Huntington DiseasePhase 3STARNCT06097780
Efficacy and Safety of NestaCell® in Huntington's Disease
A Phase 3 study testing NestaCell® in people with Huntington Disease, run by Azidus Brasil.
Trial summary
Huntington's disease (HD) is a rare neurodegenerative condition caused by increased CAG trinucleotide repeats in the HTT gene, on chromosome 4. The estimated global prevalence is 2.71 cases per 100,000 inhabitants. Read more about this study
- Who is it for?
- Ages 18 to 55 years
- Conditions the study is for
- Huntington Disease
- What is being tested?
- NestaCell®
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 120 participants
- Where
- No sites listed yet
- Study dates
- 8 Jun 2024to 9 Feb 2026
- Phase
- Phase 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 18 to 55 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Male or female;
- Age from 18 to 55 years;
- HD diagnostic confidence level (DCL) score of 3 or 4 at enrolment;
- HD manifestations begin from 4 to 8 years before enrolment;
- UHDRS Total Functional Capacity (TFC) from 7 to 12, suggesting mild-moderate functional impairment;
- Body weight at the V -1 from 50 to 90 Kg;
- CAG repeats from 40 to 50;
- ICF signature.
Who cannot join
- Juvenile Huntington's disease,
- Concomitant epilepsy;
- Decompensated psychiatric disorders;
- History of a suicide attempt;
- Other neurological or musculoskeletal disorders that might interfere with the assessments;
- Prior use of gene or cell therapy.
Show all 23Show fewer
- Confirmed or suspected cancer within the last 1 year (except operated basal cell carcinoma);
- History of allergy to imaging exams contrast, or bovine origin products;
- Current or planned use of immunosuppressants;
- Clinically significant changes in the safety exams, defined as;
- Serum transaminases (ALT, AST) increased > 2.5 × upper limit of normality (ULN).
- Absolute neutrophil count in peripheral blood < 3,000 cells/1 mm3.
- Serum creatinine > 2 × age- and sex-specific ULN.
- Positive serology for HIV 1 and 2 (Anti-HIV-1,2), HTLV I and II, HBV (HBsAg, Anti-HBc), HCV (anti-HCV-Ab), and FTA-ABS.
- Amylase, Troponin I, CKmB increased > 2.0 × ULN.
- Malignancy shown by the Total-Body PET Scan.
- Glycated hemoglobin > 6.5%.
- aPTT, TT, platelets > 2.5 x ULN.
- Pregnancy, lactation, or pregnancy plan;
- BMI less than 18.5 at enrolment;
- Participation in a clinical trial within twelve months before inclusion;
- History of surgical procedures aiming at improving symptoms of Huntington's disease, such as neural transplantations, lesions of the central nervous system, infusions of neurotrophic agents, or deep brain stimulation.
- Any medical condition that makes the patient unsuitable for the study or increases the risk of participation at the investigator's discretion.
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Phase III Efficacy and Safety of NestaCell® in Moderated Huntington's Disease
Huntington's disease (HD) is a rare neurodegenerative condition caused by increased CAG trinucleotide repeats in the HTT gene, on chromosome 4. The estimated global prevalence is 2.71 cases per 100,000 inhabitants. In Brazil, it is estimated that 13,000 to 19,000 people carry the gene and 65,000 to 95,000 are descendants at risk.
HD usually manifests itself in the fourth decade of life with motor, cognitive and behavioral symptoms, such as chorea. This condition profoundly affects quality of life and there is no treatment that can modify its course. Tetrabenazine is the only medication approved to control chorea.
Read more
A partnership between the Butantan Institute and Cellavita investigates the use of Human Dental Pulp Stem Cells (hDPSCs) to treat HD. NestaCell® was developed, a product based on these cells, which express high levels of BDNF, an important neurotrophic factor for neuronal survival.
Preclinical tests showed that NestaCell® is distributed to several organs, including the central nervous system, being well tolerated in toxicological tests in rats.
In phase I (SAVE) and phase II (ADORE) clinical trials, NestaCell® was administered to patients with HD. The results indicated a significant improvement in motor scores and functional capacity compared to placebo, demonstrating a clinically significant benefit.
