Huntington DiseasePhase 1SAVE-DHNCT02728115
Safety Evaluation of Cellavita HD Administered Intravenously in Participants With Huntington's Disease
A Phase 1 study testing Cellavita HD Lower Dose and Cellavita HD Higher dose in people with Huntington Disease, run by Azidus Brasil at 1 site in Brazil.
Trial summary
Cellavita HD is a stem-cell therapy for Huntington's Disease. This is a first-in-human, non-randomized, phase I study in which participants with Huntington's Disease will receive three intravenous injections and will be followed for 5 years to evaluate safety and tolearability of product and preliminary evidence of… Read more about this study
- Who is it for?
- Ages 21 to 65 years, men only
- Conditions the study is for
- Huntington Disease
- What is being tested?
- Cellavita HD Lower Dose and Cellavita HD Higher dose
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteBrazil
- Study dates
- 16 Oct 2017to 31 Dec 2023
- Phase
- Phase 1
- 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
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Sign and date ICF;
- Ability to follow instructions as well as ability to understand and fulfill the study requirements correctly;
- Male participant aged ≥ 21 and ≤ 65;
- Participants who submit medical report (PCR) attesting Huntington's disease with a number of CAG repeats on chromosome 4, greater than or equal to 40 and less than or equal to 50 (if the participant has not performed the examination and/or if he does not have the report available, a new exam should be done);
- Score 5 points or more in motor assessment UHDRS scale (Unified Huntington's Disease Rating Scale) at the time of enrollment;
- Score between 8 and 11 points in the functional capacity of the UHDRS scale at the time of enrollment.
Who cannot join
- Participation within 12 months in any clinical trial;
- Any medical observation data (clinical and physical) that medical research judge as a risk for subject if enrollment at the study;
- Any laboratory exam data that medical research judge as a risk for subject if enrollment at the study;
- Juvenile Huntington disease diagnosis;
- History of epilepsy;
- Diagnostic of major cognitive impairment;
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- Active decompensated psychiatric disease;
- Current or prior history of neoplasia;
- Current history of gastrointestinal, hepatic, renal, endocrine, pulmonary, hematologic, immune, metabolic pathology or severe and uncontrolled cardiovascular disease;
- Diagnostic of any active infection, be it viral, bacterial, fungal, or caused by another pathogen;
- Participants who have contraindication to undergo any of the tests performed in this study, for example, have pacemakers or surgical clip;
- History of alcohol or illegal drugs abusers;
- History of 1 or more episodes of suicide in the two years before Visit V-4;
- Active smoker or have stopped smoking less than six months prior to enrollment;
- Test positive in at least one of the serological tests: HIV 1 and 2 (Anti-HIV-1,2), HTLV I and II, HBV (HBsAg, anti-HBc), HCV (anti-HCV-Ab) and VDRL (Treponema pallidum);
- History of drug allergy, including contrasts for imaging, or bovine products;
- In use or expected use of immunosuppressive drugs or prohibited medicines for the first three months after the first administration of the investigational product;
- Any clinical changes that is interpreted by the medical researcher as a risk to participant's enrollment.
Trial locations and contacts
1 site in 1 country.
Brazil1 site
Azidus Brasil Pesquisa Científica e Desenvolvimento Ltda.
Valinhos, São Paulo, 13271-130
About this study
Official title: First in Human Study to Evaluate Safety of Cellavita HD Investigational Product After Intravenous Application in Participants With Huntington's Disease
Cellavita HD is a stem-cell therapy for Huntington's Disease. This is a first-in-human, non-randomized, phase I study in which participants with Huntington's Disease will receive three intravenous injections and will be followed for 5 years to evaluate safety and tolearability of product and preliminary evidence of effectiveness.
Read the detailed description
This is a first-in-human, non-randomized, phase I study in which participants with HD will receive three intravenous injections of one of two doses of the investigational product, one every month for three months. Safety evaluation data will be composed by the register of adverse events (including type, frequency, intensity, seriousness, severity, and action taken related to the investigational product), could be include changes in vital signs, physical and medical evaluations, laboratory or serology tests and electrocardiogram (ECG), and by the incidence of benign and malign neoplasms. Preliminary evidence of efficacy will be evaluated by global clinical improvement (CIBIS) and evolution of disease improvement (motor, cognitive and behavioral degradation) through Unified Huntington's Disease Rating Scale - UHDRS and inflammatory markers: IL-4, IL-6, IL-10 (interleukin IL) e TNF-alpha (tumoral necrosis factor alpha). CNS improvement will be assessed by magnetic resonance imaging (MRI). Fluctuation in suicide tendency grade will be evaluated by Hamilton Depression Rating Scale (HDRS).The immunological response of HD product over the administration period will be evaluated by CD4+ and CD8+ proliferation and inflammatory markers release.
