Natural History Study of Huntington’s Fully Enrolled, Triplet Says
Triplet Therapeutics announced it has completed patient enrollment in SHIELD HD, a natural history study documenting the course of Huntington’s in the absence of treatment and associated disease biomarkers.
With more than 60 people taking part, SHIELD HD (NCT04406636) has attained and exceeded its target enrollment goal ahead of time. The study, which started enrolling participants this year at nine sites across North America and Europe, is now expanding at European sites due to demand from the Huntington’s community.
“We are pleased to have completed enrollment of this natural history study faster than expected,” Irina Antonijevic, MD, PhD, chief medical officer of Triplet, said in a press release.
“We are especially encouraged by the HD [Huntington’s disease] community’s enthusiasm for the study and incredibly grateful to the participants and investigators who are helping to accelerate research that can inform and advance into disease-modifying therapies,” Antonijevic added.
“Based on what we have seen with SHIELD HD, we believe patients and families share the excitement and confidence in our approach, and we are so pleased to partner with them to drive the study forward and learn more about the early clinical and biological signs of the disease,” said Nessan Bermingham, PhD, CEO, president and founder of Triplet.
Sponsored and led by Triplet, SHIELD HD is a prospective, longitudinal study that aims to follow adults, ages 18–63, with early manifest (early stage) and premanifest (asymptomatic) Huntington’s for up to two years.
During the study, investigators will assess the evolution of a series of clinical outcomes and disease biomarkers, including changes in brain MRI scans, and in cognitive, motor, and functional measures, such as those assessed by the composite unified Huntington’s disease rating scale (cUHDRS).
Alterations in neurofilament light chain (NfL) levels in the blood and spinal fluid, and in the activity of genes involved in the DNA damage response (DDR) pathway, will also be analyzed.
NfL, a protein, has been proposed as a biomarker of nerve cell damage in people with Huntington’s and other neurodegenerative disorders, while DDR genes are thought to influence both the age of Huntington’s onset and its severity. These genes are also the targets of several therapy candidates Triplet is developing to treat disorders caused by the expansion of DNA repeats, including Huntington’s.
Findings from SHIELD HD are expected to complement data from upcoming clinical trials, and to facilitate their interpretation in the context of Huntington’s.
Natural history studies collect information about the course of a disease, from its onset onward, in the absence of an intervention. They are particularly necessary for rare diseases, and essential to therapy development.