NestaCell® also presented a good safety and tolerability profile, with few adverse events related to the product. The results support the conclusion that NestaCell® is safe and well tolerated in HD patients, within the doses tested.
Read the detailed description
This is a Phase III multicenter, prospective, randomized, double-blind, placebo-controlled clinical trial to evaluate the efficacy and safety of Nestacell® in Huntington's Disease.
After signing the Informed Consent Form (ICF) the patients will perform the V-2 and V-1 screening procedures. In the V-2 the site collects personal data and a physician clinically confirms the HD diagnosis. The patient collects blood for CAG repeats and safety exams. Females with childbearing potential perform a urine pregnancy test. In the V-1 the investigator remotely reviews the results of the V-2 exams and authorizes the performance of the V-1 radiological exams (MRI and Total-Body PET Scan) in the radiology center.
In the V0, the PI and his team perform the baseline UHDRS and body weight assessments. The patient will be randomized to Nestacell® or placebo. Those weighing 50 to 67.9 kg will receive 100 million cells (or placebo) and those weighing ≥ 68 kg will receive 136 million cells(or placebo) per administration. The doses are based on the V0 weight assessment and will not change throughout the study unless the PI asks for safety reasons (for example, a relevant weight loss).
The V1 marks the first investigational product administration. It will happen in sites unrelated to those performing the outcomes evaluation by personnel specially trained to prepare and administer the investigational product. After the females with childbearing potential performe the urine pregnancy test, NestaCell® or placebo will be administered intravenously in three cycles of three-monthly administrations with a monthly interval between cycles (total of 9 administrations). The other administrations happen at visits V2, V3, V5, V6, V7, V9, V10, and V11. To assure blindness, UHDRS and other clinical evaluations will be carried out in the research center [by the principal investigator (PI)] while the administrations will be made in the Center for the Investigational Product Administration (CIPA). The outcome evaluations by the PI and his/her team happen at V4, V8, and V12, one month after the end of each cycle. In the V13, the patients will also be directed to the radiology center to repeat the MRI exam.
Each patient participates in the trial for approximately 14 months, two months for screening, and twelve months for investigational product administration and follow-up.
- Conditions
- Huntington Disease
- Being tested
- NestaCell®
- Keywords
- STAR, NestaCell ®
- Medical subject headings
- Huntington Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NestaCell®Genetic
Human Dental Pulp Stem Cells (hDPSCs).
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Arm 1: Nine intravenous placebo administrations in twelve months. Receives: Genetic: NestaCell® |
| Intravenous NestaCell® administrations | Experimental | Arm 2: Nine intravenous NestaCell® administrations in twelve months. Receives: Genetic: NestaCell® |
What is being measured
The primary outcome is the main question the study is designed to answer.
Primary Efficacy Objective
Proportion of patients who stabilized or decreased the UHDRS-TMS from Visit 0 to Visit 12 in the Nestacell® vs. Placebo groups.
Measured: 1 year
Secondary outcomes (9)
Secondary Efficacy Objectives
Mean variation of the UHDRS-TMS mean variation from Visit 0 to Visit 12 in the groups Nestacell® and Placebo.
Measured: 1 year
Secondary Efficacy Objectives
Proportion of patients who stabilized or increased the UHDRS-TFC from Visit 0 to Visit 12 in the groups Nestacell® and Placebo.
Measured: 1 year
Secondary Efficacy Objectives
Mean variation of the UHDRS-TFC from Visit 0 to Visit 12 of the groups Nestacell® and Placebo.
Measured: 1 year
Secondary Efficacy Objectives
Proportion of patients who stabilized or increased the cUHDRS from Visit 0 to Visit 12 in the groups Nestacell® and Placebo.
Measured: 1 year
Secondary Efficacy Objectives
Mean variation of the cUHDRS from Visit 0 to Visit 12 of the groups Nestacell® and Placebo.
Measured: 1 year
Secondary Efficacy Objectives
Proportion of patients who stabilized the MRI outcomes: 7.1 white substance, 7.2 gray substance, from Visit 0 to Visit 12 in the groups Nestacell® and Placebo.
