Dose-response Evaluation of the Cellavita HD Product in Patients With Huntington's Disease (ADORE-DH)

Azidus

Status and phase: Completed; Phase 2

Conditions: Huntington Disease

Treatments: Biological: Cellavita HD higher dose; Biological: Cellavita HD lower dose; Other: Placebo

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT03252535

ADORE-DH

52375916.1.0000.5412 (Other Identifier)

Details and patient eligibility

About

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.

Full 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.

Enrollment: 35 patients

Sex: All

Ages: 21 to 65 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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35 participants in 3 patient groups, including a placebo group

Cellavita HD Lower Dose

Experimental group

Description:

The participants randomized to this group will receive a total of 9 intravenous administrations of 1x10\^6 cells/weight range divided into three administrations per cycle. Each administration will occur every 30 days and cycles every 120 days (total of 3 cycles).

Treatment:

Biological: Cellavita HD lower dose

Cellavita HD Higher Dose

Experimental group

Description:

The participants randomized to this group will receive a total of 9 intravenous administrations of 2x10\^6 cells/weight range divided into three administrations per cycle. Each administration will occur every 30 days and cycles every 120 days (total of 3 cycles).

Treatment:

Biological: Cellavita HD higher dose

Placebo Group

Placebo Comparator group

Description:

The participants randomized to this group will receive a total of 9 intravenous administrations divided into three administrations per cycle. Each administration will occur every 30 days and cycles every 120 days (total of 3 cycles).

Treatment:

Other: Placebo