Clinical trials: Spastic Paraplegias
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 Type 50
Phase 3 Efficacy Study With Concurrent Control of IT MELPIDA in SPG50.Concurrent Controls.
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).
- Trial name
- SPG50 (NCT06692712)
- Testing
- MELPIDA
- Where
- 2 sites in United States, Spain
- Volunteers
- 24 people
- Sponsor
- Elpida Therapeutics SPC
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
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)
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
Neuromuscular Diseases, Obesity (Disorder)
Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology
The ActiLiège-Adult study is a prospective, longitudinal, observational study designed to collect natural history data on adult patients with neurological or metabolic diseases affecting movement.
- Conditions
- Neuromuscular Diseases, Obesity (Disorder), Myotonic Dystrophy 1, Myasthenic Syndrome
- Trial name
- Acti-Adult (NCT07136844)
- Testing
- Syde, Dynamometric measurements of muscle strength, The 6-minute walk test (6MWT)
- Where
- Belgium
- Volunteers
- 300 people
- Sponsor
- Centre Hospitalier Universitaire de Liege
Motor Neuron Diseases, Spinocerebellar Ataxias
Rare Disease Registry Database for Movement Disorders in Southern Anhui Province
According to the World Health Organization (WHO) definition, a rare disease is a disease affecting fewer than 6.5 people per 10,000; in China, it generally refers to diseases with a prevalence of less than 1/10,000 (i.e., with a…
- Conditions
- Motor Neuron Diseases, Spinocerebellar Ataxias, Hereditary Muscular Dystrophy, Hereditary Spastic Paraplegia
- Trial name
- W-MoSCA (NCT07838883)
- Where
- China
- Volunteers
- 180 people
- Sponsor
- First Affiliated Hospital of Wannan Medical College
Amyotrophic Lateral Sclerosis, Progressive Muscular Atrophy
A Patient Centric Motor Neuron Disease Activities of Daily Living Scale
The purpose of this study is to learn about rates of patient-reported disease progression in patients with motor neuron diseases (amyotrophic lateral sclerosis, progressive muscular atrophy, primary lateral sclerosis, hereditary…
- Conditions
- Amyotrophic Lateral Sclerosis, Progressive Muscular Atrophy, Primary Lateral Sclerosis, Hereditary Spastic Paraplegia
- Trial name
- NCT02852278
- Testing
- Web-based Survey
- Volunteers
- 410 people
- Sponsor
- University of South Florida
Spastic Paraplegia Type 50
Administration of MELPIDA to Determine the Safety and Efficacy for Patients With Spastic Paraplegia Type 50
This will be a first-in-human Phase I, open-label, single dose clinical study of MELPIDA administered intrathecally (IT) through a lumbar puncture (LP) to a single subject with confirmed pathogenic mutations in the Adaptor…
- Trial name
- NCT06069687
- Testing
- MELPIDA
- Where
- Canada
- Volunteers
- 1 people
- Sponsor
- The Hospital for Sick Children
Primary Lateral Sclerosis, Hereditary Spastic Paraplegia
Nuclear Magnetic Spectroscopy Imaging to Evaluate Primary Lateral Sclerosis, Hereditary Spastic Paraplegia and Amyotrophic Lateral Sclerosis
This study will use a magnetic resonance imaging technique called nuclear magnetic spectroscopy (H-MRS) to define the pathology and progression of primary lateral sclerosis, hereditary spastic paraplegia and amyotrophic lateral…
- Conditions
- Primary Lateral Sclerosis, Hereditary Spastic Paraplegia, Amyotrophic Lateral Sclerosis
- Trial name
- NCT00023075
- Where
- United States
- Volunteers
- 55 people, healthy volunteers welcome
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Hereditary Spastic Paraplegia, Primary Lateral Sclerosis
Spastic Paraplegia - Centers of Excellence Research Network
The Spastic Paraplegia - Centers of Excellence Research Network (SP-CERN) is a collaborative research consortium dedicated to advancing the understanding, diagnosis, and treatment of hereditary spastic paraplegia (HSP) and…
- Conditions
- Hereditary Spastic Paraplegia, Primary Lateral Sclerosis, SPG4, SPG5A
- Trial name
- SP-CERN (NCT06553976)
- Where
- 11 sites in United States
- Volunteers
- 100 people
- Sponsor
- Boston Children's Hospital
Spina Bifida, Cerebral Palsy
Assessing Transition Practices for Children With Disabilities: Pathways to a Successful Adulthood
Transition to adult care is a purposeful and structured movement of youth with complex disabilities from child centered to adult oriented services. This complex and potentially challenging period requires careful planning.
