Clinical trials: Hereditary Spastic Paraplegia
Muscular Dystrophies, Muscle Myopathies
Genetic and Physical Study of Childhood Nerve and Muscle Disorders
Background: Some nerve and muscle disorders that start early in life (before age 25), like some forms of muscular dystrophy, can run in families. However, the genetic causes of these disorders are not known.
- Conditions
- Muscular Dystrophies, Muscle Myopathies, Hereditary Spastic Paraplegias, Inherited Neuropathies
- Trial name
- NCT01568658
- Where
- United States
- Volunteers
- 2,586 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Hereditary Spastic Paraplegia, Adrenomyeloneuropathy
Repetitive Transcranial Magnetic Stimulation as Therapy in Hereditary Spastic Paraplegia and Adrenomyeloneuropathy
Hereditary spastic paraplegia (HSP) is the group of inherited disorders, characterized by progressive gait disturbance. There is no established therapy. Adrenoleukodystrophy (AMN) is an x-linked hereditary disease.
- Trial name
- NCT03627416
- Testing
- rTMS
- Where
- Poland
- Volunteers
- 15 people
- Sponsor
- Jakub Antczak
Hereditary Spastic Paraplegia
A Prospective Cohort Study of ITB Treatment for HSP
The investigators conduct a prospective cohort study to explore the treatment effectiveness of continuous infusion of intrathecal baclofen (ITB) for hereditary spastic paraplegia (HSP) in China, delve into the optimal timing for…
- Trial name
- ITB-HSP (NCT06844734)
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Shanghai 6th People's Hospital
Hereditary Spastic Paraplegia
Effects of Botulinum Toxin Injections in Patients With Hereditary Spastic Paraplegia
Hereditary spastic paraplegias constitute a heterogeneous group of diseases with the common predominant feature of spasticity of the lower limbs.
- Trial name
- SPASTOX (NCT02604186)
- Testing
- Botulinum Toxin Injections
- Where
- Brazil
- Volunteers
- 55 people
- Sponsor
- University of Campinas, Brazil
Hereditary Spastic Paraplegia
Robot-assisted Walking Treatment in Hereditary Spastic Paraplegia (HSP)
Patients will undergo a combined rehabilitation treatment consisting of 15 sessions on Lokomat and 15 sessions of physical therapy over three weeks for a total of 30 sessions. Two sessions/day for 5 days a week.
- Trial name
- NCT06728787
- Testing
- Robot-Assisted Gait Training
- Where
- Italy
- Volunteers
- 50 people
- Sponsor
- IRCCS Eugenio Medea
AMN, AMN Gene Mutation
Study of Disease Progression in Adults With Inherited Forms of Spastic Paraplegia
The course of AMN-related disabilities over time is poorly or incompletely understood due to a limited number of patients and lack of treatments.
- Conditions
- AMN, AMN Gene Mutation, X-ALD
- Trial name
- CYGNET (NCT05008874)
- Testing
- Natural History Observation
- Where
- 6 sites in United States, Germany and 1 more
- Volunteers
- 65 people
- Sponsor
- SwanBio Therapeutics, Inc.
Mitochondrial Encephalomyopathies, Hereditary Spastic Paraplegia
Natural History Study of Patients with HPDL Mutations
This study uses medical records that allow retrospective data extraction of clinical manifestation to assess the natural history of HPDL mutations
- Conditions
- Mitochondrial Encephalomyopathies, Hereditary Spastic Paraplegia, Spastic Paraplegia, White Matter Disease
- Trial name
- NCT05848271
- Testing
- Patient Registry, Dry blood spots sampling
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University of California, San Diego
Hereditary Spastic Paraplegia
Calcium Folinate Treatment of Spastic Paraplegia 56
SPG56 is one of the complicated and early-onset HSP subtypes caused by genetic mutations in CYP2U1. So far, there is no standardized and specific clinical therapy for SPG56.
- Trial name
- CFT-SPG56 (NCT06478238)
- Testing
- calcium folinate
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shanghai 6th People's Hospital
Hereditary Spastic Paraplegia, Neurodegenerative Diseases
Hereditary Spastic Paraplegia Genomic Sequencing Initiative (HSPseq)
The purpose of the HSP Sequencing Initiative is to better understand the role of genetics in hereditary spastic paraplegia (HSP) and related disorders.
