Testing Miglustat Administration in Subjects With Spastic Paraplegia 11

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-06-15

Study Completion Date

2021-09-15

Brief Summary

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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.

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Detailed Description

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We will analyze the safety of Miglustat

Conditions

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Hereditary Spastic Paraparesis

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

BASIC_SCIENCE

Blinding Strategy

NONE

Study Groups

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Evaluate the safety of Miglustat administration in subjects with Spastic Paraplegia 11

100 mg of Miglustat, 3 caps per day for first 4 weeks; 100 mg of Miglustat, 6 caps per day for 8 weeks

Group Type EXPERIMENTAL

Miglustat 100 MG

Intervention Type DRUG

100mg/TID in 4w then 200mg/TID in 8 w

Interventions

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Miglustat 100 MG

100mg/TID in 4w then 200mg/TID in 8 w

Intervention Type DRUG

Other Intervention Names

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Genorph

Eligibility Criteria

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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).

Minimum Eligible Age

14 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No