Treatment in a U.S.-based Phase 1/2 clinical trial evaluating uniQure‘s gene therapy AMT-130 in people with Huntington’s disease has been temporarily postponed in response to the COVID-19 outbreak, the company announced.
The decision to postpone procedures in the two enrolled patients follows pandemic-related state-of-emergency declarations in the U.S., but the trial is “not on hold” and “remains open,” uniQure said in an email to Huntington’s Disease News.
“Due to the COVID-19 pandemic, sites may have halted screening for the time being due to their institutional policies surrounding the pandemic,” the company stated, but patients can contact enrolling clinical sites directly or send general inquires to [email protected] if they are interested in participating.
“We are following all federal and local regulations including the FDA guidance on clinical trial conduct during the COVID-19 pandemic and we are working very closely with trial investigators to ensure that patient safety remains our top priority,” Robert Gut, MD, PhD, chief medical officer at uniQure, said in a press release.
The therapy consists of an adeno-associated virus, a form of virus that has been modified to be harmless and designed to carry a small portion of genetic material — called microRNA (miRNA) — and deliver it to nerve cells.
When the miRNA enters cells, it binds to the RNA molecule carrying instructions for producing the huntingtin protein and destroys it, thereby lowering the amount of defective huntingtin protein.
Preclinical studies have shown that a single administration of AMT-130 significantly lowers the amount of this protein in the brains and cerebrospinal fluid (the fluid surrounding the brain and spinal cord) of pig models of Huntington’s disease. In mice, levels of exon 1 HTT protein were also lowered in certain regions of the brain after AMT-130 administration.
The ongoing Phase 1/2 trial (NCT04120493) is assessing the safety and effectiveness of AMT-130 in Huntington’s disease patients with early symptomatic disease. The study is expected to enroll 26 participants across multiple sites in the U.S. and to assign them randomly to one of two groups.
Patients will receive either a low or high dose of AMT-130 or a sham procedure, administered as a single dose into a brain region called the striatum (involved in motor control, and a major site of lesions in Huntington’s).
The primary aim is to assess the therapy’s safety and tolerability. Secondary measures of efficacy include the levels of AMT-130 and mutant HTT in the brain, disease severity, and changes in motor and cognitive function.
“Despite this unexpected postponement, we are encouraged by the progress that we have made with this trial since the beginning of the year, and the fact that we have patients who are highly motivated to participate,” Gut said. “We will continue our work to resume treatment in the Phase 1/2 trial as soon as it is clinically appropriate.”
AMT-130 has been granted orphan drug status and fast track designation by the U.S. Food and Drug Administration, as well as orphan medicinal product designation by the European Medicines Agency. The designations are intended to accelerate AMT-130’s development and approval for Huntington’s disease treatment.
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