Journal article
The Spastic Paraplegia-Centers of Excellence Research Network (SP-CERN): Clinical Trial Readiness for Hereditary Spastic Paraplegia
Neurology. Genetics, Vol.11(2), e200249
04/2025
DOI: 10.1212/NXG.0000000000200249
PMCID: PMC11849523
PMID: 39996129
Abstract
The primary objective of this paper was to present the establishment of the Spastic Paraplegia-Centers of Excellence Research Network (SP-CERN) aimed at promoting clinical trial readiness for hereditary spastic paraplegia (HSP). SP-CERN is unique in its approach to addressing the diagnostic and therapeutic challenges associated with HSP through a large-scale, collaborative effort.
Participants with HSP are identified through multicenter collaborations across 11 institutions in the United States. SP-CERN systematically collects longitudinal clinical data, biospecimens, and wearable device data from patients. Data are stored in a centralized REDCap database, facilitating shared access for analysis. Patients are evaluated using standardized assessment tools for motor function, biomarkers, and digital outcome measures.
SP-CERN has established a biorepository, centralized data collection methods, and standardized clinical assessments. It is conducting natural history studies for all HSP subtypes, enabling the validation of biomarkers and development of gene-based therapies.
SP-CERN's collaborative approach bridges gaps in clinical care and research for HSP by improving diagnostic capabilities and promoting clinical trial readiness. This initiative represents a framework for rare disease research, accelerating the development of novel therapies and improving patient outcomes through standardized, multi-institutional collaboration.
Details
- Title: Subtitle
- The Spastic Paraplegia-Centers of Excellence Research Network (SP-CERN): Clinical Trial Readiness for Hereditary Spastic Paraplegia
- Creators
- Luca Schierbaum - Boston Children's HospitalVicente Quiroz - Boston Children's HospitalKathryn Yang - Boston Children's HospitalJoshua Rong - Boston Children's HospitalNicole Battaglia - Boston Children's HospitalUmar Zubair - Boston Children's HospitalMichelle Christie - Texas Scottish Rite Hospital for ChildrenMarie Davis - University of WashingtonDaniel Calame - Baylor College of MedicineMatt C Danzi - Dr. John T. Macdonald FoundationRichard S Finkel - St. Jude Children's Research HospitalJoshua Burns - St. Jude Children's Research HospitalDonald L Gilbert - Cincinnati Children's Hospital Medical CenterDararat Mingbunjerdsuk - Seattle Children's HospitalGreg Pruitt - Spastic Paraplegia FoundationNorma Pruitt - Spastic Paraplegia FoundationJohn Cobb - Spastic Paraplegia FoundationReza Sadjadi - Harvard UniversityChristopher R Cashman - Harvard UniversityCraig Blackstone - Harvard UniversityJohn K Fink - University of MichiganMichael E Shy - University of IowaStephan Zuchner - Dr. John T. Macdonald FoundationDarius Ebrahimi-Fakhari - Boston Children's Hospital
- Resource Type
- Journal article
- Publication Details
- Neurology. Genetics, Vol.11(2), e200249
- DOI
- 10.1212/NXG.0000000000200249
- PMID
- 39996129
- PMCID
- PMC11849523
- NLM abbreviation
- Neurol Genet
- ISSN
- 2376-7839
- eISSN
- 2376-7839
- Publisher
- LIPPINCOTT WILLIAMS & WILKINS
- Grant note
- Spastic Paraplegia Foundation
The SP-CERN Pilot Study is supported by the Spastic Paraplegia Foundation.
- Language
- English
- Date published
- 04/2025
- Academic Unit
- Neurology; Molecular Physiology and Biophysics; Iowa Neuroscience Institute
- Record Identifier
- 9984795371102771
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