Participants who show evidence of loss of clinical benefit achieved over the course of treatment verified through worsening greater or equal to that expected for the natural course of the disease on motor, cognitive, behavioral and functional capacity symptoms assessed by the UHDRS scale, will receive additional doses of the product as long as there is clinical benefit at the Investigator's discretion and/or until the product is marketed. The same dose used by the subject during the treatment period will be administered throughout the period of additional doses.
- Conditions
- Huntington Disease
- Being tested
- Cellavita HD Lower Dose, Cellavita HD Higher dose
- Keywords
- Huntington's Disease, Stem Cell, Dental Pulp Stem Cells
- 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 HD Lower DoseBiological
Also called cellular therapy, mesenchymal stem cells
The first three participants enrolled in the study will be assigned to the lower dose arm with staggered treatment, with an interval of 30 days between the first administration of the first participant and the first administration of the second participant assigned to this arm. All participants will receive a total of 3 intravenous administration, one every 30 days.
Cellavita HD Higher doseBiological
Also called cellular therapy, mesenchymal stem cells
The last three participants enrolled in the study will be assigned to the higher dose arm with staggered treatment, with an interval of 30 days between the first administration of the first participant and the first administration of the second participant assigned to this arm. All participants will receive a total of 3 intravenous administration, one every 30 days.
| Study group | Role | What they receive |
|---|---|---|
| Cellavita HD Lower Dose | Experimental | Participants assigned to this arm will receive 3 administrations, one every 30 days, of 1x10^6 cells/weight range per administration of Cellavita HD (n= 3) . Receives: Biological: Cellavita HD Lower Dose |
| Cellavita HD Higher dose | Experimental | Participants assigned to this arm will receive 3 administrations, one every 30 days, of 2x10^6 cells/weight range per administration of Cellavita HD (n= 3). Receives: Biological: Cellavita HD Higher dose |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of Cellavita HD by periodic monitoring changes at adverse events, vital signs, laboratory tests, ECG and incidence of benign and malignant neoplasms
The safety of the investigational product will be evaluated in detail from periodic evaluations contemplating monitoring changes of:
- Adverse events including type, frequency, intensity, seriousness, severity, and action taken related to the investigational product study;
- Vital signs (BP, HR, axillary temperature), physical and medical examination (BMI, weight, height, medical condition - cardiovascular, pulmonary, digestive, musculoskeletal and peripheral, with emphasis on the neurological assessment and others);
- Laboratory tests included hematologic, biochemical, urologic and serological analysis;
- Electrocardiogram (ECG) of 12 derivations;
- Incidence and classification of benign and malignant neoplasms in the following organs/systems: CNS, lung, liver, spleen, pancreas, prostate, testicle, urinary, hematological and skeletal system through the laboratory tests, magnetic resonance imaging, computerized tomography and ultrasonography.
Measured: first year and in the following 4 years
Secondary outcomes (5)
Preliminary efficacy of Cellavita HD by UHDRS improvement and global clinical response (CIBIS)
Will be evaluated by statistical comparison of the results of each UHDRS scale component: motor, cognitive and behavior. The global clinical response will be assessed by statistical comparison between baseline score observed by the Investigator before and after Cellavita HD treatment.
Measured: first year and in the following 4 years
Preliminary efficacy of Cellavita HD by comparison of the inflammatory markers
Will be evaluated by statistical comparison of the inflammatory markers included IL-4, IL-6, IL-10 (interleukin IL) and TNF-alpha (tumoral necrosis factor alpha).
Measured: first year
Immunological Response of Cellavita HD
The immunological response induced by Cellavita HD will be evaluated by statistical comparison between baseline results of CD4+ and CD8+ proliferation and the other evaluated times.
Measured: first year
Preliminary efficacy of Cellavita HD by comparison of the CNS assessment
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.
