Research network for hereditary spastic paraplegia and primary lateral sclerosis
Spastic Paraplegia - Centers of Excellence Research Network (SP-CERN) - Natural History Study Pilot
This study is trying to learn more about hereditary spastic paraplegia and primary lateral sclerosis by enrolling people with specific types of the condition to help find better treatments and understand how these diseases progress.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 100 (estimated) |
| Sex | All |
| Sponsor | Boston Children's Hospital Academic / other |
| Locations | 11 sites (Miami, Florida and 10 other locations) |
| Trial ID | NCT06553976 on ClinicalTrials.gov |
What this trial studies
The Spastic Paraplegia - Centers of Excellence Research Network (SP-CERN) is a collaborative initiative aimed at enhancing the understanding, diagnosis, and treatment of hereditary spastic paraplegia (HSP) and primary lateral sclerosis (PLS). This observational study focuses on enrolling individuals diagnosed with hereditary spastic paraplegia type 4 (SPG4) or type 5A (SPG5A) to perform natural history studies, discover biomarkers, and develop therapeutic targets. The consortium also aims to harmonize efforts with similar international consortia to accelerate research on these conditions globally.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals of all ages with a clinical and molecular diagnosis of hereditary spastic paraplegia type 4 or type 5A.
Not a fit: Patients without a diagnosis of hereditary spastic paraplegia or primary lateral sclerosis will not benefit from this study.
Why it matters
Potential benefit: If successful, this research could lead to improved diagnosis and treatment options for patients with hereditary spastic paraplegia and primary lateral sclerosis.
How similar studies have performed: Other studies focusing on hereditary spastic paraplegia and related disorders have shown promise, but this consortium approach is relatively novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion 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). Exclusion Criteria: * Not having such a diagnosis and/or not being related to such individual.
Where this trial is running
Miami, Florida and 10 other locations
- University of Miami Miller School of Medicine — Miami, Florida, United States (Not_yet_recruiting)
- University of Iowa Carver College of Medicine — Iowa City, Iowa, United States (Not_yet_recruiting)
- Boston Children's Hospital — Boston, Massachusetts, United States (Recruiting)
- Massachusetts General Hospital — Boston, Massachusetts, United States (Not_yet_recruiting)
- University of Michigan School of Medicine — Ann Arbor, Michigan, United States (Not_yet_recruiting)
- Columbia University - Irving Medical Center — New York, New York, United States (Not_yet_recruiting)
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (Not_yet_recruiting)
- Scottish Rite for Children — Dallas, Texas, United States (Not_yet_recruiting)
- Texas Children's Hospital — Houston, Texas, United States (Not_yet_recruiting)
- Seattle Children's Hospital — Seattle, Washington, United States (Not_yet_recruiting)
- University of Washington School of Medicine — Seattle, Washington, United States (Not_yet_recruiting)
Study contacts
- Study coordinator: Darius Ebrahimi-Fakhari, MD, PhD.
- Email: hsp.research@childrens.harvard.edu
- Phone: 617-355-0097
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.