Topiramate for Cryptogenic Sensory Peripheral Neuropathy in Metabolic Syndrome (CSPN)

Phase 2 Completed 132 enrolled Virginia Commonwealth University National Institute of Neurological Disorders and Stroke (NINDS) · collab NeuroNEXT Network · collab

RandomizedParallel-groupDouble-blindTreatment

Summary

The TopCSPN trial is a double blinded randomized placebo controlled study of oral topiramate as a potential disease modifying therapy for cryptogenic sensory peripheral neuropathy (CSPN). Patients with CSPN who also have metabolic syndrome (defined by the ATPIII criteria) who do not have an alternative cause for neuropathy will be potentially eligible. The co primary outcome measures are change in the Norfolk Quality of Life - Diabetic Neuropathy (NQOL-DN) Scale and intraepidermal nerve fiber density (IEFND) at the distal thigh. The treatment phase will last 24 months.

Timeline

Start: 2018-02-12
Primary completion: 2021-09-28
Completion: 2021-09-28

Outcome

Missed primary endpoint

paper The difference in change in IENFD and NQOL-DN score was noninferior but not superior in the intention-to-treat (ITT) analysis PMID 37870862 ↗

paper Topiramate did not slow IENFD decline or affect NQOL-DN score in the primary ITT analysis. PMID 37870862 ↗

Publications

Smith AG, Singleton JR, Aperghis A, Coffey CS, Creigh P, Cudkowicz M, Conwit R, Ecklund D, Fedler JK, Gudjonsdottir A, Hauer P, Herrmann DN, Kearney M, Kissel J, Klingner E, Quick A, Revere C, Stino A; NeuroNEXT NN108 TopCSPN Study Team. Safety and Efficacy of Topiramate in Individuals With Cryptogenic Sensory Peripheral Neuropathy With Metabolic Syndrome: The TopCSPN Randomized Clinical Trial. JAMA Neurol. 2023 Dec 1;80(12):1334-1343. doi: 10.1001/jamaneurol.2023.3711.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Topiramate	Small molecule	50 mg	Oral