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NCT Number: NCT06844734

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 starting treatment, and investigate the response differences among different subtypes. The ultimate goal is to provide clinical evidence and guidance for the application of ITB in treating HSP in China, as well as improve the life expectancy and quality of life for HSP patients. The main questions it aims to answer are:

1. Changes in gait and motor function, as well as spasticity levels, compared to pre-surgery and control group after ITB surgery. 2. Changes in quality of life, pain, psychological and emotional status, and cognition compared to pre-surgery and control group after ITB surgery. 3. Complications following ITB surgery. 4. Impact of ITB surgery on the occurrence and progression of skeletal deformities. 5. Subgroup analysis: comparing surgical outcomes between different genotypes and between simple versus complex types. 6. Determine the optimal timing for ITB intervention.

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Key information

Conditions

Hereditary Spastic Paraplegia Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hereditary Sensory and Motor Neuropathy Heredodegenerative Disorders, Nervous System Nervous System Diseases Nervous System Malformations Neurodegenerative Diseases Neuromuscular Diseases Peripheral Nervous System Diseases Polyneuropathies Spastic Paraplegia, Hereditary

Age range

14 year–70 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Shanghai Sixth People's Hospital Affiliated to Shanghai Jiao Tong University School of Medicine

Shanghai, Shanghai Municipality, 213000, China

Location status: Recruiting

Location contact

Wotu Tian, phD

CONTACT

[email protected]

21 64369181

About this study

Hereditary spastic paraplegias (HSPs) are a heterogeneous group of neurodegenerative disorders characterized by progressive spasticity and weakness of the lower limbs due to retrograde axonal degeneration of the corticospinal tract. Baclofen is a selective GABA-B receptor agonist and is commonly used for the treatment of spasticity. Baclofen can be administered orally or intrathecally by the surgical implantation of a specialized pump. Intrathecal baclofen (ITB) is significantly more potent for the treatment of spasticity than the oral form. The goal of this clinical trial is to explore the efficacy and safety of ITB in the treatment of HSP patients. This study is prospective, open-label, single center, and this trial will last for 3 years. A total of 50 patients will participate. Twenty-five patients who will receive ITB and the other 25 control patients will receive oral baclofen. Patients with HSP aged 14 to 70 years, with a Modified Ashworth Scale score of ≥3 in ≥2 joints of lower limb, will be recruited. Patients who do not agree to undergo ITB and/or those who have an inadequate response during intrathecal baclofen test will receive oral baclofen therapy and undergo natural history observation. Professional clinical evaluations are conducted regularly for both groups.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Patients meet the clinical and genetic diagnostic criteria of hereditary spastic paraplegia (HSP);
  • Age: 14 to 70 years old
  • Modified Ashworth Score for lower limbs: ≥2 joints with muscle tone ≥grade 3
  • Patients are willing to participate in clinical trials and able to understand and comply with the research program

Exclusion criteria

  • Patients are allergic to the baclofen
  • Other neurological diseases likely affecting the evaluation of study treatment
  • Other medical conditions such as: heart disease, tumor, blood disease, liver disease, kidney disease, etc. in the past 1 year
  • Pregnancy or lactating women or subjects who are unable to use appropriate contraception during the trial
  • Participating in another study drug trial and used the investigational drug in the past 30 days
  • Subjects have poor compliance or other factors that are not suitable for participating in the clinical trial

Treatment and study plan

Primary outcomes

  1. Six-Minute Walk Test(6-MWT)

    Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively

    The 6-MWT measures the distance a person can walk in six minutes. A shorter distance indicates lower exercise capacity.

  2. Step length

    Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively

    Step length is measured by gait analysis device. Step length refers to the distance from the heel strike of one foot to the heel strike of the opposite foot. Longer step length indicates that the patient has improved lower limb muscle strength and coordination, and that spastic symptoms have been alleviated.

  3. walking speed

    Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively

    Walking speed is measured by gait analysis device. Walking speed refers to the distance covered by a person within a unit of time while walking, expressed in meters per minute (m/min). An increase in walking speed indicates an improvement in spasticity and motor function.

  4. knee flexion angle

    Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively

    Knee flexion angle is measured by gait analysis device.An increase in the knee flexion angle indicates improved motor function and reduced spasticity.

  5. Modified Ashworth Scale(MAS)

    Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively

    range: 0-4, higher scores mean more severe spasticity.

  6. Range of Motion (ROM) of Joints

    Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively

    ROM refers to the distance and direction a joint can move to its full potential. Flexion, extension, abduction, adduction of joints are measured.

Secondary outcomes

  1. Patient Global Impression of Change (PGIC) scale

    Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively

    range (1-7), lower scores mean a better outcome

  2. Oswestry Disability Index (ODI)

    Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively

    The ODI consists of 10 sections (items). Each section is scored on a scale of 0 to 5, with higher scores indicating greater levels of disability.

  3. Numeric Rating Scale (NRS)

    Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively

    rang 0 to 10, higher scores mean a worse outcome

  4. MMSE score

    Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively

    Mini-Mental State Examination (MMSE) : range: 0-30, higher scores mean a better outcome

  5. MoCA score

    Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively

    Montreal Cognitive Assessment (MoCA) score: range: 0-30, higher scores mean a better outcome.

  6. SDS

    Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively

    Self-Rating Depression Scale: Index score < 50 Normal range; 50-59 Mild depression; 60-69 Moderate depression; ≥70 Severe depression

  7. SAS

    Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively

    Self-Rating Anxiety Scale (SAS): 20-44 Normal range; 45-59 Mild to moderate anxiety levels; 60-74 Marked to severe anxiety levels; 75 and above Extreme anxiety levels

  8. SPRS score

    Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively

    The change in the Spastic Paraplegia Rating Scale (SPRS) score : range: 0-52, higher scores mean a worse outcome

  9. Complications

    Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively

    Catheter breakage, blockage, infection, etc.

  10. GMFM-88

    Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively

    Gross Motor Function Measure-88 (GMFM-88) score: range: 0-264, higher scores mean a better outcome

  11. GAS

    Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively

    Goal Attainment Scale (GAS) - Family Goal Selection Version: range: -2-+2, higher scores mean a better outcome

  12. Foot deformity molding

    Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively

    Utilizing CT digital 3D reconstruction technology

  13. Scoliosis assessment

    Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively

    Measurement of the Cobb angle using spinal X-ray imaging

Study contacts

Contact information is provided by the study sponsor or research team.

Li Cao, phD

CONTACT

[email protected]

+86 21 64369263

Wotu Tian, phD

CONTACT

[email protected]

+86 21 64369181

Sponsors and collaborators

Lead sponsor

Shanghai 6th People's Hospital

Other

Registry information

Official study title

Intrathecal Baclofen for the Management of Hereditary Spastic Paraparesis: a Prospective Cohort Study

Acronym: ITB-HSP

Important dates

Study start
2025
Primary completion
2028
Study completion
2028
First posted
Feb 25, 2025
Registry last updated
Feb 26, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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