Hereditary Spastic ParaplegiaITB-HSPNCT06844734
A Prospective Cohort Study of ITB Treatment for HSP
An observational study of Hereditary Spastic Paraplegia, run by Shanghai 6th People's Hospital at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 14 to 70 years
- Conditions the study is for
- Hereditary Spastic Paraplegia
- Type of treatment
- Pill or oral medicine
- How many people
- 50 participants
- Where
- 1 siteChina
- Study dates
- 1 Jan 2025to 1 Mar 2028
- How long will I take part?
- 3 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 14 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Shanghai Sixth People's Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, 213000
Wotu Tian, phD, 21 64369181, wotu_tian@163.com
About this study
Official title: Intrathecal Baclofen for the Management of Hereditary Spastic Paraparesis: a Prospective Cohort Study
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:
- Changes in gait and motor function, as well as spasticity levels, compared to pre-surgery and control group after ITB surgery.
- Changes in quality of life, pain, psychological and emotional status, and cognition compared to pre-surgery and control group after ITB surgery.
- Complications following ITB surgery.
- Impact of ITB surgery on the occurrence and progression of skeletal deformities.
- Subgroup analysis: comparing surgical outcomes between different genotypes and between simple versus complex types.
- Determine the optimal timing for ITB intervention.
Read the 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
- Keywords
- Hereditary Spastic Paraparesis, Intrathecal baclofen
- Medical subject headings
- Spastic Paraplegia, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
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
range (1-7), lower scores mean a better outcome
Measured: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
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.
Measured: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
Numeric Rating Scale (NRS)
rang 0 to 10, higher scores mean a worse outcome
Measured: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
MMSE score
Mini-Mental State Examination (MMSE) : range: 0-30, higher scores mean a better outcome
Measured: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
MoCA score
Montreal Cognitive Assessment (MoCA) score: range: 0-30, higher scores mean a better outcome.
Measured: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
SDS
Self-Rating Depression Scale: Index score < 50 Normal range; 50-59 Mild depression; 60-69 Moderate depression; ≥70 Severe depression
Measured: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
SAS
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
Measured: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
SPRS score
The change in the Spastic Paraplegia Rating Scale (SPRS) score : range: 0-52, higher scores mean a worse outcome
Measured: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
Complications
Catheter breakage, blockage, infection, etc.
Measured: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
GMFM-88
Gross Motor Function Measure-88 (GMFM-88) score: range: 0-264, higher scores mean a better outcome
Measured: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
GAS
Goal Attainment Scale (GAS) - Family Goal Selection Version: range: -2-+2, higher scores mean a better outcome
Measured: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
Foot deformity molding
Utilizing CT digital 3D reconstruction technology
Measured: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
Scoliosis assessment
Measurement of the Cobb angle using spinal X-ray imaging
Measured: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Feb 2025
- Study start1 Jan 2025
- Primary completion1 Mar 2028 (estimated)
- Study completion1 Mar 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary Spastic ParaplegiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 50 participants (estimated)
- Target duration
- 3 Years
Sponsor and investigators
- Lead sponsor
- Shanghai 6th People's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Li Cao, Shanghai 6th People's Hospital
- Investigators
- Li Cao, phD, Principal investigator, Shanghai Jiao Tong University Affiliated Sixth People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06844734
- Sponsor's protocol ID
- 2024-KY-305(K)
- Acronym
- ITB-HSP
- Status last verified
- Jan 2025
- First submitted
- 10 Feb 2025
- Last updated
- 26 Feb 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Intrathecal Baclofen for the Management of Hereditary Spastic Paraparesis: a Prospective Cohort Study
- ClinicalTrials.gov ID
- NCT06844734
- Acronym / sponsor ID
- ITB-HSP / 2024-KY-305(K)
- Conditions
- Hereditary Spastic Paraplegia
- Keywords
- Hereditary Spastic Paraparesis, Intrathecal baclofen
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Shanghai 6th People's HospitalUniversity, hospital or other
- Study start
- 1 Jan 2025 (actual)
- Primary completion
- 1 Mar 2028 (estimated)
- Study completion
- 1 Mar 2028 (estimated)
- First posted
- 25 Feb 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- Changes in gait and motor function, as well as spasticity levels, compared to pre-surgery and control group after ITB surgery.
- Changes in quality of life, pain, psychological and emotional status, and cognition compared to pre-surgery and control group after ITB surgery.
- Complications following ITB surgery.
- Impact of ITB surgery on the occurrence and progression of skeletal deformities.
- Subgroup analysis: comparing surgical outcomes between different genotypes and between simple versus complex types.
- 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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 3 Years
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Ages
- 14 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
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.
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.
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.
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.
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.
13 secondary outcomes
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
Oswestry Disability Index (ODI)
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
Numeric Rating Scale (NRS)
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
MMSE score
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
MoCA score
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
SDS
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
SAS
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
SPRS score
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
Complications
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
GMFM-88
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
GAS
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
Foot deformity molding
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
Scoliosis assessment
Time frame: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Feb 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Li Cao, phD principal investigator
Shanghai Jiao Tong University Affiliated Sixth People's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hereditary Spastic Paraplegia, run by Shanghai 6th People's Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 26 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Li Cao, phD (+86 21 64369263, caoli2000@yeah.net), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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 study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Feb 2025.