Status:
NOT_YET_RECRUITING
Efficacy and Safety of NestaCell® in Huntington's Disease
Lead Sponsor:
Azidus Brasil
Collaborating Sponsors:
Cellavita Pesquisa Científica Ltda
Conditions:
Huntington Disease
Eligibility:
All Genders
18-55 years
Phase:
PHASE3
Brief Summary
Huntington's disease (HD) is a rare neurodegenerative condition caused by increased CAG trinucleotide repeats in the HTT gene, on chromosome 4. The estimated global prevalence is 2.71 cases per 100,00...
Detailed Description
This is a Phase III multicenter, prospective, randomized, double-blind, placebo-controlled clinical trial to evaluate the efficacy and safety of Nestacell® in Huntington's Disease. After signing the ...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
June 8 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
February 9 2026
Estimated Enrollment :
120 Patients enrolled
Trial Details
Trial ID
NCT06097780
Start Date
June 8 2024
End Date
February 9 2026
Last Update
October 24 2023
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