February 2018

Features

Early Chart Reviews Can Provide Steady Flow of Potential Study Subjects, Experts Say

February 26, 2018

Beginning chart reviews before initiating a site can generate a list of prequalified candidates for outreach — and give sponsors the chance to examine the study’s inclusion and exclusion criteria, and their potential to cause enrollment issues, according to experts during two WCG webinars on acceptable methods for sites and sponsors, and best recruitment practices.

Briefs

NHGRI Launches Strategic Planning Process for Genomic Research and Funding

February 26, 2018

The National Human Genome Research Institute has begun to refocus its research and funding strategies with new emphasis on the development of precision medicines, exploring the basis for certain diseases and supporting the industry’s education in data science. The main federal institute behind the Human Genome Project, NHGRI plans to prioritize its efforts in emerging areas, such as the use of genomic information in patient care and drug development, and plans to gather input from the industry and the public over the next two years. The NHGRI’s strategic plan is expected to be finalized in October 2020, which commemorates the Human Genome Project’s 30th anniversary. The institute expects to prioritize emerging areas that are not well-defined, that will benefit from significant investments and are not specific to particular diseases. Well-established areas, such as cancer and microbial genomics, are expected to be deemphasized during the upcoming process, the institute said.

Pipeline

2018-02-26: New Progress in Treatments for Cystic Fibrosis, Skin Conditions and Cancer

February 26, 2018

Company Drug/Device Medical Condition Status
Arch Biopartners AB569 antibiotic-resistant bacterial infections in the lungs of cystic fibrosis and chronic obstructive pulmonary disease Phase I trials initiated in the U.S.
Xencor XmAb18087 neuroendocrine tumors (NET) and gastrointestinal stromal tumors (GIST) Phase I trials initiated
PQ Bypass DETOUR Long (>15 cm) superficial femoral artery (SFA) blockages Phase I trials initiated enrolling 292 subjects in the U.S. and Europe
Therachon TA-46 achondroplasia Phase I trials initiated enrolling 70 subjects in the Netherlands
OncoPep PVX-4106 metastatic triple negative breast cancer (TNBC) who are human leukocyte antigen A2 positive (HLA-A2+) Phase Ib trials initiated
Bonti EB-001 scar reduction Phase II trials initiated
AbbVie JAK1-selective inhibitor adult patients with moderate to severe atopic dermatitis Phase II trials initiated
IRX Therapeutics IRX-2 squamous cervical intraepithelial neoplasia 3 or vulvar intraepithelial neoplasia 3 Phase II trials initiated
Glenmark Pharmaceuticals GBR 830 moderate to severe atopic dermatitis Phase IIa trials initiated enrolling 64 subjects
Centrexion Therapeutics CNTX-4975 Chronic moderate to severe knee osteoarthritis Phase III trials initiated enrolling 325 subjects
AstraZeneca IMFINZI® unresectable Stage III non-small cell lung cancer following CRT Phase III trials initiated
Celgene OTEZLA \(apremilast) active Behçet’s Disease Phase III RELIEF trials initiated
Vertex Pharmaceuticals Incorporated VX-659 cystic fibrosis Phase III trials initiated enrolling 360 patients
Eli Lilly Taltz (ixekizumab) moderate to severe genital psoriasis Phase IIIb trials initiated enrolling 149 subjects
Evofem Biosciences Amphora urogenital chlamydia infection in women Fast Track designation granted by the FDA
Novartis Cosentyx (secukinumab) moderate to severe plaque psoriasis FDA approved label update
Trial Results

Celgene announced OTEZLA study Showed Improvements in Patients with Oral Ulcer

February 26, 2018

Celgene announced that data from the Phase III RELIEF clinical trial of OTEZLA(apremilast) in patients with active Behçet’s Disease with oral ulcers showed statistically significant reductions in oral ulcers with apremilast 30mg twice daily (BID) versus placebo through week 12. OTEZLA (apremilast) is Celgene’s oral selective inhibitor of phosphodiesterase 4 (PDE4). The RELIEF study is a Phase III randomized, placebo-controlled, double-blind study evaluating apremilast 30mg BID in 207 patients with active Behçet’s Disease who were previously treated with at least one topical or systemic medication. This 52-week study was conducted at 63 sites across ten countries. In the study, a total of 207 patients were randomized to apremilast 30mg BID or placebo. At week 12, the area under the curve (AUC) for the number of oral ulcers was statistically significantly reduced with apremilast 30mg BID versus placebo (129.5 vs. 222.1; P<0.0001), the trial’s primary endpoint. The most common adverse events observed in the trial were diarrhea (41.3 percent with apremilast, 19.4 percent for placebo), nausea (19.2 percent with apremilast, 10.7 percent for placebo), headache (14.4 percent for apremilast, 9.7 percent for placebo) and upper respiratory tract infection (11.5 percent for apremilast, 4.9 percent for placebo). This study primarily evaluated the effect of apremilast on recurring oral ulcers in patients with active Behçet’s Disease who were previously treated with at least one topical or systemic medication.

