tirasemtiv

[John1]

... finished year long in january, went to clinic in march and requested another trial. they said sorry you are 15 days over three years. its cut in stone, no longer than three years diagnosed and in some cases onset. bummer as i still walk, talk, eat, and breath on my own, that should be the criteria after 3 years.

I believe the reason that trials don't want PALS with a long diagnosis is that they want fast progressors in order to measure the efficacy of the treatment. Slow progressors are more difficult to evaluate.

 

[pearshoot]

our clinics reason. less than three years have greater chance of completing trial than someone over 3 years

 

[mbmurray]

Just completed my last visit for this trial (been off the drug/placebo now for a month). Some tidbits...

• Study coordinator told me Cytokinetics is closing down enrollment at end of month
• Study coordinator also told me they are expanding enrollment up to 500
• Results should be available by EOY
• As for my vitals: MVV (~120 l.m) and SVC (~88%) still same as pre-drug trial levels. Left hand strength same at ~82 lbs, right hand strength down to ~35lbs was at ~54lbs at start of trial

 

[pearshoot]

don't know how many of you read about the one year alzheimer study done by un o Miami, fl using a particular supplement which produced amazing results. I am trying to duplicate the study and will report in three months. five weeks into it

 

[dbltree]

I almost missed this PS...good find and I'm going to give aloe polymannose multinutrient complex (APMC) a trial run myself, thanks for posting!

 

[pearshoot]

dbtree recommend you wait until I finish three months, aug 11 new eden is $125 for one month. here's what my herbalist said after I started, if you are already doing supplements just start drinking aloe vera juice, same thing. un of Miami did not tell whole story, they had a lot of other things going on at same time, education, singing, dancing, memory games, ect. wellness quest, makers of new eden, only took credit for making patients healthier, of course this was learned after I started. know you are doing dp, you don't need another supplement. aloe vera has been a medicine for over 2000 years. it is suppose to improve stiffness, I use it to mix in supplement

 

[dbltree]

Rats! I already ordered it but I'll just add aloe vera juice to my regime....sounds like they feel that is the key element anyway...thanks!

 

[debra6004]

I originally signed up for ALS registry and was recently contacted by the ALS registry, and was asked to donate blood, urine, hair, and fingernail samples for biorepository. They sent out a nurse, fed-ex'd the kit, and process took about an hour. No reimbursement, but will provide samples for research, etc. They are scheduling another collection in 6 months. I have the contact info, but assumed that if you were signed up with the registry, they would contact you.

 

[mbmurray]

BIG screw-up with Cytokinitecs for this trial... Jeez...

Cytokinetics shares slide on dosing snafu in ALS drug study - FierceBiotech

 

[mbmurray]

Post went to mod... Here is article about cytokinetics sanfu....

Shares of Cytokinetics ($CYTK) plunged more than 10% this morning after the biotech awkwardly explained a dosing snafu in its sizable midstage study for an experimental treatment for ALS, or Lou Gehrig's disease. Investigators say that a "program error" in the system used to designate patients for the drug or a placebo caused 58 people in the drug arm to get a sugar pill rather than tirasemtiv. And now they are following up with regulators to see if they may need to expand on patient enrollment as the biotech ponders tinkering with the study's timeline.

"Cytokinetics is in communication with regulatory authorities regarding how best to respond to the error in drug assignment in order to preserve the intended scientific value of BENEFIT-ALS," the company said in a release. "The company continues to enroll patients in the study under the current protocol and may amend the protocol to allow increased enrollment. Following further communications with regulatory authorities, Cytokinetics expects to provide updated guidance relating to the conduct of BENEFIT-ALS, which may include revisions to the timing of publicly available results from the study as well as to the projected costs of the study."

The dosing screw-up was flagged by Cytokinetics' data management vendor, but the developer didn't finger any of the contractors that were involved by name. It's common for most biotechs engaged in clinical research to line up outside specialists for trial work like this.
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As of now, Cytokinetics says it has enrolled about 450 of the 500 patients they had sought when starting the Phase IIb study. The news jarred investors after a string of positive events, including back-to-back licensing deals with Amgen ($AMGN) and Astellas. A few days ago Astellas paid $40 million in upfront cash and near-term R&D support to team up with Cytokinetics on new research on treatments for muscle weakness while partnering on the early-stage program for CK-2127107. And the deal is back-ended with up to $450 million in milestones for successful development work.

