Ignite Creation Date:
2025-12-25 @ 2:20 AM
Ignite Modification Date:
2025-12-27 @ 11:08 PM
Study NCT ID:
NCT06844734
Status:
RECRUITING
Last Update Posted:
2025-02-26
First Post:
2025-02-10
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Prospective Cohort Study of ITB Treatment for HSP
Sponsor:
Shanghai 6th People's Hospital
Organization:
Study Overview
Official Title:
Intrathecal Baclofen for the Management of Hereditary Spastic Paraparesis: a Prospective Cohort Study
Status:
RECRUITING
Status Verified Date:
2025-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
ITB-HSP
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.
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.
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?: