Novartis to Investigate Second Death in Gene Therapy Trials

Despite positive interim trial results for its experimental gene therapy for spinal muscular atrophy in infants, Novartis AG announced last week it will investigate a second trial death that may be related to the same treatment.

The gene therapy, Zolgensma, returned encouraging interim results for 22 babies with Type 1 SMA, the most serious form of spinal muscular atrophy. An initial death in the trial was found to be unrelated to the treatment, but the second fatality — in a separate European trial — ”was deemed possibly related to treatment by the investigator,” the company says.

An autopsy has been performed and results are pending.

New App Debuts to Locate Colorectal Cancer Trials

Fight CRC and Roche’s Flatiron Health have collaborated to design and build a clinical trial search app that addresses late-stage MSS-CRC.

The original version of Clinical Trial Finder was developed by a stage IV colorectal cancer survivor, Dr. Tom Marsilje, as a curated spreadsheet of colorectal cancer clinical trials. Flatiron developed the web-based version in 2017. Since then, nearly 14,000 people have used it to conduct 24,000 unique clinical trial searches.

Reported to be first-of-its-kind, the app enables patients with late-stage microsatellite stable colorectal cancer to research relevant clinical trials via their smartphone. The app is now available for both iOS and Android operating systems.

FDA Allows Analysis of Covariance for Randomized Control Trials

Drug sponsors can use complicated covariance analyses to help determine statistical significance or sharpen estimates of treatment efficacy, but they shouldn’t apply those analyses to anything in a clinical trial that might be affected by the treatment, the FDA says in a new draft guidance.

The six-page draft guidance is the FDA’s effort to round out recommendations in ICH’s E9 Statistical Principle for Clinical Trials guidance. In it, regulators recognize that different patients have different disease prognoses and that drug sponsors would like to be able to incorporate different prognoses into statistical analyses of trial data.

When sponsors are writing out their protocols or statistical analysis plans, they should “prospectively specify the covariates and the mathematical form of the model.”

Read the draft guidance here: https://bit.ly/2vlb6pl.

Blood Better Than Biopsy in Identifying Subjects for Cancer Trials, Researchers Say

A blood test may be more effective than a biopsy in matching advanced cancer patients to clinical trials that may be their last hope, researchers in the UK have found.

Testing a small blood sample for genetic information on a patient’s cancer provides more up-to-date results than tumor biopsies, which can be months or even years old, according to a study from the University of Manchester and the Christie Hospital NHS Foundation Trust.

The study included 100 patients, 11 of whom ultimately were matched and enrolled in a clinical trial based on genetic data gleaned from their blood.

Biogen Ends Plan for Aducanumab in Alzheimer’s Prevention

Virtually out of options for the use of aducanumab given the failure of its main trial program last month, Biogen has now tabled a proposed Phase III trial of the drug for the prevention of Alzheimer’s disease (CenterWatch Weekly, March 25, 2019).

Having analyzed data from the failed Phase III program with Eisai, Biogen has decided against initiating the planned study “at this time.” When Biogen finishes closeout studies it said would end last month, there will be no active clinical trials of aducanumab listed on ClinicalTrials.gov.

NIH Announces New HGT Protocol Review Guidelines

The NIH last week updated its guidelines for research on recombinant or synthetic nucleic acid molecules, easing some protocol review requirements and clarifying others.

While the new version of NIH Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules does not alter the fundamental requirement for Institutional Biosafety Committee (IBC) approval at each trial site, it specifies that approval must be prior to enrollment rather than trial initiation. In addition, IBC oversight may now extend to biosafety issues, and IBCs may choose to set alternative endpoints for oversight based on their biosafety assessments.

The revised guidelines also eliminate the requirement for protocol review by the Recombinant DNA Advisory Committee (RAC). Now renamed Novel and Exceptional Technology and Research Advisory Committee (NExTRAC), the committee will serve the NIH director in an advisory capacity.

Read the NIH announcement here: https://bit.ly/2W7jeWo.

FDA Launches Redesigned Website

The FDA today launched a fully redesigned and updated website that it hopes will be more user-friendly and visually appealing.

Changes to the website, which is still located at fda.gov, include:

• Redesigned webpages that can be viewed on any internet-ready device (more than 50 percent of the website’s 5 million visitors a month access it through mobile devices);
• Updated navigation based on data and audience behavior;
• More streamlined search results; and
• More visuals and interactive content.
• The new website, which has been entirely rebuilt, has been several years in the making.