AzTrials
AzTrials

Huntington DiseasePhase 2/3ADORE-EXTNCT04219241

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

A Phase 2/3 study testing Cellavita-HD in people with Huntington Disease, run by Azidus Brasil at 1 site in Brazil.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages 21 to 65 years
Conditions the study is for
Huntington Disease
What is being tested?
Cellavita-HD
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
35 participants
Where
1 siteBrazil
Study dates
10 Feb 2020to Dec 2022
Phase
Phase 2/3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 21 to 65 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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;
  • Provide consent by signing in two copies of the Informed Consent Form;
  • Participant using an acceptable contraceptive method.

Who cannot join

  • History of malignant neoplasia;
  • 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;
  • known hypersensitivity to the investigational product and / or products of bovine origin;
  • 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);
  • Pregnant or lactating participant;
  • In use or anticipated use of immunosuppressive drugs as well as any other drug in which the use is restricted to this research protocol.

Trial locations and contacts

1 site in 1 country.

Brazil1 site

  • Azidus Brasil Pesquisa Científica e Desenvolvimento Ltda.

    Valinhos, São Paulo, 13271-130

Other trials in Brazil

About this study

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.

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.

Read the 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.

Being tested
Cellavita-HD
Keywords
Huntington Disease, Stem cell therapy, Dental Pulp Stem Cell
Medical subject headings
Huntington Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Cellavita-HDBiological

    Also called cellular therapy, mesenchymal stem cells

    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).

Study groups in NCT04219241
Study groupRoleWhat they receive
Cellavita-HDExperimentalThe 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).
Receives: Biological: Cellavita-HD

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Maintenance of effectiveness on clinical progression of the disease

    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.

    Measured: Two years (every study visit)

Secondary outcomes (5)

  1. Clinical neurological worsening over the treatment by UHDRS

    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.

    Measured: Two years

  2. BMI assessment

    The BMI (Body Mass Index) will be evaluated through the BMI profiles obtained during the treatment to evaluate the nutritional status.

    Measured: Two years

  3. Risk of suicidal ideation and/or behavior by C-SSRS

    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.

    Measured: Two years

  4. Risk of suicidal ideation and/or behavior by HDS

    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.

    Measured: Two years

  5. CNS assessment by MRI (Magnetic Ressonance Imaging)

    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.

    Measured: One year

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted7 Jan 2020
  2. Study start10 Feb 2020
  3. Primary completionDec 2022 (estimated)
  4. Study completionDec 2022 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2/3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
35 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04219241
Sponsor's protocol ID
ADORE-EXT
Other IDs
23917319.0.0000.5412
Acronym
ADORE-EXT
Status last verified
Oct 2022
First submitted
11 Dec 2019
Last updated
28 Oct 2022
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Undecided: It is believed that after the data analysis and presentation to the National Commission on Research Ethics, all data will become public.

Publications

  • Adachi N, Numakawa T, Richards M, Nakajima S, Kunugi H. New insight in expression, transport, and secretion of brain-derived neurotrophic factor: Implications in brain-related diseases. World J Biol Chem. 2014 Nov 26;5(4):409-28. doi: 10.4331/wjbc.v5.i4.409.

    PubMed 25426265

  • Aleynik A, Gernavage KM, Mourad YSh, Sherman LS, Liu K, Gubenko YA, Rameshwar P. Stem cell delivery of therapies for brain disorders. Clin Transl Med. 2014 Jul 19;3:24. doi: 10.1186/2001-1326-3-24. eCollection 2014.

    PubMed 25097727

  • Bachoud-Levi AC, Gaura V, Brugieres P, Lefaucheur JP, Boisse MF, Maison P, Baudic S, Ribeiro MJ, Bourdet C, Remy P, Cesaro P, Hantraye P, Peschanski M. Effect of fetal neural transplants in patients with Huntington's disease 6 years after surgery: a long-term follow-up study. Lancet Neurol. 2006 Apr;5(4):303-9. doi: 10.1016/S1474-4422(06)70381-7.

    PubMed 16545746

  • Barker RA, Mason SL, Harrower TP, Swain RA, Ho AK, Sahakian BJ, Mathur R, Elneil S, Thornton S, Hurrelbrink C, Armstrong RJ, Tyers P, Smith E, Carpenter A, Piccini P, Tai YF, Brooks DJ, Pavese N, Watts C, Pickard JD, Rosser AE, Dunnett SB; NEST-UK collaboration. The long-term safety and efficacy of bilateral transplantation of human fetal striatal tissue in patients with mild to moderate Huntington's disease. J Neurol Neurosurg Psychiatry. 2013 Jun;84(6):657-65. doi: 10.1136/jnnp-2012-302441. Epub 2013 Jan 23.

    PubMed 23345280

  • Goldberg YP, Kremer B, Andrew SE, Theilmann J, Graham RK, Squitieri F, Telenius H, Adam S, Sajoo A, Starr E, et al. Molecular analysis of new mutations for Huntington's disease: intermediate alleles and sex of origin effects. Nat Genet. 1993 Oct;5(2):174-9. doi: 10.1038/ng1093-174.

    PubMed 8252043

  • Unified Huntington's Disease Rating Scale: reliability and consistency. Huntington Study Group. Mov Disord. 1996 Mar;11(2):136-42. doi: 10.1002/mds.870110204.

    PubMed 8684382

  • Jia JM, Chen Q, Zhou Y, Miao S, Zheng J, Zhang C, Xiong ZQ. Brain-derived neurotrophic factor-tropomyosin-related kinase B signaling contributes to activity-dependent changes in synaptic proteins. J Biol Chem. 2008 Jul 25;283(30):21242-50. doi: 10.1074/jbc.M800282200. Epub 2008 May 12.

    PubMed 18474605

  • Kerkis I, Haddad MS, Valverde CW, Glosman S. Neural and mesenchymal stem cells in animal models of Huntington's disease: past experiences and future challenges. Stem Cell Res Ther. 2015 Dec 14;6:232. doi: 10.1186/s13287-015-0248-1.

    PubMed 26667114

  • Marder K, Zhao H, Myers RH, Cudkowicz M, Kayson E, Kieburtz K, Orme C, Paulsen J, Penney JB Jr, Siemers E, Shoulson I. Rate of functional decline in Huntington's disease. Huntington Study Group. Neurology. 2000 Jan 25;54(2):452-8. doi: 10.1212/wnl.54.2.452.

    PubMed 10668713

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Cellavita-HD in people with Huntington Disease, run by Azidus Brasil at 1 site in Brazil.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 28 Oct 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Brazil. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Maintenance of effectiveness on clinical progression of the disease (time frame: Two years (every study visit)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04219241, which also lists 9 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Oct 2022.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in