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

NCT04219241 | Unknown Phase 2

• 2 other identifiers: ADORE-EXT23917319.0.0000.5412
• Study Type: interventional
• Target: 35
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Huntington Disease

Keywords

Huntington Disease; Stem cell therapy; Dental Pulp Stem Cell

Outcome Measures

Primary Outcomes (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.

  Two years (every study visit)

Secondary Outcomes (5)

• Clinical neurological worsening over the treatment by UHDRS

  Two years

• BMI assessment

  Two years

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

  Two years

• Risk of suicidal ideation and/or behavior by HDS

  Two years

• CNS assessment by MRI (Magnetic Ressonance Imaging)

  One year

Other Outcomes (1)

• Safety of Cellavita-HD administration

  Two years

Study Arms (1)

Cellavita-HD

EXPERIMENTAL

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).

Biological: Cellavita-HD

Interventions

Cellavita-HD BIOLOGICAL

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).

Also known as: cellular therapy, mesenchymal stem cells

Cellavita-HD

Eligibility Criteria

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

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Azidus Brasil: lead
• Cellavita Pesquisa Científica Ltda: collaborator

Study Sites (1)

Azidus Brasil Pesquisa Científica e Desenvolvimento Ltda.

Valinhos, São Paulo, 13271-130, Brazil

Location

Related Publications (9)

• 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.

  PMID: 25426265 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

• 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

• 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

• 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.

  PMID: 8252043 BACKGROUND

• 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.

  PMID: 8684382 BACKGROUND

• 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.

  PMID: 18474605 BACKGROUND

• 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.

  PMID: 26667114 BACKGROUND

• 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.

  PMID: 10668713 BACKGROUND

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, MD

  Azidus Brasil Scientific Research and Development Ltda

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 11, 2019
• First Posted: January 7, 2020
• Study Start: February 10, 2020
• Primary Completion: December 1, 2022
• Study Completion: December 1, 2022
• Last Updated: October 28, 2022

Record last verified: 2022-10

Locations

BR (1)