Clinical Trials Logo

SPG50 clinical trials

View clinical trials related to SPG50.

Filter by:
  • Recruiting  
  • Page 1

NCT ID: NCT05518188 Recruiting - Spasticity, Muscle Clinical Trials

Melpida: Recombinant Adeno-associated Virus (Serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)

Start date: February 15, 2023
Phase: Phase 1/Phase 2
Study type: Interventional

MELPIDA is proposed for the treatment of subjects with SPG50 and targets neuronal cells to deliver a fully functional human AP4M1 cDNA copy via intrathecal injection to counter the associated neuronal loss. Outcomes will evaluate the safety and tolerability of a single dose of MELPIDA, which will be measured by the treatment-associated adverse events (AEs) and serious adverse events (SAEs). Secondarily, the trial will explore efficacy in terms of disease burden assessments.

NCT ID: NCT04712812 Recruiting - Clinical trials for Hereditary Spastic Paraplegia

Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia

HSP
Start date: April 27, 2020
Phase:
Study type: Observational [Patient Registry]

The Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia (HSP) is focused on gathering longitudinal clinical data as well as biological samples (skin and/or blood and/or saliva) from male or female patients who exhibited onset of HSP symptoms at 18 years old or younger with (1) a clinical diagnosis of hereditary spastic paraplegia and/or (2) the presence of variants in HSP related genes and/or be a relative of a person with such a diagnosis. Currently, the treatment for this disorder is generally symptomatic and available therapies improve quality of life, but are grossly inefficient in slowing the disease progression. Access to the registry information will be limited to the study staff who are responsible for recruitment and maintenance of the registry. We hope that recruitment into registry for studies will advance knowledge of the causes, clinical course, diagnosis and treatment of these conditions.