Cytokinetics has been working on small molecules that can boost body muscle where it counts. It's retained all rights for tirasemtiv, though, hoping that it is in line to complete work on a drug designed to treat a disease that typically kills its victims three to 5 years after diagnosis.

 

[dbltree]

Good grief!

 

[Nighthawk]

Ouch!

First, it was Dexpramipexole trial and...now this?!

I'm starting to believe that, right now Propofol would be our best bet (for now).



Carlos

 

[Sanky]

Does anyone know what really happened with the Cytokinetics trial or who the contractors are that they were using? I wonder what will happen now!

 

[dbltree]

This was posted on Inspire:

Boston Business Journal, 7/9/13

Non-Profit Biotech Chief: Mix-up Won't Derail ALS Drug

The head of a local non-profit biotech dedicated to advancing drugs to cure amyotrophic lateral sclerosis (ALS, or Lou Gehrig's disease) says a recent mix-up in a trial by Cytokinetics Inc. won’t derail the drug’s development, but may make it last a little longer.

Cytokinetics (Nasdaq: CYTK) announced Monday it mistakenly gave 58 patients in a 500-patient trial a placebo. They were supposed to be given its drug candidate, tirasemtiv. The company blamed the mistake on a computer error, according to an article on Bloomberg News.

Steve Perrin, CEO and Chief Scientific Officer of the Cambridge, Mass.-usits based non-profit ALS-TDI, said Cytokinetics deserves credit for announcing the error right off the bat, and says it’s lucky that the error didn’t put any patients at risk. He said the company now must decide whether to enroll another 58 patients, or go forward with a slightly smaller trial size.

“It certainly is unfortunate to lose 10 percent of the patients,” he said, but replacing those patients will “extend the timeline of the study.” It will also cost Cytokinetics more money.

The company, which initially lost stock value after the announcement but has since risen almost 5 percent from it’s opening stock price on Monday, said it’s talking to the U.S. Food and Drug Administration about what to do next in the trial.

Perrin says ALS-TDI, meanwhile, is ready to start enrolling patients for a Phase 2a trial of a drug it's developing in conjunction with Novartis called Gilyena, which is already approved for treatment of patients with multiple sclerosis. The organization also has several pre-clinical candidates in development, and hopes to advance one to the human trials before the end of the year.

 

[mbmurray]

UGH.... Raising enrollment to 680 from 500 due to error, another delay for when results will be released....


Cytokinetics Inc. will boost the size of a mid-stage Lou Gehrig's disease drug trial after a contractor mistakenly switched some patients from the drug to a placebo.

The South San Francisco company (NASDAQ: CYTK), led by CEO Robert Blum, said it now will enroll about 680 patients with amyotrophic lateral sclerosis, commonly known as Lou Gehrig's disease, in its "BENEFIT-ALS" Phase IIb trial of the drug tirasemtiv.

Some 450 of roughly 500 people expected enrollees had joined the trial when the error was identified.

Ramping up the size of the study, which is expected to be completed in the second of this year and report out results in early 2014, will increase the trial's cost by about $5 million.

Tirasemtiv isn't designed to halt ALS but to activate a muscle protein, called troponin, to make a key muscle complex more sensitive to calcium, which increases skeletal muscle force and reduces muscle fatigue. The drug could help ALS patients, whose muscles typically give out over three to five years until they succumb from the disease, to breathe, walk or simply hold a cup.

The trial could have been delayed after a programming glitch by an unidentified contract research organization, which handled electronic data capture systems, caused 58 patients who had received tirasemtiv to be switched to a placebo.

No patients were switched from placebo to tirasemtiv because of the error, Cytokinetics said. What's more, the company and all clinical trial site personnel remain blinded, meaning they don't know which patients are receiving the drug or the placebo.