Measured: 1 year
Secondary Efficacy Objectives
Mean variation of the MRI outcomes: 8.1 white substance, 8.2 gray substance, from Visit 0 to Visit 12 in the groups Nestacell® and Placebo.
Measured: 1 year
Secondary Efficacy Objectives
Proportion of patients who stabilized or decreased the NfL blood levels from Visit 0 to Visit 12 in the groups Nestacell® and Placebo.
Measured: 1 year
Secondary Efficacy Objectives
Mean variation of the NfL blood levels from Visit 0 to Visit 12 of the groups Nestacell® and Placebo.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Oct 2023
- Study start8 Jun 2024 (estimated)
- Primary completion8 Sep 2025 (estimated)
- Study completion9 Feb 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (24)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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- The trial will evaluate 120 adult patients (male and female) aged 18 to 55 years with Huntington's disease:CAG repeats from 40 to 50; HD diagnostic confidence level (DCL) score of 4;UHDRS Total Functional Capacity (TFC) from 7 to 12, suggesting mild-moderate functional impairment. Each patient participates in the trial for approximately 14 months, two months for screening, and twelve months for investigational product administration and follow-up.
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 120 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Azidus Brasil
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Cellavita Pesquisa Científica Ltda
- Responsible party
- Sponsor
- Investigators
- Luciana Ferrara, Doctor, Study director, Azidus Brasil
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06097780
- Sponsor's protocol ID
- STAR
- Other IDs
- U1111-1298-5627
- Acronym
- STAR
- Status last verified
- Oct 2023
- First submitted
- 5 Oct 2023
- Last updated
- 24 Oct 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: After the data analysis and presentation to the National Commission on Research Ethics, all data of the study will become public.
Publications
ABH - Associação Brasil Huntington. p. Perguntas Frequentes. Disponível em: https://abh.org.br/perguntas-frequentes/. Acesso em: 19/12/2022.
BARRETO, R. D. R. Características da Disfunção Congnitiva na Doença de Huntington. Orientador: JANUÁRIO, D. C. 2009. 87 f. (Mestre) - Faculdade de Medicina, Universidade de Coimbra.
Bathina S, Das UN. Brain-derived neurotrophic factor and its clinical implications. Arch Med Sci. 2015 Dec 10;11(6):1164-78. doi: 10.5114/aoms.2015.56342. Epub 2015 Dec 11.
PubMed 26788077Baydyuk M, Xu B. BDNF signaling and survival of striatal neurons. Front Cell Neurosci. 2014 Aug 28;8:254. doi: 10.3389/fncel.2014.00254. eCollection 2014.
PubMed 25221473Benedict RH, DeLuca J, Phillips G, LaRocca N, Hudson LD, Rudick R; Multiple Sclerosis Outcome Assessments Consortium. Validity of the Symbol Digit Modalities Test as a cognition performance outcome measure for multiple sclerosis. Mult Scler. 2017 Apr;23(5):721-733. doi: 10.1177/1352458517690821. Epub 2017 Feb 16.
PubMed 28206827BENTON, A. L.; HAMSHER, K. S.; SIVAN, A. B.; PSYCHOLOGICAL ASSESSMENT RESOURCES, I. Multilingual Aphasia Examination, Third Edition: Manual of instructions. PAR, 1978.
Biglan KM, Zhang Y, Long JD, Geschwind M, Kang GA, Killoran A, Lu W, McCusker E, Mills JA, Raymond LA, Testa C, Wojcieszek J, Paulsen JS; PREDICT-HD Investigators of the Huntington Study Group. Refining the diagnosis of Huntington disease: the PREDICT-HD study. Front Aging Neurosci. 2013 Apr 2;5:12. doi: 10.3389/fnagi.2013.00012. eCollection 2013.
PubMed 23565093Chen J, Marks E, Lai B, Zhang Z, Duce JA, Lam LQ, Volitakis I, Bush AI, Hersch S, Fox JH. Iron accumulates in Huntington's disease neurons: protection by deferoxamine. PLoS One. 2013 Oct 11;8(10):e77023. doi: 10.1371/journal.pone.0077023. eCollection 2013.
