Status:
COMPLETED
Testing Miglustat Administration in Subjects With Spastic Paraplegia 11
Lead Sponsor:
IRCCS Fondazione Stella Maris
Conditions:
Hereditary Spastic Paraparesis
Eligibility:
All Genders
14+ years
Phase:
PHASE2
Brief 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)...
Detailed Description
We will analyze the safety of Miglustat
Eligibility Criteria
Inclusion
- 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
- 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).
Key Trial Info
Start Date :
June 15 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 15 2021
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT04768166
Start Date
June 15 2021
End Date
September 15 2021
Last Update
April 11 2022
Active Locations (1)
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1
IRCCS Fondazione Stella Maris
Pisa, PI, Italy, 56128