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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04219241
Recruitment Status : Not yet recruiting
First Posted : January 7, 2020
Last Update Posted : January 13, 2020
Sponsor:
Collaborator:
Cellavita Pesquisa Científica Ltda
Information provided by (Responsible Party):
Azidus Brasil

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.

Condition or disease Intervention/treatment Phase
Huntington Disease Biological: Cellavita-HD Phase 2 Phase 3

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 35 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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.
Estimated Study Start Date : January 2020
Estimated Primary Completion Date : January 2022
Estimated Study Completion Date : April 2022

Resource links provided by the National Library of Medicine


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 Name: cellular therapy, mesenchymal stem cells




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.


Other Outcome Measures:
  1. 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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   21 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04219241


Contacts
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Contact: Joyce Macedo, MD +551938296160 joyce.macedo@azidusbrasil.com.br
Contact: Regina Doi, MD +551938296160 regina@azidusbrasil.com.br

Locations
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Brazil
Azidus Brasil Pesquisa Científica e Desenvolvimento Ltda.
Valinhos, São Paulo, Brazil, 13271-130
Contact: Joyce Macedo da Silva, MD    551938296160    joyce.macedo@azidusbrasil.com.br   
Contact: Regina Mayumi Doi, MD    551938296160    regina@azidusbrasil.com.br   
Principal Investigator: Joyce Macedo da Silva, MD         
Sponsors and Collaborators
Azidus Brasil
Cellavita Pesquisa Científica Ltda
Investigators
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Principal Investigator: Joyce Macedo, MD Azidus Brasil Scientific Research and Development Ltda
Publications:

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Responsible Party: Azidus Brasil
ClinicalTrials.gov Identifier: NCT04219241    
Other Study ID Numbers: ADORE-EXT
23917319.0.0000.5412 ( Registry Identifier: CAAE )
First Posted: January 7, 2020    Key Record Dates
Last Update Posted: January 13, 2020
Last Verified: January 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Plan Description: It is believed that after the data analysis and presentation to the National Commission on Research Ethics, all data will become public.

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Azidus Brasil:
Huntington Disease
Stem cell therapy
Dental Pulp Stem Cell
Additional relevant MeSH terms:
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Huntington Disease
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Dementia
Chorea
Dyskinesias
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Cognition Disorders
Neurocognitive Disorders
Mental Disorders