PubMed 24146952Dorsey ER, Beck CA, Darwin K, Nichols P, Brocht AF, Biglan KM, Shoulson I; Huntington Study Group COHORT Investigators. Natural history of Huntington disease. JAMA Neurol. 2013 Dec;70(12):1520-30. doi: 10.1001/jamaneurol.2013.4408.
PubMed 24126537FDA. Guidance for Industry: Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products. pp. 27.
Frank S, Testa C, Edmondson MC, Goldstein J, Kayson E, Leavitt BR, Oakes D, O'Neill C, Vaughan C, Whaley J, Gross N, Gordon MF, Savola JM; Huntington Study Group/ARC-HD Investigators and Coordinators. The Safety of Deutetrabenazine for Chorea in Huntington Disease: An Open-Label Extension Study. CNS Drugs. 2022 Nov;36(11):1207-1216. doi: 10.1007/s40263-022-00956-8. Epub 2022 Oct 15.
PubMed 36242718GAMARRA, L.; CINTRA, L.; GÁRATE, A. P. Relatório Final do Estudo de Biodistribuição do Produto NestaCell®. Centro de Experimentação e Treinamento em Cirurgia (CETEC) do Instituto Israelita de Ensino e Pesquisa Hospital Albert Einstein, p. 72. 2022.
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 8684382JANUÁRIO, C. Doença de Huntington. Onde estamos agora? 2011. 148 f. (Doutor) -, Universidade de Coimbra.
Muller M, Leavitt BR. Iron dysregulation in Huntington's disease. J Neurochem. 2014 Aug;130(3):328-50. doi: 10.1111/jnc.12739. Epub 2014 May 28.
PubMed 24717009
Study at a glance
From the registry record, last updated by the sponsor on 24 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase III Efficacy and Safety of NestaCell® in Moderated Huntington's Disease
- ClinicalTrials.gov ID
- NCT06097780
- Acronym / sponsor ID
- STAR / STAR
- Conditions
- Huntington Disease
- Keywords
- STAR, NestaCell ®
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 120 participants (estimated)
- Lead sponsor
- Azidus BrasilIndustry (pharma, biotech, devices)
- Collaborators
- Cellavita Pesquisa Científica Ltda
- Study start
- 8 Jun 2024 (estimated)
- Primary completion
- 8 Sep 2025 (estimated)
- Study completion
- 9 Feb 2026 (estimated)
- First posted
- 24 Oct 2023 (actual)
- 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.
Huntington's disease (HD) is a rare neurodegenerative condition caused by increased CAG trinucleotide repeats in the HTT gene, on chromosome 4. The estimated global prevalence is 2.71 cases per 100,000 inhabitants. In Brazil, it is estimated that 13,000 to 19,000 people carry the gene and 65,000 to 95,000 are descendants at risk.
HD usually manifests itself in the fourth decade of life with motor, cognitive and behavioral symptoms, such as chorea. This condition profoundly affects quality of life and there is no treatment that can modify its course. Tetrabenazine is the only medication approved to control chorea.
A partnership between the Butantan Institute and Cellavita investigates the use of Human Dental Pulp Stem Cells (hDPSCs) to treat HD. NestaCell® was developed, a product based on these cells, which express high levels of BDNF, an important neurotrophic factor for neuronal survival.
Preclinical tests showed that NestaCell® is distributed to several organs, including the central nervous system, being well tolerated in toxicological tests in rats.
In phase I (SAVE) and phase II (ADORE) clinical trials, NestaCell® was administered to patients with HD. The results indicated a significant improvement in motor scores and functional capacity compared to placebo, demonstrating a clinically significant benefit.
NestaCell® also presented a good safety and tolerability profile, with few adverse events related to the product. The results support the conclusion that NestaCell® is safe and well tolerated in HD patients, within the doses tested.
Detailed description
This is a Phase III multicenter, prospective, randomized, double-blind, placebo-controlled clinical trial to evaluate the efficacy and safety of Nestacell® in Huntington's Disease.
