Hereditary Spastic Paraplegia Type 4 (SPG4) Clinical Trials

Hereditary spastic paraplegia type 4 (SPG4) is an autosomal dominant neurodegenerative disorder caused by mutations in the SPAST gene encoding spastin, a microtubule-severing protein essential for proper intracellular transport and axonal dynamics. Spastin dysfunction impairs axonal transport capacity and microtubule dynamics, leading to progressive selective degeneration of the longest corticospinal tract axons that descend from motor cortex to spinal cord. This length-dependent axonopathy particularly affects the longest neurons because they have the greatest energy and transport demands.

The most common symptoms of Hereditary Spastic Paraplegia Type 4 include Progressive lower limb weakness and stiffness, Spasticity affecting legs and feet, Hyperreflexia and Babinski sign, Difficulty walking and balance problems, Urinary urgency and incontinence, among others. Hereditary Spastic Paraplegia Type 4 affects approximately 1 in 10,000 to 20,000 people worldwide. Symptoms can range from mild to severe and may vary between individuals.

Trial Friend lists all active clinical trials for Hereditary Spastic Paraplegia Type 4 from ClinicalTrials.gov, with eligibility criteria translated into plain English. Patients can filter by trial phase and recruitment status, search by location, and use the Match Me tool to score trials against their profile. Research into hereditary spastic paraplegia is advancing multiple promising therapeutic approaches targeting spastin function restoration, improving axonal transport capacity, and modulating neuroinflammation. Clinical trials are exploring modulators of microtubule dynamics, neuroprotective strategies, and cellular stress responses.

As of September 2026, Trial Friend's curated drug index does not include an FDA-approved treatment specific to Hereditary Spastic Paraplegia Type 4. The Treatments section of this page checks FDA drug labels live and lists any approved drug whose label names the condition, and the trials listed below cover the investigational options.

Hereditary Spastic Paraplegia Type 4 is caused by variants in the SPAST gene. SPG4 is the single most common hereditary spastic paraplegia; HSP as a whole involves dozens of genes.

It is autosomal dominant, so each child of an affected parent has a 1 in 2 chance of inheriting it. The condition affects approximately 1 in 10,000 to 20,000 people worldwide. Genetic testing confirms the diagnosis.

Yes. Genetic testing for Hereditary Spastic Paraplegia Type 4 looks at the SPAST gene. SPG4 is the single most common hereditary spastic paraplegia; HSP as a whole involves dozens of genes.

It is autosomal dominant, so each child of an affected parent has a 1 in 2 chance of inheriting it.

A confirmed variant establishes the diagnosis and is often required to enroll in gene-specific trials. The labs currently offering the test are listed in the Genetic Testing section of this page, pulled live from the NIH Genetic Testing Registry. A genetic counselor can help interpret the result and decide which relatives should be tested.

Eligibility for Hereditary Spastic Paraplegia Type 4 clinical trials depends on factors like age, diagnosis, treatment history, and overall health. Each trial sets its own inclusion and exclusion criteria. Trial Friend translates these requirements from medical language into plain English so patients can review them more easily. The Match Me tool can score your profile against active trials to help identify studies that may be a fit.

Participating in a Hereditary Spastic Paraplegia Type 4 clinical trial typically involves scheduled visits, medical tests, and close monitoring by a research team. Depending on the study phase, you may receive an investigational treatment, a placebo, or the current standard of care. Trials may last from a few weeks to several years. Study participation is voluntary, and you can withdraw at any time.