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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Thank you for explanation guys.

Hi,

I have an 8 years old boy with DMD. I was wondering if  someone tell me about the HT-100 phase 2 ? I know there are still recruiting and I was wondering if someone went to phase 1 clinical trial ? Are there any benefits, I mean if there are visible improvements and how often are the visits to the hospital ?

Thank you,
Monica


HT-100 - Akashi's Phase 1/2 Clinical Program in Duchenne


 

Frequently Asked Questions (FAQs) about this research:

 

What stage is this research?

  • This clinical trial program is actively recruiting participants.

 

What is the goal or purpose of this trial?

  • The main purpose of this study is to test the safety and tolerability of different, increasing doses of an experimental medication called HT-100 in boys and young men with Duchenne muscular dystrophy. The study medication, HT-100, is a medicine that may help promote healthy muscle regeneration, diminish inflammation and the resulting damage to muscle, and decrease the scar tissue that forms in the muscles of children with Duchenne. HT-100 does not appear to be mutation-specific, meaning it is potentially applicable to all boys and young men with Duchenne.
  • The clinical trial is also evaluating exploratory measures of efficacy of HT-100.
  • Akashi Therapeutics (formerly Halo Therapeutics) has an approved IND from the FDA allowing the company to conduct this research and has received Orphan Drug status for HT-100 for Duchenne.

 

Who is funding this research?

  • The drug is being developed for Duchenne by Akashi Therapeutics, Inc. (formerly Halo Therapeutics). This research is funded by the Nash Avery Foundation, Charley's Fund, Parent Project Muscular Dystrophy, and more than 20 other Duchenne patient foundations.

 

Who is eligible to participate in this trial?

  • This trial is open to males with Duchenne, ages 6-20 years old.  Ambulatory or non-ambulatory boys can enroll, and participants may be either corticosteroid-naive or on corticosteroid therapy for at least 12 months (stable dose and regimen).  
  • Recent, substantial change in use of cardiac medications or medications affecting muscle function and/or significantly compromised cardio-respiratory function would exclude you from this trial.

 

What do I have to do if I decide to participate in this trial?

  • Single and multiple ascending doses of HT-100 will be given to participants. Safety and tolerability will be assessed.  Pharmacokinetic sampling, or measurements of the amount of HT-100 in the bloodstream, will also be taken.
  • This initial study of safety and tolerability will be followed immediately by a 6-month, open label extension study.  All boys and young men who complete the initial study will be eligible to participate in the extension study.
  • All participants who complete the open label extension study will be eligible to participate in an open label study that will provide ongoing access to medication until HT-100 is available as a marketed product.

 

Where does this clinical trial take place?

  • There are 5 sites in the US:  UC-Davis in Sacramento, CA; Kennedy Krieger Institute in Baltimore, MD;  Washington University School of Medicine in St. Louis, MO; Nationwide Children’s Hospital in Columbus, OH; and Cincinnati Children’s Hospital in Cincinnati, OH. 

 

How many visits to the study site are necessary?

  • There are 3 HT-100 clinical protocols that allow for continuous dosing over an extended period of time:
    • For the initial study, there are 9 separate visits to the study site, some of which require an overnight stay and some of which occur over multiple days.
    • For the extension study, the participant has the option to remain in the study for up to 12 months with 7 separate single day visits to the study site, 1 every other month, with phone conversations planned between the on-site visits.
    • For the long-term extension study, the participant has the possibility of continuing for a period of years and there are 2 single day visits to the study site each year, 1 every 6 months, for as long as the participant remains in the study. At the time of the participant's last dose in either the extension or long-term extension study, the visit to the study site can occur over multiple days if it is more convenient for the participant and family.

 

Is there any funding to help pay for travel?

  • Yes, there is some funding available to help pay for travel.  In addition, Akashi Therapeutics (the sponsor) is making available a travel coordinator to assist families with travel planning.

 

Will I get paid for participating in this study?

  • No, there is no stipend for participating in this study.

 

Why should I consider participating in this study?

  • While no personal benefit can ever be guaranteed by participation in a clinical trial, there are other benefits, including allowing you to play an active role in the health care of your child, gaining access to medical specialists that are normally not available to your child, and helping others by contributing to the better understanding of Duchenne.

 

Where can I learn more about this research?

Thank you, Moein for this information, this is really encouraging

Monica

Moein said:


HT-100 - Akashi's Phase 1/2 Clinical Program in Duchenne


 

Frequently Asked Questions (FAQs) about this research:

 

What stage is this research?

  • This clinical trial program is actively recruiting participants.

 

What is the goal or purpose of this trial?

  • The main purpose of this study is to test the safety and tolerability of different, increasing doses of an experimental medication called HT-100 in boys and young men with Duchenne muscular dystrophy. The study medication, HT-100, is a medicine that may help promote healthy muscle regeneration, diminish inflammation and the resulting damage to muscle, and decrease the scar tissue that forms in the muscles of children with Duchenne. HT-100 does not appear to be mutation-specific, meaning it is potentially applicable to all boys and young men with Duchenne.
  • The clinical trial is also evaluating exploratory measures of efficacy of HT-100.
  • Akashi Therapeutics (formerly Halo Therapeutics) has an approved IND from the FDA allowing the company to conduct this research and has received Orphan Drug status for HT-100 for Duchenne.

 

Who is funding this research?

  • The drug is being developed for Duchenne by Akashi Therapeutics, Inc. (formerly Halo Therapeutics). This research is funded by the Nash Avery Foundation, Charley's Fund, Parent Project Muscular Dystrophy, and more than 20 other Duchenne patient foundations.

 

Who is eligible to participate in this trial?

  • This trial is open to males with Duchenne, ages 6-20 years old.  Ambulatory or non-ambulatory boys can enroll, and participants may be either corticosteroid-naive or on corticosteroid therapy for at least 12 months (stable dose and regimen).  
  • Recent, substantial change in use of cardiac medications or medications affecting muscle function and/or significantly compromised cardio-respiratory function would exclude you from this trial.

 

What do I have to do if I decide to participate in this trial?

  • Single and multiple ascending doses of HT-100 will be given to participants. Safety and tolerability will be assessed.  Pharmacokinetic sampling, or measurements of the amount of HT-100 in the bloodstream, will also be taken.
  • This initial study of safety and tolerability will be followed immediately by a 6-month, open label extension study.  All boys and young men who complete the initial study will be eligible to participate in the extension study.
  • All participants who complete the open label extension study will be eligible to participate in an open label study that will provide ongoing access to medication until HT-100 is available as a marketed product.

 

Where does this clinical trial take place?

  • There are 5 sites in the US:  UC-Davis in Sacramento, CA; Kennedy Krieger Institute in Baltimore, MD;  Washington University School of Medicine in St. Louis, MO; Nationwide Children’s Hospital in Columbus, OH; and Cincinnati Children’s Hospital in Cincinnati, OH. 

 

How many visits to the study site are necessary?

  • There are 3 HT-100 clinical protocols that allow for continuous dosing over an extended period of time:
    • For the initial study, there are 9 separate visits to the study site, some of which require an overnight stay and some of which occur over multiple days.
    • For the extension study, the participant has the option to remain in the study for up to 12 months with 7 separate single day visits to the study site, 1 every other month, with phone conversations planned between the on-site visits.
    • For the long-term extension study, the participant has the possibility of continuing for a period of years and there are 2 single day visits to the study site each year, 1 every 6 months, for as long as the participant remains in the study. At the time of the participant's last dose in either the extension or long-term extension study, the visit to the study site can occur over multiple days if it is more convenient for the participant and family.

 

Is there any funding to help pay for travel?

  • Yes, there is some funding available to help pay for travel.  In addition, Akashi Therapeutics (the sponsor) is making available a travel coordinator to assist families with travel planning.

 

Will I get paid for participating in this study?

  • No, there is no stipend for participating in this study.

 

Why should I consider participating in this study?

  • While no personal benefit can ever be guaranteed by participation in a clinical trial, there are other benefits, including allowing you to play an active role in the health care of your child, gaining access to medical specialists that are normally not available to your child, and helping others by contributing to the better understanding of Duchenne.

 

Where can I learn more about this research?

Hi, guys,

I just received an email from Akashi Therapeutics telling me that my son will be accepted in this trial.

I really need your advise - because this is a long an unknown road.. How many of you participated to the previous trial ? We don't live in the United States and if we come we have to live there for at least 2 years.

We now live in Romania and the doctors here don't know much about this horrible disease...I just went this week to a doctor and guess what he told me : "You have to be prepared for this disease, I supposed you read on the internet that the live expectation is around 20..you are young and you have another child"..he left me hopeless..:

I have the feeling that everything I do , I do in vain. I'm trying to get help here, but they just rise their shoulders and tell me that there is nothing that I can do...

Here is the above mail I received :

Thanks for the email. The Sponsor does offer a cursory pre-screen, which is a review of the compiled clinical documents that you provided. Based on these materials you submitted, your son is potentially eligible for our study. Final eligibility is not able to be determined until a more thorough review of his records by a clinical trial site physician and a visit to confirm your son's current physical and functional ability. We anticipate that these sites will be screening patients during the first months of 2016. Due to the frequency of visits, it is necessary to be geographically in the United States to ensure compliance with the visit timelines. As we now have your "pre-screen" review, you are on the central list of interested and potentially eligible patients. Please reach back out to us when you are settled in the United States to see how things have progressed and the potential for additional screening. Best, Ellen

I'm trying to get my son onboard too, but I don't have your challenge because there is a trial site in my city. Very tough choice
Consider that most treatments seek mainly to stabilize muscle function at the level it was measured when your son starts, it's much less likely to actually rebuild lost function. This, to me, suggests the earlier your son is enrolled (rather than waiting few years for results before enrolled) the greatest potential impact

Thank you, David. I see that your son has also been accepted and you are really lucky and I really hope this would work for you and your son. Unfortunatelly, I don't live in the US, but they also told me that there would be a trial in the Europe. I really want Darius to participate, but I don't know if that is  possible, considering that this study is for 2 years, right ? I have to abandon my job. Tell me, do you know someone who  participated last year, when they tested tollerability and had improvements ? I'm asking you all that, and hope you don't mind.

Wish you all the best,

I have no personal experience to share. We have not enrolled yet. We had hoped for another trial that has not started. We don't know anybody whose participated.

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