A Prospective Cohort Study of ITB Treatment for HSP
ConditionHereditary Spastic Paraplegia
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age14-70
SponsorShanghai 6th People's Hospital
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
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
Trial 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.
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
50 Participants
are grouped into 2 trial groupsGroup A: Intrathecal baclofen management of hereditary spastic paraparesis
Group B: The oral baclofen group
Trial outcomes
Primary outcomes
1
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.
Time frame
From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively2
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.
Time frame
From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively3
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.
Time frame
From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively4
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.
Time frame
From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectivelySecondary outcomes
1
Patient Global Impression of Change (PGIC) scale
range (1-7), lower scores mean a better outcome
Time frame
From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively2
Oswestry Disability Index (ODI)
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.
Time frame
From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively3
Numeric Rating Scale (NRS)
rang 0 to 10, higher scores mean a worse outcome
Time frame
From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively4
MMSE score
Mini-Mental State Examination (MMSE) : range: 0-30, higher scores mean a better outcome
Time frame
From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectivelySponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov