NextCell pharma is running a clinical trial of their ProTrans product. This is a stem cell product derived from "Wharton´s Jelly", which is a specific part of the umbilical cord. ProTrans is made from umbilical cords from third parties, not the person receiving the transplant. (So this is not self transplants, where the person gets stem cells which were originally harvested from themselves.)
The difference between ProTrans (the product being tested), and generic stem cells from Wharton's Jelly in the umbilical cord, is some proprietary testing. NextCells thinks they have a way of identifying which batches of stem cells will be more effective, so they test each batch of stem cells, and the ones that pass are used to make ProTrans.
The Clinical Trial
The clinical trial being run right now, can be thought of as two mini-trials, being run one after the other. They were both approved at the same time, which is why there is only one clinical trial registry. However, when you read it, it is very clear that there is a 9 person trial, followed by a 15 person trial. The first part is focused on finding the best dose, and the second part is focused on finding how effective that dose is. The researchers have published their entire study protocol, which you can read here:
Note that this published protocol description is not the same as the FDA clinical trial registry description. I'm assuming that the published protocol is correct, as it was published months after the FDA registry was last updated.
Both parts of this study are limited to adults (18-40 years old) who are within 2 years of diagnosis.
The first part will be men only, the second part will be men and women. The treatment is one intravenous infusion. No immune supressives will be given. The primary end points are safety and C-peptide generation (this measures the body's ability to generate insulin) after a year. Secondary end points include insulin independence, insulin use, A1c numbers, and more C-peptide results.
This part of the study is not blinded, and will include three groups of three people each, all of whom will get the treatment (no control group). Each group will get a different dose of stem cells. The first group will get 25 million cells, the next 100 million cells, and the third will get 200 million.
Results: No serious adverse effects as of June-2018. Initial (one-month) results expected Oct-2018.
This part of the study is double blind. Ten people will get the treatment and five will get a placebo as a control group. Everyone in this group will get the same dose, and that dose will depend on which one gives the best results in the first part of the trial.
Results: Full results expected 2020.
NextCell's web site: http://www.nextcellpharma.com/
Clinical Trial Record: https://clinicaltrials.gov/ct2/show/NCT03406585
Paper on Wharton's Jelly in general:
publicjoshualevy at gmail dot com
All the views expressed here are those of Joshua Levy, and nothing here is official JDRF, JDCA, or Bigfoot Biomedical news, views, policies or opinions. In my day job, I work in software for Bigfoot Biomedical. My daughter has type-1 diabetes and participates in clinical trials, which might be discussed here. My blog contains a more complete non-conflict of interest statement. Thanks to everyone who helps with the blog.