ChinaSCINet Update

[Fly_Pelican_Fly]

Yes, a major difference in time!

When the 27 patients are randomized to the 3 groups (cell transplantation + walking/rehab, cell transplantation + lithium + walking/rehab, walking/rehab only), the therapy will then be available.

I disagree with that Jim. I would be gobsmacked if the FDA would green light compassionate use whilst the 3 groups are receiving their interventions. They would require safety data from the first US-treated cohort before considering any kind of compassionate use. Are you being overzealous here?

[Jim]

F_P_F, I had the same thought but it is so- "Expanded access, sometimes called "compassionate use," is the use outside of a clinical trial of an investigational medical product (i.e., one that has not been approved by FDA. FDA is committed to increasing awareness of and knowledge about its expanded access programs and the procedures for obtaining access to human investigational drugs (including biologics) and medical devices."

http://www.fda.gov/NewsEvents/Public...se/default.htm

[Fly_Pelican_Fly]

F_P_F, I had the same thought but it is so- "Expanded access, sometimes called "compassionate use," is the use outside of a clinical trial of an investigational medical product (i.e., one that has not been approved by FDA. FDA is committed to increasing awareness of and knowledge about its expanded access programs and the procedures for obtaining access to human investigational drugs (including biologics) and medical devices."

http://www.fda.gov/NewsEvents/Public...se/default.htm

see http://www.fda.gov/NewsEvents/Public...s_Requirements

Safety

• Ensuring patient safety is a priority - FDA must determine that the potential patient benefit justifies the potential risk of the expanded access use of the investigational drug, and that the potential risk is not unreasonable in the context of the disease or condition to be treated. Even with safeguards, there may be significant unknowns about safety and effectiveness.
  
• Patients should understand and be willing to accept the possible risks and unknown effectiveness associated with the investigational medical product. Patients generally will need to sign an informed consent form in order to receive the investigational drug.
  
• The patient’s physician must determine that the probable risk to the patient from the investigational drug is not greater than the probable risk from the disease or condition.

Logic suggests that the FDA would find it impossible to determine the risk without the safety data to hand first. You cannot be informed without the safety data. Unless you are suggesting the Chinese safety data will be used for submission.

[Jim]

We were told this would apply to our US IIb Trial, therefore it would have to be based on the safety data of the ChinaSCINet Phase II Trial.

[Fly_Pelican_Fly]

We were told this would apply to our US IIb Trial, therefore it would have to be based on the safety data of the ChinaSCINet Phase II Trial.

Ok. Will be interesting to see whether the FDA would accept the Chinese safety data. Would that be a US-first?

[taymas]

Ok. Will be interesting to see whether the FDA would accept the Chinese safety data. Would that be a US-first?

Great question - I never thought about this, but I also remember compassionate use being for those with life threatening problems (there have beeen good arguments here on CC that argue that SCI is life threatening) hence why some might qualify for compassionate use, with a relaxed attitude around safety - cos it's life threatening.

But I would also like to know if this would be a first, and if this will hold up either the trial or compassionate use.

Also Jim, didn't you say after 1 year has lapsed for IIb, that compassionate use applies? That would make sense as that might be enough of a safety window.

But, you then also said once the trial participants are chosen, that compassionate use will be available.

Keep up the Good work Dr Wise, Jim and everyone!

[Barrington314mx]

Actually, they didn't think to include sexual function as an outcome measure. When the subjects are examined for the 3 year follow-up they will ask.
BTW, sexual function is also controlled by a central pattern generator, so the chances of return are good.

While an inpatient at the Shepherd Center we were required to take classes that covered SCI sexuality. I would guess that most of the SCI speciality hospitals have the same.

When you say "return", do you mean getting a hard-on without the use of say, Viagra for example? That probably doesn't include sensation but at least it'll be easier to keep the Mrs happy.

[taymas]

When you say "return", do you mean getting a hard-on without the use of say, Viagra for example? That probably doesn't include sensation but at least it'll be easier to keep the Mrs happy.

What goes up, must come down

Lol anyway, cant wait for a response, especially on FDA approval for compassionate use. It's the weekend so maybe we'll get a reply in the weekday.

[niallel]

Sensation, sensation, sensation - surely thats the most important (well to me anyways)

[Jim]

Ok. Will be interesting to see whether the FDA would accept the Chinese safety data. Would that be a US-first?

I would think there are many therapies that have been developed around the world and approved by FDA. Not sure at the Phase II stage though. Dr. Young had a pre-IND meeting with the FDA and they accepted the data so it also applies to expanded compassionate use.

Great question - I never thought about this, but I also remember compassionate use being for those with life threatening problems (there have beeen good arguments here on CC that argue that SCI is life threatening) hence why some might qualify for compassionate use, with a relaxed attitude around safety - cos it's life threatening.

But I would also like to know if this would be a first, and if this will hold up either the trial or compassionate use.

Also Jim, didn't you say after 1 year has lapsed for IIb, that compassionate use applies? That would make sense as that might be enough of a safety window.

But, you then also said once the trial participants are chosen, that compassionate use will be available.

Keep up the Good work Dr Wise, Jim and everyone!

Expanded compassionate use, which is new, is available once the subjects are chosen for the phase II. We are told you will be able to apply online and get an answer in 48 hours.

When you say "return", do you mean getting a hard-on without the use of say, Viagra for example? That probably doesn't include sensation but at least it'll be easier to keep the Mrs happy.

I am totally speculating because we haven't yet interviewed the trial subjects, but because sexual function is programmed in the spinal cord (CPG) just like walking, bowel, and bladder, it would make sense that it may also return. Money is now being raised to bring the subjects back for complete examinations.

[niallel]

I am totally speculating because we haven't yet interviewed the trial subjects, but because sexual function is programmed in the spinal cord (CPG) just like walking, bowel, and bladder, it would make sense that it may also return. Money is now being raised to bring the subjects back for complete examinations.

Does this mean it would be just getting it up, no sensation?

Have there been any sensory gains with this trial? Apologies if it was mentioned before.

[Scaper1]

Sensation, sensation, sensation - surely thats the most important (well to me anyways)

Gawd no. Not without return of function. Can you imagine still needing to cath and feeling everything?

[Mize]

Gawd no. Not without return of function. Can you imagine still needing to cath and feeling everything?

Aack! Do they make a coude in a French size 2?

[niallel]

Gawd no. Not without return of function. Can you imagine still needing to cath and feeling everything?

Hmmm, good point. I would imagine its not such a nice feeling, but wonder how much in comparison to the benefits of getting your rocks off.

Seriously I think these are the real things we need to know, not just for this trial but all of them.
No one really talks about the practicalities.

[Chaz19]

Congrats! Quick question: Did any quads gain arm and hand dexterity?