Hereditary Spastic ParaplegiaEarly Phase 1CFT-SPG56NCT06478238
Calcium Folinate Treatment of Spastic Paraplegia 56
A Early Phase 1 study testing calcium folinate in people with Hereditary Spastic Paraplegia, run by Shanghai 6th People's Hospital at 1 site in China.
Trial summary
SPG56 is one of the complicated and early-onset HSP subtypes caused by genetic mutations in CYP2U1. So far, there is no standardized and specific clinical therapy for SPG56. The goal of this clinical trial is to explore the efficacy and safety of calcium folinate in the treatment of SPG56 patients. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hereditary Spastic Paraplegia
- What is being tested?
- calcium folinate
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteChina
- Study dates
- 1 Jul 2024to 31 May 2030
- Phase
- Early Phase 1
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients meet the clinical diagnostic standard of hereditary spastic paraplegia (HSP);
- Spastic paraplegia type 56 (SPG56) was diagnosed by CYP2U1 pathogenic mutation;
- 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 drugs involved in the study;
- 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 6th People's Hospita
Shanghai, Shanghai Municipality
Shankai Yin, +8664369181, slxck@126.com
About this study
Official title: A Prospective Single Arm Clinical Trial of Calcium Folinate in the Treatment of Spastic Paraplegia 56
SPG56 is one of the complicated and early-onset HSP subtypes caused by genetic mutations in CYP2U1. So far, there is no standardized and specific clinical therapy for SPG56. The goal of this clinical trial is to explore the efficacy and safety of calcium folinate in the treatment of SPG56 patients.
This study is prospective, open-label and single arm and this trial will last for 6 years. A total of 10 patients will participate and they will receive calcium folinate treatment and professional clinical evaluation regularly.
- Conditions
- Hereditary Spastic Paraplegia
- Being tested
- calcium folinate
- 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.
calcium folinateDrug
Intravenous infusion and/or oral therapy
| Study group | Role | What they receive |
|---|---|---|
| calcium folinate treatment group | Experimental | Drug: calcium folinate Phase I: calcium folinate infusion intravenously for 5 consecutive days at a dose of 1mg/kg/day in two divided doses per day. Then it was changed to oral administration at a dose of 2mg/kg/day during hospitalization.
Phase II: long-term oral medication at a dose of 2mg/ kg/day in two daily doses. Receives: Drug: calcium folinate |
What is being measured
The primary outcome is the main question the study is designed to answer.
GMFM-88
The change in the Gross Motor Function Measure-88 (GMFM-88) score from baseline (range: 0-264, higher scores mean a better outcome).
Measured: At the end of the 5-year follow-up period
Secondary outcomes (7)
SPRS score
The change in the Spastic Paraplegia Rating Scale (SPRS) score from baseline (range: 0-52, higher scores mean a worse outcome).
Measured: At the end of the 5-year follow-up period
MMSE score
The change in the Mini-Mental State Examination (MMSE) score from baseline (range: 0-30, higher scores mean a better outcome).
Measured: At the end of the 5-year follow-up period
Laboratory indicators
The change in the Laboratory indicators (blood biochemistry, lipid metabolism, folate, etc) and the number of participants with abnormal laboratory indicators.
Measured: At the end of the 5-year follow-up period
Cranial CT/MRI
The change in the cranial CT/MRI from baseline.
Measured: At the end of the 5-year follow-up period
Gait examination
The change in the gait examination from baseline.
Measured: At the end of the 5-year follow-up period
MoCA score
The change in the Montreal Cognitive Assessment (MoCA) score from baseline (range: 0-30, higher scores mean a better outcome).
Measured: At the end of the 5-year follow-up period
High density electroencephalogram
The change in the high density electroencephalogram from baseline.
Measured: At the end of the 5-year follow-up period
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jun 2024
- Study start1 Jul 2024 (estimated)
- Primary completion31 May 2030 (estimated)
- Study completion31 May 2030 (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
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
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
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06478238
- Sponsor's protocol ID
- 2024-058-(1)
- Acronym
- CFT-SPG56
- Status last verified
- Jun 2024
- First submitted
- 8 Jun 2024
- Last updated
- 27 Jun 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 27 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Single Arm Clinical Trial of Calcium Folinate in the Treatment of Spastic Paraplegia 56
- ClinicalTrials.gov ID
- NCT06478238
- Acronym / sponsor ID
- CFT-SPG56 / 2024-058-(1)
- Conditions
- Hereditary Spastic Paraplegia
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Shanghai 6th People's HospitalUniversity, hospital or other
- Study start
- 1 Jul 2024 (estimated)
- Primary completion
- 31 May 2030 (estimated)
- Study completion
- 31 May 2030 (estimated)
- First posted
- 27 Jun 2024 (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.
SPG56 is one of the complicated and early-onset HSP subtypes caused by genetic mutations in CYP2U1. So far, there is no standardized and specific clinical therapy for SPG56. The goal of this clinical trial is to explore the efficacy and safety of calcium folinate in the treatment of SPG56 patients.
This study is prospective, open-label and single arm and this trial will last for 6 years. A total of 10 patients will participate and they will receive calcium folinate treatment and professional clinical evaluation regularly.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| calcium folinate treatment group | Experimental | Drug: calcium folinateDrug: calcium folinate Phase I: calcium folinate infusion intravenously for 5 consecutive days at a dose of 1mg/kg/day in two divided doses per day. Then it was changed to oral administration at a dose of 2mg/kg/day during hospitalization. Phase II: long-term oral medication at a dose of 2mg/ kg/day in two daily doses. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Patients meet the clinical diagnostic standard of hereditary spastic paraplegia (HSP);
- Spastic paraplegia type 56 (SPG56) was diagnosed by CYP2U1 pathogenic mutation;
- 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 drugs involved in the study;
- 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.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| calcium folinateIntravenous infusion and/or oral therapy | Drug | calcium folinate treatment group |
Primary outcome
GMFM-88
Time frame: At the end of the 5-year follow-up period
The change in the Gross Motor Function Measure-88 (GMFM-88) score from baseline (range: 0-264, higher scores mean a better outcome).
7 secondary outcomes
SPRS score
Time frame: At the end of the 5-year follow-up period
MMSE score
Time frame: At the end of the 5-year follow-up period
Laboratory indicators
Time frame: At the end of the 5-year follow-up period
Cranial CT/MRI
Time frame: At the end of the 5-year follow-up period
Gait examination
Time frame: At the end of the 5-year follow-up period
MoCA score
Time frame: At the end of the 5-year follow-up period
High density electroencephalogram
Time frame: At the end of the 5-year follow-up period
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 27 Jun 2024.
- Individual participant dataSharing planned: Undecided
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing calcium folinate in people with 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 3 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 27 Jun 2024. 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.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
GMFM-88 (time frame: At the end of the 5-year follow-up period). 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 27 Jun 2024.