Novartis Pauses Study of GenVec's Gene Therapy CFG166 (UPDATE May 2016: Trial Is Again Continuing)

[Samir]

I'm assuming it is due to trouble recruiting participants.

Yes, most likely so. Hopefully the new sites they open will help them recruit more patients.

Do you know if they have opened more sites since? I see only 3 sites on the clinical trial website. I suppose these are the original sites? Kansas City, Baltimore, and New York?

 

[Autumnly]

 

[Margus]

It seems that at some point estimated trial completion date has been moved to 2020 (January 30, 2020). Rather long for phase 1&2 I think. Unfortunately, but it looks like failure.

Does anyone have some more recent information?

 

[GregCA]

@Autumnly - thanks for the slide. It says the drug can be delivered IL to the cochlea. What does IL stand for? I know IT (intra-tympanic) but I'm wondering what IL means.

 

[annV]

@Autumnly - thanks for the slide. It says the drug can be delivered IL to the cochlea. What does IL stand for? I know IT (intra-tympanic) but I'm wondering what IL means.

Intra Labyrinthe...

 

[reggie green]

When they prolong a trial that means it likely has failed.

 

[Tempest]

https://www.tinnitustalk.com/threads/denver-man-gets-gene-therapy-to-restore-hearing.7312/

Well two people already got the treatment many years ago and it did nothing for either so I'd say it won't do anything.

 

[Contrast]

was the medicine updated since then?

 

[Aaron123]

Does anyone have some more recent information?

There is no new information as far as I know.

When they prolong a trial that means it likely has failed.

Not sure why you think that. I would think that it would be in there interest to terminate a failed trial early, which has happened at least a couple of times with tinnitus trials, rather than incur more expense to continue a failed trial - though they would still have to monitor for safety issues. This one has been prolonged first due to a possible safety issue and generally due to very strict inclusion criteria.

All of the outcome measures have a two year window so the trial won't end until 2 years after the last patient has been enrolled. The trials design calls for 45 people. Given the slide above, we know they have enrolled 14 people. Of course we don't know how many have been enrolled, only that at least 14 have enrolled.

was the medicine updated since then?

No. That would invalidate the trial design and, especially given the nature of the treatment in this could, would likely require new pre-clinical work as well as new safety cohorts in the trial given how it is designed.

 

[GregCA]

Intra Labyrinthe

That doesn't bring any hits in google... it doesn't seem to be a widely used terminology.

Is it the procedure that consists of drilling a hole in the cochlea to dispense meds into the bony labyrinth?

@Aaron123

 

[Margus]

Looks like Novartis are having troubles to recruit new participants, yet another extension of trial deadline, now it's
August 13, 2021

 

[Coleoptere]

That is 3 year in the future and they still continue. What should we make of that?

 

[Margus]

I guess not much without any press release or insider info. 3 years is because the study timeframe is 24 months, and apparently they plan to recruit remaining participants during next year. However I struggle understand why it takes so long to recruit 45 participants, trial is running for 4 years already, it was suspended for a while some years ago, but still.

 

[RinaRegina]

They just cut 2,000 jobs to focus on more expensive gene therapy treatments than high-volume drugs, making me think that if successful, they'll want the tinnitus treatment to be super expensive when it comes out. https://www.marketwatch.com/story/n...part-of-revamp-2018-09-25?ns=prod/accounts-mw

 

[Margus]

not much news, but some info in the article is quite encouraging.
https://www.hearinglossjournal.com/cgf166-update-on-a-promising-new-treatment-for-hearing-loss

I think in a way the trial runs for too long to allow it to succeed, I mean the patent most likely is going to expire soon so there is no financial incentive to bring it to the market, however if the approach itself is successful I believe they might soon launch slightly modified version of the same in new trial.

 

[Krolo]

I guess not much without any press release or insider info. 3 years is because the study timeframe is 24 months, and apparently they plan to recruit remaining participants during next year. However I struggle understand why it takes so long to recruit 45 participants, trial is running for 4 years already, it was suspended for a while some years ago, but still.

