2018

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 The revised draft guidance on Alzheimer’s disease noted a greater focus on evaluating drug treatments during the disease’s earliest stages, partially due to the failures of clinical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.

Pipeline

Twenty-one Drugs and Devices have Entered a New Trial Phase This Week

February 19, 2018

Company Drug/Device Medical Condition Status
Applied Therapeutics AT-001 diabetic complications Phase I trials initiated
Aravive Biologics AVB-S6-500 n/a Phase I trials initiated enrolling 40 subjects in the U.S.
Mallinckrodt ExpressGraftC9T1 skin tissue diabetic foot ulcers Phase I trials initiated enrolling six subjects
Pulmatrix Pulmazole (PUR1900) allergic bronchopulmonary aspergillosis related to asthma Phase I trials initiated enrolling 42 subjects
Vyera Pharmaceuticals TUR-006 toxoplasmosis Phase I trials initiated
MyoKardia MYK-491 dilated cardiomyopathy Phase Ib trials initiated enrolling 64 subjects
Arrowhead Pharmaceuticals ARO-HBV chronic hepatitis B virus infection Phase I/II trials planned enrolling 30 subjects in New Zealand
Audentes Therapeutics AT342 Crigler-Najjar syndrome Phase I/II trials initiated
Sienna Biopharmaceuticals SNA-125 psoriasis and the associated pruritus Phase I trials initiated enrolling 15 subjects
Imara IMR-687 sickle cell disease Phase IIa trials initiated enrolling 50 subjects in the U.S.
Nohla Therapeutics NLA101 acute myeloid leukemia Phase II trials initiated enrolling 220 subjects in the U.S.
Citius Pharmaceuticals, Inc. Mino-Lok Therapy catheter related bacteremias Phase III trials initiated enrolling 700 subjects in the U.S.
Intercept Pharmaceuticals obeticholic acid cirrhosis due to NASH Phase III trials planned enrolling 540 subjects
Pfizer PF-04965842 moderate to severe atopic dermatitis Breakthrough Therapy designation granted by the FDA
Abeona Therapeutics ABO-202 infantile Batten disease Orphan Drug designation granted by the FDA
AstraZeneca, Merck selumetinib neurofibromatosis type 1 Orphan Drug designation granted by the FDA
Xeris Pharmaceuticals ready-to-use glucagon Hyperinsulinemic Hypoglycemia Orphan Drug designation granted by the FDA
60 Degrees Pharmaceuticals Tafenoquine prevention of malaria Priority Review designation granted by the FDA
Genentech Rituxan (rituximab) pemphigus vulgaris Priority Review designation granted by the FDA
Janssen Erleada (apalutamide) non-metastatic castration-resistant prostate cancer FDA approved
NorthStar Medical Radioisotopes RadioGenix System Non-uranium Sourced Molybdenum-99 Production of Imaging Isotope Technetium-99m FDA approved
Trial Results

Eli Lilly Announces Positive Results for Ankylosing Spondylitis

February 19, 2018

Eli Lilly announced that Taltz (ixekizumab) met the primary and all key secondary endpoints in COAST-V, a Phase II study evaluating the safety and efficacy of Taltz for the treatment of ankylosing spondylitis (AS), also known as radiographic axial spondyloarthritis (axSpA). COAST-V is a multicenter, randomized, double-blind, active and placebo-controlled 16-week study. The trial included a placebo arm and an active control arm (adalimumab) for comparison with placebo, and studied patients who had never received a biologic disease-modifying anti-rheumatic drug (bDMARD). Taltz demonstrated a statistically significant improvement in the signs and symptoms of AS. Patients were required to have an established diagnosis of AS with active disease defined by a Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) Numeric Rating Scale (NRS) score ≥4 and a total back pain NRS score ≥4. During the study, ixekizumab-treated patients received a starting dose of 80mg or 160mg, followed by one of two dosing regimens: either 80mg administered subcutaneously once every two weeks or 80mg administered subcutaneously once every four weeks. The COAST-V study will also evaluate the long-term efficacy and safety of ixekizumab in patients with AS up to one year.

JobWatch

Job openings, upcoming industry conferences and interactive workshops

February 19, 2018

Twice monthly, CWWeekly provides featured listings of clinical research job openings, upcoming industry conferences and educational programs from JobWatch, CenterWatch’s online recruitment website for both clinical research employers and professionals.

  For conferences, webinars, training programs and job postings, join the LinkedIn JobWatch group.
Featured Jobs

Customer Support Representative
Verified Clinical Trials
Garden City, NY

Junior Research Scientist
New York University College of Dentistry
New York, NY

Experienced Research Coordinator
West Broward Research Institute, LLC
Ft. Lauderdale, FL

Medical Director/Principal Investigator
The West Virginia University Research Corporation
Morgantown, WV

Clinical Research Coordinator
Clintex Research Group, Inc.
Miami, FL

Clinical Research Manager
Shockwave Medical
Fremont, CA

Associate Clinical Trials Manager
Vertex
Boston, MA

Clinical Research Coordinator
eStudySite
Las Vegas, NV

Internal Clinical Trials Manager
Ascensia Diabetes Care
Valhalla, NY

Clinical Trials Coordinator
Providence Health & Services
Portland, OR

Clinical Research Coordinator II
Imaging Endpoints
Scottsdale, AZ

Clinical Trials Associate
Seattle Genetics, Inc.
Bothell, WA

Clinical Project Coordinator
General Electric
Waukesha, WI

Ascensia Diabetes Care
Associated Retinal Consultants, P.C.
Royal Oak, MI

Clinical Data Manager
Synteract
Morrisville, NC

Study Manager
CTI Clinical Trial Services, Inc.
Raleigh, NC

Clinical Research Associate I
Providence Health & Services
Santa Monica, CA

Clinical Research Coordinator
Allergy and Asthma Research Center, PA
San Antonio, TX

Nurse (Clinical/Obstetrics-Gynecology)
Department of the Army
Fort Stewart, GA

Clinical Research Coordinator
Radiology and Imaging Specialists
Lakeland, FL

Upcoming Conference

FEBRUARY 13-18, 2018
Scope Summit for Clinical OPS Executives 2018
SCOPE will offer three days of in-depth discussions in 18 different conferences, three plenary keynote sessions and interactive breakout discussions focused on advances and innovative solutions in all aspects of clinical trial planning, management and operations.
Orlando, FL

Webinars

FEBRUARY 28, 2018
Gene Therapy for Rare and Ultra-rare Diseases -Understanding Scientific and Ethical Considerations
Learn the nuances of gene therapy for rare diseases.

MARCH 20, 2018
Paying Clinical Trial Subjects Deconstructing the Most Important Ethical & Practical Considerations
Reasons to pay research participants, in regard to fairness and the role that payment plays in facilitating recruitment. Find out what the regulators think you should be doing — what’s ethical and what’s legal.

Interactive Workshop

MARCH 6-8, 2018
FDAnews ICH E6 GCP Interactive Workshops
Get all the ICH E6 (R2) training you need with three days of hands-on workshops aimed at helping you understand and comply with new requirements.
Raleigh, NC

APRIL 10-11, 2018
Clinical Quality Assurance: Roles and Responsibilities for Auditors and Managers
Register before March 11 and save $200! SIGN UP HERE

Learn what FDA investigators use to evaluate your sites, and how to develop risk-based CQA processes and compliance readiness.
Cambridge, MA