Rett Syndrome is Such a Trial… #2 Ketamine

purple 2Many parents can attest to the fact that after a procedure requiring anesthesia our children have a decrease in Rett related symptoms. For Katelin, this was a drastic decrease in behavioral issues. My other children would often say… Can we get some of that stuff to bring home?? This anecdotal evidence is so pronounced and consistent across different experiences/procedures etc. that it would be beyond the scope of “coincidence”, imo. Apparently, I’m not the only one who thinks so! So, we know what happens after just one dose, but what happens to symptoms after repeat dosing??

Rett syndrome Research Trust is conducting a trial with ketamine to find out and is the sole funding source. Dr. Jana Von Hehn gives a nice breakdown of the reasons this is a trial worth trying in this article on RSRT’s website.  This trial has some really good things going for it, which is great news for the Rett community.

Safety– Ketamine has been used for decades; it has a good safety profile and after decades of use, parents can be reassured that it’s been tried on all ages, males and females and any variety of patients with pre-existing conditions.

Scope and Convenience– Only 48 participants are needed for this trial. Currently there are five sites recruiting, with another one in the works. This means a large area of the United States is covered and within reach of many families. Also, RSRT and the Rettland Foundation are working together to assist families with the financial impact of participation.

Crossover administration– Even though this is a double-blind trial, it is one in which each participant will be given the placebo during one part of the trial and Ketamine during the other, to compare the findings between the two phases. Both the dose and order in which it is given is randomized and neither the study coordinator nor providers/parents know which is being given at any particular time.

Oral doses- A previous study for the use of Ketamine was discontinued due to a funding withdrawl. That study was IV administration. This trial is being done with oral dosing and may be given through G-tube as well.

Ketamine is already FDA approved– This is a HUGE bonus. If the trial provides positive results, the path to approval for Rett syndrome would be much easier than starting from scratch.

As you can see, this trial has a lot of good points going for it.

For more  inclusion criteria/exclusionary criteria check

A couple things of note are the ages: 6-12 and the girls cannot have had their first menses. Which I found interesting. Reaching out to Dr. Hehn, she responded with the following data:

“Currently FDA’s position is that the neurotoxicity observed in adolescent rats but not in younger animals is sufficient for them to feel cautious about exposing our patients to a semi-chronic dose of a 5-day administration, rather than a 1-time administration as ketamine is intended. The references that FDA cite for their concern are Olney et al, “Pathological changes induced in cerebrocortical neurons by phencyclidine and related drugs”, Science 244, 1360-1362 (1989) and Jevtovic-Todorovic et al, “A comparative evaluation of the neurotoxic properties of ketamine and nitrous oxide”, Brain Res. 895, 264-267 (2001).”

Does this mean if it’s found effective in the age range for the trial that it won’t be made available to older children/adults at some point?- No. The FDA makes it decisions based on verifiable data, which can change over time. So, even if at first it isn’t approved for older children/adults that doesn’t mean the FDA won’t/can’t later approve it when more data about the safety of chronic use in adults becomes available.

One thing I really like is that, in addition to the various questionnaires, participants will be wearing biosensors:

“(from 2 different non-invasive, wearable devices will be used in the study to determine changes in physiologic measures for the patient in the home environment. Biosensors will be worn continuously during the screening and treatment period to measure activity, sleep, position, heart rate, and breathing.”

These devices are being used more and more often in studies and I like that physiological data is being recorded and part of studies because it provides concrete non-subjective data. It’s one thing for a parent to say, optimistically…I THINK her breathing is better; it’s a completely different thing for a sensor to SHOW that her breathing is better or even that her breathing ISN’T better. This objective data is irrefutable and a better measure of certain things.

The main thing we, as a community, need to do is GET THIS TRIAL COMPLETED! Last time I checked, there were already 10 children enrolled and another 8 slated, that leaves only 30 left. With an anticipated completion date of by the end of 2019, this trial looks on track, but it does still need 30 children. Take a look at the trial sites and see if any are within reach for you (for those not recruiting yet, you can still call and say you’re interested for when they start up)and remember the Rettland Foundation is there to assist  you in getting there!

United States, Alabama
University of Alabama Birmingham School of Medicine
Not yet recruiting
Birmingham, Alabama, United States, 35294
Contact: Judy Combs 205-996-4935
Principal Investigator: Alan Percy, MD

United States, Colorado
Children’s Hospital Colorado
Aurora, Colorado, United States, 80045
Contact: Trusha Rajgor, PhD 720-777-8499
Principal Investigator: Timothy Benke, MD, PhD

United States, Illinois
Rush University Medical Center
Chicago, Illinois, United States, 60612
Contact: Thao Tran 312-942-2815
Principal Investigator: Elizabeth Berry-Kravis, MD, PhD

United States, Massachusetts
Boston Children’s Hospital
Boston, Massachusetts, United States, 02115
Contact: Grace Bazin 617-355-1495
Principal Investigator: David Lieberman, MD, PhD

United States, Pennsylvania
Children’s Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Contact: Casey Gorman 267-426-5171
Principal Investigator: Eric Marsh, MD, PhD

United States, Tennessee
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37203
Contact: Diana Coman;
Principal Investigator: Jeffrey Neul, MD, PhD





This entry was posted in Breathing issues in Rett syndrome, FDA, ketamine, Rett Research, Rett Syndrome, Rettland Foundation,, RSRT, Trail to a Texas Trial, Uncategorized and tagged , , , , , , , , , . Bookmark the permalink.

2 Responses to Rett Syndrome is Such a Trial… #2 Ketamine

  1. Melissa says:

    At our second visit today 🤞🏼🤞🏼

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