If you look a the inclusion criterias they are quite hard to fit in I would say. I do wondee what adverse effects they had due to the stop.

 

[Margus]

If you look a the inclusion criterias they are quite hard to fit in I would say. I do wondee what adverse effects they had due to the stop.

I think it was quite vague at the time, perhaps there was nothing bad, as I can recall the comment that it was suspended as per trial design. Maybe after they enrolled 9 participants they wanted to monitor for any adverse effects before enrolling more. As we see it's 45 participants now and from below comment it seems they could enroll even more:
"As a result, the trials have so far been limited to just 45 patients. Dr. Lustig is hopeful that the FDA may soon loosen the criteria to allow for an increase in patients."

Perhaps all the precaution because of too many unknown factors in using gene therapy.

 

[Krolo]

Perhaps all the precaution because of too many unknown factors in using gene therapy.

Could be. Hopefully there might be something in this drug after all. Too bad it is going to take so darn long though.

 

[JohnAdams]

What's this?
http://www.trials.novartis.com/en/clinical-trials/hearing-restoration-clinical-trial/

 

[JohnAdams]

PROOF IT CAN BE DONE!!!!!!!!!

"The results so far are promising. "There have been a couple patients with hearing improvement, so we are definitely encouraged."

https://www.hearinglossjournal.com/cgf166-latest-news/

And @PolishSoldier87 we were on the right track suspecting we needed to increase Atoh1. Love you brother. I still believe in a natural undiscovered method to do this, we just need to go steal to the Vatican's gold and set up a plant lab and run our experiments.

I think fx322 is going to blow this cgf166 out of the water.

 

[Don Tinny]

PROOF IT CAN BE DONE!!!!!!!!!

"The results so far are promising. "There have been a couple patients with hearing improvement, so we are definitely encouraged."

.

A couple? What about the others?

 

[katri]

PROOF IT CAN BE DONE!!!!!!!!!

"The results so far are promising. "There have been a couple patients with hearing improvement, so we are definitely encouraged."

https://www.hearinglossjournal.com/cgf166-latest-news/

And @PolishSoldier87 we were on the right track suspecting we needed to increase Atoh1. Love you brother. I still believe in a natural undiscovered method to do this, we just need to go steal to the Vatican's gold and set up a plant lab and run our experiments.

I think fx322 is going to blow this cgf166 out of the water.

There could've been a blind part and the dosage could've differed. It's out of 11. A couple is. Better than zero.

 

[JohnAdams]

That was also back in 2017 over a year and a half ago. It is happening.

 

[RB2014]

So it looks like you have to have a certain level of hearing left for this to work. Same information as we have seen on FX-322. It is encouraging and it could explain why one of the participants ended up getting a CI after being on this trial. They only tested severe to profound hearing loss just in case this didn't work. I wonder if they tested on people that had moderate hearing loss if the outcomes would have been better.

 

[Contrast]

I don't even think this trial is meant to be a drug candidate they are just doing it to see what happens.

Someone correct me if I'm wrong.

 

[JohnAdams]

I don't even think this trial is meant to be a drug candidate they are just doing it to see what happens.

Someone correct me if I'm wrong.

I had the same thought. Just applying atoh1 causes the supporting cells to grow and depletes the number of remaining supporting cells. This would not be a good drug candidate, this is however amazing news. I feel stupid for not finding this earlier.

 

[Contrast]

gene therapies are shit compared to progenitor cell activation.

 

[Krolo]

Shit. Now I need to become deaf on one ear darnit...

 

[JohnAdams]

So it looks like you have to have a certain level of hearing left for this to work. Same information as we have seen on FX-322. It is encouraging and it could explain why one of the participants ended up getting a CI after being on this trial. They only tested severe to profound hearing loss just in case this didn't work. I wonder if they tested on people that had moderate hearing loss if the outcomes would have been better.

I think all you need is lgr5+ supporting cells for FX-322 to work.

 

[Manny]

gene therapies are shit compared to progenitor cell activation.

Why?