- Conditions
- Spina Bifida, Cerebral Palsy, Spinal Cord Injury, Spastic Paraplegia
- Trial name
- NCT07706517
- Testing
- Chart review, Semi-structured interview
- Where
- France
- Volunteers
- 128 people
- Sponsor
- Centre Hospitalier Universitaire de Saint Etienne
Spastic Paraplegia
A Registered Cohort Study on Spastic Paraplegia
The aim of this study is to determine the clinical spectrum and natural progression of Hereditary Spastic Paraplegias(HSP) and related disorders in a prospective multicenter natural history study, to assess the clinical, genetic…
- Trial name
- NCT04006418
- Where
- China
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- Wan-Jin Chen
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.
Hereditary Spastic Paraplegia
Phenotypes, Biomarkers and Pathophysiology in Hereditary Spastic Paraplegias and Related Disorders
The aim of this study is to determine the clinical spectrum and natural progression of Hereditary Spastic Paraplegias (HSP) and related disorders in a prospective multicenter natural history study, identify digital, imaging and…
- Trial name
- HSP-PBP (NCT03981276)
- Testing
- Clinical rating scale to measure disease severity and progression, Next-Gen Sequencing (NGS)
- Where
- 13 sites in Austria, Germany and 1 more
- Volunteers
- 2,000 people, healthy volunteers welcome
- Sponsor
- Dr. Rebecca Schule
HSP, Hereditary Spastic Paraplegia
Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)
Safety and Efficacy of AAV9/AP4B1 For Patients with AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47): A Phase 1/2 Single-Center, Open-Label Study of Stereotactic Intra-cisterna Magna Administration.
- Conditions
- HSP, Hereditary Spastic Paraplegia, Hereditary Spastic Paraparesis, Hereditary Spastic Paraplegia Type 50
- Trial name
- NCT06948019
- Testing
- BFB-101 (AAV9-CBh-AP4B1)
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- BlackfinBio Ltd
Idiopathic Toe Walking, Cerebral Palsy
The Effect of Targeting the Plantaris Muscle-tendon in Surgical Correction of Ankle Equinus in Children
Tight ankle muscles can produce ankle equinus (limited ability to pull the foot upward) and occur often in children, significantly impacting their ability to walk.
- Conditions
- Idiopathic Toe Walking, Cerebral Palsy, Hereditary Spastic Paraparesis, Traumatic Brain Injury
- Trial name
- Plantaris Sx (NCT07090057)
- Testing
- Tendoachilles (TA) lengthening or gastrocnemius (GN) recession, then Plantaris tenotomy, Plantaris tenotomy, then Tendoachilles (TA) lengthening or gastrocnemius (GN) recession
- Where
- Canada
- Volunteers
- 42 people
- Sponsor
- University of Alberta
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
Stroke, Spastic Paraplegia
Turkish Lower-Extremity Motor Activity Log (LE-MAL)
The Motor Activity Log was developed to measure paretic upper extremity use in daily activities in the real-life context (real world) of people with different health conditions, including stroke.
- Trial name
- NCT06156813
- Testing
- Diagnostic test
- Where
- Turkey (Türkiye)
- Volunteers
- 80 people
- Sponsor
- Gaziantep Islam Science and Technology University
Hereditary Spastic Paraplegia
A Pilot Study of Shockwave Therapy in HSP
This is a pilot study of the efficacy, safety, and tolerability of shockwave therapy for the treatment of spasticity in Hereditary Spastic Paraplegia.
- Trial name
- NCT05411627
- Testing
- Shockwave Therapy
- Volunteers
- healthy volunteers welcome
- Sponsor
- Spaulding Rehabilitation Hospital