Spastic Paraplegia - Centers of Excellence Research Network

Description

The Spastic Paraplegia - Centers of Excellence Research Network (SP-CERN) is a collaborative research consortium dedicated to advancing the understanding, diagnosis, and treatment of hereditary spastic paraplegia (HSP) and primary lateral sclerosis (PLS). Aims of the consortium are to a) perform natural history studies of HSP subtypes, b) discover and validate biomarkers and clinician- and patient-reported outcome measures, c) uncover HSP's molecular pathophysiology and develop rational therapeutic targets, and d) perform sufficiently powered clinical trials. The current pilot study is aimed at enrolling 100 individuals with hereditary spastic paraplegia type 4 (SPG4) or hereditary spastic paraplegia type 5A (SPG5A).

Conditions

Hereditary Spastic Paraplegia, Primary Lateral Sclerosis, SPG4, SPG5A, Spastic Paraplegia 4, Spastic Paraplegia 5A, Early Onset Hereditary Spastic Paraplegia, Neuromuscular Diseases, Spastic Paraplegia, Hereditary

Study Overview

Study Details

Study overview

The Spastic Paraplegia - Centers of Excellence Research Network (SP-CERN) is a collaborative research consortium dedicated to advancing the understanding, diagnosis, and treatment of hereditary spastic paraplegia (HSP) and primary lateral sclerosis (PLS). Aims of the consortium are to a) perform natural history studies of HSP subtypes, b) discover and validate biomarkers and clinician- and patient-reported outcome measures, c) uncover HSP's molecular pathophysiology and develop rational therapeutic targets, and d) perform sufficiently powered clinical trials. The current pilot study is aimed at enrolling 100 individuals with hereditary spastic paraplegia type 4 (SPG4) or hereditary spastic paraplegia type 5A (SPG5A).

Spastic Paraplegia - Centers of Excellence Research Network (SP-CERN) - Natural History Study Pilot

Spastic Paraplegia - Centers of Excellence Research Network

Condition
Hereditary Spastic Paraplegia
Intervention / Treatment

-

Contacts and Locations

Miami

University of Miami Miller School of Medicine, Miami, Florida, United States, 33136

Iowa City

University of Iowa Carver College of Medicine, Iowa City, Iowa, United States, 52242

Boston

Boston Children's Hospital, Boston, Massachusetts, United States, 02215

Boston

Massachusetts General Hospital, Boston, Massachusetts, United States, 02215

Ann Arbor

University of Michigan School of Medicine, Ann Arbor, Michigan, United States, 48109

New York

Columbia University - Irving Medical Center, New York, New York, United States, 10032

Cincinnati

Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States, 45229-3039

Dallas

Scottish Rite for Children, Dallas, Texas, United States, 75219

Houston

Texas Children's Hospital, Houston, Texas, United States, 77035

Seattle

Seattle Children's Hospital, Seattle, Washington, United States, 98105

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Male or female patients of all ages with a clinical and molecular diagnosis of hereditary spastic paraplegia type 4 (SPG4, SPAST) or hereditary spastic paraplegia type 5A (SPG5A, CYP7B1).
  • * Not having such a diagnosis and/or not being related to such individual.

Ages Eligible for Study

to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Boston Children's Hospital,

Study Record Dates

2027-06-04