NCT06097780: STAR

Efficacy and Safety of NestaCell® in Huntington's Disease

Status unknown Phase 3 Last updated 24 October 2023

What this trial tests

Phase 3 trial testing NestaCell® in Huntington Disease in 120 participants. Status unknown.

Timeline

8 June 2024

Primary endpoint
8 September 2025

9 February 2026

Quick facts

Lead sponsor	Azidus Brasil
Phase	Phase 3
Status	Status unknown
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	single
Primary purpose	treatment
Enrollment	120
Start date	8 June 2024
Primary completion	8 September 2025
Estimated completion	9 February 2026

Drugs / interventions tested

• NestaCell® — full drug profile →

Conditions studied

• Huntington Disease — all drugs for Huntington Disease →

Sponsor

Azidus Brasil — full company profile →

Who can join

Adults 18 to 55, any sex, with Huntington Disease. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Polyglutamine (PolyQ) Diseases: Navigating the Landscape of Neurodegeneration.
   Tenchov R, Sasso JM, Zhou QA. · · 2024 · cited 31× · PMID 38996083 · DOI 10.1021/acschemneuro.4c00184
2. Huntington's Disease Clinical Trials Corner: March 2024.
   Estevez-Fraga C, Tabrizi SJ, Wild EJ. · · 2024 · cited 31× · PMID 38489195 · DOI 10.3233/jhd-240017
3. Towards Stem Cell Therapy for Critical-Sized Segmental Bone Defects: Current Trends and Challenges on the Path to Clinical Translation.
   Quek J, Vizetto-Duarte C, Teoh SH, Choo Y. · · 2024 · cited 13× · PMID 38921519 · DOI 10.3390/jfb15060145
4. Advances in gene and cellular therapeutic approaches for Huntington's disease.
   Piao X, Li D, Liu H, Guo Q, et al · · 2025 · cited 9× · PMID 39121016 · DOI 10.1093/procel/pwae042
5. Advances in Stem Cell Therapy for Huntington's Disease: A Comprehensive Literature Review.
   Shah S, Mansour HM, Lucke-Wold B. · · 2025 · cited 8× · PMID 39791743 · DOI 10.3390/cells14010042
6. Huntington's disease clinical trials update: March 2025.
   Farag M, Tabrizi SJ, Wild EJ. · · 2025 · cited 7× · PMID 40302443 · DOI 10.1177/18796397251337000
7. Research progress on optimization of <i>in vitro</i> isolation, cultivation and preservation methods of dental pulp stem cells for clinical application.
   Wang X, Li F, Wu S, Xing W, et al · · 2024 · cited 6× · PMID 38633667 · DOI 10.3389/fbioe.2024.1305614
8. Age-Related Neurodegenerative Diseases: A Stem Cell's Perspective.
   Calvo B, Schembri-Wismayer P, Durán-Alonso MB. · · 2025 · cited 4× · PMID 40072076 · DOI 10.3390/cells14050347