A Phase 2/3 interventional study of Cellavita-HD in Huntington Disease, sponsored by Azidus Brasil. Status unknown at 1 site in Brazil. Open to participants aged 21 Years to 65 Years. Per ClinicalTrials.gov, last updated 2022-10-28.
Sponsored by Azidus Brasil · Phase 2/3, Interventional, and Treatment
Cellavita-HD is a stem-cell therapy for Huntington's Disease. Open label, single treatment, extension study for long-term safety and efficacy evaluation of Cellavita-HD intravenous administration in Huntington's disease patients who participated of ADORE-DH trial.
This extension study will include the subjects who participated the study phase II for dose-response evaluation of Cellavita-HD intravenous administration in Huntington's disease patients (maximum 35 subjects). Because it is a extension study, with an open scenario, the subjects will receive the maximum dose tested in the last clinical trial (ADORE-DH), corresponding to 6 x 10\^6 cells/weight range for each treatment cycle planned. Throughout of 24 months, the subjects will receive total dose planned divided into three administrations per cycle (2 x 10\^6 cells/weight range), that each will occur every 30 days. The maintaining the effectiveness of intravenous administration of Cellavita-HD product over time on clinical progression Huntington's disease will be verified by comparing the UHDRS total score at the end of the 24th study month against baseline score. Secondary evidences of efficacy will be evaluated by through the data of functional state, total functional capacity, functional independence, psychiatric symptoms and cognition from UHDRS scale. Additionally, related data to clinical worsening, change of Body Mass Index (BMI), risk of suicide attempt and neurological image improvement will be evaluated. Safety evaluation will included the incidence and classification of the adverse events experienced by the subjects during the study.
285 studies on the registry are indexed under Huntington Disease; 49 are open to participants now.
This study's planned enrollment of 35 is below the median of 40 across 203 interventional studies indexed under Huntington Disease.
Browse Huntington Disease studies →Azidus Brasil is the lead sponsor of 78 studies on the registry; 5 are open to participants now.
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The participants will receive a total of 12 intravenous administrations of 2x10\^6 cells/weight range divided into three administrations per cycle. Each administration will occur every 30 days and cycles every 180 days (total of 4 cycles).
Biological: Cellavita-HD
The participants will receive a total of 12 intravenous administrations of 2x10\^6 cells/weight range divided into three administrations per cycle. Each administration will occur every 30 days and cycles every 180 days (total of 4 cycles).
Also known as: cellular therapy, mesenchymal stem cells
Maintenance of effectiveness on clinical progression of the disease
The maintenance of treatment effectiveness will be verified by comparing the total UHDRS (Unified Huntington's Disease Rating Scale) score registered at the end of the 24th month of study in relation to the baseline score.
Time frame: Two years (every study visit)
Clinical neurological worsening over the treatment by UHDRS
The clinical neurological worsening over the treatment will be evaluated by specific Unified Huntington's Disease Rating Scale - UHDRS subscales (motor, behavior, functional capacity and cognitive domain). Each specific domain have 4 categories (from 0 to 4). For motor subscale, the score ranged from 0 to 124; Therefore,highest scores indicate more severe motor impairment. Cognitive operations are assessed by aplication of three tests: verbal fluency test, Symbol Digit modalities test and the Stroop Color Word test. For all cognitive tests, highest scores translate to better cognitive ability. Behavior assessment score is obtained by summing different items related to the severity of psychiatric symptoms. Highest scores indicate greater behavioral impairment. Functional assessment includes three scales: a functional checklist, an independence scale, and a total functional capacity assessment. The maximum score of 25 points represents greater functional capacity.
Time frame: Two years
BMI assessment
The BMI (Body Mass Index) will be evaluated through the BMI profiles obtained during the treatment to evaluate the nutritional status.
Time frame: Two years
Risk of suicidal ideation and/or behavior by C-SSRS
Will be evaluated by Columbia Suicide Severity Rating Scale (C-SSRS) registered throughout the study in relation to the baseline score. The total score ranges from 2 to 25, with a higher number indicating more intense ideation and greater risk. Greater lethality or potential lethality of the behavior (endorsed on the Behavior subscale) indicates increased risk.
Time frame: Two years
Risk of suicidal ideation and/or behavior by HDS
Will be evaluated through Suicidal Domain from HDS (Hamilton Depression Scale) registered throughout the study in relation to the baseline score. Scores above 25 points characterize severely ill patients depressed; scores between 18 and 24 points, moderately depressed patients; and scores between 7 and 17 points, patients with mild depression.
Time frame: Two years
CNS assessment by MRI (Magnetic Ressonance Imaging)
Huntington's patients normally present MRI structural abnormalities, including cortical atrophy and structural white matter changes. Alterations after product administration will be evaluated througout statistical comparison of the CNS assessment through magnetic resonance imaging at cortical thickness measurements, volumes of different brain structures, especially the basal ganglia, with special attention to caudate and metabolic changes identified in proton spectroscopy.
Time frame: One year
Safety of Cellavita-HD administration
Will be evaluated from the periodical assessments including clinical and imaging exams. Any clinical relevant change will be properly recorded and judged by Investigator according to expectation, causality, intesity and severity, as well as the continued treatment administration.
Time frame: Two years
Plan to share: Undecided — It is believed that after the data analysis and presentation to the National Commission on Research Ethics, all data will become public.
This study is status unknown, as verified in Oct 2022. You cannot join it, but the record below documents what was studied.
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Azidus Brasil