After signing the Informed Consent Form (ICF) the patients will perform the V-2 and V-1 screening procedures. In the V-2 the site collects personal data and a physician clinically confirms the HD diagnosis. The patient collects blood for CAG repeats and safety exams. Females with childbearing potential perform a urine pregnancy test. In the V-1 the investigator remotely reviews the results of the V-2 exams and authorizes the performance of the V-1 radiological exams (MRI and Total-Body PET Scan) in the radiology center.
In the V0, the PI and his team perform the baseline UHDRS and body weight assessments. The patient will be randomized to Nestacell® or placebo. Those weighing 50 to 67.9 kg will receive 100 million cells (or placebo) and those weighing ≥ 68 kg will receive 136 million cells(or placebo) per administration. The doses are based on the V0 weight assessment and will not change throughout the study unless the PI asks for safety reasons (for example, a relevant weight loss).
The V1 marks the first investigational product administration. It will happen in sites unrelated to those performing the outcomes evaluation by personnel specially trained to prepare and administer the investigational product. After the females with childbearing potential performe the urine pregnancy test, NestaCell® or placebo will be administered intravenously in three cycles of three-monthly administrations with a monthly interval between cycles (total of 9 administrations). The other administrations happen at visits V2, V3, V5, V6, V7, V9, V10, and V11. To assure blindness, UHDRS and other clinical evaluations will be carried out in the research center [by the principal investigator (PI)] while the administrations will be made in the Center for the Investigational Product Administration (CIPA). The outcome evaluations by the PI and his/her team happen at V4, V8, and V12, one month after the end of each cycle. In the V13, the patients will also be directed to the radiology center to repeat the MRI exam.
Each patient participates in the trial for approximately 14 months, two months for screening, and twelve months for investigational product administration and follow-up.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
- Model description
- The trial will evaluate 120 adult patients (male and female) aged 18 to 55 years with Huntington's disease:CAG repeats from 40 to 50; HD diagnostic confidence level (DCL) score of 4;UHDRS Total Functional Capacity (TFC) from 7 to 12, suggesting mild-moderate functional impairment. Each patient participates in the trial for approximately 14 months, two months for screening, and twelve months for investigational product administration and follow-up.
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Genetic: NestaCell®Arm 1: Nine intravenous placebo administrations in twelve months. |
| Intravenous NestaCell® administrations | Experimental | Genetic: NestaCell®Arm 2: Nine intravenous NestaCell® administrations in twelve months. |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (8 listed)
- Male or female;
- Age from 18 to 55 years;
- HD diagnostic confidence level (DCL) score of 3 or 4 at enrolment;
- HD manifestations begin from 4 to 8 years before enrolment;
- UHDRS Total Functional Capacity (TFC) from 7 to 12, suggesting mild-moderate functional impairment;
Key exclusion criteria (23 listed)
- Juvenile Huntington's disease,
- Concomitant epilepsy;
- Decompensated psychiatric disorders;
- History of a suicide attempt;
- Other neurological or musculoskeletal disorders that might interfere with the assessments;
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 |
|---|---|---|
| NestaCell®Human Dental Pulp Stem Cells (hDPSCs). | Genetic | Intravenous NestaCell® administrations, Placebo |
Primary outcome
Primary Efficacy Objective
Time frame: 1 year
Proportion of patients who stabilized or decreased the UHDRS-TMS from Visit 0 to Visit 12 in the Nestacell® vs. Placebo groups.
9 secondary outcomes
Secondary Efficacy Objectives
Time frame: 1 year
Secondary Efficacy Objectives
Time frame: 1 year
Secondary Efficacy Objectives
Time frame: 1 year
Secondary Efficacy Objectives
Time frame: 1 year
Secondary Efficacy Objectives
Time frame: 1 year
Secondary Efficacy Objectives
Time frame: 1 year
Secondary Efficacy Objectives
Time frame: 1 year
Secondary Efficacy Objectives
Time frame: 1 year
Secondary Efficacy Objectives
Time frame: 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Oct 2023.
- BackgroundABH - Associação Brasil Huntington. p. Perguntas Frequentes. Disponível em: https://abh.org.br/perguntas-frequentes/. Acesso em: 19/12/2022.