Measured: first year
Risk of suicidal ideation by Hamilton Depression Rating Scale (HDRS)
Will be evaluated by suicidal domain. The classificatory pontuation may correspond to mild depression (score: 8 to 13), moderate depression (score: 19 - 22) and severe depression (score: > 23).
Measured: first year and in the following 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Apr 2016 (estimated)
- Study start16 Oct 2017
- Primary completion31 Dec 2022 (estimated)
- Study completion31 Dec 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (8)Papers the sponsor has linked to this study.
- Other trials for Huntington DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 1
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Azidus Brasil
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Cellavita Pesquisa Científica Ltda, Azidus Brasil Scientific Research and Development Ltda
- Responsible party
- Sponsor
- Investigators
- Joyce Macedo da Silva, MD, Principal investigator, Azidus Brasil Scientific Research and Development Ltda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02728115
- Sponsor's protocol ID
- SAVE-DH
- Other IDs
- 51005115.9.0000.5412
- Acronym
- SAVE-DH
- Status last verified
- Nov 2022
- First submitted
- 11 Mar 2016
- Last updated
- 2 Nov 2022
- Results posted
- No
- Data monitoring committee
- Yes
- 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 of the study will become public.
Publications
Adam OR, Jankovic J. Symptomatic treatment of Huntington disease. Neurotherapeutics. 2008 Apr;5(2):181-97. doi: 10.1016/j.nurt.2008.01.008.
PubMed 18394562Aleynik 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 25097727de Almeida FM, Marques SA, Ramalho Bdos S, Rodrigues RF, Cadilhe DV, Furtado D, Kerkis I, Pereira LV, Rehen SK, Martinez AM. Human dental pulp cells: a new source of cell therapy in a mouse model of compressive spinal cord injury. J Neurotrauma. 2011 Sep;28(9):1939-49. doi: 10.1089/neu.2010.1317. Epub 2011 Aug 8.
PubMed 21609310Bachoud-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 16545746Bachoud-Levi AC, Remy P, Nguyen JP, Brugieres P, Lefaucheur JP, Bourdet C, Baudic S, Gaura V, Maison P, Haddad B, Boisse MF, Grandmougin T, Jeny R, Bartolomeo P, Dalla Barba G, Degos JD, Lisovoski F, Ergis AM, Pailhous E, Cesaro P, Hantraye P, Peschanski M. Motor and cognitive improvements in patients with Huntington's disease after neural transplantation. Lancet. 2000 Dec 9;356(9246):1975-9. doi: 10.1016/s0140-6736(00)03310-9.
PubMed 11130527Ball LM, Bernardo ME, Roelofs H, van Tol MJ, Contoli B, Zwaginga JJ, Avanzini MA, Conforti A, Bertaina A, Giorgiani G, Jol-van der Zijde CM, Zecca M, Le Blanc K, Frassoni F, Egeler RM, Fibbe WE, Lankester AC, Locatelli F. Multiple infusions of mesenchymal stromal cells induce sustained remission in children with steroid-refractory, grade III-IV acute graft-versus-host disease. Br J Haematol. 2013 Nov;163(4):501-9. doi: 10.1111/bjh.12545. Epub 2013 Aug 31.
PubMed 23992039Barker 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 23345280Fernandes JMS, Pagani E, Wenceslau CV, Ynoue LH, Ferrara L, Kerkis I. A phase I, open-label study of intravenous human dental pulp stem cells (NestaCell(R)) at two dose levels in patients with Huntington's disease. Stem Cell Res Ther. 2025 Nov 4;16(1):611. doi: 10.1186/s13287-025-04703-w.
PubMed 41188963
Study at a glance
From the registry record, last updated by the sponsor on 2 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- First in Human Study to Evaluate Safety of Cellavita HD Investigational Product After Intravenous Application in Participants With Huntington's Disease
- ClinicalTrials.gov ID
- NCT02728115
- Acronym / sponsor ID
- SAVE-DH / SAVE-DH
- Conditions
- Huntington Disease
- Keywords
- Huntington's Disease, Stem Cell, Dental Pulp Stem Cells
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Azidus BrasilIndustry (pharma, biotech, devices)
- Collaborators
- Cellavita Pesquisa Científica Ltda, Azidus Brasil Scientific Research and Development Ltda
- Study start
- 16 Oct 2017 (actual)
- Primary completion
- 31 Dec 2022 (estimated)
- Study completion
- 31 Dec 2023 (estimated)
- First posted
- 5 Apr 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Cellavita HD is a stem-cell therapy for Huntington's Disease. This is a first-in-human, non-randomized, phase I study in which participants with Huntington's Disease will receive three intravenous injections and will be followed for 5 years to evaluate safety and tolearability of product and preliminary evidence of effectiveness.