- Conditions
- Hereditary Spastic Paraplegia, Neurodegenerative Diseases, Pediatric Disorder, Spasticity, Muscle
- Trial name
- NCT05354622
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Boston Children's Hospital
Hereditary Spastic Paraparesis
Testing Miglustat Administration in Subjects With Spastic Paraplegia 11
Hereditary spastic paraparesis type 11 (SPG11) is caused by mutations in the SPG11 gene that produces spatacsin, a protein involved in lysosomal function.
- Trial name
- TreatSPG11 (NCT04768166)
- Testing
- Miglustat 100 MG
- Where
- Italy
- Volunteers
- 10 people
- Sponsor
- IRCCS Fondazione Stella Maris
Hereditary Spastic Paraplegia
Dental-Oral and Craniofacial Characteristics of Hereditary Spastic Paraplegia: A Preliminary Study
This study aims to analyze the oral and craniofacial characteristics associated with Hereditary Spastic Paraplegia (HSP) using non-invasive complementary diagnostic tests, with the hypotesis that there is a characteristic…
- Trial name
- NCT07799233
- Where
- Spain
- Volunteers
- 50 people
- Sponsor
- University of Barcelona
Spasticity, Muscle, Microcephaly
Melpida: Recombinant Adeno-associated Virus (Serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)
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.
- Conditions
- Spasticity, Muscle, Microcephaly, Intellectual Deficiency, Growth Retardation
- Trial name
- NCT05518188
- Testing
- MELPIDA
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Elpida Therapeutics SPC
SPG4
Studying Non-motor Symptoms in HSP
Comparing the non-motor symptoms of patients with hereditary spastic paraplegia (HSP) to healthy controls (spouses, relatives or other healthy controls) by using the a number of specific questionaires
- Trial name
- NCT03204773
- Testing
- EuroQol five dimensions questionaire (EQ-5D), Modified Fatigue Impact Scale (MFI), Brief Pain Inventory (BPI)
- Where
- Germany
- Volunteers
- 236 people
- Sponsor
- University Hospital Tuebingen
Cognitive Impairment, Hereditary Spastic Paraplegia
Studying Cognition in SPG4
Comparing the cognitive levels of patients with SPG4 mutations to healthy controls.
- Trial name
- NCT03104088
- Testing
- CANTAB, MOCA
- Where
- Germany
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University Hospital Tuebingen
Hereditary Spastic Paraparesis
Evaluation of an Intensive Training Program for Patients with Hereditary Spastic Paraparesis SPG4/Spast
Hereditary spastic paraparesis is a group of inherited neurological diseases. Only symptomatic treatments exist for the moment. The Modifspa study (cf citation) carried out by the team showed that patients perceived a feeling of…
- Trial name
- WALK-up (NCT06229626)
- Testing
- intensive reeducation
- Where
- France
- Volunteers
- 50 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Hereditary Spastic Paraplegia
Disease Natural History and Biomarkers of SPG3A, SPG4A, and SPG31
Background: Hereditary spastic paraplegia (HSP) usually progresses slowly. Researchers want to learn more about how its symptoms change over time.
- Trial name
- NCT02859428
- Where
- United States
- Volunteers
- 51 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Parkinson Disease, Hereditary Spastic Paraparesis
Enhancement of Quality of Work And Life
Work participation is essential for quality of life, providing purpose, social interaction, financial security, and shaping social status.
- Conditions
- Parkinson Disease, Hereditary Spastic Paraparesis, Cerebellar Ataxia, Mitochondrial Diseases
- Trial name
- EQuAL (NCT06573866)
- Testing
- Preventive Participatory workplace intervention
- Where
- Netherlands
- Volunteers
- 124 people
- Sponsor
- Radboud University Medical Center
Hereditary Spastic Paraplegia
Neuromodulation to Enhance Motor Function in HSP
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.
- Trial name
- NCT07417943
- Testing
- transcutaneous spinal cord stimulation
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Rahul Sachdeva
Hereditary Spastic Paraplegia, Spastic Ataxia
TreatHSP Platform: Adaptive Natural History Platform for Ataxias, HSPs, and Spastic Ataxias
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…
- Trial name
- TreatHSP (NCT07798674)
- Where
- 30 sites in Austria, Czechia and 7 more
- Volunteers
- 4,000 people, healthy volunteers welcome
- Sponsor
- Heidelberg University
Hereditary Spastic Paraplegia, SPG47
Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia
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 and female…
- Conditions
- Hereditary Spastic Paraplegia, SPG47, SPG50, SPG51
- Trial name
- HSP (NCT04712812)
- Where
- United States
- Volunteers
- 700 people, healthy volunteers welcome
- Sponsor
- Boston Children's Hospital