ClinicalTrials.gov record NCT06844734

A Prospective Cohort Study of ITB Treatment for HSP

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

Intrathecal Baclofen for the Management of Hereditary Spastic Paraparesis: a Prospective Cohort Study

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeObservationalA study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.
Age14 years old to 70 years old
SexAll
LocationChina
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This is a prospective, open-label cohort study observing people with hereditary spastic paraplegia who receive intrathecal baclofen (ITB) or oral baclofen. Fifty participants are planned and will be followed for three years with assessments about every six months. The study compares walking, spasticity, quality of life, complications, skeletal changes, and outcomes across subgroups.

What it is studying. The study is testing the effects and safety of continuous intrathecal baclofen compared with oral baclofen on walking, spasticity, quality of life, complications, skeletal changes, and differences between HSP subgroups.

What the researchers hope to learn. To gather clinical evidence on gait, motor function, spasticity, quality of life, pain, mood, cognition, and complications after ITB versus oral baclofen; to assess changes in skeletal deformities; to compare outcomes by genotype and by simple versus complex HSP; and to explore the optimal timing for ITB intervention.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

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.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This 3‑year prospective study enrolls 50 people: about 25 will receive intrathecal baclofen (surgical implantation of a pump) and about 25 will receive oral baclofen with natural history observation. People considering ITB will undergo an intrathecal baclofen test; those who decline surgery or do not respond to the test will be in the oral group. Both groups receive professional clinical assessments every six months up to 36 months, including walking tests (6‑minute walk), gait analysis (step length, walking speed, knee angle), spasticity and range‑of‑motion measurements, questionnaires on pain, mood and cognition (for example MMSE, MoCA, SDS, SAS), and imaging for foot deformity (CT 3D) and scoliosis (spinal X‑ray). The ITB group will be monitored for device‑related complications (for example catheter breakage, blockage, infection).

How the study is structured. Prospective observational cohort (open-label, single-center). Participants are observed in two groups: those who undergo ITB surgery and those who receive oral baclofen (natural history/control). Clinical assessments, gait measurements, questionnaires, imaging, and complication monitoring are done at baseline and about every six months for up to three years. A total of 50 participants are planned (about 25 in each group).

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
  • The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on hereditary spastic paraplegia

Clinical trials21 trials listed for hereditary spastic paraplegiaEvery trial the registry lists for hereditary spastic paraplegia, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in hereditary spastic paraplegia researchLatest monthly update: September 2026. Includes: SPTSSA treatment signal. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Jan 1, 2025
Primary completion
Mar 1, 2028 (estimated)
Study completion
Mar 1, 2028 (estimated)
Target enrollment
50 participants (estimated)
Last updated on registry
Feb 26, 2025
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Shanghai 6th People's Hospital
Principal investigator
Li Cao, Professor
Shanghai 6th People's Hospital

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

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

    Shanghai, Shanghai Municipality, 213000 · China

Terms used on this page · explanations by MedicalUpdates.ai

prospective cohort
A group of people who share a characteristic, such as a diagnosis, and are studied together. 'Prospective' means they are followed forward from enrolment; 'retrospective' means existing records are reviewed.
intrathecal
Given by injection into the fluid around the spinal cord, usually through a lumbar puncture.
life expectancy
Some studies require that, in the investigator's judgement, a participant is likely to live long enough to complete the study period. It is a study requirement, not a prediction about any individual.
control group
The group the study treatment is compared with. It may receive a placebo, an existing treatment (active comparator), a sham procedure, or no intervention.
study drug
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
clinical trial
A study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
open-label
No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
natural history
A study that follows how a condition changes over time without assigning an experimental treatment. It may involve study visits and tests, and is often used to plan future trials.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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