Detailed description
This is a first-in-human, non-randomized, phase I study in which participants with HD will receive three intravenous injections of one of two doses of the investigational product, one every month for three months. Safety evaluation data will be composed by the register of adverse events (including type, frequency, intensity, seriousness, severity, and action taken related to the investigational product), could be include changes in vital signs, physical and medical evaluations, laboratory or serology tests and electrocardiogram (ECG), and by the incidence of benign and malign neoplasms. Preliminary evidence of efficacy will be evaluated by global clinical improvement (CIBIS) and evolution of disease improvement (motor, cognitive and behavioral degradation) through Unified Huntington's Disease Rating Scale - UHDRS and inflammatory markers: IL-4, IL-6, IL-10 (interleukin IL) e TNF-alpha (tumoral necrosis factor alpha). CNS improvement will be assessed by magnetic resonance imaging (MRI). Fluctuation in suicide tendency grade will be evaluated by Hamilton Depression Rating Scale (HDRS).The immunological response of HD product over the administration period will be evaluated by CD4+ and CD8+ proliferation and inflammatory markers release.
Participants who show evidence of loss of clinical benefit achieved over the course of treatment verified through worsening greater or equal to that expected for the natural course of the disease on motor, cognitive, behavioral and functional capacity symptoms assessed by the UHDRS scale, will receive additional doses of the product as long as there is clinical benefit at the Investigator's discretion and/or until the product is marketed. The same dose used by the subject during the treatment period will be administered throughout the period of additional doses.
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cellavita HD Lower Dose | Experimental | Biological: Cellavita HD Lower DoseParticipants assigned to this arm will receive 3 administrations, one every 30 days, of 1x10^6 cells/weight range per administration of Cellavita HD (n= 3) . |
| Cellavita HD Higher dose | Experimental | Biological: Cellavita HD Higher doseParticipants assigned to this arm will receive 3 administrations, one every 30 days, of 2x10^6 cells/weight range per administration of Cellavita HD (n= 3). |
Who may qualify
- Ages
- 21 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Sign and date ICF;
- Ability to follow instructions as well as ability to understand and fulfill the study requirements correctly;
- Male participant aged ≥ 21 and ≤ 65;
- Participants who submit medical report (PCR) attesting Huntington's disease with a number of CAG repeats on chromosome 4, greater than or equal to 40 and less than or equal to 50 (if the participant has not performed the examination and/or if he does not have the report available, a new exam should be done);
- Score 5 points or more in motor assessment UHDRS scale (Unified Huntington's Disease Rating Scale) at the time of enrollment;
Key exclusion criteria (18 listed)
- Participation within 12 months in any clinical trial;
- Any medical observation data (clinical and physical) that medical research judge as a risk for subject if enrollment at the study;
- Any laboratory exam data that medical research judge as a risk for subject if enrollment at the study;
- Juvenile Huntington disease diagnosis;
- History of epilepsy;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Cellavita HD Lower DoseAlso: cellular therapy, mesenchymal stem cellsThe first three participants enrolled in the study will be assigned to the lower dose arm with staggered treatment, with an interval of 30 days between the first administration of the first participant and the first administration of the second participant assigned to this arm. All participants will receive a total of 3 intravenous administration, one every 30 days. | Biological | Cellavita HD Lower Dose |
| Cellavita HD Higher doseAlso: cellular therapy, mesenchymal stem cellsThe last three participants enrolled in the study will be assigned to the higher dose arm with staggered treatment, with an interval of 30 days between the first administration of the first participant and the first administration of the second participant assigned to this arm. All participants will receive a total of 3 intravenous administration, one every 30 days. | Biological | Cellavita HD Higher dose |
Primary outcome
Safety of Cellavita HD by periodic monitoring changes at adverse events, vital signs, laboratory tests, ECG and incidence of benign and malignant neoplasms
Time frame: first year and in the following 4 years
The safety of the investigational product will be evaluated in detail from periodic evaluations contemplating monitoring changes of: * Adverse events including type, frequency, intensity, seriousness, severity, and action taken related to the investigational product study; * Vital signs (BP, HR, axillary temperature), physical and medical examination (BMI, weight, height, medical condition - cardiovascular, pulmonary, digestive, musculoskeletal and peripheral, with emphasis on the neurological assessment and others); * Laboratory tests included hematologic, biochemical, urologic and serological analysis; * Electrocardiogram (ECG) of 12 derivations; * Incidence and classification of benign and malignant neoplasms in the following organs/systems: CNS, lung, liver, spleen, pancreas, prostate, testicle, urinary, hematological and skeletal system through the laboratory tests, magnetic resonance imaging, computerized tomography and ultrasonography.
