A Prospective Cohort Study of ITB Treatment for HSP

NCT06844734 | Recruiting

• 1 other identifier: 2024-KY-305(K)
• Study Type: observational
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Hereditary Spastic Paraplegia

Keywords

Hereditary Spastic Paraparesis; Intrathecal baclofen

Outcome Measures

Primary Outcomes (6)

• Six-Minute Walk Test(6-MWT)

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

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

• Step length

  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.

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

• walking speed

  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.

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

• knee flexion angle

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

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

• Modified Ashworth Scale(MAS)

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

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

• Range of Motion (ROM) of Joints

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

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

Secondary Outcomes (13)

• Patient Global Impression of Change (PGIC) scale

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

• Oswestry Disability Index (ODI)

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

• Numeric Rating Scale (NRS)

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

• MMSE score

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

• MoCA score

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

Study Arms (2)

Intrathecal baclofen management of hereditary spastic paraparesis

Patients with hereditary spastic paraparesis aged 14 to 70 years, with a Modified Ashworth Scale score of greater or equal to 3 in 2 or more joints of lower limb, and agree to undergo Intrathecal baclofen surgery will be recruited. Patients will receive professional assessment every six months.

The oral baclofen group

Patients who do not agree to intrathecal baclofen treatment or those who do not achieve satisfactory therapeutic effect during the baclofen testing phase will be included in oral baclofen group. Patients will receive professional assessment every six months, with natural history observation.

Eligibility Criteria

• Age: 14 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients meet the clinical and genetic diagnostic criteria of hereditary spastic paraplegia (HSP) between 14 to 70 years old with of lower limbs. Participants suffered from severe spasticity with score ≥ 3 by Modified Ashworth assessment in ≥2 joints of lower limbs. Patients agree to participate in clinical trials and able to understand and comply with the research program.

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Shanghai 6th People's Hospital: lead

Study Sites (1)

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

Shanghai, Shanghai Municipality, 213000, China

RECRUITING

MeSH Terms

Conditions

Spastic Paraplegia, Hereditary

Condition Hierarchy (Ancestors)

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

Study Officials

• Li Cao, phD

  Shanghai Jiao Tong University Affiliated Sixth People's Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Li Cao, phD

CONTACT

+86 21 64369263; caoli2000@yeah.net

Wotu Tian, phD

CONTACT

+86 21 64369181; wotu_tian@163.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 3 Years
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: February 10, 2025
• First Posted: February 25, 2025
• Study Start: January 1, 2025
• Primary Completion (Estimated): March 1, 2028
• Study Completion (Estimated): March 1, 2028
• Last Updated: February 26, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)