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Neurological & Neuromuscular

Hereditary Spastic Paraplegia (HSP) Clinical Trials

Also called HSP, Familial Spastic Paraplegia, Strumpell-Lorrain Disease

Hereditary Spastic Paraplegia is a heterogeneous group of genetic neurological disorders unified by selective degeneration of the distal axons of corticospinal motor neurons. Over 80 genetic forms are recognized (SPG1-SPG81), each caused by mutations in different genes encoding proteins involved in various cellular processes: axonal transport, mitochondrial function, membrane trafficking, lipid metabolism, and protein degradation. The common pathological feature is degeneration of the longest corticospinal tract axons, affecting the ability to control leg movement.

In plain words, hereditary spastic paraplegia is a group of genetic conditions where the long nerves that control the legs slowly break down. Leg muscles get stiffer and weaker over time, which makes walking more and more difficult.

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Hereditary Spastic Paraplegia

Where treatments stand, from ClinicalTrials.gov and FDA labels

Also 14 studies with no phase, like observational studies.

FDA review0

No decision pending. FDA calendar

FDA-approved medicines0

No medicine's current FDA label names Hereditary Spastic Paraplegia yet.

Hereditary Spastic Paraplegia has no FDA-approved medicines, 18 open or active clinical trials (17 recruiting), and no pending FDA decision.

Labels checked October 21, 2026.Download the data

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More about Hereditary Spastic Paraplegia

HSP manifests with progressive lower extremity spasticity, weakness, and difficulty walking. The spasticity develops insidiously over months to years, with progressive stiffness of leg muscles and hyperreflexia.

Keep reading about Hereditary Spastic Paraplegia

Some patients show relatively pure HSP with only lower extremity involvement, while others (complex HSP) have associated features including cognitive impairment, seizures, ataxia, peripheral neuropathy, vision loss, or hearing loss depending on the genetic subtype.

Disease progression varies widely by subtype: some forms have static or very slowly progressive disease allowing lifelong ambulation, while others show rapidly progressive paraplegia requiring wheelchair use within years of onset. Genetic heterogeneity means that even within the same family, disease expression can vary.

What are the symptoms of Hereditary Spastic Paraplegia?

These are common symptoms of Hereditary Spastic Paraplegia, and how strong they are varies from person to person.

  • Progressive spasticity and muscle tone increase in legs
  • Weakness of lower extremities affecting gait and walking
  • Hyperreflexia and increased deep tendon reflexes
  • Extensor plantar responses
  • Progressive loss of ambulation in severe forms
  • Pain and muscle cramps

Who gets Hereditary Spastic Paraplegia?

Age of onset varies widely by genetic subtype, ranging from early childhood to adulthood. Some forms present in early childhood; others in late adulthood.

Affects males and females, with gender bias depending on inheritance pattern (X-linked forms more severe in males). Multiple inheritance patterns: autosomal dominant, autosomal recessive, and X-linked. Over 80 genetic subtypes with variable ethnic distribution.

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Side effects people have reported to the FDA

Side effects people have reported to the FDA

Real-world side effect reports from the FDA Adverse Event Reporting System (FAERS). Includes both FDA-approved drugs and investigational therapies from active clinical trials. Click any drug to see what patients reported.

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Diagnosis and genetic testing

Genetic testing for Hereditary Spastic Paraplegia

Genetic testing can confirm a diagnosis, guide treatment decisions, and identify family members who may be at risk.

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Trusted Hereditary Spastic Paraplegia resources

Reputable organizations and medical references for learning more about Hereditary Spastic Paraplegia, including disease registries, foundation resources, and clinical guidelines.

Active clinical trials for Hereditary Spastic Paraplegia

Use this Hereditary Spastic Paraplegia clinical trial finder to see the 17 studies recruiting patients and 1 not yet recruiting in the United States and worldwide, with eligibility criteria in plain English. Of these, 7 test a treatment and 11 are observational studies or registries; each card says which. Every trial comes from ClinicalTrials.gov.

Jump to the trial list, the site map or the pipeline timeline.

Note: Trial recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator before making plans.

18 active trials worldwide
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RecruitingObservational study, no treatment givenRecently updatedNCT07838883

Rare Disease Registry Database for Movement Disorders in Southern Anhui Province

Sponsor: First Affiliated Hospital of Wannan Medical College

, with a total of no more than 140,000 patients). Such diseases are mostly hereditary or congenital in origin. Patients with rare diseases often present with complex symptoms, most of which are neurological.

Ages not specified1 location
Started Sep 2026Updated 3 weeks agoEst. Sep 2029 (~2y 10m)
RecruitingObservational study, no treatment givenRecently updatedNCT07798674

TreatHSP Platform: Adaptive Natural History Platform for Ataxias, HSPs, and Spastic Ataxias

Sponsor: Heidelberg University · Horizon Europe

Ataxias, hereditary spastic paraplegias (HSP), and spastic ataxias (collectively referred to as SPAX diseases) are rare neurological conditions that cause progressive problems with walking, balance, coordination, and daily activities. Although many SPAX diseases are caused by spe...

Ages 5 Years+
Started Jul 2024Updated 2 months agoEst. Dec 2035 (~9y 1m)
RecruitingTreatment trialPhase 1/2Updated a few months agoNCT05518188

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

Intervention: MELPIDA

Sponsor: Elpida Therapeutics SPC

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 ME...

Ages 4 Months to 10 Years1 location
Started Feb 2023Updated 4 months agoEst. Oct 2028 (~1y 10m)
RecruitingTreatment trialNo updates in a whileNCT07417943

Neuromodulation to Enhance Motor Function in HSP

Intervention: transcutaneous spinal cord stimulation

Sponsor: Rahul Sachdeva

Hereditary spastic paraplegia (HSP) is a rare neurological condition that causes stiffness, weakness, and difficulty walking due to damage in the nerves that control movement. This study will test whether a noninvasive form of spinal cord stimulation, called transcutaneous spinal...

Ages 18 Years+1 location
Started Apr 2026Updated 6 months agoEst. Dec 2027 (~1y 1m)
RecruitingTreatment trialPhase 3No updates in a whileNCT06692712

Phase 3 Efficacy Study With Concurrent Control of IT MELPIDA in SPG50.Concurrent Controls.

Intervention: MELPIDA

Sponsor: Elpida Therapeutics SPC

Phase 3, open-label study to assess the efficacy and safety of a single lumbar intrathecal administration of MELPIDA in individuals with Hereditary Spastic Paraplegia Type 50 (SPG50).

Ages 4 Months to 72 Months2 locations
Started Apr 2026Updated 6 months agoEst. Feb 2032 (~5y 3m)
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Active trial locations11 cities in the US
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Trial Pipeline

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Data from ClinicalTrials.gov, U.S. National Library of Medicine.
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