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

September 1, 2025 updated by: Azidus Brasil

Dose-Response Evaluation of the Investigational Product Cellavita HD After Intravenous Administration in Patients With Huntington's Disease

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

49

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Brazil
    • São Paulo
      • Valinhos, São Paulo, Brazil, 13271-130
        • Azidus Brasil Pesquisa Científica e Desenvolvimento Ltda.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

17 years to 61 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cellavita HD Lower Dose
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).
Biological: Cellavita HD lower dose
The participants 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).
Other Names:
  • cellular therapy, mesenchymal stem cells
Experimental: Cellavita HD Higher Dose
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).
Biological: Cellavita HD higher dose
The participants 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).
Other Names:
  • cellular therapy, mesenchymal stem cells
Placebo Comparator: Placebo Group
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).
Other: Placebo
The participants will receive a total of 9 intravenous administrations of placebo divided into three administrations per cycle. Each administration will occur every 30 days and cycles every 120 days (total of 3 cycles).
Other Names:
  • physiological solution without cells

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Efficacy by UHDRS-TMS
Time Frame: monthly for eleven months
The primary endpoint was the rate of change (slope) in Unified Huntington's Disease Rating Scale - Total Motor Score from V0 to V11 (minimum 0, the best; and maximum 124, the worst). For each participant, a regression line was fitted (Y = a + bx), where b represents the individual slope, reflecting the annualised rate of motor progression. A negative slope suggests improvement or slower decline; a positive slope indicates worsening. To compare treatment efficacy, the slopes were analysed using a Mixed Model for Repeated Measures (MMRM), which accounts for intra-subject variability and missing data. This approach allowed estimation and comparison of average slopes between each treated group and placebo, providing a robust measure of motor function trajectory over time.
monthly for eleven months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Efficacy by UHDRS-TFC
Time Frame: monthly for eleven months

Assessed by the rate of change (slope) from V0 to V11 in the UHDRS-TFC.

The response of each participant or means of all participants was evaluated by the slopes of each regression line of UHDRS-TFC determined as follows:

The equation describes the regression line: Y = a + bx, where:

  • "Y" is the dependent variable.
  • "a" is the constant that represents the intersection of the line with the vertical axis.
  • "b" is the angular coefficient, that is, the slope.
  • "x" is the independent variable. Therefore, the 'b' value represents the slope of the straight line that best fits the values collected from the baseline (V0) to the value of V11, using the least squares method. This slope corresponds to the estimated annualized individual variation of UHDRS-TFC during the given period.
monthly for eleven months

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Exposure to NestaCell (Former Cellavita HD) Product
Time Frame: eleven months
Number of administrations of NestaCell (Former Cellavita HD) and Placebo
eleven months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Azidus Brasil

Collaborators

Cellavita Pesquisa Científica Ltda

Investigators

  • Principal Investigator: Joyce Macedo da Silva, MD, Azidus Brasil Scientific Research and Development Ltda

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 15, 2018

Primary Completion (Actual)

March 23, 2021

Study Completion (Actual)

April 30, 2021

Study Registration Dates

First Submitted

August 15, 2017

First Submitted That Met QC Criteria

August 16, 2017

First Posted (Actual)

August 17, 2017

Study Record Updates

Last Update Posted (Estimated)

September 19, 2025

Last Update Submitted That Met QC Criteria

September 1, 2025

Last Verified

September 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ADORE-DH
  • 52375916.1.0000.5412 (Other Identifier: CAAE)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

IPD Plan Description

It is believed that after the data analysis and presentation to the National Commission on Research Ethics, all data will become public.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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