5 secondary outcomes
Preliminary efficacy of Cellavita HD by UHDRS improvement and global clinical response (CIBIS)
Time frame: first year and in the following 4 years
Preliminary efficacy of Cellavita HD by comparison of the inflammatory markers
Time frame: first year
Immunological Response of Cellavita HD
Time frame: first year
Preliminary efficacy of Cellavita HD by comparison of the CNS assessment
Time frame: first year
Risk of suicidal ideation by Hamilton Depression Rating Scale (HDRS)
Time frame: first year and in the following 4 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Nov 2022.
- Background: PubMed 18394562Adam OR, Jankovic J. Symptomatic treatment of Huntington disease. Neurotherapeutics. 2008 Apr;5(2):181-97. doi: 10.1016/j.nurt.2008.01.008.
- Background: PubMed 25097727Aleynik 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.
- Background: PubMed 21609310de Almeida FM, Marques SA, Ramalho Bdos S, Rodrigues RF, Cadilhe DV, Furtado D, Kerkis I, Pereira LV, Rehen SK, Martinez AM. Human dental pulp cells: a new source of cell therapy in a mouse model of compressive spinal cord injury. J Neurotrauma. 2011 Sep;28(9):1939-49. doi: 10.1089/neu.2010.1317. Epub 2011 Aug 8.
- Background: PubMed 16545746Bachoud-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.
- Background: PubMed 11130527Bachoud-Levi AC, Remy P, Nguyen JP, Brugieres P, Lefaucheur JP, Bourdet C, Baudic S, Gaura V, Maison P, Haddad B, Boisse MF, Grandmougin T, Jeny R, Bartolomeo P, Dalla Barba G, Degos JD, Lisovoski F, Ergis AM, Pailhous E, Cesaro P, Hantraye P, Peschanski M. Motor and cognitive improvements in patients with Huntington's disease after neural transplantation. Lancet. 2000 Dec 9;356(9246):1975-9. doi: 10.1016/s0140-6736(00)03310-9.
- Background: PubMed 23992039Ball LM, Bernardo ME, Roelofs H, van Tol MJ, Contoli B, Zwaginga JJ, Avanzini MA, Conforti A, Bertaina A, Giorgiani G, Jol-van der Zijde CM, Zecca M, Le Blanc K, Frassoni F, Egeler RM, Fibbe WE, Lankester AC, Locatelli F. Multiple infusions of mesenchymal stromal cells induce sustained remission in children with steroid-refractory, grade III-IV acute graft-versus-host disease. Br J Haematol. 2013 Nov;163(4):501-9. doi: 10.1111/bjh.12545. Epub 2013 Aug 31.
- Background: PubMed 23345280Barker 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.
- Derived from this study: PubMed 41188963Fernandes JMS, Pagani E, Wenceslau CV, Ynoue LH, Ferrara L, Kerkis I. A phase I, open-label study of intravenous human dental pulp stem cells (NestaCell(R)) at two dose levels in patients with Huntington's disease. Stem Cell Res Ther. 2025 Nov 4;16(1):611. doi: 10.1186/s13287-025-04703-w.
- Individual participant dataSharing planned: Undecided. It is believed that after the data analysis and presentation to the National Commission on Research Ethics, all data of the study will become public.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Joyce Macedo da Silva, MD principal investigator
Azidus Brasil Scientific Research and Development Ltda
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Cellavita HD Lower Dose and Cellavita HD Higher dose 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 6 inclusion and 18 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 2 Nov 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 phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Safety of Cellavita HD by periodic monitoring changes at adverse events, vital signs, laboratory tests, ECG and incidence of benign and malignant neoplasms (time frame: first year and in the following 4 years). 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 NCT02728115, which also lists 8 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 2 Nov 2022.