FDA Recommends Early, Pre-Approval Trials in Pediatrics for Systemic Dermatitis Drugs

April 16, 2018

The FDA published a new draft guidance outlining how early Sponsors should incorporate pediatric patients, and relevant age groups, for systemic therapies for atopic dermatitis (AD). The agency previously recommended that Sponsors submit pre-approval data on the use of topical products, but did not recommend pediatric studies of systemic drugs until after adult approval. The new draft guidance recommends Sponsors initiate pediatric studies early in development, typically after obtaining initial evidence from early-phase adult studies. In addition, Sponsors should discuss the specifics of their pediatric programs with the FDA as early as possible, as pediatric study plans are required to be submitted within 60 days after an end-of-Phase II meeting. The FDA said it is important to study all relevant age groups, including children younger than two years of age. A sequential approach may not be needed, except for safety concerns, technical issues or the need for information from older subpopulations to inform further study designs. Juvenile animal toxicity studies should also be considered before enrolling human participants. If approved, Sponsors should provide as much information as possible when labeling in regard to pediatric use. The agency said it is also planning a separate guidance to address the technical aspects of drug development for pediatric patients with AD. The draft guidance is available here:

Pipeline new from Stealth Biotherapeutics, Translate Bio, Adynxx, and BiondVax Pharmaceuticals

April 16, 2018

Company Drug/Device Medical Condition Status
Abbott optical coherence tomography (OTC) high-risk, complex coronary artery disease Phase I trials initiated enrolling 3,650 subjects in North America, Europe and Asia
Translate Bio MRT5005 first-in-human clinical trial in patients with cystic fibrosis Phase I/II clearance given by the FDA
Adynxx Inc. brivoligide (AYX1) post-surgical pain Phase II results announced
GTx, Inc. enobosarm post-menopausal stress urinary incontinence (SUI) Phase II trials initiated enrolling 493 subjects at over 60 clinical trial centers across the U.S.
BiondVax Pharmaceuticals Ltd. M-001 universal flu vaccine Phase II trials initiated enrolling 9,630 subjects age 50 years and older across four-to-six countries over a period of two flu seasons
Therapix Ltd. THX-110 adult subjects with Tourette syndrome Phase IIa topline results announced
AbbVie upadacitinib moderate to severe Rheumatoid Arthritis Phase III topline results announced evaluating 37 subjects
Stealth Biotherapeutics elamipretide Barth syndrome Orphan Drug Designation granted
Clovis Oncology Inc. Rubraca (rucaparib) tablets adult patients with recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy FDA approval granted

KEYTRUDA Monotherapy Met Primary Endpoint in Phase III Study

April 16, 2018

Merck announced that the pivotal Phase III KEYNOTE-042 trial evaluating KEYTRUDA, Merck’s anti-PD-1 therapy, as monotherapy for the first-line treatment of locally advanced or metastatic non-small cell lung cancer met its primary endpoint of overall survival. KEYNOTE-042 is an international, randomized, open-label Phase III study investigating KEYTRUDA monotherapy compared to standard-of-care platinum-based chemotherapy in patients with locally advanced or metastatic PD-L1 positive (TPS ≥1 percent) NSCLC. Patients had no EGFR or ALK genomic tumor aberrations and had not previously received systemic therapy for advanced disease. The primary endpoint is OS with TPS of ≥50 percent, ≥20 percent and ≥1 percent, which were assessed sequentially. The secondary endpoints are PFS and objective response rate (ORR). The study enrolled 1,274 patients randomized 1:1 to receive either KEYTRUDA (200 mg fixed dose every three weeks). The safety profile of KEYTRUDA in this trial was consistent with that observed in previously reported monotherapy studies involving patients with advanced NSCLC.

April 16: Job openings, upcoming industry conferences, webinars and training programs

April 16, 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.
More Jobs

Clinical Trials Manager
Tufts Medical Center
Boston, MA

Associate Manager, Clinical Pharmacology Trials
Celgene Corporation
Summit, NJ

Clinical Research Coordinator
Clinical Research Institute, Inc.
Plymouth, MN

Clinical Research Coordinator
SCL Health
Billings, MT

Clinical Research Study Coordinator
Neurology Private Practice
Plymouth, MA

RN Clinical Research Coordinator
Sutter Health
San Mateo, CA

Clinical Laboratory Scientist II
NeoGenomics Laboratories
Nashville, TN

Regulatory Specialist: Clinical Trials Industry
Segal Trials
North Miami, FL

Upcoming Conference

MAY 10, 2018
West Coast Symposium on Expanded Access
An all-day event for the drug, device, diagnostics and clinical trial communities. An all-star cast of presenters will probe Expanded Access from every angle — regulatory, scientific, business, patient safety and liability.
San Francisco, CA

Training Programs

MAY 1-31, 2018
Program Phlebotomy Training— Two Day Training
Various locations


APRIL 23, 2018
Managing Cybersecurity Risks in the Medical Device and Healthcare Sectors
Do you know what the FDA, HHS, DHS and global regulators are planning? The cost of ignorance could include regulatory sanctions and liability judgments and cybersecurity attacks are only growing.

APRIL 27, 2018
Clinical Trial Portals Essential for Small, Mid-Size Biotech Firms: Are You Standing Out in an Increasingly Crowded Space?

  • How to demonstrate you’ve got the technology solutions in place to efficiently meet milestones, deadlines and startup times for your clinical trials
  • How to attract the top sites to participate in your clinical studies
  • And more

Industry Approaching Inflection Point in Consolidation Towards Large Health Systems

April 9, 2018

The clinical research business is approaching an inflection point similar to the shifts seen over the past 25 to 30 years, only now the trends are toward consolidation in larger health systems and their affiliates that can take a more patient-centric approach, according to Ken Getz, director of sponsored research programs at the Tufts University Center for the Study of Drug Development.

FDA Outlines Considerations for Enrolling Pregnant Women in Clinical Trials

April 9, 2018

The FDA published a new draft guidance on the scientific and ethical considerations for including pregnant women in clinical trials, telling sponsors that data are needed to inform safe and effective treatment during pregnancy, and that it is appropriate to enroll pregnant women in certain situations. The agency said it considers it ethically justifiable to include pregnant women in clinical trials when adequate nonclinical studies have been completed, and when the trial holds the prospect of direct benefit to the women and/or the fetus that cannot be obtained by any other means. In the postmarket setting, an established safety database in nonpregnant women should be available, and inclusion should be allowed when efficacy cannot be extrapolated or safety cannot be assessed through other study methods.