A Prospective Cohort Study of ITB Treatment for HSP

NCT ID: NCT06844734

Last Updated: 2025-02-26

Basic Information

• Recruitment Status: RECRUITING
• Total Enrollment: 50 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2025-01-01
• Study Completion Date: 2028-03-01

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. 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.

Detailed Description

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.

Conditions

Hereditary Spastic Paraplegia

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

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.

No interventions assigned to this group

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.

No interventions assigned to this group

Eligibility Criteria

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

• Minimum Eligible Age: 14 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Shanghai 6th People's Hospital

OTHER

Sponsor Role: lead

Responsible Party

Li Cao

Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Li Cao, phD

Role: PRINCIPAL_INVESTIGATOR

Shanghai Jiao Tong University Affiliated Sixth People's Hospital

Locations

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

Shanghai, Shanghai Municipality, China

Site Status: RECRUITING

Countries

China

Central Contacts

Li Cao, phD

Role: CONTACT

caoli2000@yeah.net

+86 21 64369263

Wotu Tian, phD

Role: CONTACT

wotu_tian@163.com

+86 21 64369181

Facility Contacts

Wotu Tian, phD

Role: primary

wotu_tian@163.com

21 64369181

Other Identifiers

2024-KY-305(K)

Identifier Type: -

Identifier Source: org_study_id