Testing Miglustat Administration in Subjects With Spastic Paraplegia 11 (NCT04768166) | Clinical Trial Compass
CompletedPhase 2
Testing Miglustat Administration in Subjects With Spastic Paraplegia 11
Italy10 participantsStarted 2021-06-15
Plain-language summary
Hereditary spastic paraparesis type 11 (SPG11) is caused by mutations in the SPG11 gene that produces spatacsin, a protein involved in lysosomal function. Studies performed in skin cells (fibroblasts) from SPG11 patients, mice and zebrafish models of the disease showed that the material accumulated in the lysosomes is made of glycosphingolipids (GSL).
Miglustat is a drug that inhibits an enzyme called glucosylceramide synthetase (GCS) which is used for the production of GSL. Miglustat, therefore, helps to delay the production of GSL. This study aims to collect preliminary data on the safety of miglustat on the SPG11 disease and to assess biomarkers.
Who can participate
Age range
14 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Written signed informed consent;
* Confirmed diagnosis of SPG11;
* Age \> 13 years;
* SPRS score ≥ 10 or ≤35;
* Use of effective contraceptive methods and the performance of pregnancy tests (only fertile subjects).
Exclusion Criteria:
* Diagnosis of other concomitant neurodegenerative diseases;
* Outcomes of severe pre- or peri-natal suffering;
* Age ≤ 13 years;
* SPRS score ≥ 35 or ≤10;
* Hypersensitivity or intolerance to miglustat;
* Participation in other pharmacological studies within 30 days of the first Study visit (T0);
* The inability to take the drug;
* Any additional medical conditions;
* Subjects with severe renal impairment;
* Refusal to use effective contraceptive methods and the performance of pregnancy tests (only fertile subjects).
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
1-Changes from baseline blood tests at 24 weeks 2-Changes from baseline neurophysiological tests at 24 weeks 3-Report of severe adverse events