August 2018

Features

Experts Ask FDA to Rethink Trial Exclusion Criteria

August 27, 2018

Ethical concerns about testing on vulnerable groups may be unnecessary and may be depriving trials of key critical data, a panel of experts says, urging the FDA to reconsider rigid rules excluding certain groups from clinical trials—or at least make decisions on a case-by-case basis.

Features

FDA: New Flexibility for Blinding Cancer Trials

August 27, 2018

Cancer patients in clinical trials should be told whether they’ve been given placebos or experimental drugs if their tumors reappear or get worse—and patients and researchers should be told if sponsors are worried experimental drugs may be triggering bad reactions, the FDA says in new draft guidance.

Using placebos in double-blind, randomized trials is a traditional way to protect against bias, but it also raises “both practical and ethical concerns,” the agency says. For one thing, giving a mere placebo to someone who is getting sicker could be perceived as tantamount to denying them care. For another, many experimental drugs may have toxic side effects and it can be dangerous to keep patients and/or researchers in the dark about whether a med or disease is to blame for symptoms.

“For example, in a blinded immunotherapy trial, a patient who develops adverse events … may receive unnecessary treatments,” the FDA says. “Maintaining the blind [status] after disease progression could also affect a patient’s subsequent therapy, potentially preventing a patient who had been on a placebo from receiving an approved therapy, or delaying or preventing the patient’s entry into other clinical trials.”

In an effort to assuage concerns, many recent cancer trials take an open-label approach where patients are given an already-approved treatment instead of placebos to compare against the experimental one. Other trials use an add-on approach—all patients are given standard treatment plus either a placebo or an experimental treatment.

But in some cases there aren’t other treatments available. In those rare instances, the FDA suggests trials “un-blind” patients if their disease recurs or worsens while taking placebos.

The draft guidance—released late last week—says both patients and investigators should also be “un-blinded” if an experimental treatment causes harmful side effects and the patient may need another med or even surgery. But it recommends patients be allowed to stay in trials even if they’re un-blinded.

The FDA notes that if sponsors insist on keeping trials “blind” throughout, at the least, they need to carefully explain this may happen—and the risks involved—in informed consent documents before patients sign up.

Read the draft guidance here.

Briefs

New Scale for Tumor Treatments

August 27, 2018

Europe’s largest oncologist association has approved a new scale to measure tumor DNA mutations, a move that backers hope will simplify and standardize choices for targeted cancer treatments and clinical trials.

Pipeline

New Pipeline Updates from Aravive Biologics, Biohaven Pharma and Sun Pharmaceutical

August 27, 2018

Company Drug/Device Medical Condition Status
Aravive Biologics, Inc. AVB-S6-500 Platinum-resistant ovarian cancer Granted Fast Track Designation by the FDA
Biohaven Pharma BHV-0223 Social anxiety and public speaking anxiety disorders Positive data from a proof-of-concept Phase III clinical trial
Sun Pharmaceutical Industries Ltd. Cequa (cyclosporine ophthalmic solution) 0.09 percent Keratoconjunctivitis sicca (dry eye) Approval granted by FDA
Lion TCR Pte. Ltd. LioCyx™ Relapsed liver cancer post-liver transplantation Approval granted by Health Sciences Authority (HSA), Singapore, for multicenter Phase I/II clinical trial
Taiwan Liposome Company, Ltd. TLC590, a proprietary BioSeizer™ liposomal formulation of ropivacaine Postsurgical pain Dosing of first patients in a Phase I/II trial
Teva Pharmaceuticals Generic version of EpiPen Auto-Injector Severe allergic reactions Approval granted by the FDA
BeiGene Ltd. Tislelizumab, an investigational anti-PD-1antibody, combined with chemotherapy as a potential first-line treatment Stage IIIB or IV squamous non-small cell lung cancer (NSCLC) Phase III clinical trial initiated
Eiger BioPharmaceuticals, Inc. Avexitide (formerly exendin 9-39) Post-bariatric surgical patients who experience post-bariatric hypoglycemia (PBH) Completion of enrollment in Phase II clinical trial
Escalier Biosciences, BV ESR-114 topical gel Mild-to-moderate psoriasis Phase I/IIa clinical trial initiated
Sangamo Therapeutics, Inc. SB-525, a cDNA gene therapy candidate Hemophilia A Positive preliminary data from the Phase I/II clinical trial
ADC Therapeutics ADCT-402 (loncastuximab tesirine) Relapsed or refractory diffuse large B-cell lymphoma (DLBCL) Phase II clinical trial initiated
Alexion Pharmaceuticals, Inc. ALXN1210, investigational long-acting C5 complement inhibitor Paroxysmal nocturnal hemoglobinuria (PNH) FDA accepted Biologics License Application (BLA) for approval
Merck & Co. Keytruda in combination with Alimta (pemetrexed) and platinum chemotherapy as first-line treatment Metastatic non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations Granted approval by the FDA
SPR Therapeutics SPRING endura (single lead) and extensa (dual lead) Peripheral Nerve Stimulation (PNS) Systems Pain Cleared by the FDA
Univision 360 Luminelle DTx Hysteroscopy System Hysteroscopy and cystoscopy Received 510(k) clearance from the FDA
Tolero Pharmaceuticals, Inc. Combination therapy with AbbVie’s venetoclax and Tolero’s investigational agent Relapsed/refractory acute myeloid leukemia (AML) Tolero Pharmaceuticals and AbbVie partner for trial
CWMarketplace

August: Top 10 Industry Provider Profiles

August 27, 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.

