Clinical trial NCT03252535: Dose-response Evaluation of the Cellavita HD Product in Patients With Huntington's Disease
Official title: Dose-Response Evaluation of the Investigational Product Cellavita HD After Intravenous Administration in Patients With Huntington's Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
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: 2017-08-17
Enrollment: 35
Sex: ALL
Minimum age (years): 21
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Cellavita HD is a stem-cell therapy for Huntington's Disease. This is a prospective, phase II, single-center, randomized (2:2:1), triple-blind, placebo controlled study, with two test doses of Cellavita HD product.

Detailed description: This is a phase II dose-response study in which participants with HD will receive three intravenous injections of the investigational product or placebo (one every month for three months) a total of three cycles. The subjects will be randomized in 2: 2: 1 ratio for the groups G1: lower dose (1x10\^6 cells/weight range), G2: higher dose (2x10\^6 cells/weight range) or G3: placebo. To identify the dose of the product that will provide the best clinical response, motor assessment will be performed with UHDRS scale and improvement will be evaluated by correlating before and after treatment scores. Additionally, also will be performed the combined score through the cUHDRS. Secondary evidences of efficacy will be evaluated 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. Provide a written, signed and dated Informed Consent Form;
2. Male and female subjects aged ≥ 21 and ≤ 65 years;
3. Have a confirmatory diagnosis report (PCR) of Huntington's disease with a number of CAG repeats in chromosome 4 higher than or equal to 40, and lower than or equal to 50 (if the subject did not perform the exam and/or if he/she does not have an available result for this exam, a new exam must be performed);
4. A score of 5 points or higher for the motor evaluation of the UHDRS scale (Unified Huntington's Disease Rating Scale) at enrollment;
5. Score of 8 to 11 points for the functional capacity of the UHDRS scale at enrollment.

Exclusion Criteria:

1. Subject who participated in clinical trials protocols within the last twelve (12) months (Resolution CNS 251, August 7, 1997, item III, subitem J), unless, at the investigator's opinion, the subject would have a direct benefit from it;
2. Diagnosis of juvenile Huntington's disease;
3. Diagnosis of epilepsy;
4. Diagnosis of major cognitive disorder;
5. Active decompensated psychiatric illness;
6. Current or prior history of neoplasm;
7. Current history of gastrointestinal, hepatic, renal, endocrine, pulmonary, hematological, immunological, metabolic pathology or severe uncontrolled cardiovascular diseases;
8. Diagnosis of any active infection, whether viral, bacterial, fungal or caused by another pathogen;
9. Subject with contraindication to the exams performed in this study, for example, with pacemaker or surgical clip; Alcohol and drugs abuse (previously diagnosed according to the Diagnostic and Statistical Manual of Mental Disorders - DSM V criteria);
10. Use of illegal drugs;
11. Tabagism;
12. Smoker or quit smoking for less than 6 months;
13. Positive result in one of the serum tests: HIV 1 and 2 (Anti-HIV-1,2), HTLV I and II, HBV (HBsAg, Anti-HBc), HCV (anti-HCV-Ab) and FTA-ABS (Treponema pallidum);
14. History of drug allergy, including to contrast agents used in imaging tests or bovine-derived products;
15. Using or expects to use immunosuppressant drugs or forbidden drugs (item 5.3) during the first three months after the first administration of the investigational product;
16. Any clinical change that the investigator considers a risk to subject's enrollment in the study.

Primary outcomes:
measure: Effective Dose
timeFrame: monthly for fourteen months
Secondary outcomes:
measure: Clinical neurological worsening over the treatment
timeFrame: monthly for fourteen months
measure: BMI assessment
timeFrame: monthly for fourteen months
measure: Risk of suicidal ideation
timeFrame: monthly for fourteen months
measure: CNS assessment
timeFrame: baseline and one year later
measure: Clinical Interview Based impression of Severity (CIBIS)
timeFrame: monthly for fourteen months
Other outcomes:
measure: Safety administration of Cellavita HD product
timeFrame: monthly for fourteen months
measure: Prognosis of Huntington Disease
timeFrame: baseline and one year later
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 da Silva, MD
role: PRINCIPAL_INVESTIGATOR
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