- BackgroundBARRETO, R. D. R. Características da Disfunção Congnitiva na Doença de Huntington. Orientador: JANUÁRIO, D. C. 2009. 87 f. (Mestre) - Faculdade de Medicina, Universidade de Coimbra.
- Background: PubMed 26788077Bathina S, Das UN. Brain-derived neurotrophic factor and its clinical implications. Arch Med Sci. 2015 Dec 10;11(6):1164-78. doi: 10.5114/aoms.2015.56342. Epub 2015 Dec 11.
- Background: PubMed 25221473Baydyuk M, Xu B. BDNF signaling and survival of striatal neurons. Front Cell Neurosci. 2014 Aug 28;8:254. doi: 10.3389/fncel.2014.00254. eCollection 2014.
- Background: PubMed 28206827Benedict RH, DeLuca J, Phillips G, LaRocca N, Hudson LD, Rudick R; Multiple Sclerosis Outcome Assessments Consortium. Validity of the Symbol Digit Modalities Test as a cognition performance outcome measure for multiple sclerosis. Mult Scler. 2017 Apr;23(5):721-733. doi: 10.1177/1352458517690821. Epub 2017 Feb 16.
- BackgroundBENTON, A. L.; HAMSHER, K. S.; SIVAN, A. B.; PSYCHOLOGICAL ASSESSMENT RESOURCES, I. Multilingual Aphasia Examination, Third Edition: Manual of instructions. PAR, 1978.
- Background: PubMed 23565093Biglan KM, Zhang Y, Long JD, Geschwind M, Kang GA, Killoran A, Lu W, McCusker E, Mills JA, Raymond LA, Testa C, Wojcieszek J, Paulsen JS; PREDICT-HD Investigators of the Huntington Study Group. Refining the diagnosis of Huntington disease: the PREDICT-HD study. Front Aging Neurosci. 2013 Apr 2;5:12. doi: 10.3389/fnagi.2013.00012. eCollection 2013.
- Background: PubMed 24146952Chen J, Marks E, Lai B, Zhang Z, Duce JA, Lam LQ, Volitakis I, Bush AI, Hersch S, Fox JH. Iron accumulates in Huntington's disease neurons: protection by deferoxamine. PLoS One. 2013 Oct 11;8(10):e77023. doi: 10.1371/journal.pone.0077023. eCollection 2013.
- Background: PubMed 24126537Dorsey ER, Beck CA, Darwin K, Nichols P, Brocht AF, Biglan KM, Shoulson I; Huntington Study Group COHORT Investigators. Natural history of Huntington disease. JAMA Neurol. 2013 Dec;70(12):1520-30. doi: 10.1001/jamaneurol.2013.4408.
- BackgroundFDA. Guidance for Industry: Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products. pp. 27.
- Background: PubMed 36242718Frank S, Testa C, Edmondson MC, Goldstein J, Kayson E, Leavitt BR, Oakes D, O'Neill C, Vaughan C, Whaley J, Gross N, Gordon MF, Savola JM; Huntington Study Group/ARC-HD Investigators and Coordinators. The Safety of Deutetrabenazine for Chorea in Huntington Disease: An Open-Label Extension Study. CNS Drugs. 2022 Nov;36(11):1207-1216. doi: 10.1007/s40263-022-00956-8. Epub 2022 Oct 15.
- BackgroundGAMARRA, L.; CINTRA, L.; GÁRATE, A. P. Relatório Final do Estudo de Biodistribuição do Produto NestaCell®. Centro de Experimentação e Treinamento em Cirurgia (CETEC) do Instituto Israelita de Ensino e Pesquisa Hospital Albert Einstein, p. 72. 2022.
- Individual participant dataSharing planned: No. 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.
Luciana Ferrara, Doctor study director
Azidus Brasil
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing NestaCell® in people with Huntington Disease, run by Azidus Brasil.
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 8 inclusion and 23 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 24 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. 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 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?
Contact Luciana Ferrara, Doctor (+ 55 19 981428814, luciana.ferrara@azidusbrasil.com.br), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Primary Efficacy Objective (time frame: 1 year). 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 NCT06097780, which also lists 24 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 24 Oct 2023.