NorthWest Biotherapeutics Inc (NWBO)

[longfellow95]

Beartrap. Thankyou for reminding me about the Expanded Access Arm.

I looked up the CT listing:-

Detailed Description:

Patients who are being screened under protocol 020221 who are not eligible for enrollment due to a) evidence of disease progression or pseudo-progression post chemo-radiation or b) insufficient (<5 doses) vaccine manufactured, and for whom the DCVax-L treatment was manufactured and released are eligible for this study.

Treatment Schedule:

Open label vaccine injections will be give per the associated 020221 protocol. Injections will be given at days 0, 10, 20, and at months 2, 4, 8, 12, 18, 24 and 30. There are no therapeutic restrictions, but guidelines for drug administration are recommended as per the 020221 protocol.

Data collected includes vaccine administration information, and any vaccine related adverse event. Patient MRIs will be collected centrally for future review. Patients will be followed for disease progression and survival.

https://clinicaltrials.gov/ct2/show/NCT02146066?term=dcvax-l&draw=2&rank=1

So it was a way of providing access to L treatment for up to 99 patients who failed screening for the main trial.
Not only for those who exhibited progression / pseudo-progression during the 3 months screening, but also for those for whom they were unable to produce enough L doses (probably because of insufficient tumor material).

The way I read it, this was really just a compassionate provision.
Although it's called a study, there are no formal endpoints. Though patients are followed for progression and survival.

As you say, we really know nothing else about this EAP study, and the last entry on CT was in 2016.

For sure, it will provide some additional research indications.
Such as how effective L can be on less than 5 doses.
And they can review the scans themselves rather than it being done by central review, as I doubt that a CRO is even used to follow these patients. So they can see how a patient with a scan suggesting progression actually did subsequently.
Potentially useful info and data, but I don't think it could possibly be used as standalone evidence to support approval for rGBM.
But the fact that it is officially registered as a study suggests to me that it may have some possible use as supportive or supplementary evidential data for another study (perhaps one in the making), if that makes sense.

Then again, we don't know how many patients actually received L under this expanded access. They might have got up to 99 and stopped there basically to control cost. Or they may only have only recruited a handful.
So I would applaud them for making this route available for patients who would have signed a consent form for the main trial, and undergone the leukapheresis only to fail screening, rather than just abandoning them.
But I don't really consider it a controlled trial.

And I don't know why they had both the 55 patient info arm and then created this separate expanded access provision.

Just wondering if an email to Dr Bosch might answer a few (non-material) questions?