Israel-based Teva Pharmaceutical has started enrolling patients in the Pride-HD Phase II trial designed to assess the impact of pridopidine, an investigational medication, on motor impairment in patients with Huntington's disease (HD).
Around 400 patients at 30 sites across the globe are expected to be enrolled in the randomised, double-blind, placebo-controlled trial.
The Pride-HD trial will evaluate the safety and efficacy of pridopidine 45mg, 67.5mg, 90mg, and 112.5mg twice-daily (bid) versus placebo for symptomatic treatment in patients with HD.
Ulm University Hospital professor of neurology and lead study investigator Bernhard Landwehrmeyer said Huntington's disease represents a significant unmet medical need as there are currently no treatments that improve the motor movements that are crucial for gait, balance and coordination.
"Based on previous observations using the compound, we believe pridopidine holds promise for symptomatic relief with an acceptable safety profile," Landwehrmeyer said.
The trial's primary objective will be to evaluate the efficacy of pridopidine on motor impairment after 26 weeks of treatment using the unified Huntington's disease rating scale (UHDRS) total motor score (TMS).
The trial will also examine the effect of treatment with pridopidine on the physical performance test (PPT), and also the safety and tolerability across the range of pridopidine doses in patients with HD during the 26 weeks of treatment.
Teva said that patients qualifying for the Pride-HD trial must be 21 years of age or older, with an onset of HD after age 18 and must have a diagnosis of HD based on clinical features and the presence of =36 cytosine-adenosine-guanine (CAG) repeats in the HTT gene.
Initiation of patient enrolment in the trial represents the latest milestone in the company's commitment to developing medicines to improve the quality of life for patients suffering from devastating CNS diseases, such as HD.
George-Huntington-Institute study investigator, founding director and CEO Ralf Reilmann said: "Pridopidine has shown promising results in previous advanced-stage clinical trials and merits additional study, as it has the potential to have a significant effect on total motor score, the endpoint most commonly used in the assessment of treatment efficacy in HD."
The company plans to design and complete new clinical studies of pridopidine to evaluate its potential for symptomatic relief of HD.
Earlier clinical trials of pridopidine carried out in the US, EU and Canada in patients with HD indicate a significant treatment effect on an important secondary endpoint, total motor score (TMS).
Image: A microscope image of a neuron with inclusion (stained orange) caused by HD. Photo: courtesy of Leevanjackson.