policy for registering and submitting summary results information to ClinicalTrials.gov for all NIH-funded trials, including those not subject to the final rule.

“Access to more information about clinical trials is good for patients, the public and science,” said NIH Director Francis S. Collins. “The final rule and NIH policy we have published today will help maximize the value of clinical trials, whether publicly or privately supported, and help us honor our commitments to trial participants, who do so much to help society advance knowledge and improve health.”

Expanding the registration information in ClinicalTrials.gov improves people’s ability to find clinical trials in which they may be able to participate and access investigational therapies. More information about the scientific results of trials, whether positive or negative, may help inform healthcare providers and patients regarding medical decisions. Additional information will help researchers avoid unnecessary duplication of studies, focus on areas in need of study and improve study designs, ultimately advancing the development of clinical interventions.

Requirements under the final rule apply to most interventional studies of drug, biological, and device products that are regulated by the FDA. The requirements do not apply to Phase 1 trials of drug and biological products, or small feasibility studies of device products. The final rule specifies how and when information collected in a clinical trial must be submitted to ClinicalTrials.gov. It does not dictate how clinical trials should be designed or conducted, or what data must be collected.

FDA's Woodcock Supports Duchenne Patients With Sarepta Approval

The U.S. Food and Drug Administration has approved Exondys 51 (eteplirsen) injection, the first drug approved to treat patients with Duchenne muscular dystrophy (DMD). Exondys 51 is specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13 percent of the population with DMD.

“Patients with a particular type of Duchenne muscular dystrophy will now have access to an approved treatment for this rare and devastating disease,” said Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research (CDER). “In rare diseases, new drug development is especially challenging due to the small numbers of people affected by each disease and the lack of medical understanding of many disorders. Accelerated approval makes this drug available to patients based on initial data, but we eagerly await learning more about the efficacy of this drug through a confirmatory clinical trial that the company must conduct after approval.”

DMD is a rare genetic disorder characterized by progressive muscle deterioration and weakness. It is the most common type of muscular dystrophy. DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. The first symptoms are usually seen between three and five years of age, and worsen over time. The disease often occurs in people without a known family history of the condition and primarily affects boys, but in rare cases it can affect girls. DMD occurs in about one out of every 3,600 male infants worldwide.

People with DMD progressively lose the ability to perform activities independently and often require use of a wheelchair by their early teens. As the disease progresses, life-threatening heart and respiratory conditions can occur. Patients typically succumb to the disease in their 20s or 30s; however, disease severity and life expectancy vary.

Exondys 51 was approved under the accelerated approval pathway, which provides for the approval of drugs that treat serious or life-threatening diseases and generally provide a meaningful advantage over existing treatments. Approval under this pathway can be based on adequate and well-controlled studies showing the drug has an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit to patients (how a patient feels or functions or whether they survive). This pathway provides earlier patient access to promising new drugs while the company conducts clinical trials to verify the predicted clinical benefit.

Duke Clinical Research Institute To Coordinate National Study Of Childhood Health

The Duke Clinical Research Institute (DCRI) has been named the coordinating center as part of a $157-million federal initiative involved in studying how environmental factors affect childhood health. The seven-year grant from the National Institutes of Health (NIH) will fund the organizational framework of the Environmental Influences on Child Health Outcomes (ECHO) initiative.

As the coordinating center for the research initiative, the DCRI will provide support to the study’s steering committee, lead site training for participating research teams, develop common rules and standard procedures, monitor quality controls, and establish and maintain websites and other communications tools. The ECHO coordinating center at DCRI will also include an Opportunities and Infrastructure Fund to support pilot projects, encourage development of junior investigators, and introduce new tools and technologies in the context of the ECHO program. 

 “We are certainly honored to be selected as the coordinating center for this important research initiative at the NIH,” said principal investigator Brian Smith, M.D., a Duke neonatologist and faculty member of the DCRI. “This builds on a number of our strengths in clinical research, notably in pediatric clinical research, where we have developed specific expertise.”

 The awards will build the infrastructure and capacity for the ECHO program to support multiple longitudinal studies that extend and expand existing studies of mothers and their children. ECHO research will focus on factors that may influence health outcomes around the time of birth as well as into later childhood and adolescence.  

Owlstone Medical Commences Next Phase In World’s Largest Breath-Based Clinical Trial

Diagnostics company Owlstone Medical has announced it is expanding its Lung Cancer Indicator Detection (LuCID) clinical trial. The study, originally funded by the Small Business Research Initiative for Healthcare (SBRI Healthcare), an NHS England funded initiative, aims to save 10,000 lives and save the NHS £245 million by 2020 with a quick, non-invasive and high-compliance breath test to detect lung cancer. The study will recruit up to 3,000 patients across 21 sites in the UK and Europe, making it the world’s largest breath-based study ever undertaken for early cancer detection.

If detected at stage 1, the 5-year survival rate for lung cancer is 54 percent, but this drops to just 4 percent if the cancer is detected at stage 4. Current lung cancer screening tests, such as low dose computed tomography (CT screening), have been shown to result in a high number of false-positives and therefore unnecessary, invasive follow-up procedures on healthy patients.

The mutations that drive the development of lung cancer have downstream effects on the metabolites we exhale, even in the very early stages of lung cancer. Owlstone Medical uses the Respiration Collector for In Vitro Analysis (ReCIVA), in combination with the Field Asymmetric Ion Mobility Spectrometer (FAIMS) sensor platform, to accurately and selectively detect lung cancer volatile organic compounds (VOCs) in breath.

The trial is moving to the next phase as a consequence of positive interim sensitivity and specificity data, when compared to other available tests, with over 97 percent of patients reporting the breath test to be comfortable to perform. The study is being led by Dr. Robert Rintoul, lead clinician for Thoracic Oncology and Director of the Clinical Trials Unit at Papworth Hospital, Cambridge, UK, and will recruit patients in order to validate breath biomarkers for the early detection of cancer and differentiation between benign and malignant tumors, which is a major problem in current screening approaches. The trial will compare Owlstone Medical’s platform with current CT and PET screening tests, with the aim of demonstrating its ability to reduce the number of false-positive results and complications seen from invasive biopsy follow-ups.