Understanding and modifying Fabry disease: Rationale and design of a pivotal Phase 3 study and results from a patient-reported outcome validation study.

"AcknowledgmentsThe authors thank all patients and investigators involved in the MODIFY study and the PRO validation study. The authors also thank Yosef Mansour, PhD, an employee of Idorsia Pharmaceuticals Ltd., for medical writing support during manuscript development. Editorial support was provided by Edward Johnson, PhD, of Alpharmaxim Healthcare Communications, funded by Idorsia Pharmaceuticals Ltd."

"Funding source The MODIFY study was supported by Idorsia Pharmaceuticals Ltd."

"MODIFY is a Multicenter, dOuble-blind, ranDomized, placebo-controlled, parallel-group Phase 3 study to determine the clinical effIcacy and safety of lucerastat oral monotherapy in adult patients with FabrY disease. The study is comprised of a screening period lasting 6–7 weeks and a double-blind treatment period lasting 6 months. Patients who complete the 6-month treatment period have the option to enter into an open-label extension study (conducted under a separate protocol, ClinicalTrials.gov: NCT03737214); patients who do not enter the extension study continue to the safety follow-up period (), which lasts 1 month for female patients and 3 months for male patients. MODIFY is being conducted in full compliance with the principles of the Declaration of Helsinki, International Conference on Harmonization Good Clinical Practice Guidelines, and local laws and regulations of the countries in which the study is conducted. MODIFY is registered on Clinicaltrials.gov (NCT03425539).Fig. 1MODIFY study design. *For patients who enter the OLE study, the EOS corresponds to the EOT visit. †The safety follow-up is applicable to all patients except those who enter the OLE study. EOS, end of study; EOT, end-of-treatment; OLE, open-label extension.Fig. 1"