Clinical trial NCT04219241: Clinical Extension Study for Safety and Efficacy Evaluation of Cellavita-HD Administration in Huntington's Patients.
Official title: Clinical Extension Study for Assessing the Safety and Efficacy of the Intravenous Administration of Cellavita-HD in Huntington's Disease Patients Who Participated in the ADORE-DH Study.
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
PHASE3
Conditions: Huntington Disease
Keywords: Huntington Disease
Stem cell therapy
Dental Pulp Stem Cell
Lead sponsor: Azidus Brasil
Sponsor class: INDUSTRY
Collaborators: name: Cellavita Pesquisa Científica Ltda
First posted: 2020-01-07
Enrollment: 35
Sex: ALL
Minimum age (years): 21
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

1. Phase II study participants (ADORE-DH) who express interest in participating in the extension period and who may have significant clinical benefits (motor, cognitive, behavioral and functional capacity) at the Investigator's judged after intravenous application of the Cellavita-HD product;
2. Provide consent by signing in two copies of the Informed Consent Form;
3. Participant using an acceptable contraceptive method.

Exclusion Criteria:

1. History of malignant neoplasia;
2. Present any clinical and laboratory condition or comorbidity that, at the physician's judged, may endanger the health of the research participant and prevent him / her from being part of the extension study;
3. known hypersensitivity to the investigational product and / or products of bovine origin;
4. Research participant who has participated in clinical trial protocols in the last twelve (12) months, unless the Investigator believes that there may be direct benefit to it (Resolution CNS 251 of August 7, 1997, item III, sub-item J);
5. Pregnant or lactating participant;
6. In use or anticipated use of immunosuppressive drugs as well as any other drug in which the use is restricted to this research protocol.

Primary outcomes:
measure: Maintenance of effectiveness on clinical progression of the disease
timeFrame: Two years (every study visit)
Secondary outcomes:
measure: Clinical neurological worsening over the treatment by UHDRS
timeFrame: Two years
measure: BMI assessment
timeFrame: Two years
measure: Risk of suicidal ideation and/or behavior by C-SSRS
timeFrame: Two years
measure: Risk of suicidal ideation and/or behavior by HDS
timeFrame: Two years
measure: CNS assessment by MRI (Magnetic Ressonance Imaging)
timeFrame: One year
Other outcomes:
measure: Safety of Cellavita-HD administration
timeFrame: Two years
Locations:
city: Valinhos
country: Brazil
facility: Azidus Brasil Pesquisa Científica e Desenvolvimento Ltda.
state: São Paulo
Study officials:
affiliation: Azidus Brasil Scientific Research and Development Ltda
name: Joyce Macedo, MD
role: PRINCIPAL_INVESTIGATOR
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