CWMarketplace

2018-02-26: Ten Industry service provider profiles

February 26, 2018

CWMarketPlace is a monthly section featuring a range of clinical research service providers who have Industry Provider Profile pages posted on CenterWatch.com. Included in their annual subscriptions, company profiles are randomly selected to appear in this section, providing added exposure for their products and services. To learn more about becoming an Industry Provider Profile page subscriber, contact Sales at (617) 948-5100.

Click on any provider to view the company’s complete online profile or search more profiles.

Features

Sensors and Wearables Transform Clinical Trials but Challenges Remain, Experts Say

February 19, 2018

With a multitude of sensors, wearables and mobile devices becoming available for use in clinical trials, sponsors should conduct systematic comparisons before designing protocols, according to experts at the annual SCOPE conference, who presented the work they’ve done to demonstrate the value of using digital monitoring in their studies, as well as the obstacles they encountered.

Briefs

FDA Releases Guidances on Clinical Trial Design for Neurological Treatments

February 19, 2018

The FDA has released five guidances on clinical trial designs and endpoints for developing treatments for a variety of neurological disorders, including Alzheimer’s disease, Duchenne muscular dystrophy (DMD) and ALS. The revised draft guidance on Alzheimer’s disease noted a greater focus on evaluating drug treatments during the disease’s earliest stages, partially due to the failures of clinical trials that aimed to alter later stage disease progression. Diagnostic criteria that reliably define an early Alzheimer’s population are suited for evaluating drugs that try to delay or prevent overt symptoms, the agency said. Enrollment eligibility for Alzheimer’s drug efficacy trials should be centered around current consensus diagnostic criteria, focusing on objective tests in addition to history and physical examination when appropriate, to determine the disease’s presence and rule out similar conditions. Alzheimer’s Stage 1 patients — who exhibit evidence of clinical impact — are important targets for clinical trials, although clinically meaningful benefits cannot be measured in them due to a lack of clinical impairment. However, effects on the characteristic pathophysiologic changes of AD — demonstrated by effects on biomarkers — may be measured in these patients, and the measurements may serve as the basis for an accelerated trial, as the biomarker effects would be “reasonably likely to predict clinical benefit,” with a post-approval study required for confirmation. The agency’s final guidance on DMD and related dystrophinopathies said endpoints measuring change of function in a wide range of deficits may offer advantages in the development of drugs that treat diseases like DMD and Becker muscular dystrophy, in addition to increasing the number of patients eligible for enrollment. Sponsors should consider using endpoints that are able to assess function across different stages of the disease — such as combining measures of upper body function and ambulation — and detect improvement and decline from baseline in order to capture the range of possible beneficial drug effects. Patient-reported outcomes (PRO) can be useful for assessing clinical meaningfulness of relatively small objective findings, and for contributing benefit and risk assessments. PRO instruments for dystrophinopathies should, in general, “include a limited number of items that assess the most important aspects of the outcome of interest.” The final guidance on drug development for acute treatment of migraines, which does not apply to over-the-counter products, elaborated on ideal trial design, trial population and entry criteria, dose selection, efficacy endpoints and concomitant medications, among other topics. Draft guidance issued on drugs for the treatment of partial onset seizures explained that “efficacy can be extrapolated from adults to pediatric patients when it is reasonable to assume that children, compared with adults, have a similar progression of disease, similar response of disease to treatment, and similar exposure-response relationship.” The draft guidance on developing drugs for ALS treatment addresses the clinical development of drugs intended to treat the main neuromuscular aspects of the disease and focuses on specific clinical drug development and trial design issues that are unique to the study of ALS. The guidances “signal how modernization of the new drug regulatory program includes an enhanced focus on incorporation of patient input into our thinking,” said FDA Commissioner Scott Gottlieb. Read the five guidances here: www.fdanews.com/02-15-18-Guidances.pdf.

Three Questions

Three Questions: Marie Emms, Syneos Health

February 19, 2018

CWWeekly presents this feature as a spotlight on issues faced by executives in clinical research. This week, writer Karyn Korieth spoke with Marie Emms, head of Patient Engagement at Syneos Health, who has 20 years’ experience in global patient recruitment and retention strategies for clinical trials.