Halo Therapeutics is now enrolling patients in our initial clinical trial of HT-100 for Duchenne muscular dystrophy. For more information about participating in the HT-100 clinical program, please contact us at trialinfo@halotherapeutics.com. For a description of our first clinical trial, please visit clinicaltrials.gov.

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The lead researcher at Davis Medical Center ( a participating facility, but not yet recruiting ) will be running 1/2 marathon with me and my son this weekend in Davis CA. I'll make sure to see what he can tell me about HT-100. Sure is exciting, but seems to be very little information about it compared to other treatments.

-David

I cannot sleep waiting for this trial to start in my area! Just wanted to share an email response I received from Halo, when I asked about my sons participation:

 

Is he ambulant? on or off steroids? (both are accepted, we just need to know)

If on steroids, he needs to be on them for at least 1 year, and 6 months of dose without ANY change in the dose.
If he takes any other medications, no changes should be made for 3 months before enrollment date, and no changes on any medication will be allowed during the study.
 
The study is very intense in term of visit load for the first 6 weeks, so it is important that you are either close to one of the clinical trial centers or able to travel and be flexible for the start date, and then for fixed dates on follow up visits.
If all these sound good, you should be calling the site directly, and have with you the following:
 
genetic test results.
muscle biopsy if available
last echocardiogram results
last EKG results
last forced vital capacity.

I like the response. Looks like the research team wants to hit the ground running and not redo the whole data collection again.

Hope to see the same eagerness when sharing results. They should not try to make it a secret affair and make data sharing appear like a huge media/publicity event.

halo has somewhat unique management. it seems they were founded almost exclusively to run this trial for this drug, which they purchased from another company in Israel that could not solve the nausea problem. they are DMD parents themselves, many of them.
Hey soon my son may be enrolled at UC DAVIS in California US, which is a six month tolerability study with secondary measures for functional improvement.

I don't know much more than that but I will soon.

Finally a study that does not exclude non ambulatory subjects!!
Maybe I'm too close to this but it sure does seem like HALO is actually doing what all the other companies state but rarely do -- move quickly! Look at the ground they've covered since changing management. ... astonishing

Good Luck! I will be curious to hear how the trial is going.  My son was told he was a perfect candidate for this trial, but then in July he was denied because his deletion is 8--11 and Diana Escolar told me that she could not be sure he is actually a duchenne because of where his deletion is.  I went balastic, as we know that he is an out of frame deletion and IS a duchenne based on the genetic testing.  She would only do it if a biopsy was obtained, and our Dr. (Kathryn Wagner), as well as Pat Furlong herself disagreed that this was necessary.  Dr. Escolar's arbitrary exclusions of patients is making it hard to fill the trial.  If she would change her mind I would bring him to any sight in the country that needs patients.  Our site was filled after she excluded him in July.

The side note to this is that parents whose children have an early gene deletion should really consider whether they need a biopsy to make sure they do not have a problem with future trials.  Pat Furlong and my Dr both told me I shouldn't have a problem, and that my son will be a candidate for future trials, but it still scares me that I could have this happen again.

I'm sorry you had to endure that. I'm not sure what is involved with the trial yet... I'm probably jinxing myself for posting this. Even if we can't join I'm very very happy to see the progress in real tangible steps. My son visits mda clinic at uc Davis so we feel like we are literally close to the action.

Good luck David. In which cohort is your son. My son is in 2nd Cohort. He will be restarting doing for 1 month this Tuesday.

Can someone who is in the trial let us know if they see any benefits. This drug seems to hold a large promise.

The HT-100 is a 2 part trial.  The first part last only 2 months. It is given for one day, 1 week and then 4 weeks with a wash out period after each dose regimen.  If you complete the 2 month study you can then sign on for the 6 month extension study where you receive the medicine everyday.  This extension study is just now getting started so it would be very difficult for anyone to say if they are seeing any benefits yet as no one has probably taken this for longer than 4 weeks.

Aleksandar said:

Can someone who is in the trial let us know if they see any benefits. This drug seems to hold a large promise.

The 2 month initial trial was designed to ensure tolerability at most basic level ... did the subject vomit?

This treament failed years ago because no one could keep it down.

The very fact of the extension study moving forward tells me the basic level of tolerability have been met. It will be some time before we can infer more than that.

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