CONTRACT RESEARCH ORGANIZATIONS
CROMSOURCE

CROMSOURCE
Waltham, MA
(617) 871-1128
april.mccall@cromsource.com

CROMSOURCE was among the first CROs to become active in Central & Eastern Europe and Russia. Their successful growth over has been built on stability, integrity, high levels of customer satisfaction and repeated business.

Data MATRIX

Data MATRIX
Saint Petersburg, Russia
(8812)4498633
a.shakhova@dm-matrix.com

With offices in Germany and Russia, Data MATRIX’sEDC/IWRS has been used in more than 120 international trials. Data were successfully submitted to the EMA and FDA.

Lotus Clinical Research, LLC

Lotus Clinical Research, LLC
Pasadena, CA
(626) 568-8727
general@lotuscr.com

Since 2001, Lotus has honed its study design and conduct methodology, interacts on behalf of clients with the FDA’s Analgesics Division and understands how to efficiently navigate the agency’s regulatory framework.

Medpace

Medpace
Cincinnati, OH
(513) 579-9911
info@medpace.com

Medpace employs approximately 2,500 people across 35 countries and provides Phase I-IV clinical development services to the biotechnology, pharmaceutical and medical device industries.

Pivotal S.L.

Pivotal S.L.
La Florida, Madrid
0034 679 488 022
bd@pivotal.es

Pivotal was founded in 2001 by Dr. Ibrahim Farr on the principle that strategic medical advice and support should be the backbone of all clinical trials. Pivotal currently employs 200 cross-functional professionals.

Institutional Review Board (IRB)
Biomedical Research Alliance of New York LLC

Biomedical Research Alliance of New York LLC
Lake Success, NY
(516) 470-6900
info@brany.com

BRANY provides IRB administration for more than 1,100 active research trials. It coordinates the study start-up process and manages study startup and research revenue tracking through study close-out.

Investigative Site Networks (non SMO)
Alliance for Multispecialty Research, LLC

Alliance for Multispecialty Research, LLC
Knoxville, TN
(615) 591-0211
amrteam@amrllc.com

AMR is an industry-leading clinical research company with 17 locations across the U.S., enrolling over 145,000 subjects and completing more than 7,700 Phase I-IV clinical trials.

Insearch

Insearch
Tampa, FL
(727) 544-4842
vnapoli@insearchgroup.net

Insearch is a clinical business development company that offers personalized service, at no charge, to Pharma and CROs looking for appropriate PIs/sites for their trials.

Summit Research Network Management, Inc.

Summit Research Network Management, Inc.
Portland, OR
(503) 972-9818
jhockley@summitnetwork.com

Summit Research Network is an independent medical research organization with an out-patient facility.

Technology Solutions
Techorizon

Techorizon
Verona, Italy
+39 045 8222888
silvio.severini@techorizon.com

In February 2011,Techorizon achieved the ISO Certification 9001: 2008, and it considers such accreditation the basis of the company.

Features

FDA: New Guidance for Expansion Cohort Trials

August 20, 2018

Expansion cohorts can help bring life-saving drugs to market quickly, but sponsors should be sure to update informed consent for patients and convene expert safety panels that meet regularly, the FDA says in new draft guidance.

Briefs

First Human Zika Vaccine Trial

August 20, 2018

Researchers are launching the first human trial of an experimental live Zika vaccine. The vaccine, which contains a weakened version of the virus, was de­veloped by scientists at the National Institute of Allergy and Infectious Diseases (NIAID). The trial plans to enroll a total of 28 healthy, non-pregnant adults, ages 18 to 50, at Johns Hopkins School of Public Health Center for Immunization Research and at the Vaccine Testing Center at Larner College of Medicine at the University of Vermont. Trial participants will be randomly assigned to receive a single subcutaneous dose of the experimental vaccine (20 participants) or a placebo (eight participants). Neither they nor investigators will know who is receiving the real thing. Participants will keep track of their tem­perature on diary cards at home – and return to the clinic for periodic checkups for about six months. Researchers will take blood samples during visits to see if they’re developing antibodies in response to the vaccine. Anna Durbin, a professor of International health at Hopkins School of Public Health, is leading the Phase I clinical trial. Most people infected with the Zika virus — primarily transmitted by certain mosquitoes — have mild to no symptoms. But it can cause serious birth defects and developmental problems in babies born to women infected during pregnancy. That’s why the Centers for Disease Control and Prevention advises women who are pregnant or planning to become pregnant not to travel to areas with a known Zika risk. “Zika virus infection remains a significant threat to pregnant women and their developing fetuses, and we can expect to see periodic outbreaks and cases in areas where Aedes aegypti mosquitoes thrive,” said NIAID Director Anthony Fauci. “NIAID remains committed to developing safe and effective Zika vaccines, and we are pleased to begin clinical testing of a live attenuated candidate.”

