Safety Evaluation of Cellavita HD Administered Intravenously in Participants With Huntington's Disease

NCT02728115 | Unknown Phase 1 DMC

• 2 other identifiers: SAVE-DH51005115.9.0000.5412
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief 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 effectiveness.

Conditions

Huntington Disease

Keywords

Huntington's Disease; Stem Cell; Dental Pulp Stem Cells

Outcome Measures

Primary Outcomes (1)

• 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.

  first year and in the following 4 years

Secondary Outcomes (5)

• Preliminary efficacy of Cellavita HD by UHDRS improvement and global clinical response (CIBIS)

  first year and in the following 4 years

• Preliminary efficacy of Cellavita HD by comparison of the inflammatory markers

  first year

• Immunological Response of Cellavita HD

  first year

• Preliminary efficacy of Cellavita HD by comparison of the CNS assessment

  first year

• Risk of suicidal ideation by Hamilton Depression Rating Scale (HDRS)

  first year and in the following 4 years

Study Arms (2)

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) .

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).

Biological: Cellavita HD Higher dose

Interventions

Cellavita HD Lower Dose BIOLOGICAL

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.

Also known as: cellular therapy, mesenchymal stem cells

Cellavita HD Lower Dose

Cellavita HD Higher dose BIOLOGICAL

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.

Also known as: cellular therapy, mesenchymal stem cells

Cellavita HD Higher dose

Eligibility Criteria

• Age: 21 Years - 65 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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;
• 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);

Sponsors & Collaborators

• Azidus Brasil: lead
• Cellavita Pesquisa Científica Ltda: collaborator
• Azidus Brasil Scientific Research and Development Ltda: collaborator

Study Sites (1)

Azidus Brasil Pesquisa Científica e Desenvolvimento Ltda.

Valinhos, São Paulo, 13271-130, Brazil

Location

Related Publications (8)

• Adam OR, Jankovic J. Symptomatic treatment of Huntington disease. Neurotherapeutics. 2008 Apr;5(2):181-97. doi: 10.1016/j.nurt.2008.01.008.

  PMID: 18394562 BACKGROUND

• 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.

  PMID: 25097727 BACKGROUND

• de 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.

  PMID: 21609310 BACKGROUND

• 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.

  PMID: 16545746 BACKGROUND

• Bachoud-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.

  PMID: 11130527 BACKGROUND

• Ball 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.

  PMID: 23992039 BACKGROUND

• 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.

  PMID: 23345280 BACKGROUND

• Fernandes 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.

  PMID: 41188963 DERIVED

MeSH Terms

Conditions

Huntington Disease

Interventions

Cell- and Tissue-Based Therapy

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Dementia; Chorea; Dyskinesias; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Cognition Disorders; Neurocognitive Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Biological Therapy; Therapeutics

Study Officials

• Joyce Macedo da Silva, MD

  Azidus Brasil Scientific Research and Development Ltda

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 11, 2016
• First Posted: April 5, 2016
• Study Start: October 16, 2017
• Primary Completion: December 31, 2022
• Study Completion: December 31, 2023
• Last Updated: November 2, 2022

Record last verified: 2022-11

Locations

BR (1)