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Trial record 13 of 175 for:    Huntington's Disease

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

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ClinicalTrials.gov Identifier: NCT03252535
Recruitment Status : Not yet recruiting
First Posted : August 17, 2017
Last Update Posted : August 17, 2017
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. 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.

Condition or disease Intervention/treatment Phase
Huntington Disease Biological: Cellavita HD lower dose Biological: Cellavita HD higher dose Other: Placebo Phase 2

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/kg), G2: higher dose (2x10^6 cells/kg) 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. 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 include the incidence and classification of the adverse events experienced by the subjects during the study.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 35 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Masking Description: The study drugs will be provided in identical packages to maintain the study masking. Neither the Investigator nor the study team will know which drug the subject is receiving. In addition, the external outcome evaluator will receive the results in a codified manner (concealed).
Primary Purpose: Treatment
Official Title: Dose-Response Evaluation of the Investigational Product Cellavita HD After Intravenous Administration in Patients With Huntington's Disease
Estimated Study Start Date : April 15, 2018
Estimated Primary Completion Date : April 15, 2020
Estimated Study Completion Date : September 30, 2020

Resource links provided by the National Library of Medicine


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/kg body weight 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/kg body weight divided into three administrations per cycle. Each administration will occur every 30 days and cycles every 120 days (total of 3 cycles).
Other Name: 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/kg body weight 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/kg body weight divided into three administrations per cycle. Each administration will occur every 30 days and cycles every 120 days (total of 3 cycles).
Other Name: 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 Name: Cellavita HD product without cells




Primary Outcome Measures :
  1. Effective Dose [ Time Frame: one year ]
    Consists of identifying the dose of the product Cellavita HD providing the best clinical response.


Secondary Outcome Measures :
  1. Improvement of UHDRS domains [ Time Frame: one year ]
    Will evaluate of the functional state, total functional capacity, functional independence, psychiatric symptoms and cognition through UHDRS scale before and throught the study.

  2. Clinical worsing over the treatment [ Time Frame: one year ]
    The clinical worsing over the treatment will be evaluated by specific UHDRS domain.

  3. BMI assessment [ Time Frame: one year ]
    The BMI (Body Mass Index) will be assessed through the BMI profiles obtained during the treatment.

  4. Risk of suicidal ideation [ Time Frame: one year ]
    Will be evaluated by suicidal domain from Hamilton Depression Scale (HAM-D). The classificatory punctuation may correspond to mild depression (score: 8 to 13), moderate depression (score: 19 - 22) and severe depression (score: > 23).

  5. CNS assessment [ Time Frame: one year ]
    Will be evaluated by statistical comparison of the CNS assessment through magnetic resonance image 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 administration of Cellavita HD product [ Time Frame: approximately two years ]
    Will be carefully evaluated from the periodical assessments including clinical, laboratory, and imaging exams, so that any change is properly recorded.

  2. Prognosis of Huntington Disease [ Time Frame: one year ]
    This parameter will be evaluated by statistical comparison of NF-L (biological marker) results observed at baseline period and other analysed times.



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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. 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.

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): NCT03252535


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

Sponsors and Collaborators
Azidus Brasil
Cellavita Pesquisa Científica Ltda
Investigators
Study Director: Luciana Ferrara, CEO Azidus Brasil Pesquisa Científica e Desenvolvimento Ltda

Responsible Party: Azidus Brasil
ClinicalTrials.gov Identifier: NCT03252535     History of Changes
Other Study ID Numbers: ADORE-DH
52375916.1.0000.5412 ( Other Identifier: CAAE )
First Posted: August 17, 2017    Key Record Dates
Last Update Posted: August 17, 2017
Last Verified: August 2017
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.

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:
Huntington Disease
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Cognition Disorders
Neurocognitive Disorders
Mental Disorders
Dementia
Chorea
Dyskinesias