Feasibility of Simon Two‐Stage Futility Trials in People with Early, Symptomatically Treated Parkinson's Disease
Phase 2 futility trials using the Simon two-stage methodology are feasible in early PD, including in treated and untreated patients. OFF-state scores are preferable to ON-state scores as the primary outcome measure. Futility trials offer a smaller-scale, faster, and cost-effective approach to assessing new candidate treatments in PD. © 2025 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
Authors
- Michael G. Schlossmacher (ORCID: https://orcid.org/0000-0002-0394-0300)
- Jop Mostert (ORCID: https://orcid.org/0000-0003-3640-9935)
- Marcus Koch (ORCID: https://orcid.org/0000-0001-9972-5092)
- Daryl Wile (ORCID: https://orcid.org/0000-0002-5341-9062)
- Bernard M.J. Uitdehaag (ORCID: https://orcid.org/0000-0002-9226-7364)
- Eva M. M. Strijbis (ORCID: https://orcid.org/0000-0001-6705-5864)
- Camila Aquino (ORCID: https://orcid.org/0000-0003-1643-9117)
- Tarrant McPherson
- Gary Cutter (ORCID: https://orcid.org/0000-0002-8455-980X)
- Miguel D’haeseleer (ORCID: https://orcid.org/0000-0003-4378-0089)
- Tiago Mestre (ORCID: https://orcid.org/0000-0002-6973-7479)
- Lorraine V. Kalia (ORCID: https://orcid.org/0000-0002-9384-1305)
- Justyna R. Sarna (ORCID: https://orcid.org/0000-0003-3308-1540)
Institutions
- National Multiple Sclerosis Society (US)
- University of British Columbia (CA)
- Emory University (US)
- University of Ottawa (CA)
- University of Calgary (CA)
- University of Toronto (CA)
- Toronto Western Hospital (CA)
- Multiple Sclerosis Foundation (US)
- Ottawa Hospital (CA)
- European Platform of Women Scientists (BE)
- University of Alabama at Birmingham (US)
- MIND Research Institute (US)
- Universitair Ziekenhuis Brussel (BE)
- Amsterdam University Medical Centers (NL)
- Rijnstate Hospital (NL)
- Krembil Research Institute
- uOttawa Brain and Mind Research Institute
Publication Details
- Journal
- Movement Disorders
- Published
- 2025-10-20
- DOI
- https://doi.org/10.1002/mds.70090
- Primary Topic
- Parkinson's Disease Mechanisms and Treatments
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Michael J. Fox Foundation for Parkinson's Research
- Amgen
- Bristol-Myers Squibb
- Sanofi
- Biogen
- CHDI Foundation
- Teva Pharmaceutical Industries
- Alexion Pharmaceuticals
- Celgene
- University of Pennsylvania
- CSL Behring
- International Parkinson and Movement Disorder Society
- University of Ottawa
- Fondation Brain Canada
- Regeneron Pharmaceuticals
- PTC Therapeutics
- GW Pharmaceuticals
- Horizon Pharmaceuticals
- AveXis
- Prothena
- University of Toronto
- Krembil Foundation
- Parkinson Canada
- Mitsubishi Tanabe Pharma Corporation
- Neuraxpharm
- Genentech
- Sanofi Genzyme
- Canadian Institutes of Health Research
- Cilag