NCT03252535

Dose-response Evaluation of the Cellavita HD Product in Patients With Huntington's Disease

Dose-Response Evaluation of the Investigational Product Cellavita HD After Intravenous Administration in Patients With Huntington's Disease

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 49 (actual)
• Sponsor: Azidus Brasil · Industry
• Sex: All
• Age: 21 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

Cellavita HD is a stem-cell therapy for Huntington's Disease. This is a prospective, phase II, single-center, randomized (2:2:1), triple-blind, placebo controlled study, with two test doses of Cellavita HD product.

Detailed description

This is a phase II dose-response study in which participants with HD will receive three intravenous injections of the investigational product or placebo (one every month for three months) a total of three cycles. The subjects will be randomized in 2: 2: 1 ratio for the groups G1: lower dose (1x10\^6 cells/weight range), G2: higher dose (2x10\^6 cells/weight range) or G3: placebo. To identify the dose of the product that will provide the best clinical response, motor assessment will be performed with UHDRS scale and improvement will be evaluated by correlating before and after treatment scores. Additionally, also will be performed the combined score through the cUHDRS. Secondary evidences of efficacy will be evaluated through the data of functional state, total functional capacity, functional independence, psychiatric symptoms and cognition from UHDRS scale. Additionally, related data to clinical worsening, change of Body Mass Index (BMI), risk of suicide attempt and neurological image improvement will be evaluated. Safety evaluation will included the incidence and classification of the adverse events experienced by the subjects during the study.

Conditions

• Huntington Disease

Interventions

Type	Name	Description
BIOLOGICAL	Cellavita HD lower dose	The participants will receive a total of 9 intravenous administrations of 1x10\^6 cells/weight range divided into three administrations per cycle. Each administration will occur every 30 days and cycles every 120 days (total of 3 cycles).
BIOLOGICAL	Cellavita HD higher dose	The participants will receive a total of 9 intravenous administrations of 2x10\^6 cells/weight range divided into three administrations per cycle. Each administration will occur every 30 days and cycles every 120 days (total of 3 cycles).
OTHER	Placebo	The participants will receive a total of 9 intravenous administrations of placebo divided into three administrations per cycle. Each administration will occur every 30 days and cycles every 120 days (total of 3 cycles).

Timeline

• Start date: 2018-01-15
• Primary completion: 2021-03-23
• Completion: 2021-04-30
• First posted: 2017-08-17
• Last updated: 2025-09-19
• Results posted: 2025-09-19

Locations

1 site across 1 country: Brazil