EUCTR2019-002827-14-IT
Active, not recruiting
Phase 1
Phase 2 pharmacological experimental study to test the safety of miglustat in subjects with hereditary spastic paraplegia type 11 - TreatSPG11
ConditionsSpastic paraparesis type 11 (SPG11), caused by mutations in spatacsin protein, is characterized by the association of peripheral neuropathy, parkinsonism, ataxia, cognitive impairment, hypotrophy of the corpus callosum and retinal degeneration. The disease progresses with degeneration of motor and cognitive functions up to total dependence on caregivers. At the moment there are no therapies capable of modifying its natural history and progression.MedDRA version: 20.0Level: PTClassification code 10019903Term: Hereditary spastic paraplegiaSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Spastic paraparesis type 11 (SPG11), caused by mutations in spatacsin protein, is characterized by the association of peripheral neuropathy, parkinsonism, ataxia, cognitive impairment, hypotrophy of the corpus callosum and retinal degeneration. The disease progresses with degeneration of motor and cognitive functions up to total dependence on caregivers. At the moment there are no therapies capable of modifying its natural history and progression.
- Sponsor
- IRCCS Fondazione Stella Maris
- Enrollment
- 10
- Status
- Active, not recruiting
- Last Updated
- 4 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Diagnosis of SPG11 confirmed by genetic analysis positive for biallelic mutations in the SPG11 gene.
- •Age \> 13 years.
- •Moderate degree of disease severity (SPRS disease scale \> 10 and \< 35\).
- •Signature of informed consent for enrollment in the study.
- •Acceptance of the use of contraceptive methods (for subjects of childbearing age).
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range:
- •F.1\.2 Adults (18\-64 years) yes
- •F.1\.2\.1 Number of subjects for this age range 4
- •F.1\.3 Elderly (\>\=65 years) no
Exclusion Criteria
- •Diagnosis of other concomitant neurodegenerative disease.
- •Very severe (SPRS \= 35 scale) or mild / paucisymptomatic (SPRS \=10\) SPG11 disease.
- •Outcomes of severe pre\- / peri\-natal suffering.
- •Sensitivity or intolerance to miglustat.
- •Participation in other pharmacological studies within 30 days of the first visit of this study (T0\).
- •Inability to take the study drug due to severe swallowing problems or the presence of percutaneous endoscopic gastrostomy access.
- •Other medical conditions that may interfere with the study results or the safety of the participants.
- •Intractable diarrhea in the 3 months prior to enrollment.
Outcomes
Primary Outcomes
Not specified
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