Clinical Extension Study for Safety and Efficacy Evaluation of Cellavita-HD Administration in Huntington's Patients. (ADORE-EXT)

October 26, 2022 updated by: Azidus Brasil

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.

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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Anticipated)

35

Phase

  • Phase 2
  • Phase 3

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

21 years to 65 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

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: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 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).
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).
Other Names:
  • cellular therapy, mesenchymal stem cells

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Maintenance of effectiveness on clinical progression of the disease
Time Frame: Two years (every study visit)
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.
Two years (every study visit)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Clinical neurological worsening over the treatment by UHDRS
Time Frame: Two years
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.
Two years
BMI assessment
Time Frame: Two years
The BMI (Body Mass Index) will be evaluated through the BMI profiles obtained during the treatment to evaluate the nutritional status.
Two years
Risk of suicidal ideation and/or behavior by C-SSRS
Time Frame: Two years
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.
Two years
Risk of suicidal ideation and/or behavior by HDS
Time Frame: Two years
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.
Two years
CNS assessment by MRI (Magnetic Ressonance Imaging)
Time Frame: One year
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.
One year

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Safety of Cellavita-HD administration
Time Frame: Two years
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.
Two years

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, 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)

February 10, 2020

Primary Completion (Anticipated)

December 1, 2022

Study Completion (Anticipated)

December 1, 2022

Study Registration Dates

First Submitted

December 11, 2019

First Submitted That Met QC Criteria

January 3, 2020

First Posted (Actual)

January 7, 2020

Study Record Updates

Last Update Posted (Actual)

October 28, 2022

Last Update Submitted That Met QC Criteria

October 26, 2022

Last Verified

October 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ADORE-EXT
  • 23917319.0.0000.5412 (Registry 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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