Efficacy and Safety of NestaCell® in Huntington's Disease (STAR)
Huntington Disease
About this trial
This is an interventional treatment trial for Huntington Disease focused on measuring STAR, NestaCell ®
Eligibility Criteria
Inclusion Criteria: Male or female; Age from 18 to 55 years; HD diagnostic confidence level (DCL) score of 3 or 4 at enrolment; HD manifestations begin from 4 to 8 years before enrolment; UHDRS Total Functional Capacity (TFC) from 7 to 12, suggesting mild-moderate functional impairment; Body weight at the V -1 from 50 to 90 Kg; CAG repeats from 40 to 50; ICF signature. Exclusion Criteria: Juvenile Huntington's disease, Concomitant epilepsy; Decompensated psychiatric disorders; History of a suicide attempt; Other neurological or musculoskeletal disorders that might interfere with the assessments; Prior use of gene or cell therapy. Confirmed or suspected cancer within the last 1 year (except operated basal cell carcinoma); History of allergy to imaging exams contrast, or bovine origin products; Current or planned use of immunosuppressants; Clinically significant changes in the safety exams, defined as; Serum transaminases (ALT, AST) increased > 2.5 × upper limit of normality (ULN). Absolute neutrophil count in peripheral blood < 3,000 cells/1 mm3. Serum creatinine > 2 × age- and sex-specific ULN. Positive serology for HIV 1 and 2 (Anti-HIV-1,2), HTLV I and II, HBV (HBsAg, Anti-HBc), HCV (anti-HCV-Ab), and FTA-ABS. Amylase, Troponin I, CKmB increased > 2.0 × ULN. Malignancy shown by the Total-Body PET Scan. Glycated hemoglobin > 6.5%. aPTT, TT, platelets > 2.5 x ULN. Pregnancy, lactation, or pregnancy plan; BMI less than 18.5 at enrolment; Participation in a clinical trial within twelve months before inclusion; History of surgical procedures aiming at improving symptoms of Huntington's disease, such as neural transplantations, lesions of the central nervous system, infusions of neurotrophic agents, or deep brain stimulation. Any medical condition that makes the patient unsuitable for the study or increases the risk of participation at the investigator's discretion.
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Placebo Comparator
Experimental
Placebo
Intravenous NestaCell® administrations
Arm 1: Nine intravenous placebo administrations in twelve months.
Arm 2: Nine intravenous NestaCell® administrations in twelve months.