Phase 2 pharmacological experimental study to test the safety of miglustat in subjects with hereditary spastic paraplegia type 11 - TreatSPG11

IRCCS Fondazione Stella Maris0 个研究点目标入组 10 人开始时间: 2021年5月24日最近更新: 2022年3月14日

适应症

试验速览

暂无简介。

•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
•F.1.3.1 Number of subjects for this age range

•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.

发起方