Pitolisant Clinical Trial Delayed in Response to COVID-19 Pandemic


Harmony Biosciences reports that their Phase 2 clinical trial to assess the safety, efficacy and appropriate dosing of pitolisant in treating some of the symptoms experienced by patients with Prader-Willi Syndrome (PWS) has been delayed due to the COVID-19 pandemic.

In a letter to the PWS community, Harmony Executive Vice President & Chief Medical Officer Jeffrey M. Dayno, MD, assures the community that Harmony remains committed to PWS and continues to move forward with the work required to initiate the Phase 2 clinical trial. Dr. Dayno writes in his letter that, “due to the importance of physical distancing and the current strain on healthcare resources during the COVID-19 pandemic, we cannot begin enrollment in the trial until the impact of this pandemic begins to subside, to ensure the trial can be completed under the rigors required by the protocol, including completing inpatient study procedures at the investigative sites. Based on the importance and nature of these procedures for this particular trial, there are no alternatives or contingency plans that we can put in place that would allow us to start the trial as we had hoped. We believe that during this period of uncertainty and strain on healthcare resources, we must all act responsibly and allow healthcare workers to focus on the current crisis.”

The clinical trial, when it starts, will enroll 60 patients aged 6 to 65 years. It will include an 11-week double-blind treatment phase (including a 3-week titration period and an 8-week stable dose period), and an optional open label extension phase. Patients will take pitolisant or placebo as an oral tablet once daily in the morning upon wakening. During the open label extension phase all eligible patients will receive treatment with open-label pitolisant and will undergo titration during a 3-week titration period to achieve the maximum target dose based upon their age. The open label extension phase will be multi-year in duration.

The double-blind treatment phase will include two arms. In the low dose pitolisant arm, patients will be randomized to either placebo or to pitolisant with pitolisant dosing tailored to the age of the patient. Patients aged six to less than 12 years will receive 4.45 mg pitolisant in week one, 8.9 mg pitolisant in week 2 through 11. Adolescent patients aged 12 to less than 18 years will receive 4.45 mg pitolisant week 1, 8.9 mg pitolisant week 2, and 13.35 mg pitolisant weeks three through 11. Adult patients will receive 4.45 mg pitolisant week 1, 8.9 mg pitolisant week 2, and 17.8 mg pitolisant weeks 3 through 11.

In the high dose pitolisant arm, patients will be randomized to either placebo or to higher doses of pitolisant with pitolisant dosing tailored to the age of the patient. Patients aged six to less than 12 years will receive 4.45 mg pitolisant in week one, 8.9 mg pitolisant in week two, and 17.8 mg pitolisant in weeks three through 11. Adolescent patients aged 12 to less than 18 years will receive 8.9 mg pitolisant week 1, 17.8 mg pitolisant week 2, and 26.7 mg pitolisant weeks three through 11. Adult patients will receive 8.9 mg pitolisant week 1, 17.8 mg pitolisant week 2, and 35.6 mg pitolisant weeks 3 through 11.

Further details about the clinical trial can be found at: https://clinicaltrials.gov/ct2/show/NCT04257929


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