Pipeline

New Pipeline from Ivantis, Genentech, BeiGene and Endomag

August 20, 2018

Company Drug/Device Medical Condition Status
Amicus Therapeutics Galafold (migalastat) Fabry disease Approval granted by the FDA
Arbutus Biopharma Coporation and Alnylam Pharmaceuticals, Inc. ONPATTRO (patisiran) Hereditary transthyretin amyloidosis Approval granted by the FDA
Ivantis, Inc. Hydrus® Microstent, a microinvasive glaucoma surgery (MIGS) device Mild to moderate primary open-angle glaucoma in conjunction with cataract surgery Approval granted by FDA
Genentech Xlair® (omalizumab) Prevention of severe allergic reactions following accidental exposure to foods in people with allergies Breakthrough Therapy Designation granted by the FDA
Samsung Bioepis, Co., Ltd. SB26 ulinastatin-Fc (UTI-Fc) fusion protein Severe acute pancreatitis Phase I trial set to begin
Sesen Bio, Inc. Vicinium™ Non-muscle invasive bladder cancer (NMIBC) Granted Fast Track Designation by the FDA
BeiGene Ltd. Tislelizumab, an investigational anti-PD-1 antibody, combined with chemotherapy Potential first-line treatment for patients with Stage IIIB or IV squamous non-small cell lung
cancer (NSCLC)
Phase III clinical trial initiated
Eiger BioPharmaceuticals, Inc. Avexitide (formerly exendin 9-39) Post-bariatric surgical patients who experience post-bariatric hypoglycemia (PBH) Completion of enrollment in Phase II clinical trial
Izana Bioscience Namilumab Ankylosing spondylitis Phase II proof-of-concept clinical trial initiated
Rafael Pharmaceuticals, Inc. CPI-613 in combination with bendamustine Relapsed or refractory T-Cell Lymphoma Activation of a second clinical trial site for the Phase I study
Endomag Magtrace, the first non-radioactive, dual-tracer Lymphatic mapping in patients with breast cancer undergoing a mastectomy Granted premarket approval granted by the FDA
Cannabics Pharmaceuticals, Inc. Cannabics SR 5mg, daily for 3 months Advanced Cancer and Cancer Anorexia Cachexia Syndrome (CACS) Conclusion of trial announced; officials results are being evaluated
OrthoTrophix, Inc. TPX-100 Osteoarthritis Phase II trial results: improvements in function and cartilage in 55 percent of TPX-100 treated knees
Entasis Therapeutics ETX2514SUL Complicated urinary tract infections, including acute pyelonephritis (kidney infection) in adults Positive topline results from Phase II trial
Realm Therapeutics PR022 Atopic dermatitis Phase II trial failed to reach endpoint goals
INOVA Diagnostics, Inc. QUANTA Flash® HMGCR Aids in the diagnosis of idiopathic inflammatory myopathy (IIM), a group of conditions that affect the skeletal muscles Received FDA clearance
JobWatch

August 20: Job openings, upcoming industry conferences, webinars and training programs

August 20, 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

Senior Medical Director/PI
Quotient Sciences
Miami, FL

Clinical Research Coordinator
Catalina Research Institute, LLC
Montclair, CA

Clinical Project Manager
GlycoMimetics, Inc.
Rockville, MD

Clinical Trials Analyst
Mount Sinai
New York, NY

Clinical Research Coordinator I
Emory University
Atlanta, GA

Clinical Project Manager
Integrium, LLC
Phoenix, AZ

Clinical Research Coordinator
Allergy Partners of Western North Carolina
Asheville, NC

Infectious Diseases – Tenure leading Position
UNMC College of Pharmacy
Omaha, NE

Upcoming Conference

SEPTEMBER 10-12, 2018
Mastering EU Medical Device Regulation
Philadelphia, PA

SEPTEMBER 27, 2018
Emerging Biopharmaceutical Therapies
Washington, DC

OCTOBER 9-10, 2018
Medical Device Complaint Management
Waltham, MA

OCTOBER 23-25, 2018
FDA Inspections Summit – 13th Annual
Bethesda, MD

Training Programs

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