February 4, 2016 I Dosing and new patient enrollment in all cohorts of the HALO trial, a study evaluating the compound HT-100 in patients with Duchenne muscular dystrophy, a rare disease that results in muscle degeneration and premature death, are being suspended. We are saddened to report that one of the patients in the trial, receiving 60μg/kg/d (the highest dose in the study), is experiencing serious, life-threatening health issues, and the company is working with the FDA to analyze the situation. We do not yet know to what extent the patient’s health issues are related to HT-100 and/or to other factors.
Updated February 4: It is with great sorrow that we share that the brave young man who was experiencing serious, life-threatening health issues has passed away. We offer our deepest condolences to his family and loved ones. Akashi has initiated a comprehensive investigation, beginning with detailed data reviews and in vitro studies, to evaluate the extent to which the patient’s health issues are related to HT-100 and/or to other factors. We will share updates as we reach conclusions in the investigation.
What steps is Akashi taking to analyze the situation?
Updated February 4: Akashi has initiated a comprehensive investigation, which includes a careful review of all patient records, with a particular focus on this young man’s record, and is initiating the first in a series of studies to further analyze what happened. Experts from outside the company are helping Akashi’s scientists and clinicians in these efforts. We continue to work with the FDA, and based on what we find, our research program will determine additional protective measures, if any, that should be included in the HT-100 program.
Have health issues of this kind previously been reported in the HALO trial?
None of the other patients in the clinical program have experienced a health issue of this kind or of this severity. To date, patients in the three lower-dose cohorts have been treated for between 11 and 19 months with HT-100, amounting to more than 20 patient-years of favorable data, with no other serious events related to study drug, while this patient had been receiving HT-100 at higher doses for approximately two weeks. An interim safety and efficacy analysis of HALO released in June of 2015 showed promising results for boys and young men enrolled in the HT-100 trial at lower doses, including data supporting improvement in muscle strength, with no serious adverse events related to the study drug.
Why has Akashi suspended the trial?
The trial has been suspended, based on discussions with the FDA, to allow us time to better understand the circumstances that led to this patient’s experiences. We will continue discussions with the FDA and will provide information once the investigation is complete. Our earlier-stage clinical and preclinical programs in Duchenne muscular dystrophy, with experimental compounds DT-200 and AT-300, are not affected.
When will the trial be restarted?
Our intention is to restart the trial once our investigation into the causes of this patient’s experiences is complete, and Akashi and the FDA are satisfied that any measures that might be necessary can be put in place to address these causes. As we are at the very beginning of the investigation, we cannot provide a projected timeline. As always, our first priority is patient safety.
What should patients enrolled in the trial, or planning to enroll in the trial, know?
If families of patients participating or planning to participate in the HT-100 trial have any questions, they may contact Akashi at trialinfo@akashirx.com or the principal investigator at their clinical site.
SOURCE: Akashi Therapeutics
Post Views: 31
February 4, 2016 I Dosing and new patient enrollment in all cohorts of the HALO trial, a study evaluating the compound HT-100 in patients with Duchenne muscular dystrophy, a rare disease that results in muscle degeneration and premature death, are being suspended. We are saddened to report that one of the patients in the trial, receiving 60μg/kg/d (the highest dose in the study), is experiencing serious, life-threatening health issues, and the company is working with the FDA to analyze the situation. We do not yet know to what extent the patient’s health issues are related to HT-100 and/or to other factors.
Updated February 4: It is with great sorrow that we share that the brave young man who was experiencing serious, life-threatening health issues has passed away. We offer our deepest condolences to his family and loved ones. Akashi has initiated a comprehensive investigation, beginning with detailed data reviews and in vitro studies, to evaluate the extent to which the patient’s health issues are related to HT-100 and/or to other factors. We will share updates as we reach conclusions in the investigation.
What steps is Akashi taking to analyze the situation?
Updated February 4: Akashi has initiated a comprehensive investigation, which includes a careful review of all patient records, with a particular focus on this young man’s record, and is initiating the first in a series of studies to further analyze what happened. Experts from outside the company are helping Akashi’s scientists and clinicians in these efforts. We continue to work with the FDA, and based on what we find, our research program will determine additional protective measures, if any, that should be included in the HT-100 program.
Have health issues of this kind previously been reported in the HALO trial?
None of the other patients in the clinical program have experienced a health issue of this kind or of this severity. To date, patients in the three lower-dose cohorts have been treated for between 11 and 19 months with HT-100, amounting to more than 20 patient-years of favorable data, with no other serious events related to study drug, while this patient had been receiving HT-100 at higher doses for approximately two weeks. An interim safety and efficacy analysis of HALO released in June of 2015 showed promising results for boys and young men enrolled in the HT-100 trial at lower doses, including data supporting improvement in muscle strength, with no serious adverse events related to the study drug.
Why has Akashi suspended the trial?
The trial has been suspended, based on discussions with the FDA, to allow us time to better understand the circumstances that led to this patient’s experiences. We will continue discussions with the FDA and will provide information once the investigation is complete. Our earlier-stage clinical and preclinical programs in Duchenne muscular dystrophy, with experimental compounds DT-200 and AT-300, are not affected.
When will the trial be restarted?
Our intention is to restart the trial once our investigation into the causes of this patient’s experiences is complete, and Akashi and the FDA are satisfied that any measures that might be necessary can be put in place to address these causes. As we are at the very beginning of the investigation, we cannot provide a projected timeline. As always, our first priority is patient safety.
What should patients enrolled in the trial, or planning to enroll in the trial, know?
If families of patients participating or planning to participate in the HT-100 trial have any questions, they may contact Akashi at trialinfo@akashirx.com or the principal investigator at their clinical site.
SOURCE: Akashi Therapeutics
Post Views: 31
