The 201 Trial: a placebo-controlled randomized phase 2 study of safety and tolerance of the c-Abl kinase inhibitor risvodetinib in untreated Parkinson's disease.
The 201 Trial: a placebo-controlled randomized phase 2 study of safety and tolerance of the c-Abl kinase inhibitor risvodetinib in untreated Parkinson's disease.
Where did the research take place?
The study site has not been established. Author addresses may differ from where the research occurred.
Atlanta, US · Author affiliation
ABLi Therapeutics, Atlanta, GA, USA. mhwerner@ablitherapeutics.com.Location evidence
Sarasota, US · Author affiliation
Clintrex Research, Sarasota, FL, USA.Location evidence
Camden, US · Author affiliation
Cooper Medical School at Rowan University/Cooper University Healthcare, Camden, NJ, USA.Location evidence
Boston, US · Author affiliation
Department of Neurology, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA, USA.Location evidence
IL · Author affiliation · country only
Department of Neurology, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA, USA.Location evidence
Scottsdale, US · Author affiliation
CND Life Sciences, Scottsdale, AZ, USA.Location evidence
US · Author affiliation · country only
QUEST Research Institute, Farmington, MI, USA.Location evidence
Tulsa, US · Author affiliation
The Movement Disorder Clinic of Oklahoma, Tulsa, OK, USA.Location evidence
Tampa, US · Author affiliation
University of South Florida Health Byrd Institute, Tampa, FL, USA.Location evidence
Boca Raton, US · Author affiliation
Parkinson's Disease and Movement Disorders Center, Boca Raton, FL, USA.Location evidence
Memphis, US · Author affiliation
Veracity Neuroscience, Memphis, TN, USA.Location evidence
Little Rock, US · Author affiliation
University of Arkansas for Medical Sciences, Little Rock, AR, USA.Location evidence
Miami, US · Author affiliation
University of Miami, Miami, FL, USA.Location evidence
Nashville, US · Author affiliation
Vanderbilt University Medical Center, Nashville, TN, USA.Location evidence
Round Rock, US · Author affiliation
Central Texas Neurology Consultants, Round Rock, TX, USA.Location evidence
Oregon, US · Author affiliation
Oregon Health & Science University, Portland, OR, USA.Location evidence
Los Angeles, US · Author affiliation
University of Southern California, Los Angeles, CA, USA.Location evidence
Reseda, US · Author affiliation
SC3 Research Group, Reseda, CA, USA.Location evidence
Durham, US · Author affiliation
Duke Movement Disorders Clinic, Durham, NC, USA.Location evidence
Golden Valley, US · Author affiliation
Struthers Parkinson's Center, Golden Valley, MN, USA.Location evidence
Naples, US · Author affiliation
Aqualane Clinical Research, Naples, FL, USA.Location evidence
Raleigh, US · Author affiliation
Raleigh Neurology Associates, Raleigh, NC, USA.Location evidence
GB · Author affiliation · country only
Neurology Center of New England, Foxborough, MA, USA.Location evidence
Milwaukee, US · Author affiliation
Medical College of Wisconsin, Milwaukee, WI, USA.Location evidence
Houston, US · Author affiliation
Baylor College of Medicine, Houston, TX, USA.Location evidence
Grand Rapids, US · Author affiliation
Michigan State University, Grand Rapids, MI, USA.Location evidence
New York City, US · Author affiliation
Department of Neurology and Neuroscience, Mount Sinai School of Medicine, New York, NY, USA.Location evidence
Follow the study behind this paper
A Randomized, Double-Blind, Placebo-Controlled Trial of IkT-148009 in Untreated Parkinson's Disease
The registry links this paper to the study.
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Original abstract
The nonreceptor Abelson (Abl) tyrosine kinases have been implicated as key drivers of initiation and progression in Parkinson's disease (PD). Risvodetinib, a potent, brain-penetrant, selective inhibitor of the nonreceptor Abl kinases c-Abl1 and c-Abl2/Arg (collectively, c-Abl), was evaluated in a randomized, double-blind, placebo-controlled phase 2a trial ('the 201 Trial') using once-daily 50 mg, 100 mg, 200 mg or placebo in 137 participants with early, untreated PD. The primary end points in safety and tolerability were met, with 95% of enrolled participants completing the 12-week dosing regimen and a 2-week safety follow-up. Both the average number of adverse events per person and the fraction of participants experiencing at least one treatment-emergent adverse event were similar between risvodetinib and placebo treatment groups, indicating that risvodetinib was safe and well tolerated. Although the trial was not powered to measure efficacy, 14 secondary end points evaluated motor and nonmotor features of disease in hierarchical order. The primary hierarchical secondary end point of the Movement Disorder Society revision to the Unified PD Rating Scale sum of Parts II + III did not reach statistical significance. The study findings indicate that risvodetinib is safe and well tolerated. Longer duration trials are needed to evaluate its potential clinical efficacy. ClinicalTrials